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1
LLY
Relapsed/refractory chronic lymphocytic leukemia
Approved
2023-01-27 00:00:00
FDA Approved on January 27, 2023.
1
TAK
Narcolepsy
Phase 2
2021-10-05 00:00:00
Phase 2 trial stopped dosing and ended trial due to emergence of a safety signal, noted October 5, 2021.
-1
IFRX
ANCA-associated vasculitis (AAV)
Phase 2
2021-05-11 00:00:00
Phase 2 data released May 11, 2021 - safe and well tolerated
1
AZN
Bladder cancer
Phase 3
2020-03-06 00:00:00
Phase 3 data did not meet primary endpoint.
1
IONS
Chronic hepatitis B virus (CHB)
Phase 2b
2022-06-27 00:00:00
Phase 2b data readout reported that up to 68% of patients on NA therapy and up to 65% of patients not on NA achieved HBsAg <100 IU/mL at the end of treatment, noted June 27, 2022.
1
LLY
Medullary thyroid cancer (MTC)
Phase 3
2023-10-21 00:00:00
Phase 3 data presented at ESMO reported that selpercatinib demonstrated superior PFS vs control in the ITT-pembro (HR: 0.465, 95% CI: 0.309, 0.699; p-value: 0.0002) and ITT populations (HR: 0.482, 95% CI: 0.331, 0.700; p-value: 0.0001), noted October 21, 2023.
1
AMGN
Chronic spontaneous urticaria
Phase 2b
2023-10-31 00:00:00
Phase 2b trial did not meet its primary endpoint, noted October 31, 2023.
1
AGIO
Thalassemia
Phase 2
2022-12-12 00:00:00
Phase 2 data reported durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients, noted December 12, 2022.
1
NVS
Non-small cell lung cancer (NSCLC), adjuvant
Phase 3
2022-08-15 00:00:00
Phase 3 trial did not meet the primary endpoint, noted August 15, 2022.
1
GLMD
HIV-associated lipodystrophy and nonalcoholic fatty liver disease (NAFLD)
Phase 2
2018-02-14 00:00:00
Phase 2 top-line data released February 14, 2018 did not meet primary endpoint.
1
JNJ
Multiple myeloma
Approved
2023-08-10 00:00:00
Approved August 10, 2023.
1
NVS
Acute myeloid leukemia (AML)
Approved
2017-04-28 00:00:00
Priority review granted November 14, 2016. Approval announced April 28, 2017.
1
BGNE
Nasopharyngeal cancer (RM-NPC)
Phase 3
2022-04-19 00:00:00
Additional Phase 3 data reported that progression-free survival was 15.5 months, noted April 19, 2022.
0
APLT
Diabetic cardiomyopathy (DbCM)
Phase 3
2023-06-22 00:00:00
Phase 3 baseline data to be presented at ADA showed a strong statistical correlation between elevations in the cardiac stress biomarker, NT-proBNP, reduced cardiac functional capacity and physical activity, underscoring the importance of early diagnosis via NT-proBNP and impact of DbCM on physical function and quality ...
1
NBIX
Huntington's disease
Approved
2023-08-18 00:00:00
Approved August 18, 2023.
1
TVTX
Classical homocystinuria (HCU)
Phase 1/2
2023-05-31 00:00:00
Additional Phase 2 data reported that treatment was well tolerated, with dose-dependent reductions in tHcy as previously observed in the highest BIW dosed cohorts, noted July 6, 2023.
1
INVA
cIAI (complicated intra-abdominal infections)
Approved
2018-08-27 00:00:00
FDA approval announced August 27, 2018.
1
INCY
Alopecia Areata
Approved
2022-06-14 00:00:00
Approved June 14, 2022.
1
EVO
High adenosine signature cancers
Phase 1
2023-04-19 00:00:00
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
1
APM
Neuroblastoma
Phase 1/2
2023-06-22 00:00:00
Phase 1b/2a trial has been repurposed for small molecule drug , noted June 22, 2023.
0
AGIO
Pyruvate Kinase Deficiency
Phase 3
2022-06-10 00:00:00
Phase 3 new patient outcome reported that 28/78 (35.9%) of all patients achieved a normal hemoglobin level at least once during treatment, noted June 10, 2022.
1
SELB
Chronic Refractory Gout
Phase 3
2023-03-21 00:00:00
Phase 3 top-line data reported that both trials met their primary endpoints, noted March 21, 2023.
0
GKOS
Progressive keratoconus
Phase 3
2021-02-25 00:00:00
Phase 3 trial met primary endpoint - February 25, 2021.
0
LPTX
Biliary tract cancer (BTC)
Phase 1/2
2018-12-14 00:00:00
Phase 1/2 data released December 14, 2018 noted 21.3% PR rate.
0
BMY
Multiple Myeloma
Phase 3
2020-03-09 00:00:00
Phase 3 trial did not meet progression-free survival primary endpoint.
1
ENTA
Hepatitis C virus (HCV)
Approved
2017-08-03 00:00:00
Approval announced August 3, 2017.
1
NRXP
COVID-19
Phase 2/3
2021-10-14 00:00:00
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.
1
PFE
Obsessive compulsive disorder (OCD)
Phase 2/3
2020-06-24 00:00:00
Phase 2/3 top-line data did not meet primary endpoint.
1
JAZZ
Small cell lung cancer (SCLC)
Phase 3
2020-12-03 00:00:00
Phase 3 trial did not meet overall survival endpoint - December 3, 2020.
1
FBIO
Non-small cell lung cancer (NSCLC)
Phase 1/2
2018-09-25 00:00:00
Phase 1/2 preliminary data released September 24, 2018.
0
CDXS
Phenylketonuria (PKU)
Phase 1
2018-11-08 00:00:00
Phase 1 trial met endpoints - November 8, 2018.
0
AZN
COVID-19 vaccine (variant)
Phase 2/3
2022-02-10 00:00:00
Phase 2/3 trial met primary endpoint, noted February 10, 2022.
0
SCPH
Chronic heart failure
Phase 2
2022-07-12 00:00:00
Phase 2 trial data reported a 37% reduction in the risk of a heart failure hospitalization relative to patients randomized to "treatment as usual" at day 30, noted July 12, 2022.
1
SNPX
Multiple sclerosis (MS)
Phase 1
2023-07-19 00:00:00
IND submitted to FDA, noted July 19, 2023
0
EXEL
Solid Tumors
Phase 1/2
2022-01-18 00:00:00
Phase 1/2 cohort results showed that of the 36 patients enrolled in cohort 2 of the CAMILLA trial, 29 were evaluable for the efficacy analysis. The primary outcome of investigator-assessed ORR per modified RECIST version 1.1 was 27.6%. The confirmed PR rate was 20.7%, and the DCR was 86.2%. Median PFS was 3.8 months, w...
1
OPTN
Nasal polyps
Approved
2017-09-17 00:00:00
Approval announced September 18, 2017 (prior to IPO).
1
IPSEY
Fibrodysplasia ossificans progressiva (FOP)
Approved
2023-08-16 00:00:00
FDA Approved on August 16, 2023.
1
CHRS
Wet age-related macular degeneration (wAMD), among others
Approved
2022-08-02 00:00:00
Biosimilar approved August 2, 2022.
1
ADVM
Wet age-related macular degeneration (Wet-AMD)
Phase 2
2022-11-04 00:00:00
Phase 2 end-of-study results reported that all 2E11 participants were inflammation free at the end of the study, noted November 4, 2022.
0
JNJ
Relapsing multiple sclerosis
Phase 3
2019-07-26 00:00:00
Phase 3 trial met primary endpoint.
0
NKTR
Renal Cell Carcinoma (RCC)
Phase 3
2022-04-14 00:00:00
Phase 3 data reported that trial did not met its endpoints of objective response rate (ORR) and overall survival (OS), noted April 14, 2022.
0
ONCT
Chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL)
Phase 1/2
2022-12-12 00:00:00
Phase 1/2 data reported that the ORR of 89% and CR rate of 43% for patients with MCL treated with zilovertamab plus ibrutinib compare favorably to the historical ORR of 66% and CR rate of 20% for ibrutinib monotherapy, noted December 12, 2022,
1
RGNX
Homozygous familial hypercholesterolemia (HoFH)
Phase 1/2
2020-08-06 00:00:00
Development to be discontinued - noted August 6, 2020.
-1
SGEN
Frontline Hodgkin lymphoma
Approved
2018-04-04 00:00:00
sBLA approval announced March 20, 2018.
1
PFE
Glioblastoma (GBM)
Phase 2/3
2023-10-31 00:00:00
Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.
0
NVS
Non-small cell lung cancer (NSCLC) with BRAF V600E mutation
Approved
2017-06-22 00:00:00
FDA Approval announced June 22, 2017.
1
MDGL
Heterozygous familial hypercholesterolemia (HeFH)
Phase 2a
2018-04-03 00:00:00
Phase 2 top-line data released February 8, 2018 - primary endpoint met.
0
BPMC
Non-advanced systemic mastocytosis (SM)
Approved
2023-05-22 00:00:00
FDA approval on May 22, 2023.
1
BGNE
Waldenström’s macroglobulinemia
Approved
2021-09-01 00:00:00
FDA Approval announced September 1, 2021.
1
MOR
Ulcerative colitis
Phase 2b
2022-10-24 00:00:00
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.
1
TAK
Complex Crohn's Perianal Fistulas (CPF)
Phase 3
2023-10-18 00:00:00
Phase 3 trial did not meet its primary endpoint, noted October 18, 2023.
1
AZN
Type 2 diabetes
Approved
2019-05-03 00:00:00
FDA approval announced May 3, 2019.
1
ALT
Non-alcoholic steatohepatitis (NASH) - Healthy, Overweight and Obese Volunteers
Phase 2
2023-03-21 00:00:00
Phase 2 interim 24-week data data showed that approximately 50% of subjects achieved 10% or more weight loss and approximately 20% of subjects achieved 15% or more weight loss at the 1.8 mg and 2.4 mg doses at Week 24. Treatment discontinuation rates were 28.2% in subjects receiving placebo and 24.0% in subjects receiv...
1
EYEN
Mydriasis (office-based pupil dilation)
Approved
2023-11-02 00:00:00
FDA supplemental approval on November 1, 2023.
1
GMAB
Relapsing multiple sclerosis
Approved
2020-08-20 00:00:00
FDA approval announced August 20, 2020.
1
VCNX
Resectable Stage III melanoma
Phase 1
2022-11-10 00:00:00
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.
1
CGEN
Ovarian Cancer
Phase 1
2022-11-07 00:00:00
Preliminary Phase 1 data reported a 12% ORR in 3L+ MSS-CRC patients with liver metastases, noted November 7, 2022.
0
LLY
Type 2 Diabetes
Approved
2020-01-27 00:00:00
FDA Approval announced January 27, 2020.
1
AZN
Third-line ovarian cancer
Phase 3
2018-12-20 00:00:00
Phase 3 data released December 20, 2018 met primary and secondary endpoints.
0
VIR
COVID-19 antibody
Approved
2022-02-11 00:00:00
EUA granted by FDA February 11, 2022.
0
CNCE
Alopecia areata
Phase 3
2022-11-21 00:00:00
Additional data reported that for patients with an absolute Severity of Alopecia Tool (SALT) score less than 95 at baseline, 43% and 57% of the 8 mg twice-daily and 12 mg twice-daily deuruxolitinib dose groups, respectively, achieved a SALT score of 20 or less at Week 24, noted November 21, 2022.
1
LXRX
Heart failure
Approved
2023-05-26 00:00:00
Approved May 26, 2023.
1
VRTX
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
Approved
2015-03-17 00:00:00
Approved March 17, 2015.
1
TAK
Eosinophilic esophagitis
CRL
2021-12-21 00:00:00
CRL December 21, 2021.
0
GSK
Multiple Myeloma
Phase 3
2022-11-07 00:00:00
Phase 3 trial did not meet its primary endpoint, noted November 7, 2022.
1
CYTO
COVID-19
Phase 1
2023-01-24 00:00:00
Phase 1 topline data reported that trial did not meet primary endpoint, noted January 24, 2023.
1
ALGS
Hepatitis B
Phase 1
2023-06-21 00:00:00
Phase 1 data presented at EASL resulted in dose-dependent reductions in HBsAg levels through 90 days. Importantly, the safety and PK profile of these dose levels also support further evaluation of higher dose levels, noted June 21, 2023.
1
BLUE
Sickle Cell Disease
Phase 3
2021-02-16 00:00:00
Phase 3 trial temporarily suspended due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) - February 16, 2021.
0
PFE
Acute myeloid leukemia (AML)
Approved
2018-11-21 00:00:00
FDA approval announced November 21, 2018.
1
AZN
Anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG)
Approved
2022-04-28 00:00:00
Approved April 28, 2022.
1
JAZZ
Multiple sclerosis (MS)-related spasticity
Phase 3
2022-06-28 00:00:00
Phase 3 trial did not meet the primary endpoint, noted June 28, 2022.
1
HOTH
EGFRI-induced skin toxicity
Phase 2a
2023-06-14 00:00:00
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.
1
ABBV
Crohn’s Disease
Approved
2022-06-17 00:00:00
Approved June 17, 2022.
1
FOLD
Pompe Disease
Approved
2023-09-28 00:00:00
FDA Approved on September 28, 2023.
1
NUVL
ALK non-small cell lung cancer (NSCLC)
Phase 1
2023-10-13 00:00:00
Phase 1 trial showed preliminary signs of activity observed in heavily pre-treated patients with ALK-positive NSCLC, including in subgroups of patients who have previously received a 2nd generation ALK TKI and lorlatinib, have brain metastases, or have single or compound ALK resistance mutations, noted October 13, 2023...
1
BMY
Follicular lymphoma
Phase 2
2023-06-17 00:00:00
Phase 2 data presented at ICML showed that 97% of patients with R/R FL treated with Breyanzi achieved a response, with 94% achieving a complete response, and 81.9% of responders in ongoing response at 12 months, noted June 17, 2023.
1
CRDF
Small cell lung cancer (SCLC)
Phase 2
2023-09-26 00:00:00
Phase 2 safety data from the first six patients by the institutional review board confirmed the trial can continue to enroll as planned, noted September 26, 2023.
0
IKNA
Solid tumors and urothelial carcinoma, bladder cancer
Phase 1
2022-11-10 00:00:00
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.
1
GILD
Hepatitis C virus (HCV)
Approved
2017-07-18 00:00:00
Approval announced July 18, 2017.
1
NGM
Pancreatic Cancer
Phase 1b
2021-09-16 00:00:00
Phase 1b data had all 6 patients demonstrated disease control, 3 patients had partial responses (PR), and 3 stable disease (SD). 2 patients discontinued early due to gemcitabine/Nab-paclitaxel toxicity and/or progression of the underlying disease. Phase 1a monotherapy cohort reported 3 patients (30%) in the 30 mg dosin...
1
GLPG
Cutaneous lupus erythematosus (CLE)
Phase 2
2019-10-24 00:00:00
Phase 2 trial did not meet primary endpoint.
1
BGNE
Stage II-IIIA non-small cell lung cancer (NSCLC)
Phase 3
2023-10-23 00:00:00
Phase 3 data presented at ESMO reported that MPR & pCR rates were significantly improved with TIS + CT vs CT, noted October 23, 2023.
1
LGVN
Hypoplastic Left Heart Syndrome
Phase 1b
2023-05-09 00:00:00
Phase 1b long term data reported that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B, noted May 9, 2023.
0
RHHBY
Pemphigus vulgaris
Phase 3
2019-06-13 00:00:00
Phase 3 trial met primary and secondary endpoints - June 13, 2019.
0
JNJ
Influenza
Phase 2a
2023-06-22 00:00:00
FDA has granted Fast Track designation (FTD), noted June 22, 2023.
0
OMER
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 1b
2023-11-02 00:00:00
Phase 1b data from ASH abstract reported that mean Hgb increased from baseline by 3.1 g/dL at 4 weeks, and by 9.5 g/dL at the latest timepoint of 24 weeks, noted November 2, 2023.
0
LLY
Diabetes
Approved
2020-06-15 00:00:00
FDA Approval announced June 15, 2020.
1
ATOS
Breast cancer
Phase 2
2021-06-09 00:00:00
Phase 2 final data presented June 9, 2021. Primary endpoint met.
1
JANX
Metastatic castration-resistant prostate cancer (mCPRC)
Phase 1
2023-07-17 00:00:00
Phase 1 interim clinical data showed tumor-activated T cell engagement in patients with prostate cancer representing the first-in-human data for the TRACTr platform and providing confidence for continuing clinical development of JANX007 and advancing future programs from the platform, noted July 17, 2023.
1
JNJ
Waldenström’s Macroglobulinemia
Approved
2018-08-27 00:00:00
FDA approval announced August 27, 2018.
1
BIIB
Postpartum Depression
Approved
2023-08-04 00:00:00
Approved August 4, 2023.
1
MRK
Metastatic Melanoma
Phase 2b
2023-10-31 00:00:00
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.
1
ZVRA
Niemann-Pick disease Type C (NPC)
CRL
2021-06-18 00:00:00
CRL issued June 18, 2021.
0
ONCY
HR+ / HER2- breast cancer
Phase 2
2023-06-05 00:00:00
Phase 2 data readout at ASCO reported that nine pts (33%) discontinued pelareorep and 6 (35%) discontinued avelumab due to toxicity, noted June 5, 2023.
-1
OBSV
In Vitro Fertilization (IVF)
Phase 3
2018-02-26 00:00:00
Phase 3 data released February 26,2018 - primary endpoint met. Improvement shown in ET D5 subgroup but not in ET D3.
1
BIVI
Alzheimer's disease
Phase 2
2022-12-05 00:00:00
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.
1
ADVM
Diabetic macular edema (DME)
Phase 2
2021-10-09 00:00:00
Phase 2 data from trial (June 22, 2021, cutoff date, n=34) at ASRS demonstrated that dose and disease state appear to have an impact in determining the therapeutic window which balances efficacy and safety. Dose-dependent inflammation observed may be in part related to the study population which included patients with ...
1
TSVT
Multiple Myeloma
Phase 3
2023-02-10 00:00:00
Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted...
1
RETA
CKD Patients at Risk of Rapid Progression
Phase 2
2023-05-10 00:00:00
Program discontinued, noted May 10, 2023.
-1
CUE
HPV16+ head and neck cancer
Phase 1
2023-11-03 00:00:00
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.
1