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
XNCR	COVID-19 antibody	Approved	5/26/21	FDA Emergency Use Authorization filing approval announced May 26, 2021.	6
PFE	Remicade (infliximab) biosimilar	Approved	12/13/17	Approval announced December 13, 2017.	6
GTHX	Metastatic colorectal cancer (mCRC)	Phase 3	2/13/23	Phase 3 topline data reported that achieved its co-primary endpoints related to severe neutropenia but failed to achieve a significant response in overall response rate (ORR) and preliminary measures of survival, noted February 13, 2023.	6
VRDN	Thyroid eye disease (TED)	Phase 1	8/15/22	Phase 1 study demonstrated that treatment achieved a substantially extended half-life of 30-40 days and the feasibility of a convenient, low-volume, subcutaneous injection paradigm of 2mL 300mg, noted August 15, 2022.	6
APLIF	Parasitic and anaerobic bacterial infections	Approved	9/25/23	Approved September 25, 2023.	6
SNY	Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR	Approved	8/10/18	FDA Approval announced August 10, 2018.	6
RNXT	Locally advanced pancreatic cancer (LAPC)	Phase 3	6/29/23	Phase 3 interim data presented at ESMO GI demonstrated an eight-month median PFS benefit, 15 versus 7 months, in delaying the progression of cancer for patients receiving treatment with RenovoGem versus standard-of-care, noted June 29, 2023.	6
IONS	Immunoglobulin A nephropathy (IgAN)	Phase 2	11/7/22	IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months	6
BFRI	Actinic Keratosis	Phase 1	11/21/22	Phase 1 Pharmacokinetics study and results discussed with FDA; to be submitted to FDA along with 3 tubes safety study. Phase 3 study launched, noted November 21, 2022.	5
AZN	Bladder cancer	Phase 3	3/6/20	Phase 3 data did not meet primary endpoint.	4
VNDA	Gastroparesis	Phase 3	2/4/22	Phase 3 data reported that the trial did not meet its primary endpoint, noted February 4, 2022.	6
TERN	Nonalcoholic steatohepatitis (NASH)	Phase 2a	11/12/21	Phase 2a safety and efficacy clinical data reported no differences from placebo in percentage change in low density lipoprotein (LDL) cholesterol or high density lipoprotein (HDL) cholesterol. Reductions in alanine transaminase (ALT) and MRI proton density fat fraction (MRI-PDFF) were reported in the 10 and 15 mg groups, and significant reductions in gamma-glutamyl transferase (GGT) in all dose groups, noted November 12, 2021.	4
ACAD	Parkinson’s disease psychosis (PDP)	Approved	4/29/16	Approved April 29, 2016.	6
GMAB	Cervical Cancer	Approved	9/20/21	Approved September 20, 2021.	6
NKTR	Renal Cell Carcinoma (RCC)	Phase 3	4/14/22	Phase 3 data reported that trial did not met its endpoints of objective response rate (ORR) and overall survival (OS), noted April 14, 2022.	5
GRTS	Solid tumors	Phase 1/2	9/12/22	Phase 2 portion data from Phase 1/2 trial presented at ESMO demonstrated early evidence of efficacy with a 39% molecular response rate in evaluable patients with late-line microsatellite-stable colorectal cancer (MSS-CRC) and non-small cell lung cancer (NSCLC). Data support moving trial into earlier lines of treatment, noted September 12, 2022.	6
CLNN	Parkinson's Disease	Phase 2	8/5/21	Phase 2 trial met primary endpoint - August 5, 2021.	6
SESN	Non-muscle invasive bladder cancer (NMIBC)	CRL	8/13/21	CRL announced August 13, 2021.	5
NVAX	Influenza	Phase 3	3/24/20	Phase 3 top-line data met primary endpoints - March 24, 2020.	6
NVCR	Gastric cancer	Phase 2	6/3/22	Phase 2 data reported a confirmed objective response rate of 50%, with a 10.3 month duration of response and 72% one-year survival rate, noted June 3, 2022.	5
BMRN	Duchenne Muscular Dystrophy (DMD)	CRL	1/14/16	CRL issued January 14 2016.	5
BLUE	TDT and the β0/β0genotype	Phase 3	6/11/20	Phase 3 updated data at EHA June 12, 2020. Six of eight evaluable patients achieved transfusion independence,	6
ZYNE	Adult Epilepsy Patients with Refractory Focal Seizures	Phase 2	8/7/17	Phase 2 data released August 7, 2017 - endpoints not met.	4
CLLS	Multiple myeloma	Phase 1/2	12/13/21	Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.	6
OCS	Diabetic macular edema (DME)	Phase 3	10/6/23	Phase 3 data presented at EURETINA noted that 6 times a day dosing of OCS-01 was a highly effective Loading Dose, noted October 6, 2023.	6
MITO	Mitochondrial disease associated with pathogenic nDNA mutations (nPMD)	Phase 3	12/20/19	Phase 3 data did not meet primary endpoint - December 20, 2019.	4
APTX	Parkinson’s disease cognitive impairment, healthy volunteers	Phase 2	2/27/23	Phase 2 study did not demonstrate sufficient efficacy to support further development, noted February 27, 2023.	6
CDAK	Solid tumors	Phase 1/2	6/30/22	Phase 1/2 data reported that two patients experienced tumor regressions in both injected and non-injected lesions, including a partial response in one patient, noted June 30, 2022.	5
LBPS	Solid tumors	Phase 1/2	3/23/22	Phase 1/2 part B data reported that the renal cell carcinoma (RCC) group met the primary endpoint, noted March 23, 2022.	5
IMTX	Solid tumors	Phase 1b	10/24/23	FDA Granted RMAT on October 24, 2023.	5
VTRS	Multiple sclerosis (MS) - Generic for Copaxone	Approved	10/4/17	Approval announced October 4, 2017.	6
BNTX	COVID-19 Vaccine (Booster)	Phase 1/2	5/23/22	Phase 1/2/3 booster trial in children reported that a 80.3% vaccine efficacy, noted May 23, 2022.	5
ALDX	Psoriasis / atopic asthma / COVID-19	Phase 2	3/29/22	Phase 2 top-line data reported statistically significant improvements in all three indications with no serious adverse events, noted March 29, 2022.	6
PHAR	Activated phosphoinositide 3-kinase delta syndrome (APDS)	Approved	3/24/23	Approved March 24, 2023.	6
BOLT	HER2-Expressing Solid Tumors, gastric cancer, including gastroesophageal junction cancer	Phase 2	10/23/23	Phase 2 updated safety data presented at ESMO supports the selection of 20 mg/kg q2w as the recommended Phase 2 dose (RP2D), noted October 23, 2023.	6
BMY	Spinal Muscular Atrophy (SMA)	Phase 3	9/14/23	Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.	5
ATHX	Acute Respiratory Distress Syndrome	Phase 2	8/6/21	Phase 2 open-label data released August 6, 2021. 20/28 days were ventilator free compared with 11/28 for standard therapy.	6
SNY	Chronic Spontaneous Urticaria (CSU)	Phase 3	2/18/22	Phase 3 trial did not meet the primary endpoint, noted February 18, 2022.	6
CVAC	COVID-19 vaccine	Phase 2/3	10/12/21	CVnCoV withdrawn from regulatory review due to focus on second generation candidate, noted October 12, 2021.	4
BGNE	Cervical Cancer (CC)	Phase 2	10/22/23	Phase 2 data presented at ESMO reported that the ORR was 22.5%, with 13 complete responses; 76.8% had a durable response of ≥6 mo, noted October 22, 2023.	6
INCY	COVID-19 associated cytokine storm	Phase 3	12/14/20	Phase 3 top-line did not meet primary endpoint - December 14, 2020.	4
AVXL	Mild to moderate Alzheimer’s disease	Phase 2/3	9/14/23	Phase 2b/3 full data reported that the least-squares mean (LSM) change from baseline to 48 weeks between the blarcamesine and placebo groups were −1.783; for ADAS-Cog13, and −0.456 for CDR-SB in patients with early Alzheimer's disease, noted September 14, 2023.	5
AGIO	Advanced hematologic malignancies with an IDH2 mutation	Approved	8/1/17	Approval announced August 1, 2017.	6
NOVN	Human papillomavirus (HPV)	Phase 2	11/29/16	Phase 2 data released November 29, 2016. Primary endpoint met.	6
GILD	Non-Small Cell Lung Cancer (NSCLC)	Phase 2	9/11/23	Phase 2 trial data reported that in both cohorts, the ORR was 56%, and DCR was 82%. Median duration of response (DoR) was not reached at the time of data cut-off, and DoR rate at six months was 88% in both cohorts, noted September 11, 2023.	6
PFE	COVID-19 vaccine	Phase 2/3	11/4/22	Phase 2/3 clinical trial demonstrated robust neutralizing immune response one month after a 30-µg booster dose of the COVID-19 vaccine (Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5)). Omicron BA.4/BA.5-neutralizing antibody titers increased 13.2-fold from pre-booster levels in adults older than 55 years of age and 9.5-fold in adults 18 to 55 years of age, noted November 4, 2022.	6
IMGN	Cancer - ovarian cancer and relapsed endometrial cancer	Phase 3	9/28/23	Additional Phase 3 data reported at ESGO noted that in patients with prior PARPi, ORR in the ELAHERE arm was 45%, including 7 CRs, compared to 17%, with no CRs, in the IC chemotherapy arm, noted September 28, 2023.	5
CGEM	Solid tumors	Phase 1	11/3/23	Phase 1 Initial clinical biomarker data demonstrated that CLN-619 increases MICA expression on the tumor cell surface, consistent with previously reported preclinical data and supporting the proposed mechanism of action, noted November 3, 2023.	6
NUVL	ALK non-small cell lung cancer (NSCLC)	Phase 1	10/13/23	Phase 1 trial showed preliminary signs of activity observed in heavily pre-treated patients with ALK-positive NSCLC, including in subgroups of patients who have previously received a 2nd generation ALK TKI and lorlatinib, have brain metastases, or have single or compound ALK resistance mutations, noted October 13, 2023.	6
EQRX	Stage III Non-small cell lung cancer (NSCLC)	Phase 3	8/7/22	Phase 3 data reported that treatment demonstrated a statistically significant improvement in progression-free survival versus placebo, noted August 7, 2022.	6
TARS	Lyme disease	Phase 1	12/15/22	Phase 1 data data showed that treatment was well tolerated and safety data supports progression to Phase 2a (Carpo), noted December 15, 2022.	6
BMY	First-line Renal cell carcinoma (RCC)	Phase 3	9/16/21	Additional Phase 3 survival data released at ESMO September 16, 2021. Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy. Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up.	6
BMY	Chronic lymphocytic leukemia (CLL)	Phase 1/2	6/6/23	Phase 1/2 data presented at ASCO reported that the primary endpoint of CR/CRi rate was met at 18.4%, and the ORR was 42.9% and was not statistically significant, noted June 6, 2023.	6
PLX	Fabry disease	Approved	5/10/23	Approved May 10, 2023.	6
CALT	IgA nephropathy	Approved	12/15/21	Approved December 15, 2021.	6
BMY	Stage IIA to IIIB non-small cell lung cancer (NSCLC)	Phase 3	10/21/23	Phase 3 data presented at ESMO reported that NIVO + chemo/NIVO improved pCR rates (25.3% vs 4.7%; odds ratio, 6.64 [95% CI, 3.40–12.97]) and MPR rates (35.4% vs 12.1%; odds ratio: 4.01 [2.48–6.49]) vs chemo/PBO, noted October 21, 2023.	6
VRDN	Thyroid Eye Disease (TED)	Phase 1/2	4/27/23	Phase 1/2 clinical data reported that 80% (4/5) of VRDN-001 responders showed durability of effects at 12 weeks, noted April 27, 2023.	6
FULC	Facioscapulohumeral muscular dystrophy (FSHD)	Phase 3	9/8/23	Phase 3 enrollment completed, noted September 8, 2023	5
SLS	Hematologic malignancies, acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and lymphoma	Phase 2a	10/30/23	FDA granted fast track designation, noted October 30, 2023.	5
IONS	Chronic hepatitis B virus (CHB)	Phase 2b	6/27/22	Phase 2b data readout reported that up to 68% of patients on NA therapy and up to 65% of patients not on NA achieved HBsAg <100 IU/mL at the end of treatment, noted June 27, 2022.	6
AVIR	COVID-19	Phase 2	10/19/21	Phase 2 trial did not meet primary endpoint, noted October 19, 2021.	4
CLSD	Wet AMD using suprachoroidal delivery	Phase 2	7/30/23	Phase 2 additional data reported meaningful reductions in Anti-VEGF injection burden with stable to improved CRT through month 6, noted July 30, 2023.	6
AZN	Chronic obstructive pulmonary disease (COPD)	Approved	7/24/20	FDA approval announced July 24, 2020.	6
BIIB	Lupus	Phase 2b	10/23/18	Phase 2b data released October 23, 2018 - primary endpoint not met.	5
TAK	Higher-risk myelodysplastic syndromes (HR-MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML)	Phase 3	9/1/21	Phase 3 data did not meet primary endpoint September 1, 2021.	4
IMAB	COVID-19	Phase 2	8/11/21	Phase 2 interim analysis released August 11, 2021. Mechanical ventilation free (MVF) rate (83.6% vs 76.7%) by day 30, lower mortality rate (4.9% vs 13.3%) compared to placebo.	6
CLVS	Ovarian Cancer - First-line maintenance treatment	PDUFA	6/26/23	Company went bankrupt in 2022 not information disclosed about the PDUFA.	5
AZN	Wilson disease	Phase 3	6/23/22	Phase 3 data reported at the International Liver Congress noted greater improvements in neurological scores for those treated with ALXN1840 compared to SoC, however, there were no significant differences between treatment groups observed at 48 weeks, noted June 23, 2022.	6
DRRX	COVID-19 Patients with Acute Liver or Kidney Injury	Phase 2	3/4/21	Phase 2 trial to be discontinued due to limited number of patients eligible or willing to enroll.	4
ADAP	Ovarian Cancer	Phase 1	11/8/22	Phase 1 data reported an increased ORR now 43% in ovarian cancer with one new response, noted November 8, 2022.	5
ABBV	Presbyopia	Approved	10/29/21	Approved October 29, 2021.	6
NVS	Complicated urinary tract infections (cUTI)	Approved	8/29/17	Approved August 29, 2017.	6
SNY	Coronavirus COVID-19	Phase 3	9/1/20	Phase 3 global trial did not meet primary endpoint - September 1, 2020.	4
BCRX	Paroxysmal nocturnal hemoglobinuria (PNH), Healthy Volunteers	Phase 1	10/26/23	Phase 1 PoC trial with PNHl initiated, noted October 26, 2023.	5
MRK	First-line ovarian cancer	Approved	5/8/20	FDA Approval announced May 8, 2020.	6
BMY	Tumors harboring ALK, ROS1, or NTRK1-3 alterations	Phase 1/2	10/23/21	Phase 1/2 early clinical data demonstrated confirmed responses in (3/4) TKI-naïve patients, noted October 23, 2021.	6
CCCC	Non-Small Cell Lung Cancer (NSCLC)	Phase 1a	7/5/23	IND Cleared by FDA, July 5, 2023.	5
ACRS	Common warts (verruca vulgaris)	Phase 2	10/24/19	Phase 3 data met all endpoints - second Phase 3 trial.	6
NAMS	Alzheimer's Disease	Phase 2a	9/21/23	Phase 2a data observed reductions of 11% and 12% in 24- and 27-hydroxycholesterol in cerebrospinal fluid (CSF), respectively, indicating potential improvement of cholesterol metabolism in the brain. Observed 8% increase in Aβ42/40 ratio, a key biomarker of AD risk, suggesting improvement in disease pathology, noted September 21, 2023.	6
ALBO	Alagille syndrome (ALGS)	Approved	6/13/23	FDA Approved on June 13, 2023.	6
CDXS	Exocrine pancreatic insufficiency (EPI)	Phase 1	2/23/23	Phase 1 interim data reported that no safety issues occurred, with no SAEs or discontinuations, noted February 23, 2023.	4
PLX	Fabry disease	Phase 3	2/10/21	Phase 3 final data presented February 10, 2021 - key objectives for safety and efficacy.	5
MRK	First-line ovarian cancer following platinum-based chemotherapy	Approved	12/19/18	FDA Approval announced December 19, 2018.	6
LLY	Hepatocellular Carcinoma	Approved	5/13/19	FDA approval announced May 13, 2019.	6
ONCY	Breast cancer	Phase 1	12/8/22	Additional dat presented at the San Antonio Breast Cancer Symposium (SABCS) reported that disease control, partial response (PR) or stable disease (SD), was achieved in thirteen of fourteen evaluable patients (93%), with twelve (86%) showing tumor shrinkage from baseline, with 50% achievign a partial response, noted December 8, 2022.	6
GSK	HIV	Approved	7/2/20	FDA Approval announced July 2, 2020.	6
JAZZ	COVID-19	Phase 2	12/13/21	Phase 2 data reported that treatment in patients with grade 5 WHO COVID 19 ARDS does not induce bleeding, and is associated with rapid restoration of respiratory function (73% of patients). No oxygen support was needed at discharge and a 1-month OS rate of 89% was observed, which is higher than historical controls (77%) treated in the same setting, noted December 13, 2021.	6
BMY	Non-Small Cell Lung Cancer (NSCLC)	Phase 3	9/11/23	Phase 3 data demonstrated consistent, durable benefits of Opdivo plus Yervoy vs. chemotherapy at six years, regardless of PD-L1 expression, noted September 11, 2023.	6
ASLN	Gastric cancer	Phase 2	1/13/19	Phase 2 trial did not meet primary endpoint - January 13, 2019.	4
RHHBY	Babies with pre-symptomatic spinal muscular atrophy (SMA)	Phase 3	10/4/23	Phase 3 OLE met its primary endpoint with 80% of babies sitting without support for at least 5 seconds after 1 year of Evrysdi, noted October 4, 2023.	5
EIGR	Hepatitis delta virus (HDV)	Phase 2	11/12/19	Phase 2 data presented at AASLD November 12, 2019 - Median HDV RNA decline was 3.4 log IU/mL (IQR: 2.9-4.5, p<0.0001).	6
GSK	dMMR endometrial cancer	Approved	2/10/23	Full approval granted February 10, 2023.	6
CGTX	Mild-to-Moderate Alzheimer's Disease	Phase 2	3/28/23	Phase 2 biomarker data reported that treatment resulted in a significant shift in levels of prion protein (PRPN), noted March 28, 2023.	6
AQST	Lennox-Gastaut Syndrome	Approved	8/31/18	Tentative approval announced August 31, 2018.	6
JNJ	Coronary Artery Disease or Peripheral Artery Disease	Phase 3	2/8/17	Phase 3 trial stopped early due to positive efficacy - February 8, 2017.	6
CNTX	Breast Cancer - 2nd-line	Phase 2	12/8/22	Phase 2 data reported a 4-month PFS rate of 44%, noted December 8, 2022.	5
TSVT	Multiple Myeloma	Phase 3	2/10/23	Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.	6
PTGX	Polycythemia vera	Phase 2	12/12/21	Phase 2 data reported that in all 16 erythrocytotic PV patients, rusfertide demonstrated rapid reduction of hematocrit below 45% within weeks, noted December 12, 2021.	6
RHHBY	Urothelial cancer	Phase 3	1/24/20	Phase 3 trial did not meet primary endpoint - January 24, 2020.	4
SCPH	Heart Failure	Phase 3	7/13/21	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	6

XNCR

COVID-19 antibody

Approved

5/26/21

FDA Emergency Use Authorization filing approval announced May 26, 2021.

6

PFE

Remicade (infliximab) biosimilar

Approved

12/13/17

Approval announced December 13, 2017.

6

GTHX

Metastatic colorectal cancer (mCRC)

Phase 3

2/13/23

Phase 3 topline data reported that achieved its co-primary endpoints related to severe neutropenia but failed to achieve a significant response in overall response rate (ORR) and preliminary measures of survival, noted February 13, 2023.

6

VRDN

Thyroid eye disease (TED)

Phase 1

8/15/22

Phase 1 study demonstrated that treatment achieved a substantially extended half-life of 30-40 days and the feasibility of a convenient, low-volume, subcutaneous injection paradigm of 2mL 300mg, noted August 15, 2022.

6

APLIF

Parasitic and anaerobic bacterial infections

Approved

9/25/23

Approved September 25, 2023.

6

SNY

Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR

Approved

8/10/18

FDA Approval announced August 10, 2018.

6

RNXT

Locally advanced pancreatic cancer (LAPC)

Phase 3

6/29/23

Phase 3 interim data presented at ESMO GI demonstrated an eight-month median PFS benefit, 15 versus 7 months, in delaying the progression of cancer for patients receiving treatment with RenovoGem versus standard-of-care, noted June 29, 2023.

6

IONS

Immunoglobulin A nephropathy (IgAN)

Phase 2

11/7/22

IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months

6

BFRI

Actinic Keratosis

Phase 1

11/21/22

Phase 1 Pharmacokinetics study and results discussed with FDA; to be submitted to FDA along with 3 tubes safety study. Phase 3 study launched, noted November 21, 2022.

5

AZN

Bladder cancer

Phase 3

3/6/20

Phase 3 data did not meet primary endpoint.

4

VNDA

Gastroparesis

Phase 3

2/4/22

Phase 3 data reported that the trial did not meet its primary endpoint, noted February 4, 2022.

6

TERN

Nonalcoholic steatohepatitis (NASH)

Phase 2a

11/12/21

Phase 2a safety and efficacy clinical data reported no differences from placebo in percentage change in low density lipoprotein (LDL) cholesterol or high density lipoprotein (HDL) cholesterol. Reductions in alanine transaminase (ALT) and MRI proton density fat fraction (MRI-PDFF) were reported in the 10 and 15 mg groups, and significant reductions in gamma-glutamyl transferase (GGT) in all dose groups, noted November 12, 2021.

4

ACAD

Parkinson’s disease psychosis (PDP)

Approved

4/29/16

Approved April 29, 2016.

6

GMAB

Cervical Cancer

Approved

9/20/21

Approved September 20, 2021.

6

NKTR

Renal Cell Carcinoma (RCC)

Phase 3

4/14/22

Phase 3 data reported that trial did not met its endpoints of objective response rate (ORR) and overall survival (OS), noted April 14, 2022.

5

GRTS

Solid tumors

Phase 1/2

9/12/22

Phase 2 portion data from Phase 1/2 trial presented at ESMO demonstrated early evidence of efficacy with a 39% molecular response rate in evaluable patients with late-line microsatellite-stable colorectal cancer (MSS-CRC) and non-small cell lung cancer (NSCLC). Data support moving trial into earlier lines of treatment, noted September 12, 2022.

6

CLNN

Parkinson's Disease

Phase 2

8/5/21

Phase 2 trial met primary endpoint - August 5, 2021.

6

SESN

Non-muscle invasive bladder cancer (NMIBC)

CRL

8/13/21

CRL announced August 13, 2021.

5

NVAX

Influenza

Phase 3

3/24/20

Phase 3 top-line data met primary endpoints - March 24, 2020.

6

NVCR

Gastric cancer

Phase 2

6/3/22

Phase 2 data reported a confirmed objective response rate of 50%, with a 10.3 month duration of response and 72% one-year survival rate, noted June 3, 2022.

5

BMRN

Duchenne Muscular Dystrophy (DMD)

CRL

1/14/16

CRL issued January 14 2016.

5

BLUE

TDT and the β0/β0genotype

Phase 3

6/11/20

Phase 3 updated data at EHA June 12, 2020. Six of eight evaluable patients achieved transfusion independence,

6

ZYNE

Adult Epilepsy Patients with Refractory Focal Seizures

Phase 2

8/7/17

Phase 2 data released August 7, 2017 - endpoints not met.

4

CLLS

Multiple myeloma

Phase 1/2

12/13/21

Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.

6

OCS

Diabetic macular edema (DME)

Phase 3

10/6/23

Phase 3 data presented at EURETINA noted that 6 times a day dosing of OCS-01 was a highly effective Loading Dose, noted October 6, 2023.

6

MITO

Mitochondrial disease associated with pathogenic nDNA mutations (nPMD)

Phase 3

12/20/19

Phase 3 data did not meet primary endpoint - December 20, 2019.

4

APTX

Parkinson’s disease cognitive impairment, healthy volunteers

Phase 2

2/27/23

Phase 2 study did not demonstrate sufficient efficacy to support further development, noted February 27, 2023.

6

CDAK

Solid tumors

Phase 1/2

6/30/22

Phase 1/2 data reported that two patients experienced tumor regressions in both injected and non-injected lesions, including a partial response in one patient, noted June 30, 2022.

5

LBPS

Solid tumors

Phase 1/2

3/23/22

Phase 1/2 part B data reported that the renal cell carcinoma (RCC) group met the primary endpoint, noted March 23, 2022.

5

IMTX

Solid tumors

Phase 1b

10/24/23

FDA Granted RMAT on October 24, 2023.

5

VTRS

Multiple sclerosis (MS) - Generic for Copaxone

Approved

10/4/17

Approval announced October 4, 2017.

6

BNTX

COVID-19 Vaccine (Booster)

Phase 1/2

5/23/22

Phase 1/2/3 booster trial in children reported that a 80.3% vaccine efficacy, noted May 23, 2022.

5

ALDX

Psoriasis / atopic asthma / COVID-19

Phase 2

3/29/22

Phase 2 top-line data reported statistically significant improvements in all three indications with no serious adverse events, noted March 29, 2022.

6

PHAR

Activated phosphoinositide 3-kinase delta syndrome (APDS)

Approved

3/24/23

Approved March 24, 2023.

6

BOLT

HER2-Expressing Solid Tumors, gastric cancer, including gastroesophageal junction cancer

Phase 2

10/23/23

Phase 2 updated safety data presented at ESMO supports the selection of 20 mg/kg q2w as the recommended Phase 2 dose (RP2D), noted October 23, 2023.

6

BMY

Spinal Muscular Atrophy (SMA)

Phase 3

9/14/23

Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.

5

ATHX

Acute Respiratory Distress Syndrome

Phase 2

8/6/21

Phase 2 open-label data released August 6, 2021. 20/28 days were ventilator free compared with 11/28 for standard therapy.

6

SNY

Chronic Spontaneous Urticaria (CSU)

Phase 3

2/18/22

Phase 3 trial did not meet the primary endpoint, noted February 18, 2022.

6

CVAC

COVID-19 vaccine

Phase 2/3

10/12/21

CVnCoV withdrawn from regulatory review due to focus on second generation candidate, noted October 12, 2021.

4

BGNE

Cervical Cancer (CC)

Phase 2

10/22/23

Phase 2 data presented at ESMO reported that the ORR was 22.5%, with 13 complete responses; 76.8% had a durable response of ≥6 mo, noted October 22, 2023.

6

INCY

COVID-19 associated cytokine storm

Phase 3

12/14/20

Phase 3 top-line did not meet primary endpoint - December 14, 2020.

4

AVXL

Mild to moderate Alzheimer’s disease

Phase 2/3

9/14/23

Phase 2b/3 full data reported that the least-squares mean (LSM) change from baseline to 48 weeks between the blarcamesine and placebo groups were −1.783; for ADAS-Cog13, and −0.456 for CDR-SB in patients with early Alzheimer's disease, noted September 14, 2023.

5

AGIO

Advanced hematologic malignancies with an IDH2 mutation

Approved

8/1/17

Approval announced August 1, 2017.

6

NOVN

Human papillomavirus (HPV)

Phase 2

11/29/16

Phase 2 data released November 29, 2016. Primary endpoint met.

6

GILD

Non-Small Cell Lung Cancer (NSCLC)

Phase 2

9/11/23

Phase 2 trial data reported that in both cohorts, the ORR was 56%, and DCR was 82%. Median duration of response (DoR) was not reached at the time of data cut-off, and DoR rate at six months was 88% in both cohorts, noted September 11, 2023.

6

PFE

COVID-19 vaccine

Phase 2/3

11/4/22

Phase 2/3 clinical trial demonstrated robust neutralizing immune response one month after a 30-µg booster dose of the COVID-19 vaccine (Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5)). Omicron BA.4/BA.5-neutralizing antibody titers increased 13.2-fold from pre-booster levels in adults older than 55 years of age and 9.5-fold in adults 18 to 55 years of age, noted November 4, 2022.

6

IMGN

Cancer - ovarian cancer and relapsed endometrial cancer

Phase 3

9/28/23

Additional Phase 3 data reported at ESGO noted that in patients with prior PARPi, ORR in the ELAHERE arm was 45%, including 7 CRs, compared to 17%, with no CRs, in the IC chemotherapy arm, noted September 28, 2023.

5

CGEM

Solid tumors

Phase 1

11/3/23

Phase 1 Initial clinical biomarker data demonstrated that CLN-619 increases MICA expression on the tumor cell surface, consistent with previously reported preclinical data and supporting the proposed mechanism of action, noted November 3, 2023.

6

NUVL

ALK non-small cell lung cancer (NSCLC)

Phase 1

10/13/23

Phase 1 trial showed preliminary signs of activity observed in heavily pre-treated patients with ALK-positive NSCLC, including in subgroups of patients who have previously received a 2nd generation ALK TKI and lorlatinib, have brain metastases, or have single or compound ALK resistance mutations, noted October 13, 2023.

6

EQRX

Stage III Non-small cell lung cancer (NSCLC)

Phase 3

8/7/22

Phase 3 data reported that treatment demonstrated a statistically significant improvement in progression-free survival versus placebo, noted August 7, 2022.

6

TARS

Lyme disease

Phase 1

12/15/22

Phase 1 data data showed that treatment was well tolerated and safety data supports progression to Phase 2a (Carpo), noted December 15, 2022.

6

BMY

First-line Renal cell carcinoma (RCC)

Phase 3

9/16/21

Additional Phase 3 survival data released at ESMO September 16, 2021. Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy. Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up.

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BMY

Chronic lymphocytic leukemia (CLL)

Phase 1/2

6/6/23

Phase 1/2 data presented at ASCO reported that the primary endpoint of CR/CRi rate was met at 18.4%, and the ORR was 42.9% and was not statistically significant, noted June 6, 2023.

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PLX

Fabry disease

Approved

5/10/23

Approved May 10, 2023.

6

CALT

IgA nephropathy

Approved

12/15/21

Approved December 15, 2021.

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BMY

Stage IIA to IIIB non-small cell lung cancer (NSCLC)

Phase 3

10/21/23

Phase 3 data presented at ESMO reported that NIVO + chemo/NIVO improved pCR rates (25.3% vs 4.7%; odds ratio, 6.64 [95% CI, 3.40–12.97]) and MPR rates (35.4% vs 12.1%; odds ratio: 4.01 [2.48–6.49]) vs chemo/PBO, noted October 21, 2023.

6

VRDN

Thyroid Eye Disease (TED)

Phase 1/2

4/27/23

Phase 1/2 clinical data reported that 80% (4/5) of VRDN-001 responders showed durability of effects at 12 weeks, noted April 27, 2023.

6

FULC

Facioscapulohumeral muscular dystrophy (FSHD)

Phase 3

9/8/23

Phase 3 enrollment completed, noted September 8, 2023

5

SLS

Hematologic malignancies, acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and lymphoma

Phase 2a

10/30/23

FDA granted fast track designation, noted October 30, 2023.

5

IONS

Chronic hepatitis B virus (CHB)

Phase 2b

6/27/22

Phase 2b data readout reported that up to 68% of patients on NA therapy and up to 65% of patients not on NA achieved HBsAg <100 IU/mL at the end of treatment, noted June 27, 2022.

6

AVIR

COVID-19

Phase 2

10/19/21

Phase 2 trial did not meet primary endpoint, noted October 19, 2021.

4

CLSD

Wet AMD using suprachoroidal delivery

Phase 2

7/30/23

Phase 2 additional data reported meaningful reductions in Anti-VEGF injection burden with stable to improved CRT through month 6, noted July 30, 2023.

6

AZN

Chronic obstructive pulmonary disease (COPD)

Approved

7/24/20

FDA approval announced July 24, 2020.

6

BIIB

Lupus

Phase 2b

10/23/18

Phase 2b data released October 23, 2018 - primary endpoint not met.

5

TAK

Higher-risk myelodysplastic syndromes (HR-MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML)

Phase 3

9/1/21

Phase 3 data did not meet primary endpoint September 1, 2021.

4

IMAB

COVID-19

Phase 2

8/11/21

Phase 2 interim analysis released August 11, 2021. Mechanical ventilation free (MVF) rate (83.6% vs 76.7%) by day 30, lower mortality rate (4.9% vs 13.3%) compared to placebo.

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CLVS

Ovarian Cancer - First-line maintenance treatment

PDUFA

6/26/23

Company went bankrupt in 2022 not information disclosed about the PDUFA.

5

AZN

Wilson disease

Phase 3

6/23/22

Phase 3 data reported at the International Liver Congress noted greater improvements in neurological scores for those treated with ALXN1840 compared to SoC, however, there were no significant differences between treatment groups observed at 48 weeks, noted June 23, 2022.

6

DRRX

COVID-19 Patients with Acute Liver or Kidney Injury

Phase 2

3/4/21

Phase 2 trial to be discontinued due to limited number of patients eligible or willing to enroll.

4

ADAP

Ovarian Cancer

Phase 1

11/8/22

Phase 1 data reported an increased ORR now 43% in ovarian cancer with one new response, noted November 8, 2022.

5

ABBV

Presbyopia

Approved

10/29/21

Approved October 29, 2021.

6

NVS

Complicated urinary tract infections (cUTI)

Approved

8/29/17

Approved August 29, 2017.

6

SNY

Coronavirus COVID-19

Phase 3

9/1/20

Phase 3 global trial did not meet primary endpoint - September 1, 2020.

4

BCRX

Paroxysmal nocturnal hemoglobinuria (PNH), Healthy Volunteers

Phase 1

10/26/23

Phase 1 PoC trial with PNHl initiated, noted October 26, 2023.

5

MRK

First-line ovarian cancer

Approved

5/8/20

FDA Approval announced May 8, 2020.

6

BMY

Tumors harboring ALK, ROS1, or NTRK1-3 alterations

Phase 1/2

10/23/21

Phase 1/2 early clinical data demonstrated confirmed responses in (3/4) TKI-naïve patients, noted October 23, 2021.

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CCCC

Non-Small Cell Lung Cancer (NSCLC)

Phase 1a

7/5/23

IND Cleared by FDA, July 5, 2023.

5

ACRS

Common warts (verruca vulgaris)

Phase 2

10/24/19

Phase 3 data met all endpoints - second Phase 3 trial.

6

NAMS

Alzheimer's Disease

Phase 2a

9/21/23

Phase 2a data observed reductions of 11% and 12% in 24- and 27-hydroxycholesterol in cerebrospinal fluid (CSF), respectively, indicating potential improvement of cholesterol metabolism in the brain. Observed 8% increase in Aβ42/40 ratio, a key biomarker of AD risk, suggesting improvement in disease pathology, noted September 21, 2023.

6

ALBO

Alagille syndrome (ALGS)

Approved

6/13/23

FDA Approved on June 13, 2023.

6

CDXS

Exocrine pancreatic insufficiency (EPI)

Phase 1

2/23/23

Phase 1 interim data reported that no safety issues occurred, with no SAEs or discontinuations, noted February 23, 2023.

4

PLX

Fabry disease

Phase 3

2/10/21

Phase 3 final data presented February 10, 2021 - key objectives for safety and efficacy.

5

MRK

First-line ovarian cancer following platinum-based chemotherapy

Approved

12/19/18

FDA Approval announced December 19, 2018.

6

LLY

Hepatocellular Carcinoma

Approved

5/13/19

FDA approval announced May 13, 2019.

6

ONCY

Breast cancer

Phase 1

12/8/22

Additional dat presented at the San Antonio Breast Cancer Symposium (SABCS) reported that disease control, partial response (PR) or stable disease (SD), was achieved in thirteen of fourteen evaluable patients (93%), with twelve (86%) showing tumor shrinkage from baseline, with 50% achievign a partial response, noted December 8, 2022.

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GSK

HIV

Approved

7/2/20

FDA Approval announced July 2, 2020.

6

JAZZ

COVID-19

Phase 2

12/13/21

Phase 2 data reported that treatment in patients with grade 5 WHO COVID 19 ARDS does not induce bleeding, and is associated with rapid restoration of respiratory function (73% of patients). No oxygen support was needed at discharge and a 1-month OS rate of 89% was observed, which is higher than historical controls (77%) treated in the same setting, noted December 13, 2021.

6

BMY

Non-Small Cell Lung Cancer (NSCLC)

Phase 3

9/11/23

Phase 3 data demonstrated consistent, durable benefits of Opdivo plus Yervoy vs. chemotherapy at six years, regardless of PD-L1 expression, noted September 11, 2023.

6

ASLN

Gastric cancer

Phase 2

1/13/19

Phase 2 trial did not meet primary endpoint - January 13, 2019.

4

RHHBY

Babies with pre-symptomatic spinal muscular atrophy (SMA)

Phase 3

10/4/23

Phase 3 OLE met its primary endpoint with 80% of babies sitting without support for at least 5 seconds after 1 year of Evrysdi, noted October 4, 2023.

5

EIGR

Hepatitis delta virus (HDV)

Phase 2

11/12/19

Phase 2 data presented at AASLD November 12, 2019 - Median HDV RNA decline was 3.4 log IU/mL (IQR: 2.9-4.5, p<0.0001).

6

GSK

dMMR endometrial cancer

Approved

2/10/23

Full approval granted February 10, 2023.

6

CGTX

Mild-to-Moderate Alzheimer's Disease

Phase 2

3/28/23

Phase 2 biomarker data reported that treatment resulted in a significant shift in levels of prion protein (PRPN), noted March 28, 2023.

6

AQST

Lennox-Gastaut Syndrome

Approved

8/31/18

Tentative approval announced August 31, 2018.

6

JNJ

Coronary Artery Disease or Peripheral Artery Disease

Phase 3

2/8/17

Phase 3 trial stopped early due to positive efficacy - February 8, 2017.

6

CNTX

Breast Cancer - 2nd-line

Phase 2

12/8/22

Phase 2 data reported a 4-month PFS rate of 44%, noted December 8, 2022.

5

TSVT

Multiple Myeloma

Phase 3

2/10/23

Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.

6

PTGX

Polycythemia vera

Phase 2

12/12/21

Phase 2 data reported that in all 16 erythrocytotic PV patients, rusfertide demonstrated rapid reduction of hematocrit below 45% within weeks, noted December 12, 2021.

6

RHHBY

Urothelial cancer

Phase 3

1/24/20

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

4

SCPH

Heart Failure

Phase 3

7/13/21

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

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