ticker,disease,stage,date,catalyst,label
AZN,Neurofibromatosis type 1 plexiform neurofibromas,Approved,4/13/20,"FDA Approval announced April 13, 2020.",6
GOSS,Eosinophilic asthma,Phase 2b,10/13/20,"Phase 2b trial did not meet primary endpoint - October 13, 2020.",4
AZN,"Risk of cardiovascular (CV) death, hospitalization for heart failure (hHF) and urgent heart failure (HF)",Approved,5/9/23,"Approved May 9, 2023.",6
ABBV,Upper Limb Spasticity,Approved,7/29/21,"FDA approval announced July 29, 2021.",6
INFI,Triple negative breast cancer (TNBC) and ovarian cancer,Phase 1,12/9/20,"Phase 1 initial data due presented at SABCS Annual Meeting, December 9, 2020. 2/18 response rate (partial responses - PR) for doublet regimen. 5/12 responses for triplet (1 complete response, 4 had a PR).",5
MRK,Renal cell carcinoma,Approved,4/22/19,"FDA approval announced April 22, 2019.",6
REGN,Stage II to IV Cutaneous Squamous Cell Carcinoma (CSCC),Phase 2,10/16/23,"Phase 2 data from ESMO reported a median duration of follow-up was 18.7 months, and 1 of 10 pts with MPR experienced recurrence, noted October 16, 2023.",5
IONS,C9orf72-associated Amyotrophic Lateral Sclerosis (ALS),Phase 1,3/28/22,"Phase 1 study did not meet any secondary efficacy endpoints and it did not demonstrate clinical benefit. These results do not support further development, noted March 28, 2022.",6
CYT,"Hematologic malignancies, solid tumors, triple negative breast cancer",Phase 1,6/30/23,"Phase 1 development discontinued due to dissolution, noted June 30, 2023.",4
SPPI,Non-Hodgkin’s lymphoma,Approved,9/4/09,"Approved September 4, 2009.",6
PFE,Atopic dermatitis (AD),Phase 2b,11/9/20,"Phase 2b trial did not meet primary endpoint - November 9, 2020. Phase 3 trial planned.",5
EGRX,Supraventricular tachycardia (SVT) - atrial fibrillation and atrial flutter,CRL,5/31/23,"CRL received on May 31, 2023.",5
EWTX,Duchenne Muscular Dystrophy (DMD),Phase 2,10/26/23,"Phase 2 cohort in boys initiated, October 26, 2023.",5
GNFT,Non-alcoholic steatohepatitis (NASH),Phase 3,5/11/20,"Phase 3 data did not meet primary endpoint - May 11, 2020.",4
MEIP,"Indolent B-cell Non-Hodgkin's Lymphoma (iB-NHL) without small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL), and Waldenström's macroglobulinemia (WM)",Phase 2,11/17/22,"Phase 2 data reported a 75.4% objective response rate, and 24.6% of patients achieved a complete response, noted November 17, 2022.",6
CVAC,Solid tumors,Phase 1,11/11/22,"Phase 1 data reported that five out of 30 patients (17%) experienced a partial response and no objective responses were observed in the 10 patients of the single-agent cohort, noted November 11, 2022.",6
BHC,Open angle glaucoma (OAG) or ocular hypertension (OHT),Approved,11/2/17,"Approval announced November 2, 2017.",6
ATNXQ,Metastatic breast cancer,Phase 3,11/22/21,"Phase 3 post hoc analysis showed a median survival rate of 18.9 months in patients treated with Oral Paclitaxel vs 10.1 months in those treated with IV Paclitaxel, with a hazard ratio of 0.59, noted November 22, 2021.",6
ALGS,Nonalcoholic steatohepatitis (NASH),Phase 1,3/29/23,"Phase 1 data presented at the Hepatology Conference demonstrated a favorable safety and pharmacokinetic profile in healthy volunteers and subjects with hyperlipidemia, March 29, 2023.",6
ADVM,Wet age-related macular degeneration (Wet-AMD),Phase 2,11/4/22,"Phase 2 end-of-study results reported that all 2E11 participants were inflammation free at the end of the study, noted November 4, 2022.",5
TAK,Dravet Syndrome / Lennox-Gastaut Syndrome,Phase 3,4/27/23,"Phase 3 data reported that 78% of patients tolerated the highest dose level well with sustained reduction in frequency of drop seizures, noted April 27, 2023.",6
SRPT,Duchenne muscular dystrophy,Approved,2/25/21,"FDA approval announced February 25, 2021.",6
ANAB,Palmo-plantar pustular psoriasis,Phase 2,3/8/21,"Phase 2 trial did not meet primary endpoint - March 8, 2021.",4
SLGL,Papulopustular rosacea,Phase 3,7/8/19,"Phase 3 top-line data met primary endpoints - July 8, 2019.",6
AGIO,Thalassemia,Phase 2,12/12/22,"Phase 2 data reported durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients, noted December 12, 2022.",6
AMGN,KRAS mutant non-small cell lung cancer (NSCLC),Phase 1/2,10/16/23,"Phase 1/2 initial data reported that the confirmed objective response rate (ORR) was 25% (3/12) across efficacy-evaluable patients and seen in both KRAS G12C inhibitor resistant (14.3%; 1/7) and naïve (40%; 2/5) patients, noted October 16, 2023.",6
PFE,Multiple Myeloma,Approved,8/14/23,"FDA Approved on August 14, 2023.",6
NVS,Non-small cell lung cancer (NSCLC) with BRAF V600E mutation,Approved,6/22/17,"FDA Approval announced June 22, 2017.",6
PFE,Psoriatic arthritis,Approved,12/14/17,"Approval announced December 14, 2017.",6
AZN,Acute ischaemic stroke,Approved,11/6/20,"FDA approval announced November 6, 2020.",6
FHTX,Metastatic Uveal Melanoma (mUM),Phase 1,10/22/23,"Phase 1 clinical data presented at ESMO reported that 1 patient treated at the 7.5 mg QD continuous dose level experienced a DLT of Grade 3 keratitis, noted October 22, 2023.",6
LLY,Chronic kidney disease (CKD),Approved,9/22/23,"FDA Approval on September 22, 2023.",6
ERAS,Advanced solid tumors,Phase 1b,4/18/23,"Phase 1b data presented at AACR showed that dosing was well-tolerated with favorable pharmacokinetics and no apparent drug-drug interactions, noted April 18, 2023.",6
TAK,Hereditary angioedema (HAE) attacks in patients 2 to <6 yrs,Approved,2/3/23,"FDA approved on February 3, 2023.",6
AZN,Paroxysmal Nocturnal Hemoglobinuria (PNH) - children,Approved,6/7/21,"FDA approval announced June 7, 2021.",6
ABBV,Mid-Face Injection Via Cannula,Approved,9/3/19,"FDA Approval announced September 3, 2019.",6
AQST,Epileptic seizures,Approved,8/31/22,"Tentative approval from FDA on August 31, 2022. The tentative approval was due to to an existing FDA regulatory grant of orphan drug market exclusivity for Valtoco, a diazepam nasal spray product, Libervant is not yet eligible for marketing in the United States.",6
BHC,Acne,Approved,8/24/18,"FDA Approval announced August 24, 2018.",6
AZN,COVID-19 vaccine,Phase 3,3/22/21,"Data from U.S. trial noted 76% efficacy rate - March 24, 2021.",6
FOLD,Epidermolysis Bullosa (EB),Phase 3,9/13/17,"Phase 3 top-line data released September 13, 2017 - endpoints not met.",4
BGNE,Non-squamous non-small cell lung cancer (NSCLC),Phase 3,9/17/20,Phase 3 primary endpoint met - PFS 9.7 months in Arm A compared to 7.6 months in the chemotherapy alone Arm B (p=0.0044; stratified hazard ratio [HR]=0.64.,6
BTAI,Solid Tumors,Phase 2,10/10/23,"Phase 2 trial showed a median OS of 13.6 months and a 12-month survival rate of 56.5%., noted October 10, 2023.",6
SNY,Cold agglutinin disease,CRL,11/13/20,"CRL issued November 13, 2020.",5
ALLO,Large B-cell lymphoma,Phase 1,6/15/23,"Additional data reported at ICML across all 33 patients the CR rate was 42% with 30% maintaining a CR at Month 6. Results indicated complete responses are more common with lymphodepletion regimens containing 90 mg of ALLO-647 (FCA90). Median duration of response for both the overall population and the patients treated with the Phase 2 regimen (n=12) was 23.1 months, noted June 15, 2023.",6
BCYC,Solid tumors,Phase 1/2,2/14/23,"Phase 1/2 data reported a 50% ORR and 75% benefit rate, including one complete response, noted February 14, 2023.",5
REGN,Coronavirus COVID-19,Phase 3,7/2/20,"Phase 3 trial did not meet primary endpoint - July 2, 2020.",4
RMTI,Anemia,Approved,3/27/20,"FDA approval announced date March 27, 2020.",6
GLPG,Relapsed/refractory Non-Hodgkin Lymphoma (rrNHL),Phase 1/2,12/13/22,"Phase 1/2 data reported an ORR of 86% and all responding patients achieved complete response (CR), noted December 13, 2022.",6
JNJ,Reduce the Risk of Recurrent Venous Thromboembolism (VTE),Approved,10/14/19,"FDA approval announced October 14, 2019.",6
MGTX,X-Linked Retinitis Pigmentosa,Phase 1/2,10/3/20,"Phase 1/2 nine-month data presented at EURETINA 2020 Virtual Meeting October 3, 2020.",5
PASG,GM1 gangliosidosis (GM1),Phase 1/2,8/7/23,"Phase 1/2 initial safety data from cohorts 1-4 up to 28 months showed Dose 1 and 2 of PBGM01 were well tolerated and had a favorable safety and immunological profile, noted August 7, 2023.",6
PFE,Metastatic colorectal cancer,Approved,9/28/21,"Approved September 28, 2021.",6
BHC,Psoriasis,Approved,10/8/18,"Tentative FDA Approval announced October 8, 2018. Final approval expected November 2018 pending the expiration of exclusivity for a related product.",6
PTCT,SMA type 1/2/3 switching,Phase 2,6/11/21,"Phase 2 data reported a sustained >2-fold increase in median SMN protein levels versus baseline in all patients who received prior treatment, noted June 11, 2021.",6
TGTX,Relapsing forms of Multiple Sclerosis (RMS),Phase 2a,6/18/18,"Phase 2a updated data released at EAN June 18, 2018. Annualized Relapse Rate (ARR) of 0.07.",5
BMY,Ulcerative Colitis,Approved,5/27/21,"FDA approval announced May 27, 2021.",6
CLVS,Relapsed ovarian cancer patients with BRCA mutations,Phase 3,12/21/20,"Phase 3 trial met primary endpoint - December 21, 2020.",6
VINC,Non-Hodgkin Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL),Phase 1,12/12/22,"Phase 1 data reported that efficacy signals from the 5 newly enrolled pts include 1 stable disease (SD), 2 radiologic disease progression, and 2 on study and who have not yet had first tumor assessment, noted December 12, 2022.",5
FBIO,Non-small cell lung cancer (NSCLC),Phase 1/2,9/25/18,"Phase 1/2 preliminary data released September 24, 2018.",5
NLSP,Narcolepsy,Phase 3,3/27/23,"OLE trial data reported that the mean ESS score for these patients declined by approximately 10.2 (SD=5.83) points from their DB study baseline to month 6 in the OLE, noted March 27, 2023.",5
PLUR,"Intermittent claudication, or IC",Phase 2,6/12/18,"Phase 2 data released June 12, 2018 - primary endpoint met.",6
ADIL,Alcohol use disorder,Phase 3,7/11/23,"FDA reviewed the safety data from the ONWARD trial and did not express any concerns with the data, noted July 11, 2023.",5
CINC,Treatment Resistant Hypertension (rHTN),Phase 2,11/7/22,"Phase 2 presentation at AHA reported that iomarker activity clinically demonstrate how baxdrostat mechanistically achieves selective blood pressure lowering effects with no impact on cortisol, noted November 7, 2022.",6
ZNTL,Uterine serous carcinoma (USC),Phase 1,6/6/23,"Phase 1 showed an ORR of 36.8% in heavily pretreated platinum-resistant ovarian cancer and USC patients treated with intermittent dosing, noted June 6, 2022.",6
BIIB,Alzheimer’s disease,Phase 3,9/13/19,Phase 3 trial discontinued due to unfavorable risk-benefit ratio.,4
ITRM,Complicated urinary tract infections (cUTI),Phase 3,6/1/20,"Phase 3 data did not meet primary endpoint - June 1, 2020.",4
JNJ,Newly Diagnosed Multiple Myeloma (NDMM),Approved,6/28/19,"FDA Approval announced June 28, 2019.",6
LLY,Type 2 Diabetes,Approved,5/13/22,"Approved May 13, 2022.",6
SAGE,Bipolar depression,Phase 2,7/24/19,"Phase 2 Part A data July 24, 2019 noted MADRS score of 15.5 points at Day 15 (n=23; p<0.0001) - secondary endpoint.",6
AZN,Asthma and comorbid nasal polyps,Phase 3,9/5/21,"Phase 3 data showed an 86% reduction in the annualized asthma exacerbation rate (AAER) September 5, 2021.",6
REGN,IgA nephropathy,Phase 2,8/29/22,"Phase 2 new results demonstrated an equivalent 36% reduction in 24-hour urine total protein and a higher proportion, 32 vs 13%, of patients treated in comparison to placebo, respectively, achieving greater than or equal to 50% reduction in 24-hour UPCR, noted August 29, 2022.",6
MRK,Solid tumors,Phase 1/2,8/9/23,"Phase 1/2 sixth dose escalation cohort data and agent expansion data to showed durable anti-cancer activity observed during monotherapy dose escalation without dose-limiting toxicities. Late-stage pancreatic cancer patient achieved 80% tumor shrinkage with complete regression observed in two of three metastatic lesions in the liver in response to single-agent MDNA11, noted August 9, 2023.",6
ONCY,"Colorectal and advanced anal cancers, pancreatic cancer",Phase 1/2,11/10/22,"Phase 1/2 data reported a 69% objective response rate (ORR), noted November 11, 2022.",5
GRTX,Pancreatic cancer,Phase 1/2,9/8/21,"Phase 1/2 trial final results announced - GC4419 was well tolerated, with similar rates of early and late adverse events in the active and placebo arms September 8, 2021.",6
ASND,Growth Hormone Deficiency - adults,Phase 3,9/23/23,"Phase 3 resultspresented at ESPE 2023 showed that the majority of children and adolescents treated once weekly with TransCon hGH (lonapegsomatropin) met or exceeded average parental height SDS at time of treatment completion or last visit, noted September 23, 2023.",6
LLY,Heart failure with preserved ejection fraction (HFpEF),Phase 3,12/13/19,"Phase 3 data did not meet primary endpoint - December 13, 2019.",4
MRK,Metastatic non-small cell lung cancer (NSCLC),Phase 3,9/22/23,"Phase 3 data reported that there was not an improvement in OS for patients who received KEYTRUDA plus LENVIMA compared to docetaxel, noted September 22, 2023.",6
KMDA,Pediatric patients newly diagnosed with type 1 diabetes,Phase 2,11/1/17,"Phase 2 data released November 3, 2017. No significant treatment effect noted overall.",4
VIGL,Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP),Phase 1,9/11/23,"Phase 2 data analysis demonstrated that VGL101 continued to have a favorable safety and tolerability profile and proof-of-target engagement in SAD/MAD cohorts up to 60 mg/kg with six-month interim data expected 4Q 2023, noted Septemeber 11, 2023.",6
VRTX,Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation,Approved,10/21/19,"FDA Approval announced October 21, 2019.",6
SUPN,Epilepsy,Approved,12/14/18,"FDA approval announced December 14, 2018 to expand label to include monotherapy treatment.",6
ARVN,ER+ positive / HER2- negative breast cancer,Phase 1b,12/13/21,"Phase 1 dose escalation data reported that treatment was well tolerated with no DLTs at total daily doses up to 700mg, noted December 13, 2021.",6
RHHBY,Castration-resistant prostate cancer,Phase 3,6/19/20,"Phase 3 data met one of two co-primary endpoints - June 19, 2020.",6
NVS,Multiple sclerosis (MS),Approved,8/25/23,"Approved August 25, 2023.",6
BBIO,Amyloid cardiomyopathy (ATTR-CM),Phase 3,8/27/23,"Phase 3 data presented at ESC reported an 81% survival rate on acoramidis and over 30 months, patients survived more and were hospitalized less than has been seen in prior controlled studies, noted August 27, 2023.",6
NVAX,"COVID-19, COVID-19 vaccine, Influenza",Phase 2,5/9/23,"Phase 2 data reported that the highest dose stand-alone COVID vaccine candidate achieved statistically significant anti-S IgG and neutralization responses approximately 30% higher than Novavax's prototype COVID vaccine while maintaining comparable safety, noted May 9, 2023",6
NVS,Fascioliasis,Approved,2/13/19,"FDA Approval announced February 13, 2019.",6
VTVT,Type 2 Diabetes,Phase 2,12/14/16,"Phase 2 primary endpoint met - December 14, 2016.",6
GSK,HIV,Approved,12/21/21,"Approved December 21, 2021.",6
ZLAB,Ovarian cancer,Approved,4/29/20,"FDA Approval announced April 29, 2020.",6
VALN,Lyme disease,Phase 2,9/7/23,"Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.",6
KYMR,Peripheral T-cell Lymphoma (PTCL),Phase 1a,6/14/23,"Phase 1a data continue to demonstrate robust dose-dependent target knockdown in ongoing Phase 1a dose escalation clinical trials, with no dose limiting toxicities (DLTs) observed, noted June 14, 2023.",6
MRK,Castration-resistant prostate cancer -first line,Approved,6/1/23,"FDA Approved on June 1, 2023.",6
BNOX,Social Anxiety Disorder (SAD),Phase 3,10/16/23,End of Phase 2 meeting minutes resulted in favorable outcome for the company which confirmed agreement with the FDA on its Phase 3 clinical trial expected to commence in 1Q 2024.,6
ADMP,Erectile dysfunction,NDA Filing,2/26/19,"Refusal to file letter received February 26, 2019.",5
BBIO,Autosomal Dominant Hypocalcemia Type 1 (ADH1),Phase 2b,6/13/22,"Phase 2b treatment data presented at ENDO resulted in rapid and sustained restoration of normal mineral homeostasis, with mean values of blood calcium, urinary calcium, and blood PTH within the normal range by day 5 of therapy and sustained at 24 weeks, and was well-tolerated without any reported serious adverse events. At week 24 of treatment, 92% (12/13) of participants had achieved normal trough blood calcium levels in the absence of extra-dietary calcium supplements and active vitamin D, and 77% (10/13) of participants had normal urinary calcium excretion",6
AMAM,Breast cancer,Phase 2,12/9/22,"Phase 2 safety and efficacy data demonstrated a 57.1% confirmed overall response rate (ORR) with 100% disease control rate (DCR), noted December 9, 2022.",6
BHC,Dry Eye Disease Associated With Meibomian Gland Dysfunction,Phase 3,4/13/21,"Phase 3 trial met primary endpoints - April 13, 2021.",6
RARE,Mucopolysaccharidosis 7 (MPS 7),Approved,11/15/17,"Approval announced November 15, 2017.",6
PHVS,Hereditary angioedema - prophylactic use,Phase 1,11/10/22,"Phase 1 PK data reported that food intake did not have significant effects on the time to reach therapeutic exposure, with no server or serious treatment-emergent adverse events, noted November 10, 2022.",6
RHHBY,Hemophilia A,Phase 1/2,12/12/22,"Phase 1/2 results reported a 92% reduction in ABR for all bleeds, with all-bleed ABR of 0.98 post-infusion compared to 11.62, noted December 12, 2022.",6
EIGR,Hepatitis Delta Virus (HDV) infection,Phase 3,9/12/23,"Phase 3 LIMT-2 trial discontinued due to DSMB recommendation, noted September 12, 2023.",4
MRK,Hospital-acquired bacterial pneumonia (HABP),Approved,6/3/19,"FDA approval announced June 3, 2019.",6
INCY,COVID-19,Approved,5/11/22,"FDA approval on May 11, 2022.",6
MRK,Heart Failure,Approved,1/20/21,"FDA approval announced January 20, 2021.",6
ABBV,Biosimilar candidate to Avastin (bevacizumab),Approved,9/14/17,"Approved September 14, 2017.",6
AQST,Erectile dysfunction,CRL,11/16/18,"CRL issued November 16, 2018.",5
LEGN,Relapsed/Refractory Multiple Myeloma (R/R MM),Phase 2,12/12/22,"Phase 2 26-month data reported that median PFS and OS were NR; 24-month PFS and OS rates were 52.6% and 74.2%, respectively, noted December 12, 2022.",5
BOLT,Solid Tumors,Phase 1/2,10/17/23,"Phase 1/2 initiated, noted October 17, 2023.",5
EYPT,Non-proliferative diabetic retinopathy (NPDR),Phase 2,9/11/23,"Phase 2 interim analysis of masked data showed that EYP-1901 is well tolerated with no reported drug-related ocular or systemic serious adverse events, noted September 11, 2023.",6
EXEL,Advanced Neuroendocrine Tumors,Phase 3,10/23/23,"Phase 3 data from ESMO 23 reported that the trial met the primary objective for each cohort, demonstrating that cabozantinib provided dramatic improvements in median progression-free survival (PFS) for the patients in the pNET and epNET cohorts, noted October 23, 2023.",6
AZN,"Severe, uncontrolled asthma",Approved,11/14/17,"Approval announced November 14, 2017.",6
BIIB,Chronic Kidney Disease,Phase 2,5/10/23,"Program discontinued, noted May 10, 2023.",4
CTIC,B-cell non-Hodgkin lymphoma,Phase 3,7/9/18,Phase 3 data released July 10. 2018. Primary endpoint not met.,4
BMY,Multiple Myeloma,Phase 3,3/9/20,Phase 3 trial did not meet progression-free survival primary endpoint.,4
VNDA,Bipolar Disorder,Phase 3,12/19/22,"Phase 3 trial results reported that treated patients showed a larger improvement than placebo treated patients, and this difference was highly statistically significant, noted December 19, 2022.",6
CGTX,Mild-to-Moderate Alzheimer's Disease,Phase 2,6/28/23,"Phase 2 study met its primary endpoints for safety and tolerability and showed positive effects for CT1812-treated participants as measured via quantitative electroencephalogram (qEEG), noted June 28, 2023.",6
ADAP,Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS),Phase 1,5/26/22,"Phase 1 data released May 26, 2022. The overall response rate was 36% in heavily pre-treated patients across both types of sarcomas (41% in synovial sarcoma and 10% for MRCLS), with a median duration of response of 52 weeks. As reported last year, the pivotal trial SPEARHEAD-1 met its primary endpoint for efficacy and the benefit:risk profile of afami-cel has been favorable with mainly low-grade cytokine release syndrome and tolerable/reversible hematologic toxicities.",6
NEXI,Multiple Myeloma,Phase 1/2,12/12/21,"Phase 1/2 data reported that treatment is well-tolerated without dose-limiting toxicities, noted December 12, 2021.",6
NVO,Hemophilia A,CRL,5/4/23,CRL received in 2Q 2023.,5
ABUS,Healthy volunteers,Phase 1,9/11/23,"Phase 1 discontinued, noted September 11, 2023.",4
REGN,Prurigo nodularis (PN),Approved,10/27/22,Approved in 3Q 2022.,6
KMDA,Prophylaxis of rabies disease,Approved,8/25/17,"Approval announced August 25, 2017.",6
GRCL,Diffuse large B-cell lymphoma (DLBCL),Phase 1,9/27/23,"Additional Phase 1 data presented at IMS reported an 100% overall response rate (ORR) and a 100% MRD- sCR rate among 19 transplant-eligible, high-risk NDMM patients, noted September 27, 2023.",6
MRK,Triple negative breast cancer (TNBC),Phase 3,9/19/21,"Phase 3 overall survival data showed risk of death reduced by 27%, and an increase of 6.9 months in median OS with KEYTRUDA plus chemotherapy. OS with treatment was 23 months, versus 16.1 months with chemotherapy, September 19, 2021.",6
NKTR,Solid tumors,Phase 1/2,11/12/21,"Phase 1b data from 1b/2 trial reported 1 partial response (PR) and 5 stable disease (SD). Treatment was noted to be well tolerated, November 12, 2021.",6
INCY,Myelfibrosis,Phase 2,12/12/22,"Phase 2 data reported that 28.1% (9), 3.1% (1) and 0% of patients who received daily followed by weekly dosing experienced ≥10%, ≥25% and ≥35% reduction in spleen volume, respectively, noted December 12, 2022,",6
AMAM,HER2-positive metastatic breast cancer,Phase 3,3/1/23,"Phase 3 trial met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater progression-free survival benefit compared to the control, noted March 1, 2023.",6
BCTX,Breast cancer,Phase 1/2,2/23/23,"Additional Phase 1/2 data reported that 9 of 11 (82%) patients remain alive from 2021/2022 dosing and 7 of 11 (64%) patients showed either disease control or progression-free survival, noted February 23, 2023.",6
CMRX,Allogeneic hematopoietic stem cell transplant (HCT) recipients with adenovirus (AdV),Phase 2,5/9/19,"Phase 2 trial discontinued - May 9, 2019.",4
CADL,Glioma,Phase 1,5/19/23,"Additional Phase 1 data presented at ASGCT reported a median overall survival rate at 11.8 months after a single injection, noted May 19, 2023.",6
CRDF,KRAS-Mutated Colorectal Cancer,Phase 1/2,9/10/22,"Phase 1b/2 data presented at ESMO showed durable responses to treatment, with a median duration of response (mDoR) of 11.7 months, noted September 10, 2022.",6
CERS,Chronic anemia in thalassemia patients,Phase 3,1/23/18,"Phase 3 trial met endpoints - January 23, 2018. CE mark submission due 2H 2018.",6
EIGR,Hutchinson-Gilford Progeria Syndrome (HGPS),Approved,11/20/20,"FDA approval announced November 20, 2020.",6
ITCI,Agitation in patients with dementia,Phase 2,12/18/18,"Phase 3 interim analysis December 18, 2018 noted trial to stop due to futility.",4
PCSA,Gastroparesis,Phase 2a,5/6/23,"Phase 2a data reported at DDW noted that treatment improved both gastroparesis symptom scores and gastric emptying rate, noted May 6, 2023.",6
PCRX,Postsurgical pain,Approved,10/31/11,"Approved October 31, 2011.",6
JNCE,Second-line non-small cell lung cancer (NSCLC) patients,Phase 2,8/30/22,"Phase 2 trial did not meet its primary endpoint, noted August 30, 2022.",6
MVIR,Advanced hepatocellular carcinoma (HCC),Phase 2a,10/5/23,"Phase 2a data reported that 3 patients achieved Partial Response and 10 patients Stable Disease, providing 17% Overall Response Rate (ORR) and 72% Disease Control Rate (DCR), noted October 5, 2023.",6
BMY,Non-Small Cell Lung Cancer (NSCLC),Approved,4/11/22,"Approved April 11, 2022.",6
RYTM,Alstrom Syndrome,CRL,6/16/22,"CRL issued for sNDA application for Alstrom Syndrome issued June 16, 2022.",5
PTCT,Spinal Muscular Atrophy (SMA) in babies,Approved,5/31/22,"Approved May 31, 2022.",6
RHHBY,B-Cell Non-Hodgkin's Lymphoma,Approved,6/16/23,"FDA Approved on June 16, 2023.",6
OMER,Hematopoietic stem cell-associated TMA (HSCT-TMA),CRL,10/18/21,"CRL announced October 18, 2021.",5
ABBV,Jawline definition,Approved,8/3/22,"Approved August 3, 2022.",6
AZN,Venous thromboembolism (VTE) Prevention,Approved,6/23/17,"FDA Approval noted June 23, 2017.",6
BMRN,Children under age 5 with Achondroplasia,Approved,10/23/23,"Approved October 23, 2023.",6
TAK,Complex Crohn's Perianal Fistulas (CPF),Phase 3,10/18/23,"Phase 3 trial did not meet its primary endpoint, noted October 18, 2023.",6
KALV,Diabetic macular edema (DME),Phase 2,12/9/19,"Phase 2 data did not meet primary endpoint - December 9, 2019.",4
AGEN,Solid tumors,Phase 1/2,2/9/21,"Phase 1/2 data announced February 9, 2021 - six confirmed responses.",5
LLY,Advanced solid tumors,Phase 1b,4/18/23,"Phase 1b data presented at AACR showed that dosing was well-tolerated with favorable pharmacokinetics and no apparent drug-drug interactions, noted April 18, 2023.",6
BIVI,Parkinson's disease,Phase 2a,8/28/23,"Phase 2a results reported that in comparison of Day 1 and Day 14 of treatment, PK parameters demonstrated that NE3107 administration did not affect the PK profile of levodopa, noted August 28, 2023.",6
AUPH,Lupus,Approved,1/22/21,"FDA Approval announced January 22, 2021.",6
MRK,Metastatic non-small cell lung cancer (NSCLC),Phase 1b,6/5/23,"Phase 1b presented at ASCO demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts, noted June 6, 2023",6
KMDA,AATD - Alpha-1 Antitrypsin deficiency,Phase 2,8/30/16,"Top-line data released August 30, 2016. Primary endpoint met",6
AKBA,Hyperphosphatemia,Approved,9/5/14,"Approved Sept 5, 2014.",6
ALXO,2nd line or greater gastric/gastroesophageal junction cancer,Phase 2,10/3/23,"Phase 2 data reported a confirmed overall response rate for evorpacept combination treatment was 52% compared to 22% for control treatment, noted October 3, 2023.",6
TAK,Psoriasis,Phase 2b,9/11/23,"Additional Phase 2b data reported that the safety and tolerability profile of TAK-279 in the Phase 2b trial was consistent with previous TAK-279 clinical trials, noted September 11, 2023.",6
MRK,Beta-thalassemia,Approved,11/8/19,"FDA Approval announced November 8, 2019.",6
PFE,Hemophilia B,Phase 3,12/29/22,"Phase 3 trial met primary endpoint, noted December 29, 2022.",5
GSK,Endometrial Cancer,Approved,7/31/23,"FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.",6
FGEN,Anaemia in Chronic Kidney Disease,CRL,8/11/21,"CRL announced August 11, 2021.",5
SWTX,Multiple Myeloma,Phase 1/2,6/9/23,"Phase 1/2 data presented at EHA Data from 34 patients with low-dose and 37 patients with monotherapy are presented. Patients had a median (range) age of 68 (48–81) years and a median (range) of 5 (3–14) prior lines of therapy. As of the data cutoff (Dec 9, 2022), patients received a median (range) of 4 (1–20) cycles of the combination and 3 (1–9) monotherapy cycles. For the combination and monotherapy arms, respectively, ORR was 29% (95% CI 15.1, 47.5) and 38% (22.5, 55.2) (Table). Incidence of Grade ≥3 adverse events was 76% and 65%, noted June 9, 2023.",6
HALO,Rheumatoid arthritis (RA),Approved,10/14/13,"Approved Oct 14, 2013.",6
ZLDPF,Pediatric Congenital hyperinsulinism (CHI),Phase 3,9/19/22,"Phase 3 trial additional data showed that treatment significantly reduced the requirement for intravenous (IV) glucose to maintain glycemia in neonates and infants with CHI and reduced glucose requirements to levels that potentially allow for discontinuation of IV glucose support, noted September 19, 2022.",6
NVO,Prostate cancer (CRPC),Phase 1,10/7/21,"Phase 1 initial preliminary results reported include data as of Sept. 1, 2021, from eight men enrolled in the trial. The dose was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. (3/8) patients remain on study; (5/8) patients left the study (4 due to disease progression and 1 withdrawal of consent).",5
ABBV,Rheumatoid arthritis,Approved,8/16/19,"FDA Approval announced August 16, 2019.",6
PRTA,AL Amyloidosis,Phase 3,12/12/22,"Phase 3 VITAL data reported a significant improvement in time to all-cause mortality, noted December 12, 2022.",6
PFE,ALK-positive Anaplastic Large Cell Lymphoma,Approved,1/14/21,"FDA approval announced January 14, 2021.",6
CARA,Stage 3-4 Chronic Kidney Disease-Associated Pruritus,Phase 2,12/3/19,"Phase 2 data met primary endpoint but secondary missed - December 3, 2019.",5
ACHV,Smoking cessation,Phase 3,3/3/23,"Additionnal Phase 3 data presented at the Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting confirmed that successful abstinence was observed in subgroups of smokers who received cytisinicline. Subjects who received either 6 or 12-weeks of cytisinicline treatment experienced consistently higher rates of abstinence, noted March 3, 2023.",6
BLRX,First-line pancreatic ductal adenocarcinoma (PDAC),Phase 2,9/28/23,"Phase 2 trial data presented at the AACR Special pancreatic cancer conference reported that 6 patients (55%) experienced a partial response (PR) of which 4 (36%) were confirmed PRs with one patient experiencing resolution of the hepatic (liver) metastatic lesion, noted September 28, 2023.",6
SAGE,Major Depressive Disorder (MDD),Phase 3,8/7/23,"Phase 3 additional data reported that there were no new safety signals identified, noted August 7, 2023.",5
ANAB,Endometrial Cancer,Approved,7/31/23,"FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.",6
ELTX,Acute lung injury in patients with COVID-19,Phase 3,6/29/21,"Phase 2 trial did not meet primary or secondary efficacy endpoints - June 29, 2021.",4
ACHV,Smoking cessation of cigarettes,Phase 3,5/23/23,"Phase 3 topline results reported that subjects that received treatment had 2.85 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p=0.0008). The smoking cessation rate during weeks 3 through 6 was 14.8% for cytisinicline compared to 6% for placebo, noted May 23, 2023.",6
INBX,Alpha-1 Antitrypsin Deficiency (AATD),Phase 1,11/2/22,"Phase 1 data presented at ACoP noted that treatment demonstrated the potential to achieve and maintain normal levels of functional AAT. The 120 mg/kg Q4W regimen is predicted to achieve average trough values >21.1 µM, noted November 2, 2022.",6
MRK,Complicated urinary tract infections (cUTI) and Complicated intra-abdominal infections (cIAI),Approved,7/17/19,"FDA Approval announced July 17, 2019.",6
RHHBY,Paroxysmal Nocturnal Hemoglobinuria (PNH),Phase 3,12/12/22,"Phase 3 results reported that trial met primary endpoints, noted December 12, 2022.",5
NBIX,Parkinson's disease,Approved,4/27/20,"FDA Approval announced April 27, 2020.",6
VERU,"COVID-19 / Severe Acute Respiratory Syndrome, SARS-CoV-2, Influenza A and B, Respiratory Syncytial Virus (RSV) and other viruses",Phase 3,9/26/23,"Phase 3 new study authorized by FDA, with an FDA meeting in September 2023 resulted in agreement with FDA on New Phase 3 Clinical Trial for Sabizabulin for Broader Indication, noted September 26, 2023.",6
TAK,Post-transplant Hodgkin lymphoma (HL) cancer,Approved,8/17/15,"Approved August 17, 2015 under priority review.",6
PFE,BRAF mutant melanoma cancer,Approved,6/27/18,"Approval announced June 27, 2018.",6
IMCR,Solid tumors,Phase 1,12/8/22,"Phase 1 initial data reported that one patient had a durable Partial Response (PR), with a duration of 12.7 months, one patient who had a Stable Disease (SD) converted to an unconfirmed PR after the poster data cutoff date and is still ongoing, and 5 had SD, noted December 8, 2022.",5
HRMY,Fragile X syndrome,Phase 2/3,5/13/22,"Phase 2/3 long-term data reported that sustained improvement in ABC-CFXS Social Avoidance from baseline, noted May 13, 2022.",6
ELOX,Cystic fibrosis,Phase 2,11/17/21,"Phase 2 monotherapy dosing data at 1.5mg/kg/day demonstrated a statistically significant 5.4mmol/L mean sweat chloride reduction, an established surrogate for restoration of CFTR biological activity, noted November 17, 2021.",6
SYRS,Pancreatic Cancer,Phase 1,11/14/22,"Phase 1 safety data reported that treatment was generally safe, noted November 14, 2022.",6
ENDP,Men diagnosed with hypogonadism,Approved,3/6/14,"Approved March 6, 2014.",6
SCPH,Chronic heart failure,Phase 2,7/12/22,"Phase 2 trial data reported a 37% reduction in the risk of a heart failure hospitalization relative to patients randomized to ""treatment as usual"" at day 30, noted July 12, 2022.",6
REGN,Various cancers,Phase 1,11/4/23,"Phase 1 safety data reported that a total of 11 adverse events (including one serious adverse event not considered related to SNS-101) was reported in five patients, with no dose-limiting toxicities observed, noted November 4, 2023.",6
BLPH,COVID-19,Phase 3,11/23/20,"Phase 3 trial placed on clinical hold - November 23, 2020.",4
VIR,HIV T cell vaccine,Phase 1,5/8/23,"Phase 1 initial data reported that no safety signals and no vector shedding or viremia were reported, noted June 28, 2023.",5
GSK,Chronic rhinosinusitis with nasal polyps,Approved,7/29/21,"FDA approval announced July 29, 2021.",6
IOVA,Cervical cancer,Phase 2,5/31/19,"Data released May 31, 2019 noted ORR 44%; CR 11%.",5
CORT,Pancreatic cancer,Phase 3,6/22/21,"Phase 3 initial data released June 22, 2021. 2/31 (6%) partial responses. Enrollment to be stopped. Level of benefit does not justify further study.",4
MRK,Non-small cell lung cancer (NSCLC),Approved,4/11/19,"FDA Approval announced April 11, 2019.",6
IMGO,Myelofibrosis,Phase 2,12/12/22,"Phase 2 data reported that 65% (17/26) of patients showed a decrease in Total Symptom Score (TSS), 19% (5/26) showed a ≥ 50% decrease in TSS, 66% (33/50) showed spleen volume reductions from baseline, and 28% (14/50) showed a ≥ 20% spleen volume reduction, noted December 12, 2022.",6
SONN,Healthy volunteers,Phase 1,11/2/23,"Phase 1 interim analysis presented at CTAD reported that in single doses up to 300 ng/kg and in patients with advanced cancer in repeated doses up to at least 540 ng/kg. Incorporation of a desensitizing first dose at 150-300 ng/kg and dosing every 3 weeks has been implemented to capitalize on the potential benefits of tachyphylaxis with rhIL-12, noted November 2, 2023.",6
RLMD,Major depressive disorder (MDD),Phase 3,12/7/22,"Phase 3 trial did not meet primary endpoint, noted December 7, 2022.",6
BMRN,Phenylketonuria (PKU),Approved,5/24/18,"Approval announced May 24, 2018.",6
ABBV,Atopic dermatitis,Approved,1/14/22,"Approved January 14, 2022.",6
INCY,Marginal Zone Lymphoma,NDA Filing,1/25/22,"NDA voluntarily withdrawn, noted January 25, 2022",5
RHHBY,Solid tumors and ROS1-positive non-small cell lung cancer (NSCLC),Approved,8/15/19,"FDA approval announced August 15, 2019.",6
TEVA,Schizophrenia,Approved,4/28/23,"Approved April 28, 2023.",6
LEGN,Multiple Myeloma,Phase 3,6/5/23,"Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023",6
KNSA,Prurigo nodularis,Phase 2a,4/22/20,"Phase 2a trial met primary endpoint - April 22, 2020.",6
ADCT,High-Risk Relapsed/Refractory Follicular Lymphoma,Phase 2,7/11/23,"Phase 2 trial enrollment voluntarily paused after recent review of aggregate data of the 40 patients enrolled in the trial and consultation with the Data Monitoring Committee (DMC) which signaled potentially excessive respiratory-related events, noted July 11, 2023.",4
GSK,Uncomplicated urinary tract infection (uUTI),Phase 3,4/17/23,"Both EAGLE-2 and EAGLE-3 phase 3 trials met primary endpoint of non-inferiority to nitrofurantoin; EAGLE-3 demonstrated statistical superiority, noted April 17, 2023.",6
MOR,Relapsed or refractory diffuse large B cell lymphoma,Approved,7/31/20,"FDA Approval announced July 31, 2020.",6
AZN,Dyslipidaemia,Phase 2b,4/4/22,"Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022",6
NKTR,Cancer - Metastatic Breast Cancer,Phase 3,3/17/15,"Phase 3 topline data mid March 17, 2015 did not reach primary endpoint.",4
CPRX,Spinal Muscular Atrophy (SMA) Type 3,Phase 2,1/6/21,Phase 2 trial met primary endpoint. Secondary endpoints not met. Will not pursue development further.,4
MRK,Renal cell carcinoma (RCC),Phase 3,11/1/23,"Phase 3 trial met its primary endpoint, noted November 1, 2023.",5
KYMR,Hidradenitis suppurativa / atopic dermatitis,Phase 1,12/14/22,"Phase 1 data reported that a 37% mean reduction in EASI in AD and 42% of HS patients has AN Count 0/1/2, noted December 14, 2022.",6
ABVC,Attention-Deficit Hyperactivity Disorder (ADHD),Phase 2,4/26/23,"Phase 2 study opened new sites, clinical trial continues to demonstrate that medicines derived from plants have significant therapeutic benefits with few, noted April 26, 2023.",6
AZN,Type 2 diabetes (Pediatric),Approved,7/23/21,"FDA approval announced July 23, 2021.",6
ALLO,Multiple myeloma,Phase 1/2,12/13/21,"Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.",6
FBIO,X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants,Phase 1/2,4/17/19,Phase 1/2 trial ongoing. Initial data noted in NEJM.,5
IONS,Alzheimer's disease,Phase 1/2,3/29/23,"Phase 1b trial of 1b/2 study met its primary objective of safety and tolerability. Phase 1b LTE study results showed that dosing reduced biomarkers of soluble tau in CSF (t-tau and p-tau181) in a dose-dependent and sustained manner, with all dose groups showing approximately a 60% reduction from baseline CSF tau levels by the end of the LTE .Phase 2 trial in progress, noted March 29, 2023.",6
PRAX,Major Depressive Disorder and perimenopausal depression,Phase 2a,8/16/21,"Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes.",6
IMGN,Ovarian cancer,Phase 3,11/30/21,"Phase 3 data reported that trial met primary endpoint with a confirmed objective response rate of 32.4% and median duration of response at 5.9 months, noted November 30, 2021.",6
KPTI,Diffuse Large B-Cell Lymphoma (DLBCL),Approved,6/22/20,"FDA Approval announced June 22, 2020.",6
IMAB,Acute Myeloid Leukemia / Myelodysplastic Syndrome,Phase 2,12/12/22,"Phase 2 data reported an increased CALR expression in CD33+ blasts after lemzoparlimab and AZA combination treatment and higher immune infiltrates including total, CD91+ macrophages and CD8/Treg ratio in bone marrow at baseline is associated with better clinical response, suggesting the important role of activation of tumor derived pro-phagocytic signal and effector immune cells in the anti-tumor activity mediated by combination treatment, noted December 12, 2022.",6
OPK,Benign Prostatic Hyperplasia (BPH),Phase 2b,2/1/19,"Phase 2b trial suspension announced January 31, 2019.",4
HALO,Various blood cancers,Approved,6/22/17,"Approval announced June 22, 2017.",6
ALBO,Nonalcoholic steatohepatitis (NASH),Phase 2,8/18/20,"Phase 2 trial met primary endpoint. However, development in NASH will not continue - August 18, 2020.",4
GILD,Ankylosing spondylitis,Phase 2,9/5/18,"Phase 2 data released September 6, 2018. Primary endpoint met.",6
BMY,Myelofibrosis,Approved,8/16/19,"FDA Approval announced August 16, 2019.",6
MITO,Geographic atrophy (GA) / Age-related macular degeneration (AMD),Phase 2b,5/2/22,"Phase 2b top-line data reported that trial did not meet primary endpoint, noted May 2, 2022.",6
SRRK,Spinal muscular atrophy (SMA),Phase 2,6/30/23,"Phase 2 36-month extension data presented at the Cure SMA Conference showed that continued treatment with apitegromab over the extended treatment period was associated with substantial and sustained improvement in motor function, noted June 30, 2023.",6
IONS,Dyslipidaemia,Phase 2b,4/4/22,"Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022",6
ABBV,Systemic Lupus Erythematosus (SLE),Phase 2,5/31/23,"Phase 2 data reported that trial met primary endpoint, noted May 31, 2023.",5
CYDY,COVID-19 (Mild to Moderate),Phase 2,8/11/20,"Phase 2 data released August 11, 2020 did not report primary endpoint data (TCSS at Day 14). Day 3 subgroup showed clinical improvement but not mentioned to be statistically significant. primary endpoint data for ALL treated patients NOT released. Secondary NEWS2 was met. No data on other secondary endpoints.",5
TCON,Angiosarcoma cancer,Phase 3,4/12/19,"Phase 3 interim analysis April 12, 2019 noted trial to be terminated due to futility.",4
SLGL,Acne,Approved,7/27/21,"FDA approval announced July 27, 2021.",6
TRVI,Chronic cough - Idiopathic pulmonary fibrosis (IPF),Phase 2,9/19/22,"Phase 2 full data reported that trial met primary endpoint, noted September 19, 2022.",5
BHC,Eye Brightener,Approved,12/22/17,"Approval announced December 22, 2017.",6
TNXP,Post-traumatic stress disorder (PTSD),Phase 3,12/21/20,"Phase 3 primary endpoint was not met - December 21, 2020.",4
CLLS,Acute myeloid leukemia (AML),Phase 1,5/17/23,"Phase 1 preliminary data presented at ASGCT showed that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity, noted on May 17, 2023.",6
BMY,Multiple Myeloma,Phase 3,2/10/23,"Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.",6
ARQT,Atopic dermatitis,Phase 3,9/7/23,"OLE data reported that 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively, noted September 7, 2023.",6
BLU,Chronic pruritus associated with atopic dermatitis,Phase 2b,12/13/21,"Phase 2b top-line data reported that trial did not meet the primary endpoint. Development will not continue, noted December 13, 2021.",4
IMCR,Cutaneous melanoma (mCM),Phase 1b,6/5/22,Phase 1b data to be presented at ASCO showed 1 year overall survival (OS) of approximately 75% in heavily pre-treated mCM compared to benchmark of 55%.,6
ATOS,Breast cancer,Phase 2,6/9/21,"Phase 2 final data presented June 9, 2021. Primary endpoint met.",6
ETON,Nutritional deficiencies,CRL,11/12/20,"CRL received October 2020 (announced November 12, 2020).",5
RHHBY,Cancer related diarrhea (CRD),Phase 2,12/10/21,"Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.",6
MYMD,Sarcopenia and frailty,Phase 2,10/4/23,"Phase 2 new data showed statistically significant results across Cohort 4 (1050mg), noted October 4, 2023.",6
AGTC,Achromatopsia (ACHMB3),Phase 1/2,2/8/22,"Phase 1/2 3-month data reported that there were no new Suspected Unexpected Serious Adverse Reactions (SUSARs) and that the inflammation continues to improve with steroid treatment, noted February 8, 2022.",6
ELOX,Cystic fibrosis,Phase 2,6/14/23,"Phase 2 data demonstrated clinically relevant improvement in percent predicted forced expiratory volume (ppFEV1) based on final data assessment, noted June 14, 2023.",6
VTVT,Mild Alzheimer’s disease,Phase 3,6/12/18,"Phase 3 data from Part A released April 9, 2018 did not meet endpoints. Part B data released June 12, 2018 also did not meet endpoints.",4
FRLN,Hemophilia B,Phase 1/2,12/13/21,"Phase 1/2 long term data reported a dose-dependent increase in FIX activity and FIX expression was sustained in nine of 10 patients, noted December 13, 2021.",6
SCLX,Acute lower back pain (LBP),Phase 2,9/14/23,"Phase 2 data reported that none of the subjects in the active group and 3 (8.1%) subjects in placebo group had AEs of special interest, with a meaningful reduction of pain over the first week, noted September 14, 2023.",6
AMGN,Heart failure with reduced ejection fraction (HFrEF),CRL,2/28/23,"CRL issued February 28, 2023.",5
VACC,Hepatitis B (HBV),Phase 1/2,6/21/23,"Phase 1b/2a data at EASL showed that all participants who received and experienced a >0.5 log10 reduction in HBsAg had durable responses with reductions in HBsAg persisting through to the last measurement eight months post-final dose, noted June 21, 2023.",6
IPSC,Lymphoma or Indolent Lymphoma,Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that one patient achieved a complete response that is ongoing as of five months following their first CNTY-101 infusion, noted November 2, 2023.",6
PFE,"HR+, HER2- Metastatic Breast Cancer",Approved,3/31/17,"sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.",6
ENTA,Respiratory Syncytial Virus (community-acquired),Phase 2b,10/12/23,"Phase 2b data shared at IDWeek 2023 reported that EDP-938 has a high barrier to clinical resistance, a single resistant mutation was detected in 1 of 37 sequenced EDP-938-treated participants October 12, 2023.",6
ABBV,First line unfit AML,Approved,11/21/18,"FDA approval announced November 21, 2018.",6
ABBV,Hepatitis C virus (HCV),Approved,8/3/17,"Approval announced August 3, 2017.",6
NVS,Metastatic breast cancer (mBC),Phase 2,12/10/21,"Phase 2 data reported median overall survival improvement of 26.4 months in cohort A. Primary endpoint was met in cohort C, noted December 10, 2021.",6
BIIB,Choroideremia,Phase 3,6/14/21,"Phase 3 trial did not meet primary endpoint - June 14, 2021.",4
CHRS,Metastatic or recurrent triple-negative breast cancer (TNBC),Phase 3,6/3/23,"Phase 3 data presented at ASCO reported a statistically significant improvement in PFS by BICR was demonstrated for the toripalimab arm in the PD-L1 positive subgroup, noted June 3, 2023.",6
ASND,Hypoparathyroidism,Phase 3,10/2/23,"Phase 3 clinical trial data presented at ATA, showed that 81% of adults with chronic post-surgical hypoparathyroidism treated with TransCon PTH (palopegteriparatide) achieved independence from conventional calcium and active vitamin D therapy while maintaining normal serum calcium levels during the 26-week blinded portion of the trial, compared to 6% taking placebo, noted October 2, 2023.",6
ABBV,Second-line Chronic graft-versus-host disease (GVHD),Approved,8/2/17,"Approval announced August 2, 2017.",6
INCY,Cholangiocarcinoma,Approved,4/17/20,"FDA Approval announced April 17, 2020.",6
DRMA,Acne,Phase 2b,6/27/23,"EoP2b FDA established key elements of the DMT310 Phase 3 clinical program and delineated a potential regulatory pathway for approval of DMT310, noted June 27, 2023.",6
VIRI,Long COVID-19,Phase 2,7/17/23,"Phase 2 data reported clinically and statistically significant improvements in fatigue, pain, and symptoms of autonomic dysfunction, noted July 17, 2023.",6
ESPR,Hypercholesterolemia,Approved,2/26/20,"FDA Approval announced February 26, 2020.",6
BHC,Psoriasis,Approved,4/25/19,"FDA approval announced April 25, 2019.",6
PBYI,Breast Cancer,Phase 2,6/5/23,"Phase 2 data reported an increased MYC RNA expression was observed in tumors from patients who did not derive clinical benefit from paclitaxel alone compared to those with benefit from paclitaxel alone, noted June 5, 2023.",6
CBAY,Non-alcoholic steatohepatitis (NASH),Phase 2b,5/11/20,"Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.",6
NERV,Primary insomnia,Phase 2b,6/24/19,"Phase 2b data June 24, 2019 met primary endpoint.",6
PFE,Ulcerative colitis,Phase 3,3/29/22,"Phase 3 data met the co-primary endpoints of clinical remission at both weeks 12 and 52 and all key secondary endpoints, noted March 29, 2022.",5
SNY,Rheumatoid arthritis,Approved,5/22/17,"CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.",6
ALNY,ATTR Amyloidosis,Phase 3,5/23/22,"Phase 3 18-month data reported improvements in levels of NT-proBNP and a trend towards improvement in echocardiographic parameters, noted May 23, 2022.",6
PFE,Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC),Phase 3,3/3/21,FDA approval announced March 3 2021.,6
KROS,Myelodysplastic syndromes (MDS),Phase 2,11/2/23,"Phase 2 data from ASH abstract reported that the median treatment duration was 166 days (range 6 to 649). Most participants (89.8%) had at least 1 treatment-emergent adverse event (TEAE), and 32.2% had TEAEs considered treatment-related, noted November 2, 2023.",6
SRPT,Duchenne muscular dystrophy,Phase 3,10/30/23,"Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.",6
REGN,Atopic dermatitis 6-11 year-olds,Approved,5/26/20,"FDA Approval announced May 26, 2020.",6
BMY,Hypertrophic cardiomyopathy (HCM),Approved,4/28/22,"Approved April 28, 2022.",6
HALO,Breast cancer,Approved,2/28/19,"FDA approval announced February 28, 2019.",6
MOLN,Mild to moderate COVID-19,Phase 3,3/3/21,Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.,4
ZLAB,Chronic inflammatory demyelinating polyneuropathy (CIDP),Phase 2,7/17/23,"Phase 2 trial met primary endpoints, noted July 17, 2023.",5
AVTX,COVID-19 induced Acute Respiratory Distress Syndrome (ARDS),Phase 2,3/2/21,"Phase 2 data released March 2, 2021. At both the 28-day and the 60-day final timepoints, an approximately 50% trend in mortality reduction (22.5% vs 10.8% - placebo) was observed.",6
LLY,Type 2 diabetes,Phase 3,6/4/22,Phase 3 data results presented at ADA's 82nd Scientific Sessions® and simultaneously published in the New England Journal of Medicine demonstrate A1C reductions of up to 1.5% for Trulicity compared to placebo in this underserved population.,6
CLDX,Recurrent/metastatic head and neck squamous cell cancer,Phase 1/2,6/1/19,"Phase 2 data ASCO June 1, 2019 noted 2/19 CRs + one PR.",5
INCY,Vitiligo,Approved,7/19/22,"Approved July 19, 2022.",6
VRTX,Type 1 Diabetes,Phase 1/2,10/3/23,"Phase 1/2 long term data reported that two patients with at least 1 year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1c <7.0%, noted October 3, 2023.",5
NBIX,Essential Tremor,Phase 2,8/4/22,"Phase 2 trial did not meet primary endpoints, noted August 4, 2022.",6
CLVS,Endometrial Cancer,Phase 2,6/7/22,"Phase 2 data reported that 23 subjects had clinical benefit, with 1 (4%) with CR, 9 (39%) with PR, and 13 (57%) with stable disease as best response, noted June 7, 2022.",5
GILD,Sjogren’s syndrome,Phase 2,10/24/19,Phase 2 trial did not meet primary endpoint.,4
SNY,Nasal polyps,Approved,6/26/19,"FDA Approval announced June 26, 2019.",6
DCPH,Solid tumors,Phase 1,9/10/22,"Phase 1 initial data presented at ESMO demonstrated a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested, noted September 10, 2022",6
CYTK,Heart failure with reduced ejection fraction (HFrEF),CRL,2/28/23,"CRL issued February 28, 2023.",5
SMMT,C. difficile infection (CDI),Phase 3,10/20/22,"Phase 3 trial results reported that treatment was numerically higher sustained clinical response rate (73.0% vs 70.7%) than vancomycin, but did not achieve the pre-specified superiority endpoint, noted October 20, 2022.",6
NVS,Diabetic macular edema (DME),Phase 3,8/17/21,"Phase 3 met its primary endpoints, noted August 17, 2021.",6
LNTH,Chronic pain,Approved,9/29/14,"Approved September 29, 2014.",6
COGT,Advanced Systemic Mastocytosis,Phase 2,6/10/22,"Phase 2 initial data reported that 11/11 patients achieved ≥50% reduction in serum tryptase levels by central assessment, with an 89% median reduction in serum tryptase and six of these patients achieved reduction to <20 ng/mL, noted June 10, 2022.",6
TAK,ALK+ Metastatic Non-Small Cell Lung Cancer,Approved,5/22/20,"FDA Approval announced May 22, 2020.",6
GSK,Myelofibrosis,Approved,9/15/23,"Approved September 15, 2023.",6
OBSV,Improving IVF outcomes,Phase 3,11/6/19,"Phase 3 trial did not meet primary endpoint - November 10, 2019.",4
JNJ,Ulcerative colitis,Phase 2b,10/24/22,"Phase 2b study data reported that treatment led to a mean of 52.1 percent of IV TREMFYA week 12 clinical nonresponders to achieve clinical response at week 24, noted October 24, 2022.",6
VAXX,COVID-19 prevention,Phase 3,12/2/22,"Phase 3 trial met primary and key secondary endpoint, noted December 2, 2022.",5
FWBI,Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency,Phase 2,8/18/21,"Phase 2 top line data released August 18, 2021. Clinically meaningful improvements in the primary efficacy endpoint noted.",6
VIRI,Fibromyalgia,Phase 2b,9/19/22,"Phase 2b trial did not meet primary endpoint, noted September 19, 2022.",6
MBRX,Acute myeloid leukemia (AML),Phase 1b,5/2/23,"Phase 1b data noting one CR and determined that the first cohort dose as safe, noted May 2, 2023.",5
SUPN,ADHD,Approved,4/2/21,"FDA approval announced April 2, 2021.",6
BMRN,Achondroplasia,Approved,11/19/21,"Approved November 19, 2021.",6
GILD,HIV,CRL,3/1/22,"CRL announced March 1, 2022.",5
MRK,Non-small cell lung cancer (NSCLC),Phase 2,3/16/23,"Phase 2 trial did not meet primary endpoint, noted March 16, 2023.",6
SRNE,Rheumatoid Arthritis,Phase 1b,11/13/22,"Full results of the Phase 1b proof of concept study noted that switching from once weekly 50 mg SC injections to once weekly 25 mg lymphatic delivery significantly reduced disease activity without local adverse events or drug delivery-related immune responses, noted November 14, 2022.",6
ALXO,Acute myeloid leukemia (AML),Phase 1,12/12/22,"Additional Phase 1 data reported that treatment is well-tolerated with 4 patients achieving a response, noted December 12, 2022.",6
LUMO,Pediatric Growth Hormone Deficiency (PGHD),Phase 2,9/26/23,"Phase 2 interim data analysis reported that LUM-201 restored GH secretion to similar levels seen in normally growing children, noted September 26, 2023.",5
VACC,Human papillomavirus (HPV),Phase 1/2,4/17/23,"Additional Phase 1/2a data reported that treatment was generally well-tolerated and was administered with no product-related grade 3 unsolicited adverse events and no product-related SAEs, noted April 17, 2023.",6
SAGE,Essential tremor,Phase 2,4/12/21,"Phase 2 trial met primary endpoint (P=0.049) - April 12, 2021. 62% of patients were down-titrated to from 60mg to 45 mg or 30 mg with the high dose not well tolerated.",6
GOSS,Nasal polyps / chronic rhinosinusitis,Phase 2,10/13/20,"Phase 2 trial did not meet primary endpoint - October 13, 2020.",4
LCTX,Geographic atrophy (GA),Phase 1/2,10/5/23,"Phase 1/2 results presented at EURETINA Congress reported that 5 patients had an average of 4.4 letter BCVA gain by 3 months and 12.8 letter BCVA gain by 1 year compared to baseline, noted October 5, 2023.",6
CYAD,Colorectal cancer,Phase 1b,3/2/22,"Phase 1b trial initiated December 16, 2021. Phase 1b trial voluntarily paused due to reports of two fatalities that presented with similar pulmonary findings, noted February 28, 2022. Clinical hold announced.",6
IMMP,Solid tumors,Phase 1,10/24/23,"Phase 1 additional efficacy and tolerability data reported at ESMO showed strong 71.4% Overall Response Rate, 90.5% Disease Control Rate, 10.1-month median Progression Free Survival (PFS), and median Overall Survival (OS) that has not been reached, despite 81% of patients having low or negative PD-L1 expression, noted October 24, 2023.",6
TLSA,Secondary Progressive Multiple Sclerosis (SPMS),Phase 2a,9/26/23,"Phase 2a trial in na-SPMS initiated, noted September 26, 2023.",5
ZYME,HER2-amplified biliary tract cancers,Phase 2,6/5/23,"Phase 2 pivotal trial recruitment completed, noted April 28, 2022. Phase 2 full data presented at ASCO reported a confirmed objective response rate (cORR) of 41.3%, median duration of response (DOR) of 12.9 months, and median progression-free survival (PFS) of 5.5 months, noted June 5, 2023.",6
MNOV,Progressive multiple sclerosis (progressive MS),Phase 2b,10/28/17,"Phase 2b top-line data released October 28, 2017 at Joint ECTRIMS – ACTRIMS meeting. Primary endpoint met.",6
ASND,Follicular lymphoma,Phase 2,6/17/23,"Phase 2 data presented at ICML showed that 97% of patients with R/R FL treated with Breyanzi achieved a response, with 94% achieving a complete response, and 81.9% of responders in ongoing response at 12 months, noted June 17, 2023.",6
ATNM,Acute myeloid leukemia (AML),Phase 3,6/12/23,"Additional Phase 3 data at EHA presented results demonstrate unprecedented bone marrow transplant access and outcomes with Iomab-B on June 12, 2023",6
KRTX,Acute psychosis in patients with schizophrenia,Phase 3,8/8/22,"Phase 3 trial met primary endpoint and secondary endpoints, demonstrating a statistically significant 9.6-point reduction in PANSS Total Score compared to placebo at Week 5, noted August 8, 2022.",6
BGNE,First-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL),Approved,1/19/23,"Approved January 19, 2023.",6
MREO,Osteogenesis Imperfecta,Phase 2,10/14/23,"Additional Phase 2 data reported that the annualized fracture rate across all 24 patients in the Phase 2 portion of the study was reduced by 67%, noted October 14, 2023.",6
IXHL,"Rheumatoid arthritis, inflammatory bowel disease and lung inflammation",Phase 1,5/2/23,"Phase 1 results reported no adverse events of concern and no serious adverse events reported, noted May 2, 2023.",5
CABA,Systemic Lupus Erythematosus,Phase 1,5/1/23,"Fast track designation granted by FDA on May 1, 2023.",5
PGEN,Recurrent Respiratory Papillomatosis (RRP),Phase 2,6/20/23,"Received Breakthrough Therapy Designation (BTD) by FDA on June 20, 2023.",5
EDAP,Rectal endometriosis,Phase 2,1/31/23,"Phase 2 data reported that 3.3% of treated patients presenting Clavien 2 complications and zero patients presenting Clavien 3 complications, with significant decrease of the symptoms level from the first post-treatment evaluation, and the reduction of symptoms was maintained at three and six months following HIFU treatment, noted January 31, 2023.",6
ADAG,Solid tumors,Phase 1/2,11/10/22,"Phase 1b/2 data reported two partial responses and one confirmed response, noted November 10, 2022.",5
BMY,Myelodysplastic syndromes (MDS) cancer,Approved,4/3/20,"FDA Approval announced April 3, 2020.",6
IRWD,Irritable Bowel Syndrome,Phase 2,5/27/20,"Phase 2 trial did not meet primary endpoint - May 27, 2020.",4
EXEL,BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma,Approved,7/30/20,"FDA approval announced July 30, 2020.",6
AZN,Metastatic non-small cell lung cancer (NSCLC),Phase 1b,6/5/23,"Phase 1b presented at ASCO demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts, noted June 6, 2023",6
INCY,Anemia and Post-essential Thrombocythemia Myelofibrosis,Phase 1/2,12/12/22,"Phase 1/2 data reported predominantly grade 1/2 TEAEs and no dose-limiting toxicities (DLTs), noted December 12, 2022.",5
FRTX,Primary Axillary Hyperhidrosis,Phase 3,4/23/21,"Phase 3 open-label data announced April 23, 2021 - generally well-tolerated. Clinically meaningful improvement in axillary hyperhidrosis severity.",6
VSTM,KRAS mutant non-small cell lung cancer (NSCLC),Phase 1/2,10/16/23,"Phase 1/2 initial data reported that the confirmed objective response rate (ORR) was 25% (3/12) across efficacy-evaluable patients and seen in both KRAS G12C inhibitor resistant (14.3%; 1/7) and naïve (40%; 2/5) patients, noted October 16, 2023.",6
NVO,Type 2 diabetes,Approved,8/25/17,"Approval announced August 25, 2017.",6
GTHX,Second/third-line treatment of NSCLC,Phase 2,11/3/21,"Phase 2 trial discontinued, noted November 3, 2021.",4
AMGN,Chronic Heart Failure,Approved,4/15/15,"Approved April 15, 2015.",6
ALNY,Hepatitis B (HBV),Phase 2,6/24/23,"Phase 2 24 or 48 weeks plus up to 48 weeks of PEG-IFN-⍺ (cohorts 4 and 5 combined), 26% (8/31) achieved HBsAg loss at the end of treatment and 16% (5/31) sustained HBsAg loss 24 weeks after the end of treatment, noted June 24, 2023.",6
ITOS,Solid tumors,Phase 1/2,6/4/21,"Phase 1/2 single agent data at ASCO June 4, 2021 noted 5/21 patients with stable disease in including two partial responses.",5
RVPH,Schizophrenia,Phase 3,10/30/23,"Phase 3 trial met primary endpoint, noted October 30, 2023.",5
INCY,Myelofibrosis,CRL,3/23/23,"CRL issued by the FDA on March 23, 2023.",5
PIRS,Gastric cancer,Phase 1,11/9/19,"Phase 1 data at SITC November 9, 2019 noted 2/18 response rate.",4
RHHBY,Spinal Muscular Atrophy (SMA) type 2 and 3,Phase 2/3,3/20/23,"Phase 2/3 data reported increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease, noted March 20, 2023.",6
LIFE,COVID-19,Phase 2,1/4/21,"Phase 2 data released January 4, 2021. Primary safety endpoint met. Median time to recovery of 5.5 days compared to 6 days in the placebo group.",6
GILD,Osteoarthritis,Phase 2,10/15/20,"Phase 2 trial did not meet primary endpoint - October 15, 2020.",4
RHHBY,Non-Hogkin Lymphona,Phase 1/2,12/11/21,"Phase 1b/2 results reported an objective response rate (ORR) of 65% with a median duration of response (DOR) not reached (observed DOR: 0.1+ to 15.3+ months). At data cut-off, one patient had achieved a CR after an initial CR that had lasted for 15 months, noted December 11, 2021.",6
AZN,Small cell lung cancer (SCLC),Phase 3,9/18/21,"Phase 3 data showed sustained efficacy after a median follow up of more than three years, with a 29% reduction in the risk of death versus chemotherapy alone. Updated median OS was 12.9 months versus 10.5 for chemotherapy. 17.6% of patients treated with IMFINZI plus chemotherapy were alive at three years, versus 5.8% of patients treated with chemotherapy alone, September 18, 2021.",6
PRFX,Bunionectomy,Phase 3,6/8/23,"Phase 3 clinical trial Company's supplier of the API (active pharmaceutical ingredient) has received a deficiency notice from the FDA related to its Drug Master File (DMF). As a result, the second part of the Phase 3 trial is expected to commence once the supplier has provided the required information to the FDA and the deficiency notice has been resolved, noted June 8, 2023.",5
GLMD,Non-Alcoholic Steatohepatitis (NASH),Phase 3,5/17/22,"Open label portion of study met objective, noted May 17, 2022.",5
GILD,Nonalcoholic steatohepatitis (NASH),Phase 3,4/25/19,"Phase 3 data April 25, 2019 did not meet primary endpoint.",4
TALS,COVID-19 in living donor kidney transplant (LDKT) patients,Phase 2,6/7/22,"Additional Phase 2 data presented at ATC reported that none of these 18 patients lost chimerism as a result of their COVID-19 vaccination, noted June 7, 2022.",6
FOLD,Pompe Disease,Approved,9/28/23,"FDA Approved on September 28, 2023.",6
VYNE,Acne,Phase 2,6/2/20,"Phase 2 data released June 2, 2020.",5
RHHBY,Acute Myeloid Leukemia (AML),Approved,10/16/20,"FDA approval announced October 16, 2020.",6
GILD,Hepatitis C virus (HCV),Approved,7/18/17,"Approval announced July 18, 2017.",6
RHHBY,Breast Cancer,Phase 2,5/26/22,"Phase 2 final analysis presented at ASCO reported that greater suppression of Ki67 was observed at surgery with giredestrant + P, with an ORR of 50%, noted May 26, 2022.",6
LLY,Atopic Dermatitis,Phase 1,10/13/23,"Phase 1b data presented at EADV 2023 showed dose-dependent efficacy reported across all endpoints for REZPEG demonstrating a rapid onset of action and continuing benefit for 36 weeks after 12-week treatment period, noted October 13, 2023.",6
BPMC,Indolent and smoldering systemic mastocytosis (SM),Phase 2,2/27/23,"Phase 2 data presented at AAAAI reported a statistically significant and clinically meaningful improvement in total symptom score that deepened over time, noted February 27, 2023.",6
CORT,Nonalcoholic steatohepatitis (NASH),Phase 1b,7/17/23,"Phase 1b data demonstrated that miricorilant effectively reduced liver fat, improved liver health and key metabolic and lipid measures and is well-tolerate, noted July 17, 2023.",6
IOBT,Metastatic solid tumors,Phase 2,10/23/23,"Additional Phase 2 data presented at ESMO reported that 10 out of 18 evaluable NSCLC patients (56%) had achieved a partial response as their best overall response and 3 out of 6 evaluable SCCHN patients had achieved partial response as their best overall response, noted October 23, 2023.",6
ARGX,Immune thrombocytopenia (ITP),Phase 3,5/5/22,"Phase 3 top-line data reported that trial met primary endpoint, noted May 5, 2022.",5
JNJ,Rheumatoid arthritis,CRL,9/22/17,"CRL issued September 22, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.",6
HRMY,Autism Spectrum Disorder (ASD),Phase 2,10/8/21,"Phase 2 long term data displayed a statistically significant improvement in ABC-C Subscale scores through 38 weeks. Only 7 patients (19%) experienced an AE that was deemed to be treatment related. Of the 10 treatment-related AEs reported, 7 were application site-related (application site reaction, pruritus, and dryness) and 1 each of sleep disorder, increased appetite, and pollakiuria, noted October 8, 2021.",6
MRK,Hepatocellular Carcinoma,Phase 3,2/19/19,"Phase 3 data February 19, 2019 did not meet primary endpoints.",4
NBIX,Parkinson's Disease,Phase 2,12/22/20,"Phase 2 trial placed on clinical hold - December 22, 2020.",4
RHHBY,"Hepatocellular Carcinoma (HCC), Solid tumors",Phase 1/2,4/17/23,"Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.",6
BIIB,Alzheimer’s disease,Phase 3,9/13/19,Phase 3 trial discontinued due to unfavorable risk-benefit ratio.,4
NXTC,Solid tumors,Phase 1/2,11/13/21,"Combined Phase 1 and Phase 2 data reported one complete response (CR) and one partial response (PR), with disease control rate across all tumors in both studies was 37% and a median progression-free survival (PFS) of 5.0 months, noted November 13, 2021.",5
CRSP,CD19+ B-cell malignancies,Phase 1,10/12/21,"Phase 1 data demonstrated 58% overall response rate (ORR) and 38% complete response (CR) rate with a single dose at Dose Level 2 (DL2) and above on an intent-to-treat (ITT) basis. Durable responses achieved with six-month CR rate of 21% and longest response on-going at over 18 months after initial infusion, noted October 12, 2021.",6
AZN,Non-small cell lung cancer (NSCLC),Phase 3,10/23/23,"Phase 3 reduced the risk of disease progression or death by 25% in overall population and by 37% in patients with non-squamous tumors , noted October 23, 2023.",6
OCUP,Mydriasis (RM),Approved,9/27/23,"FDA Approval on September 27, 2023.",6
EXEL,Hepatocellular Carcinoma,Phase 3,3/14/22,"Phase 3 final OS data reported no improvement, noted March 14, 2022.",6
SVRA,Nontuberculous mycobacterial (NTM) lung infection with cystic fibrosis (CF),Phase 2a,9/3/20,"Phase 2a trial to be discontinued - noted September 3, 2020.",4
RARE,GNE Myopathy,Phase 3,8/22/17,"Phase 3 data released August 22, 2017 - endpoints not met.",4
MRK,Von Hippel-Lindau Syndrome,Approved,8/13/21,"FDA approval announced August 13, 2021.",6
NVS,Secondary progressive multiple sclerosis,Approved,3/26/19,"FDA approval announced March 26, 2019.",6
MNOV,Nonalcoholic steatohepatitis (NASH),Phase 2,4/2/18,"Noted April 2, 2018 that trial has been terminated early due meeting one of two primary endpoints (data from second endpoint not released).",6
PFE,Renal cell carcinoma (RCC),Approved,5/14/19,"FDA Approval announced May 14, 2019.",6
NBIX,Tardive dyskinesia,Approved,10/5/17,"Approval for sNDA 80 mg capsules announced October 5, 2017.",6
BLUE,Sickle Cell Disease,Phase 3,2/16/21,"Phase 3 trial temporarily suspended due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) - February 16, 2021.",4
BLTE,Stargardt disease (STGD1),Phase 1/2,5/5/22,"Phase 1b/2 results reported that 69.2% of patients reported delayed dark adaptation (DDA), 61.5% reported a gain in BCVA (ETDRS score) in at least one eye, and reductions of RBP4 levels by 80-90%, noted May 5, 2022.",6
RETA,Friedreich’s ataxia (FA),Approved,6/28/23,"PAS approved June 28, 2023.",6
RDHL,Prostate Cancer,Phase 2,11/30/21,"After additional review of the unaudited data, the Phase 2 trial did not meet the primary endpoint in the combination with enzalutamide, noted November 30, 2021.",4
IONS,Spinal muscular atrophy (SMA),Approved,12/23/16,"Approved December 23, 2016.",6
VBIV,Covid-19,Phase 1,9/27/23,"Phase 1 interim data reported a 25% reduction in GMT against Wuhan after 5 months vs. peak responses, noted September 27, 2023.",6
PMCB,Pancreatic cancer,Phase 2b,10/30/20,"Phase 2b IND placed on clinical hold - October 1, 2020.",4
BMEA,Acute myeloid leukemia (AML),Phase 1,10/17/23,"Phase 1 dosing initiated, noted October 17, 2023.",5
GNFT,Primary biliary cholangitis (PBC),Phase 3,6/30/23,"Phase 3 data reported that trial met primary endpoint, noted June 30, 2023.",5
GKOS,Progressive keratoconus,Phase 3,2/25/21,"Phase 3 trial met primary endpoint - February 25, 2021.",6
MRK,Metastatic non-small cell lung cancer (NSCLC),Phase 3,2/8/22,"Phase 3 trial did not meet primary endpoints of overall survival, safety profile was consistent with that observed in the overall JAVELIN clinical development program, noted February 8, 2022.",6
PFE,ALK+ NSCLC,Approved,11/2/18,"FDA approval announced November 2, 2018.",6
MRK,Ovarian Cancer,Phase 1/2,6/6/22,"Results from Phase 1 and Phase 2 expansion cohort presented at ASCO reported that G3/G2 immune-related AE and injection site reactions were observed in 1/1 and 1/3 pts treated at DL1, and in 1/0 and 2/2 pts at DL2, respectively. At Phase 1, one patient with MSI-High clear cell subtype had an ongoing CR after 26 months of follow-up, 2 patients had PR and 6 SD. There were 1 PR and 3 SD on P2EC, of which 1 and 2, respectively, achieved response greater than 12 weeks, noted June 6, 2022.",6
EDIT,Leber Congenital Amaurosis type 10 (LCA10),Phase 1/2,9/29/21,"Phase 1/2 initial data displayed 2 (66%) subjects in the mid-dose cohort showed improvements in BCVA, FST, and/or mobility navigation. However, retinal tears were reported by 2 (50%) subjects, 1 report of hypotony in the low dose cohort, as well as 1 retinal hemorrhage. Treatment in the adult high-dose cohort continues and pediatric mid-dose cohort commencing September 29, 2021.",5
MRK,Gastric or Gastroesophageal Junction Adenocarcinoma,Approved,5/5/21,"FDA approval announced May 5, 2021.",6
RXRX,Clostridium Difficile Infection,Phase 1,9/5/23,"Phase 1 trial achieved its primary objectives of assessing the safety, tolerability and pharmacokinetic profile of REC-3964. REC-3964 has been well tolerated with no serious adverse events (SAEs) reported, noted September 5, 2023.",6
PFE,Migraine - preventative,Approved,5/27/21,"FDA approval announced May 27, 2021.",6
LLY,First-Line Nonsquamous Non-small cell lung cancer (NSCLC),Approved,6/5/18,"FDA approval (label expansion) announced June 5, 2018.",6
NVS,Acute heart failure,Phase 3,3/22/17,"Phase 3 data released March 22, 2017 - primary endpoints not met.",4
LLY,Type 2 Diabetes,Phase 3,12/9/20,"Phase 3 data released December 9, 2020 - significantly reduced A1C and body weight.",6
GSK,Lupus Nephritis,Approved,12/17/20,"FDA approval announced December 17, 2020.",6
VALN,Chikungunya vaccine,Phase 3,12/5/22,"Phase 3 trial met primary endpoint, noted December 5, 2022.",5
GSK,Severe hypereosinophilic syndrome (HES),Approved,9/25/20,"FDA approval announced September 25, 2020.",6
SNDX,Chronic Graft versus host disease (cGvHD),Phase 2,7/24/23,"Phase 2 trial results met its primary endpoint across all cohorts with an overall response rate (ORR) of 74% at a dose of 0.3 mg/kg administered every two weeks, noted July 24, 2023.",5
AVDL,Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery,Approved,6/3/13,"Approved June 3, 2013.",6
AMGN,Asthma,Approved,12/17/21,"Approved December 17, 2021.",6
NAVB,Rheumatoid arthritis,Phase 2b,9/15/22,"Phase 2b data reported that the quantitative TIL uptake in the hands and wrists of patients is proportional to the amount of macrophage involvement in an individual RA patient's joint inflammation, noted September 15, 2022.",5
OPK,Children - growth hormone deficiency,Approved,6/28/23,"FDA Approved on June 28, 2023.",6
QLGN,Lactose intolerance,Phase 3,9/12/19,Phase 3 data failed to meet primary endpoint.,4
INVA,cIAI (complicated intra-abdominal infections),Approved,8/27/18,"FDA approval announced August 27, 2018.",6
EDIT,Sickle Cell Disease,Phase 1/2,6/9/23,"Phase 1/2 data presented at EHA reported that all five patients treated with EDIT-301 successfully engrafted and all four RUBY patients treated are free of vaso-occlusive events since infusion, noted June 9, 2023.",6
SNY,Asthma,Approved,10/19/18,"FDA approval announced October 19, 2018.",6
CANF,Rheumatoid arthritis,Phase 2,10/6/20,"Phase 3 interim analysis recommended trial to be discontinued - October 6, 2020.",4
AVDL,Hypotension,Approved,5/2/16,"Approved May 2, 2016.",6
PFE,First-Line Treatment of Philadelphia Chromosome Positive Chronic Myeloid Leukemia,Approved,12/19/17,"Approval announced December 19, 2017.",6
ALDX,Proliferative vitreoretinopathy (PVR),CRL,6/21/23,"CRL issued on June 21, 2023. Aldeyra plans to have a Type-C meeting on 2H 2023.",6
BLUE,Relapsed/refractory multiple myeloma,Approved,3/26/21,"FDA approval announced March 26, 2021.",6
ZVRA,Niemann-Pick disease Type C (NPC),CRL,6/18/21,"CRL issued June 18, 2021.",5
SNY,Chronic spontaneous urticaria (CSU),CRL,10/20/23,"CRL issued by the FDA, noted October 20, 2023.",5
BGNE,Esophageal Squamous Cell Carcinoma (ESCC),Phase 3,6/4/21,"Phase 3 poster at ASCO June 4, 2021 noted median OS of 8.6 months compared to 6.3 months in the chemotherapy arm.",6
IMVT,Graves Ophthalmopathy,Phase 2a,3/30/20,"Phase 2a initial data March 30, 2020 noted 65% mean reduction in total IgG.",6
TYME,Prostate cancer,Phase 2,2/14/19,"Phase 2 updated data February 14, 2019. Median PSA doubling-time improved by 34%.",6
PTGX,Plaque psoriasis,Phase 2b,7/5/23,"Phase 2b trial met all primary and secondary efficacy endpoints, noted July 5, 2023.",5
TCON,Mesothelioma cancer,Phase 2,10/7/21,"Phase 2 data reported that 9 patients reported stable disease as best response with Grade 3 TRAEs including lymphopenia (4) and anemia (1), noted October 7, 2021.",5
ENTA,Hepatitis B,Phase 1,11/18/21,"Development discontinued, noted November 18, 2021.",4
PDSB,Head and neck cancer,Phase 2,10/23/23,"Phase 2 data presented at ESMO reported that the combination of PDS0101 and KEYTRUDA resulted in increased polyfunctionality reflected in T cells expressing 5 or more cytokines, noted October 23, 2023.",6
APM,Neuroblastoma,Phase 1/2,6/22/23,"Phase 1b/2a trial has been repurposed for small molecule drug , noted June 22, 2023.",5
ADAG,Solid tumors,Phase 1/2,4/18/23,"Dose escalation data presented at AACR showed a compelling safety profile for ADG126, with no Grade 3 or higher TRAEs reported at repeat doses up to 20 mg/kg. Across all dose levels, the disease control rate was 37% among 27 evaluable patients, noted April 18, 2023.",6
SNY,Pompe disease,Approved,8/6/21,"FDA approval announced August 6, 2021.",6
RANI,Rheumatoid arthritis,Phase 1,11/1/23,"Program paused, noted November 1, 2023.",4
QURE,Hemophilia B,Approved,11/22/22,"Approved November 22, 2022.",6
LRMR,Friedreich’s ataxia (FA),Phase 2,7/25/23,"FDA Phase 2 clearance for dose exploration trial and OLE trial, Initiation of open-label extension trial with 25 mg daily dosing expected in 1Q 2024, noted July 25, 2023. Phase 2 50mg cohort topline data expected 1H 2024. Phase 2 interim data expected 4Q 2024.",5
ADCT,Hodgkin lymphoma (HL),Phase 2,6/22/21,"Phase 2 interim data presented June 22, 2021. Overall response rate (ORR) was 66.3% (67/101 patients) with a complete response rate (CRR) of 27.7% and partial response rate (PRR) of 38.6%. Median duration of response has not been reached.",4
LIAN,Demodex blepharitis,Phase 3,10/30/23,"Phase 3 results demonstrated statistically significant mite eradication in patients treated compared to vehicle, noted October 30, 2023.",6
PFE,Glioblastoma (GBM),Phase 2/3,10/31/23,"Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.",5
MRK,Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia (HABP/VABP),Approved,6/4/20,"FDA approval announced June 4, 2020.",6
NVS,Metastatic castration-resistant prostate cancer (mCRPC),Phase 3,10/23/23,"Phase 3 trial met its primary endpoint with a clinically meaningful and statistically significant benefit in radiographic progression-free survival, noted October 23, 2023.",5
HRTX,Postoperative nausea and vomiting (PONV),Approved,9/16/22,"Approved September 16, 2022.",6
INAB,Glioblastoma,Phase 2,6/5/23,"Data presented at ASCO 2023 showed that 100% of treated patients to date have exceeded historical median progression-free survival, with two patients that received three doses remaining progression-free at 23.5 and 19.4 months, respectively noted on June 5, 2023.",6
AMGN,Secondary hyperparathyroidism (SHPT),Approved,2/7/17,"Approved February 7, 2017.",6
UTHR,Pulmonary Arterial Hypertension (PAH),CRL,4/28/20,"CRL announced April 29, 2020.",5
PFE,Colorectal cancer and gastrointestinal (GI) cancer,Phase 1/2,6/5/23,"Initial Phase 1b/2 data showed a 50% response rate, including two confirmed partial responses (cPR) and one unconfirmed partial response (uPR), and 67% disease control rate in EC-naïve response evaluable patients at the highest dose of ERAS-007 tested (100 mg BID-QW), development deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023",6
AZN,Lung cancer,Phase 3,8/21/19,"Phase 3 data released August 21, 2019 did not meet primary endpoint.",4
BMY,Ulcerative Colitis,Phase 2,10/7/21,"Phase 2 trial did not meet primary or secondary endpoints, October 7, 2021.",4
ORGS,Acute pancreatitis,Approved,8/22/18,"Approved August 22, 2018.",6
BMY,Myelofibrosis,Phase 2,12/9/19,Phase 2 data at ASH December 2019 noted primary endpoint met in 10%~32% of patients across four cohorts.,4
RARE,Angelman Syndrome,Phase 1/2,7/18/22,"Phase 1/2 UK data reported that treatment displayed a meaningful improvement in clinical disease and an acceptable safety profile, noted July 18, 2022.",6
ADPT,Relapsed/Refractory Multiple Myeloma,Phase 1/2,12/12/22,"Phase 1/2 final data reported that treatment significantly improved the degranulation of NK cells after 4-hour stimulation with K562 or PMA/Ionomycin, noted December 12, 2022.",6
MNOV,Idiopathic pulmonary fibrosis (IPF),Phase 2,8/12/21,"Phase 2 data released August 12, 2021 - no clinically meaningful trends.",4
GSK,Multiple Myeloma,Phase 1/2,6/9/23,"Phase 1/2 data presented at EHA Data from 34 patients with low-dose and 37 patients with monotherapy are presented. Patients had a median (range) age of 68 (48–81) years and a median (range) of 5 (3–14) prior lines of therapy. As of the data cutoff (Dec 9, 2022), patients received a median (range) of 4 (1–20) cycles of the combination and 3 (1–9) monotherapy cycles. For the combination and monotherapy arms, respectively, ORR was 29% (95% CI 15.1, 47.5) and 38% (22.5, 55.2) (Table). Incidence of Grade ≥3 adverse events was 76% and 65%, noted June 9, 2023.",6
PRAX,Major Depressive Disorder,Phase 2/3,6/6/22,Phase 2/3 data did not achieve statistical significance on the primary endpoint.,6
EIGR,Hyperinsulinemic hypoglycemia after bariatric or other gastrointestinal surgeries,Phase 2b,6/13/22,"Phase 2b data reported that trial met primary and secondary endpoints, noted June 13, 2022.",5
PFE,"Diffuse Large B-cell Lymphoma (DLBCL), Cutaneous T-cell lymphoma (CTCL)",Phase 1,12/11/17,Phase 1a/b updated data released at ASH 2017 - 5/18 objective response rate.,4
INCY,Triple-negative breast cancer,Phase 2,12/10/21,"Phase 2 data reported that treatment is tolerated, noted December 10, 2021.",6
VRNA,COPD,Phase 2,1/14/19,"Phase 2 trial did not meet primary endpoint - January 13, 2019.",4
COCP,Hepatitis C,Phase 2a,1/22/19,"Phase 2a initial data released January 22, 2019. SVR12 66%.",5
HEPA,Non-alcoholic steatohepatitis,Phase 2b,6/15/23,"Phase 2b Data and Safety Monitoring Board (DSMB) review and issued a ""study may proceed without modification"" clearance, study expected to be completed by 1Q 2024, noted June 15, 2023.",5
IMMP,Melanoma,Phase 1,5/30/18,"Phase 1 data released May 30, 2018 showed ORR of 61% (11/18 patients).",6
PTGX,Beta-thalassemia,Phase 2,10/11/21,"Phase 2 clinical hold lifted October 11, 2021.",5
VALN,Chikungunya Vaccine,Phase 3,12/21/21,"Phase 3 trial met its primary endpoint, demonstrating that three consecutively manufactured vaccine lots elicited equivalent immune responses measured by neutralizing antibody titer GMT ratios on Day 29 after vaccination.",6
TNXP,Fibromyalgia,Phase 3,6/15/23,"Phase 3 met the primary endpoint on its RELIEF trial, noted June 15, 2023",5
TAK,Thyroid Carcinoma,Phase 3,9/20/21,"Phase 3 data showed a median progression-free survival was 11.0 months for CABOMETYX compared with 1.9 months for placebo. Objective response rate (ORR) favored CABOMETYX at 11%, including one complete response, versus 0% for placebo. Median overall survival was 19.4 months, September 20, 2021.",6
ITCI,Bipolar depression,Phase 3,7/8/19,"Phase 3 trial did not meet primary endpoint - July 8, 2019.",4
OBSV,Uterine fibroids,Phase 3,10/20/21,"Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.",5
ESPR,Cardiovascular Diseases,Phase 3,8/26/23,"Phase 3 total event data presented at ESC reported that bempedoic acid shows 20% risk reduction in MACE-4 and 17% risk reduction in MACE-3, noted August 26, 2023.",6
GRTS,COVID-19 vaccine,Phase 1,10/11/23,"Data presented at IDweek reported that treatment was well tolerated in both HIV-negative participants and PLWH irrespective of SARS-CoV-2 serostatus at baseline, October 11, 2023.",6
MNKD,Pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease (PH-ILD),Approved,5/24/22,"Approved May 24, 2022.",6
AIM,Ovarian cancer,Phase 2,3/9/22,"Phase 2 data reported 2 complete responses, 3 partial responses, 3 stable diseases, 5 progressive diseases and a total clinical benefit rate of 61.6%, noted March 9, 2022.",5
PFE,Spinocerebellar Ataxia (SCA),Phase 3,7/27/23,"The FDA informed Biohaven that it would not review the recently submitted NDA application for troriluzole given that the study's primary endpoint was not met and thus, would not permit a substantive review. The communication from the FDA indicated that the Company may request a Type A meeting within 30 days, noted July 27, 2023.",6
CKPT,Cutaneous squamous cell carcinoma (CSCC),BLA Filing,1/4/23,"Phase 1 pivotal top-line data reported that the trial met the primary endpoint, noted January 25, 2022. Phase 1 interim efficacy results reported an objective response rate of 54.8%, noted June 16, 2022. BLA submitted to FDA, noted January 4, 2022.",5
MRK,Renal Cell Carcinoma (RCC) - cancer,Phase 2,6/12/17,"Phase 2 data released June 12, 2017 - primary endpoint not met.",4
RIGL,COVID-19,Phase 2/3,10/11/23,"Phase 2/3 trial enrollment ceased based on the recommendations of DSMB, noting that here was an extremely low likelihood of fostamatinib providing benefits related to the primary outcome, noted October 11, 2023.",5
NBRV,Complicated urinary tract infections (cUTI),CRL,6/19/20,"CRL issued April 30, 2019. Further CRL received June 19, 2020.",5
AMGN,Neuromyelitis Optica Spectrum Disorder,Approved,6/11/20,"FDA Approval announced June 11, 2020.",6
MOR,Rheumatoid Arthritis,Phase 2b,10/21/18,"Phase 2b released October 21, 2018 did not meet primary endpoint.",4
IMRA,Sickle cell disease,Phase 2a,12/13/21,"Phase 2a 12-month OLE data showed that dose was generally well-tolerated as a monotherapy as well as in combination with hydroxyurea. There were no clinically-significant changes in lab safety data, ECGs or vital signs, and no patients have discontinued the study due to adverse events",5
PRTA,Previously-treated patients with AL amyloidosis and persistent cardiac dysfunction,Phase 2b,4/23/18,"Phase 2b trial did not meet primary endpoint - April 23, 2018.",4
MITO,Barth syndrome,NDA Filing,10/20/21,"Refusal to File letter from FDA on October 20, 2021, due to issues with Phase 2 trial and open label extension.",4
PCRX,Nerve blocker in lower extremity surgeries,Phase 3,9/6/22,"Phase 3 trial met primary endpoint, noted September 7, 2022.",5
SGEN,HER2+ Metastatic Breast Cancer (MBC),Phase 3,8/19/23,"Phase 3 trial met primary endpoint, noted August 19, 2023",5
QNCX,Alzheimer’s disease,Phase 2/3,11/11/21,"Phase 2/3 data reported that the 40 mg demonstrated equivalent or better efficacy compared 80 mg, as well as a superior safety profile. Benefits were shown in ADAS-Cog11, CDR-SB, MMSE, and NPI, with increasing separation from placebo throughout the study consistent with disease modification. Benefits were not seen on ADCS-ADL at either dose, noted November 11, 2021.",6
LLY,Early Alzheimer's disease,Phase 3,6/11/18,Announced discontinuation of trial due to futility.,4
NVS,"Atherosclerotic cardiovascular disease (ASCVD), increased risk of ASCVD or heterozygous familial hypercholesterolemia (HeFH)",Phase 3,8/28/23,"Phase 3 data reported that eight in ten patients achieved target LDL-C threshold, noted August 28, 2023.",6
BPMC,EGFR exon 20 insertion-positive NSCLC,Phase 1/2,6/5/23,"Phase 1/2 initial clinical data presented at ASCO reported a partial CNS response and a CNS complete response, noted June 5, 2023.",6
OCGN,Retinitis Pigmentosa,Phase 1/2,9/13/23,"Phase 1/2 clinical study update suggested continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline, noted September 13, 2023.",6
PRQR,Retinitis pigmentosa,Phase 1/2,11/18/21,"Additional Phase 1b/2 data reported a mean benefit of 13 letters after a single injection at 72 weeks, noted November 18, 2021",5
PFE,Bosimilar to Humira (adalimumab),Approved,11/18/19,"FDA Approval announced November 18, 2019.",6
CNSP,Glioblastoma (GBM) (adult),Phase 2,8/14/23,"Phase 2 interim update demonstrated its capability to be an innovative treatment in GBM that is safe and well tolerated, which has the potential to be a novel and effective therapy for this disease, noted August 14, 2023.",6
DYN,Myotonic Dystrophy Type 1 (DM1),Phase 1/2,9/20/23,"Phase 1/2 initiated Septmember 6, 2022. Phase 1/2 Orphan Drug Designation was granted on September 20, 2023.",5
TAK,Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP),NDA Filing,6/20/23,"Phase 3 full result findings showed a clinically significant reduction in relapse rate as well as a delayed time to relapse compared to placebo when used as a maintenance therapy, noted June 20, 2023. NDA under current review by the FDA.",6
RHHBY,Non-squamous non-small cell lung cancer (NSCLC),Approved,12/6/18,"FDA Approval announced December 6, 2018.",6
VIR,Mild to moderate COVID-19,Phase 3,3/3/21,Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.,4
XENE,Adult Focal Epilepsy,Phase 2b,12/4/22,"Additional Phase 2b data reported that seizure freedom for ≥6-month and ≥12-month consecutive durations was achieved in 17.5% and 10.5% of patients, respectively, noted December 4, 2022.",6
ADMP,Anaphylaxis,Approved,9/27/18,"FDA Approval announced September 27, 2018.",6
KNSA,Pericarditis,Approved,3/18/21,"FDA approval announced March 18, 2021.",6
ABBV,Relapsed or refractory Chronic Lymphocytic Leukemia (CLL),Approved,6/11/18,"Approval announced June 11, 2018.",6
XNCR,COVID-19 antibody,Approved,5/26/21,"FDA Emergency Use Authorization filing approval announced May 26, 2021.",6
PFE,Remicade (infliximab) biosimilar,Approved,12/13/17,"Approval announced December 13, 2017.",6
GTHX,Metastatic colorectal cancer (mCRC),Phase 3,2/13/23,"Phase 3 topline data reported that achieved its co-primary endpoints related to severe neutropenia but failed to achieve a significant response in overall response rate (ORR) and preliminary measures of survival, noted February 13, 2023.",6
VRDN,Thyroid eye disease (TED),Phase 1,8/15/22,"Phase 1 study demonstrated that treatment achieved a substantially extended half-life of 30-40 days and the feasibility of a convenient, low-volume, subcutaneous injection paradigm of 2mL 300mg, noted August 15, 2022.",6
APLIF,Parasitic and anaerobic bacterial infections,Approved,9/25/23,"Approved September 25, 2023.",6
SNY,Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR,Approved,8/10/18,"FDA Approval announced August 10, 2018.",6
RNXT,Locally advanced pancreatic cancer (LAPC),Phase 3,6/29/23,"Phase 3 interim data presented at ESMO GI demonstrated an eight-month median PFS benefit, 15 versus 7 months, in delaying the progression of cancer for patients receiving treatment with RenovoGem versus standard-of-care, noted June 29, 2023.",6
IONS,Immunoglobulin A nephropathy (IgAN),Phase 2,11/7/22,IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months,6
BFRI,Actinic Keratosis,Phase 1,11/21/22,"Phase 1 Pharmacokinetics study and results discussed with FDA; to be submitted to FDA along with 3 tubes safety study. Phase 3 study launched, noted November 21, 2022.",5
AZN,Bladder cancer,Phase 3,3/6/20,Phase 3 data did not meet primary endpoint.,4
VNDA,Gastroparesis,Phase 3,2/4/22,"Phase 3 data reported that the trial did not meet its primary endpoint, noted February 4, 2022.",6
TERN,Nonalcoholic steatohepatitis (NASH),Phase 2a,11/12/21,"Phase 2a safety and efficacy clinical data reported no differences from placebo in percentage change in low density lipoprotein (LDL) cholesterol or high density lipoprotein (HDL) cholesterol. Reductions in alanine transaminase (ALT) and MRI proton density fat fraction (MRI-PDFF) were reported in the 10 and 15 mg groups, and significant reductions in gamma-glutamyl transferase (GGT) in all dose groups, noted November 12, 2021.",4
ACAD,Parkinson’s disease psychosis (PDP),Approved,4/29/16,"Approved April 29, 2016.",6
GMAB,Cervical Cancer,Approved,9/20/21,"Approved September 20, 2021.",6
NKTR,Renal Cell Carcinoma (RCC),Phase 3,4/14/22,"Phase 3 data reported that trial did not met its endpoints of objective response rate (ORR) and overall survival (OS), noted April 14, 2022.",5
GRTS,Solid tumors,Phase 1/2,9/12/22,"Phase 2 portion data from Phase 1/2 trial presented at ESMO demonstrated early evidence of efficacy with a 39% molecular response rate in evaluable patients with late-line microsatellite-stable colorectal cancer (MSS-CRC) and non-small cell lung cancer (NSCLC). Data support moving trial into earlier lines of treatment, noted September 12, 2022.",6
CLNN,Parkinson's Disease,Phase 2,8/5/21,"Phase 2 trial met primary endpoint - August 5, 2021.",6
SESN,Non-muscle invasive bladder cancer (NMIBC),CRL,8/13/21,"CRL announced August 13, 2021.",5
NVAX,Influenza,Phase 3,3/24/20,"Phase 3 top-line data met primary endpoints - March 24, 2020.",6
NVCR,Gastric cancer,Phase 2,6/3/22,"Phase 2 data reported a confirmed objective response rate of 50%, with a 10.3 month duration of response and 72% one-year survival rate, noted June 3, 2022.",5
BMRN,Duchenne Muscular Dystrophy (DMD),CRL,1/14/16,CRL issued January 14 2016.,5
BLUE,TDT and the β0/β0genotype,Phase 3,6/11/20,"Phase 3 updated data at EHA June 12, 2020. Six of eight evaluable patients achieved transfusion independence,",6
ZYNE,Adult Epilepsy Patients with Refractory Focal Seizures,Phase 2,8/7/17,"Phase 2 data released August 7, 2017 - endpoints not met.",4
CLLS,Multiple myeloma,Phase 1/2,12/13/21,"Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.",6
OCS,Diabetic macular edema (DME),Phase 3,10/6/23,"Phase 3 data presented at EURETINA noted that 6 times a day dosing of OCS-01 was a highly effective Loading Dose, noted October 6, 2023.",6
MITO,Mitochondrial disease associated with pathogenic nDNA mutations (nPMD),Phase 3,12/20/19,"Phase 3 data did not meet primary endpoint - December 20, 2019.",4
APTX,"Parkinson’s disease cognitive impairment, healthy volunteers",Phase 2,2/27/23,"Phase 2 study did not demonstrate sufficient efficacy to support further development, noted February 27, 2023.",6
CDAK,Solid tumors,Phase 1/2,6/30/22,"Phase 1/2 data reported that two patients experienced tumor regressions in both injected and non-injected lesions, including a partial response in one patient, noted June 30, 2022.",5
LBPS,Solid tumors,Phase 1/2,3/23/22,"Phase 1/2 part B data reported that the renal cell carcinoma (RCC) group met the primary endpoint, noted March 23, 2022.",5
IMTX,Solid tumors,Phase 1b,10/24/23,"FDA Granted RMAT on October 24, 2023.",5
VTRS,Multiple sclerosis (MS) - Generic for Copaxone,Approved,10/4/17,"Approval announced October 4, 2017.",6
BNTX,COVID-19 Vaccine (Booster),Phase 1/2,5/23/22,"Phase 1/2/3 booster trial in children reported that a 80.3% vaccine efficacy, noted May 23, 2022.",5
ALDX,Psoriasis / atopic asthma / COVID-19,Phase 2,3/29/22,"Phase 2 top-line data reported statistically significant improvements in all three indications with no serious adverse events, noted March 29, 2022.",6
PHAR,Activated phosphoinositide 3-kinase delta syndrome (APDS),Approved,3/24/23,"Approved March 24, 2023.",6
BOLT,"HER2-Expressing Solid Tumors, gastric cancer, including gastroesophageal junction cancer",Phase 2,10/23/23,"Phase 2 updated safety data presented at ESMO supports the selection of 20 mg/kg q2w as the recommended Phase 2 dose (RP2D), noted October 23, 2023.",6
BMY,Spinal Muscular Atrophy (SMA),Phase 3,9/14/23,"Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.",5
ATHX,Acute Respiratory Distress Syndrome,Phase 2,8/6/21,"Phase 2 open-label data released August 6, 2021. 20/28 days were ventilator free compared with 11/28 for standard therapy.",6
SNY,Chronic Spontaneous Urticaria (CSU),Phase 3,2/18/22,"Phase 3 trial did not meet the primary endpoint, noted February 18, 2022.",6
CVAC,COVID-19 vaccine,Phase 2/3,10/12/21,"CVnCoV withdrawn from regulatory review due to focus on second generation candidate, noted October 12, 2021.",4
BGNE,Cervical Cancer (CC),Phase 2,10/22/23,"Phase 2 data presented at ESMO reported that the ORR was 22.5%, with 13 complete responses; 76.8% had a durable response of ≥6 mo, noted October 22, 2023.",6
INCY,COVID-19 associated cytokine storm,Phase 3,12/14/20,"Phase 3 top-line did not meet primary endpoint - December 14, 2020.",4
AVXL,Mild to moderate Alzheimer’s disease,Phase 2/3,9/14/23,"Phase 2b/3 full data reported that the least-squares mean (LSM) change from baseline to 48 weeks between the blarcamesine and placebo groups were −1.783; for ADAS-Cog13, and −0.456 for CDR-SB in patients with early Alzheimer's disease, noted September 14, 2023.",5
AGIO,Advanced hematologic malignancies with an IDH2 mutation,Approved,8/1/17,"Approval announced August 1, 2017.",6
NOVN,Human papillomavirus (HPV),Phase 2,11/29/16,"Phase 2 data released November 29, 2016. Primary endpoint met.",6
GILD,Non-Small Cell Lung Cancer (NSCLC),Phase 2,9/11/23,"Phase 2 trial data reported that in both cohorts, the ORR was 56%, and DCR was 82%. Median duration of response (DoR) was not reached at the time of data cut-off, and DoR rate at six months was 88% in both cohorts, noted September 11, 2023.",6
PFE,COVID-19 vaccine,Phase 2/3,11/4/22,"Phase 2/3 clinical trial demonstrated robust neutralizing immune response one month after a 30-µg booster dose of the COVID-19 vaccine (Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5)). Omicron BA.4/BA.5-neutralizing antibody titers increased 13.2-fold from pre-booster levels in adults older than 55 years of age and 9.5-fold in adults 18 to 55 years of age, noted November 4, 2022.",6
IMGN,Cancer - ovarian cancer and relapsed endometrial cancer,Phase 3,9/28/23,"Additional Phase 3 data reported at ESGO noted that in patients with prior PARPi, ORR in the ELAHERE arm was 45%, including 7 CRs, compared to 17%, with no CRs, in the IC chemotherapy arm, noted September 28, 2023.",5
CGEM,Solid tumors,Phase 1,11/3/23,"Phase 1 Initial clinical biomarker data demonstrated that CLN-619 increases MICA expression on the tumor cell surface, consistent with previously reported preclinical data and supporting the proposed mechanism of action, noted November 3, 2023.",6
NUVL,ALK non-small cell lung cancer (NSCLC),Phase 1,10/13/23,"Phase 1 trial showed preliminary signs of activity observed in heavily pre-treated patients with ALK-positive NSCLC, including in subgroups of patients who have previously received a 2nd generation ALK TKI and lorlatinib, have brain metastases, or have single or compound ALK resistance mutations, noted October 13, 2023.",6
EQRX,Stage III Non-small cell lung cancer (NSCLC),Phase 3,8/7/22,"Phase 3 data reported that treatment demonstrated a statistically significant improvement in progression-free survival versus placebo, noted August 7, 2022.",6
TARS,Lyme disease,Phase 1,12/15/22,"Phase 1 data data showed that treatment was well tolerated and safety data supports progression to Phase 2a (Carpo), noted December 15, 2022.",6
BMY,First-line Renal cell carcinoma (RCC),Phase 3,9/16/21,"Additional Phase 3 survival data released at ESMO September 16, 2021. Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy. Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up.",6
BMY,Chronic lymphocytic leukemia (CLL),Phase 1/2,6/6/23,"Phase 1/2 data presented at ASCO reported that the primary endpoint of CR/CRi rate was met at 18.4%, and the ORR was 42.9% and was not statistically significant, noted June 6, 2023.",6
PLX,Fabry disease,Approved,5/10/23,"Approved May 10, 2023.",6
CALT,IgA nephropathy,Approved,12/15/21,"Approved December 15, 2021.",6
BMY,Stage IIA to IIIB non-small cell lung cancer (NSCLC),Phase 3,10/21/23,"Phase 3 data presented at ESMO reported that NIVO + chemo/NIVO improved pCR rates (25.3% vs 4.7%; odds ratio, 6.64 [95% CI, 3.40–12.97]) and MPR rates (35.4% vs 12.1%; odds ratio: 4.01 [2.48–6.49]) vs chemo/PBO, noted October 21, 2023.",6
VRDN,Thyroid Eye Disease (TED),Phase 1/2,4/27/23,"Phase 1/2 clinical data reported that 80% (4/5) of VRDN-001 responders showed durability of effects at 12 weeks, noted April 27, 2023.",6
FULC,Facioscapulohumeral muscular dystrophy (FSHD),Phase 3,9/8/23,"Phase 3 enrollment completed, noted September 8, 2023",5
SLS,"Hematologic malignancies, acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and lymphoma",Phase 2a,10/30/23,"FDA granted fast track designation, noted October 30, 2023.",5
IONS,Chronic hepatitis B virus (CHB),Phase 2b,6/27/22,"Phase 2b data readout reported that up to 68% of patients on NA therapy and up to 65% of patients not on NA achieved HBsAg <100 IU/mL at the end of treatment, noted June 27, 2022.",6
AVIR,COVID-19,Phase 2,10/19/21,"Phase 2 trial did not meet primary endpoint, noted October 19, 2021.",4
CLSD,Wet AMD using suprachoroidal delivery,Phase 2,7/30/23,"Phase 2 additional data reported meaningful reductions in Anti-VEGF injection burden with stable to improved CRT through month 6, noted July 30, 2023.",6
AZN,Chronic obstructive pulmonary disease (COPD),Approved,7/24/20,"FDA approval announced July 24, 2020.",6
BIIB,Lupus,Phase 2b,10/23/18,"Phase 2b data released October 23, 2018 - primary endpoint not met.",5
TAK,"Higher-risk myelodysplastic syndromes (HR-MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML)",Phase 3,9/1/21,"Phase 3 data did not meet primary endpoint September 1, 2021.",4
IMAB,COVID-19,Phase 2,8/11/21,"Phase 2 interim analysis released August 11, 2021. Mechanical ventilation free (MVF) rate (83.6% vs 76.7%) by day 30, lower mortality rate (4.9% vs 13.3%) compared to placebo.",6
CLVS,Ovarian Cancer - First-line maintenance treatment,PDUFA,6/26/23,Company went bankrupt in 2022 not information disclosed about the PDUFA.,5
AZN,Wilson disease,Phase 3,6/23/22,"Phase 3 data reported at the International Liver Congress noted greater improvements in neurological scores for those treated with ALXN1840 compared to SoC, however, there were no significant differences between treatment groups observed at 48 weeks, noted June 23, 2022.",6
DRRX,COVID-19 Patients with Acute Liver or Kidney Injury,Phase 2,3/4/21,Phase 2 trial to be discontinued due to limited number of patients eligible or willing to enroll.,4
ADAP,Ovarian Cancer,Phase 1,11/8/22,"Phase 1 data reported an increased ORR now 43% in ovarian cancer with one new response, noted November 8, 2022.",5
ABBV,Presbyopia,Approved,10/29/21,"Approved October 29, 2021.",6
NVS,Complicated urinary tract infections (cUTI),Approved,8/29/17,"Approved August 29, 2017.",6
SNY,Coronavirus COVID-19,Phase 3,9/1/20,"Phase 3 global trial did not meet primary endpoint - September 1, 2020.",4
BCRX,"Paroxysmal nocturnal hemoglobinuria (PNH), Healthy Volunteers",Phase 1,10/26/23,"Phase 1 PoC trial with PNHl initiated, noted October 26, 2023.",5
MRK,First-line ovarian cancer,Approved,5/8/20,"FDA Approval announced May 8, 2020.",6
BMY,"Tumors harboring ALK, ROS1, or NTRK1-3 alterations",Phase 1/2,10/23/21,"Phase 1/2 early clinical data demonstrated confirmed responses in (3/4) TKI-naïve patients, noted October 23, 2021.",6
CCCC,Non-Small Cell Lung Cancer (NSCLC),Phase 1a,7/5/23,"IND Cleared by FDA, July 5, 2023.",5
ACRS,Common warts (verruca vulgaris),Phase 2,10/24/19,Phase 3 data met all endpoints - second Phase 3 trial.,6
NAMS,Alzheimer's Disease,Phase 2a,9/21/23,"Phase 2a data observed reductions of 11% and 12% in 24- and 27-hydroxycholesterol in cerebrospinal fluid (CSF), respectively, indicating potential improvement of cholesterol metabolism in the brain. Observed 8% increase in Aβ42/40 ratio, a key biomarker of AD risk, suggesting improvement in disease pathology, noted September 21, 2023.",6
ALBO,Alagille syndrome (ALGS),Approved,6/13/23,"FDA Approved on June 13, 2023.",6
CDXS,Exocrine pancreatic insufficiency (EPI),Phase 1,2/23/23,"Phase 1 interim data reported that no safety issues occurred, with no SAEs or discontinuations, noted February 23, 2023.",4
PLX,Fabry disease,Phase 3,2/10/21,"Phase 3 final data presented February 10, 2021 - key objectives for safety and efficacy.",5
MRK,First-line ovarian cancer following platinum-based chemotherapy,Approved,12/19/18,"FDA Approval announced December 19, 2018.",6
LLY,Hepatocellular Carcinoma,Approved,5/13/19,"FDA approval announced May 13, 2019.",6
ONCY,Breast cancer,Phase 1,12/8/22,"Additional dat presented at the San Antonio Breast Cancer Symposium (SABCS) reported that disease control, partial response (PR) or stable disease (SD), was achieved in thirteen of fourteen evaluable patients (93%), with twelve (86%) showing tumor shrinkage from baseline, with 50% achievign a partial response, noted December 8, 2022.",6
GSK,HIV,Approved,7/2/20,"FDA Approval announced July 2, 2020.",6
JAZZ,COVID-19,Phase 2,12/13/21,"Phase 2 data reported that treatment in patients with grade 5 WHO COVID 19 ARDS does not induce bleeding, and is associated with rapid restoration of respiratory function (73% of patients). No oxygen support was needed at discharge and a 1-month OS rate of 89% was observed, which is higher than historical controls (77%) treated in the same setting, noted December 13, 2021.",6
BMY,Non-Small Cell Lung Cancer (NSCLC),Phase 3,9/11/23,"Phase 3 data demonstrated consistent, durable benefits of Opdivo plus Yervoy vs. chemotherapy at six years, regardless of PD-L1 expression, noted September 11, 2023.",6
ASLN,Gastric cancer,Phase 2,1/13/19,"Phase 2 trial did not meet primary endpoint - January 13, 2019.",4
RHHBY,Babies with pre-symptomatic spinal muscular atrophy (SMA),Phase 3,10/4/23,"Phase 3 OLE met its primary endpoint with 80% of babies sitting without support for at least 5 seconds after 1 year of Evrysdi, noted October 4, 2023.",5
EIGR,Hepatitis delta virus (HDV),Phase 2,11/12/19,"Phase 2 data presented at AASLD November 12, 2019 - Median HDV RNA decline was 3.4 log IU/mL (IQR: 2.9-4.5, p<0.0001).",6
GSK,dMMR endometrial cancer,Approved,2/10/23,"Full approval granted February 10, 2023.",6
CGTX,Mild-to-Moderate Alzheimer's Disease,Phase 2,3/28/23,"Phase 2 biomarker data reported that treatment resulted in a significant shift in levels of prion protein (PRPN), noted March 28, 2023.",6
AQST,Lennox-Gastaut Syndrome,Approved,8/31/18,"Tentative approval announced August 31, 2018.",6
JNJ,Coronary Artery Disease or Peripheral Artery Disease,Phase 3,2/8/17,"Phase 3 trial stopped early due to positive efficacy - February 8, 2017.",6
CNTX,Breast Cancer - 2nd-line,Phase 2,12/8/22,"Phase 2 data reported a 4-month PFS rate of 44%, noted December 8, 2022.",5
TSVT,Multiple Myeloma,Phase 3,2/10/23,"Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.",6
PTGX,Polycythemia vera,Phase 2,12/12/21,"Phase 2 data reported that in all 16 erythrocytotic PV patients, rusfertide demonstrated rapid reduction of hematocrit below 45% within weeks, noted December 12, 2021.",6
RHHBY,Urothelial cancer,Phase 3,1/24/20,"Phase 3 trial did not meet primary endpoint - January 24, 2020.",4
SCPH,Heart Failure,Phase 3,7/13/21,"Phase 3 data released July 13 2021, study halted early due to highly statistical reduction in costs of $17,753 (p<0.0001) per study subject",6
SRPT,Duchenne muscular dystrophy,Approved,9/19/16,"Approved September 19, 2016.",6
IFRX,Chronic Autoimmune and Inflammatory Diseases,Phase 1,9/11/23,"Phase 1 SAD results demonstrated an excellent safety and tolerability profile and a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile, confirming INF904´s best-in-class potential, noted September 11, 2023.",6
RIGL,COVID-19,Phase 2,4/13/21,"Phase 2 trial met primary endpoint of safety - April 13, 2021.",6
TNYA,Heart Failure with Preserved Ejection Fraction,Phase 1,10/9/23,"Phase 1 data presented at HFSA demonstrated safety and tolerability in healthy participants with dose-proportional pharmacokinetics and robust target engagement, noted October 9, 2023.",6
MRK,Prophylaxis of cytomegalovirus (CMV) disease in adult kidney transplant recipients at high risk (D+/R-),Approved,6/5/23,"Approved on June 5, 2023.",6
APLT,Galactosemia,NDA Filing,9/6/23,"Company pre-NDA meeting with the FDA completed, noted September 6, 2023. NDA submission expected 4Q 2023.",6
VRCA,Genital warts,Phase 2,11/10/20,"Phase 2 primary endpoint met - November 10, 2020.",6
OBSV,Uterine fibroids,Phase 3,10/20/21,"Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.",5
OPNT,Opioid Overdose,Approved,5/23/23,"Approved May 22, 2023.",6
VIR,Hepatitis B,Phase 2,6/24/23,"Phase 2 part A preliminary 48-week post-treatment safety, tolerability and antiviral activity data demonstrated that combination resulted in a 2.7-3.1 log10 IU/mL decline in HBsAg levels at the end of treatment, noted June 24, 2023.",6
CRNX,Acromegaly,Phase 3,9/11/23,"Phase 3 topline data reported that trial met its primary endpoint, noted September 11, 2023.",5
AZN,COVID-19 (post-exposure prophylaxis),Phase 3,6/15/21,"Phase 3 trial did not meet primary endpoint - June 15, 2021.",4
PFE,Multiple system atrophy (MSA),Phase 3,9/27/21,"Phase 3 trial did not met primary or secondary endpoints, noted September 27, 2021.",4
PFE,Streptococcus Vaccine,Phase 2,7/19/23,"Phase 2 results showed that the investigational vaccine, GBS6, was generally well-tolerated and generated robust maternal antibody responses that were efficiently transferred to infants, noted July 19, 2023.",6
NVS,Chronic myeloid leukemia (CML),Phase 3,6/10/23,"Additional Phase 3 data reported at EHA poster showed that overall MMR rates at wk 96 were higher with ASC than BOS regardless of the last prior TKI received, noted on June 9, 2023",6
PFE,HIV,Approved,7/2/20,"FDA Approval announced July 2, 2020.",6
EIGR,Secondary lymphedema,Phase 2,10/16/18,"Phase 2 data released October 16, 2018 did not meet endpoints.",4
PRTC,Ulcerative colitis (UC),Phase 2,10/4/23,"Phase 2 dosing was initiated and FDA granted fast track designation (FTD), noted October 4, 2023.",5
ARAV,Clear Cell Renal Cell Carcinoma (ccRCC),Phase 1/2,5/25/23,"Updated Phase 1b/2 data from ASCO abstract reported an objective response rate (ORR) of 50% in this population (n=12), compared to 38% (n=13) in patients with no prior VEGF-TKI, noted May 25, 2023.",5
QTRX,Superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS),Approved,4/25/23,"Approved April 25, 2023.",6
SMMT,Duchenne muscular dystrophy,Phase 2,6/27/18,"Phase 2 top-line data released June 27, 2018. Primary endpoint not met.",4
NKTR,Atopic Dermatitis,Phase 1,10/13/23,"Phase 1b data presented at EADV 2023 showed dose-dependent efficacy reported across all endpoints for REZPEG demonstrating a rapid onset of action and continuing benefit for 36 weeks after 12-week treatment period, noted October 13, 2023.",6
NKTR,Systemic lupus erythematosus (SLE),Phase 2,4/27/23,"Phase 2 to be moved to from Lupus to Atopic dermatitis after disappointing results, noted April 27, 2023.",5
SLS,Acute Myeloid Leukemia (AML),Phase 3,11/14/22,"The median OS is considerably longer than originally anticipated, and thus the overall duration of the REGAL study is now expected to be longer than initially predicted",5
RGNX,Homozygous familial hypercholesterolemia (HoFH),Phase 1/2,8/6/20,"Development to be discontinued - noted August 6, 2020.",4
ENTA,Non-alcoholic steatohepatitis (NASH),Phase 2b,10/4/21,"Phase 2b interim analysis showed 1.0 mg as the optimal dose. The monontherapy has been discontinued and priority is placed on the combinations, noted October 4, 2021.",5
XLO,Solid Tumors,Phase 1/2,11/3/23,"Phase 1/2 trial data presented at SITC showed initial evidence of dose-dependent disease control rate with 50% disease control rate at higher doses (≥2.8 mg/kg) and 31% disease control rate across all dose levels in a range of solid tumor types, including cold tumors, noted November 3, 2023.",6
LGND,Postpartum depression,Phase 2,7/23/19,"Phase 2 negative data released July 23, 2019.",4
MOR,Membranous nephropathy (MN),Phase 1/2,11/4/21,"Phase 1b/2a data reported that creatinine ratio (UPCR) results at 6 months of treatment showed a decrease in 6 of 10 patients, with 4 patients having a decrease of >=50% from baseline. The first patient who reached the 12-month time point showed a complete immunologic response and a partial clinical response, noted November 4, 2021.",5
EXEL,Renal Cell Carcinoma (RCC),Phase 3,9/8/22,"Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.",6
VTRS,Mydriasis (RM),Approved,9/27/23,"FDA Approval on September 27, 2023.",6
SGEN,Solid Tumors,Phase 1,6/3/23,"Phase 1 data at ASCO reported that treatment-emergent adverse events (TEAEs) were observed in 88.5% of pts: 50.7% were Grade ≥ 3 (21.6% related), and 37.2% were serious (8.1% related); 6.1% of pts discontinued treatment due to TEAEs, noted June 3, 2023.",6
BNTX,"Cancer, PD-(L)1-resistant non-small cell lung cancer",Phase 1/2,6/2/23,"Phase 1/2 data reported that the ORR was 29.6% and DCR was 70.4%, including one complete response, 7 partial responses and 11 patients with stable disease, noted June 2, 2023.",5
PHVS,Hereditary angioedema - on demand,Phase 2,7/21/23,"Phase 2 data reported that trial met primary and all key secondary endpoints, noted July 21, 2023.",5
VCYT,Prostate cancer,Phase 3,10/4/23,"Phase 3 trial validation of a genomic classifier presented at the ASTRO meeting noting that only the Decipher Prostate risk score was independently associated with metastasis-free survival (MFS; HR 1.12, 95% CI) and distant metastasis (DM; sHR 1.22, 95% CI), compared to standard risk factors including Gleason score, T-stage and prostate-specific antigen (PSA) level, noted October 4, 2023.",6
RHHBY,Non-small cell lung cancer (NSCLC),Phase 3,8/1/22,"Phase 3 trial met co-primary endpoints, noted August 2, 2022. Regulatory filing planned.",5
TCON,Wet age-related macular degeneration (Wet-AMD),Phase 2,3/9/20,"Development to be discontinued - March 9, 2020.",4
HRTX,Prevention of chemotherapy-induced nausea and vomiting (CINV),Approved,2/26/19,FDA additional approval allowing IV injection in addition to current IV infusion.,6
MRNS,Focal onset seizures,Phase 3,6/13/16,"Phase 3 data did not meet endpoints - June 13, 2016",4
PBLA,Type 1 Diabetes,Phase 2,6/15/23,"Phase 2 abstract presented at ENDO highlighted that β-cell ODC inhibition protects β-cell health and function in the context of the proinflammatory T1D milieu. A 3-month course of oral DFMO was well tolerated with a favorable AE profile in persons with recent-onset T1D. Higher doses were associated with greater β-function compared to placebo. DFMO may have utility as a β-cell preservation therapy in recent-onset T1D, noted June 15, 2023",6
BMY,Progressive pulmonary fibrosis (PPF),Phase 2,10/24/23,"Breakthrough Therapy Designation (BTD) granted by FDA on October 24, 2023.",5
BIIB,Parkinson's disease,Phase 2,2/3/21,"Phase 2 trial did not meet primary endpoint - February 3, 2021.",4
MRK,Non-Small Cell Lung Cancer,Phase 3,11/9/20,"Phase 3 trial to discontinue due to futility - November 9, 2020.",4
ATXS,"Hereditary angioedema (HAE), healthy volunteers",Phase 1a,2/24/23,"Phase 1a trial data reported a half-life of 117 days, supporting dosing every three months or less, noted February 24, 2023.",5
NVO,"Heart failure with preserved ejection fraction (HFpEF) and obesity, Type 2 diabetes",Phase 3,8/25/23,"Phase 3 data reported a mean change in the KCCQ-CSS of a 16.6-point increase at 52 weeks with semaglutide 2.4 mg vs 8.7 points with placebo, leading to an estimated treatment difference of 7.8 points, noted August 25, 2023.",6
CTXR,CTCL non-Hodgkin lymphoma,CRL,7/29/23,"CRL received on July 29, 2023.",5
PFE,Ankylosing spondylitis,Approved,12/14/21,"Approved December 14, 2021.",6
TNGX,MTAP-deleted tumors,Phase 1/2,5/9/23,"Safety and efficacy data reported dose-dependent decreases in tumor SDMA with minimal or no decrease in normal tissue, noted May 9, 2023.",5
LUMO,Pediatric Growth Hormone Deficiency (PGHD),Phase 2,6/21/23,"Phase 2 new data shared at KOL shows durable response after 12 months, noted June 21, 2023.",6
AZN,Non-small cell lung cancer (NSCLC),Phase 3,9/9/23,"Phase 3 data presented at WCLC reported a reduced the risk of disease progression or death by 38% compared to TAGRISSO alone and a combination extended median PFS by 8.8 months, noted September 9, 2023.",6
RHHBY,COVID-19,Phase 3,9/18/20,"Phase 3 trial met primary endpoint, with patients 44% less likely to progress to ventilation or death compared to patients who received placebo plus standard of care - September 18, 2020. No statistical difference in mortality.",6
ATHA,Mild-to-moderate Alzheimer's disease,Phase 2,4/25/23,"Phase 2 statistically significant decrease in plasma NfL levels compared with placebo, noted April 25, 2023.",5
SRNE,COVID-19,Phase 2,10/27/21,"Phase 2 trial completed. Phase 2 data from US trial noted a 20% improvement in the primary endpoint for patients in category 5, and discharge from the hospital was 2 days sooner than those in the Control Group. The Brazilian study reported a 25% improvement in primary endpoint, noted October 27, 2021.",6
NVS,Non-small cell lung cancer,Approved,5/6/20,"FDA Approval announced May 6, 2020.",6
ZYNE,Autism Spectrum Disorder (ASD),Phase 2,10/8/21,"Phase 2 long term data displayed a statistically significant improvement in ABC-C Subscale scores through 38 weeks. Only 7 patients (19%) experienced an AE that was deemed to be treatment related. Of the 10 treatment-related AEs reported, 7 were application site-related (application site reaction, pruritus, and dryness) and 1 each of sleep disorder, increased appetite, and pollakiuria, noted October 8, 2021.",6
JNJ,Psoriatic arthritis (PsA),Approved,10/20/17,"Approval announced October 20, 2017.",6
IMPL,Migraine,Approved,9/3/21,"Approved September 3, 2021.",6
IFRX,Pyoderma Gangraenosum (PG),Phase 2a,3/26/22,"Phase 2a final data reported a 69 days (mean) to target ulcer response (PGA ≤ 3) and 104 days (mean) to target ulcer closure, noted March 26, 2022.",5
PLUR,Critical limb ischemia (CLI),Phase 3,12/9/20,Phase 3 trial to be terminated following interim analysis that trial is unlikely to meet primary endpoint.,4
TAK,Congenital thrombotic thrombocytopenic purpura (cTTP),Phase 3,1/5/23,"Phase 3 data reported that treatment reduced the incidence of thrombocytopenia events by 60%, noted January 5, 2023.",6
CNTB,Atopic dermatitis (AD),Phase 2b,3/18/23,"Phase 2b data reported that baseline median Eczema Area and Severity Index (EASI) was 26.9. 54.5% of patients were considered severe, with a baseline Investigators Global Assessment (IGA) score of 4, noted March 18, 2023.",5
MRK,Microsatellite Instability-High Cancer,Approved,5/23/17,"PDUFA date March 8, 2017 extended to June 9, 2017 due to submission of extra data. Approved May 23, 2017.",6
ONTX,KRAS+ Non-Small Cell Lung Cancer,Phase 1/2,9/12/22,"Phase 1/2a data presented at ESMO showed an early signal of efficacy in an extensively pre-treated population with 1 complete response and 2 partial responses achieved in 14 evaluable patients, noted September 12, 2022.",6
AZN,Second line HER2+ breast cancer,Approved,5/5/22,"Approved May 5, 2022.",6
SAGE,Postpartum Depression (Severe),Phase 3,11/9/17,"Phase 3 data released November 9, 2017. Primary endpoint met.",6
LLY,COVID-19 Anti-body,Approved,12/3/21,"Emergency Use Authorization (EUA) expanded for children under 12 years old, noted December 3, 2021.",6
PFE,Sickle cell disease (SCD),Phase 2/3,11/2/23,"Phase 2/3 data from ASH abstract reported that safety data showed GBT021601 was well tolerated. For 27 patients with VOC at baseline, the baseline annualized VOC rate was 2.30, noted November 2, 2023.",6
MORF,Ulcerative Colitis (UC),Phase 1,10/25/22,"Phase 1 additional data showed statistically significant effects on key lymphocyte subsets, noted October 25, 2022.",6
PFE,Early stage breast cancer,Phase 3,5/29/20,"Phase 3 trial unlikely to show a statistically significant improvement in the primary endpoint following futility analysis - May 29, 2020.",4
MBRX,Acute Myeloid Leukia (AML),Phase 1b,10/2/23,"Phase 1b portion of Phase 1B/2 study demonstrated preliminary CR/CRi rate of 33%, noted Octobeer 2, 2023.",6
LQDA,Pulmonary arterial hypertension,Approved,11/8/21,"Tentative approval November 8, 2021. Final approval due in October 2022 or earlier upon resolution of on-going litigation.",6
VALN,Lyme disease vaccine for outdoor recreationists,Phase 3,2/17/23,"Phase 3 study discontinued about 50% of patients enrolled following violations of Good Clinical Practice (GCP) at certain clinical trial sites run by a third-party clinical trial site operator, noted February 17, 2023.",4
DCPH,Gastrointestinal Stromal Tumors (GIST) - second-line,Phase 3,6/1/23,"Phase 3 data reported that the mPFS (90% CI) by IRR was 6.44 (2.89, 8.31) months and ORR was 21.1%, noted June 1, 2023.",5
ABCL,COVID-19 Anti-body,Approved,12/3/21,"Emergency Use Authorization (EUA) expanded for children under 12 years old, noted December 3, 2021.",6
VRNA,Chronic obstructive pulmonary disease (COPD),Phase 2,10/13/21,"Phase 2 part B data noted when administered by hand-held pMDI over one week treatment provided clinically meaningful, statistically significant and dose-dependent bronchodilation, noted October 13, 2021.",6
PFE,Endometriosis-associated pain,Approved,8/5/22,"Approved August 5, 2022.",6
CYAD,Colorectal cancer,Phase 1,8/5/22,"Phase 1 preliminary data noted 2/15 partial responses from the dose-escalation segment. Median progression-free survival (mPFS) and median overall survival (mOS) was 3.9 months and 10.6 months, respectively.",5
AUPH,Dry eye syndrome,Phase 2/3,11/2/20,"Phase 2/3 trial did not meet primary endpoint - November 2, 2020. Development to be suspended.",4
ZLDPF,Obesity and type 2 diabetes,Phase 2,6/23/23,"Phase 2 data presented at ADA showed nearly 19% weight loss in people with overweight or obesity, noted June 23, 2023.",6
MESO,"Inflammatory bowel disease (IBD), Ulcerative colitis or Crohn’s colitis",Phase 1/2,2/20/22,"Phase 1/2 interim analysis reported that all ulcerative colitis patients had improved clinical and endoscopy scores within two weeks, and all Crohn’s colitis patients showed treatment remissions or responses by three months, noted February 20, 2022.",6
BHVN,Alzheimer’s disease,Phase 2/3,1/18/21,"Phase 2/3 top-line data did not meet co-primary endpoints - January 18, 2021.",4
BMRN,Phenylketonuria (PKU),Phase 1/2,9/6/21,"Phase 1/2 clinical hold announced September 6, 2021.",4
PFE,Transthyretin amyloid cardiomyopathy (ATTR-CM),Approved,5/6/19,"FDA approval announced May 6, 2019.",6
RHHBY,Solid tumors - NTRK gene fusion,Approved,8/15/19,"FDA approval announced August 15, 2019.",6
EGRX,Malignant hyperthermia - cancer,Approved,7/23/14,"Approved July 23, 2014 under priority review.",6
PFE,Cardiovascular (CV) risk reduction and hypertriglyceridemia,Phase 2b,11/24/21,"Phase 2b updated data demonstrated that in the dose-ranging study in subjects with elevated non-HDL-C and triglycerides (TG), the study met its primary endpoint, achieving a statistically significant reduction in non-HDL-C at all doses tested at 24 weeks, compared to placebo, noted November 24, 2021.",6
BNTX,COVID-19 vaccine,Phase 2/3,11/4/22,"Phase 2/3 clinical trial demonstrated robust neutralizing immune response one month after a 30-µg booster dose of the COVID-19 vaccine (Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5)). Omicron BA.4/BA.5-neutralizing antibody titers increased 13.2-fold from pre-booster levels in adults older than 55 years of age and 9.5-fold in adults 18 to 55 years of age, noted November 4, 2022.",6
RHHBY,Diffuse Large B-Cell Lymphoma (DLBCL),Phase 1/2,12/11/21,"Phase 1b/2 data reported an ORR of 73.0% and 51.5% complete response (CR) rate, with patients showing durable responses at ≥6 months, noted December 11, 2021.",6
ABBV,Crohn's disease,Phase 3,2/24/22,"Phase 3 trial met primary endpoint, noted February 24, 2022.",5
BMY,Relapsing Multiple Sclerosis,Phase 3,10/28/17,"Phase 3 data due released Feb 17, 2017 - primary endpoint met, disability endpoint not met. Full data released October 27, 2017 at ECTRIMS meeting.",5
SYRS,Myelodysplastic Syndrome,Phase 2,12/5/20,"Phase 2 presentation December 5, 2020 at ASH. Overall response rate (ORR) was 67% (12/18).",6
PFE,Pneumococcal conjugate vaccine (18 yrs +),Approved,6/8/21,https://www.businesswire.com/news/home/20210608006149/en/,5
TAK,Colorectal cancer,Phase 3,9/8/22,"Phase 3 data demonstrated that treatment reduced the risk of death by 34% in metastatic colorectal cancer , and Increased disease control with risk of disease progression or death reduced by 68%, noted September 8, 2022",6
INM,Epidermolysis Bullosa,Phase 2,6/22/23,"Phase 2 data from one patient were excluded from efficacy analyses due to a significant protocol deviation. Of the 18 participants assessed, chronic itch improved by a clinically meaningful amount in 12 patients (66.7%). However, the protocol-specified statistical analyses for non-wound itch were not statistically significant in favor of INM-755 due partly to the underlying control cream's clinically important anti-itch effect, noted June 22, 2023.",6
TERN,Nonalcoholic steatohepatitis (NASH),Phase 2a,8/8/23,"Phase 2a topline data reported that the trial met all primary and secondary endpoints, noted August 8, 2023.",5
PFE,Atopic dermatitis,Phase 3,8/30/21,Phase 3 trial met its co-primary and key secondary efficacy endpoints.,6
PFE,Renal cell carcinoma (RCC),Approved,11/16/17,"Approval announced 16 November, 2017.",6
JNJ,Non-small cell lung cancer,Phase 1/2,7/26/22,"Phase 1b/2 data reported an overall response rate (ORR) of 50%, with 15 out of 20 patients remaining on treatment, noted July 26, 2022.",6
CRDF,Small cell lung cancer (SCLC),Phase 2,9/26/23,"Phase 2 safety data from the first six patients by the institutional review board confirmed the trial can continue to enroll as planned, noted September 26, 2023.",5
MRNA,COVID-19,Phase 3,10/24/23,"Phase 3 trial of mRNA-1083 in adults 50 years initiated, noted October 24, 2023.",5
PFE,Lyme disease vaccine for outdoor recreationists,Phase 3,2/17/23,"Phase 3 study discontinued about 50% of patients enrolled following violations of Good Clinical Practice (GCP) at certain clinical trial sites run by a third-party clinical trial site operator, noted February 17, 2023.",4
BXRX,Acute pain following bunionectomy surgery,Approved,2/20/20,"FDA Approval announced February 20, 2020.",6
KALV,Hereditary angioedema (HAE),Phase 1,6/9/23,"Phase 1 data presented at EAACI showed that pharmacokinetic and pharmacodynamic data support globalization of the phase 3 clinical trial and use in short-term prophylaxis, noted June 9, 2023.",6
SLRX,Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML),Phase 1/2,12/12/22,"Phase 1/2 data presented at ASH reported an ORR of 50% among eight myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), noted December 12, 2022.",6
INCY,Solid Tumors,Phase 1/2,6/3/23,"Phase 1/2 data from ASCO reported that the best overall response in part 1 was stable disease, and in part 2 was a confirmed and durable partial response in 1 pt, noted June 3, 2023.",5
GOVX,Covid-19,Phase 2,9/11/23,"Phase 2 target enrollment met, noted September 11, 2023.",5
AZN,Hyperkalaemia,Approved,5/18/18,"Approval announced May 18, 2018.",6
OPK,Mild-to-moderate COVID-19,Phase 2,12/23/21,"Phase 2 top-line data reported that mean serum 25D levels increased with treatment and remained elevated for the duration of the trial, with 88% of subjects attaining the targeted level. Three symptoms related to respiratory function resolved more quickly when serum 25D was elevated at Days 7 and 14, with resolution of chest congestion occurring 3.4 days sooner, noted December 23, 2021.",6
CPRX,Congenital Myasthenic Syndromes (CMS),Phase 3,10/30/19,"Phase 3 trial did not meet primary endpoint - October 30, 2019.",4
VALN,Booster shot COVID-19 vaccine,Phase 1/2,1/19/22,"Additional data reported that all 30 samples (100%) presented neutralizing antibodies against the ancestral virus and Delta variant, and 26 samples (87%) presented neutralizing antibodies against the Omicron variant, noted January 19, 2022.",6
MESO,End-Stage Heart Failure with LVADs,Phase 2b,11/11/18,"Phase 2b trial did not meet primary endpoint - November 11, 2018.",4
RAIN,MDM2-amplified advanced solid tumors,Phase 2,5/30/23,"Phase 2 enrollment suspended, noted May 30, 2023.",4
MRK,Metastatic castration-resistant prostate cancer (mCRPC),Phase 3,8/3/22,"Phase 3 trial results were not statistically significant, noted August 3, 2022.",5
IOBT,Non-muscle invasive bladder cancer (NMIBC),Phase 1,7/28/23,"Phase 1 dosing initiated, noted July 28, 2023.",5
BIIB,Lupus,Phase 2,12/3/19,"Phase 2 data met primary endpoint - December 3, 2019.",6
IMAB,Growth hormone deficiency,Phase 3,8/17/23,"Phase 3 trial met its primary endpoint, noted August 17, 2023.",5
PFE,HIV,Approved,12/21/21,"Approved December 21, 2021.",6
PFE,Lyme disease,Phase 2,9/7/23,"Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.",6
AGIO,Sickle cell disease,Phase 2,6/26/23,"Phase 2 portion met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms, noted June 26, 2023",5
ENTX,Osteoporosis,Phase 2,10/14/23,"Phase 2 data presented at ASBMR showed that an increase in plasma ionized calcium should result in decreased secretion and plasma concentrations of endogenous PTH(1-84), noted October 14, 2023",6
ZYME,"3L+ HER2-positive, HR-positive breast cancer",Phase 2,12/9/22,"Phase 2 data reported a confirmed objective response rate (cORR) of 33%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 9.6 months, noted December 9, 2022.",5
ALKS,Recurrent or metastatic head and neck squamous cell carcinoma,Phase 2,11/13/21,"Phase 2 data showed that combination therapy was generally well tolerated (n=14), 8 had no prior response to dosing and 6 had previous best response of SD or PR. Mean (SD±) age was 62±12; AE were consistent with those observed with intravenous studies. Peripheral immune cell expansion profiles are comparable to that observed with the same regimen.",6
SRPT,Limb-girdle muscular dystrophy type 2E (LGMD2E),Phase 1/2,10/11/21,"Results from most recent study found that treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the NSAA compared to a matched natural history cohort 3 years after treatment- noted October 11, 2021.",6
GSK,"Covid-19 vaccine, omicron variant",Phase 1,1/30/23,"Phase 1 immunogenicity data reported that on day 29 at the 12µg dose level, CV0501 generated a ratio of post-boost to pre-boost serum neutralizing titers against BA.1 of 13.3, noted January 30, 2023.",5
HCM,Gastric cancer (GC) and gastroesophageal junction adenocarcinomas (GEJ),Phase 2,4/19/23,"Phase 2 data reported a confirmed ORR by IRC was 45%, and reached 50% in 16 patients with MET GCN (high) while only 1 PR was observed in 4 patients with MET GCN, noted April 19, 2023.",6
BCTX,Breast cancer,Phase 1/2,12/9/21,"Phase 1/2a data reported that the control rate was 25% and 30% for the monotherapy studies and 33% for the combination study, noted December 9, 2021.",5
UTHR,Pulmonary hypertension associated with interstitial lung disease (PH-ILD) (WHO Group 3),Approved,4/1/21,"FDA approval announced April 1, 2021.",6
GSK,HIV for adolescents 12 years and older,Approved,3/29/22,"Approved March 29, 2022.",6
GMDA,Severe aplastic anemia,Phase 2,12/6/20,Phase 2 data presented at ASH 2020 - sustained early engraftment shown.,6
EXEL,First-line BRAF wild-type metastatic or unresectable locally advanced melanoma,Phase 3,6/20/19,"Phase 3 data did not meet primary endpoint - June 20, 2019.",4
TXMD,Moderate-to-severe vaginal pain,Approved,5/30/18,"CRL announced May 8, 2017. NDA resubmitted with approval announced May 30, 2018.",6
MCRB,Mild-to-Moderate Ulcerative Colitis,Phase 2b,12/16/21,"Phase 2b microbiome analyses reported engraftment and anticipated changes in disease-relevant metabolites post-administration were not observed, noted December 16, 2021.",6
AUTL,Adult Acute lymphoblastic leukemia (ALL) / Chronic Lymphocytic Leukemia (CLL) / B-cell non-Hodgkin Lymphoma (B-NHL),Phase 1/2,12/12/22,"Longer term extension trial data reported that 35% of adult relapsed/refractory B-ALL patients treated with obe-cel in sustained complete remissions between 24 and 47 months without any need for additional anti-leukemia therapy, noted December 12, 2022.",6
MRNA,Bivalent booster COVID-19 vaccine,Phase 2/3,6/22/22,"Phase 2/3 trial data reported that neutralizing titers against BA.4/BA.5 were approximately 3-fold lower than previously reported neutralizing titers against BA.1. One month after an mRNA-1273.214 booster, neutralizing geometric mean titers (GMT) against BA.4/BA.5 were 941 (95% CI: 826, 1071) in all participants, and 727 (95% CI: 633, 836) in seronegative participants, noted June 22, 2022.",5
RVNC,Forehead lines,Phase 2,6/30/20,"Phase 2 data noted 100% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group - June 30, 2020.",6
PFE,Tick-borne encephalitis (TBE) vaccine,Approved,8/13/21,"FDA Approval announced August 13, 2021",6
TSHA,Rett syndrome,Phase 1/2,8/24/23,"FDA granted Fast Track Designation (FTD), noted August 24, 2023.",5
BIIB,Alport Syndrome,CRL,2/25/22,"CRL issued February 25, 2022.",5
BPMC,Fourth-line GIST,CRL,5/15/20,"CRL issued May 15, 2020.",5
BDRX,Children with Newly Diagnosed Diffuse Midline Gliomas (DMGs),Phase 1,7/10/23,"Phase 1 enrollment completed, noted July 10, 2023.",5
BMY,First-line Hepatocellular carcinoma (HCC) - cancer,Phase 3,6/24/19,Phase 3 trial did not meet primary endpoint.,4
RGLS,Alport Syndrome,Phase 2,7/15/22,"Phase 2 trial terminated after failing to meet pre-defined futility criteria, noted July 15, 2022.",6
RHHBY,Hemophilia,Approved,10/4/18,"FDA Approval announced October 4, 2018.",6
INVA,Hereditary Hemochromatosis,Phase 2,6/6/19,"Phase 2 data met primary endpoint - June 6, 2019.",4
BMY,Ovarian Cancer - First-line maintenance treatment,Phase 3,6/6/22,"Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.",6
ACIU,Autosomal Dominant Alzheimer's Disease (ADAD,Phase 3,8/2/22,"Additional Phase 3 data reported that cognitive test scores of API ADAD composite 22.9%, FCSRT 19.9% and RBANS total score 43.8%, noted August 2, 2022.",5
NVS,Breast cancer,Phase 3,9/9/22,"Phase 3 overall survival data reported a median OS of approximately five years, noted September 9, 2022.",5
CLDX,Chronic spontaneous urticaria (CSU),Phase 2,7/11/23,"Phase 2 data ahead of schedule, enrollment completed, noted July 11, 2023.",5
REPL,Liver and kidney transplant recipients with recurrent Cutaneous squamous cell carcinoma,Phase 1/2,3/30/22,"Phase 1b/2 data reported that two of six patients (33%) achieved a response after treatment, with one complete response and one partial response, noted March 30, 2022.",6
BDRX,Recurrent Glioblastoma (GBM),Phase 1,10/3/23,"Phase 1 Cohort A enrollment completed, noted October 3, 2023.",5
PBYI,Small Cell Lung Cancer (SCLC),Phase 1a,8/8/23,"FDA IND Clearance on August 8, 2023.",5
MGNX,Gastric cancer,Phase 2/3,9/16/21,"Phase 2/3 results had 4 patients with confirmed complete responses and 17 confirmed partial responses, September 16, 2021.",5
PPBT,2L metastatic pancreatic cancer (PDAC),Phase 1/2,10/6/23,"Phase 1 biomarker data demonstrated that CM24 treatment significantly reduced the level of NET marker in patients' serum, noted October 6, 2023.",6
CNTX,Ovarian cancer,Phase 2,6/6/22,"Phase 2 data presented at ASCO reported that a 12-month PFS rate of 20.1% and a CBR of 35.7% in patients with GCT. No objective responses were observed, noted June 6, 2022.",6
NBIX,Tourette syndrome - adults,Phase 2,1/18/17,"Phase 2 data released January 18, 2017 did not meet primary endpoint.",4
INCY,Atopic dermatitis,Approved,9/21/21,"Approved September 21, 2021.",6
AGIO,Glioma,Phase 1,6/1/18,"Phase 1 data presented at ASCO June 1, 2018. 1 minor response + 1 partial response out of 93 patients.",5
GSK,Mild to moderate COVID-19,Phase 3,3/3/21,Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.,4
LLY,Cluster headache,Approved,6/4/19,"FDA Approval announced June 4, 2019.",6
JNJ,COVID-19 vaccine - two-dose regimen,Approved,10/20/21,"EUA for second dose for adults aged 18 and older at least two months following primary vaccination with single-shot Johnson & Johnson COVID-19 vaccine; and for eligible individuals who received a different authorized or approved COVID-19 vaccine, noted October 20, 2021.",6
FNCH,Clostridioides difficile infection,Phase 2,6/1/22,"Phase 2 data presented at DDW reported that 88.2% of participants were without CDI recurrence through 8 weeks in this post-hoc analysis, noted June 1, 2022.",6
ATAI,Treatment Resistant Depression,Phase 1,10/2/23,"Phase 1 completed, noted October 2, 2023.",5
PMVP,Solid tumors,Phase 1/2,10/12/23,"Phase 1/2 updated results presented at AACR-NCI-EORTC reported that the overall response rate (ORR) was 38% (6/16 evaluable patients) at the Recommended Phase 2 Dose (RP2D) of 2000 mg daily and 34% (13/38) in the efficacious dose range, noted October 12, 2023.",6
MOR,Diffuse Large B-Cell Lymphoma (DLBCL),Phase 1b,12/12/22,"Phase 1b data reported that ORR at EoT visit and best response across all visits were higher in Arm T/L, as were 18-month DoR, PFS, and OS rates, noted December 12, 2022.",5
BMY,Stage IIB or IIC melanoma,Approved,10/16/23,"Approved October 13, 2023.",6
VRTX,Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations,Approved,3/17/15,"Approved March 17, 2015.",6
ETON,Nutritional requirements for patients with liver disease,Approved,4/11/22,"Approved April 11, 2022.",6
VIR,Influenza,Phase 2,7/20/23,"Phase 2 trial did not meet its primary endpoint, noted July 20, 2023.",6
RCKT,Danon disease,Phase 1,5/18/23,"Phase 1 additional results reported that efficacy results continue to demonstrate sustained improvement or stabilization in all patients with preserved left ventricular systolic function at time of treatment, noted May 18, 2023.",6
LPTX,Endometrial Cancer,Phase 2,3/22/21,"Phase 2 data presented at Society of Gynecologic Oncology 2021 Annual Meeting, March 19-25, 2021. PFS 5.4 months.",5
GOSS,Pulmonary arterial hypertension (PAH),Phase 2,7/25/23,"Phase 2 OLE data reported that 9 of 11 patients continued to improve, with a mean improvement of 9 PVR dual responders of 39%, and 3 patients reached a PVR below 200 dyne*s/cm5n, noted July 25, 2023.",6
BMY,Newly Diagnosed Multiple Myeloma,Phase 2a,12/10/22,"Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.",6
ORMP,COVID-19 vaccine,Phase 1,10/7/22,"Phase 1 preliminary results reported a significant antibody response (2-6 fold over baseline) and no safety issues observed, noted October 7, 2022.",6
RGLS,Autosomal Dominant Polycystic Kidney Disease (ADPKD),Phase 1,9/20/23,"Phase 1b data showed that it was well tolerated by all 9 subjects receiving active drug with no safety findings. Increases in both PC1 and PC2 biomarkers were observed. Statistically significant increases in mean PC1 levels were observed at Day 85 and Day 86 (n=9) compared to baseline (36%-41%). Numeric increases in PC2 were observed during the treatment period, although not statistically significant, noted September 20, 2023.",6
PBYI,Metastatic triple negative breast cancer with a HER2 mutation,Phase 2,10/27/22,"Phase 2 basket trial interim efficacy results showed that the objective response rate (ORR) was 35% overall, 30% in patients pretreated with TKIs, and 50% in patients not pretreated with TKIs. Response or stable disease lasting for ≥ 48 weeks was observed in 7 patients (6 PR, 1 SD), noted October 27, 2022.",6
ZYNE,Fragile X syndrome,Phase 2/3,5/13/22,"Phase 2/3 long-term data reported that sustained improvement in ABC-CFXS Social Avoidance from baseline, noted May 13, 2022.",6
RGNX,Duchenne Muscular Dystrophy (DMD),Phase 1/2,10/3/23,"Phase 1/2 additional data shared at the World Muscle Society Congress reported that in the patient aged 4.4 years old, RGX-202 microdystrophin expression was measured to be 38.8% compared to control, and in the patient aged 10.6 years old, RGX-202 microdystrophin expression was measured to be 11.1% compared to control, noted October 3, 2023.",5
SAGE,Postpartum Depression - moderate,Approved,3/19/19,"FDA approval announced March 19, 2018.",6
MRK,HIV,Approved,9/20/19,"FDA Approval for sNDA announced September 20, 2019.",6
MRK,HIV-1,Phase 2b,10/27/21,"Phase 2b data through 144 weeks demonstrated that dosing continued to maintain viral suppression, as measured by the number of study participants achieving HIV-1 RNA levels <50 copies/mL, similar to DOR/3TC/TDF, noted October 27, 2021.",6
GSK,Chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype,CRL,9/7/18,"CRL issued September 7, 2018.",5
BMY,Plaque psoriasis,Phase 3,11/3/20,"Phase 3 trial met both co-primary endpoints - November 3, 2020.",6
ARWR,Cystic Fibrosis,Phase 1/2,7/2/21,"Phase 1/2 trial paused following a trial in rats that contained unexpected signals of local lung inflammation - July 2, 2021.",4
KPTI,Quadruple Refractory Multiple Myeloma,Approved,7/3/19,"FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).",6
EXEL,BRAF V600 Mutation-Positive Advanced Melanoma - Cancer,Approved,11/10/15,"Approved November 10, 2015.",6
ROIV,Plaque psoriasis,Approved,5/24/22,"Approved May 24, 2022.",6
PFE,Atopic Dermatitis,Approved,1/14/22,"Approved January 14, 2022.",6
GERN,Myelodysplastic syndromes,Phase 3,6/2/23,"Phase 3 data presented at ASCO showed that its primary endpoint of 8-week transfusion independence (TI) continues significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders, noted June 2, 2023",6
REGN,Osteoarthritis pain of the hip or knee,Phase 3,8/5/20,"Phase 3 data achieved the co-primary endpoints for fasinumab 1 mg monthly. Fasinumab every two months did not reach statistical significance - August 5, 2020.",5
SGEN,Diffuse large B-cell lymphoma (DLBCL),Approved,6/10/19,"FDA approval announced June 10, 2019.",6
SNY,Asthma - 6-11 year-olds,Approved,10/20/21,"Approved October 20, 2021.",6
MOR,Myelofibrosis,Phase 2,6/10/22,"Phase 2 efficacy and safety data reported that over two-thirds of JAK inhibitor-naïve patients treated with the combination achieved at least a 35% reduction in spleen volume (SVR35) from baseline at week 24. Notably, 80% of patients achieved SVR35 at any time on study, noted June 10, 2022.",6
BMY,Triple-negative breast cancer,Approved,3/8/19,"FDA Approval announced March 8, 2019.",6
ALDX,Dry eye disease,Phase 3,12/20/21,"Phase 3 trial did not meet the primary endpoint, secondary endpoint was achieved, December 20, 2021.",6
PRTA,Alzheimer’s disease,Phase 1,7/17/23,"Phase 1 SAD data presented at AAIC met key pharmacokinetic (PK) and immunogenicity secondary endpoints, noted July 17, 2023",6
ADVM,Diabetic macular edema (DME),Phase 2,10/9/21,"Phase 2 data from trial (June 22, 2021, cutoff date, n=34) at ASRS demonstrated that dose and disease state appear to have an impact in determining the therapeutic window which balances efficacy and safety. Dose-dependent inflammation observed may be in part related to the study population which included patients with severe vascular and renal disease. Further drug development will focus on wet AMD and low doses (2 x 10^11 vg/eye and lower) - noted October 9, 2021",5
VTGN,Premenstrual dysphoric disorder (PMDD),Phase 2a,9/12/23,"Phase 2a exploratory analysis demonstrated statistically and clinically significant improvement versus placebo, noted Septemeber 12, 2023.",6
PYPD,Abdominal (soft tissue) sternal surgical site infections,Phase 3,9/2/22,"Phase 3 trial did not meet primary endpoint, noted September 2, 2022.",6
ZLAB,Non-Hodgkin Lymphoma (NHL),Phase 2,12/12/22,"Phase 2 data presented at ASH showed a 49% ORR in heavily pre-treated patients who were naïve to prior CAR-T, with 31% achieving a complete response (CR), noted December 12, 2022.",6
RHHBY,BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma,Approved,7/30/20,"FDA approval announced July 30, 2020.",6
PFE,"Meningococcal Vaccine, Adolescents",Approved,10/20/23,"FDA approved on October 20, 2023.",6
TAK,Type 3 von Willebrand Disease (VWD),Approved,1/31/22,"Approved January 31, 2022.",6
AXSM,Migraine,CRL,5/2/22,"CRL announced May 2, 2022.",5
MRK,Triple negative breast cancer (TNBC),Approved,7/27/21,"FDA approval announced July 27, 2021.",6
VTRS,Dry eye disease,Approved,10/18/21,"Approved October 18, 2021.",6
MIRM,Cerebrotendinous xanthomatosis (CTX),Phase 3,10/2/23,"Phase 3 data met the primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant, noted October 2, 2023.",6
BMY,Large B-cell lymphoma (LBCL),Approved,6/24/22,"Approved June 24, 2022.",6
AZN,Chronic Kidney Disease (CKD),Phase 2b,11/3/23,"Phase 2b showed statistically significant and clinically meaningful reductions in urinary albumin-to-creatinine ratio (UACR), noted November 3, 2023.",6
MRTX,Non-Small Cell Lung Cancer (NSCLC),Phase 2,10/20/23,"Phase 2 data presented at ESMO 2023 reported that in patients with PD-L1 TPS ≥50%, adagrasib + pembrolizumab demonstrated a higher response rate (63%) than expected with pembrolizumab monotherapy (39–45%3,4), noted October 20, 2023.",6
REGN,Chronic obstructive pulmonary disease (COPD),Phase 3,3/23/23,"Phase 3 demonstrated clinically meaningful and highly significant reduction (30%) in moderate or severe acute exacerbations of COPD over 52 weeks, while also demonstrating significant improvements in lung function, quality of life and COPD respiratory symptoms, noted March 23, 2023. Data expected in 2025.",6
REGN,Cutaneous squamous cell carcinoma,Approved,9/28/18,"FDA Approval announced September 28, 2018.",6
MRK,Merkel Cell Carcinoma,Approved,12/19/18,"FDA Approval announced December 19, 2018.",6
ABCL,COVID-19 Antibody,Approved,11/9/20,"Emergency Use Authorization (EUA) announced November 9, 2020.",6
NVS,"Pre-symptomatic patients with spinal muscular atrophy (SMA) Types 1, 2 and 3",Phase 3,5/5/19,"Phase 3 data May 5, 2019 noted all patients (18/18) were alive and event-free",6
BMY,Melanoma,Phase 3,3/14/22,"Phase 3 data did not meet the primary endpoints, noted March 14, 2022.",6
BLCM,Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer,Phase 1/2,7/8/19,"Phase 2 pediatric top-line data met primary endpoint of event free survival - July 8, 2019.",6
PRLD,Solid Tumors and Recurrent High-Grade Gliomas,Phase 1,10/7/21,"Phase 1 data displayed a GBM patient who experienced a PR that later evolved into a durable CR for more than 13 months. Patients were largely able to remain on therapy with few AE-related dose interruptions (13%), reductions (4%), or discontinuations (3%), noted October 7, 2021.",5
LGND,ER+/HER2- breast cancer,Phase 3,10/2/23,"Phase 3 result presented at NAMS 2023 showed that younger age, non-visceral disease, prior tamoxifen and a longer total duration of AI use may be associated with higher baseline vaginal and/or vulvar symptomatology, noted October. 2, 2023.",6
AMGN,Moderate to severe genital psoriasis,Phase 3,10/31/23,"Phase 3 data presented that treated patients across subgroups experienced greater improvement in genital psoriasis and genital itch at week 16 compared to placebo, noted October 31, 2023.",6
PFE,Complicated Intra-abdominal Infection and Hosptial Acquired Pneumonia,Phase 3,6/1/23,"Phase 3 data reported that the cure rate in the intention to treat (ITT) analysis set was 76.4% for the ATM-AVI ± MTZ treatment arm vs 74.0% for the MER ± COL treatment arm, with a treatment difference of 2.4%, noted June 1, 2023.",6
QNRX,Netherton Syndrome (NS),Phase 1,10/24/23,"Phase 1 data from 6 subjects demonstrated well defined efficacy signals across a number of study endpoints, noted October 24, 2023.",6
RARE,Ornithine Transcarbamylase (OTC) Deficiency,Phase 1/2,5/19/22,"Phase 1/2 trial safety and efficacy data reported that in the prophylactic steroid cohort, one of two patients demonstrated a complete response, noted May 19, 2022.",6
MGNX,Metastatic breast cancer,Approved,9/7/21,"Final overall survival data did not demonstrate MARGENZA plus chemotherapy had a statistically significant advantage compared to trastuzumab plus chemotherapy; September 7, 2021.",4
GILD,Multiple Myeloma,Phase 2,6/19/23,"Phase 2 clinical hold received by the FDA after a recent patient death, noted June 19, 2023.",5
ACAD,Adjunctive treatment of schizophrenia,Phase 3,7/22/19,"Phase 3 data did not meet primary endpoint - July 22, 2019.",4
VRTX,Cystic fibrosis - Two Copies of the F508del Mutation,Approved,6/21/19,"Label expanded to now include all patients over 6 years old - June 21, 2019.",6
VRNA,Maintenance treatment of COPD,Phase 2,8/5/19,"Phase 2 data met primary endpoint - August 5, 2019.",6
RUBY,Refractory Acute Myeloid Leukemia (AML) / Solid tumors,Phase 1/2,3/15/21,"Phase 1/2 data announced March 15, 2021. 1 partial response (PR), 1 unconfirmed PR.",5
IMRA,b-thalassemia,Phase 2b,11/16/21,Phase 2b interim data showed an uptrend observed in transfusion-dependent subjects treated with higher dose for reduced transfusion burden. The dose was generally well-tolerated in this patient population.,6
APVO,Relapsed or refractory peripheral T-cell lymphoma,Phase 2,11/14/18,"Development terminated - noted November 14, 2018.",4
BIIB,X-linked Retinitis Pigmentosa,Phase 2/3,10/20/21,"Phase 2/3 trial discontinued noted October 20, 2021.",4
AMGN,Chronic spontaneous urticaria,Phase 2b,10/31/23,"Phase 2b trial did not meet its primary endpoint, noted October 31, 2023.",6
DCTH,Liver-dominant ocular melanoma,Approved,8/14/23,"FDA Approved on August 14, 2023.",6
MRK,Hypercholesterolemia,Phase 2b,3/6/23,"Phase 2b trial data presented at ACC showed that study was generally well tolerated and reduced LDL-C across all dose levels compared to placebo, noted March 6, 2023.",6
TBPH,Neurogenic Orthostatic Hypotension (nOH),Phase 3,9/15/21,"Phase 3 study did not meet its primary endpoint September 15, 2021.",4
TAK,Primary immunodeficiency (PI) in children 2-16 years old,Approved,4/11/23,"Approved April 11, 2023.",6
RGNX,Neovascular Age-related macular degeneration (AMD),Phase 2,2/11/23,"Phase 2 PK data reported that treatment was well tolerated with five SAEs reported and protein concentrations in the eye were similar between the manufacturing processes, noted February 11, 2023.",6
ANIK,Osteoarthritis of the knee,Phase 3,11/1/22,"Third Phase 3 trial met primary endpoint, noted November 1, 2022.",5
BMY,Relapsing Multiple Sclerosis,Approved,3/25/20,"FDA Approval announced March 26, 2020.",6
MRK,Small cell lung cancer (SCLC),CRL,3/1/21,"FDA Approval announced June 18, 2019. Approval withdrawn March 1, 2021 due to not meeting post-marketing requirements following accelerated approval.",4
RHHBY,Neovascular age related macular degeneration (nAMD),Phase 3,1/25/21,"Phase 3 trial met primary endpoint - January 25, 2021.",6
ZYNE,Fragile X Syndrome in Children and Adolescents,Phase 3,7/18/22,"Longterm data reported improvements in ABC-CFXS Social Avoidance in the full population, noted July 18, 2022.",6
GILD,Nonalcoholic steatohepatitis (NASH),Phase 3,2/11/19,"Phase 3 trial did not meet primary endpoint - February 11, 2019.",4
APLS,Geographic atrophy (GA),Approved,2/17/23,"FDA Approval on February 17, 2023.",6
INCY,Polycythemia Vera,Approved,12/4/14,"Approved December 4, 2014.",6
ABBV,Crohn's disease (induction/maintenance),Approved,5/18/23,"Approved May 18, 2023.",6
MOR,Diffuse Large B-Cell Lymphoma,Phase 3,12/11/21,"Phase 3 data reported that trial met primary endpoint, with overall survival (OS) observed for tafasitamab plus lenalidomide at 20.1 months. The objective response rate (ORR) was 63.6% with a CR at 39.4%, noted December 11, 2021.",6
IGXT,Migraine,CRL,3/27/20,"CRL announced March 27, 2020.",5
AZN,Relapsed/refractory chronic lymphocytic leukaemia,Phase 3,12/12/21,"Phase 3 updated results reported a reduced risk of disease progression or death by 71% versus IdR/BR as assessed by investigators at three years, noted December 12, 2021.",6
HRMY,Adult cataplexy,Approved,10/14/20,"FDA approval announced October 14, 2020.",6
RHHBY,Urothelial carcinoma,Phase 3,9/30/19,Phase 3 data at ESMO 2019 noted median PFS=8.2 versus 6.3 months; hazard ratio (HR)=0.82.,5
URGN,Non-Muscle-Invasive Bladder Cancer,Phase 2b,5/13/22,"Phase 2b results reported that by 3 months, 31/44 (70%) LG NMIBC patients achieved a complete response, noted May 13, 2022.",6
PTCT,Friedreich ataxia,Phase 3,5/23/23,"Phase 3 trial did not meet its primary endpoint, noted May 23, 2023.",6
LIAN,Symptomatic Obstructive Hypertrophic Cardiomyopathy (HCM),Approved,6/16/23,"FDA Approval on June 16, 2023",6
PCVX,Pneumococcal conjugate vaccine,Phase 2,4/17/23,"Phase 2 data reported an overall improvement in immune responses vs. PCV20 relative to the results from the prior Phase 2 study in adults aged 50-64, and the trial met the OPA response non-inferiority criteria(1) for 18 of 20 STs common with PCV20 and met the superiority criteria, noted April 17, 2023.",6
SNY,Prurigo nodularis (PN),Approved,9/28/22,"Approved September 28, 2022.",6
ANIP,Urinary tract infections,Approved,4/24/23,"Approved April 24, 2023.",6
ENDP,Adhesive capsulitis (AC) of the shoulder,Phase 2,7/1/22,"Phase 2 trial did not report a significant difference between treatment and placebo, noted July 1, 2022.",6
TPST,Solid Tumors,Phase 1,5/25/23,"Phase 1 monotherapy and combination data from abstract reported a disease control rate (DCR) by RECIST v1.1 was 44% for patients on monotherapy TPST-1495 and 40.9% for patients on TPST-1495 with pembrolizumab, noted May 25, 2023.",5
ALNY,Hypercholesterolemia,Approved,12/22/21,"Approved December 22, 2021.",6
INVA,Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter),Approved,5/24/23,"Approved May 24, 2023.",6
CHRS,Cancer,CRL,9/25/23,"CRL issued September 25, 2023.",5
VTGN,Adjustment Disorder with Anxiety (AjDA),Phase 2a,3/22/23,"Phase 2a top-line data demonstrated clinically meaningful reductions in fear, anxiety and avoidance of anxiety-provoking social and performance situations in daily life, as measured by the Liebowitz Social Anxiety Scale (LSAS)",6
ALDX,Dry eye disease,Phase 3,6/8/22,"Phase 3 topline results reported that trial met both primary endpoints, noted June 8, 2022.",5
BYSI,Chemotherapy-induced neutropenia (CIN),CRL,12/1/21,"CRL announced December 1, 2021.",5
AVRO,Cystinosis,Phase 1/2,5/18/23,"Phase 1/2 data presented at ASGCT reported sustained engraftment and durable reduction in leukocyte cystine levels across all patients, noted May 18, 2023.",6
QURE,Huntington's disease,Phase 1/2,6/21/23,"Phase 1/2 early clinical data demonstrate trends consistent with a potential clinical benefit of AMT-130 at both doses, noted June 21, 2023.",6
LLY,Atopic dermatitis,PDUFA,7/16/21,"Noted FDA will not meet assigned July 2021 PDUFA date - July 16, 2021",4
PFE,Cancer pain,Phase 3,7/28/21,Phase 3 trial met primary endpoint - July 2021.,6
ENLV,COVID-19,Phase 2,2/3/21,"Phase 2 data released February 3, 2021. 14/16 (87.5%) patients recovered and were discharged from the hospital by day-28.",6
RARE,Osteogenesis Imperfecta,Phase 2b,10/16/23,"Phase 2b data demonstrated that treatment with setrusumab (UX143) significantly reduced the incidence of fractures in patients with osteogenesis imperfecta (Gr) with at least six months of follow-up and continues to demonstrate ongoing and meaningful improvements in lumbar spine bone mineral density (BMD), noted October 16, 2023.",6
GRTS,Colorectal Cancer / Gastric Cancer,Phase 1/2,9/17/21,"Phase 1/2 data showed a 44% molecular response rate (4/9) and extended median overall survival >17 months, September 17, 2021.",6
APTX,Neuropathic Pain Associated with Diabetic Peripheral Neuropathy (DPN),Phase 2,4/7/22,"Phase 2b trial did not achieve statistically significant separation from placebo on the study's primary endpoint, noted April 7, 2022.",6
ALNY,Hypertension,Phase 2,9/7/23,"Phase 2 study met the primary endpoint, noted September 7, 2023.",5
ZLDPF,Short bowel syndrome (SBS),Phase 3,9/30/22,"Phase 3 trial results reported that trial met its primary endpoint, noted September 30, 2022.",5
NVS,Inoperable progressive midgut NETs,Approved,1/26/18,"Approved January 26, 2018. Acquired from Advanced Accelerator Applications (NASDAQ: AAAP).",6
OBSV,Endometriosis,Phase 3,5/25/22,"Phase 3 data presented at SEUD reported that at 24 weeks the mean percent reduction from baseline in MBL in the non-responder groups were 72%, 76%, 75% and 74%, across all doses, noted May 25, 2022.",6
WVE,Huntington’s disease,Phase 1/2,3/29/21,https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-provides-update-phase-1b2a-precision-hd,5
IVVD,COVID-19 (prevention),Phase 2/3,3/23/23,"Phase 2/3 study suggests clinically meaningful protection can be achieved at low neutralizing antibody titers, noted March 23, 2023.",6
RARE,Tumor-induced osteomalacia (TIO),Approved,6/18/20,"FDA Approval announced June 18, 2020.",6
NVS,Non-small cell lung cancer (NSCLC),Phase 3,10/25/21,"Phase 3 trial did not meet primary endpoints, noted October 25, 2021.",4
SNY,Eosinophilic esophagitis (EoE),Approved,5/20/22,"Approved May 20, 2022.",6
CLVS,Castrate-resistant prostate cancer (mCRPC),Approved,5/15/20,"FDA Approval announced May 15, 2020.",6
RXDX,Ulcerative colitis,Phase 2,3/1/23,"Phase 2 trial met all ranked secondary endpoints, noted March 1, 2023.",5
MYOV,Contraception,Phase 3,10/26/21,"Phase 3 trial resumed following clinical hold August 2021 after study protocol amendments, noted October 26, 2021.",5
IMRX,Solid tumors,Phase 1,4/18/23,"Phase 1 data presented at AARC showed that dosing was well tolerated with no dose limiting toxicities (DLTs) or serious adverse events (SAEs) observed, noted April 18, 2023.",6
NVCR,Liver cancer,Phase 2,7/1/21,"Phase 2 final data reported July 1, 2021. Objective response rate for the intent-to-treat population was 9.5%.",4
GSK,Head and Neck Squamous Cell Carcinoma,Phase 2,4/14/21,"Phase 2 trial to be discontinued - April 14, 2021.",4
INKT,Solid Tumors,Phase 1,11/3/23,"Phase 1 trial of agenT-797 alone or in combination showed durable clinical benefit, including: Clinical response in MSI-high 3L gastric cancer patient after failure on pembrolizumab and nivolumab/FOLFOX. Long-term disease stabilization (n=10), including in testicular cancer (SD 9 months) and anti-PD-1 relapsed/refractory non-small-cell lung cancer (SD > 10 months). Tolerable safety profile with no dose-limiting toxicities and no grade >3 neurotoxicity or cytokine release syndrome, noted November 3, 2023.",6
GLPG,Psoriatic arthritis,Phase 2,5/30/18,"Phase 2 data released May 30, 2018 - primary endpoint met.",6
ALT,COVID-19,Phase 1/2,6/29/21,"Phase 1/2 enrollment to be terminated - June 29, 2021.",4
TAK,Dengue Vaccine,Phase 3,6/9/22,"Phase 3 data reported that treatment prevented 84% of hospitalizations and 61% of symptomatic dengue illness overall, with identified safety risks, noted June 9, 2022.",6
PFE,Biosimilar bevacizumab,Approved,6/28/19,"FDA Approval announced June 28, 2019.",6
SPPI,Non-Hodgkin’s lymphoma,Approved,9/4/09,"Approved September 4, 2009.",6
NBTX,Head and Neck Cancer,Phase 1,10/4/23,"Phase 1 final data presented at ASTRO reported an overall response rate (ORR) of 81.8% (36/44) with a complete response rate (CRR) of 63.6% (28/44), noted October 4, 2023.",6
MRK,Esophageal Cancer,Approved,7/31/19,"FDA Approval announced July 31, 2019.",6
TAK,Ulcerative Colitis,Approved,9/27/23,"FDA Approved on September 27, 2023.",6
PHRRF,Levodopa-induced dyskinesia in Parkinson's disease,Phase 1/2,9/15/22,"Phase 1/2 data reported a 51% reduction of UDysRS at infusion 2, and 41% reduction by 3 months post infusion. Treatment data demonstrated that 100% of subjects treated with ketamine had a reduction in dyskinesias, noted September 16, 2022.",6
MDWD,Basal Cell Carcinoma (BCC),Phase 1/2,7/10/23,"Phase 1/2 data reported that treatment was safe and well-tolerated with eleven out of fifteen patients achieved complete clearance of their BCCs, noted July 10, 2023.",6
MRK,Hepatocellular Carcinoma (HCC),Phase 3,1/18/22,"Phase 3 data reported a reduced risk of death by 21% and that the median OS was 14.6 months, noted January 18, 2022.",6
REGN,Lymphoma,Phase 1/2,5/26/22,"Phase 1/2 data released in an abstract at ASCO reported no significant adverse effects (AEs) and clinical activity in 5 of 7 patients, noted May 26, 2022.",5
JNJ,Amyloidosis,Approved,1/15/21,"FDA approval announced January 15, 2021.",6
PFE,Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine,Approved,12/8/22,"EUA granted for children under 5 December 8, 2022.",5
CNCE,Alopecia Areata,Phase 3,8/1/22,"Phase 3 topline data reported that trial met primary endpoint and key secondary endpoint, noted August 1, 2022.",5
CANF,"Advanced liver cancer, hepatocellular carcinoma",Phase 2,12/20/21,"Phase 2 data reported a complete response with that patient surviving 5 years, noted December 20, 2021.",5
OBSV,Uterine fibroids,Phase 3,10/20/21,"Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.",5
IMMP,Non-small cell lung cancer; Head and neck cancer,Phase 2,11/3/23,"Additional biomarker results shared at SITC showed statistically significant increases of Th1 biomarkers (IFN-gamma, CXCL-10), circulating immune cells (lymphocytes), and RNA levels of immune activating genes were observed and linked to improved clinical outcomes, noted November 3, 2023.",6
ANAB,Hidradenitis Suppurativa,Phase 2,8/31/22,"Phase 2 trial did not meet its primary endpoint, noted August 31, 2022.",6
EPIX,Castrate resistant prostate cancer,Phase 1,10/31/22,"Phase 1 trial enrollment suspending October 31, 2022",4
TARS,Demodex Blepharitis,Phase 2/3,6/21/21,"Phase 2b/3 top-line data released June 21, 2021. Endpoints met.",6
AMGN,REMICADE biosimilar - rheumatoid arthritis,Approved,12/6/19,"FDA Approval announced December 6, 2019.",6
JSPR,Fanconi Anemia (FA),Phase 1b,9/21/23,"Phase 1b updated results showed that all three patients treated achieved full donor engraftment and full blood count recovery. Briquilimab was well tolerated without any complications, noted September 21, 2023.",6
REGN,Atopic dermatitis,Approved,3/28/17,"Approved under priority review - March 28, 2017.",6
MRK,Myelofibrosis,Phase 2,12/9/19,Phase 2 data at ASH December 2019 noted primary endpoint met in 10%~32% of patients across four cohorts.,4
PFE,Recurrent renal cell carcinoma (RCC),Phase 3,4/10/18,Phase 3 trial discontinued due to futility.,4
RXDX,Crohn’s Disease,Phase 2a,3/3/23,"Phase 2a primary and secondary results reported efficacy in biologic-experienced subjects consistent with overall population and consistent across subgroups and irrespective of presence of ADA, noted March 3, 2023.",5
CNCE,Agitation in patients with Alzheimer's disease,Phase 3,9/27/19,"Phase 3 trial did not meet primary endpoint - September 27, 2019.",4
VBLT,Recurrent platinum resistant ovarian cancer,Phase 3,7/19/22,"Phase 3 trial did not meet the primary endpoint, noted July 19, 2022.",6
KALV,Hereditary Angioedema (HAE),Phase 1,10/31/22,"Phase 1 data from the study showed that the ODT tablet formulation has a similar PK profile to the film-coated version currently in development, noted October 31, 2022.",6
IMUX,Progressive Multiple Sclerosis,Phase 2,10/9/23,"Phase 2 24-Week data from first half of patients shows improvements in biomarker NfL, Consistent throughout the overall population as well as all subtypes, noted October 9, 2023.",6
STOK,Dravet syndrome,Phase 1/2,7/25/23,"Additional Phase 1/2s safety data showed that it has been generally well-tolerated among 74 patients treated with single and multiple doses of 10mg to 70mg, noted July 25, 2023.",6
IONS,ATTR cardiomyopathy,Phase 3,7/31/23,"Phase 3 trial initiation announced January 10, 2020. Phase 3 enrollment completed, noted July 31, 2023.",5
TSHA,Giant axonal neuropathy (GAN),Phase 1/2,1/31/22,"Phase 1/2 data demonstrated that dosing was safe and well-tolerated supported by 53 patient-years of clinical data, efficacy data for high dose cohort showed clinically and statistically significant improvement by Year 1 compared to natural history. Biopsy data in (5/6) patient samples analyzed to date confirmed active regeneration of nerve fibers, noted January 31, 2022.",6
PTCT,Duchenne muscular dystrophy (age 2-5),Approved,6/7/19,"FDA Approval announced for label expansion June 7, 2019.",6
FOLD,Fabry Disease,Approved,8/10/18,"FDA Approval announced August 10, 2018.",6
UTHR,Pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease (PH-ILD),Approved,5/24/22,"Approved May 24, 2022.",6
ZVRA,induced Vasomotor Symptoms (iVMS) associated with menopause,Phase 1,3/17/23,"Phase 2a PoC did not meet its primary endpoint of ability to decrease the frequency or severity of hot flashes in postmenopausal women. As a result, the company is pausing the program until a full dataset review, noted March 17, 2023",6
ALVO,Inflammatory diseases,CRL,6/28/23,"Third CRL issued June 28, 2023. Resubmission planned.",5
CRIS,Relapsed or Refractory Non-Hodgkin Lymphoma,Phase 1,4/4/22,"Company voluntarily paused enrollment on April 4, 2022. Partial hold placed by FDA, noted April 11, 2022.",4
PRAX,Major depressive disorder,Phase 2,11/9/22,"Phase 2 top-line data was not statistically significant, noted November 9, 2022.",5
OCUL,Ocular inflammation and pain following cataract surgery,Approved,12/3/18,"FDA approval announced December 3, 2018.",6
DVAX,COVID-19 vaccine in adolescents,Phase 2/3,8/30/22,"Phase 2/3 data reported a 12 to 61-fold increase in neutralizing antibodies against BA.5, noted August 30, 2022.",5
AMGN,Glucocorticoid-induced osteoporosis (GIOP),Approved,5/21/18,"Approval announced May 21, 2018.",6
ANIP,Carnitine supplement,Approved,12/20/22,"Approved December 20, 2022.",6
REPL,Non-melanoma (NMSC) / Non-small cell lung cancer NSCLC),Phase 1,6/5/23,"Phase 1 data reported an overall objective response rate (ORR) of 37.4%, noted June 5, 2023.",5
RHHBY,Squamous non-small cell lung cancer (NSCLC),Approved,12/3/19,"FDA Approval announced December 3, 2019.",6
AMGN,Non-small cell lung cancer (NSCLC),Approved,5/28/21,"FDA approval announced May 28, 2021.",6
EQ,Systemic lupus erythematosus (SLE) and lupus nephritis (LN),Phase 1b,9/27/22,"Phase 1b interim data demonstrated clinically meaningful response in highly proteinuric subjects: 5 of 6 (83%) subjects achieved a complete or partial response and 4 of 6 (67%) subjects achieved > 80% reduction in urine protein creatinine ratio (UPCR) by week 28, 8 of 12 (67%) subjects achieved > 50% reduction in UPCR (6 subjects still dosing). The treatment continues to demonstrate favorable safety and tolerability through six months, noted September 27, 2022.",6
IBRX,Pancreatic cancer,Phase 2,6/6/22,"Phase 2 data from ASCO reported treatment more than doubled median overall survival, noted June 6, 2022.",6
GLMD,HIV-associated lipodystrophy and nonalcoholic fatty liver disease (NAFLD),Phase 2,2/14/18,"Phase 2 top-line data released February 14, 2018 did not meet primary endpoint.",4
ALLR,Metastatic breast cancer (mBC),Phase 2,7/5/23,"Phase 2 intial data showed potential for improved clinical benefit in DRP-selected metastatic breast cancer patients, noted July 5, 2023.",6
ACIU,Alzheimer’s disease (AD),Phase 2,12/1/22,"Phase 2 pharmacodynamic effects data presented at CTAD on December 1, 2022.",6
LLY,Severe hypoglycemia in people with diabetes,Approved,7/24/19,"FDA Approval announced July 24, 2019.",6
GMAB,Amyloidosis,Approved,1/15/21,"FDA approval announced January 15, 2021.",6
NVS,Non-small cell lung cancer (NSCLC) - 2nd/3rd line,Phase 3,3/9/21,Phase 3 trial did not meet primary endpoint.,4
INFI,Front-line triple negative breast cancer (TNBC); Renal cell cancer (RCC),Phase 2,11/14/22,"Phase 2 data reported a 52% increase in one-year progression free survival rate in ITT patient population, noted November 14, 2022.",5
ZLAB,Gastrointestinal Stromal Tumors (GIST) - second-line,Phase 3,6/1/23,"Phase 3 data reported that the mPFS (90% CI) by IRR was 6.44 (2.89, 8.31) months and ORR was 21.1%, noted June 1, 2023.",5
BGNE,HER2-amplified biliary tract cancers,Phase 2,6/5/23,"Phase 2 pivotal trial recruitment completed, noted April 28, 2022. Phase 2 full data presented at ASCO reported a confirmed objective response rate (cORR) of 41.3%, median duration of response (DOR) of 12.9 months, and median progression-free survival (PFS) of 5.5 months, noted June 5, 2023.",6
INDP,Advanced/metastatic solid tumors,Phase 1,10/31/23,"Phase 1 data abstract to be presented at SITC on November 4, 2023 noted that each of the first cohort participants experienced transient activation of biomarkers associated with innate and/or adaptive immune responses, and generally expected transient adverse events, noted October 31, 2024.",6
ACST,Ataxia-Telangiectasia (A-T),Phase 3,12/28/22,"Phase 3 pharmacokinetic(PK) study met all outcome measures, noted December 28, 2022.",5
EGRX,Percutaneous Coronary Intervention (PCI) and Percutaneous Transluminal Coronary Angioplasty (PTCA),CRL,3/18/16,CRL March 18 2016. FDA requested further characterization of bivalirudin-related substances in the drug product.,5
ICVX,RSV/human Metapneumovirus (hMPV),Phase 1,5/23/23,"Phase 1 data reported that the proportion of subjects experiencing any systemic AE within seven days was 25-41%, and similar to 35% for placebo, with induced geometric mean titers (GMTs) in RSV-A neutralizing antibody titers (nAbs) of up to approximately 16,100 IU/mL compared to approximately 2,600 IU/mL for placebo at Day 28, noted May 23, 2023.",5
TENX,Pulmonary Hypertension associated with Heart Failure and preserved Ejection Fraction (PH-HFpEF),Phase 2,6/2/20,"Phase 2 top-line data released June 2, 2020. Primary endpoint (Pulmonary Capillary Wedge Pressure (PCWP) with bicycle exercise) not met. Various secondary endpoints met.",5
EPZM,Follicular lymphoma,Approved,6/18/20,"FDA approval announced June 18, 2020.",6
BHVN,Acute treatment of migraine,Approved,2/27/20,"FDA Approval announced February 27, 2020.",6
IPHA,Head and neck squamous cell carcinoma of the head and neck (SCCHN),Phase 3,8/1/22,"Phase 3 trial did not meet the futility interim analysis, noted August 1, 2022.",6
DVAX,COVID-19 vaccine,Phase 3,3/2/23,"Phase 3 data reported that the fold decline of neutralizing antibodies over six months after a second vaccination with VLA2001 was similar to the active comparator, and less pronounced than for other licensed COVID-19 vaccines, noted March 2, 2023.",5
GOVX,Head and neck squamous cell carcinoma (HNSCC),Phase 1/2,7/10/23,"Phase 1/2 study data presented at AHNS resulted on impairment of tumor growth (i.e., ""stable disease"" using RECIST 1.1 evaluation criteria) in targeted lesions was seen in 5 of 7 patients; tumor response assessment in one patient remains under study, noted July 10, 2023.",6
AKBA,Anemia due to chronic kidney disease (CKD),Phase 3,4/3/23,"Phase 3 study met primary and secondary endpoints, noted April 3, 2023.",5
BLTE,Adolescent Stargardt disease (STGD1),Phase 1/2,10/1/22,"Phase 1b/2 trial 1-year interim data from the 2-year treatment in adolescent data reported that data showed stabilization in several parameters, including halting or slowing lesion growth, preservation in retinal thickness, reduction in Ellipsoid Zone (EZ) defect width, and stabilization of visual acuity, noted October 1, 2022.",6
FOLD,Pompe disease,Phase 1/2,10/12/22,"Phase 1/2 trial long term followup data reported that ERT-experienced patients and ERT-naïve patients had durable mean improvements from baseline, noted October 12, 2022.",6
MRK,Breast cancer,Approved,1/12/18,"Approval announced January 12, 2018.",6
MRNS,CDKL5 Deficiency Disorder (CDD),Approved,3/18/22,"Approved March 18, 2022.",6
RCKT,Fanconi Anemia (FA),Phase 2,12/12/22,"Phase 2 data reported that A seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and BM vector copy numbers (VCNs), noted December 12, 2022.",6
PLX,Fabry disease,Phase 3,3/18/22,"Phase 3 final results reported that 30% of patients reported treatment-emergent adverse events (TEAEs), noted March 18, 2022.",6
MRSN,"Ovarian Cancer, Non Small Cell Lung Cancer",Phase 1/2,7/27/23,"Phase 1/2 trial did not meet its primary endpoint, noted July 27, 2023.",6
ENSC,Severe Pain Relief,Phase 3,9/26/23,"Phase 3 data presented at the PAINWeek on September 7, 2023. Phase 3 completion of site initiation visit, noted September 26, 2023.",6
ABBV,Glioblastoma (rGBM),Phase 3,5/17/19,"Phase 3 trial did not meet primary endpoint at interim analysis - May 17, 2019.",4
ACRS,Hidradenitis suppurativa,Phase 2a,3/6/23,"Phase 2a study did not meet primary and secondary endpoints, noted March 6, 2023.",6
GILD,Ulcerative colitis,Phase 2/3,5/20/20,"Phase 2b/3 data released May 20, 2020. 200mg dose met primary endpoint. 100mg did not meet clinical remission endpoint.",5
REGN,Stage II to IV resectable cutaneous squamous cell carcinoma (CSCC),Phase 2,10/23/23,"Phase 2 data from ESMO 23 reported a 89% event-free survival rate at one year, and no disease recurrence among patients who achieved a pCR (n=40), noted October 23, 2023.",6
IMGN,Platinum-Resistant Ovarian Cancer,Approved,11/14/22,"Approved November 14, 2022.",6
LLY,COVID-19 Antibody,Approved,11/9/20,"Emergency Use Authorization (EUA) announced November 9, 2020.",6
CCXI,Hidradenitis Suppurativa (HS),Phase 2b,10/28/20,"Phase 2b data released October 28, 2020. Primary endpoint across Hurley Stages 2 and 3 not met. Phase 3 trial planned in Stage 3 patients only.",4
AMAM,PSMA Expressing Tumors,Phase 1,10/20/23,"Phase 1 data presented at ESMO reported that 52% (12/23) of patients experienced a ≥50% PSA reduction, and ≥90% PSA reduction observed in 6% (1/16) and 36% (5/14) of patients at 1.4 mg/kg (Cohort 4) and 2.0 mg/kg (Cohort 6), respectively, noted October 20, 2023.",6
NTLA,Transthyretin amyloidosis (ATTR),Phase 1,11/2/23,"Phase 1 updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001, including in 29 patients who have now reached 12 months or more of follow-up, noted November 2, 2023.",6
BMY,Psoriatic arthritis,Phase 2,11/3/21,"Phase 2 reported suppressed blood biomarkers of the IL-23/IL-17 and IFN-1 pathways and key markers of joint damage. There were no clinically meaningful changes in mean levels of serum cholesterol, creatinine, neutrophils and platelets over time with deucravacitinib treatment, noted November 3, 2021.",4
ISEE,Geographic atrophy,Phase 3,3/1/23,"Additional Phase 3 data reported that post-hoc time-to-event analysis signals up to 59% risk reduction in rate of vision loss compared to sham treatment at 12 months, noted March 1, 2023.",6
LLY,Type 2 Diabetes,Approved,1/27/20,"FDA Approval announced January 27, 2020.",6
NVCT,Non-small cell lung cancer (NSCLC),Phase 1a,9/12/23,"Phase 1a dose escalation initiated, noted September 12, 2023.",5
VOR,Acute myeloid leukemia (AML),Phase 1/2,12/7/22,"Phase 1/2a trial data from first patients reported that treatment engrafted normally and blood count was successfully maintained following post-transplant treatment, noted December 7, 2022.",6
PRLD,Solid tumors and hematological malignancies,Phase 1,10/16/23,"Phase 1 data reported that 8 (27.6%) patients had stable disease (SD) as the best response and the median PFS was 1.25 months, noted October 16, 2023.",5
TEVA,Inflammatory diseases,CRL,6/28/23,"Third CRL issued June 28, 2023. Resubmission planned.",5
SYBX,Phenylketonuria (PKU),Phase 1,7/11/23,"FDA Granted Fast Track Designation on July 11, 2023.",5
BIIB,Acute ischemic stroke,Phase 2,5/12/21,"Phase 2 trial met primary objective - May 12, 2021.",6
XNCR,Relapsed or refractory diffuse large B cell lymphoma,Approved,7/31/20,"FDA Approval announced July 31, 2020.",6
HALO,Chronic inflammatory demyelinating polyneuropathy (CIDP),Phase 2,7/17/23,"Phase 2 trial met primary endpoints, noted July 17, 2023.",5
BMY,Multiple Myeloma,Phase 1,12/12/22,"Phase 1 results reported that cytokine release syndrome (CRS) occurred in 80.0% of all treated pts, with most experiencing grade 1 (63.6%) or grade 2 (14.5%); only 1 pt experienced ≥ grade 3 (grade 4) CRS, noted December 12, 2022.",6
CADL,Prostate Cancer (Active Surveillance),Phase 2,9/25/21,"Phase 2 safety data displayed 2 grade 4 events, and 3 hospitalizations due to inflection (n=365). The most common AE was flu-like symptoms reported by 21% of patients, noted September 25, 2021.",5
PFE,Metastatic hormone sensitive prostate cancer,Phase 3,9/17/21,"Phase 3 data showed a reduced risk of death by 34% compared to placebo plus ADT. The median OS was not reached in either treatment group, September 17, 2021.",6
EXEL,Medullary thyroid cancer,Approved,11/29/12,"Approved November 29, 2012.",6
HOOK,Cytomegalovirus (CMV),Phase 2,11/9/21,"Phase 2 safety, immunogenicity, and efficacy data reported 86% seroconversion and 100% CD8+ T cell responses, 41% reduction in CMV viremia, and 41% reduction in the use of antiviral therapy, noted November 9, 2021.",6
ARWR,Hepatitis B (HBV),Phase 2b,11/15/21,"Phase 2b results reported a dose dependent response with 19.1% of patients meeting the primary endpoint and 74.7% of patients achieving HBsAg <100 IU/mL, noted November 15, 2021.",6
LLY,Solid tumors that harbor a TRK fusion.,Approved,11/26/18,"FDA Approval announced November 26, 2018.",6
CRDF,Acute Myeloid Leukemia (AML),Phase 1/2,12/6/20,"Phase 1/2 data presented at ASH December 6, 2020. 9 of 45 (20%) evaluated patients achieved CR/CRi.",5
INSM,COVID-19,Phase 3,7/31/21,Phase 3 failed noted July 2021.,4
RGNX,"Batten Disease, CLN2",Phase 1,8/30/23,"Phase 1 initial interim data reported that treatment was well tolerated with no serious adverse events. Key efficacy measures demonstrated sustained levels of TPP1 along with increased intervals between enzyme replacement therapy (ERT) infusions and an 86% reduction in seizure frequency through six month, noted August 30, 2023.",6
CHRS,Pegfilgrastim biosimilar,Approved,3/6/23,"Autoinjector approved March 6, 2023.",6
VERU,Castration Resistant Prostate Cancer,Phase 1/2,6/6/22,"Phase 1/2 final analysis reported an objective response rate of 20.7% and that the median radiographic progression free survival was 11.4 months, noted June 6, 2022.",5
PRVB,Type 1 diabetes,Approved,11/17/22,"Approved November 17, 2022.",6
NRIX,Cellular therapy,Phase 1,11/10/22,"Phase 1 clinical update reported increases the antitumor activity of the transferred T cells when compared to ACT alone, noted November 10, 2022.",5
HCM,Colorectal cancer,Phase 3,9/8/22,"Phase 3 data demonstrated that treatment reduced the risk of death by 34% in metastatic colorectal cancer , and Increased disease control with risk of disease progression or death reduced by 68%, noted September 8, 2022",6
NCNA,Advanced biliary tract cancer,Phase 3,3/2/22,"Independent Data Monitoring Committee (IDMC) concluded that trial was unlikely to achieve its primary objective and that treatment did not into an overall survival benefit, noted March 2, 2022.",6
REGN,Cardiovascular events,Approved,4/26/19,"FDA approval announced April 26, 2019.",6
GSK,Non-squamous non-small cell lung cancer (NSCLC),Phase 2,10/5/22,"Phase 2 trial met primary endpoint, noted October 5, 2022.",5
LLY,Axial Spondyloarthritis,Approved,8/26/19,"FDA Approval announced August 26, 2019.",6
JAZZ,Acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL),Approved,11/18/22,"Approved November 18, 2022.",6
MRK,Unresectable hepatocellular carcinoma (uHCC),Phase 3,9/10/22,"Phase 3 trial data was not statistically significant, noted August 3, 2022. Phase 3 data presented at ESMO showed that there was a trend toward improvement for one of the study's dual primary endpoints, overall survival (OS), for patients treated; however, the results did not meet statistical significance per the pre-specified statistical plan. The median OS was 21.2 months for combination and 19.0 months monotherapy, September 10, 2022.",6
BBIO,ATTR-PN,Phase 3,2/28/22,"Phase 3 trial has been initiated. Phase 3 trial has been withdrawn, noted February 2022.",5
CLSD,Macular edema associated with uveitis,Approved,10/25/21,"Approved October 25, 2021.",6
DBVT,Peanut allergy - ages 1 to 3 years,Phase 3,2/24/23,"Phase 3 data due presented at AAAAI reported that data demonstrate that treatment with Viaskin Peanut 250 μg for up to 36 months in peanut-allergic children was generally safe and well tolerated, noted February 24, 2023.",6
AGLE,Classical Homocystinuria,Phase 1/2,4/12/23,"Phase 1/2 data reported that treatment with pegtarviliase lowered total homocysteine levels in participants when compared to baseline values, and in the third cohort (1.35 mg/kg) did not show a consistent reduction in total homocysteine levels compared to baseline, noted April 12, 2023.",6
RHHBY,Triple-negative breast cancer (TNBC),Phase 3,8/6/20,"Phase 3 trial did not meet primary endpoint - October 6, 2020.",4
INCY,Chronic Graft versus host disease (cGvHD),Phase 2,7/24/23,"Phase 2 trial results met its primary endpoint across all cohorts with an overall response rate (ORR) of 74% at a dose of 0.3 mg/kg administered every two weeks, noted July 24, 2023.",5
KPTI,Myelofibrosis - naïve,Phase 2,7/17/23,"FDA Granted Fast Track Designation on July 17, 2023.",5
MRK,First-Line Nonsquamous Non-small cell lung cancer (NSCLC),Approved,6/5/18,"FDA approval (label expansion) announced June 5, 2018.",6
GRTX,Severe Oral Mucositis (SOM),CRL,8/9/23,"FDA issued a Complete Response Letter (CRL), noted August 9, 2023.",5
TAK,NSCLC with EGFR exon 20 insertion mutations,Phase 3,10/2/23,"Phase 3 trial withdrawn voluntarily after the confirmatory trial, which did not meet its primary endpoint, noted October 2, 2023.",6
KURA,Relapsed or refractory acute myeloid leukemia (AML),Phase 1/2,6/10/23,"Phase 1/2 data presented at EHA showed that 35% CR rate (7/20) among patients with relapsed/refractory NPM1-mutant AML treated at 600 mg dose (RP2D) and 33% (2/6) of patients with FLT3 co-mutations and 50% (4/8) of patients with IDH co-mutations achieved a CR on ziftomenib, noted June 10, 2023.",6
KTRA,MGMT-unmethylated Recurrent Bevacizumab Naïve Glioblastoma Multiforme (GBM) or The Adjuvant,Phase 2,11/18/21,"Phase 2 data noted that In the adjuvant group PFS was 9.5 months and mOS was 16.5 months, noted November 18, 2021.",5
ONCY,Pancreatic cancer,Phase 2,5/20/21,Phase 2 data in ASCO abstract noted response rate 1/12 (8.3%); stable disease 42%.,4
TAK,Renal cell carcinoma,Phase 2,1/4/21,"Phase 2 top-line data did not meet primary endpoint - January 4, 2021.",4
VKTX,Obesity,Phase 1,10/18/23,"Phase 1 data demonsted that 28 days of once-weekly treatment with VK2735 resulted in reductions in subjects' liver fat content and plasma lipid levels, both from baseline and as compared to placebo, noted October 18, 2023.",6
BHC,Acne,Approved,12/19/19,"FDA Approval announced December 19, 2019.",6
NXTC,Solid tumors,Phase 1,11/7/22,"Phase 1 data reported that treatment is safe and well-tolerated with early signs of disease control in patients with advanced tumors, noted November 7, 2022.",6
ABIO,Chronic Heart Failure,Phase 2b,5/27/18,"Oral presentation of data May 27, 2018.",5
ENDP,Edematous fibrosclerotic panniculopathy (“cellulite”),Approved,7/6/20,"FDA Approval announced July 6, 2020.",6
GLTO,Myelofibrosis,Phase 2a,12/12/22,"Phase 2a data presented at ASH reported that no significant safety concerns were observed to date, noted December 12, 2022.",6
ABBV,Lower limb spasticity,Approved,10/24/19,"FDA Approval announced October 24, 2019.",6
STOK,Dravet syndrome,Phase 1/2,7/25/23,"Phase 1/2a OLE OLE data of STK-001 (30mg, 45mg) showed a Sustained reductions in convulsive seizure frequency and improvements in cognition and behavior, noted July 25, 2023.",6
AMRN,Covid-19,Phase 3,11/16/21,"Phase 3 trial did not met the primary or secondary endpoints, noted November 16, 2021.",4
INTS,Solid tumors,Phase 1/2,11/17/22,"Phase 1/2 data reported at CTOS reported that patients who had 40% or more of their tumor burden treated with INT230-6, an exploratory analysis showed that overall survival was improved when compared to historical benchmarks, noted November 17, 2022.",6
ABBV,Presbyopia,Phase 3,4/5/22,"Phase 3 trial met primary endpoint, noted April 5, 2022.",5
OPK,Adults - growth hormone deficiency,Phase 3,12/30/16,"Phase 3 data released December 30, 2016 - primary endpoint not met.",4
CARA,Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus,Approved,8/23/21,"FDA approval announced August 23, 2021.",6
VIR,COVID-19 antibody,Approved,2/11/22,"EUA granted by FDA February 11, 2022.",5
GILD,Indolent B-Cell Non-Hodgkin Lymphoma,Approved,3/5/21,"FDA approval announced March 5, 2021.",6
AKTX,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 2/3,12/11/20,"Additional data from Phase 2 and 3 trials released December 11, 2020 - reduced transfusion dependence by 79% in the 14 formerly transfusion dependent patients.",6
GLTO,Liver cirrhosis,Phase 1/2,11/8/22,"Phase 1b/2a top-line data reported that data showed statistically significant reductions in the liver enzymes ALT, AST and GGT, with encouraging reductions for ALP , after 12 weeks of treatment, noted November 8, 2022.",6
SNY,Non-small cell lung cancer (NSCLC),Approved,2/22/21,"FDA approval announced February 22, 2021.",6
BMY,Relapsed or refractory (R/R) large B-cell lymphoma,Approved,2/5/21,"FDA approval announced February 5, 2021.",6
EXEL,Metastatic renal cell cancer (RCC),Approved,4/25/16,"Approved April 25, 2016.",6
IVVD,COVID-19 (treatment),Phase 2/3,9/29/21,"Phase 2/3 new data compiled to date suggest that the 300 mg IM regimen has a projected ability to rapidly achieve and maintain target concentrations at key tissue sites of viral replication, including the ability to attain near complete (> 90%) and durable (> 28-day) SARS-CoV-2 receptor occupancy across a range of baseline viral loads, noted September 29, 2021.",6
CDTX,Candidemia,Approved,3/22/23,"FDA approval on March 22, 2023.",6
JNJ,Multiple myeloma,Approved,7/12/21,"FDA approval announced July 12, 2021.",6
BGNE,HER2-positive breast cancer,Phase 1/2,5/26/22,"Phase 1b/2 data released in abstract reported that the cORR was 90.5% and the six-month progression-free survival rate was 95.2%, noted May 26, 2022.",5
ALKS,Relapsing Remitting Multiple Sclerosis (RRMS),Phase 3,7/30/19,"Phase 3 top-line data met primary endpoint - July 30, 2019.",6
EYEN,Presbyopia,Phase 3,10/20/22,"Phase 3 data reported that trial met its primary endpoint in a modified per-protocol analysis of evaluable patients, and adverse events were reported in fewer than 3% of patients, and all were mild and/or transient, noted October 20, 2022.",6
TRVN,Moderate to severe acute pain,Approved,8/7/20,"FDA Approval announced August 7, 2020.",6
AZN,Type-2 diabetes,Approved,2/28/17,"Approval announced February 28, 2017.",6
SNY,Respiratory Syncytial Virus,Phase 2/3,6/28/21,"Phase 2/3 positive data released June 28, 2021.",6
CTXR,Hemorrhoids,Phase 2b,6/20/23,"Phase 2b enrollment completed, noted April 3, 2023. Phase 2b data provided meaningful reduction in symptom severity when compared to individual components alone, noted June 20, 2023.",6
VIR,Hepatitis B,Phase 1,6/24/23,"Phase 1 data showed that treatment has a shorter terminal half-life and was cleared faster in participants with higher baseline HBsAg, noted June 24, 2023.",6
AMGN,Scalp Psoriasis,Approved,4/30/20,FDA approval announced April 2020.,6
VRNA,Chronic obstructive pulmonary disease (COPD),Phase 3,12/20/22,"ENHANCE-1 Phase 3 topline data met its primary and key secondary endpoints, noted December 20, 2022.",5
GLTO,COVID-19,Phase 2,6/22/21,"Phase 2 top-line data released June 22, 2021. Will focus resources on other pipeline treatments.",4
BIIB,Mitochondrial myopathies (MM),Phase 2,3/1/18,"Phase 2 initial data released March 1, 2018 - endpoints not met.",4
TVTX,Classical homocystinuria (HCU),Phase 1/2,5/31/23,"Additional Phase 2 data reported that treatment was well tolerated, with dose-dependent reductions in tHcy as previously observed in the highest BIW dosed cohorts, noted July 6, 2023.",6
MRK,Solid tumors,Phase 1/2,10/12/23,"Phase 1/2 updated results presented at AACR-NCI-EORTC reported that the overall response rate (ORR) was 38% (6/16 evaluable patients) at the Recommended Phase 2 Dose (RP2D) of 2000 mg daily and 34% (13/38) in the efficacious dose range, noted October 12, 2023.",6
LLY,Primary axillary hyperhidrosis,Approved,6/29/18,"Approval announced June 29, 2018.",6
SNY,High low-density lipoprotein (LDL) cholesterol,Approved,4/25/17,"sBLA Approval announced April 25, 2017.",6
MYOV,Uterine fibroids,Approved,5/26/21,"FDA approval announced May 26, 2021.",6
AMGN,Asthma and comorbid nasal polyps,Phase 3,9/5/21,"Phase 3 data showed an 86% reduction in the annualized asthma exacerbation rate (AAER) September 5, 2021.",6
MRK,Crohn’s Disease,Phase 2a,3/3/23,"Phase 2a primary and secondary results reported efficacy in biologic-experienced subjects consistent with overall population and consistent across subgroups and irrespective of presence of ADA, noted March 3, 2023.",5
NRSN,Parkinson's disease,Phase 1,5/2/23,"Biomarker data reported statistically significant decreased levels of novel biomarker AGO2 observed in newly diagnosed people living with Parkinson's disease (PD) compared to healthy individuals, noted May 2, 2023.",6
AZN,Cancer - leukaemia,Approved,9/13/18,"Approval announced September 13, 2018.",6
CPRX,Lambert-Easton Myasthenic Syndrome (LEMS),Approved,11/28/18,"FDA approval announced November 28, 2018.",6
CRDF,Prostate cancer,Phase 2,2/11/21,Phase 2 data disease control rate at 12 weeks of 35%.,5
FBIO,Gout,Phase 1,6/29/23,"Phase 1 study dosing commenced, noted May 31, 2022. Phase 1 data show edcomparable pharmacokinetic, pharmacodynamic and safety profile between U.S. and Japanese healthy subjects, noted Jue 29, 2023. Phase 1b clinical trial in gout patients in the U.S. and expect to begin pivotal clinical trials in 2024.",6
BIVI,Alzheimer's disease,Phase 2,12/5/22,"Phase 2 final data reported that improvements in QDRS scores were statistically significantly correlated with improvements on the ADCOMS assessment, noted December 2, 2022.",6
REGN,Homozygous familial hypercholesterolemia (HoFH),Approved,2/11/21,"FDA approval announced February 11, 2021.",6
ABBV,Psoriatic Arthritis,Approved,1/21/22,"Approved January 21, 2022.",6
VXRT,Norovirus vaccine,Phase 2,7/6/23,"Phase 2 topline data reported a similar increase in serum antibody responses with no statistical difference between the medium and high dose arms, and the candidate was well tolerated, noted July 6, 2023.",6
KPTI,Myelofibrosis (MF) - previously treated,Phase 3,6/28/23,"Phase 3 dosing commenced, noted June 28, 2023.",5
RCKT,Pyruvate Kinase Deficiency (PKD),Phase 1,5/19/23,"Phase 1 data reported robust and sustained efficacy in adults and the first pediatric patient infusion of RP-L301 was well tolerated, with engraftment achieved at day +15, noted May 19, 2023.",6
NVS,Pediatric BRAF V600E low-grade glioma,Approved,3/17/23,"Approved March 17, 2023.",6
OMER,COVID-19,Phase 2,9/15/22,"Phase 2 data reported that neither the futility nor graduation criteria were met in the analysis of the randomized population at the time the narsoplimab arm was terminated, noted September 15, 2022.",4
RHHBY,Systemic sclerosis-associated interstitial lung disease (SSc-ILD),Approved,3/4/21,"FDA approval announced March 4, 2021.",6
VCNX,Huntington’s disease,Phase 2,6/10/22,"Phase 2 data reported that treatment restores the ability to benefit from experience, noted June 10, 2022.",6
MGNX,Squamous cell carcinoma of the head and neck (SCCHN),Phase 2,7/8/22,"Phase 2 trial discontinued following an internal review of safety data, which included the occurrence of seven fatalities potentially associated with hemorrhagic events in both arms of the study, noted July 8, 2022.",4
GSK,Eosinophilic Granulomatosis with Polyangiitis (EGPA),Approved,12/12/17,"Approval announced December 12, 2017.",6
PFE,Acute myeloid leukemia (AML),Approved,11/21/18,"FDA approval announced November 21, 2018.",6
DMAC,Acute ischaemic stroke (AIS),Phase 2/3,6/21/23,"Clinical hold removed, noted June 21, 2023.",5
PFE,Irritable Bowel Syndrome,Phase 2b,3/2/21,"Phase 2b trial did not meet primary endpoint - March 2, 2021.",4
ARDS,Pseudomonas aeruginosa,Phase 2,9/3/19,"Phase 2 trial did not met primary endpoint - September 3, 2019.",4
APVO,Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS),Phase 1b,7/18/23,"Phase 1b expansion trial data reported that one patient remained on study with sustained complete response for 8 cycles, noted July 18, 2023.",5
TALS,Delayed tolerance kidney transplant,Phase 2,2/16/23,"Trial was discontinued due to the pace of enrollment and the associated timeline to critical milestones, noted February 16, 2023.",4
ALPN,Advanced Malignancies,Phase 1,4/12/22,"Phase 1 dose escalation results across a range of dose regimens, tumor volume reduction was observed in 23% of evaluable participants despite heavily pretreated.",6
HOOK,Treatment-refractory HPV16+ cancers,Phase 1/2,5/26/22,"Phase 1/2 data from abstract reported that treatment was well-tolerated with adverse events being mild or moderate, noted May 26, 2022.",6
JNJ,Alzheimer’s disease,Phase 2,5/18/18,"Announced May 18, 2018 that dosing will be stopped.",4
CADL,Non-small cell lung cancer (NSCLC),Phase 2,9/26/23,"Phase 2 initial data suggests 12-month survival is consistent with an increased tail on the maturing survival curve, noted September 26, 2023.",5
ONCT,Mantle cell lymphoma (MCL),Phase 3,4/3/23,"Phase 3 trial closed due to company strategic prioritization, noted April 3, 2023.",5
OVID,CDKL5 Deficiency,Phase 2,9/30/20,"Phase 2 data released September 30, 2020. Median motor seizure frequency reduction was 24%.",6
IRWD,Chronic idiopathic constipation (CIC),Approved,1/26/17,"sNDA approval announced January 26, 2017.",6
GMAB,Multiple Myeloma,Approved,8/20/20,"FDA approval announced August 20, 2020.",6
BMY,Metastatic non-small cell lung cancer (NSCLC),Phase 3,6/5/23,"Phase 3 data reported that the dual immunotherapy-based combination continued to enhance overall survival (OS), the trial's primary endpoint, with 21% of patients treated with Opdivo plus Yervoy with two cycles of chemotherapy alive compared to 16% of patients treated with chemotherapy alone at four years , noted June 5, 2023.",6
EXEL,First-Line RCC - cancer,Approved,12/19/17,"Approval announced December 19, 2017, ahead of PDUFA date of February 15, 2018.",6
SAGE,Postpartum depression (PPD),Phase 3,10/17/22,"Additional Phase 3 data reported that a total of two participants (all in the zuranolone group) experienced four serious adverse events all of which were assessed by the investigator as unrelated to the therapy, noted October 17, 2022.",5
PCRX,Lower extremity nerve blocks,Phase 3,5/26/21,"Phase 3 data released May 26, 2021. Did not demonstrate statistical significance for primary endpoint.",4
IVA,Nonalcoholic fatty liver disease (NAFLD),Phase 2,6/13/23,"Phase 2 data of 800mg achieved the primary efficacy endpoint demonstrating a 44% reduction of hepatic fat measured by proton magnetic resonance spectroscopy (1H-MRS) following 24 weeks of treatment in patients with nonalcoholic fatty liver disease (NAFLD), noted June 13, 2023.",6
BHVN,Obsessive compulsive disorder (OCD),Phase 2/3,6/24/20,Phase 2/3 top-line data did not meet primary endpoint.,4
IFRX,ANCA-associated Vasculitis (AAV),Phase 2,11/15/21,"Phase 2 data released November 15, 2021. Clinical response as well as clinical remission were achieved in comparably high rates in all three arms: Clinical response at week 16 in evaluable patients was observed in 16 out of 18 (88.9%) patients in the treatment group receiving vilobelimab alone; in 22 out of 23 (95.7%) patients receiving SDGC; and in 10 out of 13 (76.9%) patients in the vilobelimab + RDGC group.",6
NKTR,Urothelial cancer,Phase 2,4/14/22,"Phase 2 trial did not meet the efficacy threshold in its final ORR analysis, noted April 14, 2022.",6
BMY,Renal Cell Carcinoma (RCC),Phase 3,9/8/22,"Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.",6
ABVC,Attention Deficit Hyperactivity Disorder (ADHD),Phase 2a,9/23/22,"Part 1 of Phase 2 study found that the active ingredient of ABV-1505, PDC-1421, was safe, well-tolerated and efficacious during the treatment and follow-up period with six adult patients, noted September 23, 2022.",6
RHHBY,Diffuse large B-cell lymphoma (DLBCL),Approved,6/10/19,"FDA approval announced June 10, 2019.",6
SNY,Sickle Cell Disease,Phase 1/2,12/13/21,"Phase 1/2 data reported that total hemoglobin stabilized by Week 26 after treatment in all four patients. Fetal hemoglobin level increased from 0.1-11% at screening to 14-39% in all four patients and was 38% in the longest-treated patient at 91 weeks, noted December 13, 2021.",6
DERM,Papulopustular Rosacea,Phase 3,7/11/23,"Phase 3 topline data achieved the co-primary and all secondary endpoints and subjects completed the 16-week treatment with no significant safety issues, noted July 11, 2023.",6
PFE,Urothelial Carcinoma,Approved,5/9/17,"Approval announced May 9, 2017.",6
FRLN,Fabry disease,Phase 1/2,2/8/22,"Phase 1/2 data to be presented at the Annual WORLDSymposium reported that treatment has been well tolerated with myocarditis observed in both patients, noted February 8, 2022.",6
PHAT,H. pylori,Approved,10/30/23,"Prior Approval Supplement (PAS) for the reformulation of vonoprazan tablets approved October 30, 2023.",6
SRNE,Non-small cell lung cancer (NSCLC),Phase 3,11/12/21,"Phase 3 top-line data reported an overall response rate (ORR) of 52.2%, and overall survival (OS) of 24.9 months, noted November 12, 2021.",5
YMAB,CNS/Leptomeningeal Metastases from Neuroblastoma,CRL,12/1/22,"CRL issued by FDA on December 1, 2022.",5
ZVRA,Acute pain,Approved,2/23/18,"CRL issued June 13, 2016. Resubmitted, and issued FDA Approval February 23, 2018.",6
CYAD,Multiple Myeloma,Phase 1,12/21/22,"Phase 1 additional data reported 5 partial responses, program has been discontinued, noted December 21, 2022.",4
NVO,Obesity,Phase 3,8/8/23,"Phase 3 trial met its primary endpoint, with statistically significant and superior reduction in MACE of 20% for people treated with semaglutide 2.4 mg compared to placebo, noted August 8, 2023.",6
RHHBY,Influenza in patients 5 years and older,Approved,8/11/22,"Approved August 11, 2022.",6
FLGT,Various cancers,Phase 1/2,11/3/23,"Phase 1/2 data reported that FID-007 may have anti-tumor activity in heavily pre-treated patients across various tumor types, and a subgroup analysis based on 7 patients for Head and Neck cancer and 4 patients for Ampullary/Pancreatic cancer showed 57% and 50% objective response rate, respectively, noted November 3, 2023.",6
AZN,Respiratory syncytial virus,Approved,7/17/23,"FDA Approved on July 17, 2023.",6
HARP,Metastatic castration-resistant prostate cancer,Phase 1/2,5/19/21,Phase 1/2 data in ASCO abstract noted PSA and CTC reductions and treatment duration > 24 weeks in 15/62 patients.,6
BGNE,Chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL),Phase 2,6/15/23,"Phase 2 long-term follow-up demonstrated high rates of durable undetectable minimal residual disease (MRD) in CLL/SLL patients administered BOVen. Of the 46 patients meeting MRD criteria to cease treatment, median time from end of treatment to MRD conversion was 29.8 months, noted June 15, 2023.",6
MBIO,X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants,Phase 1/2,4/17/19,Phase 1/2 trial ongoing. Initial data noted in NEJM.,5
JNJ,Non-small cell lung cancer,Phase 3,10/24/23,"Phase 3 data presented at ESMO reported that the median PFS was 11.4 months and the ORR was 73%, noted October 24, 2023.",6
MRKR,Lymphoma who have failed or are ineligible to receive anti-CD19 CAR T cell treatment,Phase 1,9/11/23,"Phase 1 clinical update showed that patients with Non-Hodgkin's Lymphoma who relapsed after anti-CD19 CAR T cell therapy tolerated initial dose level well and achieved complete response after MT-601 treatment, noted September 11, 2023.",6
VXRT,Norovirus vaccine,Phase 2,9/6/23,"Phase 2 study met 5 of 6 primary endpoints,.Vaccination led to a statistically significant reduction in infection rate, a non-statistically significant reduction in norovirus acute gastroenteritis (AGE), and a substantial reduction in viral shedding, noted September 7, 2023.",6
MRUS,Solid tumors,Phase 1,12/6/21,"Phase 1 data demonstrated preliminary evidence of antitumor activity has been observed at doses ≥25 mg biweekly, median duration was approximately 6 (1–74) weeks. TEAEs occurred in 33 patients (97.1%); treatment-related TEAEs occurred in 23 patients (67.6%), most commonly fatigue and decreased neutrophil count, noted December 6, 2021.",6
NBRV,Cystic fibrosis (CF),Phase 1,11/28/22,"Phase 1 top-line data reported that treatment was well-tolerated, noted November 28, 2022.",6
CMRX,Acute lung injury (ALI) in COVID-19,Phase 2,5/6/21,"Phase 2 initial data released May 6, 2021. One patients died in DSTAT arm.",5
NVS,HR+/HER2- advanced breast cancer,Approved,3/13/17,"Priority review granted November 1, 2016. Approval announced March 13, 2017.",6
THRX,Gastrointestinal stromal tumors (GIST),Phase 1/2,5/25/23,"Phase 1/2 dose-escalation data demonstrated encouraging safety profile through Cohort 6 (18 mg), supportive of continued dose escalation, noted May 25, 2023.",6
TAK,Alpha-1 Liver Disease,Phase 2,6/24/23,"Phase 2 data presented at EASL showed that at week 48, patients receiving 25, 100, or 200 mg fazirsiran achieved serum Z-AAT reductions of 74%, 89%, and 94%, respectively, versus an increase of 9% observed in patients receiving placebo, noted June 24, 2023.",6
ATHX,Ischemic stroke,Phase 2/3,5/20/22,"Phase 2/3 final top-line results reported that trial did not meet the primary endpoint, noted May 20, 2022.",6
SNY,Refractory Multiple Myeloma,Approved,3/2/20,"FDA Approval announced March 2, 2020.",6
PLUR,COVID-19 / Acute Respiratory Distress Syndrome (ARDS),Phase 2,12/27/21,"Phase 2 data did not meet the primary efficacy endpoint of statistically significant improvement of ventilator free days (VFD) at 28 days. Taking into consideration the baseline risk factors of the ARDS patients, no differences in the safety profile were observed between PLX-PAD and placebo.",6
CASI,Non-Hodgkin's Lymphoma,Phase 1/2,11/5/21,"Phase 1/2 data reported an objective response rate (ORR) of 50%, with three complete responses and three partial responses seen in twelve patients, noted November 5, 2021.",5
LLY,Alopecia Areata,Approved,6/13/22,"Approval announced June 13, 2022.",6
LLY,First-line gastric cancer,Phase 3,12/8/17,Phase 3 PFS data met primary endpoint but overall survival not improved. Will not seek regulatory approval.,4
ABBV,Upper limb spasticity,Approved,6/21/19,"FDA Approval announced June 21, 2019.",6
LLY,"Refractory chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), non-Hodgkin’s lymphomas (NHL); mantle cell lymphoma",Phase 1/2,12/12/21,"Phase 1/2 updated data reported an overall response rate (ORR) of 68% including 2 complete responses, noted December 12, 2021.",5
ACST,Hypertriglyceridemia,Phase 3,8/31/20,"TRILOGY 2 data August 31, 2020 also did not meet primary endpoint.",4
TCRR,Mesothelin-positive solid tumors,Phase 1/2,9/28/22,"Phase 1 translational data demonstrated tolerability and clinical benefit. Second RECIST PR in ovarian cancer supports broad potential of treatment, consistent tumor regression in 93% evaluable patients with DCR of 77%. PFS of 5.6 months and overall survival of 11.2 months suggest durability of benefit in mesothelioma. Phase 2 portion of trial underway, noted September 28, 2022.",6
PFE,Type 2 diabetes,Approved,12/20/17,"Approval announced December 20, 2017.",6
ALKS,"Schizophrenia, bipolar",Approved,6/1/21,"FDA approval announced June 1, 2021.",6
JSPR,Acute myeloid leukemia/Myelodysplastic syndrome (AML/MDS),Phase 1,11/2/23,"Phase 1 results from abstracts reported that 10 of 13 AML in CR and 14 of 16 MDS subjects had MRD at pre-HCT screening assessed by cytogenetics, flow cytometry, and/or next generation sequencing, noted November 2, 2023.",5
NVS,Psoriasis,Approved,2/8/18,"FDA approval announced February 8, 2018.",6
SAVA,Mild-to-Moderate Alzheimer's Disease,Phase 3,10/25/23,"Phase 3 MRI data suggested that simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities, or ARIA. MRIs were all analyzed for ARIA by board-certified neuroradiologists, noted October 25, 2023.",6
TRDA,Duchenne Muscular Dystrophy (DMD),Phase 1,9/21/23,"Phase 1 trial initiated, noted September 21, 2023.",5
FBRX,Atopic Dermatitis,Phase 2,9/2/21,"Phase 2 failed to meet primary endpoint September 02, 2021.",6
AZN,Lung cancer,Phase 3,9/17/21,"Phase 3 median PFS of 16.9 months was observed, September 17, 2021.",6
VTRS,Primary Progressive Multiple Sclerosis (PPMS),Phase 3,4/24/23,"Phase 3 data presented at AAN demonstrated the significant treatment effect of GA Depot in reducing ARR (30%, p=0.0066), strengthened by MRI endpoints, supports the use of GA Depot as a DMT for RMS patients, noted April 24, 2023.",6
DRMA,Rosacea,Phase 2,12/5/22,"Phase 2 top-line data reported that trial did not meet primary endpoint, noted December 5, 2022.",6
PFE,HIV,Approved,1/21/21,"FDA approval announced January 21, 2021.",6
NYMX,Benign prostatic hyperplasia (BPH),Phase 3,7/27/15,Phase 3 endpoints met in extension trial July 2015.,6
MRTX,Non-small cell lung cancer (NSCLC),Phase 3,5/24/23,"Phase 3 final analysis did not met its primary endpoint of overall survival, noted May 24, 2023.",5
MRK,BRCAm HER2-negative breast cancer,Approved,3/11/22,"Approved March 11, 2022.",6
GSK,Ovarian cancer,Approved,4/29/20,"FDA Approval announced April 29, 2020.",6
TAK,Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL),Phase 3,6/10/23,"Phase 3 data presented at EHA showed that Ponatinib was superior to imatinib in combination with reduced-intensity chemotherapy in the front-line setting for patients with Ph+ ALL, with a significantly higher MRD-neg CR rate at EOI. Ponatinib was associated with deeper and more durable responses, with a trend toward improved EFS and comparable safety vs imatinib, noted June 9, 2023.",6
RLYB,Paroxysmal nocturnal hemoglobinuria (PNH); Generalized Myasthenia Gravis (gMG),Phase 1,9/3/23,"Phase 1 multiple ascending trial data reported maximum exposures of greater than 1 µM and 3 µM, respectively, and greater than 99% reductions in free C5 concentrations, noted September 3, 2023.",6
BMY,Graft Versus Host Disease (aGvHD),Approved,12/15/21,"Approved December 15, 2021.",6
AZN,Advanced Hematological Malignancies,Phase 1/2,12/12/22,"Phase 1/2 preliminary results reported that treatment has been well tolerated, with no DLTs to date and no discontinuations due to treatment-related AEs, noted December 12, 2022.",6
MNOV,Alcohol Use Disorder (AUD),Phase 2b,6/29/23,"Phase 2b data reported that treatment was not superior to placebo, noted June 29, 2023.",6
RDHL,IBS-D,Phase 2,10/3/17,"Phase 2 top-line data released October 3, 2017 - primary endpoint met (borderline) - p=0.05.",6
LPTX,Biliary tract cancer (BTC),Phase 1/2,12/14/18,"Phase 1/2 data released December 14, 2018 noted 21.3% PR rate.",5
PFE,Oral Protease Inhibitor for COVID-19,Approved,5/25/23,"FDA approved on May 25, 2023.",6
BIIB,Alzheimer's disease,Phase 1/2,3/29/23,"Phase 1b trial of 1b/2 study met its primary objective of safety and tolerability. Phase 1b LTE study results showed that dosing reduced biomarkers of soluble tau in CSF (t-tau and p-tau181) in a dose-dependent and sustained manner, with all dose groups showing approximately a 60% reduction from baseline CSF tau levels by the end of the LTE .Phase 2 trial in progress, noted March 29, 2023.",6
MACK,Cancer - front line pancreatic cancer,Phase 2,6/25/18,"Phase 2 data released June 25, 2018 - endpoints not met.",4
TPST,"Hepatocellular Carcinoma (HCC), Solid tumors",Phase 1,4/17/23,"Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.",6
ARQT,Scalp psoriasis,Phase 2,9/30/21,"Phase 2 study met its primary endpoint with a Scalp-Investigator Global Assessment success rate of 59.1% patients compared to a vehicle rate of 11.4% (P<0.0001). Of these, 34.3% of patients on roflumilast foam achieved a status of clear - noted September 30, 2021.",6
BLUE,Cerebral Adrenoleukodystrophy (CALD),Approved,9/17/22,"Approved September 17, 2022.",6
ALPN,Systemic lupus erythematosus (SLE),Phase 1,5/20/23,"Phase 1 data shared at the International Congress on Systemic Lupus Erythematosus reported that data demonstrated dose-dependent PK/PD. Coverage of free APRIL was maintained for 2–3 and ≥4 weeks with 80 and 240 mg, respectively, corresponding to reductions in serum Ig and antibody-secreting cells on May 20, 2023.",6
DBVT,Peanut allergy - ages 4 to 11 years,CRL,8/4/20,"CRL issued August 4, 2020",5
LLY,Type 2 diabetes,Approved,2/21/20,"FDA Approval announced February 21, 2020.",6
MITO,"Healthy Volunteers, Rare neurodegenerative diseases",Phase 1,11/1/22,"Phase 1 data reported that treatment is generally safe and well tolerated, noted November 1, 2022.",6
MOR,Chronic Lymphocytic Leukemia,Phase 2,6/15/18,Phase 2 presentation at EHA June 2018. ORR 82%. CR 9%.,6
SYBX,Phenylketonuria (PKU),Phase 2,3/19/23,"Phase 2 full data presentation at SIMD reported plasma Phe reduction of -53% among responders with a response rate was 60%, noted March 19, 2023.",6
DARE,Hormone replacement therapy,Phase 1/2,4/18/23,"Results of study published at the Menopause International Society demonstrated that the DARE-HRT1 IVRs, in general, were safe and well tolerated in healthy postmenopausal women and treatment emergent adverse events profiles were comparable between the DARE-HRT1 groups and the reference oral regimen group, noted April 18, 2023.",6
GPCR,Healthy Volunteers,Phase 1,5/23/23,"First-in-human data reported that pharmacokinetic exposure was generally dose proportional with minimal accumulation after once daily dosing with ANPA-0073 across the dose range, noted May 22, 2023.",5
AZN,Type 2 Diabetes / coronary artery disease (CAD),Approved,6/1/20,"FDA approval announced June 1, 2020.",6
BHVN,Migraine - preventative,Approved,5/27/21,"FDA approval announced May 27, 2021.",6
AZN,Relapsed/Refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL),Phase 1/2,12/12/22,"Phase 1b/2a results presented at ASH reported one CR and one PR in cohort 1, with three CR and two PR in cohort 2, noted December 12, 2022.",6
JNJ,Non- Small Cell Lung Cancer,Phase 3,10/24/23,"Phase 3 data presented at ESMO reported that PFS was significantly improved with ami-chemo (HR, 0.48; 95% CI, 0.36–0.64) and ami-laz-chemo (HR, 0.44; 95% CI, 0.35–0.56) vs chemo (median PFS, 6.3 and 8.3 vs 4.2 mo), noted October 24, 2023.",6
SNY,Influenza A - children 6-35 months of age.,Approved,11/4/19,sNDA FDA Approval announced for adults over 65 years of age.,6
NVAX,Respiratory Syncytial Virus (RSV) in older adults,Phase 3,9/15/16,"Phase 3 released September 15, 2016 did not demonstrate efficacy.",4
BMY,Solid tumors,Phase 1,10/7/21,"Phase 1 updated data noted a confirmed responses for a cORR of 36% out of the 11 MET TKI-naïve NSCLC patients, cORR of 33% for the nine MET TKI-naïve GC/GEJ patients, and among the 12 patients with advanced other solid tumors, one patient with MET amplified colorectal cancer achieved a confirmed response, noted October 7, 2021.",5
ADPT,Newly Diagnosed Multiple Myeloma,Phase 2a,12/10/22,"Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.",6
MRK,Myelodysplastic syndromes (MDS) cancer,Approved,4/3/20,"FDA Approval announced April 3, 2020.",6
INO,Cervical dysplasia,Phase 3,12/14/21,"Additional Phase 3 data reported that the safety profile observed at Week 36 remained well-tolerated through Week 88. In addition, participants who met the primary endpoint at Week 36 remained clear of HPV-16 and/or HPV-18 at Week 88, noted December 14, 2021.",6
BMY,First-line Non-small cell lung cancer (NSCLC),Approved,5/15/20,"FDA Approval announced May 15, 2020.",6
ADCT,Diffuse Large B-Cell Lymphoma,Phase 2,6/9/23,"Phase 2 data presented at EHA showed that 31% of patients who achieved a complete response remained event-free for two or more years and did not need new anticancer therapy, noted June 9, 2023.",6
ADMP,Opioid overdose,Approved,10/18/21,"Approved October 18, 2021.",6
SNY,Acquired thrombotic thrombocytopenic purpura (aTTP),Approved,2/6/19,"FDA Approval announced February 6, 2019.",6
PRQR,Usher Syndrome Type 2,Phase 1/2,3/24/21,"Phase 1/2 data released March 24, 2021. Mean benefit of 6 letters was observed at week 48",5
KALV,Hereditary angioedema (HAE),Phase 2,10/4/22,Phase 2 trial has been terminated based on the observation of liver enzyme (ALT/AST) elevations in multiple patients in all treatment groups of the trial and to prioritize the development of the KONFIDENT Phase 3 trial.,6
NWBO,Glioblastoma multiforme (GBM),Phase 3,11/17/22,"Phase 3 data reported that both median survival and ""Long Tail"" of extended survival were increased in both newly diagnosed and recurrent glioblastoma, noted November 17, 2022.",5
TMBR,Rosacea,Phase 2b,6/25/19,"Phase 2b data June 25, 2019 met endpoints.",6
SNY,Refractory Multiple Myeloma,Approved,3/31/21,"FDA approval announced March 31, 2021.",6
JNJ,Plaque psoriasis,Approved,7/13/17,"Approval announced July 13, 2017.",6
BMY,Relapsed/Refractory Multiple Myeloma (RRMM),Phase 1/2,12/11/22,"Additional data presented at ASH showed promising efficacy in a highly refractory patient population. As of the data cut-off date, mezigdomide plus DEX showed a manageable safety profile, noted December 11, 2022.",6
JNJ,GFR-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer,Phase 3,10/24/23,"Phase 3 data presented at ESMO reported ami+laz showed a 30% reduction in the risk for disease progression or death vs osi, with median PFS of 23.7 mo, and an ORR of 86%, noted October 24, 2023.",6
BMY,Urothelial cancer,Phase 2,4/14/22,"Phase 2 trial did not meet the efficacy threshold in its final ORR analysis, noted April 14, 2022.",6
TALS,Transplanted Organ Rejection,Phase 3,2/16/23,"Trial was discontinued due to the pace of enrollment and the associated timeline to critical milestones, noted February 16, 2023.",4
NVS,Hypercholesterolemia,Approved,12/22/21,"Approved December 22, 2021.",6
KURA,Head and neck squamous cell carcinomas (HNSCC) with HRAS Mutations,Phase 2,5/29/20,"Phase 2 median OS of 15.4 months, median PFS of 5.9 months and ORR of 50% observed in recurrent/metastatic HRAS mutant HNSCC",5
FGEN,Chemotherapy-induced anemia (CIA),Phase 2,8/25/21,Phase 2 positive topline results released August 25 2021.,6
MOLN,Solid Tumors,Phase 1,11/3/23,"Phase 1 data presented at SITC continues to demonstrate tumor-localized CD40 activation, leading to elevated immune activity in the tumor microenvironment, noted November 3, 202",6
PGEN,Ovarian cancer,Phase 1,6/5/23,"Phase 1 data presented at ASCO demonstrated expansion and persistence when delivered via either intraperitoneal or intravenous infusion without lymphodepletion or via intravenous infusion after lymphodepletion demonstrating the effectiveness of mbIL15, noted June 5, 2023.",6
CTMX,Breast cancer,Phase 2,7/6/22,"Phase 2 initial data reported that trial met its primary endpoint. Arm B did not pass protocol-defined futility boundary (ORR was less than 10%) in patients with advanced triple-negative breast cancer (TNBC) and enrollment into Arms B and C will be discontinued, noted July 6, 2022.",4
TAK,Narcolepsy,Phase 2,10/5/21,"Phase 2 trial stopped dosing and ended trial due to emergence of a safety signal, noted October 5, 2021.",4
BHC,Overt hepatic encephalopathy,Phase 2,3/31/20,"Phase 2 data met primary endpoint - March 31, 2020.",6
ICPT,Fibrosis due to nonalcoholic steatohepatitis (NASH),CRL,6/23/23,"Study to be discontinued, noted JUne 23, 2023.",4
BMY,RET-altered NSCLC,Phase 1/2,4/5/21,"Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.",5
TCON,Sarcomas,Phase 3,9/18/23,"Phase 2 pivotal trial satisfied more than the futility threshold of 3 responses out of 46 based on the results of the second and final mandated independent data monitoring committee (IDMC) efficacy review, and the trial will continue as planned, noted September 18, 2023.",5
GRTS,Non-small cell lung cancer (NSCLC) and colorectal cancer (CRC),Phase 2,9/30/22,"Phase 2 initial data presented at ESMO noted a 39% molecular response rate (MRR), noted September 2022.",6
NVS,Spinal muscular atrophy (SMA) Type 1,Approved,5/24/19,"FDA Approval announced May 24, 2019.",6
ENSC,Overdose Protection,Phase 1,5/9/23,"Phase 1 was completed with final part B data showing that a 25 mg dose of PF614-MPAR, delivered oxycodone as designed for what may be a prescribed dose of one to two capsules, noted May 9, 2023.",6
DARE,Bacterial Vaginosis,Approved,12/7/21,"FDA Approval on December 7, 2021.",6
CNTA,Hemophilia B,Phase 2b,10/31/23,"Phase 2b initiated, noted October 31, 2023.",5
REGN,High low-density lipoprotein (LDL) cholesterol,Approved,4/25/17,"sBLA Approval announced April 25, 2017.",6
PFE,Hemophilia A or B,Phase 3,11/2/23,"Phase 3 data from ASH abstract reported that OD group reported 12 (36.4%) adverse events (AEs) during ATP vs 9 (24.3%) in OP whereas the RP group reported 62 (74.7%) AEs in ATP vs 20 (22.0%) in OP, noted November 2, 2023.",5
SGMO,Fabry disease,Phase 1/2,5/22/23,"FDA fast-track designation granted on May 22, 2023.",5
AZN,Lupus,Phase 3,8/2/21,"FDA approval announced August 2, 2021.",6
REGN,Non-Hodgkin Lymphoma (NHL),Phase 2,12/12/22,"Phase 2 data presented at ASH showed a 49% ORR in heavily pre-treated patients who were naïve to prior CAR-T, with 31% achieving a complete response (CR), noted December 12, 2022.",6
VYNE,Acne,Approved,10/18/19,"FDA Approval announced October 18, 2019.",6
ALPMY,Geographic atrophy (GA) secondary to age-related macular degeneration (AMD),Phase 3,9/19/23,"Phase 3 24 month data confirmed that trial met primary endpoint, noted September 19, 2023.",5
JAZZ,Sickle Cell Disease-Related Acute Chest Syndrome,Phase 2,12/12/22,"Phase 2 data reported that among patients who underwent Day 30 CXR or CTA, 87.5% and 92%, respectfully, showed improvement or resolution of pulmonary consolidation, noted December 12, 2022.",6
IMNN,Ovarian cancer,Phase 1/2,9/28/23,"Phase 1/2 interim data reported efficacy trends in PFS, demonstrating a delay in disease progression in the treatment arm of approximately 33% compared with the control arm, with the hazard ratio nearing the required value. Preliminary OS data follows a similar trend, showing an approximate 9-month improvement in the treatment arm over the control arm, noted September 28, 2023.",6
TBPH,Hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP),Approved,6/21/13,"Approved June 21, 2013.",6
DTIL,Relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL),Phase 1/2,7/27/23,"Phase 1/2 Type B End of Phase 1 meeting with the FDA provided clarity and direction to advance to Phase 2, noted July 27, 2023.",6
PALI,Improvement in bowel function following cardiovascular surgery,Phase 2,5/2/22,"Phase 2 trial met primary endpoint, noted May 2, 2022.",5
ARDX,Constipation-predominant irritable bowel syndrome (IBS-C),Phase 3,5/12/17,"Phase 3 data released May 12, 2017. Primary endpoint met - however, competitive concerns raised.",5
AGIO,Sickle Cell Disease,Phase 2,12/13/21,"Phase 2 data demonstrated the potential to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling. No SAEs occurred, and all AEs were mild and mostly transient. One vaso-occlusive crisis (VOC) occurred without hospital admission and did not require dose reduction or discontinuation.",6
RETA,Pulmonary arterial hypertension associated with connective tissue disease - CTD-PAH,Phase 3,3/30/20,Phase 3 trial stopped following DSMB recommendation. Primary endpoint unlikely to be met.,4
LOGC,Methylmalonic Acidemia (MMA),Phase 1/2,2/2/22,"Phase 1/2 trial placed on clinical hold due to 2 drug related SAEs, noted February 2, 2022.",5
VIR,HIV T cell vaccine,Phase 1,9/20/23,"Phase 1 dosing initiated, noted September 20, 2023.",5
LLY,"Non-small cell lung cancer, medullary thyroid cancer and thyroid cancer.",Approved,5/8/20,"FDA Approval announced May 8, 2020.",6
PPBT,Osteoarthritis and Hypertension,Approved,5/31/18,"Approval announced May 31, 2018.",6
SCYX,Candida Auris,Phase 3,3/2/21,"Phase 3 interim analyses noted 8/10 complete responses - March 2, 2021.",6
RHHBY,Paroxysmal nocturnal hemoglobinuria (PNH),BLA Filing,9/6/23,"BLA accepted by FDA, noted Septemeber 6, 2023",5
OCUP,Mydriasis in patients aged 3-11,Phase 3,4/28/22,"Phase 3 top-line data reported that trial met primary endpoint, noted April 28, 2022.",5
VRDN,Active Thyroid Eye Disease (TED),Phase 1/2,7/10/23,"Phase 1/2 preliminary data demonstrated clinically meaningful and rapid improvement in signs and symptoms of chronic TED at week 6 after receiving two infusions of VRDN-001 10 mg/kg or 3 mg/kg, noted July 10, 2023.",6
LGND,Multiple Myeloma,Approved,10/1/18,"FDA approval announced October 1, 2018.",6
REGN,Non-small cell lung cancer (NSCLC),Phase 2,10/31/23,"Phase 2 preliminary efficacy data reported a 100% Disease Control Rate (DCR), noted October 31, 2023.",5
SUPN,Impulsive Aggression in ADHD,Phase 3,11/5/19,"Phase 3 trial did not meet primary endpoint - November 5, 2019.",4
SGMO,Sickle Cell Disease,Phase 1/2,12/13/21,"Phase 1/2 data reported that total hemoglobin stabilized by Week 26 after treatment in all four patients. Fetal hemoglobin level increased from 0.1-11% at screening to 14-39% in all four patients and was 38% in the longest-treated patient at 91 weeks, noted December 13, 2021.",6
BMY,First-line Non-small cell lung cancer (NSCLC),Approved,5/26/20,"FDA Approval announced May 26, 2020.",6
PTGX,Hereditary hemochromatosis,Phase 2,11/15/21,"Phase 2 data released November 15, 2021. Phase 2 trial met primary endpoint, rusfertide-treated patients had 0.009 phlebotomies per month during the study compared to 0.28 phlebotomies per month pre-study. The average TSAT during treatment was 31.4% compared to 45.0% pre-study and the serum iron was 101 mcg/dl compared to 137 mcg/dl.",6
OMER,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 1b,11/2/23,"Phase 1b data from ASH abstract reported that mean Hgb increased from baseline by 3.1 g/dL at 4 weeks, and by 9.5 g/dL at the latest timepoint of 24 weeks, noted November 2, 2023.",5
CELC,ER+/HER2- Breast Cancer,Phase 1b,5/12/23,"Phase 1b data shared at ESMO Breast Cancer reported that for treatment-naïve patients median PFS was 45.8 months and 48.6 months in the expansion arm, noted May 12, 2023.",6
SVRA,Heart failure with preserved ejection fraction (HFpEF),Phase 2,3/11/18,"Phase 2 top line data presented March 11, 2018 failed to meet primary endpoint.",4
OCGN,COVID-19 vaccine,Phase 3,7/2/21,Phase 3 data from partner Bharat Biotech noted efficacy rate of 78%; 93% against severe disease; 65% against Delta variant,6
NKTR,Solid tumors,Phase 1/2,3/1/19,"Phase 1/2 initial data March 1, 2019 noted ORR 2/11 ( 18%) - maximum tolerated dose has not been reached.",4
IPSEY,Glabellar and lateral canthal lines,CRL,10/3/23,"CRL received October 3, 2023.",5
PFE,ER+ positive / HER2- negative breast cancer,Phase 1b,12/13/21,"Phase 1 dose escalation data reported that treatment was well tolerated with no DLTs at total daily doses up to 700mg, noted December 13, 2021.",6
EVFM,Chlamydia and gonorrhea,Phase 3,10/12/22,"Phase 3 trial did not meet its primary endpoint, noted October 12, 2022.",6
RETA,Chronic Kidney Disease,Phase 2,5/10/23,"Program discontinued, noted May 10, 2023.",4
RDHL,H. pylori,Approved,9/18/23,https://www.biopharmcatalyst.com/company/RDHL/news/159168,5
RHHBY,Wet age-related macular degeneration (nAMD) and diabetic macular edema (DME),Approved,1/31/22,"Approved January 31, 2022.",6
SRNE,Pain associated with post-herpetic neuralgia (PHN),Approved,2/28/18,"Approval announced February 28, 2018.",6
INVA,Increase blood pressure in adults with septic or other distributive shock,Approved,12/21/17,"Approval announced December 21, 2017.",6
ARDX,Constipation-predominant irritable bowel syndrome (IBS-C),Approved,9/12/19,"FDA approval announced September 12, 2019.",6
RETA,Mitochondrial myopathies (MM),Phase 2,3/1/18,"Phase 2 initial data released March 1, 2018 - endpoints not met.",4
CANF,Pancreatic Cancer,Phase 1a,6/13/23,"Preclinical studies demonstrated robust effect reached 90% inhibition of pancreatic cancer growth, IND underway, noted June 13, 2023.",6
ATAI,Cognitive Impairment Associated with Schizophrenia,Phase 2a,12/14/21,"Full Phase 2a results reported dose-related improvements on exploratory cognitive endpoints. A dose dependent response in qEEG was observed, with the greatest increases seen in 20mg and 40mg doses of RL-007, noted December 14, 2021.",6
REVB,H3N2 Influenza,Phase 2b,3/30/22,"Phase 2b trial did not meet primary efficacy endpoint, noted March 30, 2022.",6
AMGN,B-Cell Acute Lymphoblastic Leukemia (B-All),Approved,6/21/23,"FDA Approval on June 21, 2023.",6
BIIB,Multiple sclerosis (MS),Approved,10/30/19,"FDA Approval announced October 30, 2019.",6
JAZZ,Acute myeloid leukemia,Approved,3/30/21,FDA approval for label extension to include children.,6
PTCT,Duchenne muscular dystrophy caused by nonsense mutations (nmDMD),Phase 3,6/21/22,"Trial data reported a significant benefit across 6MWD, NSAA and Timed function test endpoints, with no adverse events leading to discontinuation, noted June 21, 2022. NDA planned.",4
BMY,Mesothelin-positive solid tumors,Phase 1/2,9/28/22,"Phase 1 translational data demonstrated tolerability and clinical benefit. Second RECIST PR in ovarian cancer supports broad potential of treatment, consistent tumor regression in 93% evaluable patients with DCR of 77%. PFS of 5.6 months and overall survival of 11.2 months suggest durability of benefit in mesothelioma. Phase 2 portion of trial underway, noted September 28, 2022.",6
AMYT,Acromegaly,Approved,6/26/20,"FDA approval announced June 26, 2020.",6
PLX,Gaucher disease,Approved,5/1/12,"Approved May 1, 2012.",6
URGN,Urothelial Carcinoma,Approved,4/15/20,"FDA Approval announced April 15, 2020.",6
MRK,Small cell lung cancer (SCLC),Phase 3,1/6/20,Phase 3 data met PFS primary endpoint; overall survival primary endpoint not met,4
ACOR,OFF episodes of Parkinson’s disease (PD),Approved,12/24/18,"FDA Approval announced December 22, 2018.",6
TNXP,Pediatric obesity related binge eating disorder,Phase 2,7/10/23,"Phase 2 trial initiated, noted July 10, 2022.",5
TXMD,Contraception,Approved,8/10/18,"FDA Approval announced August 10, 2018.",6
LEXX,Cardiovascular disease (CVD),Phase 1,5/23/23,"Trial data reported that trial met primary endpoint and five weeks of treatment with the patented DehydraTECH-processed cannabidiol (CBD) capsule formulation, blood-plasma levels of interleukin (IL) 8, 10, and 18 were reduced by ~19%, ~27%, and ~43%, respectively, noted May 23, 2023.",6
ATAI,Treatment Resistant Depression,Phase 1,10/2/23,"Phase 1 data reported that treatment was well-tolerated with a favorable safety profile, and dose-dependent increases in exposure were observed, noted October 02, 2023.",6
IRWD,Hyperuricemia associated with gout,Approved,8/21/17,"Approval announced August 21, 2017.",6
BIVI,Ascites,Phase 2b,3/13/23,"Phase 2b data reported that treatment resulted in a 34% reduction in ascites fluid during the 28 days after treatment initiation compared to the 28 days prior to treatment, noted March 13, 2023.",6
XOMA,CV risk reduction,CRL,10/18/18,"CRL announced October 18, 2018.",5
SGEN,Previously untreated high risk classical Hodgkin lymphoma (cHL),Approved,11/10/22,"Approved November 10, 2022.",6
PFE,Focal Epilepsy,Phase 1,9/5/23,"Examination of EEG in healthy subjects administered single doses confirmed central nervous system (CNS) activity consistent with effects observed with other antiseizure medications, noted September 5, 2023.",6
BGNE,Esophageal Squamous Cell Carcinoma (ESCC),Phase 2,10/21/23,"Phase 2 data presented at ESMO reported that INV-assessed ORR was 30.6% with O+T vs. 20.6% with P+T; hazard ratio (HR) of INV-assessed PFS was 0.93, noted October 21, 2023.",6
AMGN,Obesity and without diabetes,Phase 1,12/1/22,"Phase 1 results presented at the 20th World Congress of Insulin Resistance, Diabetes and Cardiovascular Disease (WCIRDC) showed up to 14.5% reduction in body weight at the highest dose after 12 weeks, noted December 1, 2022.",6
CNTB,Ulcerative Colitis (UC),Phase 2b,6/1/23,"Phase 2b maintenance data reported that 57% (12/21) of patients with clinical response at the end of the induction period achieved clinical remission at the end of the maintenance period, noted June 1, 2023.",6
TAK,Hepatocellular Carcinoma,Phase 3,3/14/22,"Phase 3 final OS data reported no improvement, noted March 14, 2022.",6
IDYA,Metastatic uveal melanoma (MUM),Phase 1,12/7/21,"Phase 1 data showed 100% Disease Control Rate (DCR) with (16/16) evaluable patients with >1 post-baseline scan showed tumor shrinkage as determined by target lesion size reduction , 31% ORR, 46% of patients (6/13) with > 2 post-baseline scans observed >30% tumor reduction, including one patient with an unconfirmed PR, December 7, 2021.",6
UNCY,Hyperphosphatemia,Phase 1,12/28/22,"Trial met primary endpoint, noted December 28, 2022",5
EVLO,Atopic dermatitis,Phase 2,4/26/23,"Phase 2 trial did not meet its primary endpoint, noted April 26, 2023.",6
RHHBY,Triple-negative breast cancer,Approved,3/8/19,"FDA Approval announced March 8, 2019.",6
PDSB,Metastatic Prostate Cancer,Phase 1,10/11/23,"First in human safety and immune data presented at Cytokines reported a decrease in PSA levels, and that all doses of the combination were well-tolerated with one patient experiencing Grade 4 neutropenia, noted October 11, 2023.",6
RHHBY,Relapsed or refractory multiple myeloma,Phase 3,9/29/23,"Phase 3 data reported that it showed improvement in median PFS of 9.9 months compared to 5.8 months with the combination of study comparator pomalidomide and dexamethasone; however, the results did not reach statistical significance, noted September 29, 2023.",6
HALO,Testosterone replacement therapy (TRT),Approved,3/29/22,"Approved March 29, 2022.",6
ACER,Vascular Ehlers-Danlos Syndrome,CRL,6/25/19,"CRL issued June 25, 2019.",5
MRK,Localized muscle-invasive urothelial carcinoma (MIUC),Phase 3,10/5/23,"Phase 3 trial met one of its dual primary endpoints of disease-free survival (DFS), noted October 5, 2023.",5
RIGL,IgA nephropathy,Phase 2,4/3/18,"Phase 2 data released April 3, 2018. Primary endpoint not met.",4
EVO,High adenosine signature cancers,Phase 1,4/19/23,"Phase 1 objectives were achieved in a healthy volunteer study, confirming pharmacokinetics, pharmacodynamics, safety, and tolerability, allowing selection of a starting dose for the ongoing IGNITE Phase 1/2 study ​​in combination with a PD-1 inhibitor in patients with RCC and NSCLC, noted April 19, 2023",6
NCNA,2L Colorectal cancer,Phase 2,10/13/23,"Phase 2 patients recruiting well with no new safety signals, noted October 13, 2023.",5
BBIO,Cholangiocarcinoma,Approved,6/1/21,"FDA approval announced June 1, 2021.",6
ATOS,Male breast cancer and Gynecomastia,Phase 1,9/13/18,"Phase 1 preliminary data September 13, 2018 noted drug met safety and tolerability objectives.",6
LIAN,Ulcerative Colitis (UC),Phase 1b,8/3/22,"Phase 1b top-line data reported that no serious adverse events (SAEs) were reported, noted August 3, 2022.",5
AVTX,Major depressive disorder (MDD),Phase 2,11/29/16,"Phase 2 top-line data released November 29, 2016 failed to meet primary endpoint.",4
AZN,Epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC),Approved,3/31/17,"Approval announced March 31, 2017.",6
RHHBY,Follicular lymphoma,Approved,12/23/22,"Approved December 23, 2022.",6
APRE,Solid tumors,Phase 1/2,10/16/23,"Phase 1/2a dose expansion cohort being on track to be initiated in 2Q 2024, additionally, data presented at AACR-NCI-EORTC reported that no dose-limiting toxicities have been reported to date with persistent tumor-reducing effect, noted October 16, 2023.",6
VTYX,Plaque psoriasis,Phase 1,8/15/22,"Phase 1 data reported that treatment was well-tolerated with a dose-dependent increase in expsoure and target coverage, noted August 15, 2022.",6
BMY,Non-Small Cell Lung Cancer (NSCLC),Phase 3,4/11/22,"Phase 3 results reported that treatment reduced the risk of disease recurrence, progression or death by 37%, noted April 11, 2022.",6
STTK,Acute Myeloid Leukemia (AML) and Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS),Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that in 4 evaluable pts with UnTx TP53m-MDS, the ORs were 1 complete remission (CR), 1 marrow CR, and 2 stable disease. 2 of them proceeded to allo-HCT, noted November 2, 2023.",6
PFE,Ulcerative colitis,Approved,10/13/23,"FDA Approved on October 13, 2023.",6
BNTX,COVID-19 vaccine,Approved,6/17/22,"EUA approval for children 4 years - 6 months, noted June 17, 2022.",6
MRK,Biliary Tract Cancer,Approved,11/1/23,"Approved November 1, 2023.",6
AIM,Colorectal cancer,Phase 2,4/11/22,"Phase 2 initial results presented at AACR reported that the primary endpoint was met, noted April 11, 2022.",6
REGN,Polymyalgia rheumatica (PMR),Approved,2/28/23,"Approved February 28, 2023.",6
ORTX,MPS-IIIA,Phase 1/2,5/19/23,"Initial data shared at ASGCT reported that four out of five patients continued to gain cognitive skills in line with development in healthy children, noted May 20, 2023.",5
RHHBY,Acute uncomplicated influenza (12 years and older),Approved,11/23/20,"FDA approval announced November 23, 2020.",6
NBIX,Tardive dyskinesia (TD),Phase 3,8/29/23,"Phase 3 data reported that the mean AIMS total score at baseline was 14.6 and decreased over 48 weeks, with a mean change from baseline of -10.2 , noted August 29, 2023.",5
PFE,Transthyretin amyloid cardiomyopathy (ATTR-CM),Approved,5/6/19,"FDA approval announced May 6, 2019.",6
TEVA,Inflammatory conditions,CRL,10/12/23,"CRL received October 12, 2023.",5
OYST,Dry eye disease,Approved,10/18/21,"Approved October 18, 2021.",6
AMTI,Ulcerative colitis,Phase 2,12/23/22,"Phase 2 data at week 12 resulted in lower clinical remission (CR) rate in patients treated was 17.1% (12/70 patients) compared to a CR rate of 20.0% (7/35 patients) with placebo, noted December 23, 2022.",6
NVS,Relapsed/Refractory B-Cell Acute lymphoblastic leukemia,Approved,8/30/17,"PDUFA date under priority review announced March 29, 2017. Approval announced early - August 30, 2017.",6
GBT,Sickle cell disease,Approved,11/25/19,"FDA Approval announced November 25, 2019.",6
ZYME,Gastric/gastroesophageal junction adenocarcinoma (G/GEJC),Phase 1/2,10/20/23,"Phase 1b/2 data presented at ESMO reported that the confirmed objective response rate by investigator (INV) was 75.8% and median progression-free survival was 16.7 months, noted October 20, 2023.",6
INO,Cervical high-grade squamous intraepithelial lesions (HSIL),Phase 3,3/1/23,"Phase 3 trial did not achieved in the investigational biomarker-selected population for the endpoint of lesion regression and viral clearance, noted March 1, 2023.",6
BMRN,Hemophilia A,Phase 1/2,5/31/22,"Phase 1/2 5 year data reported that the mean ABR for the 4e13 vg/kg cohort was 0.7 with a mean cumulative ABR reduction of 91%, noted May 31, 2022.",6
ARGX,Chronic inflammatory demyelinating polyneuropathy (CIDP),Phase 2,7/17/23,"Phase 2 trial met primary endpoints, noted July 17, 2023.",5
NBTX,Pancreatic cancer,Phase 1,9/28/23,"Phase 1 data reported a 92.3% local disease control rate (12/13) and a median Overall Survival of 21 months, noted September 28, 2023.",5
MRK,Ovarian cancer,Phase 1,10/23/23,"Phase 1 data presented at ESMO reported that the confirmed overall response rate in patients with measurable disease was 38% (13 of 34): 67% (including 1 CR) at 4.8 mg/kg, 33% (5/15) at 6.4 mg/kg, and 31% (4/13) at 8.0 mg/kg, with two patients with unconfirmed partial responses were still on treatment, noted October 23, 2023.",6
BIIB,Multiple sclerosis (MS),Phase 3,10/13/21,"Phase 3b data displayed that the proportion of patients with No Evidence of Disease Activity (NEDA) was 70.0 percent for Q6W and 67.4 percent for Q4W. The proportion of patients free of disability worsening was 90.0 percent in the Q6W arm and 92.0 percent in the Q4W arm. Time to first relapse with the proportion who were relapse-free at 72 weeks at 96.9 percent for Q6W and 97.6 percent for Q4W, noted October 13, 2021",6
ONCY,HR+ / HER2- breast cancer,Phase 2,6/5/23,"Phase 2 data readout at ASCO reported that nine pts (33%) discontinued pelareorep and 6 (35%) discontinued avelumab due to toxicity, noted June 5, 2023.",4
BGNE,Gastric/gastroesophageal junction adenocarcinoma (G/GEJC),Phase 1/2,10/20/23,"Phase 1b/2 data presented at ESMO reported that the confirmed objective response rate by investigator (INV) was 75.8% and median progression-free survival was 16.7 months, noted October 20, 2023.",6
SCYX,Invasive candidiasis,Phase 3,10/24/22,"Additional Phase 3 cumulative interim analysis presented at IDWeek showed 82.3% positive clinical outcomes in patients treated with treatment, all-cause mortality analysis showed 94.6% survival 30 days post-therapy in patients with invasive candidiasis or candidemia who were treated.",6
IONS,Familial Chylomicronemia Syndrome (FCS),Phase 3,9/26/23,"Phase 3 trial met its primary efficacy endpoint, noted September 26, 2023.",5
VRTX,Alpha-1 antitrypsin (AAT) deficiency,Phase 2,6/10/21,"Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.",4
RCKT,Leukocyte Adhesion Deficiency-I (LAD-I),Phase 1/2,5/19/22,"Phase 2 top-line data reported a 100% survival rate at 12-months post-infusion, with statistically significant reduction in the rate of all-cause hospitalizations and severe infections, noted May 19, 2022.",6
XENT,Recurrent sinus obstruction,Approved,12/11/17,"Approval announced December 11, 2017.",6
COLL,"Management of pain severe enough to require daily, around-the-clock, long-term opioid treatment for which alternative treatment options are inadequate",Approved,10/26/15,"Approved October 26, 2015.",6
ATXS,Duchenne muscular dystrophy (DMD),Phase 3,10/26/20,"Phase 3 trial did not meet primary endpoint - October 26, 2020.",4
MRSN,HER2 - breast cancer,Phase 1,7/19/18,"Phase 1 partial clinical hold due to patient death announced July 19, 2018.",4
SNY,Cardiovascular events,Approved,4/26/19,"FDA approval announced April 26, 2019.",6
INMB,Neuroinflammation caused by Alzheimer’s disease,Phase 2,10/24/23,"Phase 2 presentation at CTAD reported that XPro was associated with dose-dependent improvement in neurology-related proteins, noted October 24, 2023.",6
JNJ,"Tablet reduction, non-metastatic castration-resistant prostate cancer (nmCRPC)",Approved,4/3/23,"Approved April 3, 2023.",6
ANIP,Osteoporosis,Approved,5/15/23,"ANDA approved May 15, 2023.",6
RNA,Myotonic dystrophy type 1 (DM1),Phase 1/2,10/9/23,"Phase 1/2 enrollment into open label extension trial commenced August 2, 2022. Phase 1/2 new data demonstrated improvement in additional functional measures augmenting previously reported positive data that demonstrated improvements in functional assessments of myotonia (video hand opening time, or vHOT), strength (Quantitative Muscle Testing total score, or QMT) and mobility (10-meter walk run test, or 10mWRT and the Timed Up and Go test, or TUG)., noted October 9, 2023.",6
LLY,Alzheimer's Disease,CRL,1/20/23,"CRL received for accelerated approval application, noted January 20, 2023.",6
ALT,"Non-alcoholic steatohepatitis (NASH) - Healthy, Overweight and Obese Volunteers",Phase 2,3/21/23,"Phase 2 interim 24-week data data showed that approximately 50% of subjects achieved 10% or more weight loss and approximately 20% of subjects achieved 15% or more weight loss at the 1.8 mg and 2.4 mg doses at Week 24. Treatment discontinuation rates were 28.2% in subjects receiving placebo and 24.0% in subjects receiving pemvidutide, noted March 21, 2023.",6
SNY,COVID-19 vaccine,Phase 3,12/15/21,"Phase 3 data reported that the vaccine is well tolerated, with a safety profile similar to currently approved COVID-19 vaccines, noted December 15, 2021.",6
MRNA,Covid-19 Omicron Booster,Approved,9/11/23,"FDA approved on September 11, 2023.",6
MRK,Esophageal Cancer,Approved,3/23/21,"FDA approval announced March 23, 2021.",6
ALKS,Major depressive disorder,CRL,2/1/19,"CRL announced February 1, 2019.",5
TCRT,Solid tumors,Phase 1,6/18/18,"Phase 1 trial placed on clinical hold - June 18, 2018.",4
RHHBY,Duchenne muscular dystrophy (DMD),Phase 1b,10/11/21,"Phase 1b open label interim results from the first 11 participants in Cohort 1 (ambulatory boys aged 4-7 years) provided evidence of robust expression of micro-dystrophin and no new safety signals were identified, September 24, 2021. Functional results from study cohort 1 (n=11, ages 4-7) found participants improved 3.0 points on NSAA six months after treatment - noted October 11, 2021.",6
BPMC,Advanced Systemic mastocytosis (SM),Phase 1,12/12/22,"Phase 1 data reported that 17 (25%) patients had disease progression, including 7 who progressed to acute myeloid leukemia. Median OS was NR in all AdvSM, ASM, and MCL, and was 46.9 months, noted December 12, 2022.",5
AMGN,Plaque psoriasis,Phase 3,4/18/22,"Phase 3 data reported that trial met primary endpoint, noted April 18, 2022.",5
SXTP,COVID-19 patients with mild-moderate symptoms and low risk of disease progression,Phase 1a,9/18/23,"IND withdrawn, noted September 18, 2023",5
GILD,Psoriatic arthritis,Phase 2,5/30/18,"Phase 2 data released May 30, 2018 - primary endpoint met.",6
EBS,Opioid overdose,Approved,3/29/23,"Approved March 29, 2023.",6
BLCM,Metastatic castration-resistant prostate cancer (mCRPC),Phase 1/2,12/6/21,"Phase 1/2 data reported no dose-limiting toxicities, and 1 confirmed partial response, noted December 6, 2021.",6
ACIU,Alzheimer’s disease,Phase 2,9/23/20,"Phase 2 top-line data did not meet endpoints - September 23, 2020.",4
PFE,Transthyretin amyloid cardiomyopathy (ATTR-CM),Approved,5/6/19,"FDA approval announced May 6, 2019.",6
TAK,Advanced hepatocellular cancer (HCC),Approved,1/14/19,"FDA Approval announced January 14, 2019.",6
AVXL,Rett syndrome,Phase 2,6/28/23,"Phase 2 long-term clinical study results demonstrated disease modifying effect of ANAVEX2-73 (blarcamesine) for adult patients with Rett syndrome, noted June 28, 2023.",6
FRTX,Atopic dermatitis,Phase 1,3/7/23,"Phase 1 topline results reported that treatment was well tolerated and plasma concentrations within the potential therapeutic dose range were consistent with efficacious exposure levels established in nonclinical disease models, noted March 7, 2023.",6
SCNI,Universal flu vaccine,Phase 3,10/23/20,"Phase 3 trial did not meet endpoints - October 23, 2020.",4
TCDA,Chronic Kidney Disease,Phase 3,10/24/22,"Phase 3 top-line data reported that trial did not meet its primary endpoint, noted October 24, 2022.",6
ELTX,Solid Tumors,Phase 1/2,11/3/23,"Phase 1/2 data presented at SITC reported that 87% (20/23) had direct ex vivo mKRAS-specific T cell responses, and 100% (23/23) had in vitro stimulated responses, noted November 3, 2023.",6
RVMD,Solid tumors,Phase 1b,1/11/22,"Phase 1/1b data reported one patient exhibiting a confirmed partial response with a 63% reduction from baseline and the other three with stable disease, noted January 11, 2022.",6
BIVI,Alzheimer’s disease,Phase 3,10/25/23,"Phase 3 presented at CTAD showed statistically significant population changes from baseline were observed for all primary and secondary cognitive and functional assessments measured: ADAS-Cog12, ADCS-CGIC, MMSE, CDR, CDR-SB, ADCOMS, and ADL, noted October 25, 2023.",6
SRPT,Duchenne muscular dystrophy,Approved,12/12/19,"FDA Approval announced December 12, 2019.",6
GMDA,Hematologic Malignancies,Approved,4/17/23,"FDA Approved on April 17, 2023.",6
OCUP,Presbyopia,Phase 2,5/2/22,"Phase 2 efficacy data reported that 12 or more hours after treatment, 30% of subjects in the POS arm and 14% in the placebo arm had a ≥ 15 letter binocular DCNVA improvement, noted May 2, 2022.",6
BMY,Follicular lymphoma,Phase 2,6/17/23,"Phase 2 data presented at ICML showed that 97% of patients with R/R FL treated with Breyanzi achieved a response, with 94% achieving a complete response, and 81.9% of responders in ongoing response at 12 months, noted June 17, 2023.",6
ACAD,Alzheimer’s disease psychosis (ADP),CRL,8/5/22,"CRL announced August 5, 2022.",5
AMGN,Atopic dermatitis,Phase 2,10/2/21,"Phase 2 study met the primary objective, showing statistically greater improvements in EASI score at 16 weeks with all four subcutaneous doses compared with placebo, noted October 2, 2021",6
CLNN,Multiple Sclerosis,Phase 2,8/5/21,"Phase 2 trial met primary endpoint - August 5, 2021.",6
ABBV,Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL),Approved,5/19/23,"Approved May 19, 2023.",6
AMGN,Alopecia areata,Phase 2,9/13/23,"Phase 2 initiated in collaboration with Q32 Bio on September 13, 2023.",5
ASRT,Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma,Approved,3/15/16,Approved March 15 2016.,6
ARDS,Ventilator associated pneumonia (VAP) / Staphylococcus aureus,Phase 3,6/20/23,"FDA granted Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD), noted June 20, 2023. The official determination regarding using the LPAD pathway will be made after the Company's request following the filing of the biologics license application (BLA).",4
NAVB,Lymphatic-tissue tracing agent,Approved,3/13/13,"Approved March 13, 2013.",6
AMGN,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 3,8/23/22,"Phase 3 trial met its primary endpoint, noted August 23, 2022.",5
OTIC,Tinnitus,Phase 1/2,10/12/21,"Phase 1/2 clinical trial results published in Otology & Neurology, a journal in otolaryngology demonstrated that the formulation was well-tolerated and a statistically significant higher proportion of responders than placebo across consecutive visits (Weeks 4 and 8).",6
ITCI,Heart failure,Phase 1/2,6/29/20,"Phase 1/2 top-line data released June 29, 2020 noted improved cardiac output by increasing heart contractility and decreasing vascular resistance.",6
GHRS,Treatment-Resistant Depression (TRD),Phase 2,12/6/21,"Phase 2 trial met primary and secondary endpoints, noted December 6, 2021.",6
TIL,"Non-small cell lung cancer (NSCLC), ovarian cancer, and renal cell carcinoma (RCC)",Phase 1,1/9/23,"Phase 1 prioritized with initial data expected 2023. Phase 1 dosing initiated, noted October 18, 2022. Phase 2 enrollment voluntarily paused due to manufacturing issues, noted October 31, 2022. Phase 2 reopened, noted January 9, 2023.",4
ONCS,Melanoma - cancer,Phase 2b,11/9/20,"Phase 2b interim data presented at SITC November, 2020. 20% ORR.",5
REGN,Non-small cell lung cancer (NSCLC),Phase 3,4/27/20,"Phase 3 trial stopped early due to significant improvement in overall survival - April 27, 2020.",6
PRTC,Clostridioides difficile infection (CDI),Phase 2,5/9/23,"Phase 2 data reported that the high dose arm had an acceptable safety profile and compared with placebo, reduced the odds of developing rCDI through Week 8 by over 80%, noted May 9, 2023.",6
AZN,Metastatic breast cancer,Phase 3,6/12/23,"Phase 3 trial results showed the combination reduced the risk of disease progression or death by 40% vs. Faslodex alone, noted June 12, 2023",6
CLSD,Diabetic retinopathy,Phase 2,8/1/23,"Phase 2 additional data reported that 20% of patients achieved a >2-step improvement vs. 10% in control and 54% of patients achieved any DRSS improvement vs. 20% in control, noted August 1, 2023.",6
PRVB,Crohn's disease,Phase 2,10/22/19,"Phase 2a data did not meet primary endpoint - October 22, 2019.",4
PLRX,Idiopathic pulmonary fibrosis (IFP),Phase 2a,9/7/21,"Phase 2a PET positive interim data results released September 7, 2021.",6
LPCN,Liver cirrhosis,Phase 2,7/27/23,"Phase 2 trial met its primary endpoint, noted July 27, 2023.",5
IVVD,COVID-19 Prevention,Phase 3,9/11/23,"Phase 3 dosing initiated, noted September 11, 2023.",5
CYCN,"Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)",Phase 2,6/28/22,"Phase 2 data reported that treatment was well tolerated, with no reports of serious adverse events (SAEs) or treatment discontinuation due to adverse events (AEs), and showed improvements across multiple mitochondrial disease-associated biomarkers, inflammatory biomarkers, cerebral blood flow, and functional connectivity between neural networks, noted June 28, 2022.",6
NVO,Obesity,Phase 3,5/13/20,"Phase 3 data met primary endpoint - May 13, 2020.",6
CGEN,Ovarian Cancer,Phase 1,11/7/22,"Preliminary Phase 1 data reported a 12% ORR in 3L+ MSS-CRC patients with liver metastases, noted November 7, 2022.",5
ARWR,Homozygous Familial Hypercholesterolemia (HoFH),Phase 2,5/23/23,"Phase 2 interim data from two doses reported mean reductions in LDL-C (Martin-Hopkins) of 48.1% and 44.0%, mean reductions in ApoB of 39.2% and 34.5%, mean reductions in ANGPTL3 of 82.7% and 80.1%, noted May 23, 2023.",6
MRK,Prevention HPV-Related Head and Neck Cancers,Approved,6/12/20,"FDA Approval announced June 12, 2020.",6
AZN,"Hormone Receptor Positive Breast Cancer, Triple Negative Breast Cancer and Non-small Cell Lung Cancer",Phase 1,12/9/22,"Phase 1 data reported an objective response rate (ORR) of 27% with all patients achieving a partial response and 56% with stable disease, noted December 9, 2022.",6
BHVN,Glioblastoma (GBM),Phase 2/3,10/31/23,"Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.",5
RHHBY,Granulomatosis with polyangiitis (GPA) / microscopic polyangiitis (MPA),Approved,9/27/19,"FDA Approval announced September 27, 2019.",6
AMRX,Acute symptomatic hypocalcemia in pediatric and adult patients,Approved,9/6/23,"FDA ANDA Approved based on FDA's Competitive Generic Therapy (CGT) designation with 180-day exclusivity on September 6, 2023.",6
TVTX,Pantothenate kinase-associated neurodegeneration (PKAN),Phase 3,8/22/19,"Phase 3 top-line data did not meet endpoints - August 22, 2019.",4
DSGN,Friedreich ataxia (FA),Phase 1,8/14/23,"Phase 1 ascending dose data reported that DT-216 was generally well-tolerated and achieved a statistically significant and dose-related increase in frataxin (FXN) mRNA levels in skeletal muscle biopsies, noted August 14, 2023.",6
PLRX,Idiopathic Pulmonary Fibrosis (IPF),Phase 2a,5/24/23,"Phase 2a additional f\data presented at ATS 2023 showed that bexotegrast was well tolerated across all four dose groups with bexotegrast-treated participants experiencing a reduction in forced vital capacity (FVC) decline over 12 weeks versus placebo, on and off background therapy., noted May 24, 2023.",6
MLYS,Hypertension,Phase 2,11/2/23,"Phase 2 data to be presented ASN further supports obesity-associated dysregulated aldosterone as an endotype predictive of enhanced response, noted November 2, 2023.",6
SNY,Non-small cell lung cancer (NSCLC),Phase 3,4/27/20,"Phase 3 trial stopped early due to significant improvement in overall survival - April 27, 2020.",6
GLPG,Ulcerative colitis,Phase 2/3,5/20/20,"Phase 2b/3 data released May 20, 2020. 200mg dose met primary endpoint. 100mg did not meet clinical remission endpoint.",5
RIGL,Acute myeloid leukemia,Approved,12/1/22,"Approved December 1, 2022.",6
GMAB,Relapsed or Refractory (R/R) Multiple Myeloma (MM).,Phase 1/2,12/12/22,"Phase 1/2 preliminary data reported that treatment was well tolerated with no treatment related deaths or dose-limiting toxicities, noted December 12, 2022.",6
WVE,Huntington’s disease,Phase 1/2,3/29/21,Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped,4
NVS,Severe aplastic anemia (SAA),Approved,11/16/18,"sNDA approval announced November 16, 2018.",6
AFMD,EGFR-expressing solid tumors,Phase 1/2,6/3/23,"Phase 1/2 interim data presented at ASCO reported that 7 out of 15 heavily pre-treated patients with tumor reductions, including 2 confirmed partial responses and 5 patients exhibiting stable disease, noted June 3, 2023.",6
BMY,"First-line, lower-risk Myelodysplastic syndromes (MDS)",Approved,8/28/23,"Approved August 28, 2023.",6
MRK,Cancer - Third-line Gastric or Gastroesophageal Junction Adenocarcinoma,Phase 3,12/14/17,"Approved Sept. 22, 2017 under accelerated approval in patients undergoing third-line treatment following data from Phase 2 Keynote-59 trial. Phase 3 data released December 14, 2017 in patients undergoing second-line treatment, did not meet the primary endpoint of overall survival, nor did it show a significant improvement in PFS.",4
CYDY,HIV,BLA Filing,7/13/20,"Refusal to file letter announced July 13, 2020.",5
REGN,Atopic dermatitis in 6 months - 5 year-olds,Approved,6/7/22,"Approved June 7, 2022.",6
AVEO,Third line treatment of patients with renal cell cancer,Approved,3/10/21,"FDA approval announced March 10, 2021.",6
JNJ,Urothelial cancer,Approved,4/12/19,"FDA Approval announced April 12, 2019.",6
BMY,Urothelial Carcinoma,Approved,8/20/21,"FDA approval announced August 20, 2021.",6
JAZZ,Cataplexy and Excessive Daytime Sleepiness in Narcolepsy,Approved,7/22/20,"FDA Approval announced July 22, 2020.",6
CARA,Pruritus and Notalgia Paresthetica,Phase 2,9/8/22,"Phase 2 data reported a WI-NRS score of 0 or 1 for 70% of the daily non-missing WI-NRS scores for the week., noted September 8, 2022.",5
SVRA,Autoimmune pulmonary alveolar proteinosis (PAP),Phase 3,9/6/22,"Additional Phase 3 data reported that patients treated with molgramostim after undergoing WLL had greater improvements in endpoint measures of gas exchange, noted September 6, 2022.",6
VRTX,Cystic Fibrosis (6-12 mths),Approved,4/30/19,"FDA Approval announced April 30, 2019.",6
CRSP,Beta-thalassemia,Phase 1/2,6/11/21,"Phase 1/2 data presented at EHA meeting June 11, 2021. All 15 patients were transfusion independent with follow-up ranging from 4 to 26 months after CTX001 infusion and had clinically meaningful improvements in total hemoglobin from 8.9 to 16.9 g/dL and fetal hemoglobin from 67.3% to 99.6% at last visit.",6
SGEN,Multiple myeloma,Approved,8/5/20,"FDA approval granted August 5, 2020.",6
LLY,Chronic and episodic migraine,Phase 3,8/5/19,"Phase 3 trial met primary endpoint - August 5, 2019.",6
CYTK,Chronic obstructive pulmonary disease (COPD),Phase 2,10/5/18,"Phase 2 data did not meet endpoints - October 5, 2018.",4
BMY,Solid Tumors,Phase 1b,9/10/22,"Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.",6
PGEN,HPV+ solid tumors,Phase 1,6/3/23,"Phase 1 monotherapy and combination data presented at ASCO reported a 30% ORR in patients with heavily pre-treated HPV-associated cancers, noted June 3, 2023.",6
CORT,Ovarian cancer,Phase 2,6/6/22,"Phase 2 results reported a median 7.3 months of progression-free survival, a median of 5.6 months for duration of response, and 17.9 months for overall survival, noted June 6, 2022.",5
LLY,Pediatric type 2 diabetes,Approved,6/21/23,"FDA approved for 10 mg and 25 mg tablets on June 21, 2023.",6
SYRS,Acute myeloid leukemia (AML),Phase 2,12/10/22,"Phase 2 data reported a 83% composite complete response rate in newly diagnosed unfit AML patients with RARA gene overexpression, noted December 10, 2022.",5
SLS,HER2 3+ breast cancer,Phase 2b,11/30/21,"Phase 2b trial noted DFS rate of 75.2% reduction in relative risk of recurrence or death, noted November 2021.",6
IMMX,Solid tumors,Phase 1/2,7/17/23,"Phase 1b/2a additional data noted that tumor shrinkage was observed in 3 out of 4 (75%) patients; 1 out of 4 (25%) experienced tumor control as of the July 7, 2023 clinical data cutoff date. All patients had stage IV relapsed/refractory metastatic colorectal cancer and received 8 median lines of therapy prior to IMX-110 + tislelizumab, noted July 17, 2023.",6
REGN,Cervical cancer,Phase 3,3/15/21,"Phase 3 trial to be stopped early due to sufficient efficacy. 12 months survival for Libtayo compared to 8.5 months for chemotherapy; hazard ratio (HR): 0.69 - March 15, 2021.",4
AGTC,X-linked Retinitis Pigmentosa (XLRP),Phase 1/2,5/4/22,"Phase 1/2 18-month data appear to show safety and efficacy signals similar to the study's 12-month findings, including improvements to the ellipsoid zone (EZ). In patients with XLRP, the EZ, a defined region within the photoreceptor layer of the retina, degenerates over time and is eventually lost.",6
AMGN,Relapsed Multiple Myeloma,Approved,1/17/18,"sNDA approved January 17, 2018.",6
PRLD,Solid tumors,Phase 1,10/7/21,"Phase 1 interim data displayed a durable CR maintained for over 18 months in a patient with HRD+ ovarian cancer and prolonged SD persisting for over six months in five patients. The most common grade 3 or higher treatment-related adverse events (AE) occurring in at least 5% of patients were thrombocytopenia (27%) and anemia (12%), both of which were reversible upon treatment interruption, noted October 7, 2021.",6
OCUP,Dim Light or Night Vision Disturbances (DLD),Phase 3,5/19/22,"Phase 3 topline data reported that the trial met the primary endpoint and key secondary endpoints with no serious adverse event, noted May 19, 2022.",5
AZN,Type 2 diabetes,Approved,5/3/19,"FDA approval announced May 3, 2019.",6
BGNE,Esophageal squamous cell carcinoma (ESCC),Phase 3,6/30/22,"Phase 3 data reported an improvement in overall survival (OS) for patients receiving treatment with a median OS of 17.2 months versus 10.6 months with placebo, noted June 30, 2022.",6
VERU,ER+/HER2- breast cancer,Phase 3,9/11/23,"Phase 3 enrollment discontinued due to prioritization with Phase 3 data overall response rates of 12.5% are observed in the enobosarm group in a heavily pretreated population versus no responses in the standard of care active control arm. Overall response rate was 20% for enobosarm monotherapy versus 0% for standard of care active control in patients who had ≤3 lines of prior endocrine therapy in the metastatic setting, noted September 11, 2023. ARTEST clinical data further validates the evaluation of enobosarm in the Phase 3 ENABLAR-2 study.",6
LNTH,Help visualize prostate cancer by targeting prostate specific membrane antigen (PSMA),Phase 3,9/12/18,Phase 3 trial met one of two co-primary endpoints.,6
ETON,Allergic conjunctivitis,Approved,9/25/20,"FDA approval announced September 25, 2020.",6
LCTX,Non-small cell lung cancer (NSCLC),Phase 1,7/24/23,"Phase 1 data reported that five of eight patients treated (62.5%) had a best response of immune-related stable disease, and three (37.5%) demonstrated immune-related progressive disease, noted July 24, 2023.",6
AERI,Glaucoma,Approved,3/13/19,"FDA Approval announced March 12, 2019.",6
ERAS,Solid Tumors,Phase 1b,6/5/23,"Deprioritized as dose escalation safety data do not support continued evaluation of regimen tested, noted June 5, 2023.",6
AZN,Non-small cell lung cancer (NSCLC),Phase 3,12/19/22,"Phase 3 trial did not meet primary endpoint, noted December 19, 2022.",6
LYRA,Chronic rhinosinusitis,Phase 2,9/12/23,"Phase 2 top-line results met its primary endpoint with no serious adverse events observed, September 12, 2023.",6
ABUS,"Hepatitis B, healthy volunteers",Phase 1,9/11/23,"Phase 1 dosing commenced, noted September 11, 2023. Phase 1 preliminary data 1H 2024.",5
MRK,"Measles, Mumps, and Rubella Virus Vaccine Live",Approved,3/6/23,"Approved March 6, 2023.",6
DRRX,Post-operative pain relief,Approved,2/2/21,"FDA approval announced February 2, 2021.",6
ELTX,Acute Kidney Injury associated with cardiac surgery,Phase 2,12/9/21,Phase 2 data demonstrated that trial did not meet its primary endpoint of percentage increase in serum creatinine based upon the area under the curve (AUC).,4
RHHBY,Diffuse Large B-Cell Lymphoma,BLA Filing,3/10/23,"Advisory committee meeting voted 11 to 2 in favor of approval, noted March 10, 2023.",6
SLNO,Prader-Willi syndrome (PWS),Phase 3,9/26/23,"Phase 3 trial met its primary endpoint, noted September 26, 2023.",5
MRK,Metastatic Melanoma,Phase 2b,10/31/23,"Phase 2b data presented at SITC reported that a pronounced and ongoing tumor reduction was observed in a patient with progressive disease, noted October 31, 2023.",6
KROS,Pulmonary arterial hypertension (PAH) and disorders associated with bone loss,Phase 2,5/22/23,"Phase 2 biomarker reported that treatment led to changes in serum proteins involved in structural remodeling pathways and inflammation consistent with that seen in preclinical studies, noted May 22, 2023.",6
MRK,COVID-19,Phase 3,4/14/21,"Phase 3 development to be discontinued - April 15, 2021.",4
MRK,Pneumococcal conjugate vaccine,Phase 3,7/27/23,"Phase 3 trial met primary endpoint, noted July 27, 2023.",5
MRUS,"HNSCC , Solid Tumors",Phase 1,4/14/23,"Phase 1 data from AACR reported antitumor activity among the 14 patients, with 1 partial response and 3 had stable disease, noted April 14 2023.",5
VIR,Hepatitis B (HBV),Phase 2,6/24/23,"Phase 2 24 or 48 weeks plus up to 48 weeks of PEG-IFN-⍺ (cohorts 4 and 5 combined), 26% (8/31) achieved HBsAg loss at the end of treatment and 16% (5/31) sustained HBsAg loss 24 weeks after the end of treatment, noted June 24, 2023.",6
MNOV,COVID-19 / acute respiratory distress syndrome (ARDS),Phase 2,6/8/22,"Phase 2 trial met one co-primary endpoint with statistical significants with 71% of the treated group free from respiratory failure at Day 7, noted June 8, 2022.",6
IDYA,"Solid tumors with MTAP deletion, NSCLC",Phase 1,3/15/22,"Phase 1 updated data reported no drug-related SAE's or observed dose limiting toxicity (DLT) through Cohort 5, noted March 15, 2022.",6
OBSV,Severe adenomyosis,Phase 3,10/20/21,"Pivotal trial final results noted that uterine volume was reduced by 55% at 12 weeks and 32% at 24 weeks. Pelvic pain was reduced at 12 and 24 weeks with initial signs of reduction after 4 weeks, noted October 20, 2021.",6
GOVX,COVID-19 in immunocompromised patients,Phase 2,10/30/23,"Phase 2 site expansion commenced, noted Otober 30, 2023.",5
PFE,Endometriosis,Phase 3,7/28/22,"Phase 3 data reported that trials met both co-primary endpoints with 75% of women in the relugolix combination therapy group in both studies achieving a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at Week 24, respectively, noted July 28, 2022.",6
ICPT,Primary biliary cholangitis,Phase 3,11/6/22,"Phase 3/4 results from the EC group as-treated analysis showed statistical significance in both the original and the FDA-expanded primary endpoints comparing the OCA-treated subjects in COBALT with the non-OCA-treated patients in EC, noted November 6, 2022.",6
ABBV,Follicular lymphoma (FL),Phase 1/2,6/28/23,"Phase 1/2 FL cohort data reported an overall response rate (ORR) of 82% and that the median duration of response (DOR) was not reached, noted June 28, 2023.",6
EVLO,Colorectal cancer; Triple-negative breast cancer,Phase 2,12/9/20,"Phase 1/2 data to be presented at SABSC December 9, 2020 - objective response rate (ORR) of 18%.",5
ATNM,Relapsed or refractory acute myeloid leukemia (AML),Phase 1,12/12/22,"Phase 1 data reported a 59% 1-year overall survival and 32% 2-year overall survival in patients with prior Venetoclax treatment, noted December 12, 2022.",6
MOR,Crohn's disease,Phase 3,10/16/23,"Phase 3 data reported that in the combination 54% of patients reached a clinical remission, noted October 16, 2023.",6
BMY,Non-metastatic non-small cell lung cancer,Phase 3,10/17/23,"Phase 3 reported patients treated with neoadjuvant Opdivo and chemotherapy followed by surgery and adjuvant Opdivo, the risk of disease recurrence, progression or death was reduced by 42%, noted October 17, 2023.",6
CHRS,Solid tumors,Phase 1,12/6/21,"Phase 1 update June 4, 2021 noted stable disease of 7 of 19 evaluable patients (37%). Phase 1 data reported 10 of 32 evaluable patients (31%) receiving the monotherapy had disease stabilization at eight weeks, with four (12%) persisting beyond 16 weeks, noted December 6, 2021.",5
BHC,Acne vulgaris,Approved,10/20/23,"Approved on October 20, 2023.",6
ALIM,Non-infectious uveitis,Approved,10/15/18,"FDA approval announced October 15, 2018.",6
RHHBY,Non-small cell lung cancer (NSCLC),Approved,5/18/20,"FDA Approval announced May 18, 2020.",6
ARVN,Metastatic castration-resistant prostate cancer (mCRPC),Phase 1/2,10/23/23,"Phase 1/2 data presented at ESMO reported a PSA50 of 41% in patients with tumors harboring any AR LBD mutation, and a PSA50 of 50% in patients with any tumor harboring an AR L702H mutation, noted October 23, 2023.",6
TGTX,Marginal zone lymphoma (MZL),Approved,2/5/21,"FDA approval announced February 5, 2021.",6
MTEM,Relapsed/refractory diffuse large B-cell lymphoma (DLBCL),Phase 2,4/5/21,"Development to be discontinued - April 5, 2021.",4
AVDL,Clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia,Approved,6/30/14,"Approved June 30, 2014.",6
HALO,HER2-positive early breast cancer,Approved,6/29/20,"FDA Approval announced June 29, 2020.",6
MEIP,Acute Myeloid Leukemia,Phase 3,7/2/20,"Phase 3 trial discontinued due to lack of efficacy - July 2, 2020.",4
EFTR,COVID-19,Phase 1b,2/21/23,"Phase 1b data reported that only Grade 1 or 2 events were reported, noted February 21, 2023.",5
MRK,Renal Cell Carcinoma,Approved,8/11/21,"FDA approval announced August 11, 2021.",6
OCUL,Allergic conjunctivitis,Approved,10/11/21,"sNDA approved October 11, 2021.",6
SPPI,Peripheral T-Cell Lymphoma cancer,Approved,7/7/14,"Approved July 7, 2014.",6
TGTX,Relapsed or refractory Chronic Lymphocytic Leukemia (CLL),Phase 1/2,9/20/21,"Phase 1/2 updated data showed that from a total of 20 patients 91% ORR in patients with R/R CLL (n=11), 83% ORR in BTK refractory CLL patients (n=6), with 4 of 5 responders achieving a response to U2 alone at the patient's first efficacy assessment, prior to the addition of pembrolizumab, 25% ORR in patients with RT (n=8), including 25% CR rate. Data presented at iwCLL September 20, 2021.",6
BLUE,non-β0/β0 transfusion-dependent thalassemia (TDT),Phase 3,12/12/22,"Phase 3 long term data shared at ASH reported that no hematologic malignancies, insertional oncogenesis, vector-derived replication competent lentivirus, or clonal predominance was observed, noted December 12, 2022.",6
RDHL,Gastroenteritis,Phase 3,6/14/17,"Phase 3 top-line data released June 14, 2017 - primary endpoint met.",6
BIIB,Alzheimer’s disease,Phase 2,6/16/21,"Phase 2 trial did not meet primary endpoint - June 16, 2021.",4
FATE,Acute Myelogenous Leukemia / B-cell Lymphoma,Phase 1,11/3/22,"Phase 1 data reported an 80% objective response rate for CAR T Naive versus 38% for prior CAR T patients, noted December 13, 2021. Received RMAT designation November 3, 2022.",5
KTRA,Newly diagnosed MGMT-unmethylated Glioblastoma Multiform (GBM),Phase 2,9/23/23,"Additional Phase 2 case study data to be presented at EANO reported that a patient was tumor-free and has survived more than four years as of the last follow-up in March 2023, and another patient had discontinued treatment and a new lesion was found, noted September 23, 2023.",6
BMY,Chronic Lymphocytic Leukemia (CLL),Phase 1/2,1/26/23,"Phase 1/2 study met the primary endpoint of complete response rate compared to historical control in the prespecified subset of patients, noted January 26, 2023.",5
GILD,HR+/HER2- metastatic breast cancer,Approved,2/3/23,"Approved February 3, 2023.",6
ALXO,Head and Neck Squamous Cell Carcinoma,Phase 2,11/9/21,"Phase 2 clinical data showed that patients with ≥2L who have not received a prior checkpoint inhibitor (""CPI"") (n=10), long-term follow-up data shows that dosing demonstrates a 12-month OS rate of 80% with a mOS of 24.5 months, which compares favorably with standard pembrolizumab therapy in patients with 2L CPI, noted November 9, 2021.",6
VRNA,COVID-19,Phase 2,4/23/21,"Phase 2 top-line data released April 23, 2021 - safe and well tolerated. One patient death was reported in the ensifentrine treatment group.",6
HALO,Multiple myeloma,Approved,7/12/21,"FDA approval announced July 12, 2021.",6
AMLX,Alzheimer's Disease,Phase 2,4/2/22,"Phase 2 results reported that treatment was well-tolerated, with a majority of treatment-emergent adverse events (TEAEs) being gastrointestinal, noted April 2, 2022.",6
ABBV,Psoriasis,Approved,4/23/19,"FDA approval announced April 23, 2019.",6
EXEL,Recurrent or metastatic head and neck cancer,Phase 2,5/26/22,"Phase 2 data released on May 26, 2022. The trial met its primary endpoint of objective response rate per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 at 54%. The overall clinical benefit rate was 91%. At a median follow-up of 10.6 months, the one-year progression-free survival rate was 54.0%, and median progression-free survival was 14.6 months. The one-year overall survival (OS) rate was 68.4%. For the 17 patients with a PD-L1 combined positive score (CPS) under 20, the one-year OS rate was 54.9%. For the 17 patients with a CPS score of 20 or more, the one-year OS rate was 83.6%.",6
JNJ,Multiple myeloma,Approved,5/1/20,"FDA Approval announced May 1, 2020.",6
ACAD,Adjunctive treatment in patients with negative symptoms of schizophrenia,Phase 2,11/25/19,"Phase 2 data released November 25, 2019. Primary endpoint met. Key secondary missed.",5
CAPR,Myocardial infarction (heart attack),Phase 2,5/12/17,"Phase 2 data released May 12, 2017 - primary endpoint unlikely to be met.",4
ASRT,Postherpetic neuralgia - shingles,Approved,1/28/11,"Approved January 28, 2011.",6
KRTX,Schizophrenia,Phase 3,3/20/23,"Phase 3 trial met primary endpoint, noted March 20, 2023.",5
PLRX,Primary sclerosing cholangitis (PSC),Phase 2a,9/26/23,"Phase 2a met its primary and secondary endpoints at the 12-week treatment period, noted September 26. 2023.",6
BMY,Previously Treated Hepatocellular Carcinoma,Approved,3/11/20,"FDA Approval announced March 11, 2020.",6
AFMD,"EGFR Expressing Solid Tumors, Non-small cell lung cancer, and Gastric/gastroesophageal junction (GEJ) cancer",Phase 1/2,11/7/22,"Phase 1 data reported one partial response and stable disease, noted November 7, 2022.",5
MTCR,Non-alcoholic steatohepatitis (NASH),Phase 2a,10/21/21,"No pruritus-related treatment discontinuations occurred. MET642 treatment resulted in on-target mean increases in LDL-C of 5 percent with the 3 mg dose and 19 percent with the 6 mg dose, versus a decline of 10 percent with placebo, from baseline to week 16.",6
ITCI,Bipolar depression,Approved,12/20/21,"Approved December 20, 2021.",6
AMYT,Epidermolysis Bullosa,Phase 3,3/28/22,"Phase 3 trial demonstrated that body surface area percentage of partial thickness wounds reduced from 12.1% to 5.4% after at least 15 months of treatment with no new safety signals observed, noted March 28, 2022.",6
GLPG,Systemic sclerosis,Phase 2,9/10/20,"Phase 2 trial met primary endpoint - September 10, 2020.",6
FMTX,Prostate cancer (CRPC),Phase 1,10/7/21,"Phase 1 initial preliminary results reported include data as of Sept. 1, 2021, from eight men enrolled in the trial. The dose was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. (3/8) patients remain on study; (5/8) patients left the study (4 due to disease progression and 1 withdrawal of consent).",5
LUMO,Ebola,Approved,8/3/23,"Approval in children aged 12 months and older on August 3, 2023.",6
VRTX,Cystic fibrosis (CF) ages 6 and older who have the R117H mutation,Approved,12/29/14,"Approved December 29, 2014.",6
RHHBY,Non-small cell lung cancer (NSCLC),Phase 2a,10/23/23,"Phase 2a data reported that the objective tumor responses (defined as partial responses per RECIST criteria 1.1) were observed in three of five patients (60%) who received GB1211 for at least three weeks, noted October 23, 2023",6
AMGN,Palmoplantar pustulosis for Japanese patients,Phase 2,3/27/22,"Phase 2 data APR demonstrated shorter median time to PPPASI-50 response than PBO. The most common adverse events included gastrointestinal events and headache, noted March 27, 2022.",6
RHHBY,Non-small cell lung cancer (NSCLC),Phase 3,10/18/23,"Phase 3 data reported that treatment reduces the risk of disease recurrence or death by 76%, noted October 18, 2023.",6
FRTX,Autoimmune and inflammatory diseases,Phase 1,3/7/23,"Phase 1 SAD and MAD topline results reported that trial met the primary endpoint, noted March 7, 2023.",5
UTHR,Small cell lung cancer,Phase 2/3,2/3/20,"Phase 2/3 trial did not meet primary endpoint - February 3, 2020.",4
SAVA,Alzheimer's disease,Phase 3,9/18/23,"Phase 3 Data and Safety Monitoring Board (DSMB) recommended that both of Cassava Sciences' Phase 3 studies of simufilam continue as planned, without modification, noted September 18, 2023.",5
LGND,Pneumococcal disease,Approved,7/16/21,"FDA approval announced July 16, 2021.",6
FBIO,Papulopustular Rosacea,Phase 3,7/11/23,"Phase 3 topline data achieved the co-primary and all secondary endpoints and subjects completed the 16-week treatment with no significant safety issues, noted July 11, 2023.",6
NBIX,Congenital Adrenal Hyperplasia (CAH) - children,Phase 3,10/5/23,"Phase 3 trial met primary endpoint, noted October 5, 2023.",5
ERYP,Pancreatic cancer,Phase 3,10/25/21,"Phase 3 trial did not meet its primary endpoint of overall survival, noted October 25, 2021.",4
SIOX,GM1 gangliosidosis,Phase 1/2,10/21/21,"Phase 1/2 interim safety and biomarker data demonstrated consistent dose-dependent improvements across biomarker measures, normalization of serum beta-galactosidase activity and GM1 ganglioside in CSF in the high-dose cohort, no overt disease progression in (6/7) patients treated across low- and high-dose cohorts. No SAE reported in patients (10/10), noted October 21, 2021.",6
MOR,Relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL),Phase 3,11/18/19,"Phase 3 futility analysis passed November 18, 2019",6
IMGN,Ovarian cancer,Phase 1/2,9/29/22,"Phase 1/2 data reported an ORR of 44%, a median DOR of 11.8 months, and median PFS of 8.2 months, noted September 29, 2022.",5
RETA,Alport Syndrome,CRL,2/25/22,"CRL issued February 25, 2022.",5
LLY,COVID-19 antibody,Phase 3,10/26/20,"Phase 3 trial discontinued - October 26, 2020.",4
JNCE,Solid tumors,Phase 1,3/16/23,"Phase 1 data report 5 durable responses, with 4 being confirmed responses, noted March 16, 2023.",5
SNY,Autosomal dominant polycystic kidney disease (ADPKD),Phase 2/3,6/1/21,"Phase 2/3 trial stopped due to futility - June 1, 2021.",4
JNJ,Prostate Cancer,Phase 3,9/16/21,"Phase 3 data showed OS was improved by abiraterone both in the overall population (HR: 0.83; medians: 5.7 vs 4.7 yrs) and in the ADT+ docetaxel population (HR: 0.75; medians: NR vs 4.4 yrs), noted September 16, 2021.",6
DRRX,Psoriasis,Phase 2a,1/2/20,"Phase 2 top-line data did not meet endpoints - January 2, 2020.",4
CNCE,Alopecia areata,Phase 3,11/21/22,"Additional data reported that for patients with an absolute Severity of Alopecia Tool (SALT) score less than 95 at baseline, 43% and 57% of the 8 mg twice-daily and 12 mg twice-daily deuruxolitinib dose groups, respectively, achieved a SALT score of 20 or less at Week 24, noted November 21, 2022.",6
ACHL,Non-Small Cell Lung Cancer (NSCLC),Phase 1/2,12/6/22,"Phase 1/2a data reported 1 partial response (PR), and 6 stable disease with overall durable clinical benefit at 12 weeks in 71% of evaluable patients, noted December 6, 2022.",6
RYTM,Bardet-Biedl Syndrome,Approved,6/16/22,"Approved June 16, 2022.",6
ALVO,Inflammatory conditions,CRL,10/12/23,"CRL received October 12, 2023.",5
MNPR,Soft tissue sarcoma,Phase 1b,11/1/23,"t, as well, which may result in further tumor size reduction, noted August 8, 2023. Phase 1b data showed that 9 out of the 14 patients have had stable disease (SD, as defined by RECIST 1.1 criteria) after camsirubicin treatment., noted November 1, 2023.",6
NKTR,Solid Tumors,Phase 1b,9/10/22,"Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.",6
EVOK,Diabetic gastroparesis,Approved,6/19/20,"FDA Approval announced June 19, 2020.",6
ABBV,Endometriosis,Approved,7/24/18,"FDA approval announced July 24, 2018.",6
TRVN,Epilepsy and other CNS disorders,Phase 1,10/16/23,"Full safety and tolerability data showed that TRV045 was well tolerated with results generally consistent with prior first-in-human study, noted October 16, 2023.",6
JNJ,Metastatic castration-sensitive prostate cancer (mCSPC).,Approved,9/18/19,"FDA Approval announced September 18, 2019.",6
VSTM,Chronic lymphocytic leukemia (CLL)//Small Lymphocytic Lymphoma (SLL)/Follicular Lymphoma,Approved,9/24/18,"FDA Approval announced September 24, 2018.",6
TEVA,Acute Promyelocytic Leukemia,Approved,1/15/18,"Approved January 15, 2018.",6
EVLO,"Psoriasis, healthy volunteers",Phase 2,10/17/23,"Phase 2 study did not achieve its primary endpoint, noted October 17, 2023.",6
MRK,Type 2 Diabetes,CRL,4/7/17,CRL issued April 7 2017. sNDA for approved drug requested to include data on cardiovascular effects,5
EQRX,1L metastatic non-small cell lung cancer (NSCLC),Phase 3,5/26/22,"Phase 3 data reported that the median OS was 25.4 months versus 16.9 months in placebo, noted May 26, 2022.",5
MOLN,Acute myeloid leukemia (AML),Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that one of the two patients evaluable for MP0533 antitumor activity in the third treatment cohort achieved a response, noted November 2, 2023.",6
SNGX,Oral Mucositis in Patients with Head & Neck Cancer,Phase 3,12/22/20,Phase 3 trial did not meet primary endpoint.,4
NVO,Type 1 diabetes,Phase 3,6/3/22,"Phase 3a trial met its primary endpoint of demonstrating non-inferiority in reducing HbA1c at week 26 with insulin icodec compared to insulin degludec, noted June 3, 2022.",6
BXRX,Neuromuscular blockade (NMB) in patients undergoing elective surgery,Phase 2,6/7/23,"hase 2 primary efficacy endpoint was the proportion of patients that met criteria for Good or Excellent intubating conditions using a standardized scale. All patients, across regimens, met the primary efficacy criteria, noted June 7, 2023.",5
AMGN,Migraine,Approved,5/17/18,"Approval announced May 17, 2018.",6
CNTX,Endometrial Cancer,Phase 2,11/9/22,"Phase 2 interim data demonstrated preliminary 4-month PFS rate of 77.7%, noted November 9, 2022.",6
PFE,Gastric cancer,Phase 3,11/28/17,"Phase 3 data released November 28, 2017 - primary endpoint not met.",4
ORTX,Metachromatic leukodystrophy (MLD),Phase 3,8/31/23,"Announced long-term results from 39 patients that demonstrated that administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 12 years of follow-up (median 6.76 years), noted August 31, 2023.",6
GBT,Sickle cell disease - children age 4-11,Approved,12/17/21,"Approved December 17, 2021.",6
SNY,Diabetes,Approved,12/11/17,"Approval announced December 11, 2017.",6
GSK,Severe eosinophilic asthma (6-11 yrs),Approved,9/12/19,"FDA approval announced September 12, 2019.",6
KRON,Solid tumors,Phase 1/2,11/2/23,"Phase 2 initial data presented at Connective Tissue Oncology Society reported that treatment demonstrated on-mechanism, single agent anti-tumor activity and a manageable safety profile in heavily pre-treated patients with transcriptionally addicted solid tumors, noted November 2, 2023.",6
MNOV,Progressive multiple sclerosis (progressive MS),Phase 2b,10/28/17,"Phase 2b top-line data released October 28, 2017 at Joint ECTRIMS – ACTRIMS meeting. Primary endpoint met.",6
ABBV,Crohn's disease,Phase 3,10/16/23,"Phase 3 data reported that the trial met all primary and secondary endpoints, noted October 16, 2023.",5
CDXS,Phenylketonuria (PKU),Phase 1,11/8/18,"Phase 1 trial met endpoints - November 8, 2018.",6
CMPI,Lymphoma,Phase 1/2,5/26/22,"Phase 1/2 data released in an abstract at ASCO reported no significant adverse effects (AEs) and clinical activity in 5 of 7 patients, noted May 26, 2022.",5
VIRX,Epstein-Barr Virus Associated Lymphoma,Phase 1/2,8/8/23,"Phase 1b/2 data results showed complete responses achieved and ongoing durable responses observed out to approximately 36 months across multiple EBV+ lymphoma subtypes, including some of the most aggressive cancers, noted August 8, 2023.",6
TSVT,Newly Diagnosed Multiple Myeloma,Phase 2a,12/10/22,"Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.",6
VERU,Hot flashes,Phase 2,1/13/20,"Phase 2 data released January 13, 2020. 50mg dose shown to be effective (10mg ineffective).",6
EVLO,Mild to moderate psoriasis,Phase 2,3/28/22,"Phase 2 data reported that treatment was well tolerated, and the primary endpoint analysis showed that treatment was superior to placebo with 80-90% probability across each cohort, noted March 28, 2022.",6
ALT,Non-alcoholic fatty liver disease (NAFLD),Phase 2b,8/1/23,"Phase 2b IMPACT trial enrollment initiated, noted August 1, 2023.",5
CLSD,Neovascular age-related macular degeneration (wet AMD),Phase 2b,11/1/23,"Phase 2b recruitment completed, noted November 1, 2023.",5
FATE,"B-Cell Chronic Lymphoma, chronic lymphocytic leukemia",Phase 1,12/31/21,"Phase 1 interim data released August 19, 2021. 10/14 patients (71%) achieved an objective response; 7/14 (50%) complete responses. Phase 1 data reported 5 of 6 patients achieve objective response with 4 with a complete response, noted December 13, 2021.",6
LLY,Ulcerative Colitis (Maintenance data),Phase 3,5/8/23,"Additional Phase 3 data reported that bowel urgency remission directly accounted for a 44.8% and 32.5% improvement in IBDQ total score, respectively; 22.7% and 39.1% improvement, respectively, was mediated by rectal bleeding remission, and 32.5% and 28.4%, respectively, mediated by stool frequency remission, noted May 8, 2023.",6
DVAX,Hepatitis B,Approved,11/9/17,"Final approval announced November 9, 2017",6
MOR,Plaque psoriasis,Approved,2/27/19,"Approval announced February 27, 2019.",6
ETON,Injectable hospital product,Approved,10/22/19,"FDA Approval announced October 22, 2019.",6
VBLT,Recurrent glioblastoma (rGBM) cancer,Phase 3,3/8/18,"Phase 3 top-line data released March 8, 2018 - primary endpoint not met.",4
INCY,Follicular lymphoma,Phase 2,12/7/20,Phase 2 data due released at ASH December 2020 - ORR 73%.,6
ADAP,Hepatocellular carcinoma,Phase 1,9/5/21,"Phase 1 data displayed one complete response and a disease control rate of 64% with stable disease for ≥16 weeks in two patients. Phase 1 top-line data has an acceptable safety profile. Dose escalation is complete September 5, 2021.",6
BIIB,Mild Alzheimer's disease,Phase 1b,10/25/23,"Phase 1b data presented at CTAD showed favorable trends reported for the high-dose groups on multiple measures of cognition and function, noted October 25, 2023.",6
RGEN,Detection of pancreatic duct abnormalities,CRL,6/22/12,"CRL received June 22, 2012.",5
ALEC,Alzheimer’s disease,Phase 2,9/7/23,"Phase 2 enrollment to be completed, noted September 7, 2023.",5
SGEN,Squamous cell carcinoma of the head and neck (SCCHN),Phase 2,4/17/23,"Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.",6
ACET,Non-Hodgkin’s lymphoma,Phase 1,6/26/23,"Phase 1 safety and efficacy data demonstrated 71% overall response rate (ORR) and 63% complete response (CR) rate across all dose levels in patients with median 4 prior lines of therapy, a Six-month CR rate consistent with autologous CAR T cell therapy, noted June 26, 2023.",6
AMGN,Relapsed Multiple Myeloma,Approved,6/11/18,"sNDA approval announced June 11, 2018.",6
RCKT,Glaucoma,Phase 2,7/7/17,"Phase 2 trial failed - noted July 7, 2017.",4
IMVT,Graves Ophthalmopathy / Thyroid Eye Disease (TED),Phase 2b,2/2/21,"Dosing has paused due to elevated total cholesterol and LDL levels in Phase 2b ASCEND GO-2 trial - February 2, 2021.",4
HLVX,Prevention of Norovirus-Related Acute Gastroenteritis in Infants,Phase 2b,12/5/22,"Phase 2b immunogenicity results reported that Geometric Mean Titers (GMTs) of pan-IG antibodies 28 days following the second dose were 11,102.0 IU/mL and 2,185.5 IU/mL for GI.1 and GII.4, respectively, for HIL-214 compared to 59.6 IU/mL and 73.5 IU/mL for GI.1 and GII.4, respectively, for placebo, noted December 5, 2022.",5
JAZZ,Small cell lung cancer (SCLC),Phase 3,12/3/20,"Phase 3 trial did not meet overall survival endpoint - December 3, 2020.",4
BBIO,Gastric Cancer / Gastroesophageal junction adenocarcinoma,Phase 2a,10/25/23,"Phase 2a data presented at ESMO showed that infigratinib was generally well tolerated with a manageable safety profile, noted October 25, 2023.",6
PFE,Alzheimer’s disease,Phase 2/3,1/18/21,"Phase 2/3 top-line data did not meet co-primary endpoints - January 18, 2021.",4
KRYS,Epidermolysis Bullosa,Approved,5/19/23,"Approved May 19, 2023.",6
RHHBY,Triple-negative breast cancer,Phase 3,6/4/19,"Phase 3 OS update at ASCO June 4, 2019 - median OS=21.0 vs 18.7 months; HR=0.86.",5
ZYME,HER2-positive Gastroesophageal Adenocarcinoma (GEA),Phase 2,9/16/21,"Phase 2 data showed confirmed objective response rate (cORR) of 75% and disease control rate (DCR) of 89% overall, with a cORR of 93% and DCR of 100% in the proposed Phase 3 regimen noted September 16, 2021.",6
DRRX,Schizophrenia,Approved,7/27/18,"FDA Approval announced July 27, 2018.",6
VYNE,Papulopustular rosacea,Approved,5/29/20,"FDA Approval announced May 29, 2020.",6
MESO,Chronic low back pain,Phase 3,2/8/23,"Phase 3 FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation, noted February 8, 2023.",5
CVAC,"Covid-19 vaccine, omicron variant",Phase 1,1/30/23,"Phase 1 immunogenicity data reported that on day 29 at the 12µg dose level, CV0501 generated a ratio of post-boost to pre-boost serum neutralizing titers against BA.1 of 13.3, noted January 30, 2023.",5
LLY,Migraine,Approved,10/11/19,"FDA Approval announced October 11, 2019.",6
SNY,Multiple sclerosis,Phase 2,2/6/20,"Phase 2 data met primary endpoint - February 6, 2020. Data to be presented at upcoming meeting.",6
TGTX,Multiple Sclerosis,BLA Filing,9/30/21,"BLA submitted September 30, 2021.",5
PFE,Metastatic Merkel cell carcinoma,Approved,3/23/17,"Priority review announced November 29, 2016. Approval announced March 23, 2017.",6
OCUL,Post-surgical ocular inflammation and pain,Approved,6/21/19,"FDA Approval June 21, 2019.",6
EXEL,Third-line advanced or metastatic colorectal cancer,Phase 3,5/10/18,"Phase 3 data released May 10, 2018. Primary endpoint not met.",4
SIGA,Smallpox,Approved,7/13/18,"FDA approval announced July 13, 2018.",6
VOR,Acute myeloid leukemia (AML),Phase 1/2,6/9/23,"Phase 1/2a additional data presented at EHA showed that patients transplanted with trem-cel achieved primary neutrophil engraftment and high levels of myeloid donor chimerism, noted June 9, 2023.",6
AZN,Biliary-tract cancer,Phase 3,1/18/22,Phase 3 trial demonstrated a statistically significant and clinically meaningful improvement in of 58% overall survival (OS) and 81% progression-free survival (PFS) versus 66% OS and 86% PFS with chemotherapy alone as a 1st-line treatment for patients with advanced biliary tract cancer (BTC),6
MRK,Nonsquamous non-small cell lung cancer (NSCLC),Phase 3,9/22/23,"Phase 3 trial did not meet its dual primary endpoints of overall survival (OS) and progression free survival (PFS), noted September 22, 2023.",6
GMAB,Multiple myeloma - candidates for autologous stem cell transplant (ASCT),Approved,9/26/19,"FDA Approval announced September 26, 2019.",6
CRBP,Systemic Sclerosis,Phase 3,9/8/20,"Phase 3 top-line data did not meet endpoints - September 8, 2020.",4
MTEM,Solid tumors,Phase 1,11/2/23,"Phase 1 dosing initiated, noted November 2, 2023.",5
ANIP,Severe vasomotor symptoms (hot flashes and night sweats) due to menopause,Approved,8/28/23,"Approved August 28, 2023.",6
VTVT,Type 1 Diabetes,Phase 2,10/12/21,"Phase 2 mechanistic study data demonstrated that treated patients experienced no increase in ketone levels relative to placebo during a period of acute insulin withdrawal, indicating no increased risk of ketoacidosis. Study met primary endpoint, noted October 12, 2021.",6
IKT,Multiple System Atrophy (MSA),Phase 2,10/4/23,"Orphan Drug Designation granted by FDA on October 4, 2023.",5
MRK,Pneumococcal Conjugate Vaccine,Phase 3,7/27/23,"Phase 3 trial met primary endpoints, noted July 27, 2023.",5
VTRS,Pediatric Formulation of Dolutegravir (DTG),Approved,11/23/20,"FDA tentative approval announced November 23, 2020.",6
BBIO,Molybdenum cofactor deficiency type A,Approved,2/26/21,"FDA approved February 26, 2021.",6
AURA,Uveal melanoma including choroidal melanoma,Phase 2,2/16/23,"Phase 2 safety and efficacy data presented with an average of nine months of follow-up for patients treated with three cycles of therapy showed response to the therapy with 89-100% tumor control, noted February 16, 2023.",6
SNY,COVID-19 vaccine,Phase 1/2,9/28/21,"Phase 1/2 interim data noted neutralizing antibody seroconversion in 91% to 100% of study participants. No safety concern has been observed, noted September 28, 2021.",6
PBYI,Extended adjuvant treatment of early stage HER2-positive breast cancer,Phase 2,12/13/21,"Phase 2 dose escalation final findings reported that Neratinib DE + loperamide PRN during the first 2 weeks of treatment was associated with the lowest rates of grade 3 diarrhea (13.3%) and diarrhea-related discontinuations (3.3%) compared with all other anti-diarrheal strategies investigated in the trial, noted December 13, 2021.",6
RVMD,"Pancreatic cancer, non-small cell lung cancer (NSCLC), and colorectal cancer.",Phase 1,9/19/23,"Phase 1 dosing initiated, noted Septemeber 19, 2023.",5
NVS,Migraine,Approved,5/17/18,"Approval announced May 17, 2018.",6
PFE,Acute myeloid leukemia (AML),Approved,9/1/17,"Approval announced September 1, 2017.",6
VRTX,Children (6-11 years old) who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive,Approved,6/9/21,"FDA approval announced June 9, 2021.",6
LLY,Obesity or overweight with weight-related comorbidities,Phase 3,7/27/23,"Phase 3 trial met primary endpoint, noted July 27, 2023.",5
KOD,Diabetic Macular Edema (DME),Phase 3,10/13/23,"Phase 3 data presented at the Retina Society meeting reported that 1/2 of patients achieved 6-month dosing at the primary endpoint, noted October 13, 2023.",6
CERE,Schizophrenia,Phase 1,12/19/22,"Phase 1 results reported that treatment demonstrated a mean change from baseline at week eight in 24-hour ambulatory systolic blood pressure (SBP) of -2.7 mmHg for the 10 mg QD group and -0.4 mmHg for the 30 mg QD group, noted December 19, 2022.",6
INCY,Hidradenitis suppurativa,Phase 2,10/12/23,"Phase 2 secondary analysis presented at EADV reported that a large proportion of patients who received any dose of povorcitinib from Day 1 achieved T VASI50 (37.0% 45.2%) and F VASI75 (48.4% 58.6%) responses at Week 52, noted October 12, 2023.",6
VALN,COVID-19 vaccine,Phase 3,3/2/23,"Phase 3 data reported that the fold decline of neutralizing antibodies over six months after a second vaccination with VLA2001 was similar to the active comparator, and less pronounced than for other licensed COVID-19 vaccines, noted March 2, 2023.",5
SNY,Prurigo nodularis (PN),Approved,10/27/22,Approved in 3Q 2022.,6
TENX,Septic shock,Phase 2b,10/5/16,Phase 2b trial did not meet primary endpoint.,4
ERAS,Non-small-cell Lung Cancer,Phase 1b,6/5/23,"Deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023.",6
GMAB,Relapsed or refractory multiple myeloma,Approved,6/16/17,"Approval announced June 16, 2017 - note: submitted by Janssen Biotech, Inc.",6
VCEL,Focal chondral cartilage defects in the knee.,Approved,12/13/16,"FDA Approval announced December 13, 2016 ahead of PDUFA date of January 3 2017.",6
URGN,Non-Muscle-Invasive Bladder Cancer,Phase 3,7/27/23,"Phase 3 trial met primary endpoint, noted July 27, 2023.",5
IPHA,Mycosis Fungoides,Phase 2,11/3/23,"Phase 2 data from ASH abstract reported that the global confirmed ORR was 37.5% including 2 CRs, with a median time to response of 2.8 months, noted November 3, 2023.",5
GMAB,Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL),Approved,5/19/23,"Approved May 19, 2023.",6
GILD,Metastatic urothelial cancer (mUC),Phase 2,2/17/23,"Phase 2 data demonstrated the efficacy of 13.5 Months overall survival in patients, noted February 17, 2023.",6
STRO,Relapsed/refractory AML,Phase 1,12/12/22,"Phase 1 data presented at ASH reported that treatment was well-tolerated with 47% of the patients achieved complete remission and 53% of the patients achieved partial response or stable disease, noted December 12, 2022.",6
ARMP,Pseudomonas aeruginosa Infections and Cystic Fibrosis,Phase 1/2,3/6/23,"Phase 1/2 top-line data reported that treatment was well-tolerated with a proportional increase in exposure as measured in induced sputum, noted March 6, 2023.",6
ALRN,Breast cancer,Phase 1b,2/21/23,"Phase 1b trial terminated following Grade 4 SAE, noted February 21, 2023.",4
NVS,Adjuvant breast cancer,Phase 3,6/2/23,"Phase 3 data presented at ASCO significantly reduced the risk of recurrence by 25% across a broad population of patients with early breast cancer; clinically meaningful benefit was consistent across subgroups, noted June 2, 2023.",6
LVTX,"Chronic lymphocytic leukemia (CLL), multiple myeloma (MM) and acute myeloid leukemia (AML)",Phase 1/2,6/14/23,"Phase 1/2a discontinued, noted June 14, 2023.",4
PCSA,Gastrointestinal Cancer,Phase 1b,8/17/23,"Phase 1b interim analysis suggested Processa's novel chemotherapy treatment could improve safety and efficacy in more patients by individualizing the dosing regimen for each patient, noted August 17, 2023.",6
INVA,Asthma,Approved,9/9/20,"FDA approval announced September 9, 2020.",6
NTLA,Hereditary angioedema (HAE),Phase 1/2,6/11/23,"Phase 1/2 updated safety and efficacy data to be presented at EAACI showed that after a single dose of investigational CRISPR-based therapy, patients living with hereditary angioedema experienced durable elimination of their attacks, noted June 11, 2023.",6
ARWR,AATD-associated liver disease,Phase 2,6/27/22,"Phase 2 data showed noted a Fibrosis regression observed in 58% (7/12) of patients receiving 200 mg, a median reduction of 83% of Z-AAT accumulation in the liver and a reduction of 69% in histologic globule burden",6
SNY,Atopic dermatitis,Phase 2a,9/30/21,"Phase 2a data noted 80% improvement in average EASI, and patients treated with amlitelimab-HD showed 70% improvement in average EASI, compared to 49% for the placebo group. The overall rate of TEAEs was 35% for amlitelimab-LD, 17% for amlitelimab-HD and 31% for placebo. One serious adverse event was reported in the amlitelimab-LD group related by the investigator; the event was resolved, and the patient was able to complete the study. No hypersensitivity reactions were reported, noted September 30, 2021.",6
ALEC,Frontotemporal Dementia,Phase 3,10/27/23,"Phase 3 target enrollment achieved, noted October 27, 2023.",6
GRTS,Non-small cell lung cancer (NSCLC),Phase 2,9/12/22,"Phase 2 data presented at ESMO demonstrated early evidence of efficacy with a 39% molecular response rate in evaluable patients, noted September 12, 2022.",6
ISEE,Wet age-related macular degeneration (Wet-AMD),Phase 2a,11/12/18,"Phase 2a top-line data released November 12, 2018 noted that treatment was generally well tolerated.",6
KPTI,Endometrial cancer,Phase 3,7/25/23,"Phase 3 updated exploratory subgroup analysis reported a median PFS of 27.4 months compared to 5.2 months for patients with TP53 wild-type endometrial cancer receiving placebo, noted July 25, 2023.",5
PHAT,Erosive esophagitis / Gastroesophageal reflux disease (GERD),Approved,11/1/23,"FDA Approved on November 1, 2023.",6
EGRX,Increase blood pressure in adults,Approved,12/15/21,"Approved December 15, 2021.",6
MRK,Pulmonary arterial hypertension (PAH),PDUFA,9/28/23,"PDUFA date on March 26, 2024.",5
AFMD,"EGFR-expressing tumors, Non-small cell lung cancer, and Squamous cell carcinoma of the head and neck",Phase 1,8/3/23,"Phase 1 dose-escalation update reported that AFM24 showed meaningful clinical activity in heavily pretreated patients with EGFRmut NSCLC (ORR [13%], DCR [47%]) with significant tumor reductions, including two confirmed PRs and five patients exhibiting stable disease, noted August 3, 2023.",6
MRK,adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors,Approved,3/29/23,"FDA approval on March 29, 2023.",6
ACER,induced Vasomotor Symptoms (iVMS) associated with menopause,Phase 1,3/17/23,"Phase 2a PoC did not meet its primary endpoint of ability to decrease the frequency or severity of hot flashes in postmenopausal women. As a result, the company is pausing the program until a full dataset review, noted March 17, 2023",6
BLPH,Pulmonary Arterial Hypertension (PAH),Phase 3,8/7/18,"Phase 3 trial stopped due to futility - August 7, 2018.",4
IBRX,Merkel cell carcinoma (MCC),Phase 2,11/7/19,"Phase 2 data noted 2/7 responses (1 PR / 1 CR) - November 7, 2019.",5
BFRI,"Actinic keratosis on the extremities, neck and trunk.",Phase 3,1/9/23,"Phase 3 trial dosing initiated, noted January 9, 2023.",5
GSK,"Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients",Approved,9/2/16,"Approved September 2, 2015.",6
CMRX,Neuroendocrine Tumors,Phase 2,6/4/21,Phase 2 data in ASCO abstract noted ORR 5/10 (50%).,5
RHHBY,Ulcerative colitis,Phase 2b,6/22/23,"Additional Phase 2b data continue to support RVT-3101's potential as a first-in-class anti-TL1A antibody, demonstrating sustained efficacy across a broad dose range measured at 56 weeks, noted June 22, 2023",6
COYA,Alzheimer’s Disease (AD),Phase 2,10/9/23,"Phase 2 enrollment completed, noted October 9, 2023.",5
MGTA,Acute myeloid leukemia and myelodysplasia-excess blasts,Phase 1/2,1/25/23,"Company voluntarily paused trial after a Grade 5 Serious Adverse Event (SAE) (respiratory failure and cardiac arrest resulting in death of patient), noted January 25, 2023.",4
PFE,ER+/HER2- advanced breast cancer,Phase 3,6/4/22,"Phase 3 trial did not meet the primary endpoint, noted June 4, 2022.",6
AVIR,COVID-19,Phase 3,4/17/23,"Phase 3 presented at ECCMID reported a 71% relative reduction in risk of hospitalization with bemnifosbuvir versus placebo, noted April 17, 2023.",6
MRK,Biosimilar candidate of Remicade,Approved,4/21/17,"BLA acceptance announced May 23, 2016 by partner Samsung Bioepis. Approval annuonced April 21, 2017.",6
ABBV,Cancer - Chronic Lymphocytic Leukemia Who Have Received at Least One Prior Therapy,Approved,2/12/14,"Approved February 12, 2014.",6
CNTB,Atopic dermatitis,Phase 2b,3/17/23,"Phase 2b data reported that at week 2, EASI decreased by -26.3% (head/neck), -26.4% (trunk), -21.6% (upper limbs) and -23.2% (lower limbs) for patients on CBP-201 300 mg Q4W treatment vs -9.5% to -15.7% with placebo, noted March 17, 2023.",6
LLY,Relapsed/refractory chronic lymphocytic leukemia,Approved,1/27/23,"FDA Approved on January 27, 2023.",6
ARDX,Hyperphosphatemia - adjunctive therapy to phosphate binders,Phase 3,9/3/19,Phase 3 trial met primary endpoint.,6
INCY,Atopic dermatitis,PDUFA,7/16/21,"Noted FDA will not meet assigned July 2021 PDUFA date - July 16, 2021",4
PFE,Metastatic non-small cell lung cancer (NSCLC),Phase 3,2/8/22,"Phase 3 trial did not meet primary endpoints of overall survival, safety profile was consistent with that observed in the overall JAVELIN clinical development program, noted February 8, 2022.",6
EDSA,Allergic contact dermatitis (ACD),Phase 2b,3/18/23,"Phase 2b data presented at AAD reported a 60% average improvement in symptoms from baseline on the CDSI versus 39% for the vehicle treated patients, noted March 18, 2023.",6
RHHBY,Neovascular Age-Related Macular Degeneration,Approved,10/22/21,"Approved October 22, 2021.",6
MRK,Facioscapulohumeral muscular dystrophy,Phase 2,9/16/19,"Phase 2 data did not achieve statistically significant improvements in functional endpoints relative to placebo - September 16, 2019.",4
OCUL,Dry eye disease,Phase 2,12/6/21,"Phase 2 top-line results showed statistically significant improvement for primary endpoint of bulbar conjunctival hyperemia compared with vehicle hydrogel insert. Both formulations were generally well tolerated with favorable safety profiles, noted December 6, 2021.",6
PFE,BRCA-mutated breast cancer,Approved,10/16/18,"FDA Approval announced October 16, 2018.",6
AMRX,Neulasta biosimilar,Approved,5/27/22,"Approved May 27, 2022.",6
AXLA,Long COVID-19,Phase 2a,4/17/23,"Phase 2a data reported that fatigue responders showed significantly greater improvement in phosphocreatine recovery time, a marker of mitochondrial function, and a trend towards reductions in plasma FGF-21 and serum lactate at day 28 compared with placebo-treated patients, noted April 17, 2023.",6
AZN,Eosinophilic Esophagitis,Phase 3,10/25/22,"Phase 3 data reported that trial did not meet one of the two dual-primary endpoints, noted October 25, 2022.",6
BMY,Relapsed or Refractory Follicular Lymphoma,Approved,5/28/19,"FDA Approval announced May 28, 2019.",6
ALKS,Schizophrenia,Approved,6/6/17,"Approved October 5 2015. sNDA filed for 2-month option with approval announced June 6, 2017.",6
AZN,atypical Hemolytic Uremic Syndrome (aHUS),Approved,10/18/19,"FDA Approval announced October 18, 2019.",6
AGEN,RSV vaccine,Approved,5/3/23,"FDA Approved on May 3, 2023.",6
IGXT,Pain,PDUFA priority review,9/19/23,"In response to the CRL, Xiromed submitted to the FDA an Amendment to the ANDA, requesting priority review, noted September 19, 2023. In an Amendment Acknowledgement received from the FDA by the company, the FDA granted priority review with a Generic Drug User Fee Act (GDUFA) date for review of the Amendment of March 8, 2024, unless the Agency determines that an inspection is required. Alternatively, if the FDA determines that an inspection is required, the GDUFA goal date is July 8, 2024.",5
NGM,NASH cirrhosis,Phase 2b,5/8/23,"Phase 2b top-line data demonstrated statistically significant reductions in serum 7αC4 (a marker of bile acid synthesis) and fecal bile acids versus placebo in patients with idiopathic BAM with IBS-D, noted May 8, 2023.",6
PTGX,Crohn's disease,Phase 2,11/3/21,"Phase 2 trial and candidate development has been stopped, noted November 3, 2021.",4
AGIO,Frontline AML with IDH1 mutation,Approved,5/2/19,"FDA Approval announced May 2, 2019.",6
PALI,Bowel Function Following Abdominal Surgery,Phase 2,7/29/21,"Phase 2 data released July 29, 2021- statistically significant (p=0.0008) effect in accelerating the return of bowel function.",6
VBIV,Hepatitis B vaccine,Approved,12/1/21,"Approved December 1, 2021.",6
VKTX,Non-alcoholic steatohepatitis (NASH) and fibrosis,Phase 2b,5/16/23,"Phase 2b data reported that trial met primary endpoint, noted May 16, 2023.",5
CYTK,Symptomatic obstructive hypertrophic cardiomyopathy (HCM),Phase 3,6/20/23,"Phase 3 enrollment open, noted June 20, 2023.",5
LLY,Hemophilia A,Phase 1/2,7/9/21,"Phase 1/2 clinical hold announced July 9, 2021.",4
DTIL,Multiple myeloma,Phase 1/2,6/8/22,"Phase 1/2 update reported that peak expansion rates observed at DL2 plus the GSI have been equivalent to DL4 monotherapy with no dose limiting toxicities observed, noted June 8, 2022.",6
MACK,Small Cell Lung Cancer,Phase 3,8/3/22,"Phase 3 trial did not meet primary endpoint, noted August 3, 2022.",6
COCP,Influenza,Phase 1,5/4/23,"Phase 1 data reported no serious AEs, treatment discontinuations, and no clinically significant observations noted in laboratory assessments, vital signs, physical exam findings, or ECGs, noted May 4, 2023.",6
ADCT,Solid tumors,Phase 1,12/12/22,"Phase 1 presentation at ASH reported that 2 of 3 patients achieved MRD negative CR, noted December 12, 2022.",6
PTCT,Huntington's disease,Phase 2,6/28/23,"Phase 2 trial initiated March 30, 2022. Phase 2 trial data reported that treatment was well tolerated, with no treatment related serious adverse events and no adverse events leading to discontinuation, noted June 28, 2023.",6
BIIB,Painful Lumbosacral Radiculopathy (PLSR),Phase 2,9/30/18,Phase 2 data released September 2018 - primary endpoint not met.,4
KZIA,Glioblastoma multiforme,Phase 2,6/3/22,"Phase 2 final data presented at ASCO reported that the median overall survival (OS) in the intent-to-treat (ITT) population was 15.7 months, compared to 12.7 months which was historically reported with temozolomide in this patient group. Median progression-free survival (PFS) in the ITT population was 8.6 months, noted June 3, 2022.",6
IFRX,ANCA-associated vasculitis (AAV),Phase 2,5/11/21,"Phase 2 data released May 11, 2021 - safe and well tolerated",6
AZN,Anaemia of myelodysplastic syndromes (MDS),Phase 3,10/20/23,"Phase 3 data presented at ESMO reported that treatment demonstrated a statistically significant and clinically meaningful improvement in the key secondary endpoint of pathologic complete response (pCR) versus neoadjuvant chemotherapy alone, noted October 20, 2023.",6
APLS,Paroxysmal Nocturnal Hemoglobinuria (PNH),Approved,5/14/21,"FDA approval announced May 14, 2021.",6
JNJ,Ankylosing Spondylitis,Approved,10/20/17,"Approval announced October 20, 2017.",6
EXEL,Advanced hepatocellular cancer (HCC),Approved,1/14/19,"FDA Approval announced January 14, 2019.",6
AZN,Heart failure in patients with type-2 diabetes,Approved,10/21/19,"FDA Approval announced October 21, 2019.",6
SLRN,Hidradenitis Suppurativa (HS),Phase 2/3,9/11/23,"Phase 2b/3 data to did not meet statistical significance at week 16, noted Septemeber 11, 2023.",6
ALNY,Hereditary transthyretin-mediated (hATTR) amyloidosis,Approved,6/14/22,"Approved June 14, 2022.",6
PFE,Transthyretin amyloid cardiomyopathy (ATTR-CM),Approved,5/6/19,"FDA approval announced May 6, 2019.",6
HRTX,Post operative pain,Approved,5/13/21,"FDA approval announced May 13, 2021.",6
BMY,First-line Head and Neck Cancer,Phase 3,7/16/21,"Phase 3 trial did not meet primary endpoints - July 16, 2021.",4
RLFTF,Urea cycle disorder (UCD),Approved,12/23/22,"Approved December 23, 2022.",6
GILD,Refractory Large B-cell Lymphoma,Phase 1,6/5/23,"Phase 1 data presented at ASCO showed that Yescarta is the first treatment in nearly 30 years to demonstrate a significant improvement in survival in this patient population. The treatment resulted in a 27.4% Reduction in Risk of Death, Corresponding to a 38% Relative Improvement in OS, Despite 57% of Patients Subsequently Receiving Cell Therapy Off Protocol, noted June 5, 2023",6
LLY,Medullary thyroid cancer (MTC),Phase 3,10/21/23,"Phase 3 data presented at ESMO reported that selpercatinib demonstrated superior PFS vs control in the ITT-pembro (HR: 0.465, 95% CI: 0.309, 0.699; p-value: 0.0002) and ITT populations (HR: 0.482, 95% CI: 0.331, 0.700; p-value: 0.0001), noted October 21, 2023.",6
KNSA,Chronic Pruritus,Phase 2,5/11/20,"Phase 2 interim data released May 11, 2020.",5
BIIB,Postpartum Depression,Approved,8/4/23,"Approved August 4, 2023.",6
AZN,COVID-19,Phase 3,4/12/21,Phase 3 trial did not achieve statistical significance for the primary endpoint.,4
LLY,Type 2 diabetes,Phase 2,6/24/23,"Phase 2 additional data achieved a mean reduction in A1C up to 2.1% at 26 weeks, noted June 24, 2023.",6
SGEN,Cervical Cancer,Phase 3,10/23/23,"Additional data presented at ESMO that the overall survival (OS) was statistically significantly prolonged with TIVDAK, demonstrating a 30% reduction in the risk of death compared with chemotherapy, noted October 22, 2023.",6
ABBV,Ulcerative colitis,Phase 3,10/24/22,"Phase 3 data reported that after 52-weeks of maintenance treatment patients had less severe disease and were in clinical remission for approximately 1 additional month/year, noted October 24, 2022.",6
SPRC,Tourette Syndrome,Phase 2b,6/21/23,"Phase 2b IRB approval to initiate study, noted June 21, 2023.",6
IBRX,Acute myeloid leukemia (AML),Phase 1,7/10/23,"Phase 1 results presented at the AHNS indicated the potential for a new treatment approach for the disease in advanced cases that currently have extremely poor prognoses, noted July 10, 2023.",6
ALGS,Hepatitis B,Phase 1,6/21/23,"Phase 1 data presented at EASL resulted in dose-dependent reductions in HBsAg levels through 90 days. Importantly, the safety and PK profile of these dose levels also support further evaluation of higher dose levels, noted June 21, 2023.",6
NVO,Type 2 diabetes,Approved,9/20/19,"FDA approval announced September 20, 2019.",6
AZN,Non-small cell lung cancer (NSCLC) without actionable genomic alterations,Phase 1b,9/11/23,"Phase 1b data reported an objective response rates of 77% and 50% and disease control rates of 92% and 93%, respectively, noted September 11, 2023.",5
GLPG,Cutaneous lupus erythematosus (CLE),Phase 2,10/24/19,Phase 2 trial did not meet primary endpoint.,4
WVE,Amyotrophic Lateral Sclerosis and Frontotemporal Dementia,Phase 1/2,4/4/22,"Phase 1b/2a data reported that reductions in poly(GP) were observed across all active treatment groups, reaching statistical significance versus placebo after single 30 mg doses, noted April 4, 2022.",6
MRK,HER2 amplified metastatic colorectal cancer,Approved,1/19/23,"Approved January 19, 2023.",6
PFE,Ovarian cancer,Phase 3,12/21/18,Phase 3 trial to be terminated due to lack of efficacy.,4
JAZZ,Dravet Syndrome and Lennox-Gastaut syndrome,Phase 3,3/14/16,"Top line Phase 3 data released March 14, 2016 met primary endpoint.",6
BMY,Esophageal or Gastroesophageal Junction Cancer,Approved,5/20/21,"FDA approval announced May 20, 2021.",6
SNPX,Multiple sclerosis (MS),Phase 1,7/19/23,"IND submitted to FDA, noted July 19, 2023",5
MRK,Castration-Resistant Prostate Cancer,Approved,5/19/20,"FDA Approval announced May 19, 2020.",6
GANX,Parkinson's Disease,Phase 1,10/4/23,"Phase 1 trial dosing commenced, noted October 4, 2023.",5
ACRS,Atopic dermatitis,Phase 2b,10/3/23,"Phase 2b trial enrollment completed, noted October 3, 2023",5
XERS,Hypoglycemia-Associated Autonomic Failure,Phase 2,11/21/19,"Phase 2 data showed no statistically significant differences between the treatment arms. Program to be discontinued - November 21, 2019.",4
NVS,Melanoma with BRAF V600E or V600K mutations,Approved,4/30/18,"Approval announced April 30, 2018.",6
MEIP,Relapsed/refractory Follicular Lymphoma (FL) Non Hodgkin Lymphoma,Phase 2,6/4/22,"Phase 2 top-line data presented at ASCO 22 reported a 70.3% objective response rate and that 35.2% achieved a complete response. 87.5% of responses achieved in first two cycles of therapy, 75% of CRs in first four cycles, 9.9% of patients discontinued the trial due to drug related adverse events, follow-up time for response was 8.4 months, and 83% reported grade 3 AE of special interest in cycles 1-3.",6
EQRX,T-cell lymphoma / Stage IV non-small cell lung cancer (NSCLC),Phase 2,5/13/22,"Phase 2 primary analysis reported that trial met its primary endpoint, noted May 13, 2022.",5
SCNI,Universal flu vaccine,Phase 2,6/10/20,Phase 2 data met both primary endpoints.,6
SNY,Acid sphingomyelinase deficiency (ASMD),Phase 3,1/30/20,"Phase 3 data met two independent primary endpoints- January 30, 2020. Combination endpoint was not met.",5
DCPH,Gastrointestinal Stromal Tumors (GIST) - fourth-line,Approved,5/15/20,"FDA Approval announced May 15, 2020.",6
NVS,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 3,12/13/22,"Phase 3 data reported that trial met both primary endpoints, noted December 13, 2022.",5
BMY,Psoriatic Arthritis (PsA),Approved,7/6/17,"Approval announced July 6, 2017.",6
NVS,First-line use in patients with ALK+ metastatic NSCLC,Approved,5/26/17,"Priority review granted February 22, 2017. Approval announced May 26, 2017.",6
ABBV,Psoriatic Arthritis,Phase 3,9/30/21,"Phase 3 52-week data from open-label extension showed that 58% of patients achieved American College of Rheumatology 20 (ACR20) response. Among patients initially treated with risankizumab, 32% achieved ACR50 response, and 17% achieved ACR70 response at one year. Three major adverse cardiac events (MACE) were reported, noted September 30, 2021.",5
BMY,Second-line Small cell lung cancer (SCLC),Phase 3,10/12/18,"Phase 3 data released October 12, 2018 - primary endpoint not met.",4
ACER,Urea cycle disorder (UCD),Approved,12/23/22,"Approved December 23, 2022.",6
IBRX,Various tumors,Phase 2,5/20/21,Overall response rate 8% noted in ASCO abstract.,4
BLRX,Consolidation treatment for AML cancer patients,Phase 2b,11/23/20,"Phase 2b trial did not meet primary endpoint - November 23, 2020.",4
LGND,Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS),Phase 1b,7/18/23,"Phase 1b expansion trial data reported that one patient remained on study with sustained complete response for 8 cycles, noted July 18, 2023.",5
BMY,Gastric cancer,Approved,4/16/21,"FDA approval announced April 16, 2021.",6
NVCR,Mesothelioma,Phase 2,4/17/18,"Phase 2 top-line data released April 17, 2018 noted improvements in overall survival and progression free survival. Data to be presented at upcoming conference.",6
ANIP,Mycobacterium avium complex,Approved,12/21/21,"Approved December 21, 2021.",6
KROS,Iron deficiency anemia,Phase 2,12/12/22,"Phase 2 preliminary data reported that KER-047 was generally well tolerated by this patient, with no serious adverse events or dose-limiting toxicities, noted December 12, 2022.",6
AZN,Eosinophilic granulomatosis with polyangiitis (EGPA),Phase 3,9/11/23,"Phase 3 met the primary endpoint, noted September 11, 2023.",5
LBPS,Asthma,Phase 1/2,5/17/22,"Phase 1/2 data reported that 83.3% of subjects receiving treatment had improved ACQ-6 scores compared to 56.3% of those receiving placebo, noted May 17, 2022.",6
GILD,HIV,Phase 2/3,2/16/22,"Phase 2/3 one-year results reported that 83%of participants receiving lenacapavir in combination with an optimized background regimen achieved an undetectable viral load at Week 52, noted February 16, 2022.",6
LLY,"HR+, HER2-locoregionally recurrent or metastatic breast cancer (MBC)",Approved,3/26/18,"Approval announced February 26, 2018.",6
MRTX,Renal cell carcinoma (RCC),Phase 1/2,2/15/20,"Phase 2 updated data presented at ASCO GU 2020. 15/38 (39%) partial response rate, PFS 10.3 months - February 15, 2020.",5
TGTX,Follicular lymphoma (FL),Approved,2/5/21,"FDA approval announced February 5, 2021.",6
NVS,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 3,6/9/23,"Phase 3 data presented at EHA showed that monotherapy significantly improved Hb levels vs. SoC without the need for RBCTs. By maintaining intravascular hemolysis control and reducing C3 deposition leading to EVH, IPTA monotherapy led to an expected increase in PNH RBC survival to the extent that they formed >90% of the total RBC population, noted June 9, 2023",6
ADAG,Solid tumors,Phase 1/2,4/18/23,"Phase 1/2 trial data presented at AACR demonstrated that combination was well-tolerated up to 10 mg/kg Q3W or Q6W and with repeat dosing beyond 4 cycles, noted April 18, 2023.",6
SRPT,Duchenne muscular dystrophy,Phase 2,7/6/22,"Additional data reported that treated patients improved 3.1 points (unadjusted means) and 2.3 points (least squared means) in NSAA total scores from baseline, noted July 6, 2022.",6
CYTO,Vertigo,Phase 2,8/30/22,"Phase 2 data reported that trial met primary endpoint, noted August 30, 2022.",5
OCUL,Wet Age-related Macular Degeneration (AMD),Phase 1,6/10/23,"Phase 1 topline data presented at the Clinical Trials at the Summit 2023 Meeting maintained vision and CSFT comparable to aflibercept every eight weeks, with 89% reduction in treatment burden over a 12-month period,noted June 10, 2023",6
AZN,Third line HER2+ breast cancer,Phase 2,9/18/21,"Updated Phase 2 data showed an ORR of 62.0%, including one additional CR (7.1%). The median PFS was 19.4 months, September 18, 2021.",6
AQST,Amyotrophic Lateral Sclerosis,Approved,11/23/19,"FDA Approval announced November 22, 2019.",6
SGEN,Urothelial cancer,Approved,12/18/19,"FDA Approval announced December 18, 2019.",6
RMTI,Anemia,Approved,1/26/15,"Approved January 26, 2015.",6
LPCN,Testosterone replacement therapy (TRT),Approved,3/29/22,"Approved March 29, 2022.",6
RHHBY,1L Non-Small Cell Lung Cancer (NSCLC) PD-L1 high,Phase 3,8/23/23,"Phase 3 data reported that median overall survival estimates of 22.9 months in the tiragolumab plus Tecentriq arm, and 16.7 months in the Tecentriq monotherapy arm, noted August 23, 2023.",5
PFE,Nonmetastatic Prostate Cancer (nmCSPC),Phase 3,5/1/23,"Additional Phase 3 data reported that treatment reduced the risk of metastasis or death by 58% versus placebo plus leuprolide, noted May 1, 202",6
MRK,IIB or IIC Melanoma,Approved,12/3/21,"Approved December 3, 2021.",6
MYOV,Endometriosis,Phase 3,7/28/22,"Phase 3 data reported that trials met both co-primary endpoints with 75% of women in the relugolix combination therapy group in both studies achieving a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at Week 24, respectively, noted July 28, 2022.",6
INCY,Atopic dermatitis (AD) in children (age ≥2 to <12 years),Phase 3,10/13/23,"Phase 3 8- week analysis data shared at EADV in children (age ≥2 to <12 years) with atopic dermatitis (AD) achieved significant efficacy, as defined by the Investigator's Global Assessment-treatment success (IGA-TS), following eight weeks of treatment, noted October 13, 2023.",6
MRK,Hepatocellular Carcinoma,CRL,7/8/20,"CRL issued July 8, 2020.",5
MRK,Locally advanced resectable gastric and gastroesophageal junction (GEJ) adenocarcinoma,Phase 3,6/20/23,"Phase 3 study met one of its primary endpoints of pathological complete response (pCR) rate and demonstrated a statistically significant improvement in pCR rates compared with chemotherapy alone, noted June 20, 2023.",6
RLYB,Fetal and neonatal alloimmune thrombocytopenia (FNAIT),Phase 1b,6/24/23,"Phase 1b data presented at ISTH showed that subcutaneous administration produced a dose-dependent, rapid and complete elimination of transfused HPA-1a positive platelets in HPA-1a negative subjects, with both doses meeting the prespecified proof-of-concept criteria of ≥90% reduction in mean platelet elimination half-life. Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo, noted June 24, 2023.",6
ABBV,Acute bacterial skin and skin structure infections (ABSSSI) - pediatric,Approved,7/23/21,"FDA approval announced July 23, 2021.",6
VTGN,Adjunctive Treatment Major Depressive Disorder,Phase 2,11/14/19,"Phase 2 data did not meet primary endpoint - November 14, 2019.",4
ABUS,Coronavirus,Phase 1,9/11/23,"Phase 1 trial discontinued, noted September 11, 2023.",4
BMY,Melanoma,Approved,12/20/17,"Approval announced December 20, 2017.",6
CING,ADHD,Phase 3,9/9/23,"Phase 3 trial full results reported improvements on the PERMP scores with a trend towards statistical significance for PERMP ratings over the 16-hour period, noted September 9, 2023.",6
TEVA,Migraine,Approved,9/14/18,"FDA Approval announced September 14, 2018.",6
ABUS,Hepatitis B virus (HBV),Phase 2,11/1/22,"Phase 2 preliminary data from 65 patients randomized to receive AB-729+VBR+NA (n=32), AB-729+NA (n=17) or VBR+NA (n=16) for 48 weeks showed that adding VBR to AB-729+NA does not result in greater on-treatment improvements in markers of HBV infection as compared to AB-729+NA alone, noted November 1, 2022.",6
GILD,Refractory metastatic triple-negative breast cancer (TNBC),Phase 3,6/6/22,"Phase 3 final results reported that Trodelvy improved median progression-free survival versus physicians' choice of chemotherapy to 4.8 vs. 1.7 months and extended median overall survival (OS) by almost five months, noted June 6, 2022.",6
AZN,Chronic obstructive pulmonary disease (COPD),CRL,10/1/19,"CRL announced October 1, 2019.",5
JNJ,generalized myasthenia gravis (gMG),Phase 2,6/15/20,"Phase 2 trial met primary endpoint - June 15, 2020.",6
AZN,"HR Positive, HER2 Negative Breast Cancer",Phase 3,10/23/23,"Phase 3 data presented at ESMO showed reduction of the risk of disease progression or death by 37%, providing a 2-month median PFS benefit, and was well tolerated in post-endocrine therapy setting, noted October 23, 2023.",6
LLY,RET Fusion-Positive Solid Tumors,Approved,9/21/22,"Approved September 21, 2022.",6
JNJ,Psoriatic Arthritis,Approved,7/14/20,"FDA Approval announced July 14, 2020.",6
ONTX,Recessive Dystrophic Epidermolysis Bullosa-Associated Squamous Cell Carcinoma,Phase 2,10/12/23,"Phase 2 data shared at the EADV reported that two patients achieved complete cutaneous responses of 12 months or more, one patient experienced metastatic disease progression, and one patient remains on study, noted October 12, 2023.",6
EXEL,Renal Cell Carcinoma,Phase 3,3/3/23,"Phase 3 trial did not meet its primary endpoint, noted March 3, 2023.",6
RARE,X-Linked Hypophosphatemia (XLH),Approved,4/17/18,"Approval announced April 17, 2018.",6
GLPG,Ulcerative Colitis,Phase 2,7/14/21,"Phase 2 data released July 14, 2021. No differentiation from placebo.",4
KPTI,Myelofibrosis,Phase 2,12/11/21,"Additional Phase 2 results reported that of the ten patients who were on treatment for at least 24 weeks, four (40%) patients achieved SVR of ≥35% and six (60%) patients achieved SVR of ≥25%, noted December 11, 2021.",6
BSGM,COVID-19,Phase 2,10/26/20,"Phase 2 trial halted - October 26, 2020. Unlikely that the trial would meet its primary safety endpoints.",4
PHVS,Hereditary angioedema,Phase 1,6/26/23,"Phase 1 clinical hold lifted by FDA on June 26, 2023.",5
JNJ,Alzheimer’s disease,Phase 2,5/18/18,"Announced May 18, 2018 that dosing will be stopped.",4
NOVN,Fungal infections,Phase 2,4/12/17,"Phase 2 data released April 12, 2017. Primary endpoint met.",6
IOBT,Advanced melanoma,Phase 3,6/14/23,"Phase 3 trial protocol calls for an interim analysis of overall response rate (ORR) one year after 225 patients have been randomized. Additionally the number of patients to be enrolled has been increased to 380 patients, which could potentially accelerate the time to reach the primary endpoint of progression free survival, noted June 14, 2023.",6
OBSV,Pre-term labor,Phase 2a,11/16/20,"Phase 2a data released November 16, 2020. 7/56 (12.5%) of women receiving ebopiprant delivered within 48 hours of starting treatment compared to 12/55 (21.8%) receiving placebo (OR 90% CI: 0.52 (0.22, 1.23)).",6
TAK,Multiple myeloma,Phase 3,3/10/20,"Phase 3 trial did not meet primary endpoint - March 10, 2020.",4
MRK,Cutaneous squamous cell carcinoma (cSCC),Approved,6/24/20,"FDA approval announced June 24, 2020.",6
EXAI,Advanced Solid Tumors,Phase 1/2,7/10/23,"Phase 1/2 first patient enrolled, noted July 10, 2023.",5
JNJ,Pediatric Chronic Graft-Versus-Host Disease,Approved,8/24/22,"Approved August 24, 2022.",6
MRNS,Postpartum depression,Phase 2,7/23/19,"Phase 2 negative data released July 23, 2019.",4
JNJ,Major depressive disorder (MDD),Approved,8/3/20,"FDA approval announced August 3, 2020.",6
LVTX,Castration-resistant prostate cancer (mCRPC),Phase 1/2,6/14/23,"Phase 1/2 trial will continue as lead program, noted June 14, 2023.",5
LGND,Pneumococcal conjugate vaccine,Phase 3,7/27/23,"Phase 3 trial met primary endpoint, noted July 27, 2023.",5
MRSN,Platinum-sensitive Ovarian Cancer,Phase 1,6/15/23,"A clinical hold placed due to bleeding events, noted June 15, 2023.",5
PFE,COVID-19 Vaccine (Omicron Booster),Approved,9/11/23,"FDA booster approved on September 11, 2023.",6
AZN,BRCAm HER2-negative breast cancer,Approved,3/11/22,"Approved March 11, 2022.",6
NCNA,Solid tumors,Phase 2,10/16/23,"Phase 1/2 part 2 for both as a monotherapy in solid tumors and in combination with the anti-PD-1 therapy pembrolizumab in patients with metastatic cutaneous melanoma showed that treatment has been well tolerated both as a monotherapy and in combination with pembrolizumab. Encouraging signs of efficacy, including tumor volume reductions and prolonged time on treatment have been observed in both the monotherapy and combination cohorts, noted October 16, 2023.",6
REGN,Basal cell carcinoma (BCC),Approved,2/9/21,"FDA approval announced February 9, 2021.",6
SRNE,Lumbosacral radicular pain (sciatica),Phase 3,5/5/22,"Phase 3 data presented at ASIPP reported that treatment reduced sciatica pain with an extended effect for up to 99 days following a single injection, noted May 5, 2022.",6
SUPN,Epilepsy,Approved,8/19/13,"Approved Aug 19, 2013.",6
IMUX,"Celiac disease, healthy volunteers",Phase 1,10/16/23,"Phase 1 data reported that treatment has been well tolerated, with no systematic clinically relevant findings relative to safety and tolerability, noted October 16, 2023.",6
ANVS,Parkinson's Disease (PD),Phase 3,6/20/23,"Phase 3 trial completed and expected to be concluded in November 2023, DSMB recommended the company to continue the trial as originally designed, noted June 20, 2023.",5
AVXL,Parkinson’s Disease Dementia (PDD),Phase 2,3/30/23,"Phase 2 study data met primary and key secondary endpoints, noted March 30, 2023.",5
FGEN,Duchenne muscular dystrophy,Phase 3,6/7/23,"Phase 3 trial did not meet its primary endpoint, noted June 7, 2023.",6
SGEN,HER2+ Metastatic Breast Cancer (MBC),Approved,4/17/20,"FDA Approval announced April 17, 2020.",6
ETNB,Nonalcoholic Steatohepatitis (NASH),Phase 2b,9/21/23,"Phase 2b trial received Breakthrough Therapy Designation (BTD) granted by the FDA, noted September 21, 2023.",5
OCGN,Graft Versus Host Disease,Phase 3,6/1/20,Phase 3 trial stopped early due to insufficient efficacy.,4
VRDN,"Healthy volunteers, thyroid eye disease",Phase 1,11/14/22,"Phase 1 data reported that treatment was well tolerated with no reported serious adverse events, noted November 14, 2022.",6
PXMD,Human African Trypanosomiasis (HAT),Phase 2b,7/24/23,"Phase 2b trial met its primary endpoint, noted July 24, 2023.",5
NRIX,"B-cell malignancies, chronic lymphocytic leukemia (CLL)",Phase 1b,11/2/23,"Phase 1b data from ASH abstract reported that 9 PRs/PR with rebound lymphocytosis; additionally, 11 patients had SD at the time of data cut-off and 4 had PD, noted November 2, 2023.",5
TTNP,Parkinson's disease,Phase 1/2,7/2/18,"DSMB recommended trial to continue - July 2, 2018 but further enrollment has been postponed due to lack of resources.",4
JNJ,Multiple Myeloma,Phase 3,6/5/23,"Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023",6
TARS,Demodex Blepharitis,Approved,7/25/23,"FDA approved on July 25, 2023.",6
MRNA,Propionic Acidemia,Phase 1/2,5/19/23,"Phase 1/2 interim data presented at the 2023 ASGCT showed that mRNA-3927 has been generally well-tolerated at the doses administered, with encouraging early signs of dose-dependent pharmacology and potential clinical benefits, noted May 19, 2023.",6
SUPN,Motor fluctuations in Parkinson’s disease (PD),CRL,10/10/22,"CRL announced October 10, 2022.",5
LLY,Ulcerative Colitis,CRL,4/13/23,"CRL announced April 13, 2023.",5
LLY,Non-radiographic axial spondyloarthritis,Approved,6/1/20,"FDA Approval announced June 1, 2020.",6
EXEL,Castration-Resistant Prostate Cancer,Phase 3,8/21/23,"Phase 3 data reported that the trial met one of two primary endpoints, demonstrating a statistically significant improvement in progression-free survival (PFS) at the primary analysis, noted August 21, 2023.",6
CARA,Pruritus associated primary biliary cholangitis (PBC),Phase 2,11/7/22,"Phase 2 data reported no unexpected adverse events, noted November 7, 2022.",5
DCPH,Tenosynovial Giant Cell tumor,Phase 3,10/30/23,"Phase 3 study met primary endpoint with objective response rate (ORR) at week 25 of 40% compared to 0% for placebo. The study also met all key secondary endpoints at Week 25 compared to placebo, noted October 30, 2023.",5
LMNL,Congenital plasminogen deficiency (C-PLGD),Approved,6/4/21,"FDA approval announced June 4, 2021.",6
BMY,Obstructive Hypertrophic Cardiomyopathy,Phase 2/3,4/3/22,"Phase 2/3 results from EXPLORER-LTE cohort reported that resting LVOT gradient decreased from baseline by an average of -35.6 mmHg ± 32.6 mmHg at Week 48, with 4.3% of participants discontinued treatment, noted April 3, 2022.",6
LLY,Non-small cell lung cancer,Approved,5/29/20,"FDA Approval announced May 29, 2020.",6
VNDA,Social and Performance Anxiety,Phase 2,12/2/22,"Phase 2 trial results expected end of 2022, Phase 2 is full enrolled as of August 3, 2022. Phase 2 results showed that participants who received VQW-765 showed numerically lower stress levels compared to those who received placebo. In particular, female participants (approximately 70% of the total participants) reported a larger magnitude and statistically significant response, noted December 2, 2022.",6
PFE,HR+/HER2- advanced breast cancer in men,Approved,4/4/19,"FDA approval of sNDA announced April 4, 2019.",6
BIIB,Major Depressive Disorder (MDD),Phase 3,2/16/22,"Phase 3 trial met its primary and key secondary endpoints, comparing a 50 mg co-initiated with standard of care antidepressant vs. standard of care co-initiated with placebo. At the Day 3 primary endpoint, dosing with a standard of care antidepressant showed a statistically significant reduction in depressive symptoms.",6
ALDX,Chronic Cough,Phase 2,6/27/23,"Phase 2 trial demonstrated statistically significant reduction in cough frequency following administration of ADX‑629 relative to placebo, noted June 27, 2023.",6
BMY,Relapsed/Refractory Multiple Myeloma (RRMM),Phase 1,12/12/22,"Phase 1 preliminary results reported ORR was 39.6%, with 2 (2.0%) stringent complete responses, 3 (3.0%) complete responses, 18 (17.8%) very good partial responses, and 17 (16.8%) partial responses, noted December 12, 2022.",5
CGEN,Advanced cancer,Phase 1,11/12/21,"Phase 1 preliminary data from 18 patients enrolled demonstrated a favorable safety profile and that it was well-tolerated. However, a maximum tolerated dose was not reached, best response of stable disease (SD) was reported in 9 patients (50%), with 6 patients (67%) having confirmed SD and 3 patients (17%) with SD of at least 6 months, noted November 12, 2021.",6
HCM,Non-small cell lung cancer (NSCLC),Phase 2,4/19/23,"Phase 2 poster reported that the median PFS with vs without clearance was 11.0 vs 4.1 months in pts with IHC90+ or FISH10+ tumors, noted April 19, 2023.",5
ALLK,Eosinophilic Gastritis and/or eosinophilic duodenitis (EoD),Phase 3,12/21/21,"Phase 3 data reported that the trial met its histological primary endpoint but did not meet patient reported symptomatic primary endpoint, noted December 21, 2021.",6
ANAB,Mismatch repair deficient (dMMR) solid tumors,Approved,8/17/21,"FDA Approval announced August 27, 2021.",6
ABUS,Chronic Hepatitis B,Phase 2a,6/21/23,"Phase 2a preliminary data presented at EASL data suggested that the addition of IFN to AB-729 treatment was generally well tolerated and appears to result in continued HBsAg declines in some patients, noted June 21, 2023.",6
RHHBY,Spinal Muscular Atrophy (SMA) Type 1,Approved,8/7/20,"FDA Approval announced August 7, 2020.",6
ABIO,COVID-19,Phase 2b,3/31/22,"Phase 2b top-line data reported that neither doses met the primary endpoint, noted March 31, 2022.",5
NVCR,Ovarian cancer,Phase 3,8/28/23,"Phase 3 trial did not meet its primary endpoint in the final analysis, noted August 28, 2023.",6
BMY,Multiple myeloma (MM) - autologous stem cell transplant (ASCT),Approved,2/22/17,"Approved February 22, 2017.",6
ARGX,Generalized Myasthenia Gravis (gMG),Approved,6/20/23,"FDA Approved on June 20, 2023.",6
BMY,Non-transfusion-dependent beta-thalassemia,Phase 2,6/11/21,"Phase 2 data presented at EHA meeting June 11, 2021. 77.7% of patients achieved a hemoglobin increase compared to 0% in the placebo arm.",6
LLY,Rheumatoid arthritis,Approved,6/1/18,"FDA approval announced for low dose only - June 1, 2018.",6
LLY,Inherited Alzheimer's disease,Phase 2/3,2/10/20,Phase 3 trial did not meet primary endpoint.,4
STTK,Solid Tumors or Lymphomas,Phase 1,11/9/21,"Phase 1 dose escalation data reported one confirmed partial response (PR), and a second unconfirmed PR, both in patients with PD-1/L1 inhibitor pretreated non-cutaneous melanoma, noted November 9, 2021.",5
SNY,Coronavirus COVID-19,Phase 3,7/2/20,"Phase 3 trial did not meet primary endpoint - July 2, 2020.",4
ALNA,Gout in chronic kidney disease (CKD),Phase 2a,8/10/22,"Cohort A and B initial data reported that the data was not statistically significant, noted August 10, 2022.",5
OCS,Inflammation and pain following ocular surgery,Phase 3,8/8/23,"Phase 3 trial met both primary endpoints, noted August 8, 2023.",5
BNTX,Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine,Approved,12/8/22,"EUA granted for children under 5 December 8, 2022.",5
RHHBY,Pemphigus vulgaris,Approved,6/7/18,"Approval announced June 7, 2018.",6
LXRX,Type 2 diabetes,Phase 3,11/5/22,"Phase 3 data presented at ASN reported statistically significant reductions in UACR regardless of albuminuria status, noted November 5, 2022.",6
PFE,Ulcerative colitis,Approved,5/30/18,"Approval announced May 30, 2018.",6
SELB,Chronic Refractory Gout,Phase 3,3/21/23,"Phase 3 top-line data reported that both trials met their primary endpoints, noted March 21, 2023.",5
BIIB,Major depressive disorder (MDD),CRL,8/4/23,"CRL issued August 4, 2023.",5
JNJ,Multiple myeloma - candidates for autologous stem cell transplant (ASCT),Approved,9/26/19,"FDA Approval announced September 26, 2019.",6
GMAB,Cervical Cancer,Phase 3,10/23/23,"Additional data presented at ESMO that the overall survival (OS) was statistically significantly prolonged with TIVDAK, demonstrating a 30% reduction in the risk of death compared with chemotherapy, noted October 22, 2023.",6
OMER,IgA nephropathy,Phase 3,10/16/23,"Phase 3 9-month data did not meet its primary endpoint, as a result Omeros will not submit an application for approval of narsoplimab in this indication and will discontinue the ARTEMIS-IGAN clinical trial, noted October 16, 2023.",6
CRIS,Basal Cell Carcinoma,Approved,1/30/12,"Approved January 30, 2012.",6
FGEN,Idiopathic pulmonary fibrosis,Phase 3,6/26/23,"Phase 3 study did not meet primary endpoints, noted June 26, 2023.",6
MRK,Pneumococcal Vaccine in children,Approved,6/22/22,"Approved June 22, 2022.",6
MRNA,COVID-19 vaccine,Approved,1/31/22,"Full approval January 31, 2022.",6
ONCS,Triple negative breast cancer (TNBC),Phase 2,12/12/19,"Phase 2 interim data at SABCS December 12, 2019 noted ORR 28.5%.",5
LGVN,Alzheimer's Disease,Phase 2a,10/5/23,"Phase 2a topline data reported that trial met primary endpoints of safety across all study groups, noted October 5, 2023.",5
LNTH,Chronic pain,Approved,7/19/16,Approved July 19 2016.,6
MRK,House dust mite allergies,Approved,3/1/17,"Approved March 1, 2017.",6
MACK,Pancreatic cancer,Phase 3,11/9/22,"Phase 3 data met its primary endpoint demonstrating clinically meaningful and statistically significant improvement in overall survival compared to nab-paclitaxel plus gemcitabine in 770 previously untreated. Key secondary efficacy outcomes of progression-free survival (PFS) also showed significant improvement over the comparator arm, noted November 9, 2022.",6
RHHBY,Solid tumors with loss of CDKN2A Gene,Phase 1,5/30/23,"Phase 1 basket trial to be terminated, noted May 5, 2023.",4
FRLN,Gaucher disease,Phase 1/2,10/25/23,"Phase 1/2 results shared at the ESGCT reported no infusion reactions and no serious adverse events, noted October 25, 2023.",5
PCRX,Lower extremity nerve block,Phase 3,9/21/22,"Phase 3 trial data achieved the study's primary endpoint by demonstrating a statistically significant reduction in cumulative pain scores from 0 to 96 hours compared with bupivacaine HCl, noted September 21, 2022.",6
MRK,MSI-H/dMMR Advanced Endometrial Carcinoma,Approved,3/21/22,"Approved March 21, 2022.",6
XFOR,WHIM syndrome,Phase 3,5/16/23,"Phase 3 new data reported statistically significant reduction (~60%) in annualized infection rate versus placebo with 29% (5 of 17) of those on placebo experienced Grade 3 or higher infections, whereas only 7%, (1 of 14) of those on mavorixafor experiencing a Grade 3 or higher infection, noted May 16, 2023.",6
BNTX,Solid tumors,Phase 1/2,10/23/23,"Phase 1/2 additional data presented at ESMO demonstrated that the application of CARVac increases the persistence of the adoptively transferred autologous CAR-T cells, noted October 23, 2023.",6
SAGE,Alzheimer’s disease (AD) cognitive dysfunction and mild dementia,Phase 2,4/1/22,"Phase 2 study was well-tolerated and demonstrated improvement across multiple tests of executive performance as well as improvement on key tests of learning and memory, noted April 1, 2022.",6
PFE,"Claudin-18 isoform 2-positive (CLDN18.2+), HER2−, locally advanced (LA) unresectable or metastatic gastric or gastroesophageal junction (mG/GEJ) adenocarcinoma",Phase 3,10/21/23,"Phase 3 data presented at ESMO reported that median PFS was 11.0 vs 8.9 mo and median OS was 18.2 vs 15.6 mo, noted October 21, 2023.",6
LLY,Heart failure with preserved Ejection Fraction (HFpEF),Approved,2/24/22,"Approved February 24, 2022.",6
IONS,Hypertriglyceridemia,Phase 3,11/13/21,"Phase 3 data reported a significant reduction in total TRL particle (TRLP) concentration by 51%. Total LDL particle (LDLP) concentration was not changed, small LDLP numerically decreased by 39% while medium LDLP increased by 187%, noted November 13, 2021.",6
BMY,Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia,Approved,1/2/19,"FDA Approval announced January 2, 2019.",6
MRKR,Acute Myeloid Leukemia (AML),Phase 2,2/16/22,"Phase 2 data reported that one MRD positive patient converted to MRD negative following treatment, with no reported dose limiting toxicities, noted February 16, 2022.",6
AMGN,Coronary Artery Disease (CAD),Phase 3,3/4/23,"Phase 3 data presented at the ACC.23/WCC reported that treatment significantly reduced both first and recurrent CV events, with more than double the number of total events prevented as compared with 1st events only, noted March 4, 2023.",6
BMY,Plaque psoriasis,Approved,9/10/22,"FDA Approval on September 10, 2022.",6
LLY,COVID-19,Phase 3,4/8/21,"Phase 3 trial did not meet primary endpoint - April 8, 2021.",4
MRK,"Personalized cancer vaccine, high-risk melanoma",Phase 2,10/23/23,"Phase 2 additional data presented at ESMO reported that local recurrence was more common in MRs; MNRs had more distant events. This trend was supported by stronger separation in DMFS vs RFS between MRs and MNRs, noted October 23, 2023.",6
CBIO,Hemophilia B,Phase 2b,6/15/20,"Phase 2b data noted after 28 days of daily SQ dosing of DalcA achieved protective target FIX levels of >12% in all participants, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds.",6
RHHBY,Chronic rhinosinusitis with nasal polyps (CRSwNP),Approved,12/1/20,"FDA approval announced December 1, 2020.",6
ALVR,BK viremia,Phase 2,2/15/23,"Phase 2 final results showed an even greater antiviral effect with treatment in patients with BK viral load ≥10,000 copies/mL at screening and in the biweekly posoleucel dosing group, identifying a dosing regimen and patient population to advance into a future trial., noted February 15, 2023.",6
INCY,Myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement,Approved,8/26/22,"Approved August 26, 2022.",6
ABBV,Waldenström’s Macroglobulinemia,Approved,8/27/18,"FDA approval announced August 27, 2018.",6
XERS,Post-Bariatric Hypoglycemia,Phase 2b,5/22/20,"Phase 2b outpatient data released May 22, 2020.",5
BXRX,Hemophilia A,Phase 1/2,9/28/23,"FDA granted Orphan Drug Designation, noted September 28, 2023.",5
MRK,Hepatocellular carcinoma (HCC),Approved,11/9/18,"FDA approval announced November 9, 2018.",6
ZYME,HER2-positive breast cancer,Phase 1/2,5/26/22,"Phase 1b/2 data released in abstract reported that the cORR was 90.5% and the six-month progression-free survival rate was 95.2%, noted May 26, 2022.",5
BIIB,CKD Patients at Risk of Rapid Progression,Phase 2,5/10/23,"Program discontinued, noted May 10, 2023.",4
DCPH,Gastrointestinal stromal tumor (GIST),Phase 1/2,11/30/21,"Phase 1b/2 trial discontinued, noted November 30, 2021.",4
RAIN,Liposarcoma,Phase 3,5/22/23,"Phase 3 trial did not meet its primary endpoint, noted May 22, 2023.",6
NGM,Pancreatic Cancer,Phase 1b,9/16/21,"Phase 1b data had all 6 patients demonstrated disease control, 3 patients had partial responses (PR), and 3 stable disease (SD). 2 patients discontinued early due to gemcitabine/Nab-paclitaxel toxicity and/or progression of the underlying disease. Phase 1a monotherapy cohort reported 3 patients (30%) in the 30 mg dosing and 2 patients (20%) in the 100 mg dosing had stable disease (SD), September 16, 2021.",5
MRK,Advanced unresectable or metastatic gastric (GEJ) adenocarcinoma,Phase 3,2/16/23,"Phase 3 trial combination data showed statistically significant improvements in progression-free survival and objective response rate, noted February 16, 2023.",6
SNY,Basal cell carcinoma (BCC),Approved,2/9/21,"FDA approval announced February 9, 2021.",6
CYTK,Amyotrophic lateral sclerosis (ALS),Phase 2,12/8/22,hase 2 data presented at Virtual 33rd International Symposium on ALS/MND demonstrated predicted survival risk score strongly correlated with decline in ALSFRS-R.,6
BLU,Refractory Chronic Cough,Phase 2b,2/26/23,"Phase 2b data shared at AAAAI reported that similar burdens are also reported across all three LCQ subdomains in the main vs exploratory populations, noted February 26, 2023.",5
ATAI,Treatment Resistant Depression (TRD),Phase 2b,11/15/21,Phase 2b trial met primary endpoint noted November 2021.,6
CAPR,Duchenne Muscular Dystrophy (DMD),Phase 2,10/10/23,"Phase 2 data presented at the World Muscle Society reported that the mean PUL 2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7-point decline on average observed over 24-months in the placebo, noted October 10, 2023.",6
SNY,Breast Cancer,Phase 2,4/28/22,"Phase 2 pivotal trial did not meet it primary endpoint, noted April 28, 2022.",6
ETON,Epilepsy,Approved,7/18/22,"Approved July 18, 2022.",6
MRK,Clostridium difficile infections (CDI),Approved,1/27/20,"FDA Approval announced January 27, 2020.",6
LPCN,Non-alcoholic fatty liver disease (NAFLD) / Non-alcoholic steatohepatitis (NASH),Phase 2,5/12/22,"OLE extension study results reported that treatment was well-tolerated with liver injury markers reduced, noted May 12, 2022.",6
ALPMY,Untreated locally advanced or metastatic urothelial cancer (la/mUC),Phase 3,10/23/23,"Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.",6
SNY,Cervical cancer,Phase 3,3/15/21,"Phase 3 trial to be stopped early due to sufficient efficacy. 12 months survival for Libtayo compared to 8.5 months for chemotherapy; hazard ratio (HR): 0.69 - March 15, 2021.",4
FGEN,Anemia in patients receiving concurrent chemotherapy treatment for non-myeloid malignancie,Phase 3,5/18/23,"Phase 3 trial met primary endpoint, noted May 18, 2023.",5
MRK,Colorectal cancer,Phase 3,4/7/23,"Phase 3 trial did not meet primary endpoint, noted April 7, 2023.",6
AZN,Non-Small Cell Lung Cancer,Phase 3,9/12/23,"Phase 3 data presented at WCLC reported statistically significant and clinically meaningful improvement in event-free survival and pathologic complete response with this IMFINZI-based regimen versus neoadjuvant chemotherapy alone followed by surgery, noted September 12, 2023.",6
FSTX,Head and neck cancer,Phase 2,8/11/22,"Phase 2 data reported that treatment was well tolerated and cleared the pre-determined futility, noted August 11, 2022.",6
BCDA,Heart Failure,Phase 3,9/5/23,"Phase 3 review determined that the study is unlikely to meet its primary three-tiered FS efficacy endpoint, as seen from the initial 102 randomized patient set assessed at 12-month follow-up, noted September 5, 2023.",6
MRTX,Urothelial Carcinoma (Bladder),Phase 2,9/18/20,Phase 2 at ESMO September 2020 noted objective response rate of 37% (1 complete response and 10 partial responses),5
VTRS,Asthma or chronic obstructive pulmonary disease (COPD),Approved,3/15/22,"Approved March 15, 2022.",6
RHHBY,Relapsed/Refractory Multiple Myeloma (RRMM),Phase 1,12/12/22,"Phase 1 data reported that the best overall response (BOR) achieved was: stringent complete response (sCR) in 7 patients, CR in 3 patients, very good partial response (VGPR) in 5 patients, and PR in 1 patient, noted December 12, 2022.",6
IFRX,Hidradenitis Suppurativa,Phase 2b,6/5/19,"Phase 2b data June 5, 2019 did not meet primary endpoint.",4
PFE,Uterine fibroids,Approved,5/26/21,"FDA approval announced May 26, 2021.",6
SRRK,Solid Tumors,Phase 1,11/3/23,"Phase 1 biomarker data presented at SITC showed promising anti-tumor activity in heavily pretreated clear cell renal cell carcinoma (ccRCC) patients. Objective response rate (ORR) of 21.4% and disease control rate of 57%, noted November 3, 2023.",6
CPRX,Duchenne Muscular Dystrophy (DMD),Approved,10/26/23,"Approved October 26, 2023.",6
CHRS,1L Metastatic Nasopharyngeal Carcinoma,Approved,10/30/23,"Approved October 27, 2023.",6
AMGN,Uncontrolled Gout,Approved,7/8/22,"Approved July 8, 2022.",6
CRIS,Acute Myeloid Leukemia / Myelodysplastic Syndromes (MDS),Phase 1/2,7/6/23,"FDA removed partial hold, noted July 6, 2023",5
ZLAB,Myasthenia gravis (MG),Approved,12/17/21,"Approved December 17, 2021.",6
DARE,Female Sexual Arousal Disorder (FSAD),Phase 2b,11/1/23,"Phase 2b exploratory data reported that trial achieved statistical significance and clinically meaningful improvement in Phase 2b co-primary endpoint assessing arousal sensation, and statistical significance in items from Phase 2b co-primary endpoint scale evaluating concern related to difficulties with sexual arousal in subset analyses, noted November 1, 2023.",6
PTCT,Nonsense mutation cystic fibrosis,Phase 3,3/2/17,"Phase 3 data released March 2, 2017 - endpoints not met. Development to be discontinued.",4
ALPMY,Nonmetastatic Prostate Cancer (nmCSPC),Phase 3,5/1/23,"Additional Phase 3 data reported that treatment reduced the risk of metastasis or death by 58% versus placebo plus leuprolide, noted May 1, 202",6
CHRS,Rheumatoid arthritis,Phase 3,1/11/16,"Phase 3 data released January 2016. Endpoints met. However, commercialization not possible in US before 2029 due to patent issues",5
IXHL,Obstructive sleep apnoea (OSA),Phase 2,6/3/22,Complete Phase 2 analysis concluded that treatment reduced primary endpoint apnoea hypopnea index relative to baseline at all three doses that were assessed. The low dose exhibited superior safety and efficacy metrics to mid and high doses. The low dose also reduced AHI by an average of 50.7% compared to baseline with 25% of participants experiencing a reduction in the apnoea hypopnea index of greater that 80%.,6
LGND,Osteoporosis,Approved,10/7/19,"FDA approval announced October 7, 2019.",6
GSK,COVID-19 antibody,Approved,5/26/21,"FDA Emergency Use Authorization filing approval announced May 26, 2021.",6
GSK,COVID-19 antibody,Approved,2/11/22,"EUA granted by FDA February 11, 2022.",5
RHHBY,Macular edema due to RVO,Approved,10/27/23,"Approved October 27, 2023.",6
AZN,HER2-overexpressing/amplified (HER2+) metastatic colorectal cancer (mCRC),Phase 2,6/5/23,"Phase 2 primary results to be presented at ASCO demonstrated positive antitumour activity and consistent safety in patients with previously treated HER2-positive metastatic colorectal cancer, noted June 5, 2023.",6
LLY,Alzheimer’s disease,Phase 3,7/17/23,"Phase 3 data presented at AAIC reported a 60% slowing of decline compared to placebo with nearly half of participants at earlier stage of disease on donanemab had no clinical progression at 1 year, noted July 17, 2023.",6
KRYS,Transglutaminase-1 Deficient Autosomal Recessive Congenital Ichthyosis (ARCI),Phase 2,7/1/21,"Phase 2 update announced July 1, 2021 - well tolerated with no adverse events.",6
SAGE,Major Depressive Disorder (MDD),Phase 3,2/16/22,"Phase 3 trial met its primary and key secondary endpoints, comparing a 50 mg co-initiated with standard of care antidepressant vs. standard of care co-initiated with placebo. At the Day 3 primary endpoint, dosing with a standard of care antidepressant showed a statistically significant reduction in depressive symptoms.",6
LNTH,Prostate cancer,Approved,5/27/21,"FDA Approval announced May 27, 2021.",6
SGEN,HER2 amplified metastatic colorectal cancer,Approved,1/19/23,"Approved January 19, 2023.",6
AZN,Untreated advanced or metastatic triple negative breast cancer (TNBC),Phase 1/2,10/23/23,"Phase 1b/2 data reported a confirmed objective response rate (ORR) of 79% (95% confidence interval [CI]: 67-88) including six complete responses (CRs) and 43 partial responses (PRs), noted October 23, 2023.",5
KRYS,Cystic Fibrosis,Phase 1,7/3/23,"Phase 1 dosing commenced, noted July 3, 2023",5
ENTA,COVID-19,Phase 2,5/8/23,"Phase 2 trial met primary endpoint, noted May 8, 2023.",5
TEVA,Cluster headache,Phase 3,6/15/18,"Phase 3 trial to be terminated due to lack of efficacy - noted June 15, 2018.",4
LLY,Nonsquamous NSCLC,CRL,3/24/22,"CRL announced March 24, 2022.",5
BHC,Bowel cleansing,Approved,5/7/18,"Approval announced May 7, 2018.",6
AUTL,B-Cell Acute Lymphoblastic Leukemia,Phase 2,6/2/23,"Phase 2 safety and efficacy data presented at ASCO showed that 76% of patients treated achieved a response (CR/CRi), primary endpoint has been met based on previously communicated interim analysis, noted June 2, 2023",6
RPTX,Solid Tumors,Phase 1,10/13/23,"Phase 1 initial data from Module 1 and 2 presented at plenary session at AACR-NCI-EORTC International Conference suggested treatment with lunresertib in combination with camonsertib could result in efficacy outcomes for patients in the gynecological cancer setting. In this area, we're still seeing unmet patient needs despite current therapies, noted October 13, 2023.",6
MIRM,"Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome",Phase 2,11/8/22,"Phase 2 data reported that treatment was well tolerated in infants with ALGS aged <1 year, with body weight of ≥2.5 kg, and TEAEs were mostly grade 1 in severity and unrelated to MRX, noted November 8, 2022.",6
BHC,Macular edema associated with uveitis,Approved,10/25/21,"Approved October 25, 2021.",6
VNDA,Smith-Magenis Syndrome,Approved,12/1/20,"FDA approval announced December 1, 2020.",6
NVCR,Non-small cell lung cancer (NSCLC),Phase 3,10/16/23,"Phase 3 data from ESMO abstract reported that quality of life data showed no statistically significant difference between patients who received TTFields therapy together with standard systemic therapies, noted October 16, 2023.",6
BTAI,Agitation associated with schizophrenia and bipolar disorders,Approved,4/6/22,"Approval announced April 6, 2022.",6
MTEM,Solid tumors,Phase 1,6/3/23,"Phase 1 interim efficacy and safety data from ASCO reported that all cohorts had reductions in peripheral PD-L1+ MDSCs, monocytes and dendritic cells after MT-6402 without effect on PD-L1- cells with the most depletion at the highest doses, noted June 3, 2023.",6
XNCR,"Solid tumors, prostate cancer",Phase 2,11/10/22,"Phase 2 data reported that prostate-specific antigen (PSA) reductions of more than 50% from baseline (PSA50) had been observed in three of nine patients, noted November 10, 2022.",6
URGN,Low grade non-muscle invasive bladder cancer (LG-NMIBC),Phase 3,7/27/23,"Phase 3 trial met primary endpoint with 79.2% rate of complete response at 3-months following the initial treatment, noted July 27, 2023.",6
TYME,Pancreatic cancer,Phase 2/3,6/10/21,"Phase 2/3 enrollment to be terminated and trial will be closed down. Company citing slower than expected enrollment for the decision - June 10, 2021.",4
CLVS,Ovarian cancer patients with a BRCA-like mutation,Approved,4/6/18,"Approval announced April 6, 2018.",6
MTCR,Type 2 Diabetes and Nonalcoholic steatohepatitis (NASH),Phase 2a,11/2/21,"Phase 2a top-line data released November 2, 2021. All regimens had favorable safety profiles and were generally well-tolerated, with no treatment-related serious adverse events (AEs). Mild-moderate pruritus was reported in 0%-6% of patients across all groups.",6
ALDX,Retinitis Pigmentosa,Phase 2,6/29/23,"Phase 2 top-line results presented on a webcast demonstrated statistically significant improvement in retinal function across a number of different physiological and psychophysical assessments, June 29, 2023.",6
BNTX,Resected pancreatic ductal adenocarcinoma (PDAC),Phase 1,6/5/22,"Phase 1 initial preliminary data readout from 16 vaccinated patients revealed that combination was well-tolerated. Only 1 of 16 patients (6%) developed a vaccine-related Grade 3 fever and hypertension, no other Grade 3 or higher adverse events were observed. In addition, the treatment induced de-novo, neoantigen-specific T cell response in half (8/16) of these patients from undetectable levels to large fractions of all blood T cells (median 2.9%). At an early median follow-up of 18 months, patients with de-novo immune response (n=8) had a significantly longer recurrence-free survival (RFS) as compared to those without vaccine-induced immune responses (n=8) (median not reached vs. 13.4 months, HR 0.08, 95% CI 0.01-0.4, P = 0.003)., noted June 5, 2022.",6
AMYT,Maintenance treatment of adult acromegaly,Phase 3,9/7/22,"Additional Phase 3 data reported that no new or increased safety signals were identified with a shorter time to initiation of Mycapssa, noted September 7, 2022.",5
TBPH,Crohn's Disease,Phase 2,11/17/21,"Phase 2 trial voluntarily discontinued, noted November 17, 2021",4
NVS,PIK3CA-Related Overgrowth Spectrum (PROS),Approved,4/6/22,"Approval announced April 6, 2022.",6
ACOR,Parkinson’s disease (PD),Phase 3,11/15/17,"Phase 3 data due 1Q 2018. Noted November 15, 2017 seven cases of sepsis, five of which were fatal.",5
BMY,Urothelial carcinoma,Phase 3,10/17/23,"Phase 3 data presented at ESMO reported that Opdivo in combination with cisplatin-based chemotherapy reduced the risk of death by 22%, demonstrating a median OS of 21.7 months vs. 18.9 months with chemotherapy alone, noted October 17, 2023.",6
BMY,Esophageal squamous cell carcinoma (ESCC),Approved,5/27/22,"Approved May 27, 2022.",6
BIIB,Idiopathic pulmonary fibrosis (IPF),Phase 2b,8/31/19,Phase 2b trial discontinued August 2019 due to safety concerns.,4
AZN,First-line ovarian cancer,Approved,5/8/20,"FDA Approval announced May 8, 2020.",6
MTNB,Cryptococcal meningitis,Phase 1/2,10/21/22,"Phase 1/2 cohort 4 data reported that CSF yeast clearance rate exceeded the prespecified primary endpoint, with an interim survival currently 90%, while the survival rate at Week 2 was 95%, noted October 21, 2022.",5
RLFTF,COVID-19,Phase 3,5/25/22,"Phase 3 DSMB review requested that randomization to aviptadil be stopped due to not meeting the futility guidelines, noted May 25, 2022.",6
GSK,Cancer - ovarian,Approved,3/27/17,"PDUFA date under priority review was June 30, 2017. Approved March 27, 2017.",6
BIIB,C9orf72-associated Amyotrophic Lateral Sclerosis (ALS),Phase 1,3/28/22,"Phase 1 study did not meet any secondary efficacy endpoints and it did not demonstrate clinical benefit. These results do not support further development, noted March 28, 2022.",6
TYME,Pancreatic Cancer,Phase 2/3,1/26/22,"Phase 2/3 additional data reported that overall survival for SM-88 as a monotherapy was lower compared to standard of care chemotherapies, noted January 26, 2022.",5
FBIO,Menkes disease,Phase 1/2,10/14/21,"Phase 1/2 data from both pre-specified primary and secondary efficacy analyses showed treatment with CUTX-101 demonstrated a significantly greater median overall survival (OS) compared to untreated historical control patients, noted October 14, 2021.",6
PCRX,Post surgical pain,Approved,4/6/18,"Approval announced April 6, 2018.",6
AMGN,Alzheimer’s Disease,Phase 2/3,7/11/19,Phase 2/3 trial discontinued due to lack of efficacy.,4
OPTN,Chronic sinusitis,Phase 3,7/8/22,"Additional Phase 3 analysis reported a greater improvement on the sinonasal outcomes test at week 24 for patients receiving XHANCE then placebo comparator, noted July 8, 2022.",6
CLDX,"Chronic inducible urticaria (CIndU), cold urticaria (ColdU) and symptomatic dermographism (SD)",Phase 1,6/10/23,"Phase 1 data reported at EAACI 2023 include safety data through 12 weeks were 56% patients achieved a complete response (negative test) with PCE provocation testing with just one dose of barzolvolimab and most responses remained durable through to week 12, noted June 10, 2023.",6
RHHBY,Squamous non-small cell lung cancer (NSCLC),Approved,3/18/19,"FDA approval announced March 18, 2019.",6
INFI,Urothelial cancer,Phase 2,8/9/22,"Phase 2 data reported that 45% of patients were alive compared to only 24% in the control arm, noted August 9, 2022.",5
AMPH,Asthma,CRL,12/27/16,"CRL issued December 27, 2016.",5
LSTA,Type 1 diabetes,Phase 2,2/13/19,"Phase 2 top-line data did not meet primary endpoint - February 13, 2019.",4
BCRX,Acute uncomplicated influenza to pediatric patients,Approved,9/21/17,"Approved September 21, 2017.",6
GILD,Pre-exposure prophylaxis,Approved,10/3/19,"FDA Approval announced October 3, 2019.",6
ETON,Methanol poisoning,CRL,6/28/23,"CRL Announced on June 28, 2023.",5
CMRX,H3 K27M-mutant Glioma,Phase 3,8/16/23,"Phase 3 data from survival analyses published in Cancer Discovery reported that ONC201 frontline treatment, administered post-radiation therapy, demonstrated a statistically significant increase in mOS from diagnosis versus historical controls, noted August 16, 2023.",6
LLY,Heart failure with reduced ejection fraction,Approved,8/18/21,"FDA approval announced August 18, 2021.",6
SLS,Ovarian cancer,Phase 1/2,11/10/22,"Phase 1/2 data noted a Median Overall Survival (OS) of 18.4 months compared to 13.8 months, noted November 10, 2022.",5
ATOS,Pre-menopausal women w/ early-stage estrogen receptor positive and Human Epidermal Growth Factor Receptor 2 negative breast cancer,Phase 2,6/12/23,"Phase 2 enrollment of Pharmacokinetic Run-In cohort completed, noted June 12, 2023.",5
GILD,Hepatitis delta virus (HDV),Phase 3,6/23/23,"Phase 3 continued treatment with Bulevirtide showed clinical benefit and sustained viral decline with prolonged therapy in patients originally considered partial- or non-responders, noted June 23, 2023.",6
CYTK,Hypertrophic cardiomyopathy (HCM),Phase 2,9/12/21,"Phase 2 full trial data resulted in statistically significant reductions from baseline compared to placebo in the average resting left ventricular (LV) outflow tract pressure gradient and the average post-Valsalva. The observed reductions in LVOT-G were dose dependent. Patients treated with aficamten in both Cohort 1 and Cohort 2 also experienced statistically significant reductions in NT-proBNP, noted September 12, 2021.",6
MORF,Ulcerative colitis,Phase 2a,9/23/23,"Phase 2a trial achieved the primary endpoint and demonstrated clinically meaningful improvements across secondary and exploratory measures, noted September 22, 2023.",6
ACHV,E-cigarette & vape cessation,Phase 2,4/20/23,"Phase 2 topline data reported a 2.6 times higher odds, or likelihood, to have quit vaping during the last 4 weeks of treatment compared to subjects who received placebo (p=0.035). The vaping cessation rate during weeks 9-12 was 31.8% for cytisinicline compared to 15.1% for placebo, noted April 20, 2023.",6
MRK,Charcot-Marie-Tooth disease (CMT),Phase 2,3/9/20,"Phase 2 data did not meet primary endpoint - March 9, 2020.",4
BMY,Advanced hematologic malignancies with an IDH2 mutation,Approved,8/1/17,"Approval announced August 1, 2017.",6
MGNX,Localized Prostate Cancer,Phase 2,9/16/21,"Phase 2 data showed 34% of patients had pre-prostatectomy PSA declines of >10%. At 1 year post-operatively, PSA0 rate was 66%. The median time to PSA recurrence was not reached, September 16, 2021.",6
GSK,HIV-1,Phase 3,2/23/23,"Phase 3b data reported that the trial achieved primary endpoint, noted February 23, 2023.",6
BGNE,Waldenström’s macroglobulinemia,Phase 1/2,6/12/20,"Phase 1/2 updated data at EHA 2020 noted overall response rate (ORR) was 80%, with a complete response (CR) rate of 15% and a partial response (PR) rate of 65%.",6
AMPH,Radionuclide myocardial perfusion imaging (MPI),Approved,5/24/22,"Approved May 24, 2022.",6
PHAT,Non-erosive reflux disease (NERD),Phase 3,1/8/23,"Phase 3 trial met primary endpoint in both 10 mg and 20 mg doses, noted January 8, 2023.",5
CALA,Non-small cell lung cancer (NSCLC) patients with genetic mutation NRF2/KEAP1,Phase 2,11/5/21,"Phase 2 interim analysis demonstrated lack of clinical benefit among patients treated. Trial termination, noted November 5, 2021.",4
ABBV,Facial Erythema (Redness) Associated with Rosacea,Approved,1/19/17,"Approved January 19, 2017.",6
GMAB,Newly Diagnosed Multiple Myeloma (NDMM),Approved,6/28/19,"FDA Approval announced June 28, 2019.",6
LEGN,Multiple myeloma,Approved,2/28/22,"Approved February 28, 2022.",6
ASRT,Colorectal cancer,Approved,4/29/11,"Approved April 29, 2011.",6
EBS,Anthrax Vaccine Adsorbed,Approved,11/24/15,"Approved November 24, 2015.",6
APRE,"TP53 mutant Myelodysplastic syndromes (MDS), Acute Myeloid Leukemia (AML)",Phase 2,12/14/21,"Phase 2 long-term data reported that at data-cutoff by ITT analysis, ORR was 69% and CR was 43% with a median follow up of 27.8 months, median OS was 11.8 months. Biallelic TP53 mutation or complex karyotype was significantly associated with higher CR rate, and the combination therapy was well tolerated in treated patients, noted December 14, 2021.",6
JAZZ,Pediatric narcolepsy patients with cataplexy,Approved,10/26/18,"FDA Approval announced October 26, 2018.",6
TAK,Hodgkin lymphoma and Anaplastic large cell lymphoma,Approved,8/19/11,"Approval announced August 19, 2011.",6
BLUE,Childhood cerebral adrenoleukodystrophy (CCALD),Phase 2/3,3/14/21,"Phase 2/3 updated data presented March 15, 2021. 90% of evaluable patients (27/30) alive and free of major functional disabilities at two years follow-up.",6
AQST,Parkinson’s Disease with motor fluctuations,Approved,5/21/20,"FDA approval announced May 21, 2020.",6
PRTC,"Healthy volunteers, neurological and neuropsychological conditions",Phase 1,2/14/23,"Phase 1 study achieved blood levels of allopregnanolone at or above those associated with therapeutic effect in postpartum depression1 and was generally well-tolerated, noted February 14, 2023.",6
NVO,Type 1 and type 2 diabetes,Phase 3,9/29/17,"Approved September 29, 2017.",6
TOVX,Constipation-Predominant Irritable Bowel Syndrome (C-IBS),Phase 2b,10/2/20,"Phase 2b interim futility analysis released October 2, 2020. Primary endpoint unlikely to be met. Trial to be discontinued.",4
IMVIQ,Diffuse Large B Cell Lymphoma (DLBCL),Phase 2,2/13/23,"Phase 2b initial response data showed that from 6/8 patients evaluable for efficacy; 3 patients showed confirmed complete responses, 1 patient was assessed with stable disease as best response and 2 patients were assessed with progressive disease as best response; and 2 patients with poor level of baseline functionality (ECOG ≥ 2) failed to stay on study through to the first scan and therefore could not be evaluated, noted February 13, 2023.",6
EGRX,Nonsquamous Non-Small Cell Lung Cancer (NSCLC) and Mesothelioma,Approved,10/27/17,"Tentative approval announced October 27, 2017. Full approval pending patent litigation with Eli Lilly.",5
NEXI,Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome (MDS) after allogeneic HCT,Phase 1/2,6/5/23,"Phase 1/2 clinical results expected to be shared at ASCO showed that treatment was well tolerated with a favorable safety profile, including no grade > 3 treatment related events as of May 2023, noted on June 5, 2023.",6
AMGN,Oral corticosteroid dependent asthma.,Phase 3,12/22/20,"Phase 3 trial did not meet primary endpoint - December 22, 2020.",4
LLY,Obesity or overweight with weight-related comorbidities,Phase 3,7/27/23,"Phase 3 trial met both co-primary endpoints, noted July 27, 2023.",5
VNDA,Motion sickness,Phase 3,5/25/23,"Phase 3 data reported that the 170 mg and 85 mg tradipitant doses were shown to be superior to placebo in preventing vomiting with only 18.3% and 19.5% of participants experiencing vomiting on tradipitant 170 mg and 85 mg respectively, as compared to 44.3% of participants on placebo, noted May 25, 2023.",6
CMPX,Biliary Tract Cancer (cholangiocarcinoma),Phase 2,1/19/23,"Phase 2 data presented at ASCO GI reported a 37.5% overall response rate (ORR) in 24 patients, and an ORR of 63.6% in a sub-group analysis, noted January 19, 2023.",6
ABBV,Plaque psoriasis,Phase 3,3/18/23,"Phase 3 long-term data reported that short- and long-term efficacy of psoriasis signs and symptoms (sPGA 0/1) at week 16 and week 52 in a difficult-to-treat population, with no new safety signals observed, noted March 18, 2023.",6
NERV,Schizophrenia,Phase 3,9/30/21,"Phase 3 results met all key pharmacokinetic (PK) objectives and the data demonstrate bioequivalence across the various formulations - noted September 30, 2021.",6
AKTX,Atopic keratoconjunctivitis,Phase 1/2,10/25/21,"Phase 1/2 early clinical data demonstrated a clear dose response effect. The optimal dose showed a greater reduction in inflammation score than cyclosporin and dexamethasone compared to control at day 10 (79% [nomacopan], 41% [cyclosporin], and 73% [dexamethasone]), noted October 25, 2021.",6
PFE,BRAFV600E-mutant metastatic non-small cell lung cancer (NSCLC),Approved,10/12/23,"Approved October 12, 2023.",6
SDGR,B-cell lymphoma,Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that treatment showed excellent potency in the biochemical assay and strong anti-proliferative effects on ABC-DLBCL cells, noted November 2, 2023.",6
IMUX,Ulcerative colitis,Phase 2,4/5/23,"Additional Phase 2 data reported a dose-linear increase in clinical remission as compared to placebo at week 50 with an exploratory statistical analysis confirming that the 30 mg dose of vidofludimus calcium was statistically superior in achieving clinical remission, with a 33.7% absolute improvement over placebo, noted April 5, 2023.",6
IRWD,Functional Constipation in pediatric patients aged 6-17,Approved,6/12/23,"FDA Approved on June 12, 2023.",6
SAGE,Postpartum Depression,Approved,8/4/23,"Approved August 4, 2023.",6
OPT,Wet age-related macular degeneration (AMD),Phase 2b,5/5/22,"Phase 2b Polypoidal Choroidal Vasculopathy subgroup data reported that the combination achieved superior visual acuity gains and anatomic improvements, noted May 5, 2022.",6
ABBV,Ulcerative Colitis,Phase 3,6/15/23,"Phase 3 trial met primary and secondary endpoints, noted June 15, 2023.",5
NVAX,COVID-19 vaccine (booster),Phase 3,11/8/22,"Phase 3 data reported that BA.1 vaccine candidate met its primary strain-change endpoint, noted November 8, 2022.",5
FWBI,COVID-19-related gastrointestinal (GI) infections,Phase 2,4/29/22,"Phase 2 top-line data reported that trial did not meet its primary endpoint, noted April 29, 2022.",6
ABBV,Psoriatic arthritis,Approved,12/15/21,"Approved December 15, 2021.",6
MRK,Pulmonary arterial hypertension (PAH),Phase 2,5/19/21,"Phase 2 update at the American Thoracic Society 2021 International Conference May 19, 2021. Generally well tolerated, improvements in resting and exercise hemodynamics at week 24 shown.",6
GMAB,Squamous cell carcinoma of the head and neck (SCCHN),Phase 2,4/17/23,"Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.",6
AERI,Macular edema due to RVO (retinal vein occlusion),Phase 2,10/7/21,"Phase 2 data displayed that the implant achieved targeted 6-month duration of efficacy, with improvements in BCVA and macular edema demonstrated for at least 6 months. Adverse events were consistent with other corticosteroid treatments, noted October 7, 2021.",6
RGNX,Mucopolysaccharidosis Type I (MPS I),Phase 1/2,8/30/23,"Phase 1/2 initial interim analysis reported that cognitive function was higher than the age equivalent scores in the available natural history, noted August 30, 2023.",5
TAK,Relapsed/Refractory Multiple Myeloma,Phase 1/2,12/12/22,"Phase 1/2 final data reported that treatment significantly improved the degranulation of NK cells after 4-hour stimulation with K562 or PMA/Ionomycin, noted December 12, 2022.",6
LLY,"HR+, HER2-, Node-Positive, High Risk Early Breast Cancer",Approved,3/3/23,"Approved March 3, 2023.",6
TARA,Non-muscle invasive bladder cancer (NMIBC),Phase 1b,4/28/23,"Phase 1a data indicated that TARA-002 was generally well tolerated and showed anti-tumor activity in high-grade NMIBC patients, noted April 28, 2023.",6
RHHBY,Pemphigus vulgaris,Phase 3,6/13/19,"Phase 3 trial met primary and secondary endpoints - June 13, 2019.",6
IMGN,"Relapse/Refractory AML, ALL, BPDCN",Phase 2,6/9/23,"Phase 2 interim data reported that the objective response rate was 73% (22/30 patients), and an additional patient achieved a CR post-transplant, noted June 9, 2023.",6
TGTX,Chronic Lymphocytic Leukemia (CLL) and small lymphocytic lymphoma (SLL),Phase 3,9/20/21,"Phase 3 results showed that 421 patients were randomized to the U2 (n=210) or O+Chl (n=211) arms; 57% of patients were treatment-naïve and 43% had relapsed/refractory (R/R) CLL. At a median follow-up of 36.7 months, U2 significantly prolonged independent review committee (IRC) assessed progression-free survival (PFS) vs O+Chl (median 31.9 months vs 17.9 months; hazard ratio 0.546 (p<0.0001)). September 20, 2021",6
MACK,Cancer - HER2-positive locally advanced or metastatic breast cancer,Phase 2,12/21/16,"Phase 2 trial discontinued December 21, 2016 due to poor efficacy.",4
IMGN,Ovarian cancer and relapsed endometrial cancer,Phase 1/2,9/29/22,"Additional Phase 1b/2 data reported that ORR was 71% and carboplatin AUC5 had an ORR of 89%, noted September 29, 2022.",5
DRRX,Alcoholic hepatitis (AH),Phase 2b,9/7/23,"Phase 2b last patient dosed, noted September 7, 2023",5
CALA,Renal cell carcinoma,Phase 2,1/4/21,"Phase 2 top-line data did not meet primary endpoint - January 4, 2021.",4
CCCC,Synovial sarcoma,Phase 1,11/1/23,"Phase 1 deprioritized due to insufficient single agent efficacy, despite high levels of BRD9 degradation, noted November 1, 2023.",4
LLY,"Second-line hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer and HR+, HER2- breast cancer",Approved,10/4/17,"Approved October 4, 2017.",6
SGEN,Post-transplant Hodgkin lymphoma (HL) cancer,Approved,8/17/15,"Approved August 17, 2015 under priority review.",6
IMVT,Autoimmune diseases,Phase 1,9/26/23,"Phase 1 data reported no dose-related changes in serum albumin or LDL-C, and the mean total IgG reduction from baseline in this MAD cohort was 63%, noted September 26, 2023.",6
GSK,"Measles, mumps and rubella (MMR)",Approved,6/6/22,"Approved June 6, 2022.",6
APTX,Fibromyalgia,Phase 2b,8/12/22,"Phase 2b data reported that trial did not meet statistically significant, noted August 12, 2022.",6
MRK,Melanoma,Approved,2/19/19,"FDA Approval announced February 19, 2019.",6
PEPG,Duchenne muscular dystrophy (DMD),Phase 2,5/18/23,"Phase 2 received a No Objection Letter (NOL) for its Clinical Trial Application (CTA) from Health Canada to initiate the trial on 2H 2023., noted May 18, 2023.",5
CDTX,Vulvovaginal candidiasis (VVC),Phase 2,2/21/17,"Phase 2 data released February 21, 2017 - efficacy not evident, Development to be discontinued.",4
HGEN,COVID-19,Phase 3,7/6/22,"Phase 3 data showed a strong correlation between C-reactive protein (CRP) and outcomes with treatment with the greatest clinical benefit experienced by patients with baseline CRP<150 mg/L. In these patients, likelihood of survival without mechanical ventilation (SWOV) was achieved in 90% of patients treated plus standard of care compared to 79% treated with placebo plus standard of care, which was highly statistically significant. Patients had a 62% relative reduction in the risk of progression to invasive mechanical ventilation or death, noted July 6, 2022.",6
VACC,COVID-19 vaccine,Phase 3,3/22/21,"Data from U.S. trial noted 76% efficacy rate - March 24, 2021.",6
VAXX,Parkinson's disease,Phase 1,7/17/23,"Additional Phase 1 data reported that antibodies derived from UB-312, slowed the seeding of alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of patients with PD as demonstrated using multiple target engagement assays, noted July 17, 2023.",6
ABBV,Active Ankylosing Spondylitis,Approved,4/29/22,"Approved April 29, 2022.",6
GNLX,Malignant pleural effusion (MPE) due to non-small cell lung cancer (NSCLC) or breast cancer.,Phase 1,7/10/23,"Phase 1 expansion data showed a median overall survival (OS) was 19.5 months. The median OS among patients who had malignant pleural mesothelioma (MPM) was 22 months.. There were no dose-limiting toxicities or dose de-escalations, and the maximally tolerated dose was not reached. Therefore, a recommended dose was not established, noted July 10, 2023.",6
PTGX,Polycythemia Vera (PV),Phase 2,6/11/23,"Phase 2 data presented at EHA showed that PK-efficacy model adequately described the rusfertide plasma concentration-time profile and the time course of Hb, noted June 9, 2023.",6
SGEN,Metastatic cervical cancer,Phase 1/2,6/6/22,"Phase 1b/2 data reported an 41% objective response rate, noted June 6, 2022.",5
AZN,Lung cancer,Phase 3,11/16/18,"Overall survival data also did not meet primary endpoint - November 16, 2018.",4
GTHX,Triple-negative breast cancer (TNBC),Phase 2,11/12/21,"Phase 2 trial data demonstrated that patients who received trilaciclib prior to GCb showed increased T cell function as measured by greater production of inflammatory cytokines, noted November 12, 2021.",6
BHVN,Spinal Muscular Atrophy (SMA),Phase 3,9/14/23,"Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.",5
SMMT,Non-small cell lung cancer (NSCLC),Phase 2,6/5/23,"Phase 2 data reported a median progression-free survival (PFS) of 11.0 months and an overall response rate (ORR) of 67%, noted June 5, 2023.",5
NBTX,Non-small cell lung cancer (NSCLC),Phase 1/2,7/19/23,"Phase 1/2 dosing initiated, noted July 19, 2023.",5
AMGN,Cardiovascular disease / Elevated Lipoprotein,Phase 2,8/28/23,"Phase 2 data reported at ESC noted that results from the off-treatment extension period show that patients previously dosed with ≥75 mg of olpasiran sustained a ~40-50% placebo-adjusted percent reduction in Lp(a) nearly a year after the last dose, noted August 28, 2023.",6
ALNY,Hepatitis B,Phase 2,6/24/23,"Phase 2 part A preliminary 48-week post-treatment safety, tolerability and antiviral activity data demonstrated that combination resulted in a 2.7-3.1 log10 IU/mL decline in HBsAg levels at the end of treatment, noted June 24, 2023.",6
AZN,Asthma,Approved,12/17/21,"Approved December 17, 2021.",6
SLRX,Ewing's and other FET-rearranged sarcomas,Phase 1/2,12/1/22,"Phase 1/2 data appear to indicate that first- and second-relapse Ewing sarcoma patients treated with seclidemstat in combination with topotecan and cyclophosphamide who achieve disease control may have an increased time to tumor progression (TTP) compared with treatment of topotecan and cyclophosphamide alone. 60% Confirmed Disease Control Rate1 and 7.4 Months Median Time to Tumor Progression, noted December 1, 2022.",6
SEEL,Acute Suicidal Ideation and Behavior in patients with Major Depressive Disorder (ASIB in MDD),Phase 2,9/20/23,"Phase 2 Part 2 cohort data readout demonstrated early and persistent clinically meaningful reductions in symptoms of depression and acute suicidality, noted September 20, 2023.",6
SNDX,"HR+, HER2- breast cancer",Phase 3,5/21/20,"Phase 3 did not meet overall survival primary endpoint - May 21, 2020.",4
ACIU,Alzheimer’s Disease,Phase 1/2,11/12/21,"Phase 1b/2a further interim data showed that treatment led to strong induction of antibodies specific for pathological forms of Tau such as pTau and its aggregated form, enriched paired helical filaments (ePHF).",6
GMAB,Relapsing multiple sclerosis,Approved,8/20/20,"FDA approval announced August 20, 2020.",6
IMVT,Myasthenia Gravis (MG),Phase 2,8/25/20,"Phase 2 data released August 25, 2020.",5
SNY,Meningococcal meningitis,Approved,4/24/20,"FDA approval announced April 24, 2020.",6
CMRX,Smallpox,Approved,6/4/21,"FDA approval announced June 4, 2021.",6
RLAY,"PIK3CA (PI3Kα) mutant tumors, including HR+/HER2- breast cancer",Phase 1,4/18/23,"Phase 1 initial data reported that RLY-2608 reached selective target exposure at multiple doses, with a partial response in a breast cancer patient, noted April 18, 2023.",6
ZLAB,Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter),Approved,5/24/23,"Approved May 24, 2023.",6
MRK,Metastatic melanoma,Phase 3,4/7/23,"Phase 3 trial did not meet primary endpoint, noted April 7, 2023.",6
LGND,Relapsed Multiple Myeloma,Approved,6/11/18,"sNDA approval announced June 11, 2018.",6
ETNB,Nonalcoholic steatohepatitis (NASH),Phase 1/2,6/27/22,"Additional Phase 1b/2a data presented at EASL reported that in the cohort of NASH subjects PGZ led to a meaningful reductions in extra-hepatic metabolic parameters, noted June 27, 2022.",6
VNDA,Insomnia,Approved,1/31/14,"Approved January 31, 2014.",6
ACRX,Moderate-to-severe acute pain following a surgical procedure,Approved,11/2/18,"FDA Approval announced November 2, 2018.",6
ACIU,Alzheimer’s disease,Phase 3,1/30/19,Phase 3 trial terminated following interim analysis indicating primary endpoint would not be met.,4
NVS,Wet age-related macular degeneration (AMD),Approved,10/8/19,"FDA Approval announced October 8, 2019.",6
ABBV,Waldenström’s Macroglobulinemia,Approved,1/29/15,"Approved January 29, 2015 - PCYC",6
REGN,Nasal polyps,Approved,6/26/19,"FDA Approval announced June 26, 2019.",6
JNJ,Non-metastatic castration-resistant prostate cancer (CRPC),Approved,2/14/18,"Approval announced February 14, 2018.",6
PFE,Metastatic uveal melanoma (MUM),Phase 2,4/23/23,"Phase 2 darovasertib and crizotinib combination data reported a cORR of 45%, DCR of 90% and median PFS of ~7 months in 20 evaluable First-Line MUM patients, noted April 23, 2023.",6
VTRS,Children Living with HIV,Approved,9/5/23,"Tentative approval announced September 5, 2023.",6
INCY,COVID-19,Phase 3,4/8/21,"Phase 3 trial did not meet primary endpoint - April 8, 2021.",4
BCTX,"Early stage, newly diagnosed, high-risk triple negative breast cancer (TNBC)",Phase 2,9/8/23,"Phase 2 trial enrollment completed with initial results of Median overall survival of 13.5 months in vs. 6.7-9.8 months for similar patients reported in the literature, noted September 8, 2023.",5
VBIV,Hepatitis B vaccine,Phase 3,6/17/19,Phase 3 data met co-primary endpoints but missed key secondary endpoint.,5
RFL,Pancreatic Cancer,Phase 3,6/4/22,"Phase 3 data presented at ASCO reported that mOS of 11.1 months for CPI-613+mFFX vs. 11.7 months for FFX, noted June 4, 2022.",6
BIIB,Essential tremor,Phase 2,4/12/21,"Phase 2 trial met primary endpoint (P=0.049) - April 12, 2021. 62% of patients were down-titrated to from 60mg to 45 mg or 30 mg with the high dose not well tolerated.",6
LGND,Non-alcoholic steatohepatitis (NASH) and fibrosis,Phase 2b,5/16/23,"Phase 2b data reported that trial met primary endpoint, noted May 16, 2023.",5
MGNX,Anal cancer,Phase 2,9/18/20,Phase 2 data noted objective response rate of 14% and disease control rate of 49%.,5
NVS,Multiple Sclerosis (MS),Approved,5/11/18,"Approval announced May 11, 2018.",6
ALIM,Diabetic macular edema,Approved,9/26/14,"Approved September 26, 2014,",6
XBIT,Rheumatoid Arthritis,Phase 2,5/22/23,"Phase 2 initiation authorized by FDA on May 22, 2023.",5
COLL,Painful diabetic neuropathy (PDN),Phase 2b,12/13/16,"Phase 2b data released December 13, 2016 - primary endpoints not met.",4
AMGN,Rheumatoid Arthritis,Phase 2,11/14/22,"Phase 2 data reported that DAZ reduced DAS28-CRP and RF significantly vs. PBO at d113 in all dose regimens, noted November 14, 2022.",6
ABBV,Acute Myeloid Leukemia (AML),Approved,10/16/20,"FDA approval announced October 16, 2020.",6
VSTM,KRAS mutant non-small cell lung cancer (NSCLC),Phase 2,10/4/22,"Phase 2 part A data did not meet the pre-defined criteria to continue the trial, noted October 4, 2022.",6
WVE,Huntington’s disease,Phase 1/2,9/20/22,"Phase 1b/2a data reported a mean mHTT reduction across both cohorts of 22%, noted September 20, 2022.",6
VTVT,Mild Alzheimer’s disease with type 2 diabetes,Phase 2,12/15/20,"Phase 2 top-line data did not meet primary endpoint - December 15, 2020.",4
PRAX,Early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE),Phase 3,10/2/23,"Phase 3 results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients, noted October 2, 2023.",6
NGM,Geographic atrophy,Phase 2,10/17/22,"Phase 2 trial did not meet primary endpoint, noted October 17, 2022.",6
ABBV,Botox in the cheeks,Approved,5/15/23,"FDA Approved on May 15, 2023.",6
PFE,HIV,Approved,2/1/22,"Approved February 1, 2022.",6
KURA,Relapsed or refractory peripheral T-cell lymphoma,Phase 2,12/13/21,"Phase 2 final results reported an overall response rate of 56% and median overall survival of 32.8 months in patients with advanced AITL, noted December 13, 2021.",5
RVNC,Plantar fasciitis,Phase 2,11/9/20,"Phase 2 data did not meet primary endpoint - November 9, 2020.",6
ANAB,Atopic dermatitis,Phase 1,4/27/22,"Phase 1 top-line data reported that treatment was well tolerated and a reduction of cell surface BTLA expression on T cells and B cells following dosing, noted April 27, 2022.",6
IGMS,2L Colorectal cancer,Phase 1,6/5/23,"Phase 1 data reported that the median progression-free survival (PFS) in the 24 patients was 5.6 months as of the data cut-off date, noted June 5, 2023.",5
APLS,Paroxysmal Nocturnal Hemoglobinuria (PNH),Approved,10/2/23,"FDA Approved on October 2, 2023.",6
ABBV,Chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL),Approved,1/28/19,"FDA approval announced January 28, 2019.",6
APLS,Geographic atrophy (GA) associated with age-related macular degeneration (AMD),Phase 3,9/30/22,"Phase 3 24-month data reported evidence that slowing GA lesion growth has the potential to preserve visual function, noted September 30, 2022.",6
IONS,ATTR Amyloidosis,Phase 3,3/27/23,"Phase 3 66-week data reported that trial met all three primary endpoints, noted March 27, 2023.",5
RGNX,MPS II (Hunter Syndrome),BLA Filing,5/23/23,"FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation, noted May 23, 2023.",5
GSK,Mismatch repair deficient (dMMR) solid tumors,Approved,8/17/21,"FDA Approval announced August 27, 2021.",6
LSTA,Pre-dialysis chronic kidney disease,Phase 1b,2/6/23,"Phase 1b data reported that treatment was safe and well-tolerated by patients with no serious adverse events related to the therapy, noted February 6, 2023.",6
IGMS,Hepatocellular carcinoma (HCC),Phase 1/2,9/5/23,"Phase 1b/2 interim data reported complete response was recorded in 1 patient, partial response in 2 patients, and stable disease in 2 patients, noted September 5, 2023.",5
RHHBY,Chronic Lymphocytic Leukemia,Approved,5/15/19,"FDA approval announced May 15, 2019.",6
MRTX,Nonsquamous (NSQ) Non–Small-Cell Lung Cancer (NSCLC),Phase 2,9/20/21,"Phase 2 data demonstrated that the median PFS was 5.7 months and the median overall survival (OS) was 14.9 months, with 56% and 32% of these patients alive at one year and two years, respectively. ORR was 18%, with 3% CR and 15% PR. The median DOR was 12.8 months, presented at ESMO September 20, 2021.",6
ALDX,Allergic conjunctivitis,Phase 3,6/15/23,"Phase 3 top-line data met primary and all secondary endpoints, noted on June 15, 2023.",5
BMRN,Hemophilia A,Approved,6/29/23,"Approved June 29, 2023.",6
JNJ,Multiple sclerosis,Approved,4/16/15,Approved April 16 2015.,6
IRON,Polycythemia vera (PV),Phase 1,10/3/23,"Phase 1 initiated, noted October 3, 2023.",5
PFE,Vaso-occlusive crisis of sickle cell disease,Phase 3,8/2/19,"Phase 3 data failed to meet endpoints - August 2, 2019.",4
OPK,Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) and vitamin D insufficiency - Stage 3 and 4,Approved,6/21/16,"Announced FDA Approval June 21, 2016.",6
JNJ,Relapsing multiple sclerosis,Phase 3,7/26/19,Phase 3 trial met primary endpoint.,6
SLDB,Duchenne Muscular Dystrophy,Phase 1/2,3/14/22,"Phase 1/2 2-year data from first three patients in the high dose (2E14 vg/kg) cohort suggest sustained or improved motor function, pulmonary function and clinically validated patient-reported outcomes compared with expected declines reported by natural history data, noted March 14, 2022.",6
AERI,Glaucoma,Approved,12/18/17,"Approved December 18, 2017.",6
ONCY,Triple-negative breast cancer,Phase 2,12/10/21,"Phase 2 data reported that treatment is tolerated, noted December 10, 2021.",6
MEIP,Solid tumors,Phase 1,6/4/18,Phase 1 data presented at ASCO June 2018. Noted primary endpoint P=0.06.,5
ENTA,Primary biliary cholangitis (PBC),Phase 2,5/6/20,"Phase 2 top-line data did not meet primary endpoint - May 6, 2020.",4
AMGN,Heart failure with reduced ejection fraction (HFrEF),Phase 3,4/3/22,"Phase 3 results reported that there was no change in pVO2 in patients treated with omecamtiv mecarbil versus placebo, noted April 3, 2022.",6
AKTX,Bullous Pemphigoid,Phase 2,5/10/22,"Phase 2 data showed that 7/9 patients responded to treatment with three, regarded as complete responders, showing >80% reduction in BPDAI activity and four patients showing >70% reduction in pruritis by day 42; two of nine patients were non-responders. None of the nine patients reported grade three, four or five treatment-related adverse events, noted May 10, 2022.",6
GLPG,"B-cell malignancies, Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL)",Phase 1/2,6/9/23,"Phase 1/2 data presented at EHA showed that based on current results (n=7). For all 7 pts, GLPG5201 was successfully manufactured at the PoC and administered as a fresh product, with a median vein-to-vein time of 7 days. Rapid complete responses have been observed with a 100% ORR in a heavily pre-treated pt population. Ph 1 recruitment is ongoing to establish a recommended dose for Phase 2, noted June 9, 2023.",6
GNLX,Platinum-Resistant or Refractory Ovarian Cancer,Phase 2,5/25/23,"Phase 2 topline data reported that trial met primary endpoint, noted May 25, 2023.",5
BMY,Beta-thalassemia,Approved,11/8/19,"FDA Approval announced November 8, 2019.",6
VTGN,Social Anxiety Disorder (SAD),Phase 3,7/22/22,"Phase 3 trial did not meet primary endpoint, noted July 22, 2022.",6
GLYC,Healthy volunteers,Phase 1a,9/6/23,"Phase 1a first cohort dosing initiated, noted September 6, 2023.",5
NVS,ABSSSI,Approved,8/6/14,"Approved August 6, 2014 under priority review",6
LABP,Ulcerative Colitis (UC),Phase 1b,8/3/22,"Phase 1b top-line data reported that no serious adverse events (SAEs) were reported, noted August 3, 2022.",5
ZYNE,Osteoarthritis,Phase 2,8/14/17,"Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.",4
AZN,Gastric cancer,Phase 2,9/17/21,"Phase 2 trial data showed confirmed overall response rate (ORR) of 38% as assessed by independent central review (ICR). Three (3.8%) complete responses (CR) and 27 (34.2%) partial responses (PR) were observed in patients treated with ENHERTU, September 17, 2021.",6
CRSP,Sickle cell disease,Phase 1/2,6/13/22,"Data presented at EHA reported that all 31 patients with severe SCD were free of VOCs after exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months, noted June 13, 2022.",6
FREQ,Acquired Sensorineural Hearing Loss (SNHL),Phase 2b,2/13/23,"Phase 2b study failed to achieve its primary efficacy endpoint of an improvement in speech perception. The Company will now discontinue the FX-322 development program, noted February 13, 2023.",6
MACK,Cancer - second line pancreatic,Approved,10/22/15,"Approved October 22, 2015.",6
AMPE,Osteoarthritis of the Knee (OAK),Phase 3,3/2/22,"Phase 3 data reported that in the modified Intent-to-Treat (mITT) population treatment demonstrated a statistically significant reduction in pain and trended favorably toward improvement in function versus saline control, noted March 2, 2022.",6
RETA,CKD Patients at Risk of Rapid Progression,Phase 2,5/10/23,"Program discontinued, noted May 10, 2023.",4
STRO,Ovarian and Endometrial Cancer,Phase 1,10/23/23,"Additional Phase 1 data reported that a confirmed partial response (PR) was seen in 29% of patients, and the Disease Control Rate (DCR) was 86%, noted October 23, 2023.",5
SPPI,Neutropenia,Approved,9/9/22,"Approved September 9, 2022.",6
XENE,Acne,Phase 2,3/24/17,Phase 2 data released March 24 2017 - endpoints not met.,4
PFE,Solid Tumors,Phase 1,6/3/23,"Phase 1 data at ASCO reported that treatment-emergent adverse events (TEAEs) were observed in 88.5% of pts: 50.7% were Grade ≥ 3 (21.6% related), and 37.2% were serious (8.1% related); 6.1% of pts discontinued treatment due to TEAEs, noted June 3, 2023.",6
AVDL,Neonate Patients Requiring Total Parental Nutrition (TPN),Approved,12/16/19,"FDA Approval announced December 16, 2019.",6
AZN,HER low breast cancer,Approved,8/5/22,"Approved August 5, 2022.",6
CYCC,Solid tumors,Phase 1/2,6/30/22,"Phase 1/2 no dose limiting toxicities observed, noted June 30, 2022.",6
ALNA,Enteric Hyperoxaluria,Phase 3,11/7/19,"Phase 3 trial met primary endpoint, key secondary endpoint missed - November 7, 2019.",5
BHC,Ocular Inflammation,Approved,2/25/19,"FDA approval announced February 25, 2019.",6
RHHBY,Neuromyelitis optica spectrum disorder (NMOSD),Approved,8/14/20,"FDA approval announced August 14, 2020.",6
STTK,Ovarian Cancer,Phase 1a,6/5/23,"Phase 1a monotherapy data shared at ASCO reported that the best response was stable disease in 6/27 response evaluable (22%) pts, noted June 5, 2023.",6
MRK,HIV-1,Approved,5/30/17,"Approval announced May 30, 2017.",6
COLL,Severe pain,Approved,11/7/17,"Approval announced November 7, 2017.",6
GTHX,"ER+, HER2- breast cancer",Phase 1/2,6/4/21,"Phase 1/2 initial data presented at ASCO June 4, 2021. 65% of patients experienced stable disease. Median progression-free survival was 7.4 months.",5
PFE,COVID-19 Vaccine (Booster),Phase 1/2,5/23/22,"Phase 1/2/3 booster trial in children reported that a 80.3% vaccine efficacy, noted May 23, 2022.",5
IONS,NEURO-TTR - familial amyloid polyneuropathy (FAP).,Approved,10/5/18,"FDA Approval announced October 5, 2018,",6
AFMD,CD30-positive lymphomas,Phase 1/2,12/12/22,"Presentation reported a 97% ORR in HL patients, a 75% ORR in CD30+ NHL patients, and 9 patients in PR after C1 converted to a CR after C2, noted December 12, 2022.",6
VSTM,Low-grade serous ovarian cancer (LGSOC),Phase 2,5/25/23,"Additional Phase 2 data from abstract reported a confirmed objective response rates (ORR) by blinded independent central review of 45%, noted May 25, 2023.",5
TBPH,Acute Lung Injury (ALI) caused by COVID-19,Phase 2,6/21/21,"Phase 2 trial did not meet primary endpoint - June 21, 2021.",4
ANIP,Absence seizures that are refractory to other drugs,Approved,5/15/23,"ANDA approved May 15, 2023.",6
SNY,Eosinophilic esophagitis (EoE) in 1-11 year olds,Phase 3,7/14/22,"Phase 3 data reported that trial met its primary endpoint, noted July 14, 2022.",5
SCYX,Vulvovaginal candidiasis (VVC),Approved,6/2/21,"FDA approval announced June 2, 2021.",6
PTGX,Polycythemia vera,Phase 2,11/2/23,"Phase 2 data from abstract reported that treatment resulted in consistent maintenance of hematocrit below 45% and an overall decrease in erythrocyte counts, noted November 2, 2023.",6
KPRX,Persistent epithelial defects,Phase 1,4/26/23,"Phase 1 interim results reported that 62.5% of patients achieved the primary endpoint of healing over the 4-week period , noted April 26, 2023.",6
LIAN,Gastric Cancer / Gastroesophageal junction adenocarcinoma,Phase 2a,10/25/23,"Phase 2a data presented at ESMO showed that infigratinib was generally well tolerated with a manageable safety profile, noted October 25, 2023.",6
HRMY,Fragile X Syndrome in Children and Adolescents,Phase 3,7/18/22,"Longterm data reported improvements in ABC-CFXS Social Avoidance in the full population, noted July 18, 2022.",6
GERN,Myelofibrosis (MF),Phase 2/3,9/27/18,"Janssen noted September 27, 2018 not to continue collaboration.",4
ABUS,Hepatitis B (HBV),Phase 1b,6/27/22,"Phase 1a/b data reported that all seven patients reached HBsAg levels <100 IU/ml during AB-729 treatment or follow-up and two patients reached HBsAg levels <LLOQ at one or more visits, noted June 27, 2022.",6
PFE,Renal cell carcinoma,Approved,4/22/19,"FDA approval announced April 22, 2019.",6
APLS,Amyotrophic lateral sclerosis (ALS),Phase 2,5/25/23,"Phase 2 top-line results did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52. The study also did not meet key secondary efficacy endpoints. As a result the study will be discontinued, noted May 25, 2023.",6
JNJ,Influenza,Phase 2a,6/22/23,"FDA has granted Fast Track designation (FTD), noted June 22, 2023.",5
FHTX,Acute myeloid leukemia (AML),Phase 1,6/5/23,"Phase 1 is to be commenced in 3Q 2023, clinical hold lifted by FDA, noted June 5, 2023.",5
HARP,Small cell lung cancer,Phase 1/2,10/23/23,"Phase 1/2 data from ESMO reported a confirmed response rate 35% (11/31), across all tumor types and patient cohorts treated with 1 mg priming dose, and a 32% (6/19) confirmed response rate in patients with small cell lung cancer (SCLC), noted October 23, 2023.",6
ZVRA,Idiopathic hypersomnia (IH),Phase 2,10/2/23,"Phase 2 interim data reported that marked improvements in patient-reported assessments of key IH symptoms from the open-label titration phase, including excessive daytime sleepiness, sleep inertia, and brain fog, noted October 2, 2023.",6
IPHA,Solid tumors,Phase 1,12/9/22,"Phase 1 data reported that IPH5201 blocks ATP degradation and blocks CD39-mediated degradation of eATP released after chemotherapy treatment, noted December 8, 2022.",6
AZN,COVID-19 vaccine (variant),Phase 2/3,2/10/22,"Phase 2/3 trial met primary endpoint, noted February 10, 2022.",5
TAK,Multiple myeloma,Phase 3,11/8/19,"Phase 3 trial met primary endpoint - November 8, 2019.",6
BMY,(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC),Approved,7/11/18,"Approval announced July 11, 2018.",6
JNJ,Plaque psoriasis,Approved,10/13/17,"Approval announced October 13, 2017.",6
ADCT,Diffuse Large B-Cell Lymphoma (DLBCL),Phase 3,8/31/23,"Phase 3 updated data from safety lead-in portion of study reported that the overall response rate by central review was 16/20 (80%). A total of 10/20 (50%) and 6/20 (30%) patients attained complete and partial response, respectively, noted August 31, 2023.",6
UBX,Diabetic macular edema,Phase 1,5/4/22,"Phase 1 24-week data presented at ARVO reported that treatment was well-tolerated with visual acuity was improved in 7/10 patients at 4 weeks and in 5 at Week 12, noted May 4, 2022",6
LLY,Psoriatic Arthritis,Approved,12/1/17,"Approval announced December 1, 2017.",6
NVS,Sickle cell disease,Approved,11/15/19,"FDA Approval announced November 15, 2019.",6
HALO,Non-small cell lung cancer (NSCLC),Phase 3,8/1/22,"Phase 3 trial met co-primary endpoints, noted August 2, 2022. Regulatory filing planned.",5
INSM,Nontuberculous mycobacterial (NTM) lung disease,Approved,9/28/18,"FDA Approval announced September 28, 2018.",6
LGND,Maintenance treatment of COPD,Phase 2,8/5/19,"Phase 2 data met primary endpoint - August 5, 2019.",6
AKBA,Iron deficiency anemia in non-dialysis dependent CKD patients,Approved,11/7/17,"sNDA FDA Approval announced November 7, 2017.",6
NVO,Type 2 diabetes,Phase 2,8/22/22,"Phase 2 trial data noted that treatment achieved a numerically higher HbA1c reduction of 2.18%-points compared to a reduction of 1.79%-points for people treated with semaglutide and 0.93%-points with cagrilintide alone, noted August 22, 2022.",6
RHHBY,Non-Small Cell Lung Cancer,Approved,10/15/21,"Approved October 15, 2021.",6
NVAX,COVID-19 vaccine,Approved,7/13/22,"Emergency Use Authorization (EUA) granted July 13, 2022.",5
BMY,Refractory Acute myeloid leukemia (AML) - cancer,Phase 3,6/7/22,"Phase 3 overall survival data reported that in the R172 subgroup was 14.6 months with ENA vs 7.8 months with CCR and 1-yr survival rates were 62% and 30%, respectively, noted June 7, 2022.",5
VIR,Hepatitis B,Phase 2,9/6/23,"Additional Phase 2 data significantly increased HBsAg seroconversion rate is strongly associated with BRII-179 treatment and correlates with the increased rate of HBsAg loss3, noted September 6, 2023.",6
IRON,Erythropoietic Protoporphyria (EPP),Phase 2,11/2/23,"Phase 2 data from abstract reported that treatment with bitopertin resulted in mean (SD) decreases in PPIX of -39% by Day 43, noted November 2, 2023.",6
ACRV,"Platinum-resistant ovarian,(PROC) endometrial, and urothelial cancers.",Phase 2,10/13/23,"Phase 2 preliminary data shared at AACR-NCI-EORTC reported that ACR-368 that enables identification of responders to ACR-368 treatment, noted October 13, 2023",6
XNCR,Systemic Lupus Erythematosus (SLE),Phase 2,10/8/18,"Phase 2 top-line data released October 5, 2018. Primary endpoint not met.",4
BMY,First-line renal cell carcinoma,Phase 3,9/16/21,"Additional Phase 3 data presented at ESMO September 16, 2021. At a median follow-up of 23.5 months, progression free survival (PFS) and objective response rate (ORR) benefits were observed regardless of prior nephrectomy status. The magnitudes of PFS and ORR benefits were greater in the subgroup with prior nephrectomy.",6
RHHBY,Anaplastic lymphoma kinase (ALK)-positive metastatic (advanced) non-small cell lung cancer (NSCLC),Phase 3,9/29/19,Phase 3 updated data at ESMO noted HR 0.43. PFS 34.8 months vs 10.9 months for crizotinib.,5
ICPT,Primary Biliary Cholangitis (PBC),Phase 2,6/23/23,"Phase 2 interim data presented at EASL showed combination of OCA 5-10 mg + bezafibrate 400 mg normalized a range of biomarkers of PBC (ALP, total bilirubin, GGT, ALT and AST) in 58% of patients at 12 weeks, noted June 23, 2023.",6
VBIV,Hepatitis B,Phase 2,9/6/23,"Additional Phase 2 data significantly increased HBsAg seroconversion rate is strongly associated with BRII-179 treatment and correlates with the increased rate of HBsAg loss3, noted September 6, 2023.",6
BMY,COVID-19,Phase 3,6/2/22,"Phase 3 data reported that the risk of death was lower for participants who received Orencia at 11%, versus 15% for those who received placebo, and the odds of dying were 37.4% lower, noted June 2, 2022.",5
TNXP,Fibromyalgia,Phase 3,3/21/22,"Second Phase 3 trial did not meet primary endpoint, noted March 21, 2022.",6
MDNA,Recurrent Glioblastoma (GBM),Phase 2b,7/20/23,"Phase 2b trial published in the journal Neuro-Oncology, and when compared to a well-matched external control, bizaxofusp more than doubled the median survival in end-stage rGBM patients, noted July 20, 2023. Phase 3 trial planned.",5
RDHL,COVID-19,Phase 2,12/31/20,"Phase 2 initial data released December 31, 2020.",5
SPPI,Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma,Approved,3/15/16,Approved March 15 2016.,6
ASLN,Atopic dermatitis (AD),Phase 2b,10/13/23,"Phase 2b topline new data from a posthoc analysis of patients with severe disease (baseline EASI score at least 21), representing 63% of ITT patients, show monthly dosing with 600 mg eblasakimab for 16 weeks led to a 74.5% reduction in EASI score and EASI-75 of 53.6%, noted October 13, 2023.",6
AFMD,CD30-positive peripheral T cell lymphoma (PTCL),Phase 2,4/16/23,"Phase 2 presentation at AACR reported an objective response rate (ORR) of 32.4% and 10.2% complete response rate (CR) in the ITT population, noted April 16, 2023.",6
BGNE,Marginal Zone Lymphoma,Approved,9/15/21,"Approved September 15, 2021.",6
EYEN,Mydriasis - pupil dilation,Approved,5/8/23,"Approved May 8, 2023.",6
PRTA,Parkinson's disease,Phase 2,3/16/22,"Phase 2 results presented showed that participants with PD who were treated for two years (early-start group) showed slower decline of Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part 3 scores relative to participants treated with for one year (delayed-start group), noted March 16, 2022.",6
BMY,Venous thromboembolism (VTE) prevention post total knee replacement (TKR) surgery,Phase 2,11/15/21,"Phase 2 trial met both primary endpoints; the dose response for efficacy was significant, and the 12% rate of VTE was significantly lower than the prespecified benchmark rate of 30%, noted November 15, 2021.",6
EVAX,Adjuvant melanoma,Phase 1/2,4/18/23,"Phase 1/2a data presented at AACR met both primary endpoints on safety, tolerability and immunogenicity and secondary endpoint on clinical efficacy., noted April 18, 2023.",6
XERS,Endogenous Cushing’s syndrome,Phase 3,5/12/22,"Phase 3 presentation of data at AACE reported that mean total and LDL cholesterol levels were significantly increased with placebo versus treatment, noted May 12, 2022.",6
MACK,HER2 negative metastatic breast cancer,Phase 2,11/7/18,"Phase 2 trial termination announced November 7, 2018.",4
SAGE,Postpartum depression (PPD),Phase 3,1/7/19,"Phase 3 data met primary endpoint - January 7, 2019.",6
TCRX,Hematologic malignancies,Phase 1,11/2/23,"Phase 1 data from abstract reported that the safety analysis found expected post-HCT adverse events similar in treatment and control arms. Incidence of graft-versus-host disease (GvHD) was similar in control (3 events) and treatment arms, noted November 2, 2023.",6
PTGX,Ulcerative colitis,Phase 2b,8/6/18,"Phase 2b trial discontinued following interim analysis - March 26, 2018. However, company noted August 6, 2018 that human error by CRO contributed to this decision. Will meet with FDA 2H to discuss future development.",4
ALKS,Schizophrenia,Approved,7/2/18,"FDA approval announced July 2, 2018.",6
ALPMY,Severe vasomotor symptoms (VMS) due to menopause,Phase 3,6/28/23,"Phase 3b data reported that the trial met its primary endpoint, noted June 28, 2023.",5
ARTL,Cancer-Related Anorexia and Weight Loss,Phase 1b,7/19/22,"Phase 1b safety review determined that treatment is well-tolerated, noted July 19, 2022.",6
BMY,Lupus Erythematosus (SLE),Phase 2,11/8/21,"Phase 2 52-week data reported that the primary endpoint, SRI-4 response at wk 24, was achieved by 54.3% of patients on iberdomide 0.45 mg vs. 34.9% on placebo, noted November 8, 2021.",6
ARVN,Castration-resistant prostate cancer (CRPC),Phase 1/2,10/22/23,"Updated data from the ongoing Phase 1/2 trial presented at ESMO reported a median rPFS of 11.1 months in patients harboring AR 878/875 mutations and without co-occurring AR L702H mutations, and PSA50 rates of 54% in patients with tumors harboring AR 878/875 mutations and without co-occurring AR L702H, noted October 22, 2023.",6
ESPR,LDL-C and type 2 diabetes,Phase 2,8/28/19,Phase 2 data met primary endpoint.,6
BHC,Dry Eye Disease Associated With Meibomian Gland Dysfunction,Approved,6/29/23,"Approved on May 18, 2023.",6
CYTK,Hypertrophic cardiomyopathy (HCM),Phase 2,5/22/23,"Cohort 4 data reported that overall, 58% of patients experienced a clinical reduction in symptom burden: 12.5% had a small improvement (≥ 5-10 points), 20% had a moderate to large improvement (≥ 10-20 points), and 25% had a large to very large improvement, noted May 22, 2023.",6
GLPG,Inflammatory Bowel Disease,Phase 2,3/4/21,"Phase 2 interim data released March 4, 2021.",5
MNKKQ,Hemostasis,Approved,4/30/15,"Approved April 30, 2015.",6
PFE,Head and Neck Cancer,Phase 3,3/13/20,Phase 3 trial terminated - unlikely to show a statistically significant improvement in the primary endpoint of progression-free survival (PFS).,4
IMMX,Multiple myeloma,Phase 1/2,10/3/23,"Phase 1b/2a updated data reported a 100% (9/9) overall response rate and 67% (6/9) complete response rate (MRD 10-5) observed in heavily pre-treated patients, noted October 3, 2023.",6
AMGN,Gastric and gastro-esophageal junction cancer,Phase 3,7/14/23,"Trial in China initiated, noted July 14, 2023.",5
BMY,Relapsed/refractory multiple myeloma,Approved,3/26/21,"FDA approval announced March 26, 2021.",6
GSK,Chronic obstructive pulmonary disease (COPD),Approved,9/18/17,"Approval announced September 18, 2017.",6
AZN,Non-small Cell Lung Cancer (NSCLC),Approved,8/12/22,"Approved August 12, 2022.",6
MRK,Relapsed or Refractory Primary Mediastinal Large B-Cell Lymphoma (PMBCL),Approved,6/13/18,"Approval announced June 13, 2018.",6
MRK,Chronic cough,CRL,1/24/22,"CRL announced January 24, 2022.",5
QLGN,"Pancreatic cancer, Prostate cancer and other solid tumors",Phase 1a,8/1/23,"IND cleared by FDA, noted August 1, 2023",5
NRIX,B-cell malignancies / graft-versus-host disease,Phase 1a,11/2/23,"Phase 1a data from ASH abstract reported that of three evaluable patients with CLL receiving the lowest dose of 50 mg, early signs of clinical activity were observed including one confirmed partial response and 2 patients with stable disease, noted November 2, 2023.",6
OCUP,Diabetic Retinopathy (DR) / Macular Edema (DME),Phase 2,8/28/23,"Phase 2 data reported that APX3330 achieved statistical significance in preventing clinically meaningful progression of diabetic retinopathy, noted August 28, 2023.",6
AIMD,Oral warts in HIV-seropositive patients,Phase 3,9/18/23,"Ainos plans to pursue a pre-IND meeting with the FDA in advance of planned Phase 3 clinical studies, noted September 18, 2023.",6
HEPA,Non-alcoholic steatohepatitis (NASH),Phase 2,5/22/23,"Phase 2 topline data met primary and secondary endpoints, including improvement in liver function and multiple NASH biomarkers, noted May 22, 2023.",6
HALO,Overactive bladder,Approved,12/8/11,"Approved December 8, 2011.",6
ABBV,Myelofibrosis,Phase 2,12/12/22,"Phase 2 cohort 3 data reported that 35% (9/26) of patients achieved ≥ 1 grade improvement at any time during treatment with a median time-to-improvement of 12.3 weeks, noted December 12, 2022.",6
PFE,Respiratory syncytial virus (RSV) infections in infants,Phase 2,4/7/22,"Phase 2 review by the Data Safety Monitoring Committee reported that treatment showed a favorable safety and PK exposure profile to advance to Part B, noted April 7, 2022.",6
AVXL,Rett syndrome,Phase 3,2/1/22,"Phase 3 data reported that trial met primary and all secondary efficacy and safety endpoints, noted February 1, 2022.",5
ENTX,Hypoparathyroidism,Phase 2b,9/22/19,"Phase 2b results presented September 22, 2019.",5
BMY,Philadelphia Chromosome-Positive Chronic Phase Chronic Myeloid Leukemia,Approved,11/10/17,"Approval announced November 10, 2017.",6
ZLAB,Ovarian cancer,Phase 3,6/1/23,"Phase 3 data reported a complete and partial responses, both confirmed, were observed in 12 (25.5%) and 15 (31.9%) niraparib-treated patients and 3 (11.5%) and 5 (19.2%) placebo-treated patients, respectively, leading to a confirmed ORR of 57.4% with niraparib and 30.8% with placebo, noted June 1, 2023.Phase 3 data reported a complete and partial responses, both confirmed, were observed in 12 (25.5%) and 15 (31.9%) niraparib-treated patients and 3 (11.5%) and 5 (19.2%) placebo-treated patients, respectively, leading to a confirmed ORR of 57.4% with niraparib and 30.8% with placebo, noted June 1, 2023.",6
SNY,Pemphigus,Phase 3,10/29/21,"Phase 3 trial did not meet primary or key secondary endpoints, noted October 29, 2021.",4
JNJ,Diffuse large B-cell lymphoma (DLBCL),Phase 3,7/11/18,"Phase 3 trial did not meet primary endpoint - noted July 11, 2018.",4
AVRO,Gaucher disease,Phase 1/2,10/19/21,"Phase 1/2 safety data reported no SAEs and no AEs for more than 14 months post-treatment, noted October 19, 2021.",6
BCEL,Solid tumors,Phase 1b,6/5/23,"Phase 1b interim update presented at ASCO showed clinical activity observed in multiple tumor types; longer progression-free survival observed in patients with high target expression; ATRC-101 continues to be well-tolerated, noted June 5, 2023.",6
SNY,Atopic dermatitis 6-11 year-olds,Approved,5/26/20,"FDA Approval announced May 26, 2020.",6
BMY,Melanoma,Phase 2/3,10/22/23,"Phase 2/3 data presented at ESMO reported that response to NIVO+RELA was associated with increased LAG-3+ CD4+ T cells at week 4, but on-treatment modulation of peripheral CD8+ T cells was not associated with NIVO+RELA efficacy, noted October 22, 2023.",6
TMBR,Congenital Ichthyosis,Phase 3,6/20/23,"Phase 3 interim pharmacokinetic data indicated minimal systemic absorption of isotretinoin or its major metabolites in patients with moderate to severe forms of congenital ichthyosis (CI) who were treated with TMB-001 0.05% ointment, noted June 20, 2023.",6
JAZZ,VOD in high-risk patients following hematopoietic stem cell transplantation,Phase 3,12/12/22,"Phase 3 data reported that mortality from SOS was 33.3%, noted December 12, 2022.",5
GLSI,Breast cancer,Phase 2b,5/26/22,"Phase 2b abstract reported a DFS rate of 100% versus 89.4%, and just under 23% of subjects experienced an immune response to GP2 by DTH at baseline, noted May 26, 2022.",5
LLY,Obesity,Phase 2,6/24/23,"Phase 2 met primary and secondary endpoints, noted June 24, 2023.",5
PLXP,Liquid-filled aspirin capsule,Approved,3/1/21,"FDA approval announced March 1, 2021.",6
BGNE,Chronic lymphocytic leukemia/small lymphocytic leukemia,Phase 1,6/9/23,"Phase 1 data presented at EHA reported that of 39 pts available for tumor assessment, 5 with NHL and 9 with CLL/SLL achieved responses (NHL: 3 partial responses [PR] at 160 mg/d, 1 PR at320 mg/d, 1 complete response [CR] at 640 mg/d; CLL/SLL: 2 CR and 2 PR at 80 mg/d, 3 PR at 160 mg/d, 2 PR at320 mg/d), noted June 9, 2023.",6
FBIO,Cutaneous squamous cell carcinoma (CSCC),BLA Filing,1/4/23,"Phase 1 pivotal top-line data reported that the trial met the primary endpoint, noted January 25, 2022. Phase 1 interim efficacy results reported an objective response rate of 54.8%, noted June 16, 2022. BLA submitted to FDA, noted January 4, 2022.",5
CCCC,Hematologic malignancies,Phase 1/2,4/8/22,"Phase 1/2 data reported 2 dose-limiting toxicities, with 3 stable disease responses, and two progressive disease responses, noted April 8, 2022.",5
NVS,Asthma,Phase 3,10/22/19,"Phase 3 data did not meet primary endpoint - noted October 22, 2019.",4
ASRT,Peripheral T-Cell Lymphoma cancer,Approved,7/7/14,"Approved July 7, 2014.",6
CANF,Non-alcoholic steatohepatitis (NASH),Phase 2,4/7/20,"Phase 2 endpoints met - April 7, 2020.",6
MCRB,Recurrent C. Difficile infection,Approved,4/26/23,"Approved April 26, 2023.",6
OPNT,Bulimia Nervosa,Phase 2,2/21/19,"Phase 2 data released February 21, 2019. Endpoints not met.",4
JNJ,Pulmonary arterial hypertension (PAH),Phase 3,4/18/23,"Phase 3 data reported that trial met its co-primary endpoint, with PVR change with M/T STCT was significantly greater versus macitentan, noted April 18, 2023.",5
BBIO,Limb Girdle Muscular Dystrophy Type 2i (LGMD2i),Phase 2,10/9/23,"Phase 2 long-term data at Month 21 demonstrate a well-tolerated safety profile, and stabilization in 10 meter walk test (10MWT), 100-m timed test (100mTT), and the North Star Assessment (NSAD) scores, noted October 9, 2023.",6
INO,Ebola,Phase 1b,4/16/23,"Phase 1b data presented at ECCMID reported that GMTs rose significantly after boosting for each time point measured, peaking at week 2, noted April 16, 2023.",6
JAZZ,Acute lymphoblastic leukemia (ALL),Approved,6/30/21,"FDA approval announced June 30, 2021.",6
ATNXQ,Actinic keratosis,Approved,12/15/20,"FDA approval announced December 15, 2020.",6
OTLK,Wet age-related macular degeneration (wet AMD),Phase 3,8/26/20,"Phase 3 data released August 26, 2020. No statistical differences between Lucentis (ranibizumab) and ONS-5010.",5
RHHBY,Ovarian cancer,Approved,6/13/18,"FDA approval announced June 13, 2018.",6
CDTX,Candidemia,Phase 2,7/29/19,"Phase 2 data released July 29, 2019 - noted all objectives met.",6
NKTX,B-cell malignancies,Phase 1,6/10/23,"Phase 1 data presented at EHA demonstrated that 7/10 patients treated with NKX019 monotherapy at 1 billion and 1.5 billion CAR NK cells per dose achieved complete response (70% CR rate). Early safety data support potential of allogeneic CD19 CAR NK to be administered on outpatient basis, noted June 10, 2023.",6
VXRT,Condyloma,Phase 2,6/4/18,Phase 2 trial did not meet primary endpoint.,4
PFE,Obsessive compulsive disorder (OCD),Phase 2/3,6/24/20,Phase 2/3 top-line data did not meet primary endpoint.,4
GTHX,Triple-negative breast cancer,Phase 2,5/10/23,"Phase 2 additional data highlighted the potential for trilaciclib to meaningfully reduce adverse events related to use of sacituzumab. As expected, patients with PD-L1(+) tumors appear to respond earlier than patients with PD-L1(-) tumors, noted May 10, 2023",6
ALVR,AdV viremia (following allo-HSCT),Phase 2,4/26/23,"Phase 2 study follow-up long-term data demonstrate dthat the high-risk allo-HCT patients who received posoleucel experienced continued low rates of clinically significant infections and end-organ disease and 0% non-relapse mortality, noted April 26, 2023.",6
MNKKQ,Deep partial thickness severe burns - skin defects,Approved,6/15/21,"FDA approval June 15, 2021.",6
BMY,AML Maintenance,Approved,9/1/20,"FDA approval announced September 1, 2020.",6
GRCL,B-cell acute lymphoblastic leukemia (B-ALL),Phase 1,6/10/23,"Phase 1 study of the donor-derived allogeneic presented at EHA showed 100% ORR and a favorable safety profile for treatment of r/r B-ALL, noted June 10, 2023.",6
MRK,Squamous non-small cell lung cancer (sNSCLC),Approved,10/30/18,"FDA approval announced October 30, 2018.",6
ARWR,Cardiovascular disease / Elevated Lipoprotein,Phase 2,8/28/23,"Phase 2 data reported at ESC noted that results from the off-treatment extension period show that patients previously dosed with ≥75 mg of olpasiran sustained a ~40-50% placebo-adjusted percent reduction in Lp(a) nearly a year after the last dose, noted August 28, 2023.",6
ITRM,Uncomplicated urinary tract infections (uUTI),CRL,7/26/21,"CRL announced July 26, 2021.",5
TRVN,Diabetic neuropathic pain (DNP),Phase 1,9/6/23,"Phase 1 data demonstrated a statistically significant analgesic effect in the capsaicin-induced model, noted on September 6, 2023.",6
ZLAB,Solid tumors,Phase 1,10/7/21,"Phase 1 updated data noted a confirmed responses for a cORR of 36% out of the 11 MET TKI-naïve NSCLC patients, cORR of 33% for the nine MET TKI-naïve GC/GEJ patients, and among the 12 patients with advanced other solid tumors, one patient with MET amplified colorectal cancer achieved a confirmed response, noted October 7, 2021.",6
IONS,Huntington's Disease,Phase 3,3/22/21,Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.,4
AGIO,Pyruvate Kinase Deficiency,Phase 3,6/10/22,"Phase 3 new patient outcome reported that 28/78 (35.9%) of all patients achieved a normal hemoglobin level at least once during treatment, noted June 10, 2022.",6
FDMT,X-linked retinitis pigmentosa (XLRP),Phase 1/2,10/10/21,"Phase 1/2 initial results showed that dosing was well tolerated in all patients treated to-date, with no dose-limiting toxicities, no serious adverse events and no chronic inflammation, noted October 10, 2021",6
GILD,"COVID-19 in people with mild, moderate and severe hepatic impairment",Approved,8/24/23,"Approved August 24, 2023.",6
SNY,Cystic fibrosis,Phase 1/2,3/17/21,"Phase 1/2 interim analysis March 17, 2021. Safe and well tolerated with no serious adverse events but no increase in lung function (ppFEV1) evident.",5
AMGN,Multiple Myeloma,Approved,12/1/21,"Approved December 1, 2021.",6
ZLDPF,Severe hypoglycemia in diabetes,Approved,3/22/21,"FDA approval announced March 22, 2021.",6
AMGN,Non-small cell lung cancer (NSCLC),BLA Filing,10/5/23,"FDA Oncologic Drugs Advisory Committee (ODAC) voted 2-10 against the question of if the primary endpoint, progression-free survival (PFS) per blinded independent central review (BICR), be reliably interpreted in CodeBreaK200, noted October 5, 2023.",5
RHHBY,Multiple sclerosis (MS) or primary progressive MS,Phase 3,10/11/23,"Phase 3 data reported that treatment was comparable to IV infusion in providing rapid and sustained depletion of B cells and near-complete suppression of MRI lesion activity in the brain over 24 weeks, noted October 11, 2023.",6
MRK,Metastatic malignant pleural mesothelioma,Phase 2/3,6/2/23,"Phase 2/3 data reported that mOS was 17.3m vs 16.1m for CP-pembro vs CP and mPFS was 7.13m vs 7.16m, noted June 2, 2023.",5
AYTU,Attention deficit hyperactivity disorder (ADHD),Approved,1/27/16,"Approved January 27, 2016.",6
INO,Human papilloma virus (HPV)-related anal dysplasia,Phase 2,1/6/21,Phase 2 data noted 63% showed reduction of lesions of greater than 25%.,6
ELOX,Cystinosis,Phase 2,1/14/20,"Phase 2 data released January 14, 2020.",5
ATXI,Postoperative pain following bunionectomy surgery,CRL,2/15/22,"FDA AdCom Meeting resulted in a 8-14 vote against the question that the application showed that benefits outweighs the risks, noted February 15, 2022.",6
GKOS,Glaucoma,Phase 3,9/7/22,"Phase 3 trials met primary endpoint, noted September 7, 2022.",5
ZYNE,22q11.2 Deletion Syndrome,Phase 2,4/13/23,"Additional Phase 2 data reported a statistically significant improvements in PARS-R, total score and all 5 subscales of ADAMS and all 5 subscales of ABC-C, noted April 13, 2023.",6
SNY,Hematological malignancies,Phase 1/2,10/16/23,"Phase 1/2 data from ESMO abstract reported that 3/8 patients achieved complete remission (CR), noted October 16, 2023.",6
LLY,Alopecia Areata,Approved,6/14/22,"Approved June 14, 2022.",6
MRK,Relapsed or Refractory Classical Hodgkin Lymphoma,Approved,3/14/17,"Approval announced March 14, 2017.",6
SVRA,MRSA in cystic fibrosis,Phase 3,12/10/20,"Phase 3 trial missed primary endpoint - December 10, 2020. Development to be stopped.",4
CRVS,Renal cell cancer,Phase 1/2,5/29/20,"Phase 1b/2 initial data from the RCC cohort at ASCO May 29, 2020. AdenoSig positive group -ORR of 17%. AdenoSig negative group - no partial responses.",4
NBIX,Uterine Fibroids,Approved,5/29/20,"FDA approval announced May 29, 2020.",6
INSM,Bronchiectasis,Phase 2,2/3/20,"Phase 2 trial met primary endpoint - February 3, 2020.",6
LIAN,Ulcerative Colitis,Phase 2,10/4/21,"Phase 2 data displayed clinical remission in 36.1% of the 880 mg group and 35.5% of the 440 mg group during the blinded maintenance phase. Both dose cohorts had 10% of patients report an AE of ulcerative colitis worsening, noted October 4, 2021.",6
IMUX,COVID-19,Phase 2,2/17/21,"Phase 2 top-line data released February 17, 2021 - low rates of COVID-19 prevented the primary endpoint from being evaluable.",5
TEVA,Herceptin biosimilar,Approved,12/14/18,"FDA approval announced December 14, 2018.",6
REGN,Transthyretin amyloidosis (ATTR),Phase 1,11/2/23,"Phase 1 updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001, including in 29 patients who have now reached 12 months or more of follow-up, noted November 2, 2023.",6
LXRX,Diabetic Peripheral Neuropathy,Phase 2,11/14/22,"Phase 2 final results reported no evidence of withdrawal symptoms or rebound pain after treatment ended, noted November 14, 2022.",6
MNMD,Anxiety Disorders,Phase 2a,5/11/22,"Phase 2a data results demonstrated the significant, long-lasting beneficial effects of LSD and potential to safely mitigate symptoms of anxiety and depression, noted May 11, 2022.",6
GTHX,Early-stage triple negative breast cancer (TNBC),Phase 2,12/8/22,"Phase 2 data reported a trend toward an increased ratio of CD8+ T-cells to Tregs within the tumor microenvironment, indicating trilaciclib may favorably modulate the composition of immune cells to support antitumor immune responses, noted December 8, 2022.",6
ASND,Mantle cell lymphoma (MCL),Phase 1,6/17/23,"Phase 1 data presented at ICML showed that 86.5% of patients in the relapsed or refractory mantle cell lymphoma cohort achieved a response, with 74.3% achieving a complete response, noted June 17, 2023.",6
AMRX,Avastin biosimilar,Approved,4/13/22,"Approved April 13, 2022.",6
GLPG,Rheumatoid Arthritis,Phase 2a,7/14/21,"Phase 2a data released July 14, 2021 - showed no differentiation from placebo.",4
GMAB,Multiple myeloma,Approved,5/1/20,"FDA Approval announced May 1, 2020.",6
RHHBY,Inherited Alzheimer's disease,Phase 2/3,2/10/20,Phase 3 trial did not meet primary endpoint.,4
ABOS,Alzheimer’s disease (AD),Phase 1,10/27/23,"Additional Phase 1 results shared at CTAD reported that statistically significant amyloid plaque reduction that was comparable to approved and in-review therapies at similar time points, was observed in higher dose cohorts, noted October 27, 2023",6
ALNY,Gout,Phase 1,12/15/22,"Phase 1 pharmacodynamic data suggest extrahepatic contributes significantly to serum urate in humans, noted December 15, 2022.",5
ADAP,Solid tumors,Phase 1,11/21/21,"Phase 1 preliminary data reported that all Cohort 1 patients had measurable persistence of transduced cells throughout the interventional phase of the trial with some maintaining high levels of persistence beyond 6 months post infusion. Median time to peak persistence was 1.1 weeks and median peak persistence was 218,827.9 copies/ug DNA, noted November 12, 2021.",6
MRK,Advanced renal cell carcinoma (RCC),Phase 3,8/17/23,"Phase 3 trial met primary endpoint, noted August 18, 2023.",5
PALI,Reduction in adhesions following abdominal and pelvic surgery,Phase 2,8/9/23,"Phase 2 study did not achieve primary efficacy endpoint of reducing adhesions in LB1148 treated patients compared to placebo treated patients post bowel resection surgery, noted August 9, 2023.",6
ATHE,Multiple System Atrophy (MSA),Phase 2,7/26/23,"Phase 2 trial recommended by DMC on July 26, 2023.",5
NVS,Alzheimer’s Disease,Phase 2/3,7/11/19,Phase 2/3 trial discontinued due to lack of efficacy.,4
JNJ,Relapsed or refractory multiple myeloma,Approved,6/16/17,"Approval announced June 16, 2017 - note: submitted by Janssen Biotech, Inc.",6
EGRX,Chronic lymphocytic leukemia; Indolent non-Hodgkin's lymphoma - cancer,Approved,7/2/14,"Tentative approval July 2 2014. Teva has also received orphan drug and related pediatric exclusivity expiring in September 2015 and May 2016 for the CLL and NHL indications, respectively.",6
RVMD,Advanced Solid Tumors,Phase 1b,10/22/23,"Phase 1/1b data shared at ESMO reported that the objective response rate was 38 percent, with one patient achieving a complete response (CR) as a best response and 14 patients achieving a partial response, noted October 22, 2023.",6
LGND,Refractory status epilepticus (RSE),Phase 2,9/26/19,"Phase 2 data met primary endpoint - September 26, 2019.",6
ICPT,Primary biliary cirrhosis (PBC),Approved,5/27/16,Approved May 27 2016.,6
FDMT,Cystic fibrosis,Phase 2,11/1/23,"Phase 2 Dose Expansion interim data presented at the North American Cystic Fibrosis Conference showed that it was generally well tolerated with no inflammation in any lung biopsy across Cohorts 1 and 2 (1E15 & 2E15 vg; n=7) with up to 17 months follow-up November 1, 2023.",6
ABBV,Neurogenic Detrusor Overactivity,Approved,2/10/21,"FDA approval announced February 10, 2021.",6
AZN,Castration-resistant prostate cancer -first line,Approved,6/1/23,"FDA Approved on June 1, 2023.",6
FWBI,Exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP),Phase 2,7/13/23,"Phase 2 topline Initial data from the study indicated the enhanced adrulipase formulation was safe and well tolerated and demonstrated an improvement over prior formulations of adrulipase. However, the preliminary data also indicate that it is likely the primary efficacy endpoint was not achieved, noted July13,. 2023.",6
PFE,Respiratory Syncytial Virus (RSV) in infants,Approved,8/21/23,"FDA approval on August 21, 2023.",6
ARWR,Alpha-1 Liver Disease,Phase 2,6/24/23,"Phase 2 data presented at EASL showed that at week 48, patients receiving 25, 100, or 200 mg fazirsiran achieved serum Z-AAT reductions of 74%, 89%, and 94%, respectively, versus an increase of 9% observed in patients receiving placebo, noted June 24, 2023.",6
IMCR,Uveal melanoma,Approved,1/26/22,"Approved January 26, 2022.",6
VRTX,Cystic fibrosis,Approved,12/21/20,"FDA approval announced December 21, 2020.",6
LMNL,Idiopathic pulmonary fibrosis (IPF),Phase 2,5/28/21,"Phase 2 trial will no longer be initiated - May 28, 2021.",4
SAGE,SRSE,Phase 3,9/12/17,"Phase 3 data released September 12, 2017 - primary endpoint not met.",4
AZN,First-line ovarian cancer following platinum-based chemotherapy,Approved,12/19/18,"FDA Approval announced December 19, 2018.",6
MRK,Renal cell carcinoma (RCC),Approved,11/18/21,"Approved November 18, 2021.",6
AZN,Head & neck cancer,Phase 3,12/7/18,"Phase 3 data released December 7, 2018 - primary endpoints not met.",4
ARGX,Chronic inflammatory demyelinating polyneuropathy,Phase 2,7/17/23,Phase 2 top-line data met primary endpoint; VYVGART Hytrulo demonstrated 61% reduction in risk of relapse versus placebo,6
GILD,HIV,Phase 2,2/16/22,"Phase 2 data reported high rates of viral suppression by Week 54 with 90% achieving an undetectable viral load, noted February 16, 2022.",6
MRK,Pneumococcal disease,Approved,7/16/21,"FDA approval announced July 16, 2021.",6
REGN,COVID-19 (Anti-viral antibody),Phase 2/3,9/30/21,"Phase 3 trial met primary endpoint, with 36% reduced risk of death by day 29, and 56% reduced risk in patients who were seronegative. Of 2,007 patients (REGEN-COV=1,340, placebo=667) serious adverse events occurred in 21% REGEN-COV patients and 26% placebo patients, noted September 30, 2021.",6
ASND,Solid tumors,Phase 1/2,11/11/22,"Phase 1/2 data reported that early signs of clinical activity were observed with sustained target immune system engagement over weeks, noted November 11, 2022.",6
INVA,Uncomplicated gonorrhea,Phase 3,11/1/23,"Phase 3 data met the primary endpoint when compared against the combination of injectable ceftriaxone and oral azithromycin, noted November 1, 2023.",5
GMAB,Metastatic cervical cancer,Phase 1/2,6/6/22,"Phase 1b/2 data reported an 41% objective response rate, noted June 6, 2022.",5
FRTX,Axillary hyperhidrosis,Phase 3,10/7/21,"Phase 3 study demonstrated that dosing was generally well-tolerated, the proportion of subjects achieving at least a 2-point improvement in the HDSM-Ax1 score from baseline to EOT2 was 49.3% (n=173). Additionally the change in GSP3 from baseline to EOT was -129.5mg. Overall, 82% of patients who were randomized to the dose in the study completed the full six weeks of treatment. Five (2.9%) subjects who received the dose discontinued due to a TEAE. No treatment-related Serious Adverse Events (SAEs) were reported. Noted October 7, 2021.",6
REGN,Non-small cell lung cancer (NSCLC),Approved,2/22/21,"FDA approval announced February 22, 2021.",6
INCY,Merkel cell carcinoma (MCC),Approved,3/22/23,"FDA Approved on March 22, 2023.",6
EPIX,Metastatic castration-resistant prostate cancer (mCRPC),Phase 1,10/22/23,"Phase 1 updated data presented at ESMO reported a PSA50 of 88%, PSA90 of 69%, and PSA <0.2 ng/mL of 56%, noted October 22, 2023.",6
YMAB,High-risk neuroblastoma (HR-NB) commonly,Phase 2,6/6/23,"Phase 2 interim data presented at ASCO reported that the ORR was 58% in patients with refractory disease and 42% in patients with relapsed disease, noted June 6, 2023.",6
CWBR,Nonalcoholic Fatty Liver Disease / Obesity / Nonalcoholic steatohepatitis,Phase 1b,8/10/21,"Phase 1b top-line data met primary endpoint - August 10, 2021.",6
BCYC,Solid tumors,Phase 1/2,9/6/22,"Phase 1/2 dose escalation cohort data reported an overall response rates of 22% and 67% in ovarian and urothelial cancers, with one complete response, noted September 7, 2022.",5
EYPT,Wet age-related macular degeneration (AMD),Phase 2,7/27/23,"Phase 2 trial continued to demonstrate that EYP-1901 is well tolerated with no reported drug-related ocular or systemic SAEs, noted July 27, 2023.",6
PRQR,Leber Congenital Amaurosis type 10 (LCA10),Phase 1/2,11/18/21,"Phase 1/2 data reported that the vast majority of the treated eyes demonstrated responses on multiple endpoints, noted November 18, 2021.",6
SPRO,Nontuberculous mycobacterial (NTM) pulmonary disease (PD),Phase 2,2/5/21,"Phase 2a trial placed on clinical hold - February 5, 2021.",4
ORIC,Metastatic prostate cancer,Phase 1b,10/7/21,"Phase 1b initial results showed that the dose was well tolerated, with no substantial added toxicity. PK showed no evidence of drug-drug interaction. Within the key patient population (8), 75% of patients' tumors expressed moderate to high GR and 25% of patients' tumors expressed low GR, noted October 7, 2021.",6
DAWN,Low-grade Glioma,Phase 2,6/5/23,"Additional Phase 2 presented at ASCO reported an ORR of 67% and clinical benefit rate (CBR) of 93% in 69 heavily pretreated RANO-HGG evaluable patients, noted June 5, 2023.",6
ATAI,Opioid Use Disorder,Phase 1,12/23/22,"Additional results from part 2 showed that a single dose was generally well tolerated and was observed to produce analgesic effects on CPT comparable to those seen in Part 1 of this trial, noted December 23, 2022.",6
ABBV,Augmentation of the chin region,Approved,6/15/20,"FDA Approval announced June 15, 2020.",6
FGEN,Idiopathic pulmonary fibrosis (IPF),Phase 3,6/26/23,"Phase 3 after ZEPHYRUS-1 results, company decided to discontinue this study, noted June 26, 2023.",4
ENTA,Respiratory Syncytial Virus RSV-A and RSV-B,Phase 1,6/20/23,"Phase 1 data demonstrated favorable safety, tolerability, and PK supportive of once-daily dosing, with good exposure multiples, thereby supporting further clinical advancement of EDP-323, noted June 20, 2023.",6
MRK,Ulcerative colitis,Phase 2,3/1/23,"Phase 2 trial met all ranked secondary endpoints, noted March 1, 2023.",5
NVS,Psoriatic arthritis,Phase 3,11/1/19,"Phase 3 data did not meet primary endpoint - October 31, 2019.",4
SGEN,Urothelial cancer,Approved,4/3/23,"FDA Approved on April 3, 2023.",6
PFE,Postherpetic neuralgia (PHN),Approved,10/12/17,"Approval announced October 12, 2017.",6
RHHBY,Influenza,Approved,10/18/19,"FDA Approval announced October 18, 2019.",6
XAIR,Solid tumors,Phase 1,11/3/23,"Phase 1 data presented at SITC demonstrated early clinical proof of concept, noted November 3, 2023.",6
AZN,Non-small Cell Lung Carcinoma,Approved,12/21/20,"FDA approval announced December 21, 2020.",6
FBIO,B-cell Non-Hodgkin Lymphoma & Chronic Lymphocytic Leukemia (CLL),Phase 1/2,8/16/23,"Initial Phase 1/2 data reported clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy, noted August 16, 2023.",6
AZN,Metastatic Breast Cancer,Phase 1/2,12/9/22,"Phase 1/2 presentation reported that treatment significantly reduced risk of death by 36%, noted December 9, 2022.",6
BGNE,Hepatocellular carcinoma (HCC),Phase 3,6/5/23,"Phase 3 additional data presented at ASCO reported that treatment demonstrated numerically longer median overall survival (OS) versus sorafenib in the biomarker subgroups of ALBI grade 1 (19.9 months vs. 16.9 months), PLR ≤141 (19.4 months vs. 14.5 months), and NLR ≤3 (20.9 months vs. 15.2 months, noted June 5, 2023.",6
PFE,Chronic Inflammatory Demyelinating Polyneuropathy (CIDP),Approved,2/12/21,"FDA approval announced February 12, 2021.",6
SIOX,Parkinson's disease,Phase 1/2,10/6/20,"Two evaluable patients in Cohort 2 demonstrated a 21-point mean improvement in the UPDRS Part III “OFF” score, representing a 40% improvement from the baseline average score of 52 in these patients. Two other patients not evaluated.",6
HLVX,GI.1/GII.4 virus-like particle (VLP) vaccine,Phase 2,7/20/22,"Phase 2 data reported that Pan-Ig and HBGA blocking antibody responses persisted to year 5 and no adverse events reported were determined to be related to treatment, noted July 20, 2022.",6
BMY,Mesothelioma,Approved,10/2/20,"FDA approval announced October 2, 2020.",6
MRK,COVID-19,Phase 3,2/21/23,"Phase 3 trial did not meet statistical significance, noted February 21, 2023.",6
ARCT,COVID-19 vaccine,Phase 1/2,12/28/20,"Phase 1/2 data released December 28, 2020.",5
BIIB,Biosimilar (ranibizumab),Approved,9/20/21,"Approved September 20, 2021.",6
CHRS,NSCLC neoadjuvant setting,Phase 3,6/3/23,"Phase 3 Interim event-free survival (EFS) analysis presented at ASCO reported that MPR and pCR rates per BIPR were higher in the toripalimab arm, 48.5% vs 8.4% and 24.8% vs 1.0%, respectively, noted June 3, 2023.",6
CHRS,Non-small cell lung cancer (NSCLC),Phase 3,6/6/23,"Phase 3 final overall survival and biomarker analyses presented at ASCO reported a significant improvement in OS was observed for the toripalimab arm over the placebo arm: median OS 23.8 vs 17.0 months, noted June 6, 2023.",6
PIRS,Asthma,Phase 2a,6/21/23,"Phase 2a AstraZeneca communicated to Pieris its decision to discontinue and cease dosing in the ongoing clinical studies, the decision was based on lung findings from a non-clinical 13-week GLP toxicology study, which are not a concern for the active clinical studies but do not support long-term use and progression to later-stage development, noted June 21, 2023.",4
CALT,Squamous Cell Carcinoma of the Head and Neck (SCCHN),Phase 2,7/13/23,"Phase 2 biomarker data reported that 7 out of the 16 evaluable patients were progression-free with either stable disease or partial response, out of which 6 were in the setanaxib arm and 1 was in the placebo arm, noted July 13, 2023.",6
NXTC,PD-1 axis inhibitor refractory non-small cell lung cancer (NSCLC),Phase 2,9/12/23,"Phase 2 data reported that 28% of patients (5/18) had durable clinical benefit with three of these being confirmed responses, noted September 12, 2023.",5
ZLDPF,Obesity,Phase 1b,6/24/23,"Phase 1b trial data showed that after one week of treatment, mean body weight reductions reached 2.6%, 3.6% and 4.2% from baseline following a single dose of 0.7 mg, 1.4 mg or 2.4 mg ZP8396, respectively. Reductions in body weight were dose-dependent, consistent and well-sustained during the additional five weeks of observation without further doses of ZP8396. Placebo-treated participants demonstrated a mean body weight increase of 0.6% after one week that continued to increase in most participants during the follow-up period, noted June 24, 2023.",6
VCNX,Non-small cell lung cancer (NSCLC),Phase 1/2,4/27/20,"Phase 1/2 updated data presented April 27, 2020 at AACR noted 81% of immunotherapy-naïve patients (17/21) experienced disease control - partial response (5/21 patients) or stable disease (12/21 patients)",6
SUPN,Migraine,Approved,4/5/17,"Tentative approval August 19 2016, subject to the pediatric exclusivity which expires March 28, 2017. Final approval announced April 6, 2017.",6
NVS,Hidradenitis Suppurativa (HS),Approved,10/31/23,"Approved October 31, 2023.",6
AZN,Ovarian cancer,Phase 3,3/12/20,Phase 3 trial did not meet primary endpoint.,4
INBX,Chondrosarcoma and mesothelioma,Phase 1,4/26/23,"Clinical hold lifted, noted April 26, 2023. Phase 1 combination cohorts are expected to begin reading out by year end 2023 and data from the registration-enabling trial in unresectable or metastatic conventional chondrosarcoma is expected during the 2H 2024.",5
AGIO,IDH1 mutant cholangiocarcinoma - cancer,Phase 3,9/30/19,Data presented at ESMO 2019 PFS HR HR=0.37.,5
PCVX,Invasive Pneumococcal Disease (IPD),Phase 1/2,10/19/23,"IND cleared by FDA, noted October 19, 2023",5
PRTC,Solid tumors,Phase 1/2,12/12/22,"Phase 1/2 data reported no dose limiting toxicities, noted December 12, 2022.",5
NKTR,Gram-Negative Pneumonia (INHALE 1),Phase 3,11/24/17,"Phase 3 data released November 24, 2017. Endpoints not met.",4
OTIC,Ear Tube Placement Surgery,Approved,12/11/15,"Approved December 11, 2015.",6
REGN,Multiple myeloma,Phase 2,12/12/22,"Phase 2 data reported a 64% response rate, with 45% of responders achieving a very good partial response or better, noted December 12, 2022.",6
AGIO,IDH1m Relapsed/Refractory AML - cancer,Approved,7/20/18,"FDA Approval announced July 20, 2018.",6
RCUS,Metastatic castrate-resistant prostate cancer (mCRPC),Phase 1/2,8/7/23,"Phase 1/2 trial is not expected to demonstrate sufficient clinical benefit in castrate resistant prostate cancer to warrant further investment, noted August 7, 2023.",6
TAK,Renal Cell Carcinoma (RCC),Phase 3,9/8/22,"Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.",6
PFE,Metastatic Castration-Sensitive Prostate Cancer (mCSPC),Approved,6/20/23,"FDA Approved on June 20, 2023",6
ANIX,Ovarian Cancer,Phase 1,10/13/23,"Phase 1 completed treatment of the first patient cohort, noted October 13, 2023.",6
GSK,Malaria,Approved,7/23/18,"FDA Approval announced July 23, 2018.",6
VBIV,COVID-19 vaccine,Phase 1/2,6/29/21,"Phase 1/2 initial data released June 29, 2021 - well-tolerated with no safety signals observed and induced neutralization titers in 100% of participants.",6
EBS,Anthrax Vaccine,Approved,7/21/23,"Approved July 21, 2023.",6
NVS,Metastatic castration-resistant prostate cancer (mCRPC),Approved,3/23/22,"Approved March 23, 2022.",6
PRVB,Ulcerative Colitis,Phase 1b,5/8/19,"Phase 1b top-line data May 8, 2019 noted primary safety and tolerability endpoint met but secondary efficacy endpoints were not met.",5
PFE,COVID-19 vaccine,Approved,6/17/22,"EUA approval for children 4 years - 6 months, noted June 17, 2022.",6
ARQT,Chronic Hand Eczema,Phase 2,5/4/21,Phase 1/2b trial did not meet primary endpoint.,4
GILD,COVID-19 Coronavirus,Approved,1/21/22,"Approval extended to non-hospitalized and pediatric patients January 21, 2022.",6
OGEN,Oral mucositis (OM),Phase 2,4/15/20,"Phase 2 data released April 15, 2020 did not meet primary endpoint.",4
VALN,Chikungunya Vaccine for adolecents,Phase 3,8/28/23,"Phase 3 trial data reported that VLA1553 was generally safe and well tolerated in adolescents aged 12 to 17 years, regardless of previous CHIKV infection, noted August 28, 2023.",6
BIIB,Alzheimer’s disease,Phase 3,10/25/23,"Phase 3 presentation at CTAD reported a 14% greater amyloid plaque removal than biweekly IV administration as suggested in a preliminary analysis using amyloid PET at 6 months of treatment, noted October 25, 2023.",6
ENSC,Nasal Opioid Abuse,Phase 1,10/31/22,"HAP data reported that PF614 powder produced significantly lower peak ""drug liking"" when compared with intranasal crushed IR oxycodone, noted October 31, 2022.",5
TPTX,Solid tumors,Phase 1,10/7/21,"Phase 1 updated data noted a confirmed responses for a cORR of 36% out of the 11 MET TKI-naïve NSCLC patients, cORR of 33% for the nine MET TKI-naïve GC/GEJ patients, and among the 12 patients with advanced other solid tumors, one patient with MET amplified colorectal cancer achieved a confirmed response, noted October 7, 2021.",6
MNKD,Type 1/2 diabetes,Approved,6/27/14,"Approved June 27, 2014.",6
BLU,Chronic cough,Phase 1,4/5/23,"Phase 1 bioavailability equivalence data reported that treatment was well tolerated and demonstrated equivalent bioavailability to twice-daily Immediate Release formulation, noted April 5, 2023.",6
ABEO,Recessive dystrophic epidermolysis bullosa (RDEB),BLA Filing,7/28/23,"BLA filing package completed, noted July 28, 2023. FDA pre-BLA meeting in August 2023, and expected BLA submission on 3Q, 2023.",6
CFRX,Serious infections caused by Staph aureus including MRSA,Phase 3,7/13/22,"Phase 3 DSMB interim futility recommended the trial be stopped, noted July 13, 2022.",4
ICVX,Respiratory syncytial virus (RSV) vaccine,Phase 1b,8/8/23,"Phase 1b 12 month extension trial in older adults, GMTs against RSV through day 365 persisted at ~45-70% of the GMTs at day 28 (for 75 and 250 µg unadjuvanted dosages), noted August 8, 2023.",5
RARE,Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD),Approved,6/30/20,"FDA Approval announced June 30, 2020.",6
LLY,HR+ / HER2- breast cancer,Approved,10/13/21,"Approved October 13, 2021.",6
BCRX,Reduce or eliminate attacks in HAE patients,Approved,12/3/20,"FDA approval announced December 3, 2020",6
OVID,Dravet Syndrome / Lennox-Gastaut Syndrome,Phase 3,4/27/23,"Phase 3 data reported that 78% of patients tolerated the highest dose level well with sustained reduction in frequency of drop seizures, noted April 27, 2023.",6
LYRA,Chronic rhinosinusitis / Chronic Sinusitis,Phase 2,9/10/22,"Additional Phase 2 results data presented at ARS demonstrated that significantly more patients at baseline improved to mild/no symptoms at week 24 after treatment compared to control, noted September 10, 2022.",6
BLPH,Pulmonary hypertension associated with Fibrotic Interstitial Lung Disease,Phase 3,6/5/23,"Phase 3 enrollment completed, noted January 18, 2023. Phase 3 study did not meet the primary endpoint with iNO45 performing worse than placebo by 5.49 minutes per day, noted June 5, 2023.",6
ONCT,Chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL),Phase 1/2,12/12/22,"Phase 1/2 data reported that the ORR of 89% and CR rate of 43% for patients with MCL treated with zilovertamab plus ibrutinib compare favorably to the historical ORR of 66% and CR rate of 20% for ibrutinib monotherapy, noted December 12, 2022,",6
MRK,Metastatic Castration-Resistant Prostate Cancer,Phase 3,3/15/22,"Phase 3 trial stopped following the recommendation of an independent Data Monitoring Committee (DMC), noted March 15, 2022.",4
RHHBY,Urothelial carcinoma,Phase 3,8/5/19,"Phase 3 PFS co-primary endpoint met - August 5, 2019.",6
KPTI,"Myelodysplastic syndrome (MDS), hypomethylating agents (HMA) refractory",Phase 2,5/3/23,"Phase 2 data reported that treatment demonstrated a 27% overall response rate (ORR) in the intent-to-treat (ITT) population and a 31% ORR in the efficacy evaluable population, noted May 3, 2023.",6
KDNY,"Primary Hyperoxaluria, Healthy Volunteers",Phase 1,6/17/23,"Phase 1 healthy volunteer data at ERA 2023 showed that dosing was generally well tolerated in HVs who received single doses up to 500 mg and multiple doses (14 days) up to 60 mg, noted June 17, 2023",6
LLY,Pancreatic ductal adenocarcinoma (PDAC),Phase 3,10/16/19,"Phase 3 top-line data did not meet primary endpoint - October 16, 2019.",4
MNKKQ,Jaundice,CRL,8/22/18,"CRL issued August 22, 2018.",5
BNTC,Oculopharyngeal muscular dystrophy (OPMD),Phase 1/2,6/26/23,"Phase 1b/2a IND clearance on June 26, 2023.",5
PFE,Pneumonia and influenza for >65 yrs,Phase 3,9/29/21,"Phase 3 top-line results showed responses to the combination were noninferior to those elicited by the vaccines when administered one month apart. The safety profile of the combination was similar as compared to when each vaccine was administered separately, noted September 29, 2021.",5
IRON,Dry Age-related Macular Degeneration / Geographic Atrophy,Phase 2a,11/12/21,"Additional data released November 12, 2021. Through 510 intravitreal administrations of GEM 103, no ocular serious adverse events related to study drug reported, no ocular adverse events leading to study discontinuation, and no anti-GEM103 antibodies detected in plasma. 17 patients (27.4%) experienced mild to moderate ocular adverse events in the study eye including two intraocular inflammation adverse events. Inflammation was mild or moderate and the patients recovered without recurrence and no study drug interruption. The moderate case was unrelated to GEM103 and the patient had a history of autoimmune disease. No increased risk observed for Choroidal Neovascularization (CNV). Two adverse events of neovascular AMD in the study eye occurred; in both cases, there were no definitive evidence of CNV by independent reading center's analysis of the retinal imaging. These events were determined to be unrelated to GEM103 and there was no impact on vision.",6
LNTH,Left Ventricular Ejection Fraction,Phase 3,2/3/20,"Phase 3 trial did not meet primary endpoint - February 3, 2020.",4
GSK,Severe eosinophilic asthma - pediatric,Approved,6/6/19,"FDA Approval announced June 6, 2019.",6
FGEN,Pancreatic cancer,Phase 2,1/17/17,"Phase 2 abstract released January 17, 2017.",5
IONS,Familial chylomicronemia syndrome (FCS),CRL,8/27/18,"CRL issued August 27, 2018.",5
VERA,IgA Nephropathy,Phase 2b,6/17/23,"Phase 2b 36-week presented at ERA 2023 showed that atacicept 150 mg resulted in a delta of 43% versus placebo in mean proteinuria reduction in per-protocol analysis and demonstrated statistically significant stabilization of eGFR versus placebo in this high-risk population. Phase 3 initiated on June, noted June 17, 2023.",6
DMAC,Chronic Kidney Disease,Phase 2,11/4/21,"Phase 2 data presented November 4, 2021 reported statistically significant reductions (over 30% decrease) in albuminuria and early signals of potential disease modification with the APRIL and IgA1 biomarkers decreasing 35% and 22%, respectively.",6
HCM,Follicular Lymphoma and Marginal Zone Lymphoma,Phase 2,6/9/23,"Phase 2 data from ICML presentation noted that median duration of response (DoR) and progression free survival (PFS) at median follow-up duration of 22.1 months was not reached, noted June 9, 2023.",6
AMPH,Opioid overdose,Approved,3/7/23,"Approved March 8, 2023.",6
MAIA,Non-small cell lung cancer (NSCLC),Phase 2,10/31/23,"Phase 2 preliminary efficacy data reported a 100% Disease Control Rate (DCR), noted October 31, 2023.",5
TTNP,Opioid dependence,Approved,5/26/16,"CRL issued April 30, 2013. Approved May 26, 2016.",6
BPTS,COVID-19,Phase 2/3,5/25/23,"Phase 2/3 data reported that at 28 days the adjusted difference in the proportion of patients with hospital discharge of 11% showed a trend towards a statistically significant difference in BIO101 group versus placebo in ITT population, noted May 25, 2023.",6
KZIA,Glioblastoma (GBM),Phase 2/3,10/31/23,"Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.",5
ANNX,Huntington’s disease (HD),Phase 2,6/7/22,"Phase 2 data reported that treatment led to complete and durable target engagement, disease progression was stabilized in the overall HD patient population for the entire nine months, and three trial discontinuations were due to treatment, noted June 7, 2022.",6
INO,Glioblastoma (GBM),Phase 1/2,11/5/19,Phase 1/2 interim PFS data noted 80% (16/20) of MGMT gene promoter methylated patients and 75% (24/32) of unmethylated patients were progression-free at six months.,6
CMMB,Liver Fibrosis,Phase 2a,1/3/23,"Phase 2 top-line data reported that trial met its primary endpoints, noted January 3, 2023.",5
ALNY,Primary hyperoxaluria type 1 (PH1),Phase 3,11/5/21,"Phase 3 full results reported a 33 percent and 42 percent mean reduction in POx and predialysis POx from baseline to Month 6. Reduction in POx was evident by Month 1 and persisted through the end of the month 6, noted November 5, 2021.",6
CORT,Cushing's Syndrome,Approved,2/17/12,"Approved February 17, 2012.",6
CORT,Long-standing antipsychotic-induced weight gain (APIWG),Phase 2,12/8/22,"Phase 2 data reported that AIWG did not reverse, noted December 8, 2022.",5
AEZS,Adult Growth Deficiency,Approved,12/20/17,"Approval announced December 20, 2017.",6
KNSA,Giant cell arteritis (GCA),Phase 2,10/6/20,"Phase 2 trial met endpoints - October 6, 2020.",6
MTNB,Severe hypertriglyceridemia,Phase 2,2/1/21,"Phase 2 data released February 1, 2021. Primary endpoint not met.",4
ASND,Growth hormone deficiency in children,Approved,8/25/21,"FDA approval announced August 25, 2021.",6
RVMD,Solid Tumors,Phase 1,10/13/23,"Phase 1 preliminary data shared at AACR-NCI-EORTC reported a median time to response of 1.3 months, and median time on treatment of 3.5 months in NSCLC, with a median time to response of 1.4 months, and median time on treatment of 2.4 months in CRC, noted October 13, 2023.",6
IMUX,Relapsing-remitting multiple sclerosis (RMS),Phase 2,10/13/23,"Phase 2 data presented at ECTRIMS reported that VidoCa was associated with a Nfl reduction from baseline up to W6 of -3% for the pooled 30 and 45mg VidoCa group compared to +2.5% for placebo, and up to W24 of -19% and +7%, respectively, noted October 13, 2023.",6
AVIR,Dengue,Phase 2,4/17/23,"Additional Phase 2 data presented at ECCMID reported that AT-752 was well tolerated and had no clinically relevant effects on cardiac repolarization, heart rate, PR or QRS intervals, noted April 17, 2023.",6
INZY,ENPP1 deficiency,Phase 2,9/26/23,"Phase 2 interim data from cohort 1-3 reported an improvement in the Global Impression of Change (GIC) observed in all three dose cohorts in ABCC6 Deficiency (PXE) trial, noted September 26, 2023.",6
MRK,Cervical Cancer,Approved,10/13/21,"Approved October 13, 2021.",6
NVS,HR+/HER2− metastatic breast cancer,Phase 2,12/6/22,"Phase 2 data reported that treatment doubled the median PFS vs. combination CT at 24.0 months compared to 12.3 months, noted December 6, 2022.",6
RHHBY,"Hepatocellular Carcinoma (HCC), Solid tumors",Phase 1,4/17/23,"Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.",6
REGN,"Wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR)",Approved,8/18/23,"Approved August 18, 2023.",6
IONS,Cardiovascular (CV) risk reduction and hypertriglyceridemia,Phase 2b,11/24/21,"Phase 2b updated data demonstrated that in the dose-ranging study in subjects with elevated non-HDL-C and triglycerides (TG), the study met its primary endpoint, achieving a statistically significant reduction in non-HDL-C at all doses tested at 24 weeks, compared to placebo, noted November 24, 2021.",6
SVRA,Nontuberculous mycobacteria (NTM),Phase 2,3/12/20,"Phase 2 top-line data released March 12, 2020.",5
ALNY,IgA nephropathy,Phase 2,8/29/22,"Phase 2 new results demonstrated an equivalent 36% reduction in 24-hour urine total protein and a higher proportion, 32 vs 13%, of patients treated in comparison to placebo, respectively, achieving greater than or equal to 50% reduction in 24-hour UPCR, noted August 29, 2022.",6
SUPN,Bipolar depression,Phase 3,5/5/20,"Phase 3 development has been terminated - May 5, 2020.",4
DVAX,Asthma,Phase 2a,11/29/18,"Phase 2a trial did not meet primary endpoint - November 29, 2018.",4
ABBV,Non-radiographic axial spondyloarthritis,Approved,10/21/22,"FDA. Approved on October 21, 2022.",6
ACOR,Parkinson’s disease (PD),Phase 3,11/15/17,"Phase 3 data due 1Q 2018. Noted November 15, 2017 seven cases of sepsis, five of which were fatal.",5
RHHBY,Third-line advanced or metastatic colorectal cancer,Phase 3,5/10/18,"Phase 3 data released May 10, 2018. Primary endpoint not met.",4
GSK,"Meningitis, meningococcal disease caused by serogroups A, B, C, W and Y",Phase 3,5/12/23,"Phase 3 data reported that the trial met all primary endpoints, noted May 12, 2023.",5
HALO,Generalized Myasthenia Gravis (gMG),Approved,6/20/23,"FDA Approved on June 20, 2023.",6
AZN,Factor Xa inhibitor reversal agent,Approved,12/31/18,"Prior Approval Supplement (PAS) FDA Approval announced December 31, 2018.",6
VRTX,Beta-thalassemia,Phase 1/2,6/11/21,"Phase 1/2 data presented at EHA meeting June 11, 2021. All 15 patients were transfusion independent with follow-up ranging from 4 to 26 months after CTX001 infusion and had clinically meaningful improvements in total hemoglobin from 8.9 to 16.9 g/dL and fetal hemoglobin from 67.3% to 99.6% at last visit.",6
EGRX,Exertional heat stroke (EHS),CRL,8/10/20,"CRL announced August 10, 2020.",5
BCRX,Hereditary Angioedema,Phase 2,5/7/23,"Additional long-term 96 weeks at 150mg data reported that the attack-free status was consistently high with ORLADEYO 150 mg regardless of patients' age, gender and prior prophylactic treatment, noted May 7, 2023.",6
RARE,Osteogenesis Imperfecta,Phase 2,10/14/23,"Additional Phase 2 data reported that the annualized fracture rate across all 24 patients in the Phase 2 portion of the study was reduced by 67%, noted October 14, 2023.",6
KOD,Treatment-Naive Wet Age-Related Macular Degeneration (AMD),Phase 3,10/13/23,"Phase 3 data presented at the Retina Society meeting reported a favorable safety profile with low rates of intraocular inflammation and no cases of intraocular inflammation with vasculitis or vascular occlusion, noted October 13, 2023.",6
VRTX,Children with cystic fibrosis (CF) ages 1 month to less than four months old,Approved,5/3/23,"FDA approved on May 3, 2023.",6
FDMT,Wet age-related macular degeneration (AMD),Phase 1/2,7/29/23,"Phase 1/2 additional dose response data demonstrated in favor of highest tested dose of 3E10 vg/eye, including 100% reduction in supplemental anti-VEGF injections (4 of 4 evaluable patients injection-free) and a clinically meaningful reduction in central subfield thickness (CST) at 36 weeks in patient population with high anti-VEGF need, noted July 29, 2023.",6
HRMY,Excessive daytime sleepiness associated with Prader-Willi Syndrome (PWS),Phase 2,7/20/23,"EoP2 meeting completed, noted July 20, 2023",6
MBIO,B-cell Non-Hodgkin Lymphoma & Chronic Lymphocytic Leukemia (CLL),Phase 1/2,8/16/23,"Initial Phase 1/2 data reported clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy, noted August 16, 2023.",6
CADL,Pancreatic Cancer,Phase 2,11/3/23,"Phase 2 initial overall survival and immunological biomarker data presented at SITC revealed notable improvements in patients with borderline resectable pancreatic ductal adenorcarcinoma (PDAC) after experimental treatment with CAN-2409, noted November 3, 2023.",6
JNJ,metastatic hormone-naïve prostate cancer (mHNPC),Approved,2/8/18,"sNDA approval announced February 8, 2018.",6
AZN,COVID-19,Phase 3,1/26/23,"FDA no longer allows treatment to be used as Emergency Use Authorization (EUA) due to the lack of effectiveness against the currently circulating variants, noted January 26, 2023.",6
ABBV,Waldenström’s Macroglobulinemia,Approved,1/29/15,"Approved January 29, 2015 - PCYC",6
EQRX,CNS metastases and EGFR-mutated non-small cell lung cancer (NSCLC),Phase 3,6/6/22,"Phase 3 data reported that CNS PFS events were observed in 11 patients (38%) treated, and the confirmed CNS ORR were 82.8% and 75.0% in patients treated with Au and G, respectively, noted June 6, 2022.",6
AGEN,Solid cancers,Phase 1,6/4/18,Phase 1 presentation at ASCO 2018. 31% ORR.,5
AGEN,"Advanced Solid tumors, Colorectal cancer",Phase 1b,10/21/23,"Phase 1b data presented at ESMO reported an overall response rate (ORR) of 24% was observed, compared to 2.8% reported with standard of care (SOC), and a 12-month overall survival (OS) of 74% and median OS (mOS) not yet reached, noted October 21, 2023.",6
NGM,Non-alcoholic steatohepatitis (NASH),Phase 2b,5/24/21,"Phase 2b top-line data released May 24, 2021. Primary endpoint not met.",4
ALXO,Head and neck squamous cell carcinoma (HNSCC),Phase 2,11/9/21,"Phase 2 clinical data showed that patients with 1L HNSCC who have not received prior treatment for their advanced disease (n=13), the dose demonstrates an initial ORR of 38.5% with a 12-month OS rate of 87.5% and mOS not reached in combination with pembrolizumab + 5FU + platinum. These results compare favorably with benchmark survival data from standard pembrolizumab + chemotherapy, noted November 9, 2021.",6
SNDX,Triple negative breast cancer (TNBC),Phase 2,3/7/19,"Phase 2 top-line data March 7, 2019 failed to meet endpoints.",4
AZN,Non-small cell lung cancer (NSCLC),Approved,11/11/22,"Approved November 11, 2022.",6
RHHBY,Relapsed/Refractory Multiple Myeloma (RRMM),Phase 1,12/12/22,"Phase 1 1 year data reported that the best overall response (BOR) achieved was: stringent complete response (sCR) in 7 patients, CR in 3 patients, very good partial response (VGPR) in 5 patients, and PR in 1 patient, noted December 12, 2022.",6
SNY,Hidradenitis suppurativa / atopic dermatitis,Phase 1,12/14/22,"Phase 1 data reported that a 37% mean reduction in EASI in AD and 42% of HS patients has AN Count 0/1/2, noted December 14, 2022.",6
VBIV,Hepatitis B vaccine,Phase 3,9/21/22,"Additional Phase 3 data reported that treatment may be effective only in low level HBsAg carriers with a low tolerizing effect of HBsAg, noted September 21, 2022.",6
RCUS,Non-small cell lung cancer (NSCLC),Phase 2,6/5/23,"Phase 2 presentation at ASCO reported one complete response in the monotherapy and in the DZ combo, noted June 5, 2023.",6
TAK,Negative symptoms of schizophrenia,Phase 2,3/2/21,"Phase 2 trial did not meet primary endpoint - March 2, 2021.",4
GILD,Large B-cell lymphoma (LBCL) after one prior line of therapy who were deemed ineligible for high-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT),Phase 2,9/18/23,"Phase 2 trial met its primary endpoint, noted September 18, 2023.",5
CLLS,Large B-cell lymphoma,Phase 1,6/15/23,"Additional data reported at ICML across all 33 patients the CR rate was 42% with 30% maintaining a CR at Month 6. Results indicated complete responses are more common with lymphodepletion regimens containing 90 mg of ALLO-647 (FCA90). Median duration of response for both the overall population and the patients treated with the Phase 2 regimen (n=12) was 23.1 months, noted June 15, 2023.",6
XERS,Type 1 diabetes,Phase 2,6/18/20,"Phase 2 data released June 18, 2020. 62% reduction of hyperglycemia after a glucose challenge.",6
ARGX,Myasthenia gravis (MG),Approved,12/17/21,"Approved December 17, 2021.",6
ALDX,Dry eye disease,Phase 3,7/12/22,"Phase 3 crossover results reported that met both primary endpoints, noted July 12, 2022.",5
TEVA,Osteoarthritis pain of the hip or knee,Phase 3,8/5/20,"Phase 3 data achieved the co-primary endpoints for fasinumab 1 mg monthly. Fasinumab every two months did not reach statistical significance - August 5, 2020.",5
SRPT,Duchenne Muscular Dystrophy,Approved,6/22/23,"FDA acelerated Approval on June 22, 2023.",6
JNCE,Non-small cell lung cancer and bladder cancer,Phase 2,11/2/20,"Phase 2 interim analysis noted the trial will not meet pre-specified interim criteria for continuation of enrollment - November 2, 2020.",4
PFE,Spinal Muscular Atrophy (SMA),Phase 3,9/14/23,"Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.",5
ABBV,Preventive treatment of migraine,Approved,4/18/23,"Approved April 17, 2023.",6
MRK,HER2-positive gastric or Gastroesophageal junction (GEJ) adenocarcinoma,Phase 3,10/20/23,"Phase 3 trial data presented at ESMO demonstrated a statistically significant PFS improvement in advanced HER2-positive gastric or GEJ adenocarcinoma in tumors expressing PD-L1 (Combined Positive Score [CPS] ≥1) compared to trastuzumab and chemotherapy alone October 20, 2023.",6
RHHBY,Coronavirus COVID-19,Phase 3,7/29/20,"Phase 3 trial did meet primary endpoint - July 29, 2020.",6
PRQR,Autosomal dominant retinitis pigmentosa (adRP),Phase 1/2,11/18/21,"Phase 1/2 data reported 3 cases of cataract worsening, with maximum benefit in BCVA observed after 5 weeks and declined thereafter. A mean BCVA benefit of +1.4 letters was reported across all subjects. In doses 75 - 300µg, the mean BCVA benefit was +5 letters, maximum benefit observed was +7 letters, noted November 18, 2021.",6
NVS,Acute myeloid leukemia (AML),Approved,4/28/17,"Priority review granted November 14, 2016. Approval announced April 28, 2017.",6
AVTX,Non-Eosinophilic Asthma (NEA),Phase 2,6/26/23,"Phase 2 did not meet the primary endpoint measured by reduction in asthma related events compared to placebo, noted June 26, 2023.",6
TPST,Hepatocellular carcinoma (HCC),Phase 1/2,10/11/23,"Phase 1b/2 trial had a confirmed objective response rate of 30% for the TPST-1120 triplet arm versus 13.3% for the atezolizumab + bevacizumab control arm; duration of response (""DoR"") has not yet been reached, noted October 11, 2023.",6
HCM,Intrahepatic cholangiocarcinoma (IHCC),Phase 2,5/25/23,"Phase 2 first in human data reported that the best overall response by investigator assessment was confirmed partial response in seven (31.8%) pts and stable disease in an additional 12 pts, noted May 25, 2023.",5
CYCN,Alzheimer's disease with vascular features (ADv),Phase 2a,5/11/23,"Phase 2a data was inconclusive, noted May 11, 2023.",5
MRK,Gastric cancer,Phase 3,4/25/19,"Phase 3 data released April 25, 2019 noted that monotherapy arm met noninferiority primary endpoint. Combo arm did not meet OS/PFS endpoints.",5
BIIB,Small Fiber Neuropathy,Phase 2,9/16/21,"Phase 2 200mg dosing data met primary endpoint, did not meet statistical significance for secondary endpoints. Phase 2 330 mg dosing data did not meet the primary endpoint or secondary endpoints, September 16, 2021.",4
HRMY,Developmental and Epileptic Encephalopathies (DEE),Phase 2,9/18/19,"Phase 2 top-line data released September 18, 2019. 10/46 reported a serious adverse event (SAE).",4
GTHX,Second/third-line small-cell lung cancer,Approved,2/12/21,"FDA approval announced February 12, 2021.",6
VRCA,Basal cell carcinoma,Phase 2,8/10/23,"Phase 2 data presented at AAD Innovation Academy show consistency inclinical and histological clearance of treated BCC lesions was observed by Day 49 post-treatment with the 8 mg dose of VP-315, with 4 of 6 subjects (67%) showing complete tumor clearance, noted August 10, 2023.",6
AZN,Heart failure,Approved,5/5/20,"FDA Approval announced May 5, 2020.",6
ICPT,NASH patients with compensated cirrhosis,Phase 3,8/2/23,"NASH Programs discontinued, noted August 2, 2023.",4
SGEN,Frontline CD30-positive mature T-cell lymphomas - cancer,Approved,11/16/18,"FDA Approval announced November 16, 2018.",6
LGND,Postpartum Depression - moderate,Approved,3/19/19,"FDA approval announced March 19, 2018.",6
AADI,Open-angle glaucoma (OAG),Phase 2,12/11/20,Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.,6
OVID,Adolescents with Angelman syndrome,Phase 3,12/1/20,"Phase 3 trial did not meet primary endpoint - December 1, 2020.",4
LPCN,Postpartum Depression,Phase 1,5/16/23,"Pilot clinical PK bridge study data reported that treatment is well-tolerated with no hypoxia or sedation-related adverse events, noted May 16, 2023.",6
AZN,Paroxysmal nocturnal hemoglobinuria (PNH),Approved,12/21/18,"FDA approval announced December 21, 2018.",6
ZVRA,Vascular Ehlers-Danlos Syndrome,CRL,6/25/19,"CRL issued June 25, 2019.",5
HCM,Primary immune thrombocytopenia (ITP),Phase 3,8/21/23,"Phase 3 trial met is primary endpoint, noted August 21, 2023.",5
PHGE,Acne,Phase 2,11/15/21,"Program to be discontinued to focus resources on CF and AD programs, noted November 15, 2021.",4
PTCT,Spinal Muscular Atrophy (SMA) Type 1,Approved,8/7/20,"FDA Approval announced August 7, 2020.",6
MRK,COVID-19,Phase 3,4/1/22,"Phase 3 final analysis reported that at day 3 infectious SARS-CoV-2 was detected in 0.0% of treated patients, compared with 21.8% with placebo, noted April 1, 2022.",6
OTIC,Hearing loss,Phase 1/2,12/17/20,"Phase 1/2 data released December 17, 2020. 6 out of 9 (67%) patients demonstrated a clinically-meaningful improvement on at least one of the three SIN tests at both Days 57 and 85 versus 0 out of 8 (0%) for placebo.",6
OLMA,Breast Cancer,Phase 2,5/11/23,"Phase 2 data presented at ESMO Breast Cancer reported that partial responses were observed in five patients, with 59% patients remaining on treatment, noted May 11, 2023.",6
NVCT,Ovarian Clear Cell Carcinoma,Phase 1,10/13/23,"Phase 1 data presented at AACR-NCI-EORTC reported that NXP800 induced phosphorylation of eIF2a via activation of the stress activated kinase GCN2, noted October 13, 2023.",6
ABBV,Marginal zone lymphoma,Approved,1/19/17,"sNDA filing under priority review approved January 19, 2017.",6
GSK,Ovarian cancer,Approved,10/23/19,"FDA Approval announced October 23, 2019.",6
OKYO,Dry Eye Disease,Phase 2,10/5/23,"Phase 2 data reported a dropout rate at 5.4% and that treatment was well tolerated, noted October 5, 2023.",6
LIAN,Hypertrophic cardiomyopathy (HCM),Approved,4/28/22,"Approved April 28, 2022.",6
APTX,Post-traumatic stress disorder (PTSD),Phase 2,10/19/20,"Phase 2 data released October 19, 2020. Primary endpoint met.",6
PFE,Pneumococcal + Covid-19 vaccine dual administration in 65<,Phase 3,1/12/22,"Phase 3 top-line results noted that responses for all 20 serotypes and to the covid-19 vaccine were similar, with a safety profile similar to the booster dose, noted January 12, 2022.",5
PFE,Generalized anxiety disorder (GAD),Phase 3,2/10/20,Phase 3 trial did not meet primary endpoint.,4
NOVN,Acne vulgaris,Phase 3,1/27/17,"Phase 3 top-line data released January 27, 2017. Met one of three co-primary endpoints in NI-AC301 trial. Met all endpoints in NI-AC302 trial.",6
AMGN,Behçet’s Disease,Approved,7/19/19,"FDA Approval announced July 19, 2019.",6
BIIB,Rheumatoid arthritis (RA),Approved,9/29/23,"FDA Biosimilar of ACTEMRA approved on September 29, 2023.",6
SPRY,Allergic reactions (type I) including anaphylaxis,CRL,9/20/23,"CRL announced September 20, 2023.",5
CVM,Head and Neck cancer,Phase 3,10/30/23,"Phase 3 data finalized target population data resulted in risk of death cut in half at five years versus the control, 28.6% absolute 5-year overall survival benefit versus control, 0.349 hazard ratio vs control , noted October 30, 2023.",5
ALDX,Noninfectious anterior uveitis,Phase 3,6/25/19,"Phase 3 data did not meet endpoints - June 25, 2019.",4
MRK,Cervical cancer,Approved,6/12/18,"Approval announced June 12, 2018.",6
NVO,Type 2 diabetes,Approved,12/5/17,"Approval announced December 5, 2017.",6
CYTK,Amyotrophic lateral sclerosis (ALS),Phase 3,3/31/23,"DMC review recommended the discontinuation of the trial due to futility, noted March 31, 2023.",4
ERAS,Colorectal cancer and gastrointestinal (GI) cancer,Phase 1/2,6/5/23,"Initial Phase 1b/2 data showed a 50% response rate, including two confirmed partial responses (cPR) and one unconfirmed partial response (uPR), and 67% disease control rate in EC-naïve response evaluable patients at the highest dose of ERAS-007 tested (100 mg BID-QW), development deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023",6
LIFE,Pulmonary sarcoidosis,Phase 1/2,5/22/23,"Phase 1b/2a data presented at the American Thoracic Society reported that treatment led to an exposure-dependent decrease in the extent of oral corticosteroid usage, increase in lung function as measured by percent predicted forced vital capacity and improvement in clinical symptoms as measured by the King's Sarcoidosis Questionnaire-Lung score, noted May 22, 2023.",6
RAIN,Solid tumors with loss of CDKN2A Gene,Phase 1,5/30/23,"Phase 1 basket trial to be terminated, noted May 5, 2023.",4
EYPT,Diabetic macular edema,Approved,9/26/14,Approved Sept 26 2014.,6
BFRI,Actinic keratosis (AK),Approved,10/10/23,"Approved October 10, 2023.",6
LLY,Psoriasis,Phase 3,7/17/20,"Phase 3 trial met primary and all key secondary endpoints vs placebo at Week 16 and all key secondary endpoints versus Cosentyx (secukinumab) at Week 16 and Week 52 - July 17, 2020.",6
JNJ,Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).,Approved,4/21/20,"FDA Approval announced April 21, 2020.",6
ALNY,Acute hepatic porphyrias,Approved,11/20/19,"FDA approval announced November 20, 2019.",6
OTIC,Ménière's disease,Phase 3,2/22/21,Phase 3 trial did not achieve the primary endpoint.,4
ERYP,Acute lymphoblastic leukemia (ALL),Phase 2,12/6/20,"Phase 2 data presented at ASH December 6, 2020. Endpoints met.",6
SPPI,Colorectal cancer,Approved,4/29/11,"Approved April 29, 2011.",6
BGNE,B-cell malignancies intolerant to ibrutinib and/or acalabrutinib,Phase 2,6/15/23,"Phase 2 preliminary study demonstrated that treatment with BRUKINSA was well-tolerated and unlikely to lead to recurrence of intolerant adverse events experienced with prior BTK-inhibitor therapy, suggesting BRUKINSA may be a treatment option for these patients, noted June 15, 2023.",6
UTHR,Pulmonary arterial hypertension (PAH),Phase 3,10/21/21,"78 week data noted an improved health-related quality of life at month six, noted October 21, 2021.",6
PFE,Alopecia areata,Approved,6/23/23,"FDA Approved on June 23, 2023.",6
AZN,Hepatocellular carcinoma (HCC),Approved,10/24/22,"Approved October 24, 2022.",6
KYMR,Diffuse large B-cell lymphoma (DLBCL),Phase 1,6/14/23,"Phase 1 patient dose escalation data cohorts show target knockdown at or near levels associated with clinical efficacy in preclinical tumor models, with no dose limiting toxicities (DLTs) observed, noted June 14, 2023.",6
ADXS,Desmoid tumors,Phase 2/3,10/23/23,"Phase 2 data presented at ESMO showed that 1.2 mg once daily treatment achieved Overall Response Rate of 83% in the evaluable population, AL102 1.2 mg once daily treatment resulted in 88% reduction in tumor volume and 85% reduction in T2W signal intensity, noted October 23, 2023.",6
XNCR,"Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, DLBCL",Phase 1,12/12/22,"Phase 1 data reported an overall response rate (ORR) was 52.0% (13/25), and the complete response rate was 24.0% (6/25), noted December 12, 2022.",5
BGNE,Refractory chronic lymphocytic leukemia (CLL),Phase 3,6/15/23,"Phase 3 results presnted at ICML In a subgroup analysis showed improved PFS over ibrutinib in patients in China, consistent with the global study population, noted June 15, 2023.",6
MRK,Classical Hodgkin lymphoma,Approved,10/15/20,"FDA Approval announced October 15, 2020.",6
VNDA,Atopic dermatitis,Phase 3,2/25/20,"Phase 3 trial did not meet primary endpoint - February 25, 2020.",4
VNDA,Jet Lag Disorder,CRL,8/19/19,"CRL issued August 19, 2019.",5
IMVIQ,Platinum Resistant Ovarian Cancer,Phase 2,12/9/21,"Phase 2 data reported increases in both T and B cell immune infiltration, and more prominent antigen-specific humoral responses, noted December 9, 2021.",6
ABBV,Post-Operative Atrial Fibrillation (POAF) in patients undergoing open-chest cardiac surgery,Phase 2,11/7/22,"Phase 2 data reported that primary endpoint was not met, noted November 7, 2022.",5
SNY,Hemophilia A,Approved,2/23/23,"Approved February 23, 2023.",6
ARWR,Dyslipidemia,Phase 2b,11/7/22,"Phase 2b data presented at AHA reported that ARO-ANG3 is associated with relative reduction in liver fat fraction at Week 24, with no AEs related to LFT changes reported to date, noted November 7, 2022.",6
BLUE,β-Thalassemia,Approved,8/17/22,"Approved August 17, 2022.",6
AMGN,RITUXAN biosimilar - non-Hodgkin lymphoma,Approved,12/17/20,"FDA approval announced December 17, 2020.",6
KOD,Diabetic macular edema (DME),Phase 3,7/24/23,"Phase 3 trial did not meet the primary endpoint, noted July 24, 2023.",6
AXSM,Smoking cessation,Phase 2,4/15/19,"Phase 2 top-line data met primary endpoint - April 15, 2019.",6
ENTA,HCV - genotype 1,Approved,12/19/14,"Approved December 19, 2014.",6
GILD,Hepatitis C,Approved,3/19/20,"sNDA FDA Approval announced March 19, 2020.",6
KOD,Wet age-related macular degeneration (AMD),Phase 2/3,5/3/22,"Phase 2b/3 primary results reported durability with nearly 60% on Q20W dosing, however trial did not meet primary endpoint, noted May 3, 2022.",6
DNLI,Amyotrophic lateral sclerosis (ALS),Phase 1b,4/25/23,"Phase 1b data due presented at AAN reported low variability, predictable dose-related increases in exposure, and a half-life supporting once-daily dosing, noted April 25, 2023.",6
AIM,Cisplatin resistant advanced recurrent ovarian cancer,Phase 1/2,1/24/22,"Phase 1/2 IP chemo-immunotherapy combination demonstrated to be well tolerated, and associated with interferon stimulated gene changes that favor cytotoxic T lymphocytes chemo-attraction and function, noted January 24, 2022.",6
TEVA,Tourette Syndrome,Phase 2/3,2/19/20,"Phase 2/3 trial did not meet primary endpoint - February 19, 2020.",4
RYTM,POMC deficiency obesity / Leptin Receptor Deficiency Obesity,Approved,11/27/20,"FDA approval announced November 27, 2020.",6
BMY,First-line Glioblastoma (GBM) cancer,Phase 3,12/23/20,Phase 3 trial unlikely to meet OS primary endpoint,4
BYSI,Non small cell lung cancer,Phase 3,12/13/22,"Additional data reported that plinabulin demonstrated a superior benefit for Gr4N, Gr3/4N, all GrN and DSN compared to the control, noted December 13, 2022.",6
RZLT,Congenital hyperinsulinism (HI),Phase 2b,3/23/22,"Phase 2b study demonstrated highly significant improvements in hypoglycemia and good safety and tolerability, noted March 23, 2022.",6
MOLN,Age-related macular degeneration (AMD),CRL,6/26/20,"CRL issued June 26, 2020.",5
TAK,Pouchitis,Phase 3,3/30/23,"Phase 4 met primary endpoint, noted March 30, 2023.",5
AZN,Thyroid cancer,Phase 3,7/26/18,"Phase 3 trial did not meet primary endpoint - noted July 26, 2018.",4
ZYME,"HER2-expressing Gastrointestinal Cancers (Gastroesophageal Adenocarcinoma, Biliary Tract Cancer, and Colorectal Cancer)",Phase 2,1/19/23,"Phase 2 data presented at ASCO Gastrointestinal Cancers Symposium reported an overall confirmed objective response rate (cORR) of 79%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 12.5 months, noted January 19, 2023.",6
ADAP,Esophageal and esophagogastric junction cancers,Phase 2,11/8/22,"Phase 2 data reported that ORR has increased to 37% from 33%, and the median duration of response has increased to ~20 weeks from approximately 12 weeks, noted November 8, 2022.",5
AIM,CFS,CRL,2/4/13,"CRL February 4, 2013.",5
SYBX,Cirrhotic patients with elevated ammonia,Phase 1/2,8/20/19,Development to be discontinued due to lack of efficacy.,4
NAVB,Head and Neck Cancer,Approved,6/13/14,"Approved June 13, 2014.",6
LGND,ER+/HER2- Breast Cancer,Phase 2,6/5/23,"Phase 2 data presented at ASCO reported that PFS was 56.0 wks (~13 mos), CBR was 65.5%, and ORR was 55.6%, noted June 5, 2023.",6
MLTX,Hidradenitis suppurativa,Phase 2,10/16/23,"Phase 2 24-week topline data reported that 57% of patients continuously treated with 120mg achieved a HiSCR75 response and 38% achieved HiSCR90, noted October 16, 2023.",6
ABBV,B-cell Lymphoma (DLBCL),Approved,5/22/23,"Approved May 19, 2023.",6
RHHBY,Alzheimer’s disease,Phase 3,1/30/19,Phase 3 trial terminated following interim analysis indicating primary endpoint would not be met.,4
BMY,Relapsed and/or Refractory (R/R) Multiple Myeloma (MM),Phase 1,12/10/22,"Phase 1 data is presented at ASH demonstrated deep and durable responses with a manageable safety profile across all dose levels, including patients previously treated with a B-cell maturation antigen (BCMA)-directed CAR T cell therapy, noted December 10, 2022.",6
EVFM,contraceptive vaginal gel,Approved,5/22/20,"FDA Approval announced May 22, 2020.",6
VTRS,Dry eye disease,Approved,2/3/22,"Approved February 3, 2022.",6
AMGN,"Solid tumors with MTAP deletion, NSCLC",Phase 1,3/15/22,"Phase 1 updated data reported no drug-related SAE's or observed dose limiting toxicity (DLT) through Cohort 5, noted March 15, 2022.",6
PFE,Chronic low back pain,Phase 3,2/19/19,"Phase 3 data released February 19, 2019. 10mg dose met primary endpoint. 5mg dose did not meet endpoint.",5
GSK,RSV vaccine,Approved,5/3/23,"FDA Approved on May 3, 2023.",6
MOLN,COVID-19,Phase 2a,12/6/21,"Phase 2a data reported an infectivity of virus extracted through nasopharyngeal swabs was reduced to zero within 3-5 days post treatment in patients with positive titers at baseline, noted December 6, 2021.",5
APTO,Acute myeloid leukemia (AML),Phase 1/2,10/30/23,"Phase 1/2 data demonstrated 44% ORR with TUS/VEN in Difficult-to-Treat VEN Failure R/R AML Patients, noted October 30, 2023.",6
APLT,Diabetic cardiomyopathy (DbCM),Phase 3,6/22/23,"Phase 3 baseline data to be presented at ADA showed a strong statistical correlation between elevations in the cardiac stress biomarker, NT-proBNP, reduced cardiac functional capacity and physical activity, underscoring the importance of early diagnosis via NT-proBNP and impact of DbCM on physical function and quality of life, noted June 22, 2023",6
MRK,Cervical cancer,Phase 3,7/19/23,"Phase 3 data reported that trial met the PFS primary endpoint, noted July 19, 2023.",5
AZN,Relapsed or Refractory Mantle Cell Lymphoma,Approved,10/31/17,"Priority Review announced August 2, 2017. Approval announced October 31, 2017.",6
PTCT,Mitochondrial Epilepsy,Phase 2/3,6/29/23,"Phase 2/3 data reported that trial did not meet the primary endpoint, noted June 29, 2023.",6
AXSM,Narcolepsy,Phase 2,12/3/19,Phase 2 data met primary endpoint.,6
XCUR,Merkel cell carcinoma / Cutaneous squamous cell carcinoma,Phase 1/2,8/5/21,"Phase 1b/2 interim data noted overall response rate (ORR) of 21% in all evaluable MCC patients - August 5, 2021.",5
ZVRA,Urea cycle disorder (UCD),Approved,12/23/22,"Approved December 23, 2022.",6
AEZS,Endometrial cancer,Phase 3,5/1/17,"Phase 3 trial did not meet primary endpoint - May 1, 2017.",4
HCM,Myeloid hematological malignancies (HMs),Phase 1,6/9/23,"Phase 1 data reported that the best of response was 1CR in 100 mg cohort; 9 CRs and 1 PR in 150 mg QD; 1 CR in 200 mg QD; and 3 CRs in 250 mg QD; with an ORR of 33.3% (1/3), 40.0%(10/25), 14.3% (1/7) and 42.9% (3/7), respectively, noted June 9, 2023.",5
AADI,PEComa​,Approved,11/23/21,"Approved November 23, 2021.",6
ARAV,Pancreatic Adenocarcinoma,Phase 1b,5/25/23,"Phase 1b data from ASCO reported that the median overall survival (OS) for patients with trough levels above the minimal batiraxcept efficacious concentration is greater than 15 months, and one patient demonstrated a complete response from 10 months to 20 months and is still on study, noted May 25, 2023.",6
QSAM,Bone cancer,Phase 1,8/16/23,"Initial Phase 1 data reported that treatment was well-tolerated with no serious adverse events, a 56% - 64% decrease in SUV in patient one, and that the first two patients experienced significant pain relief and improved mobility within a week after treatment, noted August 16, 2023.",6
IRWD,Irritable Bowel Syndrome with Constipation (IBS-C),Phase 3,6/18/19,"Phase 3b data met all endpoints - June 18, 2019.",6
PFE,Squamous cell carcinoma of the head and neck (SCCHN),Phase 2,4/17/23,"Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.",6
AZN,Endometrial cancer (EC),Phase 3,10/23/23,"Phase 3 results presented at ESMO reported statistically significant and clinically meaningful PFS benefit vs CP in the ITT population, with the interim OS data being immature (27.7%) yet with a trend towards benefit, noted October 21, 2023.",6
MRK,Non-transfusion-dependent beta-thalassemia,Phase 2,6/11/21,"Phase 2 data presented at EHA meeting June 11, 2021. 77.7% of patients achieved a hemoglobin increase compared to 0% in the placebo arm.",6
AMGN,Osteoporosis,Approved,4/9/19,"FDA Approval announced April 9, 2019.",6
RHHBY,Duchenne muscular dystrophy,Phase 3,10/30/23,"Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.",6
CNTA,Kidney Disease,Phase 3,12/14/21,"Phase 3 data reported that ALT elevation peaked between 1.8x and 3.5x ULN and did not return to below ULN until 23 to 140 days after tolvaptan use was discontinued. Four patients were successfully titrated to a maintenance dose of lixivaptan of either 100 mg BID or 200 mg BID, noted December 14, 2021.",6
EXEL,Solid Tumors,Phase 1/2,1/18/22,"Phase 1/2 cohort results showed that of the 36 patients enrolled in cohort 2 of the CAMILLA trial, 29 were evaluable for the efficacy analysis. The primary outcome of investigator-assessed ORR per modified RECIST version 1.1 was 27.6%. The confirmed PR rate was 20.7%, and the DCR was 86.2%. Median PFS was 3.8 months, with a 6-month PFS of 34.5%. Median OS was 9.1 months, noted January 18, 2022.",6
EFTR,Solid Tumors RTK BC and KRAS NSCLC,Phase 1/2,6/6/22,"Phase 1/2 presentation at ASCO reported 2 partial responses, noted June 6, 2022.",6
CMRX,Adenovirus Infection,Phase 3,2/22/17,"Phase 3 interim analysis released May 2016. Final data released February 22, 2017.",5
BFRI,Actinic Keratosis on face & scalp,Phase 1,8/28/23,"Phase 1 data reported that treatment was generally well tolerated and all TEAEs were transient, noted August 28, 2023.",6
DRUG,Dravet Syndrome,Phase 1/2,8/8/23,"Additional Phase 1/2 cohort 4 data reported an increase in central delta power and robust reduction in central alpha and beta power in active group, as previously reported for Anti-Epileptic Drugs (AEDs) in healthy individuals, noted August 8, 2023.",6
ELDN,Kidney transplant rejection,Phase 2,11/2/23,"Phase 2 data from 11 participants demonstrates tegoprubart successfully prevented kidney transplant rejection and was generally safe and well-tolerated, noted November 2, 2023.",6
AZN,Asthma,Phase 3,5/16/22,"Phase 3 results reported that treatment reduced the risk of a severe exacerbation by 27%, noted May 16, 2022.",6
ZNTL,Ovarian cancer,Phase 1b,6/5/23,"Phase 1b dose escalation data presented at ASCO reported that confirmed ORR of 50.0% and mPFS of 7.4 months in combination with paclitaxel, and a confirmed ORR of 35.7% and mPFS of 10.4 months in combination with carboplatin, noted June 5, 2023.",6
RVNC,Lateral Canthal (Crow’s Feet) Lines,Phase 2,6/30/20,"Phase 2 top-line data released June 30, 2020. 88% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group.",6
OPK,Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) - Stage 5,Phase 2,11/2/23,"Phase 2 data presented at ASH indicated that early, sustained and effective treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression CKD in pre-dialysis patients, noted November 2, 2023.",6
BMY,(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC),Approved,8/1/17,"Approval announced August 1, 2017.",6
ATNXQ,Angiosarcoma,Phase 2,12/9/20,"Phase 2 poster at SABCS December 9, 2020 - Complete response (CR) 43% (3/7 pts), partial response (PR) 14% (1/7 pts).",5
ACAD,Adjunctive Treatment in Patients With Major Depressive Disorder,Phase 3,7/20/20,"Phase 3 trial did not meet primary endpoint - July 20, 2020.",4
BIIB,Alzheimer’s disease,Phase 3,9/13/19,Phase 3 trial discontinued due to unfavorable risk-benefit ratio.,4
GPCR,Type 2 Diabetes,Phase 1b,9/29/23,"Phase 1b results reported that GSBR-1290 demonstrated significant weight loss supporting once-daily (QD) dosing, noted September 29, 2023.",6
MRK,Triple negative breast cancer (TNBC),Phase 3,5/20/19,"Phase 3 data May 20, 2019 did not meet primary endpoint.",4
AMGN,Wild-Type RAS Metastatic Colorectal Cancer,Approved,6/29/17,"sBLA approval announced June 29, 2017.",6
JNJ,metastatic hormone-naïve prostate cancer (mHNPC),Approved,2/8/18,"sNDA approval announced February 8, 2018.",6
PDSB,Human papillomavirus (HPV) associated cancers,Phase 2,12/28/22,"Phase 2 data reported a median OS of 21 months, with 75% remain alive at a median follow-up of 27 months, noted December 28, 2022.",5
ARQT,Plaque psoriasis in children,Approved,10/6/23,"Approved for treatment of children aged 6 - 11 on October 6, 2023.",6
INO,HPV 6 and HPV 11-associated Recurrent respiratory papillomatosis,Phase 1/2,10/10/23,"Phase 1/2 received feedback from the FDA that data could support INOVIO's submission of a BLA for review under the FDA's accelerated approval program. The FDA also advised that the company's previously planned Phase 3 trial would not be required to support this submission, noted October 10, 2023.",6
ALNY,Primary Hyperoxaluria Type 1,Phase 3,9/30/20,Clinically meaningful reduction in spot urinary oxalate:creatinine ratio.,6
IPHA,Hematological malignancies,Phase 1/2,10/16/23,"Phase 1/2 data from ESMO abstract reported that 3/8 patients achieved complete remission (CR), noted October 16, 2023.",6
MRK,Metastatic solid tumors,Phase 2,10/23/23,"Additional Phase 2 data presented at ESMO reported that 10 out of 18 evaluable NSCLC patients (56%) had achieved a partial response as their best overall response and 3 out of 6 evaluable SCCHN patients had achieved partial response as their best overall response, noted October 23, 2023.",6
CYTO,COVID-19,Phase 1,1/24/23,"Phase 1 topline data reported that trial did not meet primary endpoint, noted January 24, 2023.",6
PSTV,Leptomeningeal metastases (LM),Phase 1/2,11/3/23,"FDA granted Orphan Drug Designation (ODD), noted November 3, 2023.",5
PFE,Early stage breast cancer,Phase 3,10/9/20,"Phase 3 trial did not meet primary endpoint - October 9, 2020.",4
SYBX,Enteric Hyperoxaluria,Phase 1b,12/15/22,"Phase 1b study results and proof of concept data reported a 38% reduction in urinary oxalate compared to placebo, noted December 15, 2022.",6
BMY,Stroke prevention (SSP),Phase 2,8/28/22,"Phase 2 trial results reported that a a dose response was not observed and a relative risk reduction of approximately 30% in symptomatic ischemic stroke, noted August 28, 2022.",6
MRNS,PCDH19-related epilepsy,Phase 2,3/9/21,Phase 2 trial showed a median 61.5% reduction in seizure frequency for ganaxolone compared to 24.0% for placebo (p=0.17).,6
JANX,Metastatic castration-resistant prostate cancer (mCPRC),Phase 1,7/17/23,"Phase 1 interim clinical data showed tumor-activated T cell engagement in patients with prostate cancer representing the first-in-human data for the TRACTr platform and providing confidence for continuing clinical development of JANX007 and advancing future programs from the platform, noted July 17, 2023.",6
SUPN,Epilepsy,Approved,10/22/12,"Approved October 22, 2012.",6
ALZN,Dementia related to Alzheimer's,Phase 2a,6/22/23,"Phase 2a topline results identified a safe & appropriate dose to explore the potential to distribute more lithium to the brain but at lower systemic exposure, resulting in an improved safety profile compared to currently marketed lithium, noted June 22, 2023",6
GBT,Sickle cell disease (SCD),Phase 2/3,11/2/23,"Phase 2/3 data from ASH abstract reported that safety data showed GBT021601 was well tolerated. For 27 patients with VOC at baseline, the baseline annualized VOC rate was 2.30, noted November 2, 2023.",6
NVAX,Respiratory Syncytial Virus (RSV) in healthy pregnant women - protect infants via maternal immunization,Phase 3,2/28/19,"Phase 3 top-line data released March 28, 2019 - primary endpoint not met.",4
PFE,Amyotrophic lateral sclerosis (ALS),CRL,7/19/19,"CRL issued July 19, 2019.",5
EWTX,dystrophinopathies including Duchenne Muscular Dystrophy in children and adolescent boys,Phase 2,10/26/23,"Phase 2 initiated, noted October 26, 2023.",5
AUTL,T-cell lymphoma,Phase 1,6/16/23,"Additional data presented at the ICML showed that all 4 patients at the highest dose in the study achieved a response and 2/4 remained in a complete metabolic response beyond 12 months, noted June 16, 2024.",6
NVTA,Early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE),Phase 3,10/2/23,"Phase 3 results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients, noted October 2, 2023.",6
AMGN,Cardiovascular disease,Approved,9/24/21,"Approved for pediatric patients 10 years and older September 24, 2021.",6
RHHBY,Neoadjuvant triple negative breast cancer,Phase 3,6/18/20,"Phase 3 trial met primary endpoint - June 18, 2020.",6
PFE,Respiratory syncytial virus (RSV) vaccine,Approved,6/1/23,"Approved June 1, 2023.",6
ASMB,Hepatitis B virus (HBV),Phase 2,11/1/22,"Phase 2 preliminary data from 65 patients randomized to receive AB-729+VBR+NA (n=32), AB-729+NA (n=17) or VBR+NA (n=16) for 48 weeks showed that adding VBR to AB-729+NA does not result in greater on-treatment improvements in markers of HBV infection as compared to AB-729+NA alone, noted November 1, 2022.",6
NVS,Asthma,Phase 3,12/16/19,"Phase 3 trial did not meet primary endpoint - December 16, 2019.",4
TCRT,Solid tumors,Phase 1/2,8/14/23,"Program wind down due to prioritization. Phase 1/2 interim data reported a 83% disease control rate, noted August 14, 2023.",5
ARAV,"Growth hormone deficiency, or GHD - pediatric",Phase 3,9/21/17,"Phase 3 top-line data released September 21, 2017 - primary endpoint not met.",4
GILD,Refractory Large B-cell Lymphoma (R-LBCL),Phase 2,6/6/22,"Phase 2 data presented at ASCO reported a CR rate of 65% and ORR of 88%. With a median follow-up of 17 mo, 65% of the patients had ongoing response, with 57% ongoing in CR, noted June 6, 2022.",6
GLTO,Idiopathic pulmonary fibrosis (IPF),Phase 2b,8/15/23,"Phase 2b trial did not meet its primary endpoint, noted August 15, 2023.",6
MREO,COVID-19,Phase 1/2,12/22/21,"Phase 1b/2 data reported no safety signals were observed and 62.5% of (5/8) patients had a 2-point decrease in the WHO Disease Severity score by Day 5, compared to 28.5% (2/7) patients in the placebo arm, noted December 22, 2021.",6
BMY,Myelofibrosis,Phase 1/2,12/12/22,"Phase 1/2 results presented at ASH reported that most TRAEs were grade 1/2 in severity, while grade 3 TRAEs were successfully managed with dose delays or modifications without leading to treatment discontinuation, noted December 12, 2022.",6
INAB,Allogeneic hematopoietic stem cell transplantation (HSCT),Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that treatment emergent AE's included transient WBC and ANC decreased (100% each), platelet count decreased and anemia (88.9% each) and maculopapular rash, hypomagnesemia and blood creatine increased (55.6% each), noted November 2, 2023.",6
FDMT,Wet Age-Related Macular Degeneration (wet AMD),Phase 2,9/7/23,"Phase 2 enrollment commenced, noted September 7, 2023.",5
BHVN,Amyotrophic lateral sclerosis (ALS),Phase 3,9/29/22,"Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.",6
RHHBY,1L Extensive-Stage Small Cell Lung Cancer,Phase 3,6/7/22,"Phase 3 results noted that the combination did not provide benefit in patients with untreated ES-SCLC, noted June 7, 2022.",6
DCPH,Solid tumors,Phase 1/2,11/30/21,"Program discontinued, noted November 30, 2021.",4
LIPO,Hemorrhagic cystitis,Phase 2a,4/30/23,"Phase 2a results presented at AUA reported that treatment was tolerated without report of product related serious adverse events, noted April 30, 2023.",6
BGNE,Ovarian Cancers or Triple-Negative Breast Cancer,Phase 1/2,9/17/20,"Phase 1/2 data to be presented at ESMO September 2020. ORR 64.6% and 31.6% in cohorts 1 and 2, respectively.",5
PHGE,P. aeruginosa in patients with Cystic Fibrosis (CF),Phase 1/2,9/10/23,"Phase 1b/2 Part 1 data presented at ERS reported that treatment was well-tolerated with no adverse events related to study drug, noted September 10, 2023.",6
JNJ,Type 2 diabetes,Approved,10/30/18,"FDA Approval announced October 30, 2018.",6
ORMP,Non-alcoholic steatohepatitis (NASH),Phase 2,6/15/20,Phase 2 interim data presented at ADA 2020 Scientific Sessions - mean 6.9% reduction in liver fat content.,6
LLY,Atopic Dermatitis,CRL,11/2/23,"CRL received in the US, noted November 2, 2023.",5
ALLO,Clear cell renal cell carcinoma (ccRCC),Phase 1,4/17/23,"Phase 1 presentation at AACR reported early anti-tumor activity with deepening responses over time, noted April 17, 2023.",6
BGNE,Stage II-IIIA non-small cell lung cancer (NSCLC),Phase 3,10/23/23,"Phase 3 data presented at ESMO reported that MPR & pCR rates were significantly improved with TIS + CT vs CT, noted October 23, 2023.",6
VRCA,Common warts,Phase 2,6/26/19,"Phase 2 data noted 51% of patients noted complete clearance of warts - June 26, 2019.",5
GRTS,COVID-19 booster (60+),Phase 1,10/11/23,"Additional Phase 1 data reported at IDweek noted that durable binding and high neutralizing antibodies were induced and sustained for up to at least 12 months, noted October 11, 2023.",5
RVNC,Upper limb spasticity,Phase 2,2/22/21,"Phase 2 top-line data released February 22, 2021. One of two co-primary endpoints met (one missed due to reduced enrollment).",6
CCXI,C3 glomerulopathy (C3G),Phase 2,12/21/20,"Phase 2 trial did not meet primary endpoint - December 21, 2020.",4
AXSM,Treatment resistant depression,Phase 2,8/9/21,"Phase 2 trial met primary and key secondary endpoints - August 9, 2021.",6
YS,Rabies,Phase 3,9/26/23,"Phase 3 trial initiated, noted September 26, 2023.",5
CTIC,COVID-19,Phase 3,10/1/21,"Phase 3 trial did not demonstrate a clinical benefit, trial will no longer continue.",4
CGTX,Early Alzheimer's disease,Phase 2,10/27/23,"Phase 2 data points to a role for the sigma-2 (σ-2) receptor in regulating key ""housekeeping"" processes such as autophagy that are impaired in Alzheimer's and other neurodegenerative diseases, noted October 27, 2023.",5
INO,COVID-19 vaccine,Phase 2,5/10/21,"Phase 2 data released May 10, 2021 - generally safe and well-tolerated.",6
MRUS,Solid tumors - NRG1,Phase 1/2,10/23/23,"Phase 1/2 data update presented at ESMO showed a 37% ORR and 14.9 months median DOR in 78 evaluable NRG1+ NSCLC patients and 42% ORR and 9.1 months median DOR in 33 evaluable NRG1+ PDAC patients. Sufficient clinical data expected in 1H 2024 to support potential BLA submissions, noted October 23, 2023.",6
ROIV,Atopic dermatitis (AD),Phase 3,5/16/23,"Phase 3 data reported that the trial met its primary endpoint, noted May 16, 2023.",5
MRK,HIV-1,Phase 3,10/25/21,"Phase 3 data met safety and efficacy endpoints, noted October 25, 2021.",6
MRK,First and Second line locally advanced or metastatic urothelial cancer - bladder cancer,Approved,5/18/17,"Approved May 18, 2017.",6
BLCM,HER2+ Solid Tumors,Phase 1/2,12/6/21,"Phase 1/2 initial data reported no dose-limiting toxicities with other Grade 3+ treatment-emergent adverse events being neutropenia, leukopenia, and anemia, noted December 6, 2021.",6
MRK,Solid tumors - TMB-H ≥10 mutations/megabase,Approved,6/17/20,"FDA Approval June 17, 2020.",6
BPTH,Solid tumors,Phase 1,7/17/23,"Phase 1/1b first cohort completed, noted July 17, 2023",5
INKT,Acute respiratory distress (ARDS) secondary to COVID-19,Phase 1,5/21/23,"Phase 1 data update reported a 75% survival rate in patients with severe respiratory distress treated with agenT-797, noted May 21, 2023.",6
ORTX,Mucopolysaccharidosis type I (MPS-I),Phase 1,10/26/23,"PoC data presented at ESGCT resulted in improvement 62.5% of patients or stabilization 37.5% of patients of corneal clouding at the time of last follow-up ranging from 3.14-4.58 years compared to baseline measured prior to administration with OTL-203, noted October 26, 2023.",6
ABBV,Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).,Approved,4/21/20,"FDA Approval announced April 21, 2020.",6
ALZN,Mild to Moderate Dementia of the Alzheimer's Type,Phase 1/2,3/28/22,"Phase 1 results reported that treatment was safe and well-tolerated and salicylate exposures were within safe limits, noted March 28, 2022.",6
COGT,Gastrointestinal Stromal Tumors (GIST),Phase 3,6/5/23,"Phase 3 efficacy data presented at ASCO reported a 55% Disease Control Rate (DCR) in heavily pre-treated GIST patients, including 100% DCR and 17% overall response rate (ORR) in efficacy evaluable 2nd-line GIST patients, noted June 5, 2023.",6
AZN,Chronic rhinosinusitis with nasal polyps (CRSwNP),CRL,3/14/22,"CRL announced March 14, 2022.",5
AXSM,Major Depressive Disorder,Approved,8/19/22,"FDA Approved on August 19, 2022.",6
GSK,"Prevention of chemotherapy induced nausea and vomiting, or CINV",Approved,9/2/16,"Approved September 2, 2015.",6
PBYI,Rb-deficient head and neck squamous cell cancer,Phase 1/2,10/16/23,"Phase 1/2 data reported that the median PFS was 1.4 months, and the median overall survival (OS) was 13.5 months, noted October 16, 2023.",5
BTAI,Neuroendocrine Prostate Cancer (NEPC),Phase 2a,1/11/23,"Phase 2a data reported that combination with pembrolizumab has demonstrated an encouraging response rate, noted January 11, 2023.",6
PSTV,Glioma,Phase 1/2,6/29/23,"Phase 1/2 data presented at the Society of Nuclear Medicine & Molecular Imaging's (SNMMI) Annual Meeting demonstrated efficacy signals in a prognostically unfavorable patient population. The median overall survival (OS) in all 21 patients was 11 months or a 38% increase in OS versus a median OS of approximately 8 months for standard of care in rGBM. Median OS in patients receiving >100 Gy of absorbed radiation dose was 76 weeks versus 22 weeks for those receiving <100 Gy (p=0.0002), noted June 29, 2023.",6
RGNX,Wet AMD using suprachoroidal delivery,Phase 2,7/30/23,"Phase 2 additional data reported meaningful reductions in Anti-VEGF injection burden with stable to improved CRT through month 6, noted July 30, 2023.",6
MRK,Cytomegalovirus (CMV) Infection,Approved,11/9/17,"Approval announced November 9, 2017.",6
ACOR,Post stroke deficits,Phase 3,11/21/16,"Phase 3 data released November 21, 2016 - failed to show efficacy, development to be discontinued.",4
BGNE,Waldenström’s macroglobulinemia,Approved,9/1/21,"FDA Approval announced September 1, 2021.",6
JNJ,HIV in Women,Phase 2b,8/31/21,"Phase 2b data did not meet endpoints, study will not continue - August 31, 2021.",4
ASMB,Hepatitis B virus (HBV),Phase 2,11/12/21,"Phase 2 data reported that treatment led to a deeper level of viral suppression as measured by pgRNA and HBV DNA levels, noted November 12, 2021.",6
SNY,Dengue,Approved,5/1/19,"FDA approval announced May 1, 2019.",6
RHHBY,First-line BRAF wild-type metastatic or unresectable locally advanced melanoma,Phase 3,6/20/19,"Phase 3 data did not meet primary endpoint - June 20, 2019.",4
RLYB,Fetal and neonatal alloimmune thrombocytopenia (FNAIT),Phase 1/2,12/1/21,"Phase 1/2 data reported that treatment accelerated the elimination of HPA-1ab-positive platelets through 7 days following administration compared with placebo, noted December 1, 2021.",6
AMRX,Parkinson's disease,CRL,7/3/23,"CRL issued by the FDA on July 3, 2023.",5
INCY,Alopecia Areata,Approved,6/13/22,"Approval announced June 13, 2022.",6
ARCT,COVID-19 variant vaccine,Phase 1/2,8/18/22,"Additional Phase 1/2 data reported broad neutralizing antibody response against Omicron variants of concern, including BA.5, lasting for up to at least six months after administration of low-dose (5 mcg) ARCT-154 booster, noted August 18, 2022.",5
LNTH,Pheochromocytoma,Approved,7/30/18,"FDA approval announced July 30, 2018.",6
MYOV,Advanced prostate cancer,Approved,12/18/20,"FDA approval announced Decmber 18, 2020.",6
JNJ,Plaque psoriasis,Phase 3,9/18/22,"Phase 3b data reported that patients treated with guselkumab ≤2 years after disease onset (versus >2 years) are more likely to achieve super-respondera status, noted September 18, 2022.",6
ADPT,Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL),Approved,5/19/23,"Approved May 19, 2023.",6
PBYI,Glioblastoma (GBM),Phase 2,11/20/21,"Phase 2 results reported that in the intent-to-treat population, there was no significant improvement of PFS and OS. Median PFS with neratinib was 6.0 months versus 4.7 months and median OS was 13.8 months 14.7 months, noted November 20, 2021.",4
SLS,Malignant pleural mesothelioma (MPM),Phase 1,6/28/23,"Phase 1 topline data reported that the median overall survival for patients who entered the study as Stage IV patients was 62.3 weeks, noted June 28, 2023.",5
BDTX,Glioblastoma multiforme (GBM) and NSCLC,Phase 1,10/14/23,"Phase 1 clinical data presented at AACR-NCI-EORTC meeting reported that five of the 13 patients with either intrinsic driver, acquired resistance or complex mutations had a confirmed partial response (PR), noted October 14, 2023.",6
VERA,Kidney transplant,Phase 2,6/4/22,"Phase 2 data results showed that the antiviral effect was higher in the dosed group than the placebo group. Further, the treatment was well tolerated, with a comparable frequency of adverse events and serious adverse events between groups",6
CRNX,Acromegaly,Phase 2,6/15/23,"Phase 2 2-year data presented at ENDO 2023 showed that treatment was well-tolerated, associated with stable IGF-I and symptom control relative to that achieved by iSRLs, and was preferred over injected therapy, noted June 15, 2023.",6
MRK,"Resectable stage II, IIIA or IIIB non-small cell lung cancer (NSCLC)",Phase 3,10/20/23,"Phase 3 data presented at ESMO 23 demonstrated statistically significant improvement in overall survival versus neoadjuvant placebo plus chemotherapy, noted October 20, 2023.",6
ALGS,HBeAg-positive chronic hepatitis B (CHB),Phase 1b,6/21/23,"Additional Phase 1b data presented at EASL demonstrated multi-log reductions in HBsAg, DNA, and RNA levels in subjects dosed for up to 36 weeks, noted June 21, 2023.",6
EYPT,Non-infectious uveitis,Approved,10/15/18,"FDA approval announced October 15, 2018.",6
GSK,Meningococcal disease,Approved,10/17/22,"Approved October 17, 2022.",6
LIAN,Cholangiocarcinoma,Approved,6/1/21,"FDA approval announced June 1, 2021.",6
SGEN,Non-muscle invasive bladder cancer (NMIBC),Phase 1,6/3/23,"Phase 1 interim data shared at ASCO reported that of 4 pts receiving 125mg of intravesical EV, 3 achieved CR and continue in response, noted June 3, 2023.",6
NVO,Type 2 diabetes,Phase 3,6/3/22,"Phase 3a trial met primary endpoint, noted June 3, 2022.",5
RHHBY,Prurigo nodularis,Phase 2a,4/22/20,"Phase 2a trial met primary endpoint - April 22, 2020.",6
ASND,Solid tumors,Phase 1/2,10/26/23,"Phase 1/2 updated data presented at ESMO resulted in clinical responses demonstrated as monotherapy or in combination with checkpoint inhibitor, noted October 26, 2023.",6
AMGN,Herceptin biosimilar,Approved,6/13/19,"FDA approval announced June 13, 2019.",6
HCM,Gastric Cancer,Phase 2,11/14/22,"Phase 3 trial met primary endpoint regarding progression-free survival. The trial did not meet the the overall survival primary endpoint, noted November 14, 2022.",6
ALNY,Wild-type ATTR amyloidosis patients with cardiomyopathy,CRL,10/9/23,"CRL issued by the FDA on October 9, 2023. As a result of the CRL, the Company will no longer pursue an expanded indication for patisiran in the US.",4
CTMX,"Solid tumors / Diffuse large B-cell lymphoma (DLBCL), squamous non-small cell lung cancer (sqNSCLC), head and neck squamous cell carcinoma, and esophageal/gastro-esophageal junction (E/GEJ) cancer",Phase 2,12/20/21,"Phase 2 initial data reported an objective response rate of 18.8% and disease control rate of 87.5% in squamous non-small cell lung cancer. In advanced head and neck squamous cell carcinoma with objective response rate of 4.0 percent and disease control rate of 56.0 percent, noted December 20, 2021.",5
CYTK,Amyotrophic lateral sclerosis (ALS),Phase 3,11/21/17,"Phase 3 data released November 21, 2017 - primary endpoint not met.",4
RVNC,Cervical dystonia,Phase 3,11/9/21,"Open label data reported a median duration of effect ranging from 19.9 weeks to 26.0 weeks across doses. The most common treatment-related adverse events were muscular weakness (4.9%), dysphagia (4.2%) and injection site pain (2.7%), noted November 9, 2021.",6
IPSEY,Castration-Resistant Prostate Cancer,Phase 3,8/21/23,"Phase 3 data reported that the trial met one of two primary endpoints, demonstrating a statistically significant improvement in progression-free survival (PFS) at the primary analysis, noted August 21, 2023.",6
XERS,Hypoglycemia,Approved,8/23/21,"FDA approval August 23, 2021.",6
SNY,ER+/HER2- advanced breast cancer,Phase 3,8/17/22,"Phase 3 trial did not meet the prespecified boundary for continuation following a IDMC review, noted August 17, 2022.",6
MNKD,Pediatric type 1/2 diabetes,Phase 3,4/27/22,"Phase 3 proof-of-concept study demonstrated that a simplified 2x dose provided significant reductions in post-prandial glucose excursions (PPGE) versus the current label dosage guidelines, with no new safety concerns, noted April 27, 2022.",6
VIGL,Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP),Phase 1,12/14/22,"Phase 1 trial achieved favorable safety, tolerability and PK profiles in single ascending dose and multiple ascending dose cohorts. VGL101 achieved dose dependent, robust and durable decreases in CSF sTREM2 demonstrating proof of target engagement further validating its mechanism of action, noted December 14, 2022.",6
KNSA,COVID-19-related acute respiratory distress syndrome (ARDS),Phase 2/3,12/28/21,Phase 3 portion of the trial did not meet the primary efficacy endpoint of proportion of patients alive and free of mechanical ventilation at Day 29.,6
AZN,Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD),Approved,6/27/19,"FDA Approval announced June 27, 2019.",6
AUPH,Lupus Nephritis,Phase 3,11/3/23,"Phase 3 data from ASN reported that treated patients in the overall AURORA 2 cohort maintained stable renal function over the last two years of the study, as measured by eGFR analysis and experienced numerically greater mean reductions in urine protein creatinine ratio (UPCR), compared to patients in the control arm, noted November 3, 2023.",6
IOVA,Refractory metastatic melanoma,Phase 2,11/10/22,"Additional Phase 2 data reported that 42% of responses lasted more than 24 months, noted November 10, 2022.",5
OPTN,Chronic sinusitis,Phase 3,2/16/23,"Phase 3 trial met both co-primary endpoints, noted March 7, 2022. Phase 3 additional analysis reported that patients experienced a spectrum of benefits on symptoms and quality of life, as well improvement in objective measures of disease, relative to patients receiving a placebo comparator, noted July 8, 2022. Phase 3 pre-planned analysis of pooled data revealed 66% reduction in sinusitis exacerbations (flares) with treatment compared to placebo, noted July 13, 2022. sNDA submitted to FDA on February 16, 2023",6
VECT,Short bowel syndrome - colon-in-continuity,Phase 2,10/16/23,"Additional Phase 2 data reported that 33% of the patients achieving clinical response and PS volume reduction reached a statistically significant 40% at week 24 and the effect was maintained with a 52% volume reduction at week 52, noted October 16, 2023.",6
PRQR,Leber's Congenital Amaurosis (LCA) - Genetic blindness,Phase 2/3,4/13/22,"Phase 2/3 analysis found that benefit was observed in mean change from baseline in BCVA, noted April 13, 2022.",6
CUE,HPV+ head and neck squamous cell carcinoma (HNSCC),Phase 1b,6/5/23,"Phase 1b additional data presented at ASCO reported that among 12 evaluable RP2D combination patients to date, 5 PRs, and 2 durable SDs have been observed, with 71% (5/7) of patients expressing PD-L1 CPS ≤ 20, noted June 5, 2023.",6
EBS,Zika vaccine,Phase 1,11/19/18,"Phase 1 data released November 19, 2018 - primary endpoint met with favorable safety profile.",6
RDHL,COVID-19,Phase 2/3,2/7/22,"Phase 2/3 data reported significantly reduced mortality when given to patients who received standard-of-care (SoC), as well as a significant benefit in time to recovery, noted February 7, 2022.",6
GILD,Diffuse Large B-Cell Lymphoma (DLBCL),Phase 2,12/13/21,"Phase 2 primary analysis reported 89% of evaluable patients achieved a response (ORR), including 78% of patients with a complete response (CR) at a median follow-up of 15.9 months, noted December 13, 2021.",6
REGN,Melanoma,Phase 1,5/25/23,"Additional Phase 1 data from ASCO reported an ORR of 63% (25 of 40 patients), including 6 complete responses (CR) and 19 partial responses (PR) in the initial cohort, with a ORR of 63% in the confirmatory cohort, and 56% in the Prior neo/adjuvant systemic therapy cohort, noted May 25, 2023.",5
PULM,Acute migraine,Phase 1,7/11/23,"IND submitted to FDA to initiate Phase 2 trial, noted July 11, 2023.",5
NBIX,Tardive dyskinesia,Approved,4/11/17,"Approved April 11, 2017.",6
BPTS,Sarcopenia,Phase 2b,10/4/21,"Phase 2b data displayed that the highest dose (350 mg bid) showed a clinically meaningful improvement in the 400-Meter Walk Test (400MWT), the primary endpoint of the study, including in sub-populations at higher risk of mobility disability, after 6 months of treatment. Safety profile at the doses of 175 mg bid and of 350 mg bid showed no Serious Adverse Events (SAE) related to treatment, noted October 4, 2021.",6
IMMP,Metastatic breast cancer (MBC),Phase 2b,5/4/22,"Phase 2b biomarker data reported a statistically significant increase in innate and adaptive immune response biomarkers and six patient subgroups with improvements in Overall Survival (OS), noted May 4, 2022.",6
VCEL,Severe burns,CRL,6/29/21,"CRL announced June 29, 2021.",5
VIRX,Nasopharyngeal carcinoma and Epstein-Barr Virus-Positive (EBV+) Solid Tumors,Phase 1/2,12/9/22,"Preliminary Phase 1b/2 safety data reported no DLTS with 2/6 patients achieving stable disease, noted November 30, 2022.",6
GRTX,Pancreatic Cancer,Phase 2b,10/31/23,"Phase 2b did not pass futility analysis, so trial halted to preserve cash, noted October 31, 2023.",6
RCKT,Arrhythmogenic Cardiomyopathy,Phase 1,6/8/23,https://www.biopharmcatalyst.com/company/RCKT/news/140814,5
NVO,Hemophilia A and B,CRL,5/4/23,"CRL received noted May 4, 2023.",5
TAK,Crohn's Disease,Phase 3,7/22/19,"Phase 3 trial met primary endpoint - July 22, 2020.",6
ALLK,Eosinophilic esophagitis,Phase 2/3,12/21/21,"Phase 2/3 top-line data reported that the trial met its histologic co-primary endpoints, but did not achieve statistical significance on the patient reported symptomatic co-primary endpoints, noted December 21, 2021.",6
HOTH,Atopic Dermatitis (AD),Phase 1b,9/6/23,"Phase 1b final results reported that 78% of patients reported that their overall health had improved since starting BioLexa as compared to 22% stating that there was no change in their health, or their health was worse, noted September 6, 2023.",6
PGEN,"Myelodysplastic Syndromes, Acute Myeloid Leukemia",Phase 1b,12/13/22,"Phase 1/1b data reported a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients, noted December 13, 2022.",6
FGEN,Duchenne Muscular Dystrophy,Phase 3,8/29/23,"Phase 3 topline data reported that the trial did not meet its primary endpoint, noted August 29, 2023.",6
PBYI,Extended adjuvant HER2-positive early stage breast cancer,Approved,7/17/17,"Approval announced July 17, 2017.",6
BLRX,Pancreatic cancer,Phase 2,12/16/20,"Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.",5
STOK,Dravet Syndrome,Phase 1/2,7/25/23,"Phase 1/2a new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population, noted July 25, 2024",6
INCY,Graft versus host disease,Approved,5/24/19,"FDA approval announced May 24, 2019.",6
NVS,Spinal muscular atrophy (SMA) Type 2,Phase 3,8/12/22,"Phase 3 data reported two patient deaths, noted August 12, 2022",5
KPRX,Retinitis Pigmentosa (RP),Phase 1,5/1/23,"Phase 1 abstract presented at ASNR23 on May 1, 2023",6
SNDX,Relapsed/refractory (R/R) acute leukemias,Phase 2,10/2/23,"Phase 2 trial met its primary endpoint at the protocol-defined interim analysis stage with a complete remission (CR) or a CR with partial hematological recovery (CRh) rate of 23% among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort, noted October 2, 2023.",6
GILD,Refractory diffuse large B-cell lymphoma (DLBCL),Approved,4/1/22,"Approved April 1, 2022.",6
KDNY,IgA nephropathy,Phase 1/2,6/12/23,"Phase 1/2 interim results demonstrated rapid and sustained reductions in mechanistic biomarkers, including IgA and Gd-IgA1 levels, which correspond to clinically meaningful proteinuria reductions in patients with IgAN across Cohorts 1 and 2 due, noted June 12, 2023.",6
ENTA,Hepatitis C virus (HCV),Approved,8/3/17,"Approval announced August 3, 2017.",6
DBTX,Cisplatin-induced hearing loss,Phase 1b,6/1/23,"Phase 1b data reported that ear pain (15/19 subjects, 78.9%) and tinnitus (8/19 subjects, 42.1%) were common and the overall study objectives were met at this interim analysis, noted June 1, 2023.",5
IVA,Psoriasis,Phase 2b,10/31/22,"Development stopped noted October 31, 2022.",4
EIGR,Pulmonary arterial hypertension (PAH),Phase 2,1/16/18,"Phase 2 trial did not meet endpoints - January 16, 2018.",4
ASLN,Biliary tract cancer,Phase 2/3,11/11/19,"Phase 3 trial did not meet primary endpoint - November 11, 2019.",4
TNXP,Post-traumatic stress disorder (PTSD),Phase 3,7/27/18,"Phase 3 trial halted due to insufficient efficacy - July 27, 2018.",4
JAZZ,Spasticity due multiple sclerosis,Phase 2,2/7/17,"Phase 2 trial did not meet primary endpoint - noted February 7, 2017.",4
GILD,Higher risk-Myelodysplastic Syndrome (MDS),Phase 3,7/21/23,"Phase 3 ENHANCE study been discontinued due to futility based on a planned analysis, noted July 21, 2023.",4
ISEE,Wet age-related macular degeneration (Wet-AMD),Phase 3,8/14/17,"Phase 3 data released December 12, 2016 - primary endpoint not met. Data from third trial released August 14, 2017- primary endpoint not met.",4
ARQT,Atopic Dermatitis - ages of 2-5 years old,Phase 3,9/19/23,"Phase 3 top-line data reported that the trial met all its primary and secondary endpoints, noted September 19, 2023.",5
ESPR,Hypercholesterolemia,Approved,2/21/20,"FDA Approval announced February 21, 2020.",6
RLMD,Major Depressive Disorder (MDD),Phase 3,10/13/22,"Phase 3 trial did not meet its primary endpoint, noted October 13, 2022.",6
NVS,Diffuse large B-cell lymphoma (DLBCL) - 2nd line,Phase 3,8/23/21,"Phase 3 trial did not meet primary endpoint - August 23, 2021.",4
ADCT,Diffuse Large B-Cell (DLBCL) or Mantle Cell Lymphoma (MCL),Phase 2,12/13/21,"Phase 2 interim data reported an ORR of 57.1% in the overall DLBCL cohort, and 45.5% and 76.9% in the non-GCB DLBCL and GCB DLBCL cohorts, respectively. Median duration of response in the overall DLBCL cohort was 5.49 months and was not reached in the non-GCB DLBCL or GCB DLBCL cohorts, noted December 13, 2021.",6
ATNF,Dupuytren’s disease (DD),Phase 2/3,12/1/21,"Phase 2b data from 2b/3 trial reported that trial met its primary and secondary endpoint with no related severe adverse events, noted December 1, 2021.",6
FDMT,Fabry Disease,Phase 1/2,10/30/23,"Phase 1/2 FDA meeting to lift clinical hold and continue trial developments, noted October 30, 2023.",6
MESO,Heart failure,Phase 3,12/6/21,Phase 3 results showed greatest treatment benefit in major adverse cardiovascular events (MACE) of cardiovascular mortality or irreversible morbidity (non-fatal heart attack or stroke) in patients with diabetes and/or myocardial ischemia (72% of total treated population),6
BMY,Ulcerative colitis (UC),Phase 2,10/26/23,"Phase 2 trial was unsuccessful, noted October 26, 2023.",5
KPTI,Multiple myeloma,Approved,12/18/20,"FDA approval announced December 18, 2020.",6
MRNA,Heart Failure,Phase 2a,11/15/21,"Phase 2a study met the primary endpoint of safety and tolerability; numerical trends observed in endpoints in the heart failure efficacy domains compared with placebo, including increase in LVEF and patient reported outcomes, noted November 15, 2021.",6
NBIX,Congenital Adrenal Hyperplasia (CAH) - adults,Phase 3,9/12/23,"Phase 3 study met its primary endpoint at Week 24, Noted September 12, 2023",5
MNOV,Non-alcoholic fatty liver disease (NAFLD) with Type 2 Diabetes Mellitus and Hypertriglyceridemia,Phase 2,12/7/22,"Additional Phase 2 data reported that T2DM group showed a greater reduction in serum triglyceride levels at Week 8 and Mean HDL increase was significantly greater in subjects with T2DM than subjects without T2DM at Week 8, noted December 7, 2022.",6
CFRX,Gram-negative infections,Phase 1a,10/16/23,"IND safety review completed by FDA on October 16, 2023.",5
MGTA,Multiple Myeloma,Phase 2,12/14/21,"Phase 2 investigator-initiated clinical trial data confirmed previously reported positive topline clinical data where it achieved the primary endpoint for collection of hematopoietic stem cells (HSCs), was well-tolerated and mobilized HSCs which were able to successfully engraft with positive 100-day outcomes.",6
LLY,Type 1 Diabetes,CRL,3/20/20,"CRL announced March 20, 2020.",5
OBSV,Uterine fibroids,Phase 3,10/20/21,"Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.",5
WINT,Cardiogenic Shock in severe acute heart failure,Phase 2,9/26/22,"Phase 2 dose response data demonstrated that study met its primary endpoint of significantly improved systolic blood pressure over the first 6 hours of study drug infusion. The study also met several other secondary endpoint assessments of cardiac function, noted on September 26, 2022.",6
ATOS,Mammographic breast density (MBD),Phase 2,6/27/19,"Phase 2 data met primary endpoint - June 27, 2019. High dropout rate noted.",6
JAZZ,Fibromyalgia,CRL,10/11/10,"CRL received October 11, 2010.",5
GSK,Pediatric Lupus Nephritis,Approved,7/27/22,"Approved July 27, 2022.",6
BIIB,Alzheimer’s disease,Phase 3,9/13/19,Phase 3 trial discontinued due to unfavorable risk-benefit ratio.,4
GRTX,Non-Small Cell Lung Cancer (NSCLC),Phase 1/2,10/31/23,"Phase 1/2 halted after GRECO-2 failed futility analysis, so trial halted to preserve cash, noted October 31, 2023.",6
CGEM,Acute myeloid leukemia (AML),Phase 1,6/8/23,Preliminary safety data from an ongoing first-in-human study were published in abstract form as part of the 2023 EHA Congress in June 2023.,5
MRK,Head and neck squamous cell carcinoma (HNSCC),Phase 2,11/9/21,"Phase 2 clinical data showed that patients with 1L HNSCC who have not received prior treatment for their advanced disease (n=13), the dose demonstrates an initial ORR of 38.5% with a 12-month OS rate of 87.5% and mOS not reached in combination with pembrolizumab + 5FU + platinum. These results compare favorably with benchmark survival data from standard pembrolizumab + chemotherapy, noted November 9, 2021.",6
CANF,Psoriasis,Phase 3,6/29/23,"Phase 3 top-line data reported that trial met primary endpoint, noted June 29, 2022. Additionally in their Phase 3 the FDA encouraged the Company to enroll adolescent patients due to the strong safety profile of the drug demonstrated over the development history and prior clinical studies, noted June 29, 2023",6
ARWR,Healthy volunteers and Asthma,Phase 1/2,9/11/23,"Phase 1/2 preliminary clinical data achieved mean target gene knockdown of up to 90% with a maximum of 95% after a single inhaled administration, noted Septemeber 11, 2023.",6
IDYA,"Solid tumors, ovarian cancer",Phase 1,9/11/23,"Phase 1 data reported tumor shrinkage observed in multiple HRD solid tumor patients, including an endometrial cancer subject with a first imaging assessment of a partial response and an 87% reduction of the CA-125 tumor marker, noted September 11, 2023.",6
LLY,Moderate-to-severe atopic dermatitis (AD),CRL,10/2/23,"CRL issued October 2, 2023.",5
ADCT,Diffuse Large B-Cell Lymphoma,Approved,4/23/21,"FDA approval announced April 23, 2021.",6
HOWL,Solid Tumors,Phase 1b,11/3/23,"Phase 1/1b preliminary data provided compelling early evidence of dose-dependent biomarker and antitumor activity in patients, including two patients with ongoing unconfirmed partial responses (uPR) in the highest dose tested to date, cohort 4 (12 mg), noted November 3, 2023.",5
IMGO,Essential Thrombocythemia,Phase 2,12/12/22,"Phase 2 data reported that 100% of patients treated with bomedemstat for ≥24 weeks achieved a platelet count in the normal range and 89% (25/28) achieved a durable (≥12 weeks) platelet count, noted December 12, 2022.",6
RHHBY,COVID-19 (Anti-viral antibody),Phase 2/3,9/30/21,"Phase 3 trial met primary endpoint, with 36% reduced risk of death by day 29, and 56% reduced risk in patients who were seronegative. Of 2,007 patients (REGEN-COV=1,340, placebo=667) serious adverse events occurred in 21% REGEN-COV patients and 26% placebo patients, noted September 30, 2021.",6
FBIO,Postoperative pain following bunionectomy surgery,CRL,2/15/22,"FDA AdCom Meeting resulted in a 8-14 vote against the question that the application showed that benefits outweighs the risks, noted February 15, 2022.",6
RCUS,Triple negative breast cancer (TNBC) and ovarian cancer,Phase 1,12/9/20,"Phase 1 initial data due presented at SABCS Annual Meeting, December 9, 2020. 2/18 response rate (partial responses - PR) for doublet regimen. 5/12 responses for triplet (1 complete response, 4 had a PR).",5
RGNX,Diabetic retinopathy,Phase 2,8/1/23,"Phase 2 additional data reported that 20% of patients achieved a >2-step improvement vs. 10% in control and 54% of patients achieved any DRSS improvement vs. 20% in control, noted August 1, 2023.",6
EFTR,relapsed/refractory Acute Myeloid Leukemia (AML),Phase 1,10/24/23,"Phase 1 initiated, noted October 24, 2023.",5
ETON,Seizures/migraine,Approved,11/8/21,"FDA Approval November 8, 2021.",6
MRTX,Non-Small Cell Lung Cancer / Colorectal Cancer,Phase 1b,9/10/23,"Phase 1/1b two-year follow-up data presented at the World Conference on Lung Cancer reported a durable efficacy with a median overall survival (OS) of 14.1 months and a 2-year OS rate of 31% in patients with previously treated KRASG12C-mutated NSCLC, noted September 10, 2023.",6
MDWD,Venous leg ulcers (VLU’s),Phase 2,5/12/22,"Phase 2 results reported that both primary and key secondary endpoints were met, noted May 12, 2022.",5
PFE,"CD20-positive, low tumor burden, follicular lymphoma.",Approved,7/23/19,"FDA approval announced July 23, 2019.",6
NCNA,Pancreatic cancer,Phase 3,8/20/19,"Phase 3 enrolment suspended due to futility - August 20, 2019.",4
RHHBY,Huntington's Disease,Phase 3,3/22/21,Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.,4
APRE,Solid tumors,Phase 1/2,10/16/23,"Phase 1/2a data presented at AACR-NCI-EORTC reported that no dose-limiting toxicities have been reported to date with persistent tumor-reducing effect, noted October 16, 2023.",6
TAK,Frontline Hodgkin lymphoma,Approved,4/4/18,"sBLA approval announced March 20, 2018.",6
ITCI,Bipolar depression,Phase 3,7/8/19,"Phase 3 trial met primary endpoint - July 8, 2019.",6
AZN,Amyotrophic lateral sclerosis (ALS),Phase 3,8/20/21,Phase 3 trial discontinued due to lack of efficacy.,4
IPHA,Non-small cell lung cancer (NSCLC),Phase 2,9/17/21,"Phase 2 trial met primary endpoint of confirmed ORR of 30%, and for durvalumab plus monalizumab 36%. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone, September 17, 2021.",6
AMGN,Acute lymphoblastic leukemia (ALL) - pediatric,Phase 3,9/24/19,Phase 3 enrolment terminated due to treatment benefit over chemo. Primary endpoint of event-free survival met.,6
ABBV,Alzheimer’s disease,Phase 2,9/7/23,"Phase 2 enrollment to be completed, noted September 7, 2023.",5
GILD,Urothelial Cancer,Approved,4/13/21,"FDA approval announced April 13, 2021.",6
CGEM,Non-Small Cell Lung Cancer,Phase 1/2,12/16/21,"Updated Phase 1/2a reported that 14 patients achieved a confirmed PR for a 39% confirmed response rate and one additional patient had a PR that was pending confirmation at the time of the data cut-off. The median duration of response was >15 months and the median progression free survival was 12 months in the initial cohort of Phase 1 patients, noted December 16, 2021.",6
ABBV,Age-related macular degeneration (AMD),CRL,6/26/20,"CRL issued June 26, 2020.",5
NVS,"Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis",Approved,3/21/23,"Approved March 21, 2023.",6
REGN,Homozygous familial hypercholesterolemia (HoFH) in children,Approved,3/22/23,"Approved March 22, 2023.",6
INCY,Non-Small-Cell Lung Cancer (NSCLC),Phase 2,5/19/21,Phase 2 data in ASCO abstract noted ORR of 65.6% for the treatment-naive expansion Cohort 7.,6
LLY,Cancer pain,Phase 3,7/28/21,Phase 3 trial met primary endpoint - July 2021.,6
MRNA,Ovarian cancer,Phase 2,8/5/21,"Development has been discontinued - noted August 5, 2021.",4
ANNX,Geographic atrophy,Phase 2,7/30/23,"Phase 2 results presented at ASRS demonstrated dose and time-dependent protection of visual function in GA across multiple measures, noted July 30, 2023.",6
SWTX,Desmoid tumors,Phase 3,9/10/22,"Phase 3 data presented at ESMO resulted in rapid, sustained and statistically significant Improvements in primary and all Key secondary efficacy endpoints, noted September 10, 2022.",6
BPTH,Acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS),Phase 2,8/1/23,"Phase 2 interim analysis reported that in cohort 1, twelve of the fourteen evaluable patients (86%) achieved complete remission (CR/CRi) and two (14%) achieved partial remission (PR), and in cohort 2 eight of the fourteen evaluable patients (57%) achieved complete remission (CR/CRi), two (14%) achieved partial remission (PR) and three (22%) achieved stable disease, noted August 1, 2023.",6
PCRX,Postoperative Pain Management,Phase 3,9/21/21,"Phase 3 study found the PK profile was comparable across age groups and generally consistent with the profile in adult patients. No safety concerns were identified at a dose of 4 mg/kg, noted September 21, 2021.",6
TNGX,Solid Tumors,Phase 1a,9/6/23,"IND cleared by FDA, noted September 6, 2023. Phase 1/2 clinical trial to commence 1H 2024.",5
ETON,"Lennox-Gastaut syndrome, epilepsy",CRL,5/27/22,"CRL announced May 27, 2022.",5
GILD,Adult patients with r/r ALL - cancer,Approved,10/1/21,"Approved October 1, 2021.",6
CALC,Wet age-related macular degeneration,Phase 2b,11/15/21,"Additional Phase 2b data reported that the anatomical control was similar to aflibercept over 18 months with BCVA maintained in extension study, noted November 15, 2021.",5
BPMC,Advanced Systemic mastocytosis (SM),Approved,6/16/21,"FDA approval announced June 16, 2021.",6
VYNE,Vitiligo,Phase 1b,10/30/23,"Phase 1b clinical proof-of-concept achieved with statistically significant dose dependent reduction in F-VASI score from baseline for the VYN201 1.0% and 2.0% cohorts compared to the 0.5% cohort, noted October 30, 2023.",6
KTRA,Glioblastoma (GBM),Phase 2/3,10/31/23,"Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.",5
AKRO,Nonalcoholic steatohepatitis (NASH),Phase 2b,10/10/23,"Phase 2b top line data showed that a trend was observed for the primary endpoint of fibrosis improvement at 36 weeks, with 22% and 24% of the 28mg and 50mg EFX-treated groups, respectively, compared with 14% for placebo, noted October 10, 2023.",6
ALEC,Alzheimer’s disease,Phase 1,11/10/21,"Phase 1 data demonstrated target engagement of CD33 in both blood and central nervous system (CNS) compartments at well tolerated doses in healthy volunteers, noted November 10, 2021.",6
AERI,Open Angle Glaucoma,Phase 3,9/24/20,"Phase 3 top-line data released September 24, 2020. Non-inferiority to Ganfort achieved.",6
GILD,Nonalcoholic steatohepatitis (NASH),Phase 2,12/16/19,"Phase 2 trial did not meet primary endpoint - December 16, 2019.",4
CGTX,Alzheimer's Disease,Phase 1/2,3/28/23,"Additional biomarker data revealed 28 biomarkers that were significantly altered as a result of treatment with CT1812, 11 of which are priority biomarkers of Alzheimer's biology, noted March 28, 2023.",6
ALLO,Non-Hodgkin lymphoma (NHL),Phase 1,6/15/23,"Phase 1 data update presented at ICML and ASCO 23 reported that 7 of 12 (58%) patients achieved a CR and five (42%) maintained a CR through Month 6. Of the five patients who were in CR at 6 months, four (80%) remained in CR, noted June 15, 2023.",6
AGEN,Cervical cancer,BLA Filing,10/22/21,"BLA voluntarily withdrawn October 22, 2021.",4
CLSD,Uveal melanoma including choroidal melanoma,Phase 2,2/16/23,"Phase 2 safety and efficacy data presented with an average of nine months of follow-up for patients treated with three cycles of therapy showed response to the therapy with 89-100% tumor control, noted February 16, 2023.",6
IGXT,Acute migraine,Approved,4/17/23,"Approved April 17, 2023.",6
AQST,Allergic reactions (anaphylaxis),Phase 2,2/24/23,"Phase 2 pharmacokinetic and pharmacodynamic data due presented at AAAAI reported rapid absorption following sublingual administration with the fastest observed median Tmax (12 min forAQST-109, 23 min for EpiPen, and 45 min for IM injection), noted February 24, 2023.",6
AVEO,Hepatocellular carcinoma (HCC),Phase 1/2,1/20/22,"Phase 1b/2 data reported a 27.8% partial response (PR) rate and disease control rate (PR + stable disease) of 67.8%, with a median PFS of 7.3 months and a 1-year OS of 76%, noted January 20, 2022.",5
NKTX,Acute myeloid leukemia / myelodysplastic syndromes,Phase 1,6/27/23,"Additional Phase 1 data demonstrated that 4 of 6 patients achieved complete response (67% CR/CRi, 50% CR rate), and 2 CRs with MRD negativity 1 patient deepened response to MRD negative CRi with additional cycles, notedJune 27, 2023.",6
MRK,Ebola,Approved,8/3/23,"Approval in children aged 12 months and older on August 3, 2023.",6
JAGX,Cancer related diarrhea (CRD),Phase 2,12/10/21,"Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.",6
ACRS,Rheumatoid arthritis (RA),Phase 2a,1/19/21,"Phase 2a 12-week data released January 19, 2021 - generally well tolerated.",6
EOLS,Aesthetics,Phase 2,1/28/23,"Phase 2 interim data presented at the 2023 IMCAS World Congress on indicated that the ""extra-strength"" formulation at 40U (units) achieved a duration profile of 6 months or 26 weeks, noted January 28, 2023.",6
SELB,Tophaceous gout,Phase 2,9/30/20,"Phase 2 top-line data released September 30, 2020. Numerically higher response rate on the primary endpoint during months 3 and 6 combined, but did not meet the primary endpoint of statistical superiority.",4
INDV,Bulimia Nervosa,Phase 2,2/21/19,"Phase 2 data released February 21, 2019. Endpoints not met.",4
BLRX,Pancreatic ductal adenocarcinoma (PDAC),Phase 2,7/17/23,"Phase 2b trial initiated, noted July 17, 2023.",5
AZN,COVID-19,Phase 3,11/18/21,Phase 3 separate treatment trial showed an 88% reduced risk of severe COVID-19 or death when treated within three days of symptom onset.,6
ATAI,Generalized Anxiety Disorder (GAD),Phase 1,4/28/23,"Pharmacodynamic data indicated potential for anxiolytic activity without the significant sedative side effects associated with benzodiazepines, noted April 28, 2023.",5
ORIC,Tumors - EGFR/HER2 Exon 20 Inhibitor,Phase 1b,10/23/23,"Phase 1b initial data presented at ESMO reported responses observed in 2 of 3 EGFR exon 20 patients previously treated with amivantamab (67% ORR), including a confirmed complete response, note October 23, 2023.",6
ANIP,Menopause symptoms,Approved,9/12/23,"Approved September 12, 2023.",6
LGND,Chronic obstructive pulmonary disease (COPD),Phase 3,12/20/22,"ENHANCE-1 Phase 3 topline data met its primary and key secondary endpoints, noted December 20, 2022.",5
GMAB,Non-Hodgkin Lymphoma (NHL),Phase 1/2,12/14/21,"Phase 1/2 data reported that 25% of patients reported Grade 4 Neutropenia, with a 73% CMR and a 27% PMR, noted December 14, 2021.",5
PFE,HIV for adolescents 12 years and older,Approved,3/29/22,"Approved March 29, 2022.",6
HRTX,Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV),Approved,8/10/16,"CRL Mar 28 2013. Approved August 10, 2016.",6
VTRS,Multiple sclerosis - Tecfidera generic,Approved,8/17/20,"FDA approved August 17, 2020.",6
OPK,Obesity and diabetes,Phase 2b,3/21/19,"Phase 2b top-line data March 21, 2019 met primary endpoint.",6
ONCT,Metastatic castration-resistant prostate cancer (mCRPC),Phase 1/2,8/3/23,"Phase 1/2 study received ""Study May Proceed"" letter by FDA, noted August 3, 2023.",5
PFE,Pneumococcal Disease,Approved,4/27/23,"Approved on April 27, 2023.",6
CTMX,Solid tumors,Phase 2,3/30/20,Phase 2 trial has been terminated.,4
CYT,"Solid tumors, Ovarian cancer",Phase 1,10/12/23,"Additional Phase 1 data reported that two patients had a confirmed partial response, one additional patient achieved an unconfirmed partial response, seven patients had stable disease and one patient had progressive disease, noted October 12, 2023.",6
SNY,Non-small cell lung cancer (NSCLC),Phase 3,9/19/21,"Phase 3 data presented at ESMO September 19, 2021 showed trial met primary and all secondary endpoints, with an objective response rate of 43%.",6
AGEN,"Solid tumors, melanoma",Phase 1,6/3/23,"Phase 1 data at ASCO reported an overall response rate (ORR) was 11% (n=2); 37% had SD (n=7) resulting in a disease control rate (DCR) (CR, PR or SD) of 47%, noted June 3, 2023.",5
ANAB,Generalized pustular psoriasis,Phase 3,10/9/23,"Phase 3 top-line data met its primary endpoint in the study population achieving rapid clearance of pustulation, erythema and scaling through Week 4 after a single dose of 750mg IV imsidolimab. Top-line data also demonstrate a favorable safety and tolerability profile, noted October 9, 2023.",6
ANAB,Acne,Phase 2,3/14/22,"Phase 2 data reported that the trial did not meet the primary or secondary endpoints, noted March 14, 2022.",6
BPMC,RET-fusion non-small cell lung cancer (NSCLC),Approved,9/4/20,"FDA Approval announced September 4, 2020.",6
AZN,Second-line ovarian cancer,Approved,8/17/17,"Approval announced August 17, 2017.",6
PULM,Allergic bronchopulmonary aspergillosis in patients with asthma.,Phase 1,7/17/18,"Phase 1 Part 3 data released July 17, 2018 - safe and well tolerated",6
CPIX,Pain and fever in patients three months to six months of age,Approved,5/15/23,"Expanded label approval by FDA on May 15, 2023.",6
MRK,Neurofibromatosis type 1 plexiform neurofibromas,Approved,4/13/20,"FDA Approval announced April 13, 2020.",6
NBIX,Endometriosis,Approved,7/24/18,"FDA approval announced July 24, 2018.",6
SNGX,Cutaneous T-Cell Lymphoma (CTCL) cancer,Phase 3,5/4/23,"Phase 3 data results confirm and extend response results, noted May 4, 2023.",5
BMY,Non-small cell lung cancer (NSCLC),Phase 3,10/23/23,"Phase 3 data presented at ESMO reported that 3-y EFS and OS rates were 72% vs 47% and 85% vs 66%, respectively, noted October 23, 2023.",6
BHVN,Spinocerebellar Ataxia (SCA),Phase 3,7/27/23,"The FDA informed Biohaven that it would not review the recently submitted NDA application for troriluzole given that the study's primary endpoint was not met and thus, would not permit a substantive review. The communication from the FDA indicated that the Company may request a Type A meeting within 30 days, noted July 27, 2023.",6
TCDA,Chronic kidney disease,CRL,8/24/20,"CRL announced August 24, 2020.",5
MNPR,Oral mucositis,Phase 2/3,3/27/23,"Phase 2b/3 interim analysis conducted by an independent Data Safety Monitoring Board (DSMB), which informed the Company that the trial did not meet the pre-defined threshold for efficacy of a 15% absolute difference in SOM prevention between Validive and placebo. Based on not meeting the pre-specified efficacy threshold, the company will be discontinuing the study, noted March 27, 2023.",6
TGTX,Chronic Lymphocytic Leukemia,Phase 1/2,9/20/21,"Phase 1/2 data demonstrated that 47 patients have now been treated as of the data cutoff with 57% of patients previously exposed to a BTK inhibitor, best Overall Response Rate (ORR) was 100% amongst evaluable patients (n=46), including 37% complete response (CR) rate presented at iwCLL September 20, 2021.",6
ARDS,Cystic Fibrosis,Phase 2a,3/13/23,"Phase 2a trial met primary and secondary endpoints, noted March 13, 2023.",5
NVO,Obesity,Phase 2,6/18/20,"Phase 2 primary endpoint met - June 18, 2020.",6
LGND,Chronic obstructive pulmonary disease (COPD),Phase 2,10/13/21,"Phase 2 part B data noted when administered by hand-held pMDI over one week treatment provided clinically meaningful, statistically significant and dose-dependent bronchodilation, noted October 13, 2021.",6
IONS,Amyotrophic lateral sclerosis (SOD1-ALS),Approved,4/25/23,"FDA granted accelerated approval on April 25, 2023.",6
EIGR,COVID-19,Phase 3,3/17/22,"Phase 3 top-line data reported a probability of superiority of 99.91%, with a 60% reduced risk of COVID-19-related death, noted March 17, 2022.",6
TPST,"Hepatocellular Carcinoma (HCC), Solid tumors",Phase 1/2,4/17/23,"Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.",6
REGN,Eosinophilic esophagitis (EoE) in 1-11 year olds,Phase 3,7/14/22,"Phase 3 data reported that trial met its primary endpoint, noted July 14, 2022.",5
MRK,Pancreatic cancer,Approved,12/30/19,"FDA Approval announced December 30, 2019.",6
XFOR,Waldenström macroglobulinemia,Phase 1b,12/13/21,"Phase 1b data of 16 patients enrolled demonstrated that 14 were evaluable as of the data cut, all at low- (200 mg) and mid-level (400 mg) dosing; median duration of treatment was 272.5 days (range 33-435 days); 12 patients remain on study. CXCR4 antagonism proof of concept established, with 100% overall response rate.",6
LLY,Crohn's disease,Phase 3,10/12/23,"Phase 3 trial met the co-primary and all major secondary endpoints, noted October 12, 2023.",5
FATE,B-cell Leukemias and Lymphomas,Phase 1,12/10/22,"Phase 1 interim data reported that 1 of 2 patients naïve to CAR T-cell therapy achieved an objective response (1 CR) at Day 30, and 2 of 6 patients previously treated with CAR T-cell therapy achieved an objective response (1 CR, 1 PR) at Day 30, noted December 12, 2022.",6
AMGN,Immune Thrombocytopenia (Pediatric),Approved,12/14/18,"FDA approval announced December 14, 2018.",6
ERAS,Glioblastoma Multiforme,Phase 1,6/22/23,"DA granted orphan drug designation (ODD), noted June 22, 2023.",5
LUMO,Pediatric Growth Hormone Deficiency (PGHD),Phase 2,6/21/23,"Phase 2 interim data shared at KOL demonstrate a durable response to LUM-201 from 6 to 12 months, noted June 21, 2023.",6
LPTX,Gastric / Gastroesophageal Junction Cancer,Phase 2,6/5/23,"Phase 2 data shared at ASCO reported that the median overall survival (OS) was 19.5 months and median progression-free survival (PFS) was 11.3 months, noted June 5, 2023.",5
INVA,Beta thalassemia patients suffering from iron overload,Phase 3,11/25/19,"Development to be discontinued due to lack of efficacy - November 25, 2019.",4
MNKKQ,Hepatorenal syndrome (HRS) Type 1,CRL,2/22/22,"CRL reissued February 22, 2022.",5
HRMY,Adult Epilepsy Patients with Refractory Focal Seizures,Phase 2,8/7/17,"Phase 2 data released August 7, 2017 - endpoints not met.",4
MYOV,Endometriosis-associated pain,Approved,8/5/22,"Approved August 5, 2022.",6
VTGN,Major depressive disorder (MDD),Phase 1,6/21/23,"Phase 1 study results showed that dosing was well-tolerated and demonstrated a favorable safety and tolerability profile across single and multiple dose intranasal administrations, noted June 21, 2023.",6
DTIL,Relapsed or refractory B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma,Phase 1/2,6/8/22,"Phase 1/2 clinical update reported an 100% ORR, 73% CR rate and 50% durable response rate greater than six months, noted June 8, 2022.",5
BMY,Symptomatic Obstructive Hypertrophic Cardiomyopathy (HCM),Approved,6/16/23,"FDA Approval on June 16, 2023",6
PFE,Amyotrophic lateral sclerosis (ALS),Phase 3,9/29/22,"Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.",6
BNOX,Post-Traumatic Stress Disorder (PTSD),Phase 2b,9/28/23,"Phase 2b topline data met its primary endpoint showing BNC210 treatment led to a statistically significant reduction in total PTSD symptom severity at 12 weeks. Statistically significant secondary endpoints showed improvements in depressive symptoms and sleep, noted September 28, 2023.",6
TAK,Eosinophilic esophagitis,CRL,12/21/21,"CRL December 21, 2021.",5
ARGX,Pemphigus foliaceous and vulgaris (PV),Phase 2,6/20/18,"Phase 2 interim data released June 20, 2018, disease control observed in 4/6 patients.",6
GILD,Hepatitis B virus (HBV) infection in pediatric patients 12 years of age and older,Approved,11/2/22,"Approved November 2, 2022.",6
JNJ,Waldenström’s Macroglobulinemia,Approved,8/27/18,"FDA approval announced August 27, 2018.",6
HALO,Multiple Myeloma,Approved,12/1/21,"Approved December 1, 2021.",6
PFE,Non-metastatic high risk hormone-sensitive prostate cancer,Approved,7/13/18,"FDA approval announced July 13, 2018.",6
AMTI,Pouchitis,Phase 2,3/3/23,"Additional Phase 3 data demonstrated oral's gut-restricted profile with tissue-level pharmacodynamics (PD) effects and no systemic exposure by design. Translational analysis reveals IL-10 biological responses in 3mg and 10mg dose arms, noted March 3, 2023.",6
ARQT,Plaque psoriasis,Phase 3,9/30/21,"Phase 3 data met its primary endpoint of Investigator Global Assessment Success rate at week 8 in 37.5% compared to a vehicle rate of 6.9%, noted September 30, 2021.",6
VYGR,Parkinson's Disease,Phase 2,12/22/20,"Phase 2 trial placed on clinical hold - December 22, 2020.",4
LLY,Obesity,Phase 3,6/24/23,"Phase 3 efficacy and safety data shared at ADA met both co-primary endpoints and all key secondary endpoints compared to placebo for both estimands, with those taking tirzepatide achieving a mean weight reduction of 13.4% on 10 mg and 15.7% on 15 mg compared to 3.3% on placebo for the efficacy estimand, noted June 24, 2023.",6
JNJ,COVID-19 vaccine (single dose),Phase 3,9/21/21,"Phase 3 data showed 75 percent overall efficacy against severe/critical COVID-19, across all ages and countries. 74 percent efficacy in the U.S. against severe/critical COVID-19; 89 percent against hospitalization; 83 percent against COVID-19-related death, September 21, 2021.",6
ANEB,Cannabinoid Intoxication,Phase 2a,3/28/23,"Phase 2 met the primary endpoint in all cochorts, noted March 28, 2023",5
NRXP,COVID-19,Phase 3,5/25/22,"Phase 3 DSMB review requested that randomization to aviptadil be stopped due to not meeting the futility guidelines, noted May 25, 2022.",6
TAK,Chronic Myeloid Leukemia,Approved,12/18/20,"FDA approval announced December 18, 2020.",6
MDWD,Severe burns,CRL,6/29/21,"CRL announced June 29, 2021.",5
ACIU,Alzheimer's disease-like characteristics in individuals with Down syndrome,Phase 1/2,1/26/23,"Phase 1b/2 trial early results from the first cohort showed that low dose could elicit an anti-Abeta antibody response as soon as week 6 (2 weeks after the second injection). The data show that ACI-24.060 vaccination has been safe and well tolerated to date, noted January 26, 2023",6
VRTX,Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation,Approved,7/2/15,"Approved July 2, 2015.",6
AYLA,Adenoid cystic carcinoma,Phase 2,6/6/22,"Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.",6
NBTX,Colorectal cancer,Phase 1/2,1/15/21,More than 70% of patients in the study showed objective tumor response after CCRT.,6
CDTX,Influenza,Phase 2a,6/22/23,"FDA has granted Fast Track designation (FTD), noted June 22, 2023.",5
BMY,First-line Small cell lung cancer (SCLC),Phase 3,11/26/18,"Phase 3 trial did not meet primary endpoint - November 26, 2018.",4
HRTX,Total knee arthroplasty,Phase 2b,6/21/18,"Phase 2 data released June 21, 2018. Primary endpoint met.",6
EXEL,Solid Tumors,Phase 1b,9/10/22,"Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.",6
NKTR,"Urothelial carcinoma, Melanoma, Renal Cell Carcinoma and Non-Small Cell Lung Cancers",Phase 1/2,11/9/19,"Phase 1/2 updated data at SITC November 9, 2019 noted ORR was 53% (20/38) with 34% (13/38) complete response (CR) rate.",5
LLY,Treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC),Approved,9/21/22,"FDA Approval on September 21, 2022.",6
ITRM,Complicated intra-abdominal infections (cIAI),Phase 3,12/10/19,"Phase 3 data missed primary endpoint - December 10, 2019.",4
GLPG,Osteoarthritis,Phase 2,10/15/20,"Phase 2 trial did not meet primary endpoint - October 15, 2020.",4
BIIB,Epilepsy,Phase 2,4/22/20,Phase 2 primary endpoint not met.,4
AZN,Chronic obstructive pulmonary disease (COPD),Phase 3,5/11/18,"Phase 3 trial did not meet primary endpoint - noted May 11, 2018.",4
UBX,Diabetic macular edema / Age-related macular degeneration,Phase 2a,4/24/23,"Phase 2 48-week data demonstrated that a single injection of UBX1325 treatment led to a statistically significant improvement in vision lasting for the duration of the study (48 weeks), marked by a gain of +6.2 ETDRS letters from baseline, representing a difference of +5.6 ETDRS letters compared to sham-treated patients, noted April 24, 2023.",6
MGTX,Xerostomia,Phase 1,10/24/23,"Phase 1 OLE data presented at ESGCT showed that dosing was safe and well-tolerated at all dose levels, with no treatment-related serious adverse events or dose limiting toxicity, noted October 24, 2023.",6
IONS,End-stage renal disease (ESRD),Phase 2b,11/4/22,"Additional Phase 2b data reported that treatment also achieved dose-dependent and sustained median reductions in steady-state FXI levels of 53.1%, 72.2% and 86.6% in the 40 mg, 80 mg, and 120 mg doses of fesomersen, respectively, administered once every 4 weeks, noted November 4, 2022.",6
IBRX,BCG-Unresponsive Bladder Cancer Carcinoma,BLA Filing,10/23/23,"BLA Resubmitted to the FDA, noted October 23, 2023.",5
RHHBY,Ulcerative colitis,Phase 3,8/10/20,"HIBISCUS I induction study, in people without prior anti-tumor necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint. HIBISCUS II induction study, including people without prior anti-TNF treatment, did not meet its primary endpoint.",5
TPTX,RET-altered NSCLC,Phase 1/2,4/5/21,"Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.",5
GSK,"Respiratory syncytial virus (RSV) disease, Vaccine",Phase 3,10/25/23,"Phase 3 trial met its co-endpoint, eliciting an immune response, noted October 25, 2023.",5
NVS,Gastroenteropancreatic neuroendocrine tumors (GEP-NETs),Phase 3,9/25/23,"Phase 3 trial met primary endpoint of improvement in progression-free survival (PFS) and key secondary endpoint of objective response rate (ORR), noted September 25, 2023.",6
EIGR,Post-Bariatric Hypoglycemia (PBH),Phase 2,10/16/18,"Phase 2 data released October 16, 2018 - endpoints met.",6
REGN,Wet age-related macular degeneration (AMD),Phase 2,8/10/23,"Phase 2 two-year longer-term data demonstrated that the vast majority of aflibercept 8 mg patients with wAMD could maintain or further extend their dosing intervals. Among those who completed the two-year follow-up, noted August 10, 2023.",6
HCM,Neuroendocrine Tumors,Phase 3,1/20/20,Phase 3 China trial stopped early. Primary endpoint met.,6
ATRA,Multiple sclerosis,Phase 2,10/26/22,"Phase 2 OLE data reported that all with stable EDSS remaining in the OLE continued to have a stable EDSS for a median of 41.2 months, noted October 26, 2022.",5
GILD,Relapsed or Refractory Mantle Cell Lymphoma (r/r MCL) - cancer,Approved,7/24/20,"FDA approval announced July 24, 2020.",6
LCTX,Dry age-related macular degeneration (AMD),Phase 1/2,5/2/22,"Phase 1/2a full results presented at the Association for Research in Vision and Ophthalmology on May 2, 2022. All 24 treated patients reported at least one adverse event (AE) and at least one ocular AE. The majority of AEs reported with OpRegen were mild (Cohort 1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was well tolerated. Preliminary evidence of improvement in visual function was observed in patients with GA and impaired vision at baseline (Cohort 4 [n=12]) Patients in Cohort 4 had an average 7.6 letter gain in visual acuity at 12 months in the study eye Three patients in Cohort 4 (25%) had a 15 letter or greater gain in visual acuity at 12 months in the study eye.",6
HRMY,Osteoarthritis,Phase 2,8/14/17,"Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.",4
REGN,Rheumatoid arthritis,Approved,5/22/17,"CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.",6
GILD,HIV,Approved,10/18/21,"Approved for pediatric patients October 18, 2021.",6
BLTE,Dry Age-Related Macular Degeneration (AMD),Phase 2/3,4/25/23,"Phase 2 data reported that safe and well tolerated in adolescent Stargardt Disease (STGD1), noted April 25, 2023.",6
AZN,Solid tumors,Phase 1,12/9/22,"Phase 1 data reported that IPH5201 blocks ATP degradation and blocks CD39-mediated degradation of eATP released after chemotherapy treatment, noted December 8, 2022.",6
MRK,Endometrial cancer,Approved,9/17/19,"FDA Approval announced September 17, 2019.",6
GSK,COVID-19 vaccine,Phase 2/3,9/22/21,"Phase 2/3 interim analysis showed primary and secondary endpoints met. 100% efficacy against severe COVID-19 & hospitalization and 84% efficacy against moderate-to-severe COVID-19 caused by any strain of SARS-CoV-2 in SPECTRA, and 79% efficacy against COVID-19 of any severity caused by the globally dominant Delta variant, noted September 22, 2021.",6
SCPH,Heart failure,CRL,12/7/20,"CRL announced December 7, 2020.",5
IONS,Hereditary Angioedema (HAE),Phase 2,2/21/23,"Phase 2 open-label study data reinforce the treatment's potential to be a best-in-class prophylactic treatment for patients living with hereditary angioedema, noted February 21, 2023.",6
ITCI,Bipolar Depression and Major depressive disorder (MDD),Phase 3,9/11/23,"Phase 3 data continues to demonstrate statical significance. Lumateperone 42mg also met the key secondary endpoint by demonstrating a statistically significant and clinically meaningful reduction in the Clinician's Global Impression scale or CGI compared to placebo at Week 6 in these three populations, noted September 11, 2023.",6
UBX,Osteoarthritis of the knee,Phase 2,8/17/20,"Phase 2 top-line data failed to meet primary endpoint - August 17, 2020.",4
BMY,Mantle cell lymphoma (MCL),Phase 1,6/17/23,"Phase 1 data presented at ICML showed that 86.5% of patients in the relapsed or refractory mantle cell lymphoma cohort achieved a response, with 74.3% achieving a complete response, noted June 17, 2023.",6
MRK,Pulmonary arterial hypertension (PAH),Phase 3,9/11/23,"Phase 3 interim results from OLE study represent longest safety and efficacy analysis of sotatercept to date; safety profile of sotatercept consistent with previous studies and efficacy improvements maintained after one year of therapy, noted September 11, 2023.",6
REGN,Asthma - 6-11 year-olds,Approved,10/20/21,"Approved October 20, 2021.",6
ZLAB,Non-small-cell lung cancer (NSCLC) and solid tumors,Phase 1/2,4/12/22,"Phase 1/2 top-line data reported that in the ROS1-positive TKI-naïve advanced NSCLC population the cORR was 79%, with 4 patients receiving a complete response, and 52 having a partial response. With DOR ranging from 1.4+ to 35.1+ months and PFS ranging from 0+ to 40.4+ months, noted April 12, 2022.",5
TERN,Obesity,Phase 1a,6/26/23,"Preclinical data demonstrated the ability of TERN-601 to significantly improve glucose tolerance, suppress food intake and slow gastric emptying in transgenic mice, noted June 26, 2023",6
AZN,Paroxysmal nocturnal hemoglobinuria (PNH),Phase 3,9/16/22,"Phase 3 trial met primary endpoint at interim analysis, noted September 16, 2022.",5
COLL,Opioid dependence,Approved,6/6/14,"Approved June 6, 2014.",6
AZN,Chronic lymphocytic leukaemia,Approved,11/21/19,"FDA Approval announced November 21, 2019.",6
BCAB,Solid Tumors,Phase 2,1/10/23,"Phase 2 part 1 interim results in PD-1 failure NSCLC continues to show strong antitumor activity in a highly refractory population with additional patients enrolled, noted January 10, 2023.",6
ACRS,Healthy volunteers,Phase 1,9/18/23,"Phase 1 data reported that treatment was was generally well tolerated at all doses, noted September 18, 2023.",6
AGEN,Soft Tissue Sarcoma,Phase 2,9/17/21,"Phase 2 data showed in 125 patients, the objective response rate (ORR) was 26%, with 9% of patients achieving a complete response (CR), and 17% of patients achieving a partial response (PR), noted September 17, 2021.",5
GMAB,Non-Hodgkin lymphoma (NHL),Phase 1/2,6/9/23,"Additional data shared at EHA reported an overall response rate (ORR) of 98 percent and complete metabolic response (CMR) of 87 percent in response evaluable patients, noted June 9, 2023.",6
ALBO,Progressive familial intrahepatic cholestasis (PFIC),Approved,7/20/21,"FDA approval announced July 20, 2021.",6
NVO,Hemophilia A,Phase 1/2,7/11/22,"Phase 1/2 trial met primary endpoint, noted July 11, 2022.",5
CRBP,Dermatomyositis,Phase 3,6/24/21,"Phase 3 trial did not meet primary endpoint - June 24, 2021.",4
GNPX,Non-small cell lung cancer (NSCLC),Phase 1,6/3/23,"Phase 1 data shared at ASCO reported that treatment was well tolerated, and there were no dose limiting toxicities, noted June 3, 2023.",6
TAK,Acute Graft-Versus-Host Disease (aGvHD) in patients allo-HSCT,Phase 3,2/18/23,"Phase 3 study met primary endpoint in lower gastrointestinal (GI) aGvHD-free survival by Day 180 after allo-HSCT with no relevant differences in safety profile versus placebo, noted February 18, 2023.",5
VBIV,Hepatitis B,Phase 2,9/6/23,"Phase 2a/2b top-line data showed increased HBsAg loss rate at the end of treatment and 12 weeks follow up, noted September 6, 2023.",6
LGVN,Hypoplastic Left Heart Syndrome,Phase 1b,5/9/23,"Phase 1b long term data reported that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B, noted May 9, 2023.",5
RHHBY,Spinal Muscular Atrophy (SMA) in babies,Approved,5/31/22,"Approved May 31, 2022.",6
GSK,"Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients",CRL,10/26/17,"CRL January 11 2017. FDA Approval announced October 25, 2017 following resubmission.",6
ADXS,Adenoid cystic carcinoma,Phase 2,6/6/22,"Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.",6
ICPT,FXR Effect on Severe Alcohol-Associated Hepatitis,Phase 1,11/8/22,"Phase 1 data reported that treatment was generally well tolerated in healthy subjects. No serious adverse events were reported, noted November 8, 2022.",6
MGTX,RPE65-Deficiency,Phase 1/2,5/14/19,Phase 1/2 data met primary endpoint of safety and tolerability.,6
NGM,Solid Tumors,Phase 1/2,12/8/22,"Phase 1/2 data to be presented at the ESMO IO reported that treatment was well tolerated and six patients had reduced target lesion size including a maximum decrease in one patient of 70%, noted December 8, 2022.",6
FATE,Acute graft-versus-host disease (GvHD) undergoing hematopoietic cell transplantation (HCT),Phase 2,5/5/21,Phase 2 trial did not meet primary endpoint.,4
VRTX,Cystic fibrosis (CF) in patients aged 12 to <24 months,Approved,9/2/22,"Approved September 2, 2022.",6
INDV,Opioid Overdose,Approved,5/23/23,"Approved May 22, 2023.",6
CLVS,Ovarian Cancer - First-line maintenance treatment,Phase 3,6/6/22,"Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.",6
PFE,Persistent Pulmonary Hypertension (PPHN),Phase 3,6/28/19,"Phase 3 trial did not meet primary endpoint - June 28, 2019.",4
CALT,Alport Syndrome,Phase 2,9/27/23,"Phase 2 to be intiated in 4Q 2023. FDA granted Orphan Drug Designation, noted September 27, 2023.",5
KZIA,Brain Metastases,Phase 1,7/6/23,"FDA awarded Fast Track Designation, noted July 6, 20223.",5
PGEN,Type 1 diabetes,Phase 1/2,10/1/21,"Phase 1b interim data showed HbA1c was below the 7% target for 100% of adult patients (9) and 92% of the patients aged 17+ (11). A stabilization of IDAA1c below 9 was demonstrated in 78% of adult patients (7) and in 75% of patients aged 17+ (9). Phase 2a interim data showed stabilization of HbA1c below the ADA target of 7% was demonstrated in 70% of adult patients (7). HbA1c levels were below target in 75% of the adolescent patients (3). Stabilization of IDAA1c was demonstrated in 100% of adult patients (10) and in 75% of adolescent patients (3), noted October 1, 2021.",6
BPMC,Non-advanced systemic mastocytosis (SM),Approved,5/22/23,"FDA approval on May 22, 2023.",6
BTAI,Opioid Withdrawal Symptoms,Phase 1/2,3/31/21,"Phase 1b/2 top-line data released March 31, 2021. Primary endpoint met. Retention secondary endpoint not met.",5
HALO,Multiple sclerosis (MS) or primary progressive MS,Phase 3,10/11/23,"Phase 3 data reported that treatment was comparable to IV infusion in providing rapid and sustained depletion of B cells and near-complete suppression of MRI lesion activity in the brain over 24 weeks, noted October 11, 2023.",6
ALNA,Enteric Hyperoxaluria,Phase 3,3/18/22,"Phase 3 interim analysis due late 1Q 2022. Based on the results by an independent DSMB has recommended that the trial size be increased from the maximum allowed number of 400 subjects under the pre-specified rules. However, even with this maximum recommended sample size increase, the power to detect an effect vs. placebo would still be less than 80% based on the available data. Based upon this recommendation, the company has decided to terminate the study and plans to promptly initiate the process of closing the study.",4
JAZZ,Excessive sleepiness associated with narcolepsy,Approved,3/20/19,"FDA Approval announced March 20, 2019.",6
OTLK,Wet age-related macular degeneration (wet AMD),BLA Filing,11/2/23,"The FDA informed the company that an additional adequate and well-controlled clinical trial would be required for the approval of ONS-5010 for wet AMD. Resubmission of the ONS-5010 BLA as early year end 2024, noted November 2, 2023.",6
CLLS,Clear cell renal cell carcinoma (ccRCC),Phase 1,4/17/23,"Phase 1 presentation at AACR reported early anti-tumor activity with deepening responses over time, noted April 17, 2023.",6
BGNE,Follicular Non-Hodgkin Lymphoma,Phase 2,6/9/23,"Phase 2 data presented at EHA reported that the OS rate at 24 months was 77.3%(zanubrutinib plus obinutuzumab) and 71.4% (obinutuzumab), with median OS not reached (zanubrutinib plusobinutuzumab) and 34.6 months, noted June 9, 2023.",6
MRK,Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC),Phase 3,7/24/17,"Phase 3 trial did not meet primary endpoint - July 24, 2017. Awarded accelerated approval in 2016.",4
CYDY,Non-alcoholic steatohepatitis (NASH),Phase 2,1/5/22,"Phase 2 trial met primary and secondary endpoint, noted January 5, 2022.",5
RHHBY,HER2-positive early breast cancer,Approved,6/29/20,"FDA Approval announced June 29, 2020.",6
RPTX,Solid tumors,Phase 1/2,4/18/23,"Phase 1/2 initial data reported a 48% CBR in patients with unmet medical needs and a 32% overall response, 58% CBR and mPFS of approximately 7 months, noted April 18, 2023.",5
CRDF,Pancreatic ductal adenocarcinoma (PDAC),Phase 2,9/26/23,"Phase 2 additional data reported an objective response rate (ORR) of 19% and median progression-free survival (mPFS) of 5.0 months, noted September 26, 2023.",5
ALKS,Solid tumors,Phase 1/2,6/1/22,"Phase 1/2 monotherapy data reported that six patients achieved a partial response (PR), of which three were confirmed, with stable disease (SD) observed in 31 patients. Combination data reported two patients achieved a CR, four patients achieved a PR and SD was observed in 14 patients, noted June 1, 2022.",6
SGEN,Untreated locally advanced or metastatic urothelial cancer (la/mUC),Phase 3,10/23/23,"Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.",6
ORMP,Type 2 Diabetes,Phase 3,1/12/23,https://www.biopharmcatalyst.com/company/ORMP/news/110598,5
BIIB,Multiple sclerosis (MS),Phase 2,10/21/20,Phase 2b trial did not meet primary endpoint.,4
BMY,Relapsed/refractory multiple myeloma,Approved,11/6/18,"FDA approval announced November 6, 2018.",6
ATHX,Ischemic stroke,Phase 3,10/10/23,"Phase 3 trial was insufficiently powered to achieve the primary endpoint, noted October 10, 2023.",6
SYRS,Solid tumors,Phase 1b,5/25/23,"Phase 1/1b data from ASCO reported that in solid tumors, data demonstrated a 100% disease control rate (DCR) with 10 mg SY-5609 monotherapy, with the PDAC patient experiencing a 10% tumor reduction, noted May 25, 2023.",6
CYCN,Heart failure HFpEF,Phase 2,10/30/19,"Phase 2 trial did not meet primary endpoint - October 30, 2019.",4
CLVS,FAP-positive tumors,Phase 1/2,10/17/22,"Phase 1 update at EANM noted that of 5 patients 45.5% had a Grade >3 treatment related adverse event, noted October 17, 2022.",6
ASRT,Hot flashes,CRL,5/31/13,"CRL issued May 31, 2013.",5
TBPH,Neurogenic orthostatic hypotension (nOH),Phase 3,8/28/23,"Additional subgroup data reported benefits of ampreloxetine ranged from 0.5 to 2.2 point improvements relative to placebo across all subgroup categories and were demonstrated on the OHSA and OHQ composite scores, noted August 28, 2023.",6
MACK,Cancer - Heregulin Positive Non-Small Cell Lung Cancer,Phase 2,10/19/18,"Phase 2 trial discontinued due to futility - noted October 19, 2018.",4
GSK,Non-small Cell Lung Cancer,Phase 3,1/20/21,Phase 3 trial unlikely to meet primary endpoint - to be discontinued.,4
GRTX,Esophagitis,Phase 2,10/27/22,"Phase 2 Open-Label data reported that 14 of 29 evaluable patients (48.3%) experienced acute esophagitis at week 6, noted October 27, 2022.",6
NVS,Non-radiographic axial spondyloarthritis,Approved,6/16/20,"FDA Approval announced June 16, 2020.",6
IMUX,Primary Sclerosing Cholangitis,Phase 2,2/18/21,"Phase 2 data released February 18, 2021. Primary objective of therapeutic benefit achieved in 27.3% of patients.",5
PBYI,HER2 positive breast cancer with brain metastases,Phase 2,12/8/22,"Phase 2 data reported an Objective Response Rate by RANO-BM was 33.3% of patients in cohort 4A, 29.4% in cohort 4B, and 28.6% in cohort 4C, noted December 8, 2022.",5
PTCT,Phenylketonuria (PKU),Phase 3,5/17/23,"Phase 3 results met primary endpoint, noted May 17, 2023.",5
MDGL,Non-alcoholic steatohepatitis (NASH) with liver fibrosis,PDUFA priority review,9/13/23,https://www.biopharmcatalyst.com/company/MDGL/news/158576,5
HCM,Neuroendocrine carcinoma,Phase 2,9/29/21,"Updated Phase 2 data reported median OS was 10.3 months. PFS was 4.14 months. The confirmed ORR was 23.8% and DCR was 71.4%. All patients experienced TRAEs, including 9 (42.9%) who experienced Grade 3 or above TRAEs, noted September 29, 2021.",5
ASND,Achondroplasia,Phase 2,11/14/22,"Phase 2 top-line data reported that trial met primary endpoint, noted November 14, 2022.",5
BGNE,Relapsed or refractory mantle cell lymphoma (MCL),Approved,11/14/19,"FDA Approval announced November 14, 2019.",6
MRK,Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC),Phase 3,8/25/23,"Phase 3 data reported that at the second analysis, the trial did not demonstrate an improvement in OS compared to KEYTRUDA plus placebo, noted August 25, 2023.",6
TAK,Cytomegalovirus (CMV),Approved,11/23/21,"Approved November 23, 2021.",6
KPTI,Relapsed/Refractory Acute Myeloid Leukemia (AML),Phase 2,3/2/17,"Data Safety Monitoring Board (DSMB) noted March 2, 2017 that trial will not meet primary endpoint.",4
BGNE,Hepatocellular Carcinoma (HCC),Phase 2,10/21/23,"Phase 2 poster presented at ESMO reported that NV-assessed ORR was 35.5% with O+T+B vs. 37.5% with T+B, noted October 21, 2023.",6
DTIL,Non-Hodgkin Lymphoma (NHL),Phase 1,5/31/23,"Phase 1 update reported a 71% ORR and 43% CR rate, noted May 31, 2023.",5
CLRB,Various lymphoma,Phase 2,6/28/23,"Phase 2 data from MM cohort reported an overall response rate (ORR) of 32%, a clinical benefit rate (CBR) of 75% and a disease control rate (DCR) of 85.7%, noted June 28, 2023.",5
AZN,Metastatic non-squamous non-small cell lung cancer (NSCLC),Phase 2,9/9/23,"Phase 2 data presented at WCLC reported that objective response rates of 49% and 56% with 5.4 mg/kg and 6.4 mg/kg doses, respectively, in primary analysis, noted September 9, 2023.",6
AMTI,Ulcerative colitis,Phase 2,7/6/22,"Phase 2 top-line data reported a remission rate of 31.8% in the treatment group, versus 33.3% in the placebo group, noted July 6, 2022.",6
REGN,Non-small cell lung cancer (NSCLC),Phase 3,9/19/21,"Phase 3 data presented at ESMO September 19, 2021 showed trial met primary and all secondary endpoints, with an objective response rate of 43%.",6
NVS,Cancer - Diffuse Large B-Cell Lymphoma (DLBCL),Approved,5/1/18,"Approval announced May 1, 2018.",6
APLS,COVID-19 / Acute respiratory distress syndrome (ARDS),Phase 1/2,3/4/21,Phase 1/2 trial to be discontinued following Independent data monitoring committee (DMC) review which found no meaningful reduction in the overall mortality rate.,4
RARE,Glut1 DS patients with the movement disorder phenotype,Phase 3,10/26/18,"Phase 3 data released October 26, 2018 - endpoints not met.",4
CHRS,"Wet age-related macular degeneration (wAMD), among others",Approved,8/2/22,"Biosimilar approved August 2, 2022.",6
MRK,Head and Neck Squamous Cell Carcinoma,Phase 2,11/9/21,"Phase 2 clinical data showed that patients with ≥2L who have not received a prior checkpoint inhibitor (""CPI"") (n=10), long-term follow-up data shows that dosing demonstrates a 12-month OS rate of 80% with a mOS of 24.5 months, which compares favorably with standard pembrolizumab therapy in patients with 2L CPI, noted November 9, 2021.",6
CRMD,Hemodialysis patients with central venous catheters,PDUFA,6/21/23,"PDUFA date after resubmission on November 15, 2023.",5
AZN,Multiple HER2-expressing advanced solid tumors,Phase 2,10/23/23,"Phase 2 data presented at ESMO 23 continued to demonstrate clinically meaningful and durable responses with a a median progression-free survival of 6.9 months and median overall survival of 13.4 months in the overall trial population, noted October 23, 2023.",6
FIXX,Phenylketonuria (PKU),Phase 1/2,11/6/20,"Data released November 6, 2020 noted 2/6 showed meaningful reductions in Phe.",6
AFMD,"T-Cell, Cutaneous Lymphoma",Phase 1/2,12/7/20,Phase 1/2 data presented at ASH 2020. Objective Response Rate (ORR) of 42%(6/14).,5
AZN,Chronic Kidney Disease,Approved,4/30/21,"FDA approval announced April 30, 2021.",6
LLY,Pediatric Patients with Moderate to Severe Plaque Psoriasis,Approved,3/30/20,"FDA Approval announced March 30, 2020.",6
ABBV,Platysma prominence associated with platysma muscle activity (M21-310),Phase 3,9/18/23,"Phase 3 trial met all primary and secondary endpoints, noted September 18, 2023.",5
EIGR,Chronic hepatitis delta virus (HDV),Phase 3,6/29/23,"Phase 3 data presented at EASL showed that at week 48 the trial met its primary endpoint in both lonafarnib arms vs placebo, also 24-week post-treatment data demonstrated that both lonafarnib arms showed a statistically significant difference in composite response rate compared to placebo, noted June 24, 2023.",6