roymgabriel/BioPharma · Datasets at Hugging Face

ticker stringlengths 2 5	disease stringlengths 3 196	stage stringclasses 15 values	date stringlengths 6 8	catalyst stringlengths 20 963	label int64 4 6
PFE	Non-metastatic high risk hormone-sensitive prostate cancer	Approved	7/13/18	FDA approval announced July 13, 2018.	6
AMTI	Pouchitis	Phase 2	3/3/23	Additional Phase 3 data demonstrated oral's gut-restricted profile with tissue-level pharmacodynamics (PD) effects and no systemic exposure by design. Translational analysis reveals IL-10 biological responses in 3mg and 10mg dose arms, noted March 3, 2023.	6
ARQT	Plaque psoriasis	Phase 3	9/30/21	Phase 3 data met its primary endpoint of Investigator Global Assessment Success rate at week 8 in 37.5% compared to a vehicle rate of 6.9%, noted September 30, 2021.	6
VYGR	Parkinson's Disease	Phase 2	12/22/20	Phase 2 trial placed on clinical hold - December 22, 2020.	4
LLY	Obesity	Phase 3	6/24/23	Phase 3 efficacy and safety data shared at ADA met both co-primary endpoints and all key secondary endpoints compared to placebo for both estimands, with those taking tirzepatide achieving a mean weight reduction of 13.4% on 10 mg and 15.7% on 15 mg compared to 3.3% on placebo for the efficacy estimand, noted June 24, 2023.	6
JNJ	COVID-19 vaccine (single dose)	Phase 3	9/21/21	Phase 3 data showed 75 percent overall efficacy against severe/critical COVID-19, across all ages and countries. 74 percent efficacy in the U.S. against severe/critical COVID-19; 89 percent against hospitalization; 83 percent against COVID-19-related death, September 21, 2021.	6
ANEB	Cannabinoid Intoxication	Phase 2a	3/28/23	Phase 2 met the primary endpoint in all cochorts, noted March 28, 2023	5
NRXP	COVID-19	Phase 3	5/25/22	Phase 3 DSMB review requested that randomization to aviptadil be stopped due to not meeting the futility guidelines, noted May 25, 2022.	6
TAK	Chronic Myeloid Leukemia	Approved	12/18/20	FDA approval announced December 18, 2020.	6
MDWD	Severe burns	CRL	6/29/21	CRL announced June 29, 2021.	5
ACIU	Alzheimer's disease-like characteristics in individuals with Down syndrome	Phase 1/2	1/26/23	Phase 1b/2 trial early results from the first cohort showed that low dose could elicit an anti-Abeta antibody response as soon as week 6 (2 weeks after the second injection). The data show that ACI-24.060 vaccination has been safe and well tolerated to date, noted January 26, 2023	6
VRTX	Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation	Approved	7/2/15	Approved July 2, 2015.	6
AYLA	Adenoid cystic carcinoma	Phase 2	6/6/22	Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.	6
NBTX	Colorectal cancer	Phase 1/2	1/15/21	More than 70% of patients in the study showed objective tumor response after CCRT.	6
CDTX	Influenza	Phase 2a	6/22/23	FDA has granted Fast Track designation (FTD), noted June 22, 2023.	5
BMY	First-line Small cell lung cancer (SCLC)	Phase 3	11/26/18	Phase 3 trial did not meet primary endpoint - November 26, 2018.	4
HRTX	Total knee arthroplasty	Phase 2b	6/21/18	Phase 2 data released June 21, 2018. Primary endpoint met.	6
EXEL	Solid Tumors	Phase 1b	9/10/22	Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.	6
NKTR	Urothelial carcinoma, Melanoma, Renal Cell Carcinoma and Non-Small Cell Lung Cancers	Phase 1/2	11/9/19	Phase 1/2 updated data at SITC November 9, 2019 noted ORR was 53% (20/38) with 34% (13/38) complete response (CR) rate.	5
LLY	Treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC)	Approved	9/21/22	FDA Approval on September 21, 2022.	6
ITRM	Complicated intra-abdominal infections (cIAI)	Phase 3	12/10/19	Phase 3 data missed primary endpoint - December 10, 2019.	4
GLPG	Osteoarthritis	Phase 2	10/15/20	Phase 2 trial did not meet primary endpoint - October 15, 2020.	4
BIIB	Epilepsy	Phase 2	4/22/20	Phase 2 primary endpoint not met.	4
AZN	Chronic obstructive pulmonary disease (COPD)	Phase 3	5/11/18	Phase 3 trial did not meet primary endpoint - noted May 11, 2018.	4
UBX	Diabetic macular edema / Age-related macular degeneration	Phase 2a	4/24/23	Phase 2 48-week data demonstrated that a single injection of UBX1325 treatment led to a statistically significant improvement in vision lasting for the duration of the study (48 weeks), marked by a gain of +6.2 ETDRS letters from baseline, representing a difference of +5.6 ETDRS letters compared to sham-treated patients, noted April 24, 2023.	6
MGTX	Xerostomia	Phase 1	10/24/23	Phase 1 OLE data presented at ESGCT showed that dosing was safe and well-tolerated at all dose levels, with no treatment-related serious adverse events or dose limiting toxicity, noted October 24, 2023.	6
IONS	End-stage renal disease (ESRD)	Phase 2b	11/4/22	Additional Phase 2b data reported that treatment also achieved dose-dependent and sustained median reductions in steady-state FXI levels of 53.1%, 72.2% and 86.6% in the 40 mg, 80 mg, and 120 mg doses of fesomersen, respectively, administered once every 4 weeks, noted November 4, 2022.	6
IBRX	BCG-Unresponsive Bladder Cancer Carcinoma	BLA Filing	10/23/23	BLA Resubmitted to the FDA, noted October 23, 2023.	5
RHHBY	Ulcerative colitis	Phase 3	8/10/20	HIBISCUS I induction study, in people without prior anti-tumor necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint. HIBISCUS II induction study, including people without prior anti-TNF treatment, did not meet its primary endpoint.	5
TPTX	RET-altered NSCLC	Phase 1/2	4/5/21	Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.	5
GSK	Respiratory syncytial virus (RSV) disease, Vaccine	Phase 3	10/25/23	Phase 3 trial met its co-endpoint, eliciting an immune response, noted October 25, 2023.	5
NVS	Gastroenteropancreatic neuroendocrine tumors (GEP-NETs)	Phase 3	9/25/23	Phase 3 trial met primary endpoint of improvement in progression-free survival (PFS) and key secondary endpoint of objective response rate (ORR), noted September 25, 2023.	6
EIGR	Post-Bariatric Hypoglycemia (PBH)	Phase 2	10/16/18	Phase 2 data released October 16, 2018 - endpoints met.	6
REGN	Wet age-related macular degeneration (AMD)	Phase 2	8/10/23	Phase 2 two-year longer-term data demonstrated that the vast majority of aflibercept 8 mg patients with wAMD could maintain or further extend their dosing intervals. Among those who completed the two-year follow-up, noted August 10, 2023.	6
HCM	Neuroendocrine Tumors	Phase 3	1/20/20	Phase 3 China trial stopped early. Primary endpoint met.	6
ATRA	Multiple sclerosis	Phase 2	10/26/22	Phase 2 OLE data reported that all with stable EDSS remaining in the OLE continued to have a stable EDSS for a median of 41.2 months, noted October 26, 2022.	5
GILD	Relapsed or Refractory Mantle Cell Lymphoma (r/r MCL) - cancer	Approved	7/24/20	FDA approval announced July 24, 2020.	6
LCTX	Dry age-related macular degeneration (AMD)	Phase 1/2	5/2/22	Phase 1/2a full results presented at the Association for Research in Vision and Ophthalmology on May 2, 2022. All 24 treated patients reported at least one adverse event (AE) and at least one ocular AE. The majority of AEs reported with OpRegen were mild (Cohort 1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was well tolerated. Preliminary evidence of improvement in visual function was observed in patients with GA and impaired vision at baseline (Cohort 4 [n=12]) Patients in Cohort 4 had an average 7.6 letter gain in visual acuity at 12 months in the study eye Three patients in Cohort 4 (25%) had a 15 letter or greater gain in visual acuity at 12 months in the study eye.	6
HRMY	Osteoarthritis	Phase 2	8/14/17	Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.	4
REGN	Rheumatoid arthritis	Approved	5/22/17	CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.	6
GILD	HIV	Approved	10/18/21	Approved for pediatric patients October 18, 2021.	6
BLTE	Dry Age-Related Macular Degeneration (AMD)	Phase 2/3	4/25/23	Phase 2 data reported that safe and well tolerated in adolescent Stargardt Disease (STGD1), noted April 25, 2023.	6
AZN	Solid tumors	Phase 1	12/9/22	Phase 1 data reported that IPH5201 blocks ATP degradation and blocks CD39-mediated degradation of eATP released after chemotherapy treatment, noted December 8, 2022.	6
MRK	Endometrial cancer	Approved	9/17/19	FDA Approval announced September 17, 2019.	6
GSK	COVID-19 vaccine	Phase 2/3	9/22/21	Phase 2/3 interim analysis showed primary and secondary endpoints met. 100% efficacy against severe COVID-19 & hospitalization and 84% efficacy against moderate-to-severe COVID-19 caused by any strain of SARS-CoV-2 in SPECTRA, and 79% efficacy against COVID-19 of any severity caused by the globally dominant Delta variant, noted September 22, 2021.	6
SCPH	Heart failure	CRL	12/7/20	CRL announced December 7, 2020.	5
IONS	Hereditary Angioedema (HAE)	Phase 2	2/21/23	Phase 2 open-label study data reinforce the treatment's potential to be a best-in-class prophylactic treatment for patients living with hereditary angioedema, noted February 21, 2023.	6
ITCI	Bipolar Depression and Major depressive disorder (MDD)	Phase 3	9/11/23	Phase 3 data continues to demonstrate statical significance. Lumateperone 42mg also met the key secondary endpoint by demonstrating a statistically significant and clinically meaningful reduction in the Clinician's Global Impression scale or CGI compared to placebo at Week 6 in these three populations, noted September 11, 2023.	6
UBX	Osteoarthritis of the knee	Phase 2	8/17/20	Phase 2 top-line data failed to meet primary endpoint - August 17, 2020.	4
BMY	Mantle cell lymphoma (MCL)	Phase 1	6/17/23	Phase 1 data presented at ICML showed that 86.5% of patients in the relapsed or refractory mantle cell lymphoma cohort achieved a response, with 74.3% achieving a complete response, noted June 17, 2023.	6
MRK	Pulmonary arterial hypertension (PAH)	Phase 3	9/11/23	Phase 3 interim results from OLE study represent longest safety and efficacy analysis of sotatercept to date; safety profile of sotatercept consistent with previous studies and efficacy improvements maintained after one year of therapy, noted September 11, 2023.	6
REGN	Asthma - 6-11 year-olds	Approved	10/20/21	Approved October 20, 2021.	6
ZLAB	Non-small-cell lung cancer (NSCLC) and solid tumors	Phase 1/2	4/12/22	Phase 1/2 top-line data reported that in the ROS1-positive TKI-naïve advanced NSCLC population the cORR was 79%, with 4 patients receiving a complete response, and 52 having a partial response. With DOR ranging from 1.4+ to 35.1+ months and PFS ranging from 0+ to 40.4+ months, noted April 12, 2022.	5
TERN	Obesity	Phase 1a	6/26/23	Preclinical data demonstrated the ability of TERN-601 to significantly improve glucose tolerance, suppress food intake and slow gastric emptying in transgenic mice, noted June 26, 2023	6
AZN	Paroxysmal nocturnal hemoglobinuria (PNH)	Phase 3	9/16/22	Phase 3 trial met primary endpoint at interim analysis, noted September 16, 2022.	5
COLL	Opioid dependence	Approved	6/6/14	Approved June 6, 2014.	6
AZN	Chronic lymphocytic leukaemia	Approved	11/21/19	FDA Approval announced November 21, 2019.	6
BCAB	Solid Tumors	Phase 2	1/10/23	Phase 2 part 1 interim results in PD-1 failure NSCLC continues to show strong antitumor activity in a highly refractory population with additional patients enrolled, noted January 10, 2023.	6
ACRS	Healthy volunteers	Phase 1	9/18/23	Phase 1 data reported that treatment was was generally well tolerated at all doses, noted September 18, 2023.	6
AGEN	Soft Tissue Sarcoma	Phase 2	9/17/21	Phase 2 data showed in 125 patients, the objective response rate (ORR) was 26%, with 9% of patients achieving a complete response (CR), and 17% of patients achieving a partial response (PR), noted September 17, 2021.	5
GMAB	Non-Hodgkin lymphoma (NHL)	Phase 1/2	6/9/23	Additional data shared at EHA reported an overall response rate (ORR) of 98 percent and complete metabolic response (CMR) of 87 percent in response evaluable patients, noted June 9, 2023.	6
ALBO	Progressive familial intrahepatic cholestasis (PFIC)	Approved	7/20/21	FDA approval announced July 20, 2021.	6
NVO	Hemophilia A	Phase 1/2	7/11/22	Phase 1/2 trial met primary endpoint, noted July 11, 2022.	5
CRBP	Dermatomyositis	Phase 3	6/24/21	Phase 3 trial did not meet primary endpoint - June 24, 2021.	4
GNPX	Non-small cell lung cancer (NSCLC)	Phase 1	6/3/23	Phase 1 data shared at ASCO reported that treatment was well tolerated, and there were no dose limiting toxicities, noted June 3, 2023.	6
TAK	Acute Graft-Versus-Host Disease (aGvHD) in patients allo-HSCT	Phase 3	2/18/23	Phase 3 study met primary endpoint in lower gastrointestinal (GI) aGvHD-free survival by Day 180 after allo-HSCT with no relevant differences in safety profile versus placebo, noted February 18, 2023.	5
VBIV	Hepatitis B	Phase 2	9/6/23	Phase 2a/2b top-line data showed increased HBsAg loss rate at the end of treatment and 12 weeks follow up, noted September 6, 2023.	6
LGVN	Hypoplastic Left Heart Syndrome	Phase 1b	5/9/23	Phase 1b long term data reported that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B, noted May 9, 2023.	5
RHHBY	Spinal Muscular Atrophy (SMA) in babies	Approved	5/31/22	Approved May 31, 2022.	6
GSK	Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients	CRL	10/26/17	CRL January 11 2017. FDA Approval announced October 25, 2017 following resubmission.	6
ADXS	Adenoid cystic carcinoma	Phase 2	6/6/22	Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.	6
ICPT	FXR Effect on Severe Alcohol-Associated Hepatitis	Phase 1	11/8/22	Phase 1 data reported that treatment was generally well tolerated in healthy subjects. No serious adverse events were reported, noted November 8, 2022.	6
MGTX	RPE65-Deficiency	Phase 1/2	5/14/19	Phase 1/2 data met primary endpoint of safety and tolerability.	6
NGM	Solid Tumors	Phase 1/2	12/8/22	Phase 1/2 data to be presented at the ESMO IO reported that treatment was well tolerated and six patients had reduced target lesion size including a maximum decrease in one patient of 70%, noted December 8, 2022.	6
FATE	Acute graft-versus-host disease (GvHD) undergoing hematopoietic cell transplantation (HCT)	Phase 2	5/5/21	Phase 2 trial did not meet primary endpoint.	4
VRTX	Cystic fibrosis (CF) in patients aged 12 to <24 months	Approved	9/2/22	Approved September 2, 2022.	6
INDV	Opioid Overdose	Approved	5/23/23	Approved May 22, 2023.	6
CLVS	Ovarian Cancer - First-line maintenance treatment	Phase 3	6/6/22	Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.	6
PFE	Persistent Pulmonary Hypertension (PPHN)	Phase 3	6/28/19	Phase 3 trial did not meet primary endpoint - June 28, 2019.	4
CALT	Alport Syndrome	Phase 2	9/27/23	Phase 2 to be intiated in 4Q 2023. FDA granted Orphan Drug Designation, noted September 27, 2023.	5
KZIA	Brain Metastases	Phase 1	7/6/23	FDA awarded Fast Track Designation, noted July 6, 20223.	5
PGEN	Type 1 diabetes	Phase 1/2	10/1/21	Phase 1b interim data showed HbA1c was below the 7% target for 100% of adult patients (9) and 92% of the patients aged 17+ (11). A stabilization of IDAA1c below 9 was demonstrated in 78% of adult patients (7) and in 75% of patients aged 17+ (9). Phase 2a interim data showed stabilization of HbA1c below the ADA target of 7% was demonstrated in 70% of adult patients (7). HbA1c levels were below target in 75% of the adolescent patients (3). Stabilization of IDAA1c was demonstrated in 100% of adult patients (10) and in 75% of adolescent patients (3), noted October 1, 2021.	6
BPMC	Non-advanced systemic mastocytosis (SM)	Approved	5/22/23	FDA approval on May 22, 2023.	6
BTAI	Opioid Withdrawal Symptoms	Phase 1/2	3/31/21	Phase 1b/2 top-line data released March 31, 2021. Primary endpoint met. Retention secondary endpoint not met.	5
HALO	Multiple sclerosis (MS) or primary progressive MS	Phase 3	10/11/23	Phase 3 data reported that treatment was comparable to IV infusion in providing rapid and sustained depletion of B cells and near-complete suppression of MRI lesion activity in the brain over 24 weeks, noted October 11, 2023.	6
ALNA	Enteric Hyperoxaluria	Phase 3	3/18/22	Phase 3 interim analysis due late 1Q 2022. Based on the results by an independent DSMB has recommended that the trial size be increased from the maximum allowed number of 400 subjects under the pre-specified rules. However, even with this maximum recommended sample size increase, the power to detect an effect vs. placebo would still be less than 80% based on the available data. Based upon this recommendation, the company has decided to terminate the study and plans to promptly initiate the process of closing the study.	4
JAZZ	Excessive sleepiness associated with narcolepsy	Approved	3/20/19	FDA Approval announced March 20, 2019.	6
OTLK	Wet age-related macular degeneration (wet AMD)	BLA Filing	11/2/23	The FDA informed the company that an additional adequate and well-controlled clinical trial would be required for the approval of ONS-5010 for wet AMD. Resubmission of the ONS-5010 BLA as early year end 2024, noted November 2, 2023.	6
CLLS	Clear cell renal cell carcinoma (ccRCC)	Phase 1	4/17/23	Phase 1 presentation at AACR reported early anti-tumor activity with deepening responses over time, noted April 17, 2023.	6
BGNE	Follicular Non-Hodgkin Lymphoma	Phase 2	6/9/23	Phase 2 data presented at EHA reported that the OS rate at 24 months was 77.3%(zanubrutinib plus obinutuzumab) and 71.4% (obinutuzumab), with median OS not reached (zanubrutinib plusobinutuzumab) and 34.6 months, noted June 9, 2023.	6
MRK	Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC)	Phase 3	7/24/17	Phase 3 trial did not meet primary endpoint - July 24, 2017. Awarded accelerated approval in 2016.	4
CYDY	Non-alcoholic steatohepatitis (NASH)	Phase 2	1/5/22	Phase 2 trial met primary and secondary endpoint, noted January 5, 2022.	5
RHHBY	HER2-positive early breast cancer	Approved	6/29/20	FDA Approval announced June 29, 2020.	6
RPTX	Solid tumors	Phase 1/2	4/18/23	Phase 1/2 initial data reported a 48% CBR in patients with unmet medical needs and a 32% overall response, 58% CBR and mPFS of approximately 7 months, noted April 18, 2023.	5
CRDF	Pancreatic ductal adenocarcinoma (PDAC)	Phase 2	9/26/23	Phase 2 additional data reported an objective response rate (ORR) of 19% and median progression-free survival (mPFS) of 5.0 months, noted September 26, 2023.	5
ALKS	Solid tumors	Phase 1/2	6/1/22	Phase 1/2 monotherapy data reported that six patients achieved a partial response (PR), of which three were confirmed, with stable disease (SD) observed in 31 patients. Combination data reported two patients achieved a CR, four patients achieved a PR and SD was observed in 14 patients, noted June 1, 2022.	6
SGEN	Untreated locally advanced or metastatic urothelial cancer (la/mUC)	Phase 3	10/23/23	Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.	6
ORMP	Type 2 Diabetes	Phase 3	1/12/23	https://www.biopharmcatalyst.com/company/ORMP/news/110598	5
BIIB	Multiple sclerosis (MS)	Phase 2	10/21/20	Phase 2b trial did not meet primary endpoint.	4
BMY	Relapsed/refractory multiple myeloma	Approved	11/6/18	FDA approval announced November 6, 2018.	6

PFE

Non-metastatic high risk hormone-sensitive prostate cancer

Approved

7/13/18

FDA approval announced July 13, 2018.

6

AMTI

Pouchitis

Phase 2

3/3/23

Additional Phase 3 data demonstrated oral's gut-restricted profile with tissue-level pharmacodynamics (PD) effects and no systemic exposure by design. Translational analysis reveals IL-10 biological responses in 3mg and 10mg dose arms, noted March 3, 2023.

6

ARQT

Plaque psoriasis

Phase 3

9/30/21

Phase 3 data met its primary endpoint of Investigator Global Assessment Success rate at week 8 in 37.5% compared to a vehicle rate of 6.9%, noted September 30, 2021.

6

VYGR

Parkinson's Disease

Phase 2

12/22/20

Phase 2 trial placed on clinical hold - December 22, 2020.

4

LLY

Obesity

Phase 3

6/24/23

Phase 3 efficacy and safety data shared at ADA met both co-primary endpoints and all key secondary endpoints compared to placebo for both estimands, with those taking tirzepatide achieving a mean weight reduction of 13.4% on 10 mg and 15.7% on 15 mg compared to 3.3% on placebo for the efficacy estimand, noted June 24, 2023.

6

JNJ

COVID-19 vaccine (single dose)

Phase 3

9/21/21

Phase 3 data showed 75 percent overall efficacy against severe/critical COVID-19, across all ages and countries. 74 percent efficacy in the U.S. against severe/critical COVID-19; 89 percent against hospitalization; 83 percent against COVID-19-related death, September 21, 2021.

6

ANEB

Cannabinoid Intoxication

Phase 2a

3/28/23

Phase 2 met the primary endpoint in all cochorts, noted March 28, 2023

5

NRXP

COVID-19

Phase 3

5/25/22

Phase 3 DSMB review requested that randomization to aviptadil be stopped due to not meeting the futility guidelines, noted May 25, 2022.

6

TAK

Chronic Myeloid Leukemia

Approved

12/18/20

FDA approval announced December 18, 2020.

6

MDWD

Severe burns

CRL

6/29/21

CRL announced June 29, 2021.

5

ACIU

Alzheimer's disease-like characteristics in individuals with Down syndrome

Phase 1/2

1/26/23

Phase 1b/2 trial early results from the first cohort showed that low dose could elicit an anti-Abeta antibody response as soon as week 6 (2 weeks after the second injection). The data show that ACI-24.060 vaccination has been safe and well tolerated to date, noted January 26, 2023

6

VRTX

Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation

Approved

7/2/15

Approved July 2, 2015.

6

AYLA

Adenoid cystic carcinoma

Phase 2

6/6/22

Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.

6

NBTX

Colorectal cancer

Phase 1/2

1/15/21

More than 70% of patients in the study showed objective tumor response after CCRT.

6

CDTX

Influenza

Phase 2a

6/22/23

FDA has granted Fast Track designation (FTD), noted June 22, 2023.

5

BMY

First-line Small cell lung cancer (SCLC)

Phase 3

11/26/18

Phase 3 trial did not meet primary endpoint - November 26, 2018.

4

HRTX

Total knee arthroplasty

Phase 2b

6/21/18

Phase 2 data released June 21, 2018. Primary endpoint met.

6

EXEL

Solid Tumors

Phase 1b

9/10/22

Phase 1b data presented at ESMO demonstrated preliminary clinical activity similar to that observed with cabozantinib in Phase 1 across a range of solid tumors and dose levels, with a manageable safety profile, noted September 10, 2022.

6

NKTR

Urothelial carcinoma, Melanoma, Renal Cell Carcinoma and Non-Small Cell Lung Cancers

Phase 1/2

11/9/19

Phase 1/2 updated data at SITC November 9, 2019 noted ORR was 53% (20/38) with 34% (13/38) complete response (CR) rate.

5

LLY

Treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC)

Approved

9/21/22

FDA Approval on September 21, 2022.

6

ITRM

Complicated intra-abdominal infections (cIAI)

Phase 3

12/10/19

Phase 3 data missed primary endpoint - December 10, 2019.

4

GLPG

Osteoarthritis

Phase 2

10/15/20

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

4

BIIB

Epilepsy

Phase 2

4/22/20

Phase 2 primary endpoint not met.

4

AZN

Chronic obstructive pulmonary disease (COPD)

Phase 3

5/11/18

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

4

UBX

Diabetic macular edema / Age-related macular degeneration

Phase 2a

4/24/23

Phase 2 48-week data demonstrated that a single injection of UBX1325 treatment led to a statistically significant improvement in vision lasting for the duration of the study (48 weeks), marked by a gain of +6.2 ETDRS letters from baseline, representing a difference of +5.6 ETDRS letters compared to sham-treated patients, noted April 24, 2023.

6

MGTX

Xerostomia

Phase 1

10/24/23

Phase 1 OLE data presented at ESGCT showed that dosing was safe and well-tolerated at all dose levels, with no treatment-related serious adverse events or dose limiting toxicity, noted October 24, 2023.

6

IONS

End-stage renal disease (ESRD)

Phase 2b

11/4/22

Additional Phase 2b data reported that treatment also achieved dose-dependent and sustained median reductions in steady-state FXI levels of 53.1%, 72.2% and 86.6% in the 40 mg, 80 mg, and 120 mg doses of fesomersen, respectively, administered once every 4 weeks, noted November 4, 2022.

6

IBRX

BCG-Unresponsive Bladder Cancer Carcinoma

BLA Filing

10/23/23

BLA Resubmitted to the FDA, noted October 23, 2023.

5

RHHBY

Ulcerative colitis

Phase 3

8/10/20

HIBISCUS I induction study, in people without prior anti-tumor necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint. HIBISCUS II induction study, including people without prior anti-TNF treatment, did not meet its primary endpoint.

5

TPTX

RET-altered NSCLC

Phase 1/2

4/5/21

Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.

5

GSK

Respiratory syncytial virus (RSV) disease, Vaccine

Phase 3

10/25/23

Phase 3 trial met its co-endpoint, eliciting an immune response, noted October 25, 2023.

5

NVS

Gastroenteropancreatic neuroendocrine tumors (GEP-NETs)

Phase 3

9/25/23

Phase 3 trial met primary endpoint of improvement in progression-free survival (PFS) and key secondary endpoint of objective response rate (ORR), noted September 25, 2023.

6

EIGR

Post-Bariatric Hypoglycemia (PBH)

Phase 2

10/16/18

Phase 2 data released October 16, 2018 - endpoints met.

6

REGN

Wet age-related macular degeneration (AMD)

Phase 2

8/10/23

Phase 2 two-year longer-term data demonstrated that the vast majority of aflibercept 8 mg patients with wAMD could maintain or further extend their dosing intervals. Among those who completed the two-year follow-up, noted August 10, 2023.

6

HCM

Neuroendocrine Tumors

Phase 3

1/20/20

Phase 3 China trial stopped early. Primary endpoint met.

6

ATRA

Multiple sclerosis

Phase 2

10/26/22

Phase 2 OLE data reported that all with stable EDSS remaining in the OLE continued to have a stable EDSS for a median of 41.2 months, noted October 26, 2022.

5

GILD

Relapsed or Refractory Mantle Cell Lymphoma (r/r MCL) - cancer

Approved

7/24/20

FDA approval announced July 24, 2020.

6

LCTX

Dry age-related macular degeneration (AMD)

Phase 1/2

5/2/22

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

6

HRMY

Osteoarthritis

Phase 2

8/14/17

Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.

4

REGN

Rheumatoid arthritis

Approved

5/22/17

CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.

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GILD

HIV

Approved

10/18/21

Approved for pediatric patients October 18, 2021.

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BLTE

Dry Age-Related Macular Degeneration (AMD)

Phase 2/3

4/25/23

Phase 2 data reported that safe and well tolerated in adolescent Stargardt Disease (STGD1), noted April 25, 2023.

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AZN

Solid tumors

Phase 1

12/9/22

Phase 1 data reported that IPH5201 blocks ATP degradation and blocks CD39-mediated degradation of eATP released after chemotherapy treatment, noted December 8, 2022.

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MRK

Endometrial cancer

Approved

9/17/19

FDA Approval announced September 17, 2019.

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GSK

COVID-19 vaccine

Phase 2/3

9/22/21

Phase 2/3 interim analysis showed primary and secondary endpoints met. 100% efficacy against severe COVID-19 & hospitalization and 84% efficacy against moderate-to-severe COVID-19 caused by any strain of SARS-CoV-2 in SPECTRA, and 79% efficacy against COVID-19 of any severity caused by the globally dominant Delta variant, noted September 22, 2021.

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SCPH

Heart failure

CRL

12/7/20

CRL announced December 7, 2020.

5

IONS

Hereditary Angioedema (HAE)

Phase 2

2/21/23

Phase 2 open-label study data reinforce the treatment's potential to be a best-in-class prophylactic treatment for patients living with hereditary angioedema, noted February 21, 2023.

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ITCI

Bipolar Depression and Major depressive disorder (MDD)

Phase 3

9/11/23

Phase 3 data continues to demonstrate statical significance. Lumateperone 42mg also met the key secondary endpoint by demonstrating a statistically significant and clinically meaningful reduction in the Clinician's Global Impression scale or CGI compared to placebo at Week 6 in these three populations, noted September 11, 2023.

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UBX

Osteoarthritis of the knee

Phase 2

8/17/20

Phase 2 top-line data failed to meet primary endpoint - August 17, 2020.

4

BMY

Mantle cell lymphoma (MCL)

Phase 1

6/17/23

Phase 1 data presented at ICML showed that 86.5% of patients in the relapsed or refractory mantle cell lymphoma cohort achieved a response, with 74.3% achieving a complete response, noted June 17, 2023.

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MRK

Pulmonary arterial hypertension (PAH)

Phase 3

9/11/23

Phase 3 interim results from OLE study represent longest safety and efficacy analysis of sotatercept to date; safety profile of sotatercept consistent with previous studies and efficacy improvements maintained after one year of therapy, noted September 11, 2023.

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REGN

Asthma - 6-11 year-olds

Approved

10/20/21

Approved October 20, 2021.

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ZLAB

Non-small-cell lung cancer (NSCLC) and solid tumors

Phase 1/2

4/12/22

Phase 1/2 top-line data reported that in the ROS1-positive TKI-naïve advanced NSCLC population the cORR was 79%, with 4 patients receiving a complete response, and 52 having a partial response. With DOR ranging from 1.4+ to 35.1+ months and PFS ranging from 0+ to 40.4+ months, noted April 12, 2022.

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TERN

Obesity

Phase 1a

6/26/23

Preclinical data demonstrated the ability of TERN-601 to significantly improve glucose tolerance, suppress food intake and slow gastric emptying in transgenic mice, noted June 26, 2023

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AZN

Paroxysmal nocturnal hemoglobinuria (PNH)

Phase 3

9/16/22

Phase 3 trial met primary endpoint at interim analysis, noted September 16, 2022.

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COLL

Opioid dependence

Approved

6/6/14

Approved June 6, 2014.

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AZN

Chronic lymphocytic leukaemia

Approved

11/21/19

FDA Approval announced November 21, 2019.

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BCAB

Solid Tumors

Phase 2

1/10/23

Phase 2 part 1 interim results in PD-1 failure NSCLC continues to show strong antitumor activity in a highly refractory population with additional patients enrolled, noted January 10, 2023.

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ACRS

Healthy volunteers

Phase 1

9/18/23

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

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AGEN

Soft Tissue Sarcoma

Phase 2

9/17/21

Phase 2 data showed in 125 patients, the objective response rate (ORR) was 26%, with 9% of patients achieving a complete response (CR), and 17% of patients achieving a partial response (PR), noted September 17, 2021.

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GMAB

Non-Hodgkin lymphoma (NHL)

Phase 1/2

6/9/23

Additional data shared at EHA reported an overall response rate (ORR) of 98 percent and complete metabolic response (CMR) of 87 percent in response evaluable patients, noted June 9, 2023.

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ALBO

Progressive familial intrahepatic cholestasis (PFIC)

Approved

7/20/21

FDA approval announced July 20, 2021.

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NVO

Hemophilia A

Phase 1/2

7/11/22

Phase 1/2 trial met primary endpoint, noted July 11, 2022.

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CRBP

Dermatomyositis

Phase 3

6/24/21

Phase 3 trial did not meet primary endpoint - June 24, 2021.

4

GNPX

Non-small cell lung cancer (NSCLC)

Phase 1

6/3/23

Phase 1 data shared at ASCO reported that treatment was well tolerated, and there were no dose limiting toxicities, noted June 3, 2023.

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TAK

Acute Graft-Versus-Host Disease (aGvHD) in patients allo-HSCT

Phase 3

2/18/23

Phase 3 study met primary endpoint in lower gastrointestinal (GI) aGvHD-free survival by Day 180 after allo-HSCT with no relevant differences in safety profile versus placebo, noted February 18, 2023.

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VBIV

Hepatitis B

Phase 2

9/6/23

Phase 2a/2b top-line data showed increased HBsAg loss rate at the end of treatment and 12 weeks follow up, noted September 6, 2023.

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LGVN

Hypoplastic Left Heart Syndrome

Phase 1b

5/9/23

Phase 1b long term data reported that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B, noted May 9, 2023.

5

RHHBY

Spinal Muscular Atrophy (SMA) in babies

Approved

5/31/22

Approved May 31, 2022.

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GSK

Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients

CRL

10/26/17

CRL January 11 2017. FDA Approval announced October 25, 2017 following resubmission.

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ADXS

Adenoid cystic carcinoma

Phase 2

6/6/22

Phase 2 results reported an overall disease control rate of 69%, 14.6% in the 4mg group achieved a partial response, 8.3% in the 6 mg group, noted June 6, 2022.

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ICPT

FXR Effect on Severe Alcohol-Associated Hepatitis

Phase 1

11/8/22

Phase 1 data reported that treatment was generally well tolerated in healthy subjects. No serious adverse events were reported, noted November 8, 2022.

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MGTX

RPE65-Deficiency

Phase 1/2

5/14/19

Phase 1/2 data met primary endpoint of safety and tolerability.

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NGM

Solid Tumors

Phase 1/2

12/8/22

Phase 1/2 data to be presented at the ESMO IO reported that treatment was well tolerated and six patients had reduced target lesion size including a maximum decrease in one patient of 70%, noted December 8, 2022.

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FATE

Acute graft-versus-host disease (GvHD) undergoing hematopoietic cell transplantation (HCT)

Phase 2

5/5/21

Phase 2 trial did not meet primary endpoint.

4

VRTX

Cystic fibrosis (CF) in patients aged 12 to <24 months

Approved

9/2/22

Approved September 2, 2022.

6

INDV

Opioid Overdose

Approved

5/23/23

Approved May 22, 2023.

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CLVS

Ovarian Cancer - First-line maintenance treatment

Phase 3

6/6/22

Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.

6

PFE

Persistent Pulmonary Hypertension (PPHN)

Phase 3

6/28/19

Phase 3 trial did not meet primary endpoint - June 28, 2019.

4

CALT

Alport Syndrome

Phase 2

9/27/23

Phase 2 to be intiated in 4Q 2023. FDA granted Orphan Drug Designation, noted September 27, 2023.

5

KZIA

Brain Metastases

Phase 1

7/6/23

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

5

PGEN

Type 1 diabetes

Phase 1/2

10/1/21

Phase 1b interim data showed HbA1c was below the 7% target for 100% of adult patients (9) and 92% of the patients aged 17+ (11). A stabilization of IDAA1c below 9 was demonstrated in 78% of adult patients (7) and in 75% of patients aged 17+ (9). Phase 2a interim data showed stabilization of HbA1c below the ADA target of 7% was demonstrated in 70% of adult patients (7). HbA1c levels were below target in 75% of the adolescent patients (3). Stabilization of IDAA1c was demonstrated in 100% of adult patients (10) and in 75% of adolescent patients (3), noted October 1, 2021.

6

BPMC

Non-advanced systemic mastocytosis (SM)

Approved

5/22/23

FDA approval on May 22, 2023.

6

BTAI

Opioid Withdrawal Symptoms

Phase 1/2

3/31/21

Phase 1b/2 top-line data released March 31, 2021. Primary endpoint met. Retention secondary endpoint not met.

5

HALO

Multiple sclerosis (MS) or primary progressive MS

Phase 3

10/11/23

Phase 3 data reported that treatment was comparable to IV infusion in providing rapid and sustained depletion of B cells and near-complete suppression of MRI lesion activity in the brain over 24 weeks, noted October 11, 2023.

6

ALNA

Enteric Hyperoxaluria

Phase 3

3/18/22

Phase 3 interim analysis due late 1Q 2022. Based on the results by an independent DSMB has recommended that the trial size be increased from the maximum allowed number of 400 subjects under the pre-specified rules. However, even with this maximum recommended sample size increase, the power to detect an effect vs. placebo would still be less than 80% based on the available data. Based upon this recommendation, the company has decided to terminate the study and plans to promptly initiate the process of closing the study.

4

JAZZ

Excessive sleepiness associated with narcolepsy

Approved

3/20/19

FDA Approval announced March 20, 2019.

6

OTLK

Wet age-related macular degeneration (wet AMD)

BLA Filing

11/2/23

The FDA informed the company that an additional adequate and well-controlled clinical trial would be required for the approval of ONS-5010 for wet AMD. Resubmission of the ONS-5010 BLA as early year end 2024, noted November 2, 2023.

6

CLLS

Clear cell renal cell carcinoma (ccRCC)

Phase 1

4/17/23

Phase 1 presentation at AACR reported early anti-tumor activity with deepening responses over time, noted April 17, 2023.

6

BGNE

Follicular Non-Hodgkin Lymphoma

Phase 2

6/9/23

Phase 2 data presented at EHA reported that the OS rate at 24 months was 77.3%(zanubrutinib plus obinutuzumab) and 71.4% (obinutuzumab), with median OS not reached (zanubrutinib plusobinutuzumab) and 34.6 months, noted June 9, 2023.

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MRK

Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC)

Phase 3

7/24/17

Phase 3 trial did not meet primary endpoint - July 24, 2017. Awarded accelerated approval in 2016.

4

CYDY

Non-alcoholic steatohepatitis (NASH)

Phase 2

1/5/22

Phase 2 trial met primary and secondary endpoint, noted January 5, 2022.

5

RHHBY

HER2-positive early breast cancer

Approved

6/29/20

FDA Approval announced June 29, 2020.

6

RPTX

Solid tumors

Phase 1/2

4/18/23

Phase 1/2 initial data reported a 48% CBR in patients with unmet medical needs and a 32% overall response, 58% CBR and mPFS of approximately 7 months, noted April 18, 2023.

5

CRDF

Pancreatic ductal adenocarcinoma (PDAC)

Phase 2

9/26/23

Phase 2 additional data reported an objective response rate (ORR) of 19% and median progression-free survival (mPFS) of 5.0 months, noted September 26, 2023.

5

ALKS

Solid tumors

Phase 1/2

6/1/22

Phase 1/2 monotherapy data reported that six patients achieved a partial response (PR), of which three were confirmed, with stable disease (SD) observed in 31 patients. Combination data reported two patients achieved a CR, four patients achieved a PR and SD was observed in 14 patients, noted June 1, 2022.

6

SGEN

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

Phase 3

10/23/23

Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.

6

ORMP

Type 2 Diabetes

Phase 3

1/12/23

https://www.biopharmcatalyst.com/company/ORMP/news/110598

5

BIIB

Multiple sclerosis (MS)

Phase 2

10/21/20

Phase 2b trial did not meet primary endpoint.

4

BMY

Relapsed/refractory multiple myeloma

Approved

11/6/18

FDA approval announced November 6, 2018.

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