quant-chef/BioPharmaCatalysts · Datasets at Hugging Face

ticker stringlengths 2 5	disease stringlengths 3 196	stage stringclasses 15 values	date stringlengths 19 19	catalyst stringlengths 18 760	label int64 -1 1
ADCT	Diffuse Large B-Cell Lymphoma	Approved	2021-04-23 00:00:00	FDA approval announced April 23, 2021.	1
AZN	Metastatic non-squamous non-small cell lung cancer (NSCLC)	Phase 2	2023-09-09 00:00:00	Phase 2 data presented at WCLC reported that objective response rates of 49% and 56% with 5.4 mg/kg and 6.4 mg/kg doses, respectively, in primary analysis, noted September 9, 2023.	1
PRTC	Solid tumors	Phase 1/2	2022-12-12 00:00:00	Phase 1/2 data reported no dose limiting toxicities, noted December 12, 2022.	0
ANAB	Palmo-plantar pustular psoriasis	Phase 2	2021-03-08 00:00:00	Phase 2 trial did not meet primary endpoint - March 8, 2021.	1
TBPH	Asthma	Phase 2	2020-11-05 00:00:00	Phase 2a trial did not meet primary endpoint - November 5, 2020.	1
NVS	Metastatic breast cancer (mBC)	Phase 2	2021-12-10 00:00:00	Phase 2 data reported median overall survival improvement of 26.4 months in cohort A. Primary endpoint was met in cohort C, noted December 10, 2021.	1
MRK	Head and Neck Squamous Cell Carcinoma	Phase 2	2021-11-09 00:00:00	Phase 2 clinical data showed that patients with ≥2L who have not received a prior checkpoint inhibitor ("CPI") (n=10), long-term follow-up data shows that dosing demonstrates a 12-month OS rate of 80% with a mOS of 24.5 months, which compares favorably with standard pembrolizumab therapy in patients with 2L CPI, noted November 9, 2021.	1
CYTK	Chronic obstructive pulmonary disease (COPD)	Phase 2	2018-10-05 00:00:00	Phase 2 data did not meet endpoints - October 5, 2018.	1
NBIX	Endometriosis	Approved	2018-07-24 00:00:00	FDA approval announced July 24, 2018.	1
LCTX	Dry age-related macular degeneration (AMD)	Phase 1/2	2022-05-02 00:00:00	Phase 1/2a full results presented at the Association for Research in Vision and Ophthalmology on May 2, 2022. All 24 treated patients reported at least one adverse event (AE) and at least one ocular AE. The majority of AEs reported with OpRegen were mild (Cohort 1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was well tolerated. Preliminary evidence of improvement in visual function was observed in patients with GA and impaired vision at baseline (Cohort 4 [n=12]) Patients in Cohort 4 had an average 7.6 letter gain in visual acuity at 12 months in the study eye Three patients in Cohort 4 (25%) had a 15 letter or greater gain in visual acuity at 12 months in the study eye.	1
VBIV	Hepatitis B	Phase 2	2023-09-06 00:00:00	Additional Phase 2 data significantly increased HBsAg seroconversion rate is strongly associated with BRII-179 treatment and correlates with the increased rate of HBsAg loss3, noted September 6, 2023.	1
CPRX	Congenital Myasthenic Syndromes (CMS)	Phase 3	2019-10-30 00:00:00	Phase 3 trial did not meet primary endpoint - October 30, 2019.	1
BTAI	Agitation associated with schizophrenia and bipolar disorders	Approved	2022-04-06 00:00:00	Approval announced April 6, 2022.	1
CADL	Prostate Cancer (Active Surveillance)	Phase 2	2021-09-25 00:00:00	Phase 2 safety data displayed 2 grade 4 events, and 3 hospitalizations due to inflection (n=365). The most common AE was flu-like symptoms reported by 21% of patients, noted September 25, 2021.	0
PBYI	HER2 positive breast cancer with brain metastases	Phase 2	2022-12-08 00:00:00	Phase 2 data reported an Objective Response Rate by RANO-BM was 33.3% of patients in cohort 4A, 29.4% in cohort 4B, and 28.6% in cohort 4C, noted December 8, 2022.	0
LLY	Moderate-to-severe atopic dermatitis (AD)	CRL	2023-10-02 00:00:00	CRL issued October 2, 2023.	0
SVRA	Nontuberculous mycobacterial (NTM) lung infection with cystic fibrosis (CF)	Phase 2a	2020-09-03 00:00:00	Phase 2a trial to be discontinued - noted September 3, 2020.	-1
MORF	Ulcerative Colitis (UC)	Phase 1	2022-10-25 00:00:00	Phase 1 additional data showed statistically significant effects on key lymphocyte subsets, noted October 25, 2022.	1
IMVT	Graves Ophthalmopathy / Thyroid Eye Disease (TED)	Phase 2b	2021-02-02 00:00:00	Dosing has paused due to elevated total cholesterol and LDL levels in Phase 2b ASCEND GO-2 trial - February 2, 2021.	-1
STOK	Dravet syndrome	Phase 1/2	2023-07-25 00:00:00	Phase 1/2a OLE OLE data of STK-001 (30mg, 45mg) showed a Sustained reductions in convulsive seizure frequency and improvements in cognition and behavior, noted July 25, 2023.	1
IRWD	Functional Constipation in pediatric patients aged 6-17	Approved	2023-06-12 00:00:00	FDA Approved on June 12, 2023.	1
GSK	Malaria	Approved	2018-07-23 00:00:00	FDA Approval announced July 23, 2018.	1
AFMD	EGFR Expressing Solid Tumors, Non-small cell lung cancer, and Gastric/gastroesophageal junction (GEJ) cancer	Phase 1/2	2022-11-07 00:00:00	Phase 1 data reported one partial response and stable disease, noted November 7, 2022.	0
TEVA	Inflammatory diseases	CRL	2023-06-28 00:00:00	Third CRL issued June 28, 2023. Resubmission planned.	0
MACK	HER2 negative metastatic breast cancer	Phase 2	2018-11-07 00:00:00	Phase 2 trial termination announced November 7, 2018.	-1
MOR	Diffuse Large B-Cell Lymphoma (DLBCL)	Phase 1b	2022-12-12 00:00:00	Phase 1b data reported that ORR at EoT visit and best response across all visits were higher in Arm T/L, as were 18-month DoR, PFS, and OS rates, noted December 12, 2022.	0
MMM	Venous leg ulcers (VLU’s)	Phase 2	2022-05-12 00:00:00	Phase 2 results reported that both primary and key secondary endpoints were met, noted May 12, 2022.	0
ASND	Achondroplasia	Phase 2	2022-11-14 00:00:00	Phase 2 top-line data reported that trial met primary endpoint, noted November 14, 2022.	0
RLMD	Major depressive disorder (MDD)	Phase 3	2022-12-07 00:00:00	Phase 3 trial did not meet primary endpoint, noted December 7, 2022.	1
KMDA	Prophylaxis of rabies disease	Approved	2017-08-25 00:00:00	Approval announced August 25, 2017.	1
ASND	Solid tumors	Phase 1/2	2023-10-26 00:00:00	Phase 1/2 updated data presented at ESMO resulted in clinical responses demonstrated as monotherapy or in combination with checkpoint inhibitor, noted October 26, 2023.	1
PFE	Uterine fibroids	Approved	2021-05-26 00:00:00	FDA approval announced May 26, 2021.	1
ATHA	Alzheimer’s disease	Phase 3	2022-10-17 00:00:00	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.	0
RHHBY	Non-small cell lung cancer (NSCLC)	Approved	2020-05-18 00:00:00	FDA Approval announced May 18, 2020.	1
ALRN	Breast cancer	Phase 1b	2023-02-21 00:00:00	Phase 1b trial terminated following Grade 4 SAE, noted February 21, 2023.	-1
MRK	HIV-1	Phase 3	2021-10-25 00:00:00	Phase 3 data met safety and efficacy endpoints, noted October 25, 2021.	0
BMY	Myelofibrosis	Phase 1/2	2022-12-12 00:00:00	Phase 1/2 results presented at ASH reported that most TRAEs were grade 1/2 in severity, while grade 3 TRAEs were successfully managed with dose delays or modifications without leading to treatment discontinuation, noted December 12, 2022.	1
RHHBY	Urothelial cancer	Phase 3	2020-01-24 00:00:00	Phase 3 trial did not meet primary endpoint - January 24, 2020.	1
GERN	Myelodysplastic syndromes	Phase 3	2023-06-02 00:00:00	Phase 3 data presented at ASCO showed that its primary endpoint of 8-week transfusion independence (TI) continues significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders, noted June 2, 2023	1
AGLE	Arginase I deficiency	Phase 3	2022-04-11 00:00:00	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.	1
NVS	Spinal muscular atrophy (SMA) Type 2	Phase 3	2022-08-12 00:00:00	Phase 3 data reported two patient deaths, noted August 12, 2022	0
CHRS	1L Metastatic Nasopharyngeal Carcinoma	Approved	2023-10-30 00:00:00	Approved October 27, 2023.	1
MRK	Bladder cancer	Approved	2021-08-31 00:00:00	FDA approval announced August 31, 2021.	1
SPPI	Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2	Phase 2	2022-09-10 00:00:00	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.	1
ENDP	Edematous fibrosclerotic panniculopathy (“cellulite”)	Approved	2020-07-06 00:00:00	FDA Approval announced July 6, 2020.	1
GSK	Myelofibrosis	Approved	2023-09-15 00:00:00	Approved September 15, 2023.	1
BMY	Myelofibrosis	Approved	2019-08-16 00:00:00	FDA Approval announced August 16, 2019.	1
VIRI	Long COVID-19	Phase 2	2023-07-17 00:00:00	Phase 2 data reported clinically and statistically significant improvements in fatigue, pain, and symptoms of autonomic dysfunction, noted July 17, 2023.	1
SNY	Diabetes	Approved	2017-12-11 00:00:00	Approval announced December 11, 2017.	1
CLDX	Eosinophilic esophagitis (EoE)	Phase 2	2023-07-06 00:00:00	Phase 2 dosing initiated, noted July 6, 2023.	0
TAK	Renal Cell Carcinoma (RCC)	Phase 3	2022-09-08 00:00:00	Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.	1
HGEN	COVID-19	Phase 3	2022-07-06 00:00:00	Phase 3 data showed a strong correlation between C-reactive protein (CRP) and outcomes with treatment with the greatest clinical benefit experienced by patients with baseline CRP<150 mg/L. In these patients, likelihood of survival without mechanical ventilation (SWOV) was achieved in 90% of patients treated plus standard of care compared to 79% treated with placebo plus standard of care, which was highly statistically significant. Patients had a 62% relative reduction in the risk of progression to invasive mechanical ventilation or death, noted July 6, 2022.	1
ALNY	ATTR Amyloidosis	Phase 3	2022-05-23 00:00:00	Phase 3 18-month data reported improvements in levels of NT-proBNP and a trend towards improvement in echocardiographic parameters, noted May 23, 2022.	1
LLY	Severe hypoglycemia in people with diabetes	Approved	2019-07-24 00:00:00	FDA Approval announced July 24, 2019.	1
UBX	Wet age-related macular degeneration (AMD)	Phase 2	2023-09-27 00:00:00	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.	1
CYCN	Alzheimer's disease with vascular features (ADv)	Phase 2a	2023-05-11 00:00:00	Phase 2a data was inconclusive, noted May 11, 2023.	0
DCPH	Solid tumors	Phase 1	2022-09-10 00:00:00	Phase 1 initial data presented at ESMO demonstrated a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested, noted September 10, 2022	1
HCM	Neuroendocrine Tumors	Phase 3	2020-01-20 00:00:00	Phase 3 China trial stopped early. Primary endpoint met.	-1
JAGX	Cancer related diarrhea (CRD)	Phase 2	2021-12-10 00:00:00	Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.	0
HALO	Testosterone replacement therapy (TRT)	Approved	2022-03-29 00:00:00	Approved March 29, 2022.	1
BHC	Overt hepatic encephalopathy	Phase 2	2020-03-31 00:00:00	Phase 2 data met primary endpoint - March 31, 2020.	0
ALDX	Noninfectious anterior uveitis	Phase 3	2019-06-25 00:00:00	Phase 3 data did not meet endpoints - June 25, 2019.	1
AKBA	Hyperphosphatemia	Approved	2014-09-05 00:00:00	Approved Sept 5, 2014.	1
MDGL	Non-Alcoholic Fatty Liver Disease	Phase 3	2022-06-27 00:00:00	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.	1
KZIA	Brain Metastases	Phase 1	2023-07-06 00:00:00	FDA awarded Fast Track Designation, noted July 6, 20223.	0
IVA	Psoriasis	Phase 2b	2022-10-31 00:00:00	Development stopped noted October 31, 2022.	-1
SGEN	Urothelial cancer	Approved	2019-12-18 00:00:00	FDA Approval announced December 18, 2019.	1
IRON	Erythropoietic Protoporphyria (EPP)	Phase 2	2023-11-02 00:00:00	Phase 2 data from abstract reported that treatment with bitopertin resulted in mean (SD) decreases in PPIX of -39% by Day 43, noted November 2, 2023.	1
ANIP	Osteoporosis	Approved	2023-05-15 00:00:00	ANDA approved May 15, 2023.	1
CTIC	B-cell non-Hodgkin lymphoma	Phase 3	2018-07-09 00:00:00	Phase 3 data released July 10. 2018. Primary endpoint not met.	0
AZN	Biliary-tract cancer	Phase 3	2022-01-18 00:00:00	Phase 3 trial demonstrated a statistically significant and clinically meaningful improvement in of 58% overall survival (OS) and 81% progression-free survival (PFS) versus 66% OS and 86% PFS with chemotherapy alone as a 1st-line treatment for patients with advanced biliary tract cancer (BTC)	1
ADXS	Desmoid tumors	Phase 2/3	2023-10-23 00:00:00	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.	1
DCPH	Solid tumors	Phase 1/2	2021-11-30 00:00:00	Program discontinued, noted November 30, 2021.	-1
ACRS	Healthy volunteers	Phase 1	2023-09-18 00:00:00	Phase 1 data reported that treatment was was generally well tolerated at all doses, noted September 18, 2023.	1
EXEL	Castration-Resistant Prostate Cancer	Phase 3	2023-08-21 00:00:00	Phase 3 data reported that the trial met one of two primary endpoints, demonstrating a statistically significant improvement in progression-free survival (PFS) at the primary analysis, noted August 21, 2023.	1
PFE	HIV	Approved	2020-07-02 00:00:00	FDA Approval announced July 2, 2020.	1
FBIO	X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants	Phase 1/2	2019-04-17 00:00:00	Phase 1/2 trial ongoing. Initial data noted in NEJM.	0
MGNX	Solid tumors	Phase 1	2023-02-14 00:00:00	Phase 1 reported that nine of the 35 patients (25.7%) achieved confirmed partial responses (cPR), noted February 14, 2023.	1
APLS	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Approved	2023-10-02 00:00:00	FDA Approved on October 2, 2023.	1
SCPH	Heart Failure	Phase 3	2021-07-13 00:00:00	Phase 3 data released July 13 2021, study halted early due to highly statistical reduction in costs of $17,753 (p<0.0001) per study subject	1
CMRX	Allogeneic hematopoietic stem cell transplant (HCT) recipients with adenovirus (AdV)	Phase 2	2019-05-09 00:00:00	Phase 2 trial discontinued - May 9, 2019.	-1
BMY	First-line Hepatocellular carcinoma (HCC) - cancer	Phase 3	2019-06-24 00:00:00	Phase 3 trial did not meet primary endpoint.	1
GILD	Osteoarthritis	Phase 2	2020-10-15 00:00:00	Phase 2 trial did not meet primary endpoint - October 15, 2020.	1
ABBV	Endometriosis	Approved	2018-07-24 00:00:00	FDA approval announced July 24, 2018.	1
EYPT	Wet age-related macular degeneration (AMD)	Phase 2	2023-07-27 00:00:00	Phase 2 trial continued to demonstrate that EYP-1901 is well tolerated with no reported drug-related ocular or systemic SAEs, noted July 27, 2023.	1
GILD	Triple-negative breast cancer	Approved	2020-04-22 00:00:00	FDA Approval announced April 22, 2020.	1
ERAS	Solid Tumors	Phase 1b	2023-06-05 00:00:00	Deprioritized as dose escalation safety data do not support continued evaluation of regimen tested, noted June 5, 2023.	1
SRPT	Duchenne muscular dystrophy	Phase 3	2023-10-30 00:00:00	Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.	1
KRYS	Cystic Fibrosis	Phase 1	2023-07-03 00:00:00	Phase 1 dosing commenced, noted July 3, 2023	0
PBLA	Type 1 Diabetes	Phase 2	2023-06-15 00:00:00	Phase 2 abstract presented at ENDO highlighted that β-cell ODC inhibition protects β-cell health and function in the context of the proinflammatory T1D milieu. A 3-month course of oral DFMO was well tolerated with a favorable AE profile in persons with recent-onset T1D. Higher doses were associated with greater β-function compared to placebo. DFMO may have utility as a β-cell preservation therapy in recent-onset T1D, noted June 15, 2023	1
MTCR	Non-alcoholic steatohepatitis (NASH)	Phase 2a	2021-10-21 00:00:00	No pruritus-related treatment discontinuations occurred. MET642 treatment resulted in on-target mean increases in LDL-C of 5 percent with the 3 mg dose and 19 percent with the 6 mg dose, versus a decline of 10 percent with placebo, from baseline to week 16.	1
ALPMY	Untreated locally advanced or metastatic urothelial cancer (la/mUC)	Phase 3	2023-10-23 00:00:00	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.	1
AMGN	Glucocorticoid-induced osteoporosis (GIOP)	Approved	2018-05-21 00:00:00	Approval announced May 21, 2018.	1
GERN	Myelofibrosis (MF)	Phase 2/3	2018-09-27 00:00:00	Janssen noted September 27, 2018 not to continue collaboration.	0
SLGL	Papulopustular rosacea	Phase 3	2019-07-08 00:00:00	Phase 3 top-line data met primary endpoints - July 8, 2019.	0
AZN	Chronic obstructive pulmonary disease (COPD)	Phase 3	2018-05-11 00:00:00	Phase 3 trial did not meet primary endpoint - noted May 11, 2018.	1
BIIB	Acute ischemic stroke	Phase 2b	2018-04-03 00:00:00	Phase 2b data released February 8, 2018 - primary endpoint not met.	0
MRK	Esophageal Cancer	Approved	2021-03-23 00:00:00	FDA approval announced March 23, 2021.	1
PFE	Amyotrophic lateral sclerosis (ALS)	Phase 3	2022-09-29 00:00:00	Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.	1
PRAX	Essential tremor	Phase 2	2023-08-08 00:00:00	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.	1

ADCT

Diffuse Large B-Cell Lymphoma

Approved

2021-04-23 00:00:00

FDA approval announced April 23, 2021.

1

AZN

Metastatic non-squamous non-small cell lung cancer (NSCLC)

Phase 2

2023-09-09 00:00:00

Phase 2 data presented at WCLC reported that objective response rates of 49% and 56% with 5.4 mg/kg and 6.4 mg/kg doses, respectively, in primary analysis, noted September 9, 2023.

1

PRTC

Solid tumors

Phase 1/2

2022-12-12 00:00:00

Phase 1/2 data reported no dose limiting toxicities, noted December 12, 2022.

0

ANAB

Palmo-plantar pustular psoriasis

Phase 2

2021-03-08 00:00:00

Phase 2 trial did not meet primary endpoint - March 8, 2021.

1

TBPH

Asthma

Phase 2

2020-11-05 00:00:00

Phase 2a trial did not meet primary endpoint - November 5, 2020.

1

NVS

Metastatic breast cancer (mBC)

Phase 2

2021-12-10 00:00:00

Phase 2 data reported median overall survival improvement of 26.4 months in cohort A. Primary endpoint was met in cohort C, noted December 10, 2021.

1

MRK

Head and Neck Squamous Cell Carcinoma

Phase 2

2021-11-09 00:00:00

Phase 2 clinical data showed that patients with ≥2L who have not received a prior checkpoint inhibitor ("CPI") (n=10), long-term follow-up data shows that dosing demonstrates a 12-month OS rate of 80% with a mOS of 24.5 months, which compares favorably with standard pembrolizumab therapy in patients with 2L CPI, noted November 9, 2021.

1

CYTK

Chronic obstructive pulmonary disease (COPD)

Phase 2

2018-10-05 00:00:00

Phase 2 data did not meet endpoints - October 5, 2018.

1

NBIX

Endometriosis

Approved

2018-07-24 00:00:00

FDA approval announced July 24, 2018.

1

LCTX

Dry age-related macular degeneration (AMD)

Phase 1/2

2022-05-02 00:00:00

Phase 1/2a full results presented at the Association for Research in Vision and Ophthalmology on May 2, 2022. All 24 treated patients reported at least one adverse event (AE) and at least one ocular AE. The majority of AEs reported with OpRegen were mild (Cohort 1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was well tolerated. Preliminary evidence of improvement in visual function was observed in patients with GA and impaired vision at baseline (Cohort 4 [n=12]) Patients in Cohort 4 had an average 7.6 letter gain in visual acuity at 12 months in the study eye Three patients in Cohort 4 (25%) had a 15 letter or greater gain in visual acuity at 12 months in the study eye.

1

VBIV

Hepatitis B

Phase 2

2023-09-06 00:00:00

Additional Phase 2 data significantly increased HBsAg seroconversion rate is strongly associated with BRII-179 treatment and correlates with the increased rate of HBsAg loss3, noted September 6, 2023.

1

CPRX

Congenital Myasthenic Syndromes (CMS)

Phase 3

2019-10-30 00:00:00

Phase 3 trial did not meet primary endpoint - October 30, 2019.

1

BTAI

Agitation associated with schizophrenia and bipolar disorders

Approved

2022-04-06 00:00:00

Approval announced April 6, 2022.

1

CADL

Prostate Cancer (Active Surveillance)

Phase 2

2021-09-25 00:00:00

Phase 2 safety data displayed 2 grade 4 events, and 3 hospitalizations due to inflection (n=365). The most common AE was flu-like symptoms reported by 21% of patients, noted September 25, 2021.

0

PBYI

HER2 positive breast cancer with brain metastases

Phase 2

2022-12-08 00:00:00

Phase 2 data reported an Objective Response Rate by RANO-BM was 33.3% of patients in cohort 4A, 29.4% in cohort 4B, and 28.6% in cohort 4C, noted December 8, 2022.

0

LLY

Moderate-to-severe atopic dermatitis (AD)

CRL

2023-10-02 00:00:00

CRL issued October 2, 2023.

0

SVRA

Nontuberculous mycobacterial (NTM) lung infection with cystic fibrosis (CF)

Phase 2a

2020-09-03 00:00:00

Phase 2a trial to be discontinued - noted September 3, 2020.

-1

MORF

Ulcerative Colitis (UC)

Phase 1

2022-10-25 00:00:00

Phase 1 additional data showed statistically significant effects on key lymphocyte subsets, noted October 25, 2022.

1

IMVT

Graves Ophthalmopathy / Thyroid Eye Disease (TED)

Phase 2b

2021-02-02 00:00:00

Dosing has paused due to elevated total cholesterol and LDL levels in Phase 2b ASCEND GO-2 trial - February 2, 2021.

-1

STOK

Dravet syndrome

Phase 1/2

2023-07-25 00:00:00

Phase 1/2a OLE OLE data of STK-001 (30mg, 45mg) showed a Sustained reductions in convulsive seizure frequency and improvements in cognition and behavior, noted July 25, 2023.

1

IRWD

Functional Constipation in pediatric patients aged 6-17

Approved

2023-06-12 00:00:00

FDA Approved on June 12, 2023.

1

GSK

Malaria

Approved

2018-07-23 00:00:00

FDA Approval announced July 23, 2018.

1

AFMD

EGFR Expressing Solid Tumors, Non-small cell lung cancer, and Gastric/gastroesophageal junction (GEJ) cancer

Phase 1/2

2022-11-07 00:00:00

Phase 1 data reported one partial response and stable disease, noted November 7, 2022.

0

TEVA

Inflammatory diseases

CRL

2023-06-28 00:00:00

Third CRL issued June 28, 2023. Resubmission planned.

0

MACK

HER2 negative metastatic breast cancer

Phase 2

2018-11-07 00:00:00

Phase 2 trial termination announced November 7, 2018.

-1

MOR

Diffuse Large B-Cell Lymphoma (DLBCL)

Phase 1b

2022-12-12 00:00:00

Phase 1b data reported that ORR at EoT visit and best response across all visits were higher in Arm T/L, as were 18-month DoR, PFS, and OS rates, noted December 12, 2022.

0

MMM

Venous leg ulcers (VLU’s)

Phase 2

2022-05-12 00:00:00

Phase 2 results reported that both primary and key secondary endpoints were met, noted May 12, 2022.

0

ASND

Achondroplasia

Phase 2

2022-11-14 00:00:00

Phase 2 top-line data reported that trial met primary endpoint, noted November 14, 2022.

0

RLMD

Major depressive disorder (MDD)

Phase 3

2022-12-07 00:00:00

Phase 3 trial did not meet primary endpoint, noted December 7, 2022.

1

KMDA

Prophylaxis of rabies disease

Approved

2017-08-25 00:00:00

Approval announced August 25, 2017.

1

ASND

Solid tumors

Phase 1/2

2023-10-26 00:00:00

Phase 1/2 updated data presented at ESMO resulted in clinical responses demonstrated as monotherapy or in combination with checkpoint inhibitor, noted October 26, 2023.

1

PFE

Uterine fibroids

Approved

2021-05-26 00:00:00

FDA approval announced May 26, 2021.

1

ATHA

Alzheimer’s disease

Phase 3

2022-10-17 00:00:00

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.

0

RHHBY

Non-small cell lung cancer (NSCLC)

Approved

2020-05-18 00:00:00

FDA Approval announced May 18, 2020.

1

ALRN

Breast cancer

Phase 1b

2023-02-21 00:00:00

Phase 1b trial terminated following Grade 4 SAE, noted February 21, 2023.

-1

MRK

HIV-1

Phase 3

2021-10-25 00:00:00

Phase 3 data met safety and efficacy endpoints, noted October 25, 2021.

0

BMY

Myelofibrosis

Phase 1/2

2022-12-12 00:00:00

Phase 1/2 results presented at ASH reported that most TRAEs were grade 1/2 in severity, while grade 3 TRAEs were successfully managed with dose delays or modifications without leading to treatment discontinuation, noted December 12, 2022.

1

RHHBY

Urothelial cancer

Phase 3

2020-01-24 00:00:00

Phase 3 trial did not meet primary endpoint - January 24, 2020.

1

GERN

Myelodysplastic syndromes

Phase 3

2023-06-02 00:00:00

Phase 3 data presented at ASCO showed that its primary endpoint of 8-week transfusion independence (TI) continues significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders, noted June 2, 2023

1

AGLE

Arginase I deficiency

Phase 3

2022-04-11 00:00:00

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.

1

NVS

Spinal muscular atrophy (SMA) Type 2

Phase 3

2022-08-12 00:00:00

Phase 3 data reported two patient deaths, noted August 12, 2022

0

CHRS

1L Metastatic Nasopharyngeal Carcinoma

Approved

2023-10-30 00:00:00

Approved October 27, 2023.

1

MRK

Bladder cancer

Approved

2021-08-31 00:00:00

FDA approval announced August 31, 2021.

1

SPPI

Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2

2022-09-10 00:00:00

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.

1

ENDP

Edematous fibrosclerotic panniculopathy (“cellulite”)

Approved

2020-07-06 00:00:00

FDA Approval announced July 6, 2020.

1

GSK

Myelofibrosis

Approved

2023-09-15 00:00:00

Approved September 15, 2023.

1

BMY

Myelofibrosis

Approved

2019-08-16 00:00:00

FDA Approval announced August 16, 2019.

1

VIRI

Long COVID-19

Phase 2

2023-07-17 00:00:00

Phase 2 data reported clinically and statistically significant improvements in fatigue, pain, and symptoms of autonomic dysfunction, noted July 17, 2023.

1

SNY

Diabetes

Approved

2017-12-11 00:00:00

Approval announced December 11, 2017.

1

CLDX

Eosinophilic esophagitis (EoE)

Phase 2

2023-07-06 00:00:00

Phase 2 dosing initiated, noted July 6, 2023.

0

TAK

Renal Cell Carcinoma (RCC)

Phase 3

2022-09-08 00:00:00

Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.

1

HGEN

COVID-19

Phase 3

2022-07-06 00:00:00

Phase 3 data showed a strong correlation between C-reactive protein (CRP) and outcomes with treatment with the greatest clinical benefit experienced by patients with baseline CRP<150 mg/L. In these patients, likelihood of survival without mechanical ventilation (SWOV) was achieved in 90% of patients treated plus standard of care compared to 79% treated with placebo plus standard of care, which was highly statistically significant. Patients had a 62% relative reduction in the risk of progression to invasive mechanical ventilation or death, noted July 6, 2022.

1

ALNY

ATTR Amyloidosis

Phase 3

2022-05-23 00:00:00

Phase 3 18-month data reported improvements in levels of NT-proBNP and a trend towards improvement in echocardiographic parameters, noted May 23, 2022.

1

LLY

Severe hypoglycemia in people with diabetes

Approved

2019-07-24 00:00:00

FDA Approval announced July 24, 2019.

1

UBX

Wet age-related macular degeneration (AMD)

Phase 2

2023-09-27 00:00:00

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.

1

CYCN

Alzheimer's disease with vascular features (ADv)

Phase 2a

2023-05-11 00:00:00

Phase 2a data was inconclusive, noted May 11, 2023.

0

DCPH

Solid tumors

Phase 1

2022-09-10 00:00:00

Phase 1 initial data presented at ESMO demonstrated a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested, noted September 10, 2022

1

HCM

Neuroendocrine Tumors

Phase 3

2020-01-20 00:00:00

Phase 3 China trial stopped early. Primary endpoint met.

-1

JAGX

Cancer related diarrhea (CRD)

Phase 2

2021-12-10 00:00:00

Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.

0

HALO

Testosterone replacement therapy (TRT)

Approved

2022-03-29 00:00:00

Approved March 29, 2022.

1

BHC

Overt hepatic encephalopathy

Phase 2

2020-03-31 00:00:00

Phase 2 data met primary endpoint - March 31, 2020.

0

ALDX

Noninfectious anterior uveitis

Phase 3

2019-06-25 00:00:00

Phase 3 data did not meet endpoints - June 25, 2019.

1

AKBA

Hyperphosphatemia

Approved

2014-09-05 00:00:00

Approved Sept 5, 2014.

1

MDGL

Non-Alcoholic Fatty Liver Disease

Phase 3

2022-06-27 00:00:00

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.

1

KZIA

Brain Metastases

Phase 1

2023-07-06 00:00:00

FDA awarded Fast Track Designation, noted July 6, 20223.

0

IVA

Psoriasis

Phase 2b

2022-10-31 00:00:00

Development stopped noted October 31, 2022.

-1

SGEN

Urothelial cancer

Approved

2019-12-18 00:00:00

FDA Approval announced December 18, 2019.

1

IRON

Erythropoietic Protoporphyria (EPP)

Phase 2

2023-11-02 00:00:00

Phase 2 data from abstract reported that treatment with bitopertin resulted in mean (SD) decreases in PPIX of -39% by Day 43, noted November 2, 2023.

1

ANIP

Osteoporosis

Approved

2023-05-15 00:00:00

ANDA approved May 15, 2023.

1

CTIC

B-cell non-Hodgkin lymphoma

Phase 3

2018-07-09 00:00:00

Phase 3 data released July 10. 2018. Primary endpoint not met.

0

AZN

Biliary-tract cancer

Phase 3

2022-01-18 00:00:00

Phase 3 trial demonstrated a statistically significant and clinically meaningful improvement in of 58% overall survival (OS) and 81% progression-free survival (PFS) versus 66% OS and 86% PFS with chemotherapy alone as a 1st-line treatment for patients with advanced biliary tract cancer (BTC)

1

ADXS

Desmoid tumors

Phase 2/3

2023-10-23 00:00:00

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.

1

DCPH

Solid tumors

Phase 1/2

2021-11-30 00:00:00

Program discontinued, noted November 30, 2021.

-1

ACRS

Healthy volunteers

Phase 1

2023-09-18 00:00:00

Phase 1 data reported that treatment was was generally well tolerated at all doses, noted September 18, 2023.

1

EXEL

Castration-Resistant Prostate Cancer

Phase 3

2023-08-21 00:00:00

Phase 3 data reported that the trial met one of two primary endpoints, demonstrating a statistically significant improvement in progression-free survival (PFS) at the primary analysis, noted August 21, 2023.

1

PFE

HIV

Approved

2020-07-02 00:00:00

FDA Approval announced July 2, 2020.

1

FBIO

X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants

Phase 1/2

2019-04-17 00:00:00

Phase 1/2 trial ongoing. Initial data noted in NEJM.

0

MGNX

Solid tumors

Phase 1

2023-02-14 00:00:00

Phase 1 reported that nine of the 35 patients (25.7%) achieved confirmed partial responses (cPR), noted February 14, 2023.

1

APLS

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Approved

2023-10-02 00:00:00

FDA Approved on October 2, 2023.

1

SCPH

Heart Failure

Phase 3

2021-07-13 00:00:00

Phase 3 data released July 13 2021, study halted early due to highly statistical reduction in costs of $17,753 (p<0.0001) per study subject

1

CMRX

Allogeneic hematopoietic stem cell transplant (HCT) recipients with adenovirus (AdV)

Phase 2

2019-05-09 00:00:00

Phase 2 trial discontinued - May 9, 2019.

-1

BMY

First-line Hepatocellular carcinoma (HCC) - cancer

Phase 3

2019-06-24 00:00:00

Phase 3 trial did not meet primary endpoint.

1

GILD

Osteoarthritis

Phase 2

2020-10-15 00:00:00

Phase 2 trial did not meet primary endpoint - October 15, 2020.

1

ABBV

Endometriosis

Approved

2018-07-24 00:00:00

FDA approval announced July 24, 2018.

1

EYPT

Wet age-related macular degeneration (AMD)

Phase 2

2023-07-27 00:00:00

Phase 2 trial continued to demonstrate that EYP-1901 is well tolerated with no reported drug-related ocular or systemic SAEs, noted July 27, 2023.

1

GILD

Triple-negative breast cancer

Approved

2020-04-22 00:00:00

FDA Approval announced April 22, 2020.

1

ERAS

Solid Tumors

Phase 1b

2023-06-05 00:00:00

Deprioritized as dose escalation safety data do not support continued evaluation of regimen tested, noted June 5, 2023.

1

SRPT

Duchenne muscular dystrophy

Phase 3

2023-10-30 00:00:00

Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.

1

KRYS

Cystic Fibrosis

Phase 1

2023-07-03 00:00:00

Phase 1 dosing commenced, noted July 3, 2023

0

PBLA

Type 1 Diabetes

Phase 2

2023-06-15 00:00:00

Phase 2 abstract presented at ENDO highlighted that β-cell ODC inhibition protects β-cell health and function in the context of the proinflammatory T1D milieu. A 3-month course of oral DFMO was well tolerated with a favorable AE profile in persons with recent-onset T1D. Higher doses were associated with greater β-function compared to placebo. DFMO may have utility as a β-cell preservation therapy in recent-onset T1D, noted June 15, 2023

1

MTCR

Non-alcoholic steatohepatitis (NASH)

Phase 2a

2021-10-21 00:00:00

No pruritus-related treatment discontinuations occurred. MET642 treatment resulted in on-target mean increases in LDL-C of 5 percent with the 3 mg dose and 19 percent with the 6 mg dose, versus a decline of 10 percent with placebo, from baseline to week 16.

1

ALPMY

Untreated locally advanced or metastatic urothelial cancer (la/mUC)

Phase 3

2023-10-23 00:00:00

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.

1

AMGN

Glucocorticoid-induced osteoporosis (GIOP)

Approved

2018-05-21 00:00:00

Approval announced May 21, 2018.

1

GERN

Myelofibrosis (MF)

Phase 2/3

2018-09-27 00:00:00

Janssen noted September 27, 2018 not to continue collaboration.

0

SLGL

Papulopustular rosacea

Phase 3

2019-07-08 00:00:00

Phase 3 top-line data met primary endpoints - July 8, 2019.

0

AZN

Chronic obstructive pulmonary disease (COPD)

Phase 3

2018-05-11 00:00:00

Phase 3 trial did not meet primary endpoint - noted May 11, 2018.

1

BIIB

Acute ischemic stroke

Phase 2b

2018-04-03 00:00:00

Phase 2b data released February 8, 2018 - primary endpoint not met.

0

MRK

Esophageal Cancer

Approved

2021-03-23 00:00:00

FDA approval announced March 23, 2021.

1

PFE

Amyotrophic lateral sclerosis (ALS)

Phase 3

2022-09-29 00:00:00

Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.

1

PRAX

Essential tremor

Phase 2

2023-08-08 00:00:00

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.

1