ticker
stringlengths 2
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| disease
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| stage
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| catalyst
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| label
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|---|---|---|---|---|---|
ZLAB
|
Non-small cell lung cancer (NSCLC) with a KRASG12C Mutation
|
Approved
|
2022-12-13 00:00:00
|
Approved December 13, 2022.
| 1 |
MRK
|
Castration-Resistant Prostate Cancer
|
Approved
|
2020-05-19 00:00:00
|
FDA Approval announced May 19, 2020.
| 1 |
AYTU
|
Attention deficit hyperactivity disorder (ADHD)
|
Approved
|
2016-01-27 00:00:00
|
Approved January 27, 2016.
| 1 |
MRK
|
HIV
|
Approved
|
2019-09-20 00:00:00
|
FDA Approval for sNDA announced September 20, 2019.
| 1 |
BIIB
|
Alzheimer’s disease
|
Phase 3
|
2019-09-13 00:00:00
|
Phase 3 trial discontinued due to unfavorable risk-benefit ratio.
| 1 |
SNY
|
Hematological malignancies
|
Phase 1/2
|
2023-10-16 00:00:00
|
Phase 1/2 data from ESMO abstract reported that 3/8 patients achieved complete remission (CR), noted October 16, 2023.
| 1 |
ATHX
|
Ischemic stroke
|
Phase 2/3
|
2022-05-20 00:00:00
|
Phase 2/3 final top-line results reported that trial did not meet the primary endpoint, noted May 20, 2022.
| 1 |
NGM
|
Geographic atrophy
|
Phase 2
|
2022-10-17 00:00:00
|
Phase 2 trial did not meet primary endpoint, noted October 17, 2022.
| 1 |
BMY
|
Relapsed/Refractory Multiple Myeloma (RRMM)
|
Phase 1/2
|
2022-12-11 00:00:00
|
Additional data presented at ASH showed promising efficacy in a highly refractory patient population. As of the data cut-off date, mezigdomide plus DEX showed a manageable safety profile, noted December 11, 2022.
| 1 |
VYNE
|
Vitiligo
|
Phase 1b
|
2023-10-30 00:00:00
|
Phase 1b clinical proof-of-concept achieved with statistically significant dose dependent reduction in F-VASI score from baseline for the VYN201 1.0% and 2.0% cohorts compared to the 0.5% cohort, noted October 30, 2023.
| 1 |
TPTX
|
Solid tumors
|
Phase 1
|
2021-10-07 00:00:00
|
Phase 1 updated data noted a confirmed responses for a cORR of 36% out of the 11 MET TKI-naïve NSCLC patients, cORR of 33% for the nine MET TKI-naïve GC/GEJ patients, and among the 12 patients with advanced other solid tumors, one patient with MET amplified colorectal cancer achieved a confirmed response, noted October 7, 2021.
| 1 |
NXTC
|
PD-1 axis inhibitor refractory non-small cell lung cancer (NSCLC)
|
Phase 2
|
2023-09-12 00:00:00
|
Phase 2 data reported that 28% of patients (5/18) had durable clinical benefit with three of these being confirmed responses, noted September 12, 2023.
| 0 |
TALS
|
Transplanted Organ Rejection
|
Phase 3
|
2023-02-16 00:00:00
|
Trial was discontinued due to the pace of enrollment and the associated timeline to critical milestones, noted February 16, 2023.
| -1 |
ALNY
|
Primary Hyperoxaluria Type 1
|
Phase 3
|
2020-09-30 00:00:00
|
Clinically meaningful reduction in spot urinary oxalate:creatinine ratio.
| 1 |
CRNX
|
Acromegaly
|
Phase 3
|
2023-09-11 00:00:00
|
Phase 3 topline data reported that trial met its primary endpoint, noted September 11, 2023.
| 0 |
BPMC
|
RET-mutant medullary thyroid cancer
|
Approved
|
2020-12-01 00:00:00
|
FDA approval announced December 1, 2020.
| 1 |
INM
|
Epidermolysis Bullosa
|
Phase 2
|
2023-06-22 00:00:00
|
Phase 2 data from one patient were excluded from efficacy analyses due to a significant protocol deviation. Of the 18 participants assessed, chronic itch improved by a clinically meaningful amount in 12 patients (66.7%). However, the protocol-specified statistical analyses for non-wound itch were not statistically significant in favor of INM-755 due partly to the underlying control cream's clinically important anti-itch effect, noted June 22, 2023.
| 1 |
ABBV
|
Ulcerative Colitis
|
Approved
|
2022-03-16 00:00:00
|
Approved March 16, 2022.
| 1 |
AGRX
|
Contraceptive patch
|
Approved
|
2020-02-14 00:00:00
|
FDA Approval announced February 14, 2020.
| 1 |
ASLN
|
Atopic dermatitis (AD)
|
Phase 2b
|
2023-10-13 00:00:00
|
Phase 2b topline new data from a posthoc analysis of patients with severe disease (baseline EASI score at least 21), representing 63% of ITT patients, show monthly dosing with 600 mg eblasakimab for 16 weeks led to a 74.5% reduction in EASI score and EASI-75 of 53.6%, noted October 13, 2023.
| 1 |
ALLO
|
Multiple myeloma
|
Phase 1/2
|
2021-12-13 00:00:00
|
Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.
| 1 |
STRO
|
Ovarian and Endometrial Cancer
|
Phase 1
|
2023-10-23 00:00:00
|
Additional Phase 1 data reported that a confirmed partial response (PR) was seen in 29% of patients, and the Disease Control Rate (DCR) was 86%, noted October 23, 2023.
| 0 |
ANIP
|
Acute exacerbations of multiple sclerosis, rheumatoid arthritis, systemic lupus erythematous and ulcerative colitis
|
Approved
|
2023-10-02 00:00:00
|
1-ml vial approved October 2, 2023.
| 1 |
AFMD
|
EGFR-expressing tumors, Non-small cell lung cancer, and Squamous cell carcinoma of the head and neck
|
Phase 1
|
2023-08-03 00:00:00
|
Phase 1 dose-escalation update reported that AFM24 showed meaningful clinical activity in heavily pretreated patients with EGFRmut NSCLC (ORR [13%], DCR [47%]) with significant tumor reductions, including two confirmed PRs and five patients exhibiting stable disease, noted August 3, 2023.
| 1 |
ACHV
|
Smoking cessation of cigarettes
|
Phase 3
|
2023-05-23 00:00:00
|
Phase 3 topline results reported that subjects that received treatment had 2.85 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p=0.0008). The smoking cessation rate during weeks 3 through 6 was 14.8% for cytisinicline compared to 6% for placebo, noted May 23, 2023.
| 1 |
HALO
|
Pancreatic cancer
|
Phase 3
|
2019-11-04 00:00:00
|
Phase 3 data November 4, 2019 did not meet primary endpoint.
| 1 |
CDTX
|
Influenza
|
Phase 2a
|
2023-06-22 00:00:00
|
FDA has granted Fast Track designation (FTD), noted June 22, 2023.
| 0 |
REGN
|
Nasal polyps
|
Approved
|
2019-06-26 00:00:00
|
FDA Approval announced June 26, 2019.
| 1 |
BTAI
|
Agitation associated with bipolar I or II disorder or schizophrenia
|
Phase 3
|
2023-05-25 00:00:00
|
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.
| 1 |
XBIO
|
Anemia in patients with chronic kidney disease (CKD)
|
Phase 3
|
2020-12-09 00:00:00
|
Phase 3 data using PolyXen technology reported by partner - non-inferior to Aranesp (darbepoetin alfa) - Russia trial.
| 0 |
AMGN
|
Multiple Myeloma
|
Approved
|
2018-10-01 00:00:00
|
FDA approval announced October 1, 2018.
| 1 |
RHHBY
|
Spinal Muscular Atrophy (SMA) type 2 and 3
|
Phase 2/3
|
2023-03-20 00:00:00
|
Phase 2/3 data reported increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease, noted March 20, 2023.
| 1 |
SCYX
|
Candida Auris
|
Phase 3
|
2021-03-02 00:00:00
|
Phase 3 interim analyses noted 8/10 complete responses - March 2, 2021.
| 0 |
GNLX
|
Malignant pleural effusion (MPE) due to non-small cell lung cancer (NSCLC) or breast cancer.
|
Phase 1
|
2023-07-10 00:00:00
|
Phase 1 expansion data showed a median overall survival (OS) was 19.5 months. The median OS among patients who had malignant pleural mesothelioma (MPM) was 22 months.. There were no dose-limiting toxicities or dose de-escalations, and the maximally tolerated dose was not reached. Therefore, a recommended dose was not established, noted July 10, 2023.
| 1 |
PFE
|
CD20-positive, low tumor burden, follicular lymphoma.
|
Approved
|
2019-07-23 00:00:00
|
FDA approval announced July 23, 2019.
| 1 |
IMAB
|
Growth hormone deficiency
|
Phase 3
|
2023-08-17 00:00:00
|
Phase 3 trial met its primary endpoint, noted August 17, 2023.
| 0 |
AZN
|
Endometrial cancer (EC)
|
Phase 3
|
2023-10-23 00:00:00
|
Phase 3 results presented at ESMO reported statistically significant and clinically meaningful PFS benefit vs CP in the ITT population, with the interim OS data being immature (27.7%) yet with a trend towards benefit, noted October 21, 2023.
| 1 |
OCGN
|
Retinitis Pigmentosa
|
Phase 1/2
|
2023-09-13 00:00:00
|
Phase 1/2 clinical study update suggested continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline, noted September 13, 2023.
| 1 |
CWBR
|
Cancer vaccine for merkel cell carcinoma (MCC)
|
Phase 1b
|
2023-06-05 00:00:00
|
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
| 1 |
KRYS
|
Transglutaminase-1 Deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
|
Phase 2
|
2021-07-01 00:00:00
|
Phase 2 update announced July 1, 2021 - well tolerated with no adverse events.
| 1 |
PTGX
|
Polycythemia vera
|
Phase 2
|
2021-12-12 00:00:00
|
Phase 2 data reported that in all 16 erythrocytotic PV patients, rusfertide demonstrated rapid reduction of hematocrit below 45% within weeks, noted December 12, 2021.
| 1 |
PBYI
|
Breast Cancer
|
Phase 2
|
2023-06-05 00:00:00
|
Phase 2 data reported an increased MYC RNA expression was observed in tumors from patients who did not derive clinical benefit from paclitaxel alone compared to those with benefit from paclitaxel alone, noted June 5, 2023.
| 1 |
BLRX
|
Pancreatic cancer
|
Phase 2
|
2020-12-16 00:00:00
|
Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.
| 0 |
GALT
|
Melanoma
|
Phase 1b
|
2018-09-20 00:00:00
|
Phase 1b data released September 20, 2019. ORR 50%.
| 0 |
RIGL
|
Warm Autoimmune hemolytic anemia (WAIHA)
|
Phase 3
|
2022-12-12 00:00:00
|
Phase 3 data presented at ASH reported that the ORR was 48%, noted December 12, 2022.
| 1 |
PRQR
|
Leber Congenital Amaurosis type 10 (LCA10)
|
Phase 1/2
|
2021-11-18 00:00:00
|
Phase 1/2 data reported that the vast majority of the treated eyes demonstrated responses on multiple endpoints, noted November 18, 2021.
| 1 |
VBIV
|
Hepatitis B vaccine
|
Phase 3
|
2019-06-17 00:00:00
|
Phase 3 data met co-primary endpoints but missed key secondary endpoint.
| 0 |
RHHBY
|
HER2-positive early breast cancer
|
Approved
|
2020-06-29 00:00:00
|
FDA Approval announced June 29, 2020.
| 1 |
OTIC
|
Tinnitus
|
Phase 1/2
|
2021-10-12 00:00:00
|
Phase 1/2 clinical trial results published in Otology & Neurology, a journal in otolaryngology demonstrated that the formulation was well-tolerated and a statistically significant higher proportion of responders than placebo across consecutive visits (Weeks 4 and 8).
| 1 |
HALO
|
Multiple sclerosis (MS) or primary progressive MS
|
Phase 3
|
2023-10-11 00:00:00
|
Phase 3 data reported that treatment was comparable to IV infusion in providing rapid and sustained depletion of B cells and near-complete suppression of MRI lesion activity in the brain over 24 weeks, noted October 11, 2023.
| 1 |
RHHBY
|
Solid tumors with loss of CDKN2A Gene
|
Phase 1
|
2023-05-30 00:00:00
|
Phase 1 basket trial to be terminated, noted May 5, 2023.
| -1 |
REGN
|
Fibrodysplasia ossificans progressiva (FOP)
|
Phase 2
|
2020-01-09 00:00:00
|
Phase 2 trial did not meet primary endpoint p=0.07. Patient-reported flare-ups were reduced by 50% (nominal p=0.03).
| 1 |
MRK
|
Metastatic solid tumors
|
Phase 2
|
2023-10-23 00:00:00
|
Additional Phase 2 data presented at ESMO reported that 10 out of 18 evaluable NSCLC patients (56%) had achieved a partial response as their best overall response and 3 out of 6 evaluable SCCHN patients had achieved partial response as their best overall response, noted October 23, 2023.
| 1 |
PLRX
|
Idiopathic pulmonary fibrosis (IFP)
|
Phase 2a
|
2021-09-07 00:00:00
|
Phase 2a PET positive interim data results released September 7, 2021.
| 0 |
SGEN
|
Non-muscle invasive bladder cancer (NMIBC)
|
Phase 1
|
2023-06-03 00:00:00
|
Phase 1 interim data shared at ASCO reported that of 4 pts receiving 125mg of intravesical EV, 3 achieved CR and continue in response, noted June 3, 2023.
| 1 |
VRTX
|
Cystic fibrosis (CF) in patients aged 12 to <24 months
|
Approved
|
2022-09-02 00:00:00
|
Approved September 2, 2022.
| 1 |
NAMS
|
Alzheimer's Disease
|
Phase 2a
|
2023-09-21 00:00:00
|
Phase 2a data observed reductions of 11% and 12% in 24- and 27-hydroxycholesterol in cerebrospinal fluid (CSF), respectively, indicating potential improvement of cholesterol metabolism in the brain. Observed 8% increase in Aβ42/40 ratio, a key biomarker of AD risk, suggesting improvement in disease pathology, noted September 21, 2023.
| 1 |
ZLAB
|
Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter)
|
Approved
|
2023-05-24 00:00:00
|
Approved May 24, 2023.
| 1 |
CNTB
|
Atopic dermatitis (AD)
|
Phase 2b
|
2023-03-18 00:00:00
|
Phase 2b data reported that baseline median Eczema Area and Severity Index (EASI) was 26.9. 54.5% of patients were considered severe, with a baseline Investigators Global Assessment (IGA) score of 4, noted March 18, 2023.
| 0 |
ADXS
|
Adenoid cystic carcinoma
|
Phase 2
|
2022-06-06 00:00:00
|
Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.
| 1 |
MRK
|
Pneumococcal Conjugate Vaccine
|
Phase 3
|
2021-05-20 00:00:00
|
Phase 3 trial met primary immunogenicity and safety endpoints - May 20, 2021.
| 0 |
QSAM
|
Bone cancer
|
Phase 1
|
2023-08-16 00:00:00
|
Initial Phase 1 data reported that treatment was well-tolerated with no serious adverse events, a 56% - 64% decrease in SUV in patient one, and that the first two patients experienced significant pain relief and improved mobility within a week after treatment, noted August 16, 2023.
| 1 |
ACIU
|
Alzheimer’s disease
|
Phase 2
|
2020-09-23 00:00:00
|
Phase 2 top-line data did not meet endpoints - September 23, 2020.
| 1 |
VINC
|
Hematologic malignancies
|
Phase 1
|
2023-09-14 00:00:00
|
IND cleared August 22, 2023. Phase 1 study dosing commenced, noted on September 14, 2023.
| 0 |
RGLS
|
Autosomal dominant polycystic kidney disease (ADPKD)
|
Phase 1b
|
2021-10-12 00:00:00
|
Termination of development in order to prioritize RGLS8429, noted October 12, 2021.
| -1 |
ZLAB
|
Non-small cell lung cancer (NSCLC)
|
Phase 3
|
2023-10-16 00:00:00
|
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.
| 1 |
AKBA
|
Anemia due to chronic kidney disease (CKD)
|
NDA Filing
|
2023-08-24 00:00:00
|
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.
| 0 |
TBPH
|
Neurogenic orthostatic hypotension (nOH)
|
Phase 3
|
2023-08-28 00:00:00
|
Additional subgroup data reported benefits of ampreloxetine ranged from 0.5 to 2.2 point improvements relative to placebo across all subgroup categories and were demonstrated on the OHSA and OHQ composite scores, noted August 28, 2023.
| 1 |
BGNE
|
Esophageal Squamous Cell Carcinoma (ESCC)
|
Phase 2
|
2023-10-21 00:00:00
|
Phase 2 data presented at ESMO reported that INV-assessed ORR was 30.6% with O+T vs. 20.6% with P+T; hazard ratio (HR) of INV-assessed PFS was 0.93, noted October 21, 2023.
| 1 |
LNTH
|
Left Ventricular Ejection Fraction
|
Phase 3
|
2020-02-03 00:00:00
|
Phase 3 trial did not meet primary endpoint - February 3, 2020.
| 1 |
TYME
|
Pancreatic Cancer
|
Phase 2/3
|
2022-01-26 00:00:00
|
Phase 2/3 additional data reported that overall survival for SM-88 as a monotherapy was lower compared to standard of care chemotherapies, noted January 26, 2022.
| 0 |
FGEN
|
Duchenne Muscular Dystrophy
|
Phase 3
|
2023-08-29 00:00:00
|
Phase 3 topline data reported that the trial did not meet its primary endpoint, noted August 29, 2023.
| 1 |
INMB
|
Immune mediated complications from COVID-19
|
Phase 2
|
2021-11-03 00:00:00
|
Phase 2 trial discontinued, noted November 3, 2021.
| -1 |
MRK
|
Merkel Cell Carcinoma
|
Approved
|
2018-12-19 00:00:00
|
FDA Approval announced December 19, 2018.
| 1 |
SNY
|
Cervical cancer
|
Phase 3
|
2021-03-15 00:00:00
|
Phase 3 trial to be stopped early due to sufficient efficacy. 12 months survival for Libtayo compared to 8.5 months for chemotherapy; hazard ratio (HR): 0.69 - March 15, 2021.
| -1 |
URGN
|
Urothelial Carcinoma
|
Approved
|
2020-04-15 00:00:00
|
FDA Approval announced April 15, 2020.
| 1 |
FHTX
|
Metastatic Uveal Melanoma (mUM)
|
Phase 1
|
2023-10-22 00:00:00
|
Phase 1 clinical data presented at ESMO reported that 1 patient treated at the 7.5 mg QD continuous dose level experienced a DLT of Grade 3 keratitis, noted October 22, 2023.
| 1 |
REGN
|
Cervical cancer
|
Phase 3
|
2021-03-15 00:00:00
|
Phase 3 trial to be stopped early due to sufficient efficacy. 12 months survival for Libtayo compared to 8.5 months for chemotherapy; hazard ratio (HR): 0.69 - March 15, 2021.
| -1 |
BMY
|
First-line Renal cell carcinoma (RCC)
|
Phase 3
|
2021-09-16 00:00:00
|
Additional Phase 3 survival data released at ESMO September 16, 2021. Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy. Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up.
| 1 |
PLX
|
Fabry disease
|
Phase 3
|
2022-03-18 00:00:00
|
Phase 3 final results reported that 30% of patients reported treatment-emergent adverse events (TEAEs), noted March 18, 2022.
| 1 |
LIFE
|
Pulmonary sarcoidosis
|
Phase 1/2
|
2023-05-22 00:00:00
|
Phase 1b/2a data presented at the American Thoracic Society reported that treatment led to an exposure-dependent decrease in the extent of oral corticosteroid usage, increase in lung function as measured by percent predicted forced vital capacity and improvement in clinical symptoms as measured by the King's Sarcoidosis Questionnaire-Lung score, noted May 22, 2023.
| 1 |
NVS
|
Psoriatic arthritis
|
Phase 3
|
2019-11-01 00:00:00
|
Phase 3 data did not meet primary endpoint - October 31, 2019.
| 1 |
LGND
|
Multiple Myeloma
|
Approved
|
2018-10-01 00:00:00
|
FDA approval announced October 1, 2018.
| 1 |
CFRX
|
Gram-negative infections
|
Phase 1a
|
2023-10-16 00:00:00
|
IND safety review completed by FDA on October 16, 2023.
| 0 |
KPTI
|
Myelofibrosis (MF) - previously treated
|
Phase 3
|
2023-06-28 00:00:00
|
Phase 3 dosing commenced, noted June 28, 2023.
| 0 |
IFRX
|
Chronic Autoimmune and Inflammatory Diseases
|
Phase 1
|
2023-09-11 00:00:00
|
Phase 1 SAD results demonstrated an excellent safety and tolerability profile and a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile, confirming INF904´s best-in-class potential, noted September 11, 2023.
| 1 |
BIIB
|
Amyotrophic lateral sclerosis (SOD1-ALS)
|
Approved
|
2023-04-25 00:00:00
|
FDA granted accelerated approval on April 25, 2023.
| 1 |
ESPR
|
Hypercholesterolemia
|
Approved
|
2020-02-21 00:00:00
|
FDA Approval announced February 21, 2020.
| 1 |
AIM
|
Cisplatin resistant advanced recurrent ovarian cancer
|
Phase 1/2
|
2022-01-24 00:00:00
|
Phase 1/2 IP chemo-immunotherapy combination demonstrated to be well tolerated, and associated with interferon stimulated gene changes that favor cytotoxic T lymphocytes chemo-attraction and function, noted January 24, 2022.
| 1 |
SNY
|
Eosinophilic esophagitis (EoE)
|
Approved
|
2022-05-20 00:00:00
|
Approved May 20, 2022.
| 1 |
AMGN
|
Multiple Myeloma
|
Approved
|
2021-12-01 00:00:00
|
Approved December 1, 2021.
| 1 |
DYAI
|
COVID-19 booster vaccine
|
Phase 1
|
2023-07-24 00:00:00
|
Phase 1 safety data reporter no Serious Adverse Events or Adverse Events of Special Interest have been reported to date, noted July 24, 2023
| 0 |
MRK
|
Hepatocellular Carcinoma
|
Phase 3
|
2019-02-19 00:00:00
|
Phase 3 data February 19, 2019 did not meet primary endpoints.
| 1 |
SCYX
|
Vulvovaginal candidiasis (VVC)
|
Approved
|
2021-06-02 00:00:00
|
FDA approval announced June 2, 2021.
| 1 |
RYTM
|
POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders
|
Phase 2
|
2021-09-22 00:00:00
|
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.
| 1 |
BNTC
|
Oculopharyngeal muscular dystrophy (OPMD)
|
Phase 1/2
|
2023-06-26 00:00:00
|
Phase 1b/2a IND clearance on June 26, 2023.
| 0 |
MRNS
|
Focal onset seizures
|
Phase 3
|
2016-06-13 00:00:00
|
Phase 3 data did not meet endpoints - June 13, 2016
| 1 |
PFE
|
Spinal Muscular Atrophy (SMA)
|
Phase 3
|
2023-09-14 00:00:00
|
Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.
| 0 |
COGT
|
Gastrointestinal Stromal Tumors (GIST)
|
Phase 3
|
2023-06-05 00:00:00
|
Phase 3 efficacy data presented at ASCO reported a 55% Disease Control Rate (DCR) in heavily pre-treated GIST patients, including 100% DCR and 17% overall response rate (ORR) in efficacy evaluable 2nd-line GIST patients, noted June 5, 2023.
| 1 |
RVNC
|
Upper limb spasticity
|
Phase 2
|
2021-02-22 00:00:00
|
Phase 2 top-line data released February 22, 2021. One of two co-primary endpoints met (one missed due to reduced enrollment).
| 1 |
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