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
ALNY	Wild-type ATTR amyloidosis patients with cardiomyopathy	CRL	10/9/23	CRL issued by the FDA on October 9, 2023. As a result of the CRL, the Company will no longer pursue an expanded indication for patisiran in the US.	4
CTMX	Solid tumors / Diffuse large B-cell lymphoma (DLBCL), squamous non-small cell lung cancer (sqNSCLC), head and neck squamous cell carcinoma, and esophageal/gastro-esophageal junction (E/GEJ) cancer	Phase 2	12/20/21	Phase 2 initial data reported an objective response rate of 18.8% and disease control rate of 87.5% in squamous non-small cell lung cancer. In advanced head and neck squamous cell carcinoma with objective response rate of 4.0 percent and disease control rate of 56.0 percent, noted December 20, 2021.	5
CYTK	Amyotrophic lateral sclerosis (ALS)	Phase 3	11/21/17	Phase 3 data released November 21, 2017 - primary endpoint not met.	4
RVNC	Cervical dystonia	Phase 3	11/9/21	Open label data reported a median duration of effect ranging from 19.9 weeks to 26.0 weeks across doses. The most common treatment-related adverse events were muscular weakness (4.9%), dysphagia (4.2%) and injection site pain (2.7%), noted November 9, 2021.	6
IPSEY	Castration-Resistant Prostate Cancer	Phase 3	8/21/23	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.	6
XERS	Hypoglycemia	Approved	8/23/21	FDA approval August 23, 2021.	6
SNY	ER+/HER2- advanced breast cancer	Phase 3	8/17/22	Phase 3 trial did not meet the prespecified boundary for continuation following a IDMC review, noted August 17, 2022.	6
MNKD	Pediatric type 1/2 diabetes	Phase 3	4/27/22	Phase 3 proof-of-concept study demonstrated that a simplified 2x dose provided significant reductions in post-prandial glucose excursions (PPGE) versus the current label dosage guidelines, with no new safety concerns, noted April 27, 2022.	6
VIGL	Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP)	Phase 1	12/14/22	Phase 1 trial achieved favorable safety, tolerability and PK profiles in single ascending dose and multiple ascending dose cohorts. VGL101 achieved dose dependent, robust and durable decreases in CSF sTREM2 demonstrating proof of target engagement further validating its mechanism of action, noted December 14, 2022.	6
KNSA	COVID-19-related acute respiratory distress syndrome (ARDS)	Phase 2/3	12/28/21	Phase 3 portion of the trial did not meet the primary efficacy endpoint of proportion of patients alive and free of mechanical ventilation at Day 29.	6
AZN	Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)	Approved	6/27/19	FDA Approval announced June 27, 2019.	6
AUPH	Lupus Nephritis	Phase 3	11/3/23	Phase 3 data from ASN reported that treated patients in the overall AURORA 2 cohort maintained stable renal function over the last two years of the study, as measured by eGFR analysis and experienced numerically greater mean reductions in urine protein creatinine ratio (UPCR), compared to patients in the control arm, noted November 3, 2023.	6
IOVA	Refractory metastatic melanoma	Phase 2	11/10/22	Additional Phase 2 data reported that 42% of responses lasted more than 24 months, noted November 10, 2022.	5
OPTN	Chronic sinusitis	Phase 3	2/16/23	Phase 3 trial met both co-primary endpoints, noted March 7, 2022. Phase 3 additional analysis reported that patients experienced a spectrum of benefits on symptoms and quality of life, as well improvement in objective measures of disease, relative to patients receiving a placebo comparator, noted July 8, 2022. Phase 3 pre-planned analysis of pooled data revealed 66% reduction in sinusitis exacerbations (flares) with treatment compared to placebo, noted July 13, 2022. sNDA submitted to FDA on February 16, 2023	6
VECT	Short bowel syndrome - colon-in-continuity	Phase 2	10/16/23	Additional Phase 2 data reported that 33% of the patients achieving clinical response and PS volume reduction reached a statistically significant 40% at week 24 and the effect was maintained with a 52% volume reduction at week 52, noted October 16, 2023.	6
PRQR	Leber's Congenital Amaurosis (LCA) - Genetic blindness	Phase 2/3	4/13/22	Phase 2/3 analysis found that benefit was observed in mean change from baseline in BCVA, noted April 13, 2022.	6
CUE	HPV+ head and neck squamous cell carcinoma (HNSCC)	Phase 1b	6/5/23	Phase 1b additional data presented at ASCO reported that among 12 evaluable RP2D combination patients to date, 5 PRs, and 2 durable SDs have been observed, with 71% (5/7) of patients expressing PD-L1 CPS ≤ 20, noted June 5, 2023.	6
EBS	Zika vaccine	Phase 1	11/19/18	Phase 1 data released November 19, 2018 - primary endpoint met with favorable safety profile.	6
RDHL	COVID-19	Phase 2/3	2/7/22	Phase 2/3 data reported significantly reduced mortality when given to patients who received standard-of-care (SoC), as well as a significant benefit in time to recovery, noted February 7, 2022.	6
GILD	Diffuse Large B-Cell Lymphoma (DLBCL)	Phase 2	12/13/21	Phase 2 primary analysis reported 89% of evaluable patients achieved a response (ORR), including 78% of patients with a complete response (CR) at a median follow-up of 15.9 months, noted December 13, 2021.	6
REGN	Melanoma	Phase 1	5/25/23	Additional Phase 1 data from ASCO reported an ORR of 63% (25 of 40 patients), including 6 complete responses (CR) and 19 partial responses (PR) in the initial cohort, with a ORR of 63% in the confirmatory cohort, and 56% in the Prior neo/adjuvant systemic therapy cohort, noted May 25, 2023.	5
PULM	Acute migraine	Phase 1	7/11/23	IND submitted to FDA to initiate Phase 2 trial, noted July 11, 2023.	5
NBIX	Tardive dyskinesia	Approved	4/11/17	Approved April 11, 2017.	6
BPTS	Sarcopenia	Phase 2b	10/4/21	Phase 2b data displayed that the highest dose (350 mg bid) showed a clinically meaningful improvement in the 400-Meter Walk Test (400MWT), the primary endpoint of the study, including in sub-populations at higher risk of mobility disability, after 6 months of treatment. Safety profile at the doses of 175 mg bid and of 350 mg bid showed no Serious Adverse Events (SAE) related to treatment, noted October 4, 2021.	6
IMMP	Metastatic breast cancer (MBC)	Phase 2b	5/4/22	Phase 2b biomarker data reported a statistically significant increase in innate and adaptive immune response biomarkers and six patient subgroups with improvements in Overall Survival (OS), noted May 4, 2022.	6
VCEL	Severe burns	CRL	6/29/21	CRL announced June 29, 2021.	5
VIRX	Nasopharyngeal carcinoma and Epstein-Barr Virus-Positive (EBV+) Solid Tumors	Phase 1/2	12/9/22	Preliminary Phase 1b/2 safety data reported no DLTS with 2/6 patients achieving stable disease, noted November 30, 2022.	6
GRTX	Pancreatic Cancer	Phase 2b	10/31/23	Phase 2b did not pass futility analysis, so trial halted to preserve cash, noted October 31, 2023.	6
RCKT	Arrhythmogenic Cardiomyopathy	Phase 1	6/8/23	https://www.biopharmcatalyst.com/company/RCKT/news/140814	5
NVO	Hemophilia A and B	CRL	5/4/23	CRL received noted May 4, 2023.	5
TAK	Crohn's Disease	Phase 3	7/22/19	Phase 3 trial met primary endpoint - July 22, 2020.	6
ALLK	Eosinophilic esophagitis	Phase 2/3	12/21/21	Phase 2/3 top-line data reported that the trial met its histologic co-primary endpoints, but did not achieve statistical significance on the patient reported symptomatic co-primary endpoints, noted December 21, 2021.	6
HOTH	Atopic Dermatitis (AD)	Phase 1b	9/6/23	Phase 1b final results reported that 78% of patients reported that their overall health had improved since starting BioLexa as compared to 22% stating that there was no change in their health, or their health was worse, noted September 6, 2023.	6
PGEN	Myelodysplastic Syndromes, Acute Myeloid Leukemia	Phase 1b	12/13/22	Phase 1/1b data reported a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients, noted December 13, 2022.	6
FGEN	Duchenne Muscular Dystrophy	Phase 3	8/29/23	Phase 3 topline data reported that the trial did not meet its primary endpoint, noted August 29, 2023.	6
PBYI	Extended adjuvant HER2-positive early stage breast cancer	Approved	7/17/17	Approval announced July 17, 2017.	6
BLRX	Pancreatic cancer	Phase 2	12/16/20	Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.	5
STOK	Dravet Syndrome	Phase 1/2	7/25/23	Phase 1/2a new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population, noted July 25, 2024	6
INCY	Graft versus host disease	Approved	5/24/19	FDA approval announced May 24, 2019.	6
NVS	Spinal muscular atrophy (SMA) Type 2	Phase 3	8/12/22	Phase 3 data reported two patient deaths, noted August 12, 2022	5
KPRX	Retinitis Pigmentosa (RP)	Phase 1	5/1/23	Phase 1 abstract presented at ASNR23 on May 1, 2023	6
SNDX	Relapsed/refractory (R/R) acute leukemias	Phase 2	10/2/23	Phase 2 trial met its primary endpoint at the protocol-defined interim analysis stage with a complete remission (CR) or a CR with partial hematological recovery (CRh) rate of 23% among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort, noted October 2, 2023.	6
GILD	Refractory diffuse large B-cell lymphoma (DLBCL)	Approved	4/1/22	Approved April 1, 2022.	6
KDNY	IgA nephropathy	Phase 1/2	6/12/23	Phase 1/2 interim results demonstrated rapid and sustained reductions in mechanistic biomarkers, including IgA and Gd-IgA1 levels, which correspond to clinically meaningful proteinuria reductions in patients with IgAN across Cohorts 1 and 2 due, noted June 12, 2023.	6
ENTA	Hepatitis C virus (HCV)	Approved	8/3/17	Approval announced August 3, 2017.	6
DBTX	Cisplatin-induced hearing loss	Phase 1b	6/1/23	Phase 1b data reported that ear pain (15/19 subjects, 78.9%) and tinnitus (8/19 subjects, 42.1%) were common and the overall study objectives were met at this interim analysis, noted June 1, 2023.	5
IVA	Psoriasis	Phase 2b	10/31/22	Development stopped noted October 31, 2022.	4
EIGR	Pulmonary arterial hypertension (PAH)	Phase 2	1/16/18	Phase 2 trial did not meet endpoints - January 16, 2018.	4
ASLN	Biliary tract cancer	Phase 2/3	11/11/19	Phase 3 trial did not meet primary endpoint - November 11, 2019.	4
TNXP	Post-traumatic stress disorder (PTSD)	Phase 3	7/27/18	Phase 3 trial halted due to insufficient efficacy - July 27, 2018.	4
JAZZ	Spasticity due multiple sclerosis	Phase 2	2/7/17	Phase 2 trial did not meet primary endpoint - noted February 7, 2017.	4
GILD	Higher risk-Myelodysplastic Syndrome (MDS)	Phase 3	7/21/23	Phase 3 ENHANCE study been discontinued due to futility based on a planned analysis, noted July 21, 2023.	4
ISEE	Wet age-related macular degeneration (Wet-AMD)	Phase 3	8/14/17	Phase 3 data released December 12, 2016 - primary endpoint not met. Data from third trial released August 14, 2017- primary endpoint not met.	4
ARQT	Atopic Dermatitis - ages of 2-5 years old	Phase 3	9/19/23	Phase 3 top-line data reported that the trial met all its primary and secondary endpoints, noted September 19, 2023.	5
ESPR	Hypercholesterolemia	Approved	2/21/20	FDA Approval announced February 21, 2020.	6
RLMD	Major Depressive Disorder (MDD)	Phase 3	10/13/22	Phase 3 trial did not meet its primary endpoint, noted October 13, 2022.	6
NVS	Diffuse large B-cell lymphoma (DLBCL) - 2nd line	Phase 3	8/23/21	Phase 3 trial did not meet primary endpoint - August 23, 2021.	4
ADCT	Diffuse Large B-Cell (DLBCL) or Mantle Cell Lymphoma (MCL)	Phase 2	12/13/21	Phase 2 interim data reported an ORR of 57.1% in the overall DLBCL cohort, and 45.5% and 76.9% in the non-GCB DLBCL and GCB DLBCL cohorts, respectively. Median duration of response in the overall DLBCL cohort was 5.49 months and was not reached in the non-GCB DLBCL or GCB DLBCL cohorts, noted December 13, 2021.	6
ATNF	Dupuytren’s disease (DD)	Phase 2/3	12/1/21	Phase 2b data from 2b/3 trial reported that trial met its primary and secondary endpoint with no related severe adverse events, noted December 1, 2021.	6
FDMT	Fabry Disease	Phase 1/2	10/30/23	Phase 1/2 FDA meeting to lift clinical hold and continue trial developments, noted October 30, 2023.	6
MESO	Heart failure	Phase 3	12/6/21	Phase 3 results showed greatest treatment benefit in major adverse cardiovascular events (MACE) of cardiovascular mortality or irreversible morbidity (non-fatal heart attack or stroke) in patients with diabetes and/or myocardial ischemia (72% of total treated population)	6
BMY	Ulcerative colitis (UC)	Phase 2	10/26/23	Phase 2 trial was unsuccessful, noted October 26, 2023.	5
KPTI	Multiple myeloma	Approved	12/18/20	FDA approval announced December 18, 2020.	6
MRNA	Heart Failure	Phase 2a	11/15/21	Phase 2a study met the primary endpoint of safety and tolerability; numerical trends observed in endpoints in the heart failure efficacy domains compared with placebo, including increase in LVEF and patient reported outcomes, noted November 15, 2021.	6
NBIX	Congenital Adrenal Hyperplasia (CAH) - adults	Phase 3	9/12/23	Phase 3 study met its primary endpoint at Week 24, Noted September 12, 2023	5
MNOV	Non-alcoholic fatty liver disease (NAFLD) with Type 2 Diabetes Mellitus and Hypertriglyceridemia	Phase 2	12/7/22	Additional Phase 2 data reported that T2DM group showed a greater reduction in serum triglyceride levels at Week 8 and Mean HDL increase was significantly greater in subjects with T2DM than subjects without T2DM at Week 8, noted December 7, 2022.	6
CFRX	Gram-negative infections	Phase 1a	10/16/23	IND safety review completed by FDA on October 16, 2023.	5
MGTA	Multiple Myeloma	Phase 2	12/14/21	Phase 2 investigator-initiated clinical trial data confirmed previously reported positive topline clinical data where it achieved the primary endpoint for collection of hematopoietic stem cells (HSCs), was well-tolerated and mobilized HSCs which were able to successfully engraft with positive 100-day outcomes.	6
LLY	Type 1 Diabetes	CRL	3/20/20	CRL announced March 20, 2020.	5
OBSV	Uterine fibroids	Phase 3	10/20/21	Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.	5
WINT	Cardiogenic Shock in severe acute heart failure	Phase 2	9/26/22	Phase 2 dose response data demonstrated that study met its primary endpoint of significantly improved systolic blood pressure over the first 6 hours of study drug infusion. The study also met several other secondary endpoint assessments of cardiac function, noted on September 26, 2022.	6
ATOS	Mammographic breast density (MBD)	Phase 2	6/27/19	Phase 2 data met primary endpoint - June 27, 2019. High dropout rate noted.	6
JAZZ	Fibromyalgia	CRL	10/11/10	CRL received October 11, 2010.	5
GSK	Pediatric Lupus Nephritis	Approved	7/27/22	Approved July 27, 2022.	6
BIIB	Alzheimer’s disease	Phase 3	9/13/19	Phase 3 trial discontinued due to unfavorable risk-benefit ratio.	4
GRTX	Non-Small Cell Lung Cancer (NSCLC)	Phase 1/2	10/31/23	Phase 1/2 halted after GRECO-2 failed futility analysis, so trial halted to preserve cash, noted October 31, 2023.	6
CGEM	Acute myeloid leukemia (AML)	Phase 1	6/8/23	Preliminary safety data from an ongoing first-in-human study were published in abstract form as part of the 2023 EHA Congress in June 2023.	5
MRK	Head and neck squamous cell carcinoma (HNSCC)	Phase 2	11/9/21	Phase 2 clinical data showed that patients with 1L HNSCC who have not received prior treatment for their advanced disease (n=13), the dose demonstrates an initial ORR of 38.5% with a 12-month OS rate of 87.5% and mOS not reached in combination with pembrolizumab + 5FU + platinum. These results compare favorably with benchmark survival data from standard pembrolizumab + chemotherapy, noted November 9, 2021.	6
CANF	Psoriasis	Phase 3	6/29/23	Phase 3 top-line data reported that trial met primary endpoint, noted June 29, 2022. Additionally in their Phase 3 the FDA encouraged the Company to enroll adolescent patients due to the strong safety profile of the drug demonstrated over the development history and prior clinical studies, noted June 29, 2023	6
ARWR	Healthy volunteers and Asthma	Phase 1/2	9/11/23	Phase 1/2 preliminary clinical data achieved mean target gene knockdown of up to 90% with a maximum of 95% after a single inhaled administration, noted Septemeber 11, 2023.	6
IDYA	Solid tumors, ovarian cancer	Phase 1	9/11/23	Phase 1 data reported tumor shrinkage observed in multiple HRD solid tumor patients, including an endometrial cancer subject with a first imaging assessment of a partial response and an 87% reduction of the CA-125 tumor marker, noted September 11, 2023.	6
LLY	Moderate-to-severe atopic dermatitis (AD)	CRL	10/2/23	CRL issued October 2, 2023.	5
ADCT	Diffuse Large B-Cell Lymphoma	Approved	4/23/21	FDA approval announced April 23, 2021.	6
HOWL	Solid Tumors	Phase 1b	11/3/23	Phase 1/1b preliminary data provided compelling early evidence of dose-dependent biomarker and antitumor activity in patients, including two patients with ongoing unconfirmed partial responses (uPR) in the highest dose tested to date, cohort 4 (12 mg), noted November 3, 2023.	5
IMGO	Essential Thrombocythemia	Phase 2	12/12/22	Phase 2 data reported that 100% of patients treated with bomedemstat for ≥24 weeks achieved a platelet count in the normal range and 89% (25/28) achieved a durable (≥12 weeks) platelet count, noted December 12, 2022.	6
RHHBY	COVID-19 (Anti-viral antibody)	Phase 2/3	9/30/21	Phase 3 trial met primary endpoint, with 36% reduced risk of death by day 29, and 56% reduced risk in patients who were seronegative. Of 2,007 patients (REGEN-COV=1,340, placebo=667) serious adverse events occurred in 21% REGEN-COV patients and 26% placebo patients, noted September 30, 2021.	6
FBIO	Postoperative pain following bunionectomy surgery	CRL	2/15/22	FDA AdCom Meeting resulted in a 8-14 vote against the question that the application showed that benefits outweighs the risks, noted February 15, 2022.	6
RCUS	Triple negative breast cancer (TNBC) and ovarian cancer	Phase 1	12/9/20	Phase 1 initial data due presented at SABCS Annual Meeting, December 9, 2020. 2/18 response rate (partial responses - PR) for doublet regimen. 5/12 responses for triplet (1 complete response, 4 had a PR).	5
RGNX	Diabetic retinopathy	Phase 2	8/1/23	Phase 2 additional data reported that 20% of patients achieved a >2-step improvement vs. 10% in control and 54% of patients achieved any DRSS improvement vs. 20% in control, noted August 1, 2023.	6
EFTR	relapsed/refractory Acute Myeloid Leukemia (AML)	Phase 1	10/24/23	Phase 1 initiated, noted October 24, 2023.	5
ETON	Seizures/migraine	Approved	11/8/21	FDA Approval November 8, 2021.	6
MRTX	Non-Small Cell Lung Cancer / Colorectal Cancer	Phase 1b	9/10/23	Phase 1/1b two-year follow-up data presented at the World Conference on Lung Cancer reported a durable efficacy with a median overall survival (OS) of 14.1 months and a 2-year OS rate of 31% in patients with previously treated KRASG12C-mutated NSCLC, noted September 10, 2023.	6
MDWD	Venous leg ulcers (VLU’s)	Phase 2	5/12/22	Phase 2 results reported that both primary and key secondary endpoints were met, noted May 12, 2022.	5
PFE	CD20-positive, low tumor burden, follicular lymphoma.	Approved	7/23/19	FDA approval announced July 23, 2019.	6
NCNA	Pancreatic cancer	Phase 3	8/20/19	Phase 3 enrolment suspended due to futility - August 20, 2019.	4
RHHBY	Huntington's Disease	Phase 3	3/22/21	Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.	4
APRE	Solid tumors	Phase 1/2	10/16/23	Phase 1/2a data presented at AACR-NCI-EORTC reported that no dose-limiting toxicities have been reported to date with persistent tumor-reducing effect, noted October 16, 2023.	6
TAK	Frontline Hodgkin lymphoma	Approved	4/4/18	sBLA approval announced March 20, 2018.	6
ITCI	Bipolar depression	Phase 3	7/8/19	Phase 3 trial met primary endpoint - July 8, 2019.	6
AZN	Amyotrophic lateral sclerosis (ALS)	Phase 3	8/20/21	Phase 3 trial discontinued due to lack of efficacy.	4

ALNY

Wild-type ATTR amyloidosis patients with cardiomyopathy

CRL

10/9/23

CRL issued by the FDA on October 9, 2023. As a result of the CRL, the Company will no longer pursue an expanded indication for patisiran in the US.

4

CTMX

Solid tumors / Diffuse large B-cell lymphoma (DLBCL), squamous non-small cell lung cancer (sqNSCLC), head and neck squamous cell carcinoma, and esophageal/gastro-esophageal junction (E/GEJ) cancer

Phase 2

12/20/21

Phase 2 initial data reported an objective response rate of 18.8% and disease control rate of 87.5% in squamous non-small cell lung cancer. In advanced head and neck squamous cell carcinoma with objective response rate of 4.0 percent and disease control rate of 56.0 percent, noted December 20, 2021.

5

CYTK

Amyotrophic lateral sclerosis (ALS)

Phase 3

11/21/17

Phase 3 data released November 21, 2017 - primary endpoint not met.

4

RVNC

Cervical dystonia

Phase 3

11/9/21

Open label data reported a median duration of effect ranging from 19.9 weeks to 26.0 weeks across doses. The most common treatment-related adverse events were muscular weakness (4.9%), dysphagia (4.2%) and injection site pain (2.7%), noted November 9, 2021.

6

IPSEY

Castration-Resistant Prostate Cancer

Phase 3

8/21/23

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.

6

XERS

Hypoglycemia

Approved

8/23/21

FDA approval August 23, 2021.

6

SNY

ER+/HER2- advanced breast cancer

Phase 3

8/17/22

Phase 3 trial did not meet the prespecified boundary for continuation following a IDMC review, noted August 17, 2022.

6

MNKD

Pediatric type 1/2 diabetes

Phase 3

4/27/22

Phase 3 proof-of-concept study demonstrated that a simplified 2x dose provided significant reductions in post-prandial glucose excursions (PPGE) versus the current label dosage guidelines, with no new safety concerns, noted April 27, 2022.

6

VIGL

Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP)

Phase 1

12/14/22

Phase 1 trial achieved favorable safety, tolerability and PK profiles in single ascending dose and multiple ascending dose cohorts. VGL101 achieved dose dependent, robust and durable decreases in CSF sTREM2 demonstrating proof of target engagement further validating its mechanism of action, noted December 14, 2022.

6

KNSA

COVID-19-related acute respiratory distress syndrome (ARDS)

Phase 2/3

12/28/21

Phase 3 portion of the trial did not meet the primary efficacy endpoint of proportion of patients alive and free of mechanical ventilation at Day 29.

6

AZN

Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)

Approved

6/27/19

FDA Approval announced June 27, 2019.

6

AUPH

Lupus Nephritis

Phase 3

11/3/23

Phase 3 data from ASN reported that treated patients in the overall AURORA 2 cohort maintained stable renal function over the last two years of the study, as measured by eGFR analysis and experienced numerically greater mean reductions in urine protein creatinine ratio (UPCR), compared to patients in the control arm, noted November 3, 2023.

6

IOVA

Refractory metastatic melanoma

Phase 2

11/10/22

Additional Phase 2 data reported that 42% of responses lasted more than 24 months, noted November 10, 2022.

5

OPTN

Chronic sinusitis

Phase 3

2/16/23

Phase 3 trial met both co-primary endpoints, noted March 7, 2022. Phase 3 additional analysis reported that patients experienced a spectrum of benefits on symptoms and quality of life, as well improvement in objective measures of disease, relative to patients receiving a placebo comparator, noted July 8, 2022. Phase 3 pre-planned analysis of pooled data revealed 66% reduction in sinusitis exacerbations (flares) with treatment compared to placebo, noted July 13, 2022. sNDA submitted to FDA on February 16, 2023

6

VECT

Short bowel syndrome - colon-in-continuity

Phase 2

10/16/23

Additional Phase 2 data reported that 33% of the patients achieving clinical response and PS volume reduction reached a statistically significant 40% at week 24 and the effect was maintained with a 52% volume reduction at week 52, noted October 16, 2023.

6

PRQR

Leber's Congenital Amaurosis (LCA) - Genetic blindness

Phase 2/3

4/13/22

Phase 2/3 analysis found that benefit was observed in mean change from baseline in BCVA, noted April 13, 2022.

6

CUE

HPV+ head and neck squamous cell carcinoma (HNSCC)

Phase 1b

6/5/23

Phase 1b additional data presented at ASCO reported that among 12 evaluable RP2D combination patients to date, 5 PRs, and 2 durable SDs have been observed, with 71% (5/7) of patients expressing PD-L1 CPS ≤ 20, noted June 5, 2023.

6

EBS

Zika vaccine

Phase 1

11/19/18

Phase 1 data released November 19, 2018 - primary endpoint met with favorable safety profile.

6

RDHL

COVID-19

Phase 2/3

2/7/22

Phase 2/3 data reported significantly reduced mortality when given to patients who received standard-of-care (SoC), as well as a significant benefit in time to recovery, noted February 7, 2022.

6

GILD

Diffuse Large B-Cell Lymphoma (DLBCL)

Phase 2

12/13/21

Phase 2 primary analysis reported 89% of evaluable patients achieved a response (ORR), including 78% of patients with a complete response (CR) at a median follow-up of 15.9 months, noted December 13, 2021.

6

REGN

Melanoma

Phase 1

5/25/23

Additional Phase 1 data from ASCO reported an ORR of 63% (25 of 40 patients), including 6 complete responses (CR) and 19 partial responses (PR) in the initial cohort, with a ORR of 63% in the confirmatory cohort, and 56% in the Prior neo/adjuvant systemic therapy cohort, noted May 25, 2023.

5

PULM

Acute migraine

Phase 1

7/11/23

IND submitted to FDA to initiate Phase 2 trial, noted July 11, 2023.

5

NBIX

Tardive dyskinesia

Approved

4/11/17

Approved April 11, 2017.

6

BPTS

Sarcopenia

Phase 2b

10/4/21

Phase 2b data displayed that the highest dose (350 mg bid) showed a clinically meaningful improvement in the 400-Meter Walk Test (400MWT), the primary endpoint of the study, including in sub-populations at higher risk of mobility disability, after 6 months of treatment. Safety profile at the doses of 175 mg bid and of 350 mg bid showed no Serious Adverse Events (SAE) related to treatment, noted October 4, 2021.

6

IMMP

Metastatic breast cancer (MBC)

Phase 2b

5/4/22

Phase 2b biomarker data reported a statistically significant increase in innate and adaptive immune response biomarkers and six patient subgroups with improvements in Overall Survival (OS), noted May 4, 2022.

6

VCEL

Severe burns

CRL

6/29/21

CRL announced June 29, 2021.

5

VIRX

Nasopharyngeal carcinoma and Epstein-Barr Virus-Positive (EBV+) Solid Tumors

Phase 1/2

12/9/22

Preliminary Phase 1b/2 safety data reported no DLTS with 2/6 patients achieving stable disease, noted November 30, 2022.

6

GRTX

Pancreatic Cancer

Phase 2b

10/31/23

Phase 2b did not pass futility analysis, so trial halted to preserve cash, noted October 31, 2023.

6

RCKT

Arrhythmogenic Cardiomyopathy

Phase 1

6/8/23

https://www.biopharmcatalyst.com/company/RCKT/news/140814

5

NVO

Hemophilia A and B

CRL

5/4/23

CRL received noted May 4, 2023.

5

TAK

Crohn's Disease

Phase 3

7/22/19

Phase 3 trial met primary endpoint - July 22, 2020.

6

ALLK

Eosinophilic esophagitis

Phase 2/3

12/21/21

Phase 2/3 top-line data reported that the trial met its histologic co-primary endpoints, but did not achieve statistical significance on the patient reported symptomatic co-primary endpoints, noted December 21, 2021.

6

HOTH

Atopic Dermatitis (AD)

Phase 1b

9/6/23

Phase 1b final results reported that 78% of patients reported that their overall health had improved since starting BioLexa as compared to 22% stating that there was no change in their health, or their health was worse, noted September 6, 2023.

6

PGEN

Myelodysplastic Syndromes, Acute Myeloid Leukemia

Phase 1b

12/13/22

Phase 1/1b data reported a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients, noted December 13, 2022.

6

FGEN

Duchenne Muscular Dystrophy

Phase 3

8/29/23

Phase 3 topline data reported that the trial did not meet its primary endpoint, noted August 29, 2023.

6

PBYI

Extended adjuvant HER2-positive early stage breast cancer

Approved

7/17/17

Approval announced July 17, 2017.

6

BLRX

Pancreatic cancer

Phase 2

12/16/20

Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.

5

STOK

Dravet Syndrome

Phase 1/2

7/25/23

Phase 1/2a new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population, noted July 25, 2024

6

INCY

Graft versus host disease

Approved

5/24/19

FDA approval announced May 24, 2019.

6

NVS

Spinal muscular atrophy (SMA) Type 2

Phase 3

8/12/22

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

5

KPRX

Retinitis Pigmentosa (RP)

Phase 1

5/1/23

Phase 1 abstract presented at ASNR23 on May 1, 2023

6

SNDX

Relapsed/refractory (R/R) acute leukemias

Phase 2

10/2/23

Phase 2 trial met its primary endpoint at the protocol-defined interim analysis stage with a complete remission (CR) or a CR with partial hematological recovery (CRh) rate of 23% among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort, noted October 2, 2023.

6

GILD

Refractory diffuse large B-cell lymphoma (DLBCL)

Approved

4/1/22

Approved April 1, 2022.

6

KDNY

IgA nephropathy

Phase 1/2

6/12/23

Phase 1/2 interim results demonstrated rapid and sustained reductions in mechanistic biomarkers, including IgA and Gd-IgA1 levels, which correspond to clinically meaningful proteinuria reductions in patients with IgAN across Cohorts 1 and 2 due, noted June 12, 2023.

6

ENTA

Hepatitis C virus (HCV)

Approved

8/3/17

Approval announced August 3, 2017.

6

DBTX

Cisplatin-induced hearing loss

Phase 1b

6/1/23

Phase 1b data reported that ear pain (15/19 subjects, 78.9%) and tinnitus (8/19 subjects, 42.1%) were common and the overall study objectives were met at this interim analysis, noted June 1, 2023.

5

IVA

Psoriasis

Phase 2b

10/31/22

Development stopped noted October 31, 2022.

4

EIGR

Pulmonary arterial hypertension (PAH)

Phase 2

1/16/18

Phase 2 trial did not meet endpoints - January 16, 2018.

4

ASLN

Biliary tract cancer

Phase 2/3

11/11/19

Phase 3 trial did not meet primary endpoint - November 11, 2019.

4

TNXP

Post-traumatic stress disorder (PTSD)

Phase 3

7/27/18

Phase 3 trial halted due to insufficient efficacy - July 27, 2018.

4

JAZZ

Spasticity due multiple sclerosis

Phase 2

2/7/17

Phase 2 trial did not meet primary endpoint - noted February 7, 2017.

4

GILD

Higher risk-Myelodysplastic Syndrome (MDS)

Phase 3

7/21/23

Phase 3 ENHANCE study been discontinued due to futility based on a planned analysis, noted July 21, 2023.

4

ISEE

Wet age-related macular degeneration (Wet-AMD)

Phase 3

8/14/17

Phase 3 data released December 12, 2016 - primary endpoint not met. Data from third trial released August 14, 2017- primary endpoint not met.

4

ARQT

Atopic Dermatitis - ages of 2-5 years old

Phase 3

9/19/23

Phase 3 top-line data reported that the trial met all its primary and secondary endpoints, noted September 19, 2023.

5

ESPR

Hypercholesterolemia

Approved

2/21/20

FDA Approval announced February 21, 2020.

6

RLMD

Major Depressive Disorder (MDD)

Phase 3

10/13/22

Phase 3 trial did not meet its primary endpoint, noted October 13, 2022.

6

NVS

Diffuse large B-cell lymphoma (DLBCL) - 2nd line

Phase 3

8/23/21

Phase 3 trial did not meet primary endpoint - August 23, 2021.

4

ADCT

Diffuse Large B-Cell (DLBCL) or Mantle Cell Lymphoma (MCL)

Phase 2

12/13/21

Phase 2 interim data reported an ORR of 57.1% in the overall DLBCL cohort, and 45.5% and 76.9% in the non-GCB DLBCL and GCB DLBCL cohorts, respectively. Median duration of response in the overall DLBCL cohort was 5.49 months and was not reached in the non-GCB DLBCL or GCB DLBCL cohorts, noted December 13, 2021.

6

ATNF

Dupuytren’s disease (DD)

Phase 2/3

12/1/21

Phase 2b data from 2b/3 trial reported that trial met its primary and secondary endpoint with no related severe adverse events, noted December 1, 2021.

6

FDMT

Fabry Disease

Phase 1/2

10/30/23

Phase 1/2 FDA meeting to lift clinical hold and continue trial developments, noted October 30, 2023.

6

MESO

Heart failure

Phase 3

12/6/21

Phase 3 results showed greatest treatment benefit in major adverse cardiovascular events (MACE) of cardiovascular mortality or irreversible morbidity (non-fatal heart attack or stroke) in patients with diabetes and/or myocardial ischemia (72% of total treated population)

6

BMY

Ulcerative colitis (UC)

Phase 2

10/26/23

Phase 2 trial was unsuccessful, noted October 26, 2023.

5

KPTI

Multiple myeloma

Approved

12/18/20

FDA approval announced December 18, 2020.

6

MRNA

Heart Failure

Phase 2a

11/15/21

Phase 2a study met the primary endpoint of safety and tolerability; numerical trends observed in endpoints in the heart failure efficacy domains compared with placebo, including increase in LVEF and patient reported outcomes, noted November 15, 2021.

6

NBIX

Congenital Adrenal Hyperplasia (CAH) - adults

Phase 3

9/12/23

Phase 3 study met its primary endpoint at Week 24, Noted September 12, 2023

5

MNOV

Non-alcoholic fatty liver disease (NAFLD) with Type 2 Diabetes Mellitus and Hypertriglyceridemia

Phase 2

12/7/22

Additional Phase 2 data reported that T2DM group showed a greater reduction in serum triglyceride levels at Week 8 and Mean HDL increase was significantly greater in subjects with T2DM than subjects without T2DM at Week 8, noted December 7, 2022.

6

CFRX

Gram-negative infections

Phase 1a

10/16/23

IND safety review completed by FDA on October 16, 2023.

5

MGTA

Multiple Myeloma

Phase 2

12/14/21

Phase 2 investigator-initiated clinical trial data confirmed previously reported positive topline clinical data where it achieved the primary endpoint for collection of hematopoietic stem cells (HSCs), was well-tolerated and mobilized HSCs which were able to successfully engraft with positive 100-day outcomes.

6

LLY

Type 1 Diabetes

CRL

3/20/20

CRL announced March 20, 2020.

5

OBSV

Uterine fibroids

Phase 3

10/20/21

Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.

5

WINT

Cardiogenic Shock in severe acute heart failure

Phase 2

9/26/22

Phase 2 dose response data demonstrated that study met its primary endpoint of significantly improved systolic blood pressure over the first 6 hours of study drug infusion. The study also met several other secondary endpoint assessments of cardiac function, noted on September 26, 2022.

6

ATOS

Mammographic breast density (MBD)

Phase 2

6/27/19

Phase 2 data met primary endpoint - June 27, 2019. High dropout rate noted.

6

JAZZ

Fibromyalgia

CRL

10/11/10

CRL received October 11, 2010.

5

GSK

Pediatric Lupus Nephritis

Approved

7/27/22

Approved July 27, 2022.

6

BIIB

Alzheimer’s disease

Phase 3

9/13/19

Phase 3 trial discontinued due to unfavorable risk-benefit ratio.

4

GRTX

Non-Small Cell Lung Cancer (NSCLC)

Phase 1/2

10/31/23

Phase 1/2 halted after GRECO-2 failed futility analysis, so trial halted to preserve cash, noted October 31, 2023.

6

CGEM

Acute myeloid leukemia (AML)

Phase 1

6/8/23

Preliminary safety data from an ongoing first-in-human study were published in abstract form as part of the 2023 EHA Congress in June 2023.

5

MRK

Head and neck squamous cell carcinoma (HNSCC)

Phase 2

11/9/21

Phase 2 clinical data showed that patients with 1L HNSCC who have not received prior treatment for their advanced disease (n=13), the dose demonstrates an initial ORR of 38.5% with a 12-month OS rate of 87.5% and mOS not reached in combination with pembrolizumab + 5FU + platinum. These results compare favorably with benchmark survival data from standard pembrolizumab + chemotherapy, noted November 9, 2021.

6

CANF

Psoriasis

Phase 3

6/29/23

Phase 3 top-line data reported that trial met primary endpoint, noted June 29, 2022. Additionally in their Phase 3 the FDA encouraged the Company to enroll adolescent patients due to the strong safety profile of the drug demonstrated over the development history and prior clinical studies, noted June 29, 2023

6

ARWR

Healthy volunteers and Asthma

Phase 1/2

9/11/23

Phase 1/2 preliminary clinical data achieved mean target gene knockdown of up to 90% with a maximum of 95% after a single inhaled administration, noted Septemeber 11, 2023.

6

IDYA

Solid tumors, ovarian cancer

Phase 1

9/11/23

Phase 1 data reported tumor shrinkage observed in multiple HRD solid tumor patients, including an endometrial cancer subject with a first imaging assessment of a partial response and an 87% reduction of the CA-125 tumor marker, noted September 11, 2023.

6

LLY

Moderate-to-severe atopic dermatitis (AD)

CRL

10/2/23

CRL issued October 2, 2023.

5

ADCT

Diffuse Large B-Cell Lymphoma

Approved

4/23/21

FDA approval announced April 23, 2021.

6

HOWL

Solid Tumors

Phase 1b

11/3/23

Phase 1/1b preliminary data provided compelling early evidence of dose-dependent biomarker and antitumor activity in patients, including two patients with ongoing unconfirmed partial responses (uPR) in the highest dose tested to date, cohort 4 (12 mg), noted November 3, 2023.

5

IMGO

Essential Thrombocythemia

Phase 2

12/12/22

Phase 2 data reported that 100% of patients treated with bomedemstat for ≥24 weeks achieved a platelet count in the normal range and 89% (25/28) achieved a durable (≥12 weeks) platelet count, noted December 12, 2022.

6

RHHBY

COVID-19 (Anti-viral antibody)

Phase 2/3

9/30/21

Phase 3 trial met primary endpoint, with 36% reduced risk of death by day 29, and 56% reduced risk in patients who were seronegative. Of 2,007 patients (REGEN-COV=1,340, placebo=667) serious adverse events occurred in 21% REGEN-COV patients and 26% placebo patients, noted September 30, 2021.

6

FBIO

Postoperative pain following bunionectomy surgery

CRL

2/15/22

FDA AdCom Meeting resulted in a 8-14 vote against the question that the application showed that benefits outweighs the risks, noted February 15, 2022.

6

RCUS

Triple negative breast cancer (TNBC) and ovarian cancer

Phase 1

12/9/20

Phase 1 initial data due presented at SABCS Annual Meeting, December 9, 2020. 2/18 response rate (partial responses - PR) for doublet regimen. 5/12 responses for triplet (1 complete response, 4 had a PR).

5

RGNX

Diabetic retinopathy

Phase 2

8/1/23

Phase 2 additional data reported that 20% of patients achieved a >2-step improvement vs. 10% in control and 54% of patients achieved any DRSS improvement vs. 20% in control, noted August 1, 2023.

6

EFTR

relapsed/refractory Acute Myeloid Leukemia (AML)

Phase 1

10/24/23

Phase 1 initiated, noted October 24, 2023.

5

ETON

Seizures/migraine

Approved

11/8/21

FDA Approval November 8, 2021.

6

MRTX

Non-Small Cell Lung Cancer / Colorectal Cancer

Phase 1b

9/10/23

Phase 1/1b two-year follow-up data presented at the World Conference on Lung Cancer reported a durable efficacy with a median overall survival (OS) of 14.1 months and a 2-year OS rate of 31% in patients with previously treated KRASG12C-mutated NSCLC, noted September 10, 2023.

6

MDWD

Venous leg ulcers (VLU’s)

Phase 2

5/12/22

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

5

PFE

CD20-positive, low tumor burden, follicular lymphoma.

Approved

7/23/19

FDA approval announced July 23, 2019.

6

NCNA

Pancreatic cancer

Phase 3

8/20/19

Phase 3 enrolment suspended due to futility - August 20, 2019.

4

RHHBY

Huntington's Disease

Phase 3

3/22/21

Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.

4

APRE

Solid tumors

Phase 1/2

10/16/23

Phase 1/2a data presented at AACR-NCI-EORTC reported that no dose-limiting toxicities have been reported to date with persistent tumor-reducing effect, noted October 16, 2023.

6

TAK

Frontline Hodgkin lymphoma

Approved

4/4/18

sBLA approval announced March 20, 2018.

6

ITCI

Bipolar depression

Phase 3

7/8/19

Phase 3 trial met primary endpoint - July 8, 2019.

6

AZN

Amyotrophic lateral sclerosis (ALS)

Phase 3

8/20/21

Phase 3 trial discontinued due to lack of efficacy.

4