ticker,disease,stage,date,catalyst,label
RLAY,Cholangiocarcinoma (CCA),Phase 1,10/12/23,"Phase 1 data from non-CCA expansion cohort presented at AACR-NCI-EORTC reported a 35% overall response rate (ORR), and nine of 26 patients experienced a partial response (PR), noted October 12, 2023.",6
GSK,Rheumatoid Arthritis,Phase 2b,10/21/18,"Phase 2b released October 21, 2018 did not meet primary endpoint.",4
ABBV,Episodic Migraine,Phase 3,4/21/23,"Phase 3 trial met its primary and secondary endpoints, noted April 21, 2023.",5
CRSP,Advanced clear cell renal cell carcinoma,Phase 1,11/10/22,"Phase 1 result reported a 77% disease control rate in a heavily pretreated RCC patient population. The longest duration of stable disease achieved was observed for 7.8 months, noted November 10, 2022.",6
RHHBY,Follicular lymphoma,Phase 3,6/10/22,"Phase 3 final analysis reported that PFS was significantly improved with Gazyva plus chemotherapy (63.4%) compared with Rituxan plus chemotherapy (55.7%), noted June 10, 2022.",6
KMDA,COVID-19,Phase 1/2,3/31/21,"Phase 1/2 top-line data released March 31, 2020. 11/12 patients recovered.",5
MRKR,Pancreatic cancer,Phase 1/2,7/19/19,Phase 1/2 data at AACR noted 5/9 patients with stable disease with one complete response.,5
AGRX,Contraceptive patch,Approved,2/14/20,"FDA Approval announced February 14, 2020.",6
REGN,Retinopathy of Prematurity (ROP),Approved,2/8/23,"FDA approval on February 8, 2023.",6
AMRX,Attention Deficit Hyperactivity Disorder (ADHD),Approved,9/6/23,"ANDA approved September 6, 2023.",6
INCY,Rheumatoid arthritis,Approved,6/1/18,"FDA approval announced for low dose only - June 1, 2018.",6
ERYP,Triple-Negative Breast Cancer (TNBC),Phase 2,9/19/22,"Phase 2 data reported that no clinical benefit was demonstrated, noted September 19, 2022.",6
ANAB,Non-squamous non-small cell lung cancer (NSCLC),Phase 2,10/5/22,"Phase 2 trial met primary endpoint, noted October 5, 2022.",5
FGEN,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
NCNA,Colorectal Cancer,Phase 1/2,10/13/23,"Phase 1b/2 achieved a longer progression-free survival as compared to their first-line treatment with 5-FU-based Therapy, noted October 13, 2023.",6
ANIP,Tranxene generic,Approved,6/22/22,"Approved June 22, 2022.",6
XFOR,Severe Congenital Neutropenia (SCN),Phase 2,8/10/23,"Phase 2 data reported that three participants receiving G-CSF and once-daily oral mavorixafor showing robust increases in ANC, maintenance of ANC levels in the normal range, and the ability to reduce G-CSF dose earlier than anticipated, noted August 10, 2023.",6
CBAY,Primary biliary cholangitis (PBC),Phase 3,9/21/23,"Phase 3/4 study initiated, noted September 21, 2023.",5
EBS,Anthrax Vaccine,Approved,5/17/12,"Approved May 17, 2012.",6
DERM,"Microbial flora, papulopustular rosacea",Phase 1,6/13/23,"Phase 1 study achieved all primary objectives and no significant safety issues were noted during the study, noted June 13, 2023.",6
ACLX,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
BGNE,Nasopharyngeal cancer (RM-NPC),Phase 3,4/19/22,"Additional Phase 3 data reported that progression-free survival was 15.5 months, noted April 19, 2022.",5
APLT,Sorbitol Dehydrogenase Deficiency (SORD Deficiency),Phase 2,5/25/23,"Phase 2 EMA granted Orphan medicinal product designation, noted May 25, 2023.",5
RHHBY,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
REGN,Atopic dermatitis 12-17 year-olds,Approved,3/11/19,"FDA approval announced March 11, 2019.",6
AZN,"Chronic Lymphocytic Leukemia (CLL), small lymphocytic lymphoma (SLL) and refractory mantle cell lymphoma (MCL)",Approved,8/5/22,"Approved August 5, 2022.",6
RHHBY,Multiple sclerosis (MS),Phase 2,10/13/23,"Phase 2 showed that fenebrutinib crosses the blood-brain barrier with the potential to act directly on the chronic inflammation related to multiple sclerosis (MS). More than 90% relative reduction in new/enlarging T2 lesions and new T1 gadolinium-enhancing (Gd+) lesions with fenebrutinib beginning at eight week, noted October 13, 2023.",6
MRK,Cutaneous squamous cell carcinoma (cSCC),Approved,7/6/21,"FDA approval announced July 6, 2021.",6
FBIO,AL amyloidosis,Phase 1/2,12/11/17,Phase 1/2 data at ASH December showed 67% response rate.,6
RHHBY,Duchenne Muscular Dystrophy,Approved,6/22/23,"FDA acelerated Approval on June 22, 2023.",6
XFOR,Renal Cell Carcinoma (RCC),Phase 2a,9/30/19,Phase 2a data presented at ESMO 2019 - TKI therapy PFS 7.4 months; ORR 18%.,5
CGEN,Platinum resistant ovarian cancer,Phase 1/2,6/5/23,"Phase 1/2 data presented at ASCO reported an objective response rate (ORR) of 2/9 [22%] pts; 2 pts with SD. Disease control rate [CR + PR + SD] 4/9 [44%], noted June 5, 2023.",6
PDSB,Cervical cancer,Phase 2a,10/2/23,"Additional Phase 2a data to be reported at the American Society for Radiation Oncology (ASTRO 2023) demonstrated that patients treated with PDS0101 had greater clearance of cfDNA as compared to those treated with SOC, noted October 2, 2023.",6
NVO,haemophilia B,Approved,5/31/17,"Approval announced May 31, 2017.",6
IKNA,"Solid tumors and urothelial carcinoma, bladder cancer",Phase 1,11/10/22,"Phase 1 data presented at SITC showed durable antitumor activity with a 20% ORR and 40% DCR in heavily pretreated urothelial carcinoma. Monotherapy activity observed; confirmed partial response with DoR of 14.9 months and ongoing, noted November 10, 2022.",6
AZN,Amyloidosis,Phase 2,12/12/22,"Phase 2 1-year study results reported that treatment has been generally well tolerated without evidence of organ toxicity. Organ response persisted even after cessation of anti-PCD treatment, noted December 12, 2022.",6
LXRX,Heart failure,Approved,5/26/23,"Approved May 26, 2023.",6
MMM,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
CYBN,Major Depressive Disorder (MDD) and Alcohol use disorder (AUD),Phase 1/2,10/31/23,"Phase 1/2a topline data reported that the reduction in major depressive disorder (""MDD"") symptoms, defined as change from baseline in MADRS total score, was superior in participants assigned to CYB003 compared to the participants who received placebo by 14.08 points, noted October 31, 2023.",6
JNJ,Second-line Chronic graft-versus-host disease (GVHD),Approved,8/2/17,"Approval announced August 2, 2017.",6
IVA,Non-alcoholic steatohepatitis (NASH),Phase 2b,6/24/23,"Phase 2b additional data presented on EASL showed that lanifibranor significantly increases the levels of adiponectin and that this increase is correlated with the improvements of cardiometabolic markers, including insulin resistance, glycemic control, lipid metabolism, and systemic inflammation, noted June 24, 2023.",6
CBIO,Hemophilia,Phase 3,11/12/21,"Program to be discontinued, Phase 3 data reported that the trial successfully treated bleeds with subcutaneous (SQ) MarzAA and has not observed any treatment-related adverse or thrombotic events, noted November 12, 2021.",6
CLNN,Amyotrophic Lateral Sclerosis (ALS),Phase 2/3,9/25/23,"Phase 2/3 post hoc results showed significantly improved survival with a 49% decreased risk of death for the covariate risk-adjusted analyses compared to the largest U.S. clinical database of previous amyotrophic lateral sclerosis (ALS) trials (PRO-ACT), noted September 25, 2023.",6
IMVIQ,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
PRVB,Coxsackievirus B (CVB) vaccine,Phase 1,10/26/21,"Phase 1 interim data met the primary endpoint and secondary efficacy endpoint as it induced high titers of viral-neutralizing antibodies against all CVB serotypes, noted October 26, 2021.",6
ATNXQ,Neoadjuvant breast cancer,Phase 2,12/20/22,"Phase 2 data reported that oral treatment was associated with less neuropathy and was not associated with an increase in febrile neutropenia, noted December 20, 2022.",6
MRK,Newly Diagnosed Classical Hodgkin Lymphoma (cHL),Phase 2,12/12/22,"Phase 2 data reported that after a median follow-up of 15.5 months, 22 patients had objective response (ORR, 73% [95% CI, 54-88]; complete response, 9 [30%]; partial response, 13 [43%]), noted December 12, 2022.",5
NVS,Reduction of thrombotic cardiovascular events including stent thrombosis in patients with coronary artery disease undergoing percutaneous coronary intervention (PCI),CRL,4/30/14,CRL April 30 2014.,5
ENDP,"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
LGND,Acute lymphoblastic leukemia (ALL),Approved,6/30/21,"FDA approval announced June 30, 2021.",6
CLVS,Urothelial carcinoma,Phase 2,4/12/19,"Phase 2 trial to be discontinued due to lack of efficacy - April 12, 2019.",4
IMMP,Ulcerative Colitis,Phase 2,1/21/21,"Phase 2 trial discontinued following interim analysis - January 21, 2021.",4
SABS,Type A and Type B influenza,Phase 2a,5/4/23,"Phase 2a first fully-human multi-epitope binding broadly neutralizing immunoglobulin antibody therapeutic being developed for treatment of high-risk patients demonstrates safety and efficacy in Phase 1 and 2a trials, noted May 4, 2023",6
TVTX,Cystinuria,Approved,6/28/19,"FDA Approval announced June 28, 2019.",6
EWTX,Duchenne Muscular Dystrophy (DMD),Approved,10/26/23,"Approved October 26, 2023.",6
ALNA,Hyperuricemia in chronic kidney disease (CKD),Phase 2a,1/4/22,"Phase 2a initial data reported statistically significant reduction in serum uric acid (sUA) from baseline compared to placebo, noted January 4, 2022.",6
SPRB,Classic Congenital Adrenal Hyperplasia,Phase 2,6/17/23,"Phase 2 data presented at ENDO highlighted that gonadal axis disruption is common in men with 21OHD and suggests that modulation of adrenal androgen production via CRFR1 antagonism may improve testicular function, noted June 17, 2023.",6
PFE,BRAF-Mutant Colorectal Cancer,Approved,4/8/20,"FDA Approval announced April 8, 2020.",6
TSVT,Acute myeloid leukemia (AML),Phase 1,6/14/23,"Phase 1 paused after recent Grade 5 (fatal) serious adverse event (SAE) and was followed by the required notification to the FDA, noted June 14, 2023.",4
ABBV,Chronic Lymphocytic Leukemia,Phase 3,12/10/22,"Phase 3 reported that treatment reduced the risk of progression or death by 79 percent among older and/or unfit patients with previously untreated chronic lymphocytic leukemia (CLL) compared to patients treated with chemoimmunotherapy, noted December 10, 2022.",6
BTAI,Agitation associated with Alzheimer’s Disease,Phase 3,6/29/23,"Phase 3 topline data reported that only 60mcg dose met primary endpoint, with a 7.5 point reduction versus 5.4 point reduction in the placebo group, noted June 29, 2023.",6
GTHX,Neoadjuvant breast cancer,Phase 2,12/20/22,"Phase 2 data reported that oral treatment was associated with less neuropathy and was not associated with an increase in febrile neutropenia, noted December 20, 2022.",6
INCY,Alopecia Areata,Approved,6/14/22,"Approved June 14, 2022.",6
VTGN,Social Anxiety Disorder (SAD),Phase 3,8/7/23,"Phase 3 topline data met its primary endpoint, noted August 7, 2023.",5
ADGI,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
ARQT,Plaque psoriasis,Phase 3,3/25/22,"Phase 3 pooled data reported that 40% of patients treated with roflumilast cream achieved IGA success and and 48% of patients treated with roflumilast cream achieved IGA status of clear, noted March 25, 2022.",6
HALO,Multiple myeloma,Approved,5/1/20,"FDA Approval announced May 1, 2020.",6
ABBV,Crohn’s Disease,Approved,6/17/22,"Approved June 17, 2022.",6
AIM,Early-stage triple negative breast cancer,Phase 1,9/11/23,"Phase 1 complete topline results confirmed treatment was well tolerated, with promising clinical activity of pathologic complete response (pCR) + microinvasive residual disease (ypTmic) at 66%, comparable to pembrolizumab/neoadjuvant chemotherapy (NAC), noted September 11, 2023.",6
AIMD,Sjögren's syndrome,Phase 3,11/3/22,"Phase 3 results showed increased unstimulated whole saliva and improvements in mouth dryness and seven other SS symptoms, noted November 3, 2022.",6
FENC,"Ototoxicity in non-metastatic, solid tumors",Approved,9/20/22,"Approved September 20, 2022.",6
JNJ,High-Risk Non-Muscle Invasive Bladder Cancer (NMIBC),Phase 2b,10/24/23,"Phase 2b data presented at ESMO reported that centrally confirmed CR was achieved in 23/30 pts, noted October 24, 2023.",6
RLMD,Major Depressive Disorder (MDD),Phase 3,9/20/23,"Phase 3 results showed that patients newly treated with REL-1017 for up to one year experienced rapid, clinically meaningful, and sustained improvements in depressive symptoms and associated functional impairment, noted Septemeber 20, 2023.",6
NKTR,1L Non-small cell lung cancer (NSCLC),Phase 2,12/13/21,"Phase 2 data reported an overall response rate (ORR) of 17%, including 2 complete responses (CR) in the highest PD-L1 status cohort, noted December 13, 2021.",5
BIIB,Friedreich’s ataxia (FA),Approved,6/28/23,"PAS approved June 28, 2023.",6
KNSA,COVID-19,Phase 2,12/22/20,"Phase 2 trial did not meet primary endpoint - December 22, 2020.",4
XOMA,Hyperprolactinemia,Phase 2,4/24/17,"Phase 2 proof-of-concept results released April 24, 2017.",5
FNCH,C. difficile Infection,Phase 2,10/24/22,"Phase 2 biomarker data presented at ACG 2022 showed that administration of treatment after standard-of-care (SOC) CDI antibiotics, there was a significant increase in microbiome diversity from baseline through week 8 and week 24, noted October 24, 2022.",6
NRBO,Homozygous Familial Hypercholesterolemia (HoFH),Phase 2b,6/28/17,"Phase 2b data released June 28, 2017 - primary endpoint met.",6
DVAX,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
MRK,Bladder cancer,Approved,8/31/21,"FDA approval announced August 31, 2021.",6
UTHR,Pulmonary arterial hypertension (PAH),Phase 3,4/8/19,"Phase 3 data released April 8, 2019 did not meet primary endpoint.",4
LGVN,Aging Frailty,Phase 2b,9/29/21,"Phase 2b biomarker data showed Lomecel-B had statistically significant reduction in serum levels of soluble TIE-2 in a dose-dependent fashion at Day 270 compared to placebo. The levels fell by -243.13 ± 1073.21 pg/mL compared to an increase of 356.03 ± 1018.95 pg/mL in the placebo group, noted September 29, 2021.",6
JNJ,Multiple myeloma,Approved,8/10/23,"Approved August 10, 2023.",6
OCUL,Dry eye disease (DED),Phase 2,10/22/21,"Phase 2 data did not show separation between the OTX-CSI treated subjects and the vehicle treated subjects for the primary endpoint of increased tear production, noted October 22, 2021.",4
HALO,Pancreatic cancer,Phase 3,11/4/19,"Phase 3 data November 4, 2019 did not meet primary endpoint.",4
BLUE,Sickle cell disease,PDUFA,6/21/23,"PDUFA Date assigned for December 20, 2023.",5
FHTX,Synovial sarcoma,Phase 1,11/2/23,"Phase 1 preliminary results shared at the Connective Tissue Oncology Society Annual Meeting reported that FHD-609 treatment led to dose-dependent BRD9 degradation in tumor tissue, noted November 2, 2023.",6
IDYA,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
NVS,CV risk reduction,CRL,10/18/18,"CRL announced October 18, 2018.",5
TPTX,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
SLRX,Hematological cancers and solid tumors,Phase 1,6/12/23,"Phase 1 clinical trial data revealed that in Jeko-1 (lymphoma) cells, SP-3164 induces twice the degradation of target proteins I/A at 1/10th the dose of lenalidomide, providing further support for the development of SP-3164 in relapsed refractory non-Hodgkin lymphoma, noted June 12, 2023.",5
INMB,Immune mediated complications from COVID-19,Phase 2,11/3/21,"Phase 2 trial discontinued, noted November 3, 2021.",4
RYTM,Hypothalamic obesity,Phase 2,6/19/23,"Phase 2 data demonstrated a mean BMI reduction of 21.0% from baseline observed in 13 patients at six months, showing progression from 16.8% mean BMI reduction at 16 weeks, noted June 19 2023.",6
GSK,Anaemia in chronic kidney disease for patients on dialysis,Approved,2/1/23,"Approved February 1, 2023.",6
IOVA,Non-small cell lung cancer (NSCLC),Phase 2,11/12/21,"Phase 2 data results showed a 21.4% overall response rate (ORR) in heavily pre-treated patients (n=28), including one complete response and five partial responses, noted November 12, 2021.",5
GBT,Hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest,Phase 2a,10/23/17,"Phase 2a discontinuation announced October 23, 2017 due to lack of efficacy.",4
BIIB,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
RHHBY,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
VRTX,alpha-1 antitrypsin (AAT) deficiency,Phase 2,10/14/20,Phase 2 trial and development to be discontinued due to safety.,4
ANTX,Treatment-Refractory MAC Lung Disease,Phase 2/3,9/13/23,"Phase 2/3 enrollment completed, noted September 13, 2023.",5
DNLI,Frontotemporal dementia (FTD) caused by mutations in the granulin gene (GRN),Phase 1/2,11/1/22,"Phase 1/2 part A interim results showed that single doses of treatment result in substantial increases in CSF progranulin levels suggesting brain delivery of DNL593 was achieved and has the potential to address progranulin deficiency, noted November 1, 2022.",6
MTEM,Multiple Myeloma,Phase 1,4/7/23,"Partial clinical hold placed April 7, 2023.",5
BHC,Allergic conjunctivitis,Approved,9/25/20,"FDA approval announced September 25, 2020.",6
ARCT,COVID-19 variant vaccine,Phase 1/2,1/24/22,"Additional Phase 1/2 data reported neutralization against several variants of concern after day 15 and 29, noted January 24, 2022.",5
LLY,Migraine prevention,Approved,9/28/18,"FDA approval announced September 27, 2018.",6
KDNY,Chronic kidney disease,Phase 2,11/3/22,"Interim data reported that of the 20 patients enrolled, ten patients remained on study, nine patients completed 52 weeks of treatment and one patient discontinued after 13 weeks of treatment due to a headache. Treatment demonstrated mean reductions in 24-hour urine protein creatinine ratio (UPCR) of 38.1% at six weeks of treatment, 48.3% at 12 weeks of treatment and 54.7% at 24 weeks of treatment, noted November 3, 2022.",6
FULC,Sickle Cell Disease,Phase 1,2/24/23,"IND placed on hold February 24, 2023.",5
AMRX,Advanced prostatic cancer,Approved,11/2/22,"FDA Abbreviated New Drug Application (ANDA) approval on November 2, 2022.",6
NRXP,COVID-19,Phase 2/3,10/14/21,"Data from ineligible subjects for the Phase 2b/3 trial recorded a 60-day survival in 81% of those treated with aviptadil, compared to 21% survival among those who received standard of care treatment, noted October 14, 2021.",6
CAPR,COVID-19,Phase 2,3/28/22,"Phase 2 met safety primary endpoint, with an overall mortality of 20% and 5 deaths in the treatment group, noted March 28, 2022.",6
AMGN,Thyroid Eye Disease (TED),Approved,1/21/20,"FDA Approval announced January 21, 2020.",6
CLLS,Relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL),Phase 2,6/8/23,"Phase 2 data presented at EHA reported that treatment was well tolerated and clinical responses were achieved, noted June 8, 2023.",6
BIIB,Mild cognitive impairment (MCI) due to Alzheimer's disease (AD),Approved,7/6/23,"Traditional approval received July 6, 2023.",6
AZN,"Monotherapy for expanded use in women with HR+, HER2- advanced breast cancer",Approved,8/28/17,"Expanded approval announced August 28, 2017.",6
FMTX,Acute myeloid leukemia,Approved,12/1/22,"Approved December 1, 2022.",6
HOTH,"Aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN).",Phase 1a,9/13/23,"Preclinical Results of Novel HT-KIT Therapeutic Performed at NC State University demonstrated that gastrointestinal Stromal Tumors Cells Rapidly Died Between 48 and 72 Hours After Exposure to HT-KIT. HT-KIT Shows Positive Results in AML, noted September 13, 2023.",6
ATAI,Healthy volunteers,Phase 1,8/8/23,"Phase 1 was generally well-tolerated with no SAE reported. At the 60mg IV dose, rates of sedation and dissociation were consistent with prior studies of PCN-101 at this dose-level, noted August 8, 2023.",6
VTYX,Ulcerative Colitis (UC),Phase 2,10/9/23,"Phase 2 top-line data results showed that at 60 mg achieved the primary endpoint of clinical remission with a high rate of complete endoscopic remission, noted on October 9, 2023.",6
ALKS,Multiple sclerosis (MS),Approved,10/30/19,"FDA Approval announced October 30, 2019.",6
NVO,Type 2 Diabetes,Approved,3/28/22,"Approved March 28, 2022.",6
GRTS,1L Microsatellite-Stable Colorectal Cancer (MSS-CRC),Phase 1/2,1/13/22,"Phase 1/2 data demonstrated that patients who experienced a molecular response, continue to have an OS advantage compared to those patients who did not have a molecular response. All patients alive at the time of the ESMO 2021 data presentation remain alive after an additional 22 weeks of follow-up, noted January 13, 2021.",6
RAPT,Advanced Cancer,Phase 1/2,11/3/23,"Phase 1/2 update SITC showed a confirmed objective response rate (ORR) of 40% in PD-L1 positive (TPS ≥1%) patients with no prior checkpoint inhibitor therapy (CPI). Median progression-free survival (PFS) of 6.3 months in PD-L1 positive patients at time of data cut off, noted November 3, 2023",6
ABBV,Forehead lines,Approved,10/3/17,"Approval (third indication) announced October 3, 2017.",6
VBIV,Recurrent glioblastoma multiforme (rGBM),Phase 2b,9/7/23,"Phase 2b treatment in the primary GBM expected to commenced, noted September 7, 2023.",6
REGN,Asthma,Approved,10/19/18,"FDA approval announced October 19, 2018.",6
GSK,HIV,Approved,4/8/19,"FDA approval announced April 8, 2019.",6
INCY,Myelofibrosis,Approved,11/16/11,"Approved November 16, 2011.",6
ACIU,Neurodegenerative diseases (NDD),Phase 1,7/18/23,"Updated biomarker data reported that ACI-15916shows significantly improved specific binding to pathological a-syn aggregates in brain sections from different a-synucleinopathy cases, noted July 18, 2023.",6
ZLAB,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
REGN,Chronic spontaneous urticaria (CSU),CRL,10/20/23,"CRL issued by the FDA, noted October 20, 2023.",5
RIGL,Immune Thrombocytopenic Purpura (ITP),Approved,4/17/18,"Approved April 17, 2018.",6
RDHL,Crohn’s disease,Phase 3,7/30/18,"Phase 3 data released July 30, 2018. Primary endpoint met but further trial required.",5
TVTX,IgA nephropathy,Approved,2/17/23,"FDA accelerated approval on February 17, 2023.",6
ACST,Subarachnoid hemorrhage (SAH),Phase 3,5/8/23,"PK study met all endpoints, noted May 18, 2022. Phase 3 trial to be initiated in 2H 2023. If the Phase 3 study meets the primary endpoint, an NDA filing for GTX-104 under Section 505(b)(2) is expected to follow, noted April 4, 2023. The company successfully submited GTX-104 Pivotal Phase 3 protocol IND amendment to the FDA with expectation for first patient dosed in calendar 4Q 2023, noted May 8, 2023.",6
JAZZ,Idiopathic Hypersomnia,Approved,8/12/21,"FDA approval announced August 12, 2021.",6
REGN,Wet AMD,CRL,8/13/18,"CRL issued August 13, 2018 - ongoing labeling discussions.",5
AZN,Paroxysmal nocturnal hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS),Phase 3,6/24/20,"Phase 3 trial met primary endpoint - June 24, 2020",6
MRSN,Ovarian Cancer,Phase 3,6/15/23,"A clinical hold placed due to bleeding events, noted June 15, 2023.",5
AUTL,Relapsed or refractory diffuse large B-celllymphoma (DLBCL),Phase 2,12/7/20,"Phase 2 updated data at ASH December 7, 2020 noted objective response rate (ORR) of 65% and a CR rate of 51%.",6
ACAD,Rett Syndrome,Approved,3/10/23,"Approved March 10, 2023.",6
NVO,Hemophilia A,Approved,2/19/19,"FDA Approval announced February 19, 2019.",6
CKPT,Non-small cell lung cancer (NSCLC),Phase 1/2,9/25/18,"Phase 1/2 preliminary data released September 24, 2018.",5
CYBN,Major Depressive Disorder (MDD),Phase 1b,9/27/23,"Phase 1b data reported that 100% of patients in the SSRI cohort responded to SPL026 (DMT) with 92% of patients in remission from depression, noted September 27, 2023.",5
URGN,Overactive Bladder (OAB),Phase 2,8/27/20,"Phase 2 trial did not meet primary endpoint - August 27, 2020.",4
CCXI,Focal Segmental Glomerulosclerosis,Phase 2,5/18/20,"Phase 2 trial did not meet primary endpoint - May 18, 2020.",4
GLTO,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
CLVS,Mutant EGFR T790M-positive lung cancer,CRL,6/28/16,"CRL June 28, 2016. Phase 3 TIGER-3 also terminated.",4
ADPT,Multiple Myeloma,Approved,8/14/23,"FDA Approved on August 14, 2023.",6
JNJ,HIV,Approved,7/18/18,"FDA Approval announced July 17, 2018.",6
MRUS,Solid Tumors,Phase 3,10/26/22,"Phase 1/2 update reported that treatment was well-tolerated, with 2 confirmed partial responses observed and 4 additional patients had >20% tumor shrinkage, noted October 26, 2022.",6
XERS,Severe hypoglycemia,Approved,9/10/19,"FDA Approval issued September 10, 2019.",6
SIGA,Smallpox,Approved,5/19/22,"Approved May 19, 2022.",6
ADMA,Primary Immune Deficiency Diseases,Approved,12/13/22,FDA supplemental approval for room temperature (25°C) storage conditions for up to 4 weeks during the first 24 months of the 36-month approved shelf life.,6
JAZZ,Small cell lung cancer (SCLC),Approved,6/15/20,"FDA Approval announced June 15, 2020.",6
EGRX,Severe Community-Acquired Bacterial Pneumonia (SCABP),Phase 2/3,6/14/23,"FDA granted Qualified Infectious Disease Product (QIDP) Designation under the Generating Antibiotic Incentives Now (GAIN) Act and Fast Track Designation, noted June 14, 2023. QIDP designation entitles Eagle to an additional five years of exclusivity upon NDA approval.",6
OCUL,Glaucoma and ocular hypertension,Phase 3,5/20/19,"Phase 3 data May 20, 2019 did not meet primary endpoint.",4
KPTI,Dedifferentiated liposarcoma,Phase 3,11/2/20,"Phase 3 trial met primary endpoint - November 2, 2020.",6
INCY,Cancer - first-line metastatic melanoma,Phase 3,4/6/18,"Phase 3 trial did not meet primary endpoint - noted April 6, 2018.",4
MRK,"First-line, lower-risk Myelodysplastic syndromes (MDS)",Approved,8/28/23,"Approved August 28, 2023.",6
ONTX,2nd-line HR-MDS (high risk myelodysplastic syndromes),Phase 3,8/24/20,"Phase 3 trial did not meet primary endpoint - August 24, 2020.",4
BIIB,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
INCY,Anal cancer,Phase 2,9/18/20,Phase 2 data noted objective response rate of 14% and disease control rate of 49%.,5
EFTR,Metastatic breast cancer (mBC),Phase 2a,5/25/23,"Phase 2a data observed partial responses seen in 26% of patients, is substantially higher than we would expect for treating such heavily pretreated patients with just fulvestrant and abemaciclib, noted May 25, 2023",6
ALXO,Myelodysplastic syndromes (MDS),Phase 1b,8/10/23,"Phase 1b data reported that the evorpacept combination did not substantially improve upon the historical activity of azacitidine alone, noted August 10, 2023.",6
NKTR,Melanoma,Phase 3,3/14/22,"Phase 3 data did not meet the primary endpoints, noted March 14, 2022.",6
RVNC,Moderate to severe glabellar (frown) lines,Approved,9/8/22,"Approved September 8, 2022.",6
NVS,Pegfilgrastim biosimilar,Approved,11/5/19,"FDA Approval announced November 5, 2019.",6
SNY,Immune thrombocytopenic purpura (ITP),Phase 2,12/7/19,Phase 2 data at ASH December 2019 noted 39% of patients achieved primary endpoint.,5
SGEN,Frontline Hodgkin lymphoma,Approved,4/4/18,"sBLA approval announced March 20, 2018.",6
AERI,Dry eye disease,Phase 2b,4/26/22,"Phase 2b results reported that statistically significant improvements were reported with signs and symptoms following treatment, noted April 24, 2022.",6
TEVA,Rituxan (rituximab) biosimilar,Approved,11/28/18,"FDA Approval announced November 28, 2018.",6
NVS,IgA nephropathy (IgAN),Phase 3,10/2/23,"Phase 3 data reported that met its pre-specified interim analysis primary endpoint, noted October 2, 2023.",5
KA,Solid tumors,Phase 1/2,11/3/23,"Phase 1/2 biomarker data to be presented at SITC, noted Noember 3 2023.",6
CHRS,Esophageal Squamous Cell Carcinoma,Phase 3,9/17/21,"Phase 3 trial met the co-primary endpoints of progression free survival (PFS) and overall survival (OS) for patients treated with the toripalimab and chemotherapy combination compared to chemotherapy alone, September 17, 2021.",6
MOR,Psoriatic Arthritis,Approved,7/14/20,"FDA Approval announced July 14, 2020.",6
MRK,Head and neck squamous cell carcinoma (HNSCC),Phase 3,7/20/22,"Phase 3 trial did not meet primary endpoint, noted June 20, 2022.",6
REGN,Homozygous familial hypercholesterolemia (HoFH),Approved,4/1/21,"FDA approval announced April 1, 2021.",6
AMGN,Hematopoietic Syndrome of Acute Radiation Syndrome,Approved,2/2/21,"FDA approval announced in earnings release - February 2, 2021.",6
BYSI,Docetaxel-induced neutropenia in breast cancer,Phase 2/3,12/13/22,"Additional data reported that plinabulin was superior vs no-treatment for DIN and hematologic complications, noted December 13, 2022.",6
AZN,"Severe, uncontrolled asthma",Phase 3,5/10/17,"Phase 3 trial did not meet primary endpoint - May 10, 2017.",4
RHHBY,Hepatocellular carcinoma,Phase 3,4/17/23,"Additional Phase 3 data reported that the combination reduced the risk of cancer returning by 28%, compared with active surveillance, at a median follow-up of 17.4 months, noted April 17, 2023.",6
TENX,Reduce the incidence of low cardiac output syndrome during cardiac surgery,Phase 3,1/31/17,"Phase 3 top line data released January 31, 2017 - primary endpoints not met.",4
AZN,Neuromyelitis Optica Spectrum Disorder (NMOSD),CRL,9/6/23,"CRL issued on September 6, 2023.",5
INCY,Relapsed or refractory diffuse large B cell lymphoma,Approved,7/31/20,"FDA Approval announced July 31, 2020.",6
ABBV,Major depressive disorder (MDD),Approved,12/19/22,"Approved December 19, 2022.",6
JAZZ,Acute Myeloid Leukemia (AML),Phase 1b,12/12/22,"Phase 1b subgroup analysis reported that complete remission (CR) was achieved by 14/17 (82%) patients with a FLT3 ITD mutation and by 5/6 (83%) patients with a FLT3 TKD mutation, noted December 12, 2022.",6
EVAX,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
ANNX,Retinal vein occlusion (RVO),Phase 2,8/11/23,"Phase 2 data reported a long duration of effect with a single dose of standard of care anti-VEGF treatment in two of five treated patients. These two patients also had some positive effects on visual acuity and the retina already during the four weeks before standard of care treatment was used, noted August 11, 2023.",6
TEVA,Tardive dyskinesia (TD),Approved,2/17/23,"FDA Approval on February 17, 2023.",6
REGN,Chaple,Approved,8/18/23,"Approved August 18, 2023.",6
PTN,Dry eye disease (DED),Phase 2,12/15/20,Phase 2 trial met the primary endpoints in the moderate to severe patient population but not in the overall population including mild patients.,5
KURA,Head and neck squamous cell carcinomas (HNSCC),Phase 2,10/21/23,"Phase 2 data presented at ESMO reported that the Tipifarnib ORR was higher in 2L setting than 3L+ setting (29% vs. 15%), noted October 21, 2023.",6
TAK,Medullary thyroid cancer,Approved,11/29/12,"Approved November 29, 2012.",6
MREO,Solid tumors,Phase 1/2,11/30/21,"Phase 1b/2 interim data showed that the dose was well tolerated with a favorable safety profile. One complete response, one partial response and four cases of stable disease observed among 15 patients in the efficacy analysis set",6
XERS,Endogenous Cushing’s syndrome,Approved,12/30/21,"Approved December 30, 2021.",6
AMGN,Ph+ R/R ALL,Approved,7/11/17,"PDUFA date under priority review August 14, 2017. Approval announced July 11, 2017.",6
FREQ,Acquired Sensorineural Hearing Loss (SNHL),Phase 2a,3/23/21,"Phase 2a trial did not show improvement versus placebo - March 23, 2021.",4
DYAI,COVID-19 booster vaccine,Phase 1,7/24/23,"Phase 1 safety data reporter no Serious Adverse Events or Adverse Events of Special Interest have been reported to date, noted July 24, 2023",5
JSPR,Sickle Cell Disease and Beta Thalassemia,Phase 1/2,2/16/23,"Phase 1/2 follow up data reported that two sickle cell disease participants have achieved 100% donor myeloid chimerism through 100 days follow-up, noted February 16, 2023.",6
IMGN,Acute Myeloid Leukemia (AML),Phase 1/2,12/12/21,"Phase 1b/2 data reported a objective response rate (ORR) of 48%, with a composite complete remission (CCR) rate of 30%, noted December 12, 2021.",5
JNJ,Relapsed or refractory (R/R) multiple myeloma.,Approved,10/26/22,"Approved October 26, 2022.",6
CYDY,Triple Negative Breast Cancer,Phase 1/2,11/3/21,"Phase 1/2 data reported 75% of patients with a lower level of circulating cells after leronlimab (86%) or at baseline (14%) exhibited a 3600% increase in 12-month Overall Survival, as compared to a 980% reported in August 2021. A 580% increase in 12-month Progression Free Survival was reported as well, November 3, 2021.",6
INCY,Squamous Cell Carcinoma of the Anal Canal (SCAC),CRL,7/23/21,"CRL announced July 23, 2021",5
OMER,Cataract surgery,Approved,6/2/14,"Approved June 2, 2014.",6
PFE,Relapsed or Refractory Acute Lymphoblastic Leukemia in Adults,Approved,8/17/17,"Approval announced August 17, 2017.",6
NVIV,Complete Thoracic AIS A Spinal Cord Injury,Phase 3,3/9/23,"Pivotal trial did not meet the primary endpoint, noted March 9, 2023.",6
GKOS,Dry Eye Disease (DED),Phase 2a,1/10/23,"Phase 2a outcomes for GLK-301 demonstrated improvement in the quality of tear film, noted January 10, 2023.",6
VERU,Benign prostatic hyperplasia (BPH),Approved,12/13/21,"Approved December 13, 2021.",6
FIXX,Phenylketonuria (PKU),Phase 1,7/21/23,"Phase 1 initial data reported that participant 2 has experienced a meaningful plasma Phe reduction of 49% at 17, and participant 1 had a reduction in plasma phenylalanine (Phe) levels to below the ACMG PKU treatment guideline threshold of <360 μmol/L, noted July 27, 2023.",6
BLRX,Stem-cell mobilization for autologous transplantation,Approved,9/11/23,"FDA Approved on September 11, 2023.",6
BPMC,Treatment-resistant EGFR-driven NSCLC,Phase 1/2,6/5/23,"Phase 1/2 initial clinical data presented at ASCO reported confirmed partial responses observed in patients whose tumors had progressed following treatment with osimertinib, note June 5, 2023.",6
ARAV,Platinum Resistant Ovarian Cancer (PROC),Phase 3,8/2/23,"Phase 3 topline data did not meet primary endpoint of progression-free survival, noted August 2, 2023.",6
MRTX,Non-small cell lung cancer (NSCLC) with a KRASG12C Mutation,Approved,12/13/22,"Approved December 13, 2022.",6
AVRO,Fabry disease,Phase 2,10/19/21,"Phase 2 safety data noted that no adverse events (AEs) or serious adverse events (SAEs) were reported, October 19, 2021.",6
CINC,Uncontrolled Hypertension (rHTN),Phase 1,11/28/22,"Phase 1 topline data reported that trial did not meet primary endpoint, noted November 28, 2022.",6
GSK,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
BIIB,Acute ischemic stroke,Phase 2b,4/3/18,"Phase 2b data released February 8, 2018 - primary endpoint not met.",4
MRK,Colorectal cancer (CRC),Approved,6/29/20,"FDA Approval announced June 29, 2020.",6
CLVS,Cancer - ovarian second line,Approved,12/19/16,"Approved December 19, 2016. Original PDUFA date under priority review was February 23, 2017.",6
ABEO,Sanfilippo syndrome type A (MPS IIIA),Phase 1/2,2/12/21,"Phase 1/2 updated safety data released February 12, 2021. Well tolerated.",6
REGN,Ebola,Approved,10/14/20,"FDA Approval announced October 14, 2020.",6
AGLE,Arginase I deficiency,Phase 3,4/11/22,"Phase 3 results reported that 65% of patients treated reached or exceeded prespecified response criteria for at least one mobility assessment, with 6 exceeding for tow mobility outcomes, noted April 11, 2022.",6
VALN,Chikungunya Virus Infection,Phase 3,8/5/21,"Phase 3 trial met primary endpoint - August 5, 2021.",6
BYSI,Multiple Myeloma,Phase 2,8/29/22,"Phase 2 data reported that the median white blood count (WBC) on Day 0, 1 and 2 was 7.6. Six patients had fever at median of eight days post AHCT. Five patients with engraftment syndrome were treated with steroids, and there was one patient with non-engraftment-related neutropenic fever, noted August 29, 2022.",6
ARQT,Vitiligo,Phase 2b,7/1/21,"Phase 2a trial to be terminated - noted July 1, 2021.",4
ABBV,HCV - genotype 1,Approved,12/19/14,"Approved December 19, 2014.",6
GMAB,Solid tumors,Phase 1/2,11/10/21,"Phase 1/2 data demonstrated biologic and early antitumor activity with a favorable safety profile in patients with advanced solid tumors. Expansion cohorts, including combination therapy with PD-1 inhibitors, are currently enrolling, noted November 10, 2021.",6
VIR,COVID-19 antibody,Approved,5/26/21,"FDA Emergency Use Authorization filing approval announced May 26, 2021.",6
ANIP,"Acute exacerbations of multiple sclerosis, rheumatoid arthritis, systemic lupus erythematous and ulcerative colitis",Approved,10/2/23,"1-ml vial approved October 2, 2023.",6
BNTX,Solid tumors,Phase 1/2,11/10/21,"Phase 1/2 data demonstrated biologic and early antitumor activity with a favorable safety profile in patients with advanced solid tumors. Expansion cohorts, including combination therapy with PD-1 inhibitors, are currently enrolling, noted November 10, 2021.",6
NKTR,Hemophilia A,Approved,11/16/15,"Approved November 16, 2015.",6
MIRM,Alagille Syndrome (ALGS),Approved,9/29/21,"Approved September 29, 2021.",6
OBSV,Endometriosis,Phase 3,1/10/21,Phase 3 trial discontinued due to enrollment challenges.,4
GLSI,HER2/neu breast cancer,Phase 2b,4/19/22,"Phase 2b trial data reported that treatment safely elicited a potent immune response, as evidenced by injection site reactions that correlate to and may serve as a complement to immune response data such as DTH, noted April 19, 2022.",6
CWBR,Cancer vaccine for merkel cell carcinoma (MCC),Phase 1b,6/5/23,"Phase 1b data presented at ASCO demonstrated to have a promising safety profile at all 3 dose schedules tested 71% of patients achieved durable systemic anti-tumor responses following IFx-Hu2.0 therapy and rechallenge with an IFx-Hu2.0 demonstrated to have a promising safety profile at all 3 dose schedules tested, noted June 5, 202",6
NVS,Wet macular degeneration,Phase 3,8/15/23,"Phase 3 trial met its primary endpoint, noted August 15, 2023.",5
NBIX,Negative symptoms of schizophrenia,Phase 2,3/2/21,"Phase 2 trial did not meet primary endpoint - March 2, 2021.",4
TNXP,Major depressive disorder (MDD),Phase 2,11/1/23,"Phase 2 topline data reported that the trial did not meet its primary endpoint, noted November 1, 2023.",6
SAGE,Insomnia,Phase 1/2,1/31/18,"Phase 1/2 trial met primary endpoint - January 31, 2018.",6
NVAX,Covid-19,Approved,10/3/23,"EUA approved October 3, 2023.",6
RHHBY,Relapsed or refractory Chronic Lymphocytic Leukemia (CLL),Approved,6/11/18,"Approval announced June 11, 2018.",6
ADAP,Urothelial Cancer,Phase 1,11/8/22,"Phase 1 data reported increased ORR now 57% in urothelial cancer with one new complete response, noted November 8, 2022.",5
ALNY,Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR,Approved,8/10/18,"FDA Approval announced August 10, 2018.",6
TAK,Non-small cell lung cancer (NSCLC),Approved,9/15/21,"Approved September 15, 2021.",6
MRK,Endometrial Cancer,Phase 3,7/22/21,"FDA approval announced July 22, 2021.",6
LSTA,Solid Tumors,Phase 2a,9/12/23,"Phase 2a dosing initiated on head and neck squamous cell carcinoma cohort, noted Septemeber 12, 2023.",5
MRK,First-Line Treatment of Metastatic Non-Squamous Non-Small Cell Lung Cancer,Approved,5/10/17,"Approved May 10, 2017.",6
MRK,NMIBC Bladder cancer,Approved,1/8/20,"FDA Approval announced January 8, 2020.",6
AMLX,Amyotrophic Lateral Sclerosis (AML),Phase 3,2/2/23,"Phase 3 trial enrollment completed, noted February 2, 2023.",5
RHHBY,HER2-positive breast cancer - residual invasive disease,Approved,5/6/19,"FDA approval announced May 6, 2019.",6
IPHA,Squamous Cell Carcinoma of the Head and Neck (SCCHN),Phase 2,10/16/23,"Phase 2 results reported that D+M 1 partial response (PR) was recorded and stable disease (SD) was observed in 11 pts (26%), noted October 16, 2023.",6
BCRX,Acute Influenza,Approved,12/22/14,"Approved December 22, 2014.",6
MDNA,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
AYTU,Attention deficit hyperactivity disorder (ADHD),Approved,9/15/17,"Approval announced September 15, 2017.",6
MESO,"Pediatric participants with acute graft versus host disease (aGVHD), following allogeneic hematopoietic stem cell transplant (HSCT)",Phase 3,9/21/23,"Type A meeting noted the lack of a suitable potency assay for the RYONCIL product used during the Phase 3 trial, new potency assay data to be generated, noted September 21, 2023.",6
ABBV,Relapsed or refractory MCL mantle cell lymphoma,Approved,11/13/13,"Approved November 13, 2013.",6
SLN,Cardiovascular diseases,Phase 1,11/1/23,"Phase 1 data showed very significant and durable reductions in lipoprotein(a) of up to 99%. Lp(a) levels remained around 90% lower than baseline at study endpoint, noted November 1, 2023.",6
AMGN,Immune thrombocytopenia (ITP),Approved,10/18/19,"FDA Approval announced October 18, 2019.",6
OTIC,Meniere's disease,Phase 3,8/30/17,Phase 3 data released August 30 failed to meet primary and key secondary endpoints.,4
KALA,Post‑operative inflammation and pain following cataract surgery,Approved,8/23/18,"FDA approval announced August 23, 2018.",6
IDYA,Non-metastatic uveal melanoma (UM),Phase 1/2,5/9/23,"Phase 2 data reported that treatment showed evidence of anti-tumor activity, noted May 9, 2023.",6
ZLAB,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
CTIC,Myelofibrosis,Approved,2/28/22,"Approved February 28, 2022.",6
LLY,Metastatic colorectal cancer,Approved,9/28/21,"Approved September 28, 2021.",6
KYMR,Cutaneous T-cell lymphoma (CTCL),Phase 1,9/18/23,"FDA granted Fast Track Designation in ongoing Phase 1 trial, noted September 18, 2023.",5
ASRT,Neutropenia,Approved,9/9/22,"Approved September 9, 2022.",6
WINT,COVID-19 / Acute Lung Injury/Acute Respiratory Distress Syndrome (ARDS),Phase 2,3/22/22,"Phase 2 data reported that treatment was safe and well tolerated with fast administration and considered feasible, noted March 22, 2022.",6
ITCI,Schizophrenia,Approved,12/23/19,"FDA Approval announced December 23, 2019.",6
KNTE,Solid Tumors,Phase 1,9/18/23,https://www.biopharmcatalyst.com/company/KNTE/news/159347,5
INCY,Vitiligo,Phase 2,10/11/23,"Phase 2 trial initiated March 2021. Phase 2b study results shared at EADV reported that the total body depigmentation improvement from baseline as measured by the Total Vitiligo Area Scoring Index (T-VASI) at Week 52 for povorcitinib 15-to-75 mg, 45 mg, 75 mg and placebo-to-75 mg were 40.7%, 42.7%, 41.3% and 18.1%, respectively, noted October 11, 2023.",6
ATHX,Acute Respiratory Distress Syndrome,Phase 1/2,2/2/22,"Phase 1/2 data reported a median 18.5 ventilator-free days compared to 6.5 days for placebo. 25% of the treatment group reported mortality compared to 40% in placebo, noted February 2, 2022.",6
FSTX,Advanced malignancies,Phase 1,12/6/22,"Phase 1 interim data reported one complete response, 6 stable disease (SD), and 16 progressive disease, noted December 6, 2022.",5
RHHBY,Hepatocellular carcinoma,Approved,5/29/20,"FDA Approval announced May 29, 2020.",6
TBPH,Ulcerative Colitis,Phase 2b,8/23/21,"Phase 2b data did not meet primary endpoint - August 23, 2021.",4
ZLDPF,Post bariatric surgery hypoglycemia,Phase 2,3/30/20,"Phase 2 data released March 30, 2020.",5
RVNC,Cervical Dystonia,Approved,8/14/23,"Approved on August 14, 2023.",6
SONN,Solid tumors,Phase 1,4/18/23,"Phase 1 data presented at AACR reported no dose limiting toxicities, and 5 of the first 14 patients (36%) have evidence of clinical benefit, noted April 18, 2023.",6
HGEN,Chronic myelomonocytic leukemia (CMML),Phase 2,4/14/23,"Phase 2 preliminary study results reported that 6 subjects demonstrated clinical benefit, with ten grade 3/4 Serious Adverse Events, noted April 14, 2023.",6
ZLAB,Non-small cell lung cancer (NSCLC) with a KRASG12C Mutation,Approved,12/13/22,"Approved December 13, 2022.",6
AXLA,Non-alcoholic steatohepatitis (NASH) / non-alcoholic fatty liver disease (NAFLD),Phase 2b,9/29/22,"Additional Phase 2b data reported statistically significant improvements in the liver stiffness measurement (LSM) compared to placebo in the high dose arm for all subjects, noted September 29, 2022.",6
JNJ,BRCA-positive mCRPC,Approved,8/11/23,"Approved August 11, 2023.",6
BTAI,Adjunctive Treatment for Major Depressive Disorder (MDD) for At-Home Use,Phase 1b,5/16/23,"Phase 1b data topline data showed that BXCL501 was well tolerated across a broad dose range from 30mcg to 140mcg administered chronically, noted May 16, 2023.",6
BLCO,Dry Eye Disease Associated With Meibomian Gland Dysfunction,Approved,6/29/23,"Approved on May 18, 2023.",6
BIIB,Alzheimer’s disease,Approved,6/7/21,"FDA approval announced June 7, 2021.",6
REGN,Genetic hearing loss,Phase 1/2,10/26/23,"Phase 1/2 data reported that one patient experienced improvements in auditory responses through week 6 compared to baseline, noted October 26, 2023.",6
EYEN,Mydriasis (office-based pupil dilation),Approved,11/2/23,"FDA supplemental approval on November 1, 2023.",6
INO,HPV-associated squamous cell carcinoma of the head & neck (SCCHN),Phase 1,11/10/17,"Presentation at SITC Meeting - November 10, 2017.",6
SNDX,Ovarian cancer,Phase 1/2,3/7/19,"Phase 2 top-line data March 7, 2019 failed to meet endpoints.",4
REGN,Nonproliferative Diabetic Retinopathy,Approved,5/13/19,"FDA Approval announced May 13, 2019.",6
RIGL,COVID-19,Phase 2,8/13/21,"Announced August 12, 2021 that data are insufficient for FDA emergency use authorization (EUA).",4
SNPX,Alzheimer's disease,Phase 2b,9/9/23,"Phase 2b data presented at IBRO reported that nearly all pre-specified secondary endpoints were achieved with statistical significance in the server cohort, noted September 9, 2023.",6
INCY,Treatment-naïve acute GVHD,Phase 3,1/2/20,"Phase 3 trial did not meet primary endpoint - January 2, 2020.",4
OLMA,"HR+, HER2- Breast Cancer",Phase 1/2,10/22/23,"Phase 1/2 monotherapy data presented at ESMO reported that the median PFS was 4.6 months with a CBR of 40%; in patients with ESR1 mutations at baseline, the median PFS was 5.6 months with a CBR of 52%, noted October 22, 2023.",6
TNGX,Solid Tumors,Phase 1,7/24/23,"IND cleared by FDA, noted April 3, 2023. Phase 1/2 dosing initiated, noted July 24, 2023.",5
SAVA,Alzheimer’s disease,Phase 2b,9/22/21,"Phase 2b open label data showed ADAS-Cog11 scores improved an average of 3.2 points from baseline, 68% (n=50) improved on ADAS-Cog at 12 months, an average of 6.8 points. 20% declined less than 5 points on ADAS-Cog at 12 months, an average of 2.5 points, noted September 22, 2021.",6
RHHBY,Diabetic Macular Edema,Approved,1/28/22,"FDA Approval announced January 28, 2022.",6
VERA,IgA nephropathy (IgAN),Phase 2a,11/5/22,"Phase 2a final data presented at the American Society of Nephrology Kidney Week reported that atacicept reduced immune complex levels in patients with IgA nephropathy (IgAN), noted November 5, 2022.",6
BIVI,Alzheimer's disease,Phase 2,12/5/22,"Additional Phase 2 data reported that 18 of 22 patients with abnormal baseline scans showed improvement in one or more brain regions as seen from advanced functional MRI studies, noted December 5, 2022.",6
AZN,Breast cancer,Approved,1/12/18,"Approval announced January 12, 2018.",6
NBRV,Moderate to severe Community Acquired Bacterial Pneumonia (CABP),Approved,8/19/19,"FDA approval announced August 19, 2019.",6
TAK,Frontline CD30-positive mature T-cell lymphomas - cancer,Approved,11/16/18,"FDA Approval announced November 16, 2018.",6
JAZZ,Excessive sleepiness associated with obstructive sleep apnea,Phase 3,3/20/17,"Phase 3 data released March 20, 2017 - primary endpoints met.",6
ZYNE,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
NBIX,Classic congenital adrenal hyperplasia (CAH),Phase 2,6/13/22,"Phase 2 data reported that median percent reductions ranged from -57.1% to -76.2% and a greater than 50% reduction from baseline for ACTH, 17-OHP, and androstenedione (males and females), testosterone (females), and androstenedione/testosterone ratio (males) was achieved by 63%, 75%, 50%, 60%, and 67% of participants, respectively, noted June 13, 2022.",6
LLY,Obesity,Phase 2,6/26/23,"Phase 2 24-week data met the primary endpoint for the efficacy estimand in participants living with obesity or overweight without diabetes, demonstrating a mean weight reduction up to 17.5%. In a secondary endpoint, retatrutide showed a mean weight reduction up to 24.2% at 48 weeks, noted June 26, 2023.",6
GSK,HIV,Approved,11/21/17,"Approval announced November 21, 2017.",6
NVS,Chronic spontaneous urticaria (CSU),Phase 2,10/27/21,"Phase 2 trial met primary endpoint, noted October 27, 2021.",6
AKBA,ARDS in subjects with COVID-19 and hypoxemia,Phase 2,8/4/22,"Phase 2 trial data reported that at Day 14, the proportions of subjects who had a 6, 7 or 8 on the NIAID-OS were 13.3% for vadadustat versus 16.9% for placebo, missing its primary endpoint, noted August 4, 2022.",5
ABBV,Atopic Dermatitis,Approved,1/14/22,"Approved January 14, 2022.",6
LIAN,Demodex Blepharitis,Approved,7/25/23,"FDA approved on July 25, 2023.",6
CHRS,Nasopharyngeal carcinoma (NPC),Approved,10/27/23,"FDA Approval on October 27, 202",6
MIST,Paroxysmal supraventricular tachycardia (PSVT),Phase 3,11/7/22,"Phase 3 additional data reported that median time to conversion was 17 minutes for patients treated with etripamil, three times faster than placebo, noted November 7, 2022.",6
NRIX,Solid tumors,Phase 1a,11/10/22,"Phase 1a biomarker and safety reported dose-responsive target engagement and a mean half-life of 6 to 8 hours at doses ranging from 5 mg to 50 mg, noted November 10, 2022.",5
AZN,Head & neck cancer,Phase 3,2/5/21,"Phase 3 trial did not meet the primary endpoint - February 5, 2021.",4
AUTL,Pediatric Acute lymphoblastic leukemia (ALL),Phase 1,9/5/23,"Phase 1 data presented at the Blood journal showed that of 10 responding patients, 5 had emergence of MRD (2) or frank relapse (3) with CD19 and CD22 expressing disease associated with loss of CAR T-cell persistence. Importantly, there were no cases of relapse due to antigen-negative escape, with a median follow-up of 8.7 months. Overall survival was 75% at 6 and 12 months. Six and 12-month event free survival (EFS) were 75% and 60% respectively, noted September 5, 2023.",6
OCGN,COVID-19 (within U.S.),Phase 2/3,1/9/23,"Phase 2/3 Immuno-bridging and Broadening study met both co-primary endpoints, noted January 9, 2023.",5
ZLAB,Gastric and gastro-esophageal junction cancer,Phase 3,7/14/23,"Trial in China initiated, noted July 14, 2023.",5
CARA,Pruritus in patients with Atopic dermatitis,Phase 2,3/28/22,"Phase 2 presentation at AAD reported that within a sub-study treatment altered expression of multiple individual pruritus- and AD-related genes, noted March 28, 2022.",6
ZURA,Primary Sjogren's Syndrome,Phase 2,1/27/22,"Phase 2 study withdrawn noted January 27, 2022.",5
DNLI,Healthy Volunteers,Phase 1,10/6/21,"Phase 1 data noted that treatment was well tolerated for up to 14 days of dosing, with robust distribution in the central nervous system (CNS) and predictable dose-related increases in exposure with a PK profile supporting once daily dosing, noted October 6, 2021.",6
AZN,Pancreatic cancer,Approved,12/30/19,"FDA Approval announced December 30, 2019.",6
ARWR,"Familial chylomicronemia syndrome (FCS), Hypertriglyceridemia",Phase 1/2,11/15/21,"Phase 1/2 date reported APOC3 was reduced by 98% in FCS patients and 96% in MCM patients. Both groups showed similar maximum median reductions in TG of 91% and 90%, respectively, noted November 15, 2021.",6
CYTH,Niemann-Pick Disease Type C,Phase 1,10/18/22,"Phase 1 data showed that treatment overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the central nervous system (CNS), noted October 18, 2022.",6
AVIR,COVID-19,Phase 2,4/25/23,"Fast Track Designation granted on April 25, 2023.",5
ADMP,Emergency treatment of anaphylaxis,Approved,6/15/17,"CRL issued March 27 2015. CRL issued again June 6, 2016. Approval announced June 15, 2017 following third submission.",6
LLY,Primary Sjogren's Syndrome,Phase 2,1/27/22,"Phase 2 study withdrawn noted January 27, 2022.",5
IPSEY,Fibrodysplasia ossificans progressiva (FOP),Approved,8/16/23,"FDA Approved on August 16, 2023.",6
SNSE,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
INTS,Advanced Soft Tissue Sarcoma,Phase 2,9/7/23,"Orphan Drug Designation granted by the FDA noted Septemeber 7, 2023.",5
TRVI,Prurigo Nodularis (PN),Phase 2/3,10/13/23,"Phase 2/3 long-term data also supports the continued effectiveness of Haduvio in reducing itch over 52 weeks for the participants who remained in the study, noted October 13, 2023.",6
GSK,Systemic lupus erythematosus (SLE),Approved,7/21/17,"Approval announced July 21, 2017.",6
PTPI,Erectile Dysfunction,Approved,4/27/12,"Approved April 27, 2012.",6
BCLI,Progressive Multiple Sclerosis (MS),Phase 2,3/24/21,"Phase 2 top-line data released March 24, 2021 - safety primary endpoint met.",6
MRK,Cancer - first-line metastatic melanoma,Phase 3,4/6/18,"Phase 3 trial did not meet primary endpoint - noted April 6, 2018.",4
RDHL,COVID-19 (outpatients),Phase 2/3,1/3/23,"Phase 2 trial met primary endpoint, noted January 3, 2023.",5
ATRA,Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT),Phase 3,11/4/21,"Phase 3 data reported a 50% objective response rate (ORR), noted November 4, 2021.",5
FULC,COVID-19,Phase 3,3/4/21,"Phase 3 trial to be discontinued - March 4, 2021.",4
ACAD,Parkinson’s disease psychosis (PDP),Approved,4/29/16,"Approved April 29, 2016.",6
KPRX,Later-stage Retinitis Pigmentosa,Phase 2,4/27/23,"Phase 2 interim results presented at ARVO reported patient-reported improvement in the ability to perceive a contrast between light and dark at days seven 14 and 29, noted April 27, 2023.",6
JNJ,Plaque psoriasis,Approved,2/27/19,"Approval announced February 27, 2019.",6
BGNE,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
NBIX,Tourette syndrome - juvenile,Phase 2b,12/12/18,"Phase 2 top-line data released December 12, 2018 did not meet primary endpoint.",4
VIRX,Epstein Barr Virus-associated cancers,Phase 2,10/4/23,"Phase 2 trial demonstrated overall and complete response rates of 40%; follow-up from the Phase 1b/2 study demonstrated median duration of response extended to 17.3 months, noted October 4, 2023.",6
BHVN,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
HCM,Solid Tumors,Phase 2,5/25/23,"Additional data from ASCO reported that among evaluable radioactive iodine-refractory differentiated thyroid cancer patients, median PFS was 10.9 months and median OS was not reached, and in endometrial cancer patients, the median PFS was 5.4 months and 12-month OS rate was 71.0%, noted May 25, 2023.",6
BMY,Non-small cell lung cancer (NSCLC),Approved,3/4/22,"Approved March 4, 2022.",6
ASMB,Hepatitis B,Phase 1b,12/19/22,"Phase 1b data reported that in the cohort of 50 mg, six of eight HBV patients on treatment reached lower limit of quantification for HBV DNA by day 21 and patients showed a mean reduction of 3.1 logs in plasma HBV DNA, noted December 19, 2022.",6
GSK,Shingles,Approved,10/20/17,"Approval announced October 20, 2017.",6
AYTU,Attention deficit hyperactivity disorder (ADHD),Approved,6/19/17,"Approval announced June 19, 2017.",6
SRPT,Duchenne muscular dystrophy,Phase 3,10/14/22,"Phase 3 results presented at the World Muscle Society reported that mean dystrophin levels significantly increased from baseline after 48 weeks of casimersen treatment, noted October 14, 2022.",6
PPBT,Squamous cell carcinoma of the head & neck (SCCHN),Phase 1/2,10/3/23,"Phase 1/2 data showed that 2 out of 4 SCCHN patients at the highest dose cohort demonstrated a confirmed partial response, noted October 3, 2023.",6
MRK,"Stage IB, II or IIIA non-small cell lung cancer (NSCLC)",Approved,1/27/23,"Approved January 27, 2023.",6
GRCL,Relapsed/refractory multiple myeloma (RRMM),Phase 1,9/10/23,"Phase 1 updated data at EHA demonstrated 100% minimal residual disease (MRD) negativity and 82.8% MRD negative stringent complete response (sCR) in a predominantly high-risk RRMM population, noted June 10, 2023.",6
GSK,Multiple Myeloma,Phase 3,11/7/22,"Phase 3 trial did not meet its primary endpoint, noted November 7, 2022.",6
BHVN,Amyotrophic lateral sclerosis (ALS),CRL,7/19/19,"CRL issued July 19, 2019.",5
AMRX,Metastatic non-squamous non-small cell lung cancer,Approved,6/14/23,"FDA 505(b)(2) Approval on June 14, 2023.",6
JNJ,Diabetic Kidney Disease,Approved,9/30/19,"FDA Approval announced September 30, 2019.",6
AZN,Non-squamous non-small cell lung cancer (NSCLC),Phase 2,9/11/22,"Phase 2 data to be presented at ESMO demonstrated continued durable activity across patient subtypes, noted September 11, 2022.",6
FATE,Relapsed/refractory Multiple Myeloma,Phase 1,12/12/22,"Phase 1 data presented at ASH reported that one patient in Regimen A achieved a partial response, and one patient achieving a minor response in regimen B, noted December 12, 2022.",6
ABBV,Multiple myeloma,Phase 3,12/13/21,"Phase 3 overall survival results reported that there were 78 (40%) deaths in the treatment arm versus 36 (37%). Median OS was not reached in the treatment or placebo arm among all patients, noted December 13, 2021.",4
PRTA,Psoriatic Arthritis,Phase 1b,9/28/17,"Phase 1b data released September 28, 2017 - insufficient meaningful clinical effect for further development.",4
ADGI,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
BIOR,Ulcerative Colitis (UC),Phase 1a,9/25/23,"IND submitted to FDA, noted September 25, 2023.",5
SRNE,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
MEIP,High-risk Myelodysplastic Syndrome (MDS),Phase 2,5/31/18,"Phase 2 commencement of dosing announced June 14, 2017 - analysis from first stage released May 31, 2018 - trial to expand enrollment.",5
RHHBY,Diffuse Large B-Cell Lymphoma,Approved,4/19/23,"FDA Approved on April 19, 2023.",6
CRVS,Peripheral T-cell lymphoma (PTCL),Phase 1b,6/15/23,"Phase 1/1b antitumor response data presented at the ICML demonstrated the potential of ITK inhibition to provide a novel immunotherapy mechanism of action for cancer therapy, noted June 15, 2023. Company plans to meet with FDA during 3Q 2023 to discuss Phase 3 design.",6
GILD,Acute Myeloid Leukemia (AML),Phase 1/2,8/21/23,"Partial hold on enrollment by FDA, noted August 21, 2023.",5
AZN,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
ABBV,Migraine,Approved,12/23/19,"FDA Approval announced December 23, 2019.",6
EBS,Chikungunya virus,Phase 2,11/22/19,"Phase 2 interim analysis noted 98% of study participants produced a neutralizing antibody response against the chikungunya virus (CHIKV) within seven days after vaccination - November 22, 2019.",6
REGN,Coronavirus COVID-19,Phase 3,9/1/20,"Phase 3 global trial did not meet primary endpoint - September 1, 2020.",4
LABP,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
COLL,Pain,Approved,10/25/15,"Approved October 26, 2015.",6
NVO,Type 2 diabetes,Phase 3,10/3/22,"Phase 3a trial met primary endpoint, noted October 3, 2022.",5
NKTR,Opioid-induced constipation (OIC),Approved,9/16/14,"Approved September 16, 2014.",6
CBAY,Primary biliary cholangitis (PBC),Phase 3,9/7/23,"Phase 3 registration trial achieved the primary and all key secondary endpoints, noted on September 7, 2023.",6
UTHR,Pulmonary Arterial Hypertension,Phase 2,7/10/17,"Phase 2 data released July 10, 2017. Primary endpoint met. Secondary missed.",5
NVS,"Non-small cell lung cancer (NSCLC), adjuvant",Phase 3,8/15/22,"Phase 3 trial did not meet the primary endpoint, noted August 15, 2022.",6
CGTX,Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (AMD),Phase 1,7/11/23,"Phase 2 trial dosing initiated, noted July 11, 2023.",5
MESO,ARDS due to COVID-19,Phase 3,4/29/21,"Remestemcel-L reduced mortality through day 60 by 46% in the pre-specified group below age 65, but not in patients 65 or older.",6
GMDA,Follicular and diffuse large B cell lymphomas,Phase 1,10/17/23,"Phase 1 data showed promising early evidence of anti-tumor activity in patients with relapsed/refractory B cell non-Hodgkin lymphoma, noted October 16, 2023.",6
SNY,Atopic dermatitis,Approved,3/28/17,"Approved under priority review - March 28, 2017.",6
LEGN,Molluscum Contagiosum,Phase 3,9/23/21,"Phase 3 safety data showed TEAEs reported in greater than 5% of subjects in the SB206 treated groups were all at the application site (pain, erythema, pruritis, exfoliation, and dermatitis) with the high majority of these TEAEs being mild or moderate by severity, noted September 23, 2021.",6
YMAB,Neuroblastoma,Approved,11/25/20,"FDA approval announced November 25, 2020.",6
OTIC,Acute otitis externa,Approved,3/2/18,"Approval announced March 2, 2018.",6
VTRS,Trastuzumab biosimilar,Approved,12/1/17,"Approval announced December 1, 2017.",6
RHHBY,First line unfit AML,Approved,11/21/18,"FDA approval announced November 21, 2018.",6
JAZZ,Multiple sclerosis (MS)-related spasticity,Phase 3,6/28/22,"Phase 3 trial did not meet the primary endpoint, noted June 28, 2022.",6
EWTX,Becker muscular dystrophy (BMD),Phase 1b,6/26/23,"Phase 1b 12-month study supported the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies, associated with administration, has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies, noted June 26, 2023.",6
AXGN,Severed peripheral nerves,Phase 3,5/4/22,"Phase 3 trial met primary endpoint, noted May 4, 2022.",5
VINC,Hematologic malignancies,Phase 1,9/14/23,"IND cleared August 22, 2023. Phase 1 study dosing commenced, noted on September 14, 2023.",5
NVS,Post-acute myocardial infarction,Phase 3,10/26/21,"Phase 3 did not meet primary endpoint, trial will not continue to regulatory submission, noted October 26, 2021.",4
KURA,Head and Neck Squamous Cell Carcinoma,Phase 1/2,10/26/22,"Phase 1/2 data reported that one patient experienced experienced an 81% reduction in target lesions after one cycle, noted October 26, 2022.",6
VCNX,Resectable Stage III melanoma,Phase 1,11/10/22,"Phase 1 data presented at SITC showed that 100% of patients who received the triple combination were recurrence free at 24 months. This contrasted with recurrence free survival of less than 40% in patients who received the dual combination of pepinemab and nivolumab or pepinemab and ipilimumab, noted November 10, 2022.",6
NKTR,Bronchiectasis,Phase 3,4/5/17,"Phase 3 second trial did not meet endpoint - April 5, 2017.",4
BNTX,COVID-19 Vaccine (Omicron Booster),Approved,9/11/23,"FDA booster approved on September 11, 2023.",6
GALT,Non-alcoholic steatohepatitis (NASH) with cirrhosis,Phase 2/3,10/3/23,"4th DSMB concluded that NAVIGATE can continue as designed, without modifications, noted October 3, 2023.",5
HOOK,HPV16+ Squamous cell head and neck cancers (HNSCC),Phase 1,11/3/23,"Phase 1 biomarker results demonstrated a robust increase in tumor-specific CD8+ T cells in all evaluable patients with HPV16+ head and neck cancers, noted November 3, 2023. Randomized trial planned for 2024.",6
RYTM,"POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders",Phase 2,9/22/21,"Phase 2 data showed across all SRC1 deficiency patients, the mean overall weight loss among adults was -4.0%, and the mean overall BMI-Z score reduction among patients younger than 18 years was -0.21. Across all SH2B1 deficiency patients, the mean overall weight loss among adults was -3.1%, and the mean overall BMI-Z score reduction among patients younger than 18 years was -0.15, noted September 22, 2021.",6
MRNA,Respiratory syncytial virus (RSV) vaccine,BLA Filing,7/5/23,"BLA rolling submission, noted July 5, 2023.",5
CYCN,Sickle Cell disease,Phase 2,10/14/20,"Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.",4
SRPT,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
SCYX,"Recurrent Vulvovaginal candidiasis (rVVC), Vaginal yeast infections",Approved,12/1/22,"FDA Approved on December 1, 2022.",6
GILD,Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia,Phase 1/2,6/5/23,"Additional Phase 1/2 data presented at ASCO reported that median (95% CI) OS was 14.2 mo (3.2-26.0) for pts with prior blina and NR (18.6-NE) for pts without prior blina; Grade ≥3 TRAEs occurred in 80% and 97% of pts, respectively, noted June 5, 2023.",6
CPRX,Pediatric Lambert-Eaton myasthenic syndrome (LEMS),Approved,9/29/22,"sNDA approved September 29, 2022.",6
CHRS,Psoriasis,Approved,12/20/21,"Approved December 20, 2021.",6
BGNE,Squamous non-small cell lung cancer (NSCLC),Phase 3,1/21/20,Phase 3 trial met primary endpoint of PFS at interim analyses.,6
SGEN,Cancer - ALCANZA trial for relapsed CD30-positive cutaneous T-cell lymphoma,Approved,11/9/17,"Approval announced November 9, 2017.",6
ADVM,Wet age-related macular degeneration (AMD),Phase 2,9/26/23,"Phase 2 14-week data reported that has been generally well-tolerated, with 90% of subjects were still receiving corticosteroid prophylactic taper, noted September 26, 2023.",6
REGN,Fibrodysplasia ossificans progressiva (FOP),Phase 2,1/9/20,Phase 2 trial did not meet primary endpoint p=0.07. Patient-reported flare-ups were reduced by 50% (nominal p=0.03).,4
GSK,HIV,Approved,8/6/20,"FDA approval announced August 6, 2020.",6
JNJ,Plaque psoriasis,Phase 2b,7/5/23,"Phase 2b trial met all primary and secondary efficacy endpoints, noted July 5, 2023.",5
RANI,Neuroendocrine tumors (NETs) and acromegaly,Phase 1,11/1/23,"Program discontinued, noted November 1, 2023.",4
HRMY,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
RHHBY,Influenza,Approved,10/24/18,"FDA Approval announced October 24, 2018.",6
ACRS,Seborrheic keratosis (SK),Approved,12/15/17,"Approval announced December 15, 2017.",6
ZLAB,Generalized Myasthenia Gravis (gMG),Approved,6/20/23,"FDA Approved on June 20, 2023.",6
IPSEY,Alagille syndrome (ALGS),Approved,6/13/23,"FDA Approved on June 13, 2023.",6
VNDA,Pneumonia associated with COVID-19,Phase 3,8/18/20,"Phase 3 data August 18, 2020 noted statistically significant improvement by Day 7. However, improvement was not statistically significant at Day 28.",5
VTRS,Neulasta biosimilar,Approved,6/4/18,"Approval announced June 4, 2018.",6
CLNN,Multiple Sclerosis,Phase 2,4/25/23,"Phase 2 topline data presentated at AAN reported that the primary BC-LCLA outcome LS-mean difference was 3.13 and 2 of 3 key secondary outcomes were significant, noted April 25, 2023.",6
AFMD,Hodgkin Lymphoma - Cancer,Phase 1b,2/1/18,"Phase 1b preliminary data released February 1, 2018 - Objective response rate (ORR) of 89% (8/9), 4/9 CRs and 4/9 PRs. 3-month data due mid-2018.",6
RNAZ,Metastatic solid tumors,Phase 1a,10/24/23,"Phase 0 preliminary data showed that radioactivity consistent with accumulation of TTX-MC138 was detected by noninvasive imaging in the regions of the metastatic lesions previously identified by fluorodeoxyglucose (FDG)/positron emission tomography (PET) (FDG/PET), noted October 24, 2023.",6
SAGE,Parkinson Disease,Phase 2a,3/15/22,"Phase 2a data reported that sustained effects and improving trends were seen out to Day 28, noted March 15, 2022.",6
ABBV,Parkinson's disease,CRL,3/22/23,"CRL issued by FDA on March 22, 2023.",5
BGNE,Gastric or gastroesophageal junction (G/GEJ) adenocarcinoma,Phase 3,10/21/23,"Phase 3 data presented at ESMO reported that OS in the TIS arm was significantly improved compared with the PBO arm in the ITT population (median OS: 15.0 mo vs 12.9 mo, respectively; HR=0.80 [95% CI: 0.70, 0.92]; 1-sided P=0.0011), noted October 21, 2023.",6
BHVN,Generalized anxiety disorder (GAD),Phase 3,2/10/20,Phase 3 trial did not meet primary endpoint.,4
BIIB,Alzheimer’s disease,Phase 3,3/21/19,Phase 3 trial discontinued due to futility - unlikely to meet primary endpoint upon completion.,4
NVS,HR + Metastatic breast cancer (MBC),Phase 3,8/23/18,"Phase 3 trial met primary endpoint - August 23, 2018 with data to be presented at an upcoming medical congress.",6
BMY,"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
EDIT,Transfusion-Dependent Beta Thalassemia (TDT),Phase 1/2,6/9/23,"Phase 1/2 first patient demonstrated successful neutrophil and platelet engraftment, and, at one and a half months post-infusion, the patient's response resembles that of the first four RUBY patients, noted June 9, 2023.",6
KOD,Retinal vein occlusion (RVO),Phase 3,10/13/23,"Phase 3 one year primary data from the Retina Society meeting reported that Tarcocimab treated patients had a ~30% higher chance of not requiring any additional doses versus aflibercep, noted October 13, 2023.",6
NBIX,Huntington's disease,Approved,8/18/23,"Approved August 18, 2023.",6
ACXP,C. difficile Infection,Phase 2b,11/2/23,"Phase 2a and 2b data reported an observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96%, noted November 2, 2023.",6
ELDN,Amyotrophic Lateral Sclerosis,Phase 2a,5/31/22,"Phase 2a topline data showed that treatment met the primary endpoints of safety and tolerability. Adverse events were equally distributed across dose levels, noted May 31, 2022.",6
GMAB,B-cell Lymphoma (DLBCL),Approved,5/22/23,"Approved May 19, 2023.",6
JAZZ,Tuberous Sclerosis Complex,Approved,8/3/20,"FDA approval announced August 3, 2020.",6
FUSN,Solid tumors,Phase 1,6/26/23,"Phase 1 pre-administration data reported a favorable gain in tumor lesion uptake versus normal tissue, noted June 14, 2022.",6
PFE,Bladder cancer,Approved,6/30/20,"FDA approval announced June 30, 2020.",6
AZN,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,Metastatic castrate-resistant prostate cancer (mCRPC),Phase 1b,10/31/23,"Phase 1b initial data reported that efficacy was greater at higher doses (doses ≥0.75 mg target dose) where PSA50 was 59% (n=44) and RECIST objective response rate was 41% (n=37). The safety profile was clinically manageable, with CRS that was generally low grade and primarily in cycle 1, noted October 31, 2023.",5
GILD,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
VRTX,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
TOVX,Head and Neck Squamous Cell Carcinoma,Phase 1,10/23/23,"Phase 1 data shared at ESMO 23 showed enhanced patient survival, correlating with VCN-01 mediated increases in the CPS score, a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies, noted October 23, 2023.",6
LIAN,Obstructive hypertrophic cardiomyopathy (oHCM),Phase 3,8/28/23,"Phase 3 data presented at ESC reported that treatment demonstrated improvement in Valsalva LVOT peak gradient, LVOT obstruction, clinical symptoms, health status, cardiac biomarkers, and cardiac structure in Chinese oHCM patients, noted August 28, 2023.",6
NERV,Major Depressive Disorder,Phase 2b,5/13/19,"Phase 2b data May 13, 2019 noted p-value was 0.083.",5
ABBV,Functional Constipation in pediatric patients aged 6-17,Approved,6/12/23,"FDA Approved on June 12, 2023.",6
AZN,Third-line ovarian cancer,Phase 3,12/20/18,"Phase 3 data released December 20, 2018 met primary and secondary endpoints.",6
ZLAB,Gastric cancer,Phase 1/2,6/1/23,"Phase 1/2 data reported that the most common treatment-related adverse events (TRAEs) were gastrointestinal disorders, including nausea (11/19, 57.9%), vomiting (6/19, 31.6%), and abdominal pain (3/19, 15.8%), with no Grade ≥ 4 TRAEs were reported, noted June 1, 2023.",6
EBS,Anthrax,PDUFA,3/25/15,"Approved March 25, 2015.",6
EGRX,"Breast, non-small cell lung, prostate gastric adenocarcinoma, and head and neck cancer",Approved,12/24/15,"Approved December 24, 2015.",6
STOK,Dravet Syndrome,Phase 1/2,7/25/23,"Phase 1/2 data presented showed that patients treated with 2 or 3 initial doses experienced substantial and sustained reductions in convulsive seizure frequency; Median reductions at 3 months after last dose of 80% and 89% at 6 months after last dose, compared to baseline, noted July 25, 2023.",6
MRNA,"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
FREQ,Sensorineural hearing loss progression (SNHL),Phase 1b,2/13/23,"Phase 1b dosing completed, development to be discontinued, noted February 13, 2023.",4
MDGL,Non-Alcoholic Fatty Liver Disease,Phase 3,6/27/22,"Phase 3 52-week primary data analysis reported the treatment was safe and well tolerated and provided significant reductions in liver fat, noted June 27, 2022.",6
HOTH,EGFRI-induced skin toxicity,Phase 2a,6/14/23,"Received written approval from the Institutional Review Board (IRB) to proceed with its First-in-Human (FIH) Phase 2a clinical trial of HT-001, noted June 14, 2024.",6
CYCC,Advanced cancers,Phase 1/2,10/26/22,"Phase 1/2 dose escalation data reported that of the 18 patients evaluable for response, two out of three T cell lymphoma patients treated achieved partial response and 11 out of 15 patients with various solid tumors achieved stable disease, noted October 26, 2022.",6
PFE,"Influenza and COVID-19, Omicron BA.4/BA.5-adapted bivalent",Phase 1/2,10/26/23,"Phase 1/2 data demonstrated robust immune responses to influenza A, influenza B, and SARS-CoV-2 strains, noted October 26, 2023.",6
CRBP,Cystic Fibrosis,Phase 2b,10/6/20,"Phase 2b trial did not meet primary endpoint - October 6, 2020.",4
ICPT,Primary Biliary Cholangitis (PBC),Phase 2a,6/23/23,"Phase 2a data presented at EASL showed that the Combination was well-tolerated with lowest rates of pruritus observed in OCA 5-10 mg + bezafibrate 400 mg treatment arm, noted June 23, 2023.",6
XERS,Exercise-Induced Hypoglycemia,Phase 2b,6/15/20,"Phase 2b trial met primary endpoints - June 15, 2020.",6
AMLX,Amyotrophic lateral sclerosis (ALS),Approved,9/29/22,"Approved September 29, 2022.",6
CMPS,Treatment Resistant Depression (TRD),Phase 2b,5/26/23,"Phase 2b more than half of the patients demonstrated remission of depression at 18 months, after a single dose of 25mg investigational COMP360 psilocybin with psychological support, noted May 26, 2023.",6
NVS,Rheumatoid Arthritis,Approved,10/31/18,"FDA approval announced October 31, 2018.",6
ZVRA,ADHD,Approved,3/2/21,"FDA approval announced March 2, 2021.",6
AYLA,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
LLY,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
PFE,Migraine,Approved,3/10/23,"Approved March 10, 2023.",6
EXAI,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
BHC,Psoriasis,Approved,2/15/17,"Approved February 15, 2017.",6
MDGL,Heterozygous familial hypercholesterolemia (HeFH),Phase 2a,4/3/18,"Phase 2 top-line data released February 8, 2018 - primary endpoint met.",6
GTHX,Urothelial carcinoma,Phase 2,8/2/23,"Phase 2 PFS data reported that PFS was similar between patients receiving trilaciclib prior to gemcitabine/platinum + avelumab and patients receiving gemcitabine/platinum + avelumab alone (median PFS=6.0 months and 6.1 months), noted August 2, 2023.",5
OBSV,In Vitro Fertilization (IVF),Phase 3,2/26/18,"Phase 3 data released February 26,2018 - primary endpoint met. Improvement shown in ET D5 subgroup but not in ET D3.",6
RIGL,Warm Autoimmune hemolytic anemia (WAIHA),Phase 3,12/12/22,"Phase 3 data presented at ASH reported that the ORR was 48%, noted December 12, 2022.",6
TNGX,Solid Tumors,Phase 1/2,7/10/23,"Phase 1/2 dosing initiated, noted July 10, 2023.",5
ZYME,Solid Tumors,Phase 1,4/18/23,"Additional data reported that anti-tumor activity was observed in breast cancer and gastric cancer PDXs representing a range of HER2 expression, noted April 18, 2023.",6
FUSN,Solid tumors,Phase 1,6/26/23,"Phase 1 multi-dose safety and imaging data presented at SNMMI with Cold/Hot dosing regimen demonstrated potential to improve therapeutic index and safety profile, noted June 26, 2023.",6
ALPN,"Lupus nephritis, IgA nephropathy",Phase 1/2,11/2/23,"Phase 1b/2a data presented at ASN reported that treatment reduced UPCR by greater than 50% in IgA nephropathy, noted November 2, 2023.",6
GALT,Melanoma,Phase 1b,9/20/18,"Phase 1b data released September 20, 2019. ORR 50%.",5
IPSEY,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
KZR,Lupus nephritis (LN),Phase 2,11/3/22,"Phase 2 complete results reported that zetomipzomib added to stable background therapy reduced proteinuria by 50% or greater (ORR) in 11 of 17 patients (64.7%) reaching end of treatment at Week 25, noted November 3, 2022.",6
BPMC,RET-mutant medullary thyroid cancer,Approved,12/1/20,"FDA approval announced December 1, 2020.",6
HALO,Subcutaneous treatment for patients with primary immunodeficiency (PI),Approved,9/12/14,"Approved September 12, 2014.",6
EOLS,Glabellar lines,Approved,2/1/19,"FDA Approval announced February 1, 2019.",6
CCXI,Associated vasculitis (AAV),Approved,10/8/21,"FDA approval announced October 8, 2021.",6
IPHA,COVID-19,Phase 2,7/6/21,"Phase 2 trial did not meet primary endpoints - July 6, 2021.",4
MRNA,COVID-19 vaccine - pediatric,Phase 2/3,3/23/22,"Phase 2/3 data from children 6 months to under 6 years met primary endpoint, noted March 23, 2022.",5
IMVIQ,Metastatic Bladder and Micro-Satellite Instability High (MSI-H) Solid Tumors,Phase 2,9/30/19,Phase 2 data at ESMO 2019 noted 2/23 PRs.,5
MRNA,Seasonal influenza,Phase 3,9/13/23,"Phase 3 trial met all co-primary endpoints, noted September 13, 2023.",5
MREO,Severe alpha-1 antitrypsin deficiency (AATD),Phase 2,5/9/22,Phase 2 study demonstrated to be statistically significant inhibition of blood neutrophil elastase activity of up to 90% in patients in both high and low dose groups throughout the 12-week dosing period.,6
BIIB,Amyotrophic lateral sclerosis (SOD1-ALS),Approved,4/25/23,"FDA granted accelerated approval on April 25, 2023.",6
AZN,Advanced ER-positive Breast Cancer,Phase 2,12/8/22,"Phase 2 presentation at SABCS reported that treatment had a statistically significant and clinically meaningful improvement in progression-free survival (PFS) at both 75mg and 150mg dose levels, noted December 8, 2022.",6
PFE,C. difficile Infection,Phase 3,3/1/22,"Phase 3 trial did not meet primary endpoint, noted March 1, 2022.",6
ABBV,Diffuse large B-cell lymphoma (DLBCL),Phase 3,7/11/18,"Phase 3 trial did not meet primary endpoint - noted July 11, 2018.",4
BMRN,(MPS IVA) Morquio A Syndrome,Approved,2/14/14,"Approved February 14, 2014.",6
ABBV,Chronic migraine,Approved,9/28/21,"Approved September 28, 2021.",6
IFRX,COVID-19,Phase 3,4/4/23,"FDA granted an Emergency Use Authorization (EUA), noted April 4, 2023.",5
REGN,Respiratory syncytial virus (RSV),Phase 3,8/14/17,"Phase 3 data released August 14, 2017 - primary endpoint not met.",4
GSK,Multiple myeloma,Approved,8/5/20,"FDA approval granted August 5, 2020.",6
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 63.9% (n=180). Additionally the change in GSP3 from baseline to EOT was -145.9mg. Overall, 82% of patients who were randomized to the dose in the study completed the full six weeks of treatment. Nine (5.0%) subjects who received the dose discontinued due to a TEAE. No treatment-related Serious Adverse Events (SAEs) were reported, noted October 7, 2021.",6
OYST,Dry eye disease,Phase 2,1/12/20,"Phase 2 top-line data met primary endpoint - January 12, 2020.",6
KRYS,Alpha-1 antitrypsin deficiency (AATD),Phase 1a,9/5/23,"FDA granted Orphan Drug Designation, noted September 5, 2023.",5
AXSM,Agitation in patients with Alzheimer’s disease (AD),Phase 3,10/24/23,"Phase 3 results shared at the CTAD demonstrated rapid and sustained clinical response in patients with ADA during the open-label AXS-05 treatment. During the placebo-controlled randomized withdrawal phase, AXS-05 statistically significantly delayed the time to relapse of agitation symptoms as compared to placebo, noted October 24, 2023.",6
RLMD,Major depressive disorder (MDD),Phase 3,2/23/22,"Phase 3 top-line results showed that all three doses (25 mg, 75 mg, and 150 mg, the therapeutic, supratherapeutic and maximum tolerated doses, respectively) tested in recreational drug users, demonstrated a substantial (30+ points) and statistically significant difference vs. intravenous ketamine 0.5 mg/kg over 40 minutes, and were statistically equivalent to placebo, noted February 23, 2022.",6
TBPH,Asthma,Approved,4/30/15,"Approved April 30, 2015.",6
GILD,Triple-negative breast cancer,Approved,4/22/20,"FDA Approval announced April 22, 2020.",6
AZN,Early Alzheimer's disease,Phase 3,6/11/18,Announced discontinuation of trial due to futility.,4
SUPN,ADHD,Phase 3,12/22/20,"Phase 3 trial met primary endpoint - December 22, 2020.",6
SUPN,Attention Deficit Hyperactivity Disorder (ADHD),Phase 3,2/25/20,Phase 3 trial did not meet primary endpoint.,4
AGIO,Pyruvate kinase deficiency,Approved,2/17/22,"Approved February 17, 2022.",6
CING,Healthy volunteers in fed and fasted states,Phase 1,2/23/23,"Phase 1 data demonstrated that CTx-1301 can be taken with or without food, noted February 23, 2023.",6
TBPH,COPD,Approved,11/9/18,"FDA Approval announced November 9, 2018.",6
ABBV,Uterine Fibroids,Approved,5/29/20,"FDA approval announced May 29, 2020.",6
BDRX,Diffuse Midline Glioma (DMG),Phase 1,7/6/23,"Phase 1 trial reported a median overall survival (OS) of 26.1 month, noted July 6, 2023.",5
AMGN,Multiple Myeloma,Approved,10/1/18,"FDA approval announced October 1, 2018.",6
XBIO,Anemia in patients with chronic kidney disease (CKD),Phase 3,12/9/20,Phase 3 data using PolyXen technology reported by partner - non-inferior to Aranesp (darbepoetin alfa) - Russia trial.,6
AMGN,KRAS G12C-mutated solid tumors,Phase 1b,10/7/21,"Phase 1b results displayed that patients with CRC who were KRASG12C inhibitor naïve, 9% achieved PR (1 of 11), and 82% achieved disease control (9 of 11). In patients who were previously treated with a KRASG12C inhibitor, 14% achieved PR (1 of 7), and 86% achieved disease control (6 of 7). In patients with NSCLC who were KRASG12C inhibitor naïve, 20% achieved PR (3 of 15) and 87% achieved disease control (13 of 15). In patients who were previously treated with a KRASG12C inhibitor, 67% achieved disease control (2 of 3), noted October 7, 2021.",6
ACRX,Post-operative pain following open abdominal surgery and hip or knee replacement surgery,Phase 3,8/1/17,"Phase 2 data released August 1, 2017.",5
BLRX,Solid tumors,Phase 1/2,1/24/22,"Phase 1/2a enrollment completed January 24, 2022.",5
EDSA,"COVID-19, Acute Respiratory Distress Syndrome (ARDS)",Phase 2/3,10/19/21,"Phase 2/3 additional results demonstrated the DSMB noted another mortality benefit in 136 hospitalized COVID-19 patients receiving supplemental oxygen (28-day mortality rate of 8.2% (5/61) in the EB05 + SOC arm versus 12.0% (9/75) in the placebo + SOC arm, Hazard Ratio (HR): 1.52 placebo vs. EB05, n=136). Phase 3 portion of the study to focus on critically ill population, noted October 19, 2021.",5
SGEN,Hodgkin lymphoma and Anaplastic large cell lymphoma,Approved,8/19/11,"Approval announced August 19, 2011.",6
SNY,Cutaneous squamous cell carcinoma,Approved,9/28/18,"FDA Approval announced September 28, 2018.",6
AMGN,Multiple Myeloma,Approved,1/5/18,"sBLA approval announced January 5, 2017.",6
SNSE,Squamous Cell Carcinoma of the Head and Neck,Phase 1/2,8/4/21,"Development has been discontinued - noted August 4, 2021.",4
BMY,Resectable Stage III melanoma,Phase 1,11/10/22,"Phase 1 data presented at SITC showed that 100% of patients who received the triple combination were recurrence free at 24 months. This contrasted with recurrence free survival of less than 40% in patients who received the dual combination of pepinemab and nivolumab or pepinemab and ipilimumab, noted November 10, 2022.",6
SYBX,Homocystinuria (HCU),Phase 1,3/12/23,"Phase 1 data presented at SIMD reported that no serious adverse events or deaths related to SYNB1618 or SYNB1934, noted March 19, 2023.",6
IRON,Myelofibrosis (MF) patients with anemia,Phase 1/2,11/2/23,"Phase 1b/2 data from abstracts reported dose-dependent reductions in serum hepcidin-25 and increases in serum iron levels, noted November 2, 2023.",6
MESO,Steroid-refractory acute graft versus host disease (aGVHD) in children,CRL,8/3/23,"Additional CRL announced August 3, 2023.",5
MOR,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
SLNO,Prader-Willi syndrome (PWS),Phase 3,9/8/21,"Phase 3 extension study data showed mean (SE) improvement in hyperphagia by a decrease in the HQ-CT total score, of -9.9 (0.77), was highly significant (p<0.0001) after receiving DCCR for 52 weeks, noted September 8, 2021.",6
PRAX,Healthy volunteers,Phase 1,12/4/22,"Phase 1 data reported that there were no clinically significant safety findings in vital signs, clinical laboratory results, physical exams, ECGs, or C-SSRS. TEAEs were mostly mild or moderate, noted December 4, 2022.",5
MOLN,COVID-19,Phase 2/3,1/10/22,"Phase 2b/3 part A met primary endpoint, noted January 10, 2022.",5
TBPH,Gastroparesis,Phase 2b,8/2/17,"Phase 2b data released August 2, 2017. Primary endpoint not met.",4
MRK,Human Papilloma virus vaccine,Approved,10/9/18,"Approval announced October 5, 2018.",6
CLDX,Eosinophilic esophagitis (EoE),Phase 2,7/6/23,"Phase 2 dosing initiated, noted July 6, 2023.",5
LLY,COVID-19 antibody,Approved,2/11/22,"EUA granted by FDA February 11, 2022.",5
DRRX,Post-operative pain relief,CRL,2/12/14,"CRL issued February 12, 2014.",5
REGN,Eosinophilic esophagitis (EoE),Approved,5/20/22,"Approved May 20, 2022.",6
JNJ,Ulcerative colitis,Approved,10/21/19,"FDA Approval announced October 21, 2019.",6
CNTX,"ER+, HER2-, ESR1-mutated Breast Cancer",Approved,1/31/23,"Approved January 31, 2023.",6
AMGN,Plaque psoriasis,Approved,12/20/21,"Approved December 20, 2021.",6
XLO,Solid Tumors,Phase 1,5/25/23,"Phase 1 preliminary anti-tumor activity observed in a patient with PD-L1 negative advanced non-small cell lung cancer, favorable preliminary safety profile for XTX101 observed at the recommended Phase 2 dose of 150 mg once every six weeks, noted May 25, 2023.",6
ZLAB,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
PFE,Juvenile idiopathic arthritis (JIA),Phase 3,9/28/20,"FDA approval announced September 28, 2020.",6
MRK,"Resectable Stage II, IIIA or IIIB Non-Small Cell Lung Cancer (NSCLC)",Approved,10/16/23,"Approved on October 16, 2023.",6
JAZZ,Rett syndrome,Phase 3,11/12/20,"Phase 3 trial stopped due to COVID-19 - November 12, 2020.",4
ABBV,Multiple Myeloma,Phase 1/2,12/13/21,"Phase 1/2 safety and preliminary efficacy data reported that treatment of patients with t(11;14) RRMM using VenDd and those with RRMM with VenDvd demonstrated a tolerable safety profile and an overall response rate (ORR) of 95.8% and 91.7%, respectively, noted December 13, 2021.",6
INCY,Non-small cell lung cancer,Approved,5/6/20,"FDA Approval announced May 6, 2020.",6
INVA,Chronic obstructive pulmonary disease (COPD),Approved,9/18/17,"Approval announced September 18, 2017.",6
TFFP,Invasive Pulmonary Aspergillosis (IPA),Phase 2,11/1/22,"Phase 2 trial data showed that second patient treated to clinically benefit from administration in compassionate use program, noted November 1, 2022.",6
LLY,Patients evaluated for Alzheimer's Disease,Approved,5/28/20,"FDA Approval announced May 28, 2020.",6
PSTX,Multiple myeloma,Phase 2,12/13/21,"Phase 2 results showed that dosage was well tolerated and demonstrated strong anti-tumor activity in advanced, late line R/R MM patients, with an overall response rate (ORR) of 78%, a VGPR/sCR rate of 43% and 100% overall survival at the time of the data cutoff, noted December 13, 2021.",6
SAGE,Major depressive disorder (MDD),CRL,8/4/23,"CRL issued August 4, 2023.",5
BTAI,Agitation associated with bipolar I or II disorder or schizophrenia,Phase 3,5/25/23,"Phase 3 part 1 topline data clinically meaningful efficacy results were observed with half (60mcg) of the lowest approved dose of IGALMI. In addition, greater than 50% PEC response rate was achieved, with responder rate dose-proportionally consistent with those observed in SERENITY I and II trials. Although the primary efficacy endpoint was not statistically significant at 2 hours (p=0.077), BXCL501 separated from placebo at 4 hours (p=0.049), noted May 25, 2023.",6
ABEO,Sanfilippo syndrome type B (MPS IIIB),Phase 1/2,2/12/21,"Phase 1/2 updated data announced February 12, 2021.",5
MCRB,"Gastrointestinal infections, bacteremia and graft versus host disease (GvHD)",Phase 1b,5/9/23,"Phase 1 initial safety and PK data from cohort 1 showed the successful engraftment of SER-155 bacterial strains and a substantial reduction in the cumulative incidence of pathogen domination, a biomarker associated with the risk of serious enteric infections and bloodstream infections, as well as GvHD, noted on May 9, 2023",6
SPRO,Complicated urinary tract infections (cUTI),CRL,6/27/22,"CRL announced June 27, 2022.",5
VRTX,Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.,CRL,2/5/16,"CRL issued February 5, 2016.",5
ABBV,Maintenance Treatment of Schizophrenia,Approved,11/13/17,"Approval announced November 13, 2017.",6
LGND,Relapsed Multiple Myeloma,Approved,1/17/18,"sNDA approved January 17, 2018.",6
REGN,Chronic Spontaneous Urticaria (CSU),Phase 3,2/18/22,"Phase 3 trial did not meet the primary endpoint, noted February 18, 2022.",6
GILD,Covid-19,Approved,7/14/23,"Approved July 14, 2023.",6
AGTC,Achromatopsia,Phase 1/2,6/24/21,"Phase 1/2 12-month data presented June 24, 2021. Company noted at this time point consistent evidence not shown of biologic activity, patient-reported anecdotes continue to be encouraging.",6
NERV,Major Depressive Disorder,Phase 2b,12/18/19,"Phase 2b data failed to meet endpoints - December 18, 2019. Development to be discontinued in MDD.",4
NVS,Relapsing Multiple Sclerosis,Phase 3,4/20/23,"Phase 3 5-year data reported that more than 80% of patients remained free of six-month CDW, noted April 20, 2023.",5
GLYC,Vaso-occlusive crisis of sickle cell disease,Phase 3,8/2/19,"Phase 3 data failed to meet endpoints - August 2, 2019.",4
PFE,Serious Bacterial Infection,Phase 3,6/1/23,"Phase 3 data reported that 5/12 (41.7%) of the ATM-AVI ± MTZ patients with infections due to confirmed MBL-producing Gram-negative bacteria were cured at TOC versus 0/3 (0%) of those on best available therapy (BAT), noted June 1, 2023.",5
PBYI,Third-line HER2-positive metastatic breast cancer,Approved,2/26/20,"FDA Approval announced February 26, 2020.",6
RNA,Myotonic dystrophy type 1 (DM1),Phase 1/2,9/9/23,"Phase 1/2 topline data analysis presented at the Myotonic Dystrophy Foundation Annual Conference reported directional improvements in myotonia (vHOT) as early as 6 weeks after dosing with a sustained effect at Month 6, noted September 9, 2023.",6
NVS,Solid tumors in adult and pediatric patients,Approved,6/22/22,"Approved June 22, 2022.",6
ARGX,Acute myeloid leukemia (AML),Phase 2,1/8/21,"Phase 2 interim data released January 8, 2021. Complete remission (CR) rate 27% (14/52) and composite complete remission (CRc), including CRs with incomplete hematologic recovery 40% (21/52).",5
ABBV,Ulcerative Colitis,Approved,3/16/22,"Approved March 16, 2022.",6
BNTX,"Influenza and COVID-19, Omicron BA.4/BA.5-adapted bivalent",Phase 1/2,10/26/23,"Phase 1/2 data demonstrated robust immune responses to influenza A, influenza B, and SARS-CoV-2 strains, noted October 26, 2023.",6
PFE,Atopic dermatitis in adolescents,Approved,2/10/23,"Approved February 10, 2023.",6
ROIV,Atopic Dermatitis,Phase 3,3/15/23,"Phase 3 study met primary of Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) and all secondary endpoints, noted March 15, 2023.",5
MIRM,Progressive Familial Intrahepatic Cholestasis (PFIC),Phase 3,10/24/22,"Phase 3 top-line data reported that trial met primary endpoint, noted October 24, 2022.",5
IKT,Parkinson’s disease,Phase 2,10/16/23,"Phase 2 update showed that at the end of study timepoint, participants administered the 200 mg dose had a combined Part 2 and Part 3 score that was lower by an average of -8.7 points. By contrast, the combined placebo score increased by an average of +1.7 points, a -10.4 point spread between actively treated versus placebo participants, noted October 16, 2023.",6
NVS,Relapsing multiple sclerosis,Approved,8/20/20,"FDA approval announced August 20, 2020.",6
JAGX,Chemotherapy-induced diarrhea (CID) in dogs,Approved,12/21/21,"FDA conditionally approved on December 21, 2021.",6
MBRX,Soft tissue sarcoma lung metastases,Phase 1/2,7/28/22,"Preliminary Phase 1b data reported that treatment demonstrated clinical activity, defined as stable disease or better, noted July 28, 2022.",6
BMY,Small cell lung cancer (SCLC),Approved,8/17/18,"FDA approval announced August 17, 2018.",6
AZN,Castration-Resistant Prostate Cancer,Approved,5/19/20,"FDA Approval announced May 19, 2020.",6
MGTX,X-linked retinitis pigmentosa (XLRP),Phase 1/2,10/1/22,"Full Phase 1/2 data presented at AAO reported an improvement in mean retinal sensitivity in the treated eye compared to untreated eyes in the randomized concurrent control arm, noted October 1, 2022.",6
RFL,Acute myeloid leukemia (AML),Phase 3,10/28/21,"Phase 3 trial to be stopped due to lack of efficacy based on recommendation from independent data monitoring committee, noted October 28, 2021.",4
PRAX,Essential tremor,Phase 2,8/8/23,"Phase 2 patients dosed with ulixacaltamide up to 14 weeks showed maintained or improved efficacy results, as measured by mean changes in the modified Activities of Daily Living 11 (mADL111). Patients withdrawing from ulixacaltamide to placebo experienced worsening in mADL11, noted August 8, 2023.",6
MIST,Paroxysmal supraventricular tachycardia (PSVT),Phase 3,11/15/21,"Phase 3 top-line data showed that the nasal dose significantly decreased heart rate during supraventricular tachycardia (SVT) episodes and independent of conversion to sinus rhythm (SR). Heart rate changes occurred within 5 minutes and were sustained for 60 minutes, noted November 15, 2021.",6
BBIO,Achondroplasia,Phase 2,6/20/23,"Phase 2 cohort 5 data reported at ENDO showed that at the Cohort 5 dose level resulted in a significant and robust increase in AHV compared to BL, with a change of +3.38cm/year, noted June 20, 2023.",6
BMRN,Batten Disease,Approved,4/27/17,"PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.",6
BPMC,Gastrointestinal stromal tumors (GIST),Phase 3,4/28/20,"Phase 3 data did not meet primary endpoint - April 28, 2020.",4
ZLAB,Immune thrombocytopenia (ITP),Phase 3,5/5/22,"Phase 3 top-line data reported that trial met primary endpoint, noted May 5, 2022.",5
HGEN,COVID-19,Phase 2/3,7/13/22,"Phase 2/3 top-line data showed that trial did not achieve statistical significance on the primary endpoint. The data also showed a non-significant trend toward a reduction in mortality in the overall patient population, noted July 13, 2022.",6
PLXP,Mild infections of diabetic foot ulcers,Phase 3,10/25/16,"Phase 3 top-line data did not meet endpoints - October 25, 2016 - TICKER CHANGED FROM DPRX FOLLOWING MERGER",4
AKRO,Nonalcoholic Steatohepatitis,Phase 2b,10/4/23,"Phase 2b results published at the Lancet Gastroenterology & Hepatology showed that both EFX doses achieved statistical significance on primary and secondary histopathology endpoints after 24 weeks, noted October 4, 2023.",6
AZN,Refractory generalized myasthenia gravis (gMG),Approved,10/23/17,"Approval announced October 23, 2017.",6
BCLI,Amytrophic lateral sclerosis (ALS),PDUFA,9/27/23,"Advisory Committee meeting voted 17-1 that NurOwn did not demonstrate substantial evidence of effectiveness for the treatment of mild to moderate ALS, noted September 27, 2023.",6
RHHBY,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
TNXP,fibromyalgia-type Long COVID,Phase 2,9/5/23,"Phase 2 topline data did not achieve the pre-specified primary endpoint of improving Long COVID pain intensity scores at Week 14, noted September 5, 2023.",6
NOVN,Molluscum Contagiosum,Phase 3,9/23/21,"Phase 3 safety data showed TEAEs reported in greater than 5% of subjects in the SB206 treated groups were all at the application site (pain, erythema, pruritis, exfoliation, and dermatitis) with the high majority of these TEAEs being mild or moderate by severity, noted September 23, 2021.",6
IOVA,anti-PD-1 therapy for advanced (unresectable or metastatic) Non-small Cell Lung Cancer (NSCLC),Phase 2,7/10/23,"Phase 2 preliminary data in the trial showed that the median duration of response wasn't reached. Treatrdent-emergent adverse events were consistent with the underlying disease and known adverse event profiles of non-myeloablative lymphodepletion and interleukin-2, noted July 10, 2023.",6
LPTX,Esophagogastric adenocarcinoma,Phase 1/2,8/6/19,"Phase 1/2 data August 6, 2019 noted 50% ORR.",5
GLPG,Idiopathic pulmonary fibrosis (IPF),Phase 2,11/30/20,"Phase 2 data released November 30, 2020 - forced vital capacity (FVC) decline of 42mL across treatment groups at 26 weeks. Phase 2b trial planned.",6
EQ,Celiac disease,Phase 1,5/15/23,"Phase 1 data suggested that selective blockade of IL-15 and IL-21 inhibits the synergistic signaling that mediates NK and T cell responses in multiple immune disorders including celiac disease, noted May 15, 2023.",5
CUE,Wilms' Tumor (WT1)-expressing cancers,Phase 1,11/3/23,"Phase 1 data presented at SITC demonstrated a 75%-80% DCR at the 4mg/kg and 2mg/kg doses respectively with two patients demonstrating tumor reductions of -30% and -29%, noted November 3, 2023.",6
AMGN,Plaque psoriasis in pediatric patients,Phase 3,9/9/22,"Phase 3 primary endpoint was met, noted September 8, 2022.",5
MRK,Pneumococcal Conjugate Vaccine,Phase 3,5/20/21,"Phase 3 trial met primary immunogenicity and safety endpoints - May 20, 2021.",6
PHIO,Melanoma,Phase 1b,3/22/23,"Phase 1b safety data reported no dose-limiting toxicity, and no drug-related severe adverse events or serious adverse events, noted March 22, 2023.",5
JNJ,Frontline multiple myeloma (non-transplant),Approved,5/7/18,"Approval announced May 7, 2018.",6
TERN,Non-alcoholic steatohepatitis (NASH),Phase 1,11/4/22,"Phase 1 data reported that TERN-501 was generally well tolerated and exhibited dose-dependent pharmacokinetics with low variability, noted November 4, 2022",6
ACIU,Alzheimer’s disease (AD),Phase 2,6/2/21,"Phase 2 interim analysis June 2, 2021. Good safety confirmed.",6
BMY,Melanoma,Approved,3/18/22,"Approved March 18, 2022.",6
AZN,B-cell non-Hodgkin lymphoma,Phase 1,12/12/22,"Phase 1 trial interim results presented at ASH reported that response-evaluable FL subjects treated at ≥ 800µg, the ORR was 88% (7/8 subjects); all responding subjects achieved a CR, noted December 12, 2022.",6
ALNY,Primary Hyperoxaluria Type 1 (PH1),Approved,11/24/20,"FDA approval announced November 24, 2020.",6
ALPMY,"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
VRCA,Molluscum contagiosum,Approved,7/21/23,"Approved July 21, 2023.",6
BMY,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
BPMC,CCNE-aberrant cancers,Phase 1/2,6/5/23,"Phase 1/2 data presented at ASCO reported a confirmed partial response, noted June 5, 2023.",6
LGND,Pneumococcal Conjugate Vaccine,Phase 3,7/27/23,"Phase 3 trial met primary endpoints, noted July 27, 2023.",5
CABA,Pemphigus vulgaris,Phase 1,5/18/23,"Phase 1 presented at ASGCT reported that no DLTs were observed and no clear pattern of changes in anti-DSG3 autoantibody levels across dosing cohorts were observed despite increased DSG3-CAART cell persistence, noted May 18, 2023.",6
BLPH,Pulmonary Hypertension-Sarcoidosis (PH-Sarc),Phase 2,12/17/21,"Phase 2 top-line data reported that all 8 subjects demonstrated decreases in mean pulmonary arterial pressure (mPAP) and pulmonary vascular resistance (PVR). Treatment resulted in a median drop of 20% in PVR, compared to a median baseline PVR of 329 dyne/cm.sec-5; a reduction of 20% or more in PVR, noted December 17, 2021.",6
SPPI,Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2,Phase 2,9/10/22,"Phase 2 cohorts 2 and 4 data presented at ESMO showed that 12/14 patients were evaluable and all had partial response, resulting in an ORR of 85.7% (95% CI, 57.2, 98.2) and a median DOR of 5.5 months . The ORR was 100% in the previously treated C2 patients, and 71.4% in the treatment naïve C4 patients. Median DOR was 5.3 months for the C2 patients and 8.9 months for those in C4, noted September 10, 2022.",6
BMY,Melanoma,Phase 3,10/2/20,Statistically significant improvement in RFS in the all-comer (intent-to-treat) population failed to show.,4
MDWD,Lower leg ulcers,Phase 2,4/12/22,"Phase 2 top-line data presented at the SAWC 2022 showed treatment to debride wounds and promote wound area reduction, while reducing biofilm and bacterial bioburden safely and effectively, noted April 12, 2022.",6
EXEL,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
TBPH,Asthma,Phase 2,11/5/20,"Phase 2a trial did not meet primary endpoint - November 5, 2020.",4
RARE,Glycogen Storage Disease Type Ia (GSDIa),Phase 1/2,5/19/22,"Phase 1/2 week 52 clinical efficacy and safety data reported that patients reached a mean total daily reduction of cornstarch intake of 70%, noted May 19, 2022.",6
SNY,Atopic dermatitis 12-17 year-olds,Approved,3/11/19,"FDA approval announced March 11, 2019.",6
GSK,HIV,Approved,1/21/21,"FDA approval announced January 21, 2021.",6
BMEA,"Healthy volunteers, type 2 diabetes",Phase 2,6/24/23,"Additional Phase 2 data presented at ADA of eight weeks after completing treatment with BMF-219, patients with type 2 diabetes (T2D) showed an increase of C-peptide and an improvement of HOMA-B, measured during oral glucose tolerance testing (OGTT), supporting improved beta cell function for these patients, June 24, 2023.",6
REGN,Prurigo nodularis (PN),Approved,9/28/22,"Approved September 28, 2022.",6
IPSEY,Primary biliary cholangitis (PBC),Phase 3,6/30/23,"Phase 3 data reported that trial met primary endpoint, noted June 30, 2023.",5
ASMB,Hepatitis B,Phase 1a,4/18/23,"Phase 1a clinical data for the remaining 200 mg multiple dose cohort for ABI-4334 continue to demonstrate encouraging clinical profile with strong projected activity against both viral replication and cccDNA formation, noted April 18, 2023.",6
TVTX,IgA nephropathy (IgAN),Phase 3,11/3/23,"Additional Phase 3 data presented at ASN showed one of the slowest rates of kidney function decline in IgAN trials, consistent treatment effects across baseline eGFR and proteinuria, and higher rates of complete remission compared to maximally tolerated dose of irbesartan through 110 weeks of treatment, noted November 3, 2023.",6
PFE,Gastric cancer,Phase 3,11/8/19,"Phase 3 endpoint not met - November 8, 2019.",4
EXEL,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
ALLK,Eosinophilic Gastritis and/or eosinophilic duodenitis (EoD),Phase 3,9/9/22,"Phase 3 24-week data showed that the company met its histologic co-primary endpoint but missed symptomatic co-primary endpoint, noted September 9, 2022.",6
CRNX,Congenital adrenal hyperplasia and Cushing’s disease,Phase 1,5/25/22,"Data from MAD cohort results showed serum cortisol below normal levels and a marked reduction in 24-hour urine free cortisol excretion in the presence of sustained, disease-like ACTH concentrations, noted May 25, 2022.",6
CLNN,Amyotrophic Lateral Sclerosis,Phase 2,8/29/23,"Phase 2 OLE data reported a significant median survival benefit of 19.3 months using the rank-preserving structural failure time model (RPSFTM) and a significant 52% decreased risk of ALS clinical worsening events, noted August 29, 2023.",6
FDMT,Choroideremia,Phase 1,10/25/21,"Phase 1 initial data from 6 patients enrolled in one of two dose cohorts: 3E11 vg/eye (cohort 1; n=3) and 1E12 vg/eye (cohort 2; n=3). The study showed that at the 3E11 vg/eye dose (Cohort 1), 4D-110 was well-tolerated with no dose-limiting toxicities or serious adverse events. At the 1E12 vg/eye dose, pigment dispersion (iris transillumination) was observed in 3 patients in the 1E12 vg/eye cohort ~7 to 9 months following treatment, 2 cases were asymptomatic and 1 patient reported mild glare.",5
CYTK,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
ALNY,Alzheimer's Disease and Cerebral Amyloid Angiopathy,Phase 1,10/25/23,"Phase 1 achieve sustained pharmacodynamic activity up to 10 months after administration, noted October 25, 2023.",6
DNLI,Hunter syndrome MPS II,Phase 1/2,8/30/23,"Additional data from SSIEM reported that data demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers, noted August 30, 2023.",6
GSK,Asthma,Approved,9/9/20,"FDA approval announced September 9, 2020.",6
MRK,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
CPRX,MuSK-antibody positive myasthenia gravis (MuSK-MG),Phase 3,8/10/20,"Phase 3 trial did not meet primary endpoint - August 10, 2020.",4
MRNS,Tuberous Sclerosis Complex,Phase 2,8/17/21,"Phase 2 top-line data released August 17, 2021. Primary endpoint showed a median 16.6% reduction in 28-day seizure frequency relative to baseline.",6
UBX,Wet age-related macular degeneration (AMD),Phase 2,9/27/23,"Phase 2 patients on combination treatment with UBX1325 and aflibercept from weeks 24-48 maintained vision gains achieved at week 24 on aflibercept alone, with greater vision improvement in patients with more severe disease. 40% of UBX1325-treated patients did not require anti-VEGF treatment through 48 weeks and 64% achieved an anti-VEGF treatment-free interval of over 24 weeks, noted Septemeber 27, 2023.",6
IMAB,Ulcerative colitis,Phase 2,4/26/21,Phase 2 trial met primary endpoint.,6
ATHA,Alzheimer’s disease,Phase 3,10/17/22,"Phase 3 interim analysis reported that an additional enrollment of ~150 patients will be well powered for the primary endpoint analysis, noted October 17, 2022.",5
MGNX,Solid tumors,Phase 1,2/14/23,"Phase 1 reported that nine of the 35 patients (25.7%) achieved confirmed partial responses (cPR), noted February 14, 2023.",6
AZN,COPD,Phase 3,5/30/18,"Phase 3 data released May 30, 2018. Primary endpoint not met.",4
JNJ,Psoriatic Arthritis in Pediatric Patients,Approved,8/1/22,"Approved August 1, 2022.",6
ALEC,Frontotemporal Dementia,Phase 2,8/3/23,"Additional Phase 2 data considered to be uninformative regarding treatment effect, noted August 3, 2023.",6
TBPH,Asthma,Approved,9/9/20,"FDA approval announced September 9, 2020.",6
BIIB,Cognitive Impairment Associated With Schizophrenia (CIAS),Phase 2b,7/20/22,"Phase 2b trial did not meet its primary endpoint, and program will be discontinued, noted July 20, 2022.",6
CRBU,B-Cell Non-Hodgkin Lymphoma,Phase 1,7/13/23,"Phase 1 update reported a 94% overall response rate (ORR), 69% complete response (CR) rate, and 44% CR rate at ≥6 months following a single dose of CB-010, with 24 months as the longer CR maintained to date, noted July 13, 2023.",5
CALT,IgA Nephropathy,Phase 3,6/21/23,"Phase 3 data presented at ERA continue to show that study study met its primary endpoint with Nefecon demonstrating a highly statistically significant benefit over placebo. Additionally the company submitted its sNDA based on this results to the FDA, noted June 21, 2023.",6
BGNE,Non-small cell lung cancer (NSCLC),Phase 3,4/12/21,"Phase 3 trial met primary endpoint of overall survival - November 17, 2020. Data presented at AACR meeting April 12, 2021. ORR 21.9% vs 7.0% for docetaxel; PFS 23.3% vs 5.7% in docetaxel arm; OS 17.2 months vs to 11.9 months in the docetaxel arm.",6
ORIC,Multiple myeloma,Phase 1b,11/2/23,"Phase 1b data from ASH abstract reported that ORIC-533 was very well tolerated, with 5 patients experiencing a total of 10 treatment-related adverse events (TRAEs), noted November 2, 2023.",6
GILD,Acute myeloid leukemia (AML),Phase 3,8/21/23,"Partial hold by the FDA on enrollment, noted August 21, 2023.",5
GMAB,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
XENE,Postherpetic neuralgia,Phase 2b,6/27/17,"Phase 2b data released June 27, 2017. Endpoints not met.",4
ETNB,Severe hypertriglyceridemia,Phase 2,6/24/23,"Phase 2 data presented at EASL showed consistent and significant reductions in triglycerides, atherogenic lipoproteins and liver fat, and pegozafermin's favorable safety and tolerability profile, noted June 24, 2023.",6
BHVN,Multiple system atrophy (MSA),Phase 3,9/27/21,"Phase 3 trial did not met primary or secondary endpoints, noted September 27, 2021.",4
SAGE,Major Depressive Disorder (MDD),Phase 3,12/5/19,"Phase 3 data did not meet primary endpoint - December 5, 2019.",4
AMGN,Colorectal Cancer (CRC),Phase 1b,9/12/22,"Phase 1b data presented at ESMO showed that treatment achieved a 30% confirmed objective response rate in patients with KRAS G12C-mutated metastatic colorectal cancer. Treatment response rates can be as low as 2% in this patient population, and the current standard of care offers a median progression-free survival benefit of 2 months, noted September 12, 2022.",6
FATE,Non Hodgkin Lymphoma,Phase 1,12/4/20,"Phase 1 interim data released December 4, 2020. 2/4 complete responses and one partial response.",5
APRE,TP53 mutated myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) - post transplant,Phase 2,12/13/21,"Phase 2 data from 33 patients enrolled demonstrated a 60% relapse free survival and 79% overall survival, both at 1 year post-transplant. Median overall survival was 20.6 months.",6
LLY,Diabetes,Approved,6/15/20,"FDA Approval announced June 15, 2020.",6
MRK,Non-muscle invasive bladder cancer (NMIBC),Phase 1,7/28/23,"Phase 1 dosing initiated, noted July 28, 2023.",5
CUE,HPV16+ head and neck cancer,Phase 1,11/3/23,"Phase 1 data shared at SITC showed an overall response rate (ORR) of 47% and disease control rate (DCR) of 65% in first line (1L) HNSCC patients treated with CUE-101 and pembrolizumab. ORR of 56% in patients with low expression of PD-L1 in the tumor, noted November 3, 2023.",6
AZN,Anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG),Approved,4/28/22,"Approved April 28, 2022.",6
SGEN,Acute myeloid leukemia (AML) - cancer,Phase 3,6/19/17,"Phase 3 trial discontinued due to higher rate of deaths - June 19, 2017.",4
MEIP,B-Cell Malignancies or Acute Myeloid Leukemia (AML),Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that at 200 mg, 5 of 10 AML pts (50%) had stable disease, noted November 2, 2023.",5
HARP,Multiple myeloma,Phase 1,11/2/23,"Phase 1 data from ASH abstract reported that among the 12mg and 24mg cohorts, 55% (22/40) of efficacy-evaluable pts currently have had a confirmed response (PR or better) and among pts with a response, 73% (16/22) have had a confirmed response of VGPR or better, noted November 2, 2023.",6
NVS,"Various blood cancers, rheumatoid arthritis.",CRL,5/2/18,"CRL issued May 2, 2018.",5
KPRX,Rheumatoid Arthritis,Phase 1b,4/25/22,"Phase 1b results demonstrated safety and tolerability of treatment as well as statistically significant improvements in conjunctival hyperemia in the 0.15% arm compared to vehicle control, noted April 25, 2022.",6
SNDX,"Recurrent non-small cell lung cancer (NSCLC), colorectal cancer",Phase 2,9/24/18,"Phase 2 updated data to be presented September 24, 2018 did not meet prespecified ORR endpoint.",4
SQZ,Solid Tumors,Phase 1/2,12/9/21,"Phase 1/2 monotherapy data reported that of the five patients in this cohort, one patient showed a radiographic response and symptomatic improvement and reported a complete response, noted December 9, 2021.",5
VRTX,Acute Pain following bunionectomy or abdominoplasty surgery,Phase 2,3/31/22,"Phase 2 trial met primary endpoint, noted March 31, 2022.",5
MRSN,Non-small cell lung cancer (NSCLC) adenocarcinoma and ovarian cancer,Phase 1,8/6/21,Phase 1 top-line data due 4Q 2021.,5
AADI,Soft Tissue Sarcoma,Phase 1,6/5/23,"Phase 1 data at ASCO reported that 3 of the 18 evaluable patients having achieved a partial response, and 13 achieving stable disease, noted June 5, 2023.",6
INFI,Solid tumors,Phase 1,11/10/18,Further data due at SITC November 2018 noted 2/44 PRs.,5
LLY,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
HALO,Chronic inflammatory demyelinating polyneuropathy (CIDP),Phase 2,7/17/23,"Phase 2 trial met primary endpoints, noted July 17, 2023.",5
GLUE,MYC-driven solid tumors,Phase 1/2,10/17/23,"Phase 1/2 initial data study demonstrated favorable tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles in heavily pre-treated patients with lung and high-grade neuroendocrine cancers., noted October 17, 2023",6
SRPT,Duchenne muscular dystrophy,Phase 2,5/3/21,"Phase 2 30 mg/kg arm data presented May 3, 2021 - mean exon skipping of 10.79% and mean dystrophin expression of 6.55%.",5
PFE,Children - growth hormone deficiency,Approved,6/28/23,"FDA Approved on June 28, 2023.",6
CLSD,Wet age-related macular degeneration,Phase 1/2,10/16/23,"Phase 1/2a results demonstrated the key benefits targeting the site of disease at the back of the eye, noted October 16, 2023.",6
IXHL,Anxiety,Phase 2,3/15/23,"Phase 2 data reported that the company found that there is a high probability that the total study will show a statistically significant benefit for the psilocybin treatment arm over the placebo treatment arm. This projection is made by assuming the effect size observed in the interim analysis for 29 participants is representative of the effect size through the remaining 43 participants, noted March 15, 2023.",6
ANAB,dMMR endometrial cancer,Approved,2/10/23,"Full approval granted February 10, 2023.",6
CLDX,Metastatic triple negative breast cancers that overexpress gpNMB,Phase 2b,4/16/18,"Phase 2b data released April 16, 2018 - primary endpoint not met.",4
SWTX,Neurofibromas,Phase 2b,2/25/21,Phase 2b interim data noted 10/20 Patients (50%) achieved an Objective Response,5
TXMD,Moderate to severe vasomotor symptoms (VMS),Approved,10/29/18,"FDA approval announced October 29, 2018.",6
ALT,Flu vaccine,Phase 2,3/19/19,"Phase 2 additional data released March 19, 2019 noted all patients remained seroprotected.",6
EQ,Acute graft-versus-host disease,Phase 3,4/26/23,"Phase 3 results reported that response at Day 29 was associated with improved progression-free survival through 1 year, noted April 26, 2023.",6
BBIO,Canavan Disease,Phase 1/2,5/22/23,"Phase 1/2 data presented at AGSCT reported percent decreases in NAA from baseline ranged between 70% and 95% in cerebrospinal fluid (CSF), 29% and 88% in urine, and 8% and 75% in brain, noted May 22, 2023.",6
CORT,Antipsychotic-induced weight gain (APIWG),Phase 2,12/8/22,"Phase 2 data reported that AIWG did not reverse, noted December 8, 2022.",5
MRK,"High-Risk, Early-Stage ER+/HER2- Breast Cancer",Phase 3,10/20/23,"Phase 3 data presented at ESMO reported that treatment significantly improved pCR rate compared to neoadjuvant placebo plus chemotherapy (24.3% vs. 15.6%, respectively), noted October 20, 2023.",6
RVNC,Upper facial lines,Phase 2,12/17/20,Efficacy of none or mild wrinkle severity seen in greater than 92% of subjects after four weeks.,6
IOVA,Solid Tumors,Phase 2,9/12/23,"Phase 2 data presented at WCLC reported that the ORR was 42.1%, noted September 12, 2023.",6
NVS,Acute postoperative pain,Approved,4/30/15,"Approved April 30, 2015.",6
LPCN,Men with low testosterone (Low T),Approved,12/8/20,"FDA tentative approval announced December 8, 2020.",6
NVO,Growth Hormone Deficiency (GHD),Approved,4/28/23,"Approved April 28, 2023.",6
PLX,Fabry disease,Phase 3,5/31/22,"Phase 3 data reported that most treatment emergent adverse events were mild to moderate, noted May 31, 2022.",6
AZN,HPV-associated squamous cell carcinoma of the head & neck (SCCHN),Phase 1,11/10/17,"Presentation at SITC Meeting - November 10, 2017.",5
MRK,Endometrial carcinoma,Phase 3,3/23/23,"Additional Phase 3 data reported that KEYTRUDA plus chemotherapy reduced risk of disease progression or death by 46% in patients whose cancer was mismatch repair proficient (pMMR) and by 70% in patients whose cancer was mismatch repair deficient (dMMR), compared to chemotherapy alone, noted March 23, 2023.",6
ETON,Pediatric adrenal insufficiency (AI),Approved,9/29/20,"FDA approval announced September 29, 2020.",6
ADMA,Primary humoral immunodeficiency,Approved,12/13/22,FDA supplemental approval for room temperature (25°C) storage conditions for up to 4 weeks during the first 24 months of the 36-month approved shelf life.,6
GILD,Cutaneous lupus erythematosus (CLE),Phase 2,10/24/19,Phase 2 trial did not meet primary endpoint.,4
AZN,Anaemia in Chronic Kidney Disease,CRL,8/11/21,"CRL announced August 11, 2021.",5
OTLK,Wet age-related macular degeneration (wet AMD),Phase 3,10/12/21,"Phase 3 safety data showed no intraocular inflammation or vasculitis, and the frequency and incidence of adverse events and ocular adverse events were low. The most common adverse event in the study eye conjunctival hemorrhage related to injection procedure, not to ONS-5010, and there were no additional serious adverse events associated with these injections, noted October 12, 2021.",6
TYRA,Achondroplasia,Phase 2,8/1/23,"FDA Orphan drug designation (ODD) granted on August 1, 2023.",5
RGLS,Autosomal dominant polycystic kidney disease (ADPKD),Phase 1b,10/12/21,"Termination of development in order to prioritize RGLS8429, noted October 12, 2021.",4
EPZM,Epithelioid Sarcoma,Approved,1/23/20,"FDA approval announced January 23, 2020.",6
OPTN,Nasal polyps,Approved,9/17/17,"Approval announced September 18, 2017 (prior to IPO).",6
VSTM,KRAS mutant advanced solid tumors,Phase 1/2,9/28/23,"Phase 1/2 data reported an overall response rate (ORR) of 42% (11 of 26) in evaluable patients with low grade serous ovarian cancer (LGSOC), noted September 28, 2023.",6
MRTX,MTAP-deleted cancers,Phase 1,8/8/23,"Phase 1 data reported a safety profile and early signs of clinical activity, noted August 8, 2023.",5
AMGN,Thyroid Eye Disease (TED) - chronic,Phase 3,4/10/23,"Phase 4 topline data reported that treatment achieved a statistically significant reduction in proptosis from baseline compared to those receiving placebo, noted April 10, 2023.",6
VNDA,Maintenance treatment of schizophrenia in adults,Approved,5/26/16,"Approved May 26, 2016.",6
ONCT,Relapsed or refractory Ewing sarcoma,Phase 1/2,11/15/21,"Phase 1/2 interim data showed that the drug remains generally well tolerated. 2 CR in heavily pretreated patients. As of the October 1, 2021 data cutoff date, the most common drug-related adverse events included myelosuppression, fatigue, alopecia, nausea, pyrexia, and decreased appetite. No unexpected off-target toxicities have been observed, noted November 15, 2021.",5
ELTX,Acute kidney injury (AKI) / Delayed Graft Function,Phase 3,10/26/21,"Phase 3 trial did not met the primary endpoint, noted October 26, 2021.",4
ANVS,Alzheimer’s disease (AD),Phase 2/3,10/30/23,"Phase 2/3 trial DSMB feedback resulted in no drug-related SAEs, each AE < 5% and a very low dropout rate of 4.7%, noted October 30, 2023.",5
AVDL,Excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy,Approved,5/1/23,"Final approval granted by FDA on May 1, 2023.",6
ARVN,Breast cancer,Phase 2,11/22/22,"Phase 2 data continued to show activity in heavily pre-treated patients with locally advanced or metastatic ER+/HER2- breast cancer, Median PFS of 3.7 months in all patients and 5.7 months in patients with ESR1 mutant tumors support the initiation of two Phase 3 registrational trials, noted November 22, 2022. Phase 3 monotherapy trial enrollment to be completed by 2H 2024.",6
ABBV,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
GRTS,COVID-19 vaccine,Phase 1,10/11/23,"Phase 1 data reported that all doses were well tolerated in both HIV-negative participants and PLWH irrespective of SARS-CoV-2 serostatus at baseline, noted October 11, 2023.",6
PFE,Non-small cell lung cancer (NSCLC),Phase 3,9/28/18,"FDA Approval announced September 27, 2018.",6
AKBA,Anemia due to chronic kidney disease (CKD),NDA Filing,8/24/23,"NDA to be resubmitted, noted August 25, 2023. Upon acceptance of the NDA, Akebia expects the FDA to set a PDUFA date of six months from the date of submission.",5
LQDA,Interstitial lung disease (PH-ILD),PDUFA,9/25/23,"FDA confirmed resubmission as Class II, PDUFA date assigned on January 24, 2024. Additionally, the FDA may not grant final approval of the PH-ILD indication until after the new clinical investigation exclusivity granted to Tyvaso expires on March 31, 2024, noted September 25, 2023.",6
JNJ,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
AIM,Metastatic triple-negative breast cancer,Phase 1/2,4/11/22,"Phase 1 data presented at AACR reported that trial met primary endpoint, noted April 11, 2022.",6
GSK,HIV,Approved,2/1/22,"Approved February 1, 2022.",6
TVTX,Focal segmental glomerulosclerosis (FSGS),Phase 3,11/3/23,"Phase 3 presentations at ASN showed that sparsentan delivered a clinically meaningful benefit at 108 weeks with significant proteinuria reduction, higher rates of partial and complete remission, and lower rates of end-stage kidney disease, noted November 3, 2023.",6
CRNX,Congenital hyperinsulinism (HI),Phase 1,9/15/21,"Phase 1 preliminary data demonstrated dose-dependent reductions in glucose-stimulated and sulfonylurea-induced Insulin secretion September 15, 2021.",6
TOVX,Allogeneic hematopoietic cell transplantation (HCT) for the prevention of acute graft-versus-host-disease (aGVHD),Phase 1/2,2/16/23,"Phase 1b/2a data reported that no AEs or SAEs were determined to be related to study drug treatment by the investigators, noted February 16, 2023.",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. NDA filing due in 2H 2023.,6
HEPA,NASH and Liver Cancer,Phase 2a,1/8/22,"Phase 2a data reported that treatment was well-tolorated with ALT decreased in 50%, 67%, and 87% of the subjects in the placebo, 75 mg, and 225 mg cohorts, respectively, noted January 8, 2022.",6
ETON,Injectable hospital product,Approved,6/15/21,"FDA approval announced June 15, 2021.",6
MRK,Type 2 diabetes,Approved,12/20/17,"Approval announced December 20, 2017.",6
MRNS,Refractory status epilepticus (RSE),Phase 2,9/26/19,"Phase 2 data met primary endpoint - September 26, 2019.",6
GLYC,Acute myeloid leukemia (AML) newly diagnosed and refractory - cancer,Phase 1/2,12/12/17,Phase 3 trial to be initiated mid-2018. Phase 1/2 data released at ASH 2017 showed clinical remission (CR+CRi) was 43%. Median overall survival was 9.4 months.,5
ARQT,Seborrheic dermatitis,Phase 3,9/9/22,"Additional Phase 3 data presented at EADV reported that more than 60% of patients treated with roflumilast foam achieved an itch response at Week 8, noted September 9, 2022.",6
MRK,Head and neck squamous cell carcinoma (HNSCC),Approved,6/11/19,"FDA Approval announced June 11, 2019.",6
ADTX,Covid-19,Phase 3,11/12/21,"Phase 3 top-line data reported that the trial did not meet its primary endpoint, noted November 12, 2021.",4
BPMC,PDGFRA exon 18 mutant Gastrointestinal stromal tumors (GIST),Approved,1/9/20,"FDA Approval announced January 9, 2020.",6
TPTX,"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
AMGN,Sjögren’s syndrome,Phase 2,5/31/23,"Additional Phase 2 data reported that all ESSDAI responder analyses (pre-specified and post-hoc) favored dazodalibep over placebo, with greater numerical differences for the highest levels of response and patients treated with dazodalibep experienced numerically greater improvements in ESSPRI score and fatigue compared to those who received placebo at Day 169, noted May 31, 2023.",6
SGEN,Cervical Cancer,Approved,9/20/21,"Approved September 20, 2021.",6
IRWD,Gastroesophageal reflux disease (GERD),Phase 3,9/29/20,"Phase 3 trial did not meet primary endpoint - September 29, 2020.",4
GLPG,Ankylosing spondylitis,Phase 2,9/5/18,"Phase 2 data released September 6, 2018. Primary endpoint met.",6
ELEV,Solid tumors,Phase 1,6/3/23,"Phase 1 data presented at ASCO reported a 47.1% ORR in patients with resistant/refractory gastric cancer expressing Claudin 18.2, with a well-tolerated safety profile, noted June 3, 2023.",6
ARDX,Serum phosphorus - chronic kidney disease (CKD) on dialysis,Approved,10/17/23,"Approved October 17, 2023.",6
LLY,Psoriatic Arthritis,Phase 3,12/17/18,Phase 3 trial met primary and key secondary endpoints.,6
MNMD,Major Depressive Disorder (MDD),Phase 2,4/14/23,"Phase 2 topline data reported that trial met primary endpoint, noted April 14, 2023.",5
ELOX,Alport syndrome,Phase 2,9/18/23,"Phase 2 additional independent confirmation reported that all three patients treated with ELX-02 showed a visual improvement in podocyte foot process effacement post-treatment in kidney biopsies demonstrating the disease modifying effect of ELX-02, noted September 18, 2023.",6
MRTX,Colorectal Cancer,Phase 1/2,10/25/20,Phase 1/2 updated data noted (23/51) 45% ORR in NSCLC patients.,5
NVCR,Recurrent glioblastoma,Phase 2,12/12/22,"Phase 2 trial data reported that median progression-free survival was 4.5 months compared to 2.2 months in the historical control, noted December 12, 2022.",5
MNKKQ,Pain,CRL,12/12/18,"CRL issued December 12, 2018.",5
VIGL,Healthy volunteers,Phase 1,10/17/23,"Phase 1 trial dosing initiated, noted October 17, 2023.",5
OVID,Fragile X syndrome,Phase 2,4/19/21,"Development to be discontinued - April 19, 2021.",4
JNJ,Treatment resistant depression,Approved,3/5/19,"FDA approval announced March 5, 2019.",6
PFE,Herceptin biosimilar,Approved,3/15/19,"FDA Approval March 11, 2019.",6
JNJ,Multiple Myeloma,Approved,8/20/20,"FDA approval announced August 20, 2020.",6
ABBV,Chronic Lymphocytic Leukemia,Approved,5/15/19,"FDA approval announced May 15, 2019.",6
HALO,Testosterone deficiency,Approved,10/1/18,"FDA Approval announced October 1, 2018.",6
LBPH,Developmental and epileptic encephalopathies (DEEs),Phase 1,12/5/22,"Phase 1 initial data reported favorable safety and tolerability results were observed in this study, with adverse events (AEs) generally consistent with previous clinical studies, noted December 5, 2022.",6
AMGN,Biosimilar candidate to Avastin (bevacizumab),Approved,9/14/17,"Approved September 14, 2017.",6
AMGN,Rheumatoid arthritis (RA),Approved,6/6/22,"Approved June 6, 2022.",6
PFE,Acute treatment of migraine,Approved,2/27/20,"FDA Approval announced February 27, 2020.",6
ASRT,Adrenocortical insufficiency screening,CRL,10/21/19,"CRL announced October 21, 2019.",5
BMEA,Acute myeloid leukemia (AML) / acute lymphocytic leukemia (ALL) / Diffuse large B-cell lymphoma (DLBCL) / Multiple myeloma (MM),Phase 1,7/24/23,"Phase 1 clinical update of AML cohort revealed 2 complete responses (CRs) out of 5 relapsed/refractory AML patients carrying menin-dependent mutations treated at Dose Level 4, noted July 24, 2023.",6
BMY,Kaposi’s sarcoma,Approved,5/15/20,"FDA Approval announced May 15, 2020.",6
RYZB,Gastroenteropancreatic neuroendocrine tumors,Phase 1b,10/22/23,"Phase 1b data presented at ESMO reported that the confirmed ORR was 29.4%, with all five patients having a confirmed PR, noted October 22, 2023.",6
AURA,Indeterminate lesions (ILs) and choroidal melanoma (CM),Phase 1/2,6/22/22,"Phase 1b/2 reported results from retrospective matched case control study, treatment achieved statistically significant vision preservation compared current standard of care, noted June 22, 2022.",6
INZY,ABCC6 deficiency,Phase 1/2,10/13/23,"Phase 1/2 interim clinical data presented at ASBMR reported that treatment was generally well-tolerated on October 13, 2023.",6
PRAX,Epilepsy,Phase 1,8/7/23,"Phase 1 study demonstrated pharmacodynamic activity across all dose levels for study subjects who received PRAX-628 at first administration as compared with subjects who received a placebo, noted August 7, 2023. Based on these studies and the preclinical results, Praxis intends to advance PRAX-628 into a Phase 2 study in focal epilepsy in the 1H 2024.",6
MDWD,Severe burns - pediatric,Phase 3,7/20/21,"Phase 3 top-line data met primary endpoints - July 20, 2021.",6
INCY,Steroid-refractory chronic GVHD (Graft versus host disease),Approved,9/22/21,"Approved September 22, 2021.",6
SABS,COVID-19,Phase 3,4/26/23,"Phase 3 trial demonstrated benefit in sustained symptom resolution in study participants with COVID-19 caused by Omicron as compared to participants who received a monoclonal antibody combination, REGEN-COV. Specifically, 66% of participants treated with SAB-185 reached full symptom resolution for at least 4 consecutive days by Day 28, while only 50% of participants on REGEN-COV met this endpoint, and the median time to symptom resolution for at least 4 consecutive days was 7 days shorter for SAB-185, noted April 26, 2023.",6
HALO,Amyloidosis,Approved,1/15/21,"FDA approval announced January 15, 2021.",6
KYMR,"AML, Lymphomas, and Uveal melanoma",Phase 1,6/22/23,"FDA granted orphan drug designation on June 22, 2023.",5
NVCR,Glioblastoma (GBM),Phase 2,3/24/22,"Phase 2 updated results reported a median progression-free survival was 12.1 months with median overall survival of 25.2 months, noted March 24, 2022.",5
BBIO,"Venous, Lymphatic, and Venolymphatic Malformations",Phase 1/2,6/10/22,"Phase 1/2 preliminary results noted that treatment was generally well-tolerated with local self-limited rash was the only adverse experience reported, noted June 10, 2022.",6