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
LIFE	Pulmonary sarcoidosis	Phase 1/2	5/22/23	Phase 1b/2a data presented at the American Thoracic Society reported that treatment led to an exposure-dependent decrease in the extent of oral corticosteroid usage, increase in lung function as measured by percent predicted forced vital capacity and improvement in clinical symptoms as measured by the King's Sarcoidosis Questionnaire-Lung score, noted May 22, 2023.	6
RAIN	Solid tumors with loss of CDKN2A Gene	Phase 1	5/30/23	Phase 1 basket trial to be terminated, noted May 5, 2023.	4
EYPT	Diabetic macular edema	Approved	9/26/14	Approved Sept 26 2014.	6
BFRI	Actinic keratosis (AK)	Approved	10/10/23	Approved October 10, 2023.	6
LLY	Psoriasis	Phase 3	7/17/20	Phase 3 trial met primary and all key secondary endpoints vs placebo at Week 16 and all key secondary endpoints versus Cosentyx (secukinumab) at Week 16 and Week 52 - July 17, 2020.	6
JNJ	Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).	Approved	4/21/20	FDA Approval announced April 21, 2020.	6
ALNY	Acute hepatic porphyrias	Approved	11/20/19	FDA approval announced November 20, 2019.	6
OTIC	Ménière's disease	Phase 3	2/22/21	Phase 3 trial did not achieve the primary endpoint.	4
ERYP	Acute lymphoblastic leukemia (ALL)	Phase 2	12/6/20	Phase 2 data presented at ASH December 6, 2020. Endpoints met.	6
SPPI	Colorectal cancer	Approved	4/29/11	Approved April 29, 2011.	6
BGNE	B-cell malignancies intolerant to ibrutinib and/or acalabrutinib	Phase 2	6/15/23	Phase 2 preliminary study demonstrated that treatment with BRUKINSA was well-tolerated and unlikely to lead to recurrence of intolerant adverse events experienced with prior BTK-inhibitor therapy, suggesting BRUKINSA may be a treatment option for these patients, noted June 15, 2023.	6
UTHR	Pulmonary arterial hypertension (PAH)	Phase 3	10/21/21	78 week data noted an improved health-related quality of life at month six, noted October 21, 2021.	6
PFE	Alopecia areata	Approved	6/23/23	FDA Approved on June 23, 2023.	6
AZN	Hepatocellular carcinoma (HCC)	Approved	10/24/22	Approved October 24, 2022.	6
KYMR	Diffuse large B-cell lymphoma (DLBCL)	Phase 1	6/14/23	Phase 1 patient dose escalation data cohorts show target knockdown at or near levels associated with clinical efficacy in preclinical tumor models, with no dose limiting toxicities (DLTs) observed, noted June 14, 2023.	6
ADXS	Desmoid tumors	Phase 2/3	10/23/23	Phase 2 data presented at ESMO showed that 1.2 mg once daily treatment achieved Overall Response Rate of 83% in the evaluable population, AL102 1.2 mg once daily treatment resulted in 88% reduction in tumor volume and 85% reduction in T2W signal intensity, noted October 23, 2023.	6
XNCR	Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, DLBCL	Phase 1	12/12/22	Phase 1 data reported an overall response rate (ORR) was 52.0% (13/25), and the complete response rate was 24.0% (6/25), noted December 12, 2022.	5
BGNE	Refractory chronic lymphocytic leukemia (CLL)	Phase 3	6/15/23	Phase 3 results presnted at ICML In a subgroup analysis showed improved PFS over ibrutinib in patients in China, consistent with the global study population, noted June 15, 2023.	6
MRK	Classical Hodgkin lymphoma	Approved	10/15/20	FDA Approval announced October 15, 2020.	6
VNDA	Atopic dermatitis	Phase 3	2/25/20	Phase 3 trial did not meet primary endpoint - February 25, 2020.	4
VNDA	Jet Lag Disorder	CRL	8/19/19	CRL issued August 19, 2019.	5
IMVIQ	Platinum Resistant Ovarian Cancer	Phase 2	12/9/21	Phase 2 data reported increases in both T and B cell immune infiltration, and more prominent antigen-specific humoral responses, noted December 9, 2021.	6
ABBV	Post-Operative Atrial Fibrillation (POAF) in patients undergoing open-chest cardiac surgery	Phase 2	11/7/22	Phase 2 data reported that primary endpoint was not met, noted November 7, 2022.	5
SNY	Hemophilia A	Approved	2/23/23	Approved February 23, 2023.	6
ARWR	Dyslipidemia	Phase 2b	11/7/22	Phase 2b data presented at AHA reported that ARO-ANG3 is associated with relative reduction in liver fat fraction at Week 24, with no AEs related to LFT changes reported to date, noted November 7, 2022.	6
BLUE	β-Thalassemia	Approved	8/17/22	Approved August 17, 2022.	6
AMGN	RITUXAN biosimilar - non-Hodgkin lymphoma	Approved	12/17/20	FDA approval announced December 17, 2020.	6
KOD	Diabetic macular edema (DME)	Phase 3	7/24/23	Phase 3 trial did not meet the primary endpoint, noted July 24, 2023.	6
AXSM	Smoking cessation	Phase 2	4/15/19	Phase 2 top-line data met primary endpoint - April 15, 2019.	6
ENTA	HCV - genotype 1	Approved	12/19/14	Approved December 19, 2014.	6
GILD	Hepatitis C	Approved	3/19/20	sNDA FDA Approval announced March 19, 2020.	6
KOD	Wet age-related macular degeneration (AMD)	Phase 2/3	5/3/22	Phase 2b/3 primary results reported durability with nearly 60% on Q20W dosing, however trial did not meet primary endpoint, noted May 3, 2022.	6
DNLI	Amyotrophic lateral sclerosis (ALS)	Phase 1b	4/25/23	Phase 1b data due presented at AAN reported low variability, predictable dose-related increases in exposure, and a half-life supporting once-daily dosing, noted April 25, 2023.	6
AIM	Cisplatin resistant advanced recurrent ovarian cancer	Phase 1/2	1/24/22	Phase 1/2 IP chemo-immunotherapy combination demonstrated to be well tolerated, and associated with interferon stimulated gene changes that favor cytotoxic T lymphocytes chemo-attraction and function, noted January 24, 2022.	6
TEVA	Tourette Syndrome	Phase 2/3	2/19/20	Phase 2/3 trial did not meet primary endpoint - February 19, 2020.	4
RYTM	POMC deficiency obesity / Leptin Receptor Deficiency Obesity	Approved	11/27/20	FDA approval announced November 27, 2020.	6
BMY	First-line Glioblastoma (GBM) cancer	Phase 3	12/23/20	Phase 3 trial unlikely to meet OS primary endpoint	4
BYSI	Non small cell lung cancer	Phase 3	12/13/22	Additional data reported that plinabulin demonstrated a superior benefit for Gr4N, Gr3/4N, all GrN and DSN compared to the control, noted December 13, 2022.	6
RZLT	Congenital hyperinsulinism (HI)	Phase 2b	3/23/22	Phase 2b study demonstrated highly significant improvements in hypoglycemia and good safety and tolerability, noted March 23, 2022.	6
MOLN	Age-related macular degeneration (AMD)	CRL	6/26/20	CRL issued June 26, 2020.	5
TAK	Pouchitis	Phase 3	3/30/23	Phase 4 met primary endpoint, noted March 30, 2023.	5
AZN	Thyroid cancer	Phase 3	7/26/18	Phase 3 trial did not meet primary endpoint - noted July 26, 2018.	4
ZYME	HER2-expressing Gastrointestinal Cancers (Gastroesophageal Adenocarcinoma, Biliary Tract Cancer, and Colorectal Cancer)	Phase 2	1/19/23	Phase 2 data presented at ASCO Gastrointestinal Cancers Symposium reported an overall confirmed objective response rate (cORR) of 79%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 12.5 months, noted January 19, 2023.	6
ADAP	Esophageal and esophagogastric junction cancers	Phase 2	11/8/22	Phase 2 data reported that ORR has increased to 37% from 33%, and the median duration of response has increased to ~20 weeks from approximately 12 weeks, noted November 8, 2022.	5
AIM	CFS	CRL	2/4/13	CRL February 4, 2013.	5
SYBX	Cirrhotic patients with elevated ammonia	Phase 1/2	8/20/19	Development to be discontinued due to lack of efficacy.	4
NAVB	Head and Neck Cancer	Approved	6/13/14	Approved June 13, 2014.	6
LGND	ER+/HER2- Breast Cancer	Phase 2	6/5/23	Phase 2 data presented at ASCO reported that PFS was 56.0 wks (~13 mos), CBR was 65.5%, and ORR was 55.6%, noted June 5, 2023.	6
MLTX	Hidradenitis suppurativa	Phase 2	10/16/23	Phase 2 24-week topline data reported that 57% of patients continuously treated with 120mg achieved a HiSCR75 response and 38% achieved HiSCR90, noted October 16, 2023.	6
ABBV	B-cell Lymphoma (DLBCL)	Approved	5/22/23	Approved May 19, 2023.	6
RHHBY	Alzheimer’s disease	Phase 3	1/30/19	Phase 3 trial terminated following interim analysis indicating primary endpoint would not be met.	4
BMY	Relapsed and/or Refractory (R/R) Multiple Myeloma (MM)	Phase 1	12/10/22	Phase 1 data is presented at ASH demonstrated deep and durable responses with a manageable safety profile across all dose levels, including patients previously treated with a B-cell maturation antigen (BCMA)-directed CAR T cell therapy, noted December 10, 2022.	6
EVFM	contraceptive vaginal gel	Approved	5/22/20	FDA Approval announced May 22, 2020.	6
VTRS	Dry eye disease	Approved	2/3/22	Approved February 3, 2022.	6
AMGN	Solid tumors with MTAP deletion, NSCLC	Phase 1	3/15/22	Phase 1 updated data reported no drug-related SAE's or observed dose limiting toxicity (DLT) through Cohort 5, noted March 15, 2022.	6
PFE	Chronic low back pain	Phase 3	2/19/19	Phase 3 data released February 19, 2019. 10mg dose met primary endpoint. 5mg dose did not meet endpoint.	5
GSK	RSV vaccine	Approved	5/3/23	FDA Approved on May 3, 2023.	6
MOLN	COVID-19	Phase 2a	12/6/21	Phase 2a data reported an infectivity of virus extracted through nasopharyngeal swabs was reduced to zero within 3-5 days post treatment in patients with positive titers at baseline, noted December 6, 2021.	5
APTO	Acute myeloid leukemia (AML)	Phase 1/2	10/30/23	Phase 1/2 data demonstrated 44% ORR with TUS/VEN in Difficult-to-Treat VEN Failure R/R AML Patients, noted October 30, 2023.	6
APLT	Diabetic cardiomyopathy (DbCM)	Phase 3	6/22/23	Phase 3 baseline data to be presented at ADA showed a strong statistical correlation between elevations in the cardiac stress biomarker, NT-proBNP, reduced cardiac functional capacity and physical activity, underscoring the importance of early diagnosis via NT-proBNP and impact of DbCM on physical function and quality of life, noted June 22, 2023	6
MRK	Cervical cancer	Phase 3	7/19/23	Phase 3 data reported that trial met the PFS primary endpoint, noted July 19, 2023.	5
AZN	Relapsed or Refractory Mantle Cell Lymphoma	Approved	10/31/17	Priority Review announced August 2, 2017. Approval announced October 31, 2017.	6
PTCT	Mitochondrial Epilepsy	Phase 2/3	6/29/23	Phase 2/3 data reported that trial did not meet the primary endpoint, noted June 29, 2023.	6
AXSM	Narcolepsy	Phase 2	12/3/19	Phase 2 data met primary endpoint.	6
XCUR	Merkel cell carcinoma / Cutaneous squamous cell carcinoma	Phase 1/2	8/5/21	Phase 1b/2 interim data noted overall response rate (ORR) of 21% in all evaluable MCC patients - August 5, 2021.	5
ZVRA	Urea cycle disorder (UCD)	Approved	12/23/22	Approved December 23, 2022.	6
AEZS	Endometrial cancer	Phase 3	5/1/17	Phase 3 trial did not meet primary endpoint - May 1, 2017.	4
HCM	Myeloid hematological malignancies (HMs)	Phase 1	6/9/23	Phase 1 data reported that the best of response was 1CR in 100 mg cohort; 9 CRs and 1 PR in 150 mg QD; 1 CR in 200 mg QD; and 3 CRs in 250 mg QD; with an ORR of 33.3% (1/3), 40.0%(10/25), 14.3% (1/7) and 42.9% (3/7), respectively, noted June 9, 2023.	5
AADI	PEComa	Approved	11/23/21	Approved November 23, 2021.	6
ARAV	Pancreatic Adenocarcinoma	Phase 1b	5/25/23	Phase 1b data from ASCO reported that the median overall survival (OS) for patients with trough levels above the minimal batiraxcept efficacious concentration is greater than 15 months, and one patient demonstrated a complete response from 10 months to 20 months and is still on study, noted May 25, 2023.	6
QSAM	Bone cancer	Phase 1	8/16/23	Initial Phase 1 data reported that treatment was well-tolerated with no serious adverse events, a 56% - 64% decrease in SUV in patient one, and that the first two patients experienced significant pain relief and improved mobility within a week after treatment, noted August 16, 2023.	6
IRWD	Irritable Bowel Syndrome with Constipation (IBS-C)	Phase 3	6/18/19	Phase 3b data met all endpoints - June 18, 2019.	6
PFE	Squamous cell carcinoma of the head and neck (SCCHN)	Phase 2	4/17/23	Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.	6
AZN	Endometrial cancer (EC)	Phase 3	10/23/23	Phase 3 results presented at ESMO reported statistically significant and clinically meaningful PFS benefit vs CP in the ITT population, with the interim OS data being immature (27.7%) yet with a trend towards benefit, noted October 21, 2023.	6
MRK	Non-transfusion-dependent beta-thalassemia	Phase 2	6/11/21	Phase 2 data presented at EHA meeting June 11, 2021. 77.7% of patients achieved a hemoglobin increase compared to 0% in the placebo arm.	6
AMGN	Osteoporosis	Approved	4/9/19	FDA Approval announced April 9, 2019.	6
RHHBY	Duchenne muscular dystrophy	Phase 3	10/30/23	Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.	6
CNTA	Kidney Disease	Phase 3	12/14/21	Phase 3 data reported that ALT elevation peaked between 1.8x and 3.5x ULN and did not return to below ULN until 23 to 140 days after tolvaptan use was discontinued. Four patients were successfully titrated to a maintenance dose of lixivaptan of either 100 mg BID or 200 mg BID, noted December 14, 2021.	6
EXEL	Solid Tumors	Phase 1/2	1/18/22	Phase 1/2 cohort results showed that of the 36 patients enrolled in cohort 2 of the CAMILLA trial, 29 were evaluable for the efficacy analysis. The primary outcome of investigator-assessed ORR per modified RECIST version 1.1 was 27.6%. The confirmed PR rate was 20.7%, and the DCR was 86.2%. Median PFS was 3.8 months, with a 6-month PFS of 34.5%. Median OS was 9.1 months, noted January 18, 2022.	6
EFTR	Solid Tumors RTK BC and KRAS NSCLC	Phase 1/2	6/6/22	Phase 1/2 presentation at ASCO reported 2 partial responses, noted June 6, 2022.	6
CMRX	Adenovirus Infection	Phase 3	2/22/17	Phase 3 interim analysis released May 2016. Final data released February 22, 2017.	5
BFRI	Actinic Keratosis on face & scalp	Phase 1	8/28/23	Phase 1 data reported that treatment was generally well tolerated and all TEAEs were transient, noted August 28, 2023.	6
DRUG	Dravet Syndrome	Phase 1/2	8/8/23	Additional Phase 1/2 cohort 4 data reported an increase in central delta power and robust reduction in central alpha and beta power in active group, as previously reported for Anti-Epileptic Drugs (AEDs) in healthy individuals, noted August 8, 2023.	6
ELDN	Kidney transplant rejection	Phase 2	11/2/23	Phase 2 data from 11 participants demonstrates tegoprubart successfully prevented kidney transplant rejection and was generally safe and well-tolerated, noted November 2, 2023.	6
AZN	Asthma	Phase 3	5/16/22	Phase 3 results reported that treatment reduced the risk of a severe exacerbation by 27%, noted May 16, 2022.	6
ZNTL	Ovarian cancer	Phase 1b	6/5/23	Phase 1b dose escalation data presented at ASCO reported that confirmed ORR of 50.0% and mPFS of 7.4 months in combination with paclitaxel, and a confirmed ORR of 35.7% and mPFS of 10.4 months in combination with carboplatin, noted June 5, 2023.	6
RVNC	Lateral Canthal (Crow’s Feet) Lines	Phase 2	6/30/20	Phase 2 top-line data released June 30, 2020. 88% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group.	6
OPK	Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) - Stage 5	Phase 2	11/2/23	Phase 2 data presented at ASH indicated that early, sustained and effective treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression CKD in pre-dialysis patients, noted November 2, 2023.	6
BMY	(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC)	Approved	8/1/17	Approval announced August 1, 2017.	6
ATNXQ	Angiosarcoma	Phase 2	12/9/20	Phase 2 poster at SABCS December 9, 2020 - Complete response (CR) 43% (3/7 pts), partial response (PR) 14% (1/7 pts).	5
ACAD	Adjunctive Treatment in Patients With Major Depressive Disorder	Phase 3	7/20/20	Phase 3 trial did not meet primary endpoint - July 20, 2020.	4
BIIB	Alzheimer’s disease	Phase 3	9/13/19	Phase 3 trial discontinued due to unfavorable risk-benefit ratio.	4
GPCR	Type 2 Diabetes	Phase 1b	9/29/23	Phase 1b results reported that GSBR-1290 demonstrated significant weight loss supporting once-daily (QD) dosing, noted September 29, 2023.	6
MRK	Triple negative breast cancer (TNBC)	Phase 3	5/20/19	Phase 3 data May 20, 2019 did not meet primary endpoint.	4
AMGN	Wild-Type RAS Metastatic Colorectal Cancer	Approved	6/29/17	sBLA approval announced June 29, 2017.	6
JNJ	metastatic hormone-naïve prostate cancer (mHNPC)	Approved	2/8/18	sNDA approval announced February 8, 2018.	6
PDSB	Human papillomavirus (HPV) associated cancers	Phase 2	12/28/22	Phase 2 data reported a median OS of 21 months, with 75% remain alive at a median follow-up of 27 months, noted December 28, 2022.	5
ARQT	Plaque psoriasis in children	Approved	10/6/23	Approved for treatment of children aged 6 - 11 on October 6, 2023.	6
INO	HPV 6 and HPV 11-associated Recurrent respiratory papillomatosis	Phase 1/2	10/10/23	Phase 1/2 received feedback from the FDA that data could support INOVIO's submission of a BLA for review under the FDA's accelerated approval program. The FDA also advised that the company's previously planned Phase 3 trial would not be required to support this submission, noted October 10, 2023.	6
ALNY	Primary Hyperoxaluria Type 1	Phase 3	9/30/20	Clinically meaningful reduction in spot urinary oxalate:creatinine ratio.	6

LIFE

Pulmonary sarcoidosis

Phase 1/2

5/22/23

Phase 1b/2a data presented at the American Thoracic Society reported that treatment led to an exposure-dependent decrease in the extent of oral corticosteroid usage, increase in lung function as measured by percent predicted forced vital capacity and improvement in clinical symptoms as measured by the King's Sarcoidosis Questionnaire-Lung score, noted May 22, 2023.

6

RAIN

Solid tumors with loss of CDKN2A Gene

Phase 1

5/30/23

Phase 1 basket trial to be terminated, noted May 5, 2023.

4

EYPT

Diabetic macular edema

Approved

9/26/14

Approved Sept 26 2014.

6

BFRI

Actinic keratosis (AK)

Approved

10/10/23

Approved October 10, 2023.

6

LLY

Psoriasis

Phase 3

7/17/20

Phase 3 trial met primary and all key secondary endpoints vs placebo at Week 16 and all key secondary endpoints versus Cosentyx (secukinumab) at Week 16 and Week 52 - July 17, 2020.

6

JNJ

Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

Approved

4/21/20

FDA Approval announced April 21, 2020.

6

ALNY

Acute hepatic porphyrias

Approved

11/20/19

FDA approval announced November 20, 2019.

6

OTIC

Ménière's disease

Phase 3

2/22/21

Phase 3 trial did not achieve the primary endpoint.

4

ERYP

Acute lymphoblastic leukemia (ALL)

Phase 2

12/6/20

Phase 2 data presented at ASH December 6, 2020. Endpoints met.

6

SPPI

Colorectal cancer

Approved

4/29/11

Approved April 29, 2011.

6

BGNE

B-cell malignancies intolerant to ibrutinib and/or acalabrutinib

Phase 2

6/15/23

Phase 2 preliminary study demonstrated that treatment with BRUKINSA was well-tolerated and unlikely to lead to recurrence of intolerant adverse events experienced with prior BTK-inhibitor therapy, suggesting BRUKINSA may be a treatment option for these patients, noted June 15, 2023.

6

UTHR

Pulmonary arterial hypertension (PAH)

Phase 3

10/21/21

78 week data noted an improved health-related quality of life at month six, noted October 21, 2021.

6

PFE

Alopecia areata

Approved

6/23/23

FDA Approved on June 23, 2023.

6

AZN

Hepatocellular carcinoma (HCC)

Approved

10/24/22

Approved October 24, 2022.

6

KYMR

Diffuse large B-cell lymphoma (DLBCL)

Phase 1

6/14/23

Phase 1 patient dose escalation data cohorts show target knockdown at or near levels associated with clinical efficacy in preclinical tumor models, with no dose limiting toxicities (DLTs) observed, noted June 14, 2023.

6

ADXS

Desmoid tumors

Phase 2/3

10/23/23

Phase 2 data presented at ESMO showed that 1.2 mg once daily treatment achieved Overall Response Rate of 83% in the evaluable population, AL102 1.2 mg once daily treatment resulted in 88% reduction in tumor volume and 85% reduction in T2W signal intensity, noted October 23, 2023.

6

XNCR

Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, DLBCL

Phase 1

12/12/22

Phase 1 data reported an overall response rate (ORR) was 52.0% (13/25), and the complete response rate was 24.0% (6/25), noted December 12, 2022.

5

BGNE

Refractory chronic lymphocytic leukemia (CLL)

Phase 3

6/15/23

Phase 3 results presnted at ICML In a subgroup analysis showed improved PFS over ibrutinib in patients in China, consistent with the global study population, noted June 15, 2023.

6

MRK

Classical Hodgkin lymphoma

Approved

10/15/20

FDA Approval announced October 15, 2020.

6

VNDA

Atopic dermatitis

Phase 3

2/25/20

Phase 3 trial did not meet primary endpoint - February 25, 2020.

4

VNDA

Jet Lag Disorder

CRL

8/19/19

CRL issued August 19, 2019.

5

IMVIQ

Platinum Resistant Ovarian Cancer

Phase 2

12/9/21

Phase 2 data reported increases in both T and B cell immune infiltration, and more prominent antigen-specific humoral responses, noted December 9, 2021.

6

ABBV

Post-Operative Atrial Fibrillation (POAF) in patients undergoing open-chest cardiac surgery

Phase 2

11/7/22

Phase 2 data reported that primary endpoint was not met, noted November 7, 2022.

5

SNY

Hemophilia A

Approved

2/23/23

Approved February 23, 2023.

6

ARWR

Dyslipidemia

Phase 2b

11/7/22

Phase 2b data presented at AHA reported that ARO-ANG3 is associated with relative reduction in liver fat fraction at Week 24, with no AEs related to LFT changes reported to date, noted November 7, 2022.

6

BLUE

β-Thalassemia

Approved

8/17/22

Approved August 17, 2022.

6

AMGN

RITUXAN biosimilar - non-Hodgkin lymphoma

Approved

12/17/20

FDA approval announced December 17, 2020.

6

KOD

Diabetic macular edema (DME)

Phase 3

7/24/23

Phase 3 trial did not meet the primary endpoint, noted July 24, 2023.

6

AXSM

Smoking cessation

Phase 2

4/15/19

Phase 2 top-line data met primary endpoint - April 15, 2019.

6

ENTA

HCV - genotype 1

Approved

12/19/14

Approved December 19, 2014.

6

GILD

Hepatitis C

Approved

3/19/20

sNDA FDA Approval announced March 19, 2020.

6

KOD

Wet age-related macular degeneration (AMD)

Phase 2/3

5/3/22

Phase 2b/3 primary results reported durability with nearly 60% on Q20W dosing, however trial did not meet primary endpoint, noted May 3, 2022.

6

DNLI

Amyotrophic lateral sclerosis (ALS)

Phase 1b

4/25/23

Phase 1b data due presented at AAN reported low variability, predictable dose-related increases in exposure, and a half-life supporting once-daily dosing, noted April 25, 2023.

6

AIM

Cisplatin resistant advanced recurrent ovarian cancer

Phase 1/2

1/24/22

Phase 1/2 IP chemo-immunotherapy combination demonstrated to be well tolerated, and associated with interferon stimulated gene changes that favor cytotoxic T lymphocytes chemo-attraction and function, noted January 24, 2022.

6

TEVA

Tourette Syndrome

Phase 2/3

2/19/20

Phase 2/3 trial did not meet primary endpoint - February 19, 2020.

4

RYTM

POMC deficiency obesity / Leptin Receptor Deficiency Obesity

Approved

11/27/20

FDA approval announced November 27, 2020.

6

BMY

First-line Glioblastoma (GBM) cancer

Phase 3

12/23/20

Phase 3 trial unlikely to meet OS primary endpoint

4

BYSI

Non small cell lung cancer

Phase 3

12/13/22

Additional data reported that plinabulin demonstrated a superior benefit for Gr4N, Gr3/4N, all GrN and DSN compared to the control, noted December 13, 2022.

6

RZLT

Congenital hyperinsulinism (HI)

Phase 2b

3/23/22

Phase 2b study demonstrated highly significant improvements in hypoglycemia and good safety and tolerability, noted March 23, 2022.

6

MOLN

Age-related macular degeneration (AMD)

CRL

6/26/20

CRL issued June 26, 2020.

5

TAK

Pouchitis

Phase 3

3/30/23

Phase 4 met primary endpoint, noted March 30, 2023.

5

AZN

Thyroid cancer

Phase 3

7/26/18

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

4

ZYME

HER2-expressing Gastrointestinal Cancers (Gastroesophageal Adenocarcinoma, Biliary Tract Cancer, and Colorectal Cancer)

Phase 2

1/19/23

Phase 2 data presented at ASCO Gastrointestinal Cancers Symposium reported an overall confirmed objective response rate (cORR) of 79%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 12.5 months, noted January 19, 2023.

6

ADAP

Esophageal and esophagogastric junction cancers

Phase 2

11/8/22

Phase 2 data reported that ORR has increased to 37% from 33%, and the median duration of response has increased to ~20 weeks from approximately 12 weeks, noted November 8, 2022.

5

AIM

CFS

CRL

2/4/13

CRL February 4, 2013.

5

SYBX

Cirrhotic patients with elevated ammonia

Phase 1/2

8/20/19

Development to be discontinued due to lack of efficacy.

4

NAVB

Head and Neck Cancer

Approved

6/13/14

Approved June 13, 2014.

6

LGND

ER+/HER2- Breast Cancer

Phase 2

6/5/23

Phase 2 data presented at ASCO reported that PFS was 56.0 wks (~13 mos), CBR was 65.5%, and ORR was 55.6%, noted June 5, 2023.

6

MLTX

Hidradenitis suppurativa

Phase 2

10/16/23

Phase 2 24-week topline data reported that 57% of patients continuously treated with 120mg achieved a HiSCR75 response and 38% achieved HiSCR90, noted October 16, 2023.

6

ABBV

B-cell Lymphoma (DLBCL)

Approved

5/22/23

Approved May 19, 2023.

6

RHHBY

Alzheimer’s disease

Phase 3

1/30/19

Phase 3 trial terminated following interim analysis indicating primary endpoint would not be met.

4

BMY

Relapsed and/or Refractory (R/R) Multiple Myeloma (MM)

Phase 1

12/10/22

Phase 1 data is presented at ASH demonstrated deep and durable responses with a manageable safety profile across all dose levels, including patients previously treated with a B-cell maturation antigen (BCMA)-directed CAR T cell therapy, noted December 10, 2022.

6

EVFM

contraceptive vaginal gel

Approved

5/22/20

FDA Approval announced May 22, 2020.

6

VTRS

Dry eye disease

Approved

2/3/22

Approved February 3, 2022.

6

AMGN

Solid tumors with MTAP deletion, NSCLC

Phase 1

3/15/22

Phase 1 updated data reported no drug-related SAE's or observed dose limiting toxicity (DLT) through Cohort 5, noted March 15, 2022.

6

PFE

Chronic low back pain

Phase 3

2/19/19

Phase 3 data released February 19, 2019. 10mg dose met primary endpoint. 5mg dose did not meet endpoint.

5

GSK

RSV vaccine

Approved

5/3/23

FDA Approved on May 3, 2023.

6

MOLN

COVID-19

Phase 2a

12/6/21

Phase 2a data reported an infectivity of virus extracted through nasopharyngeal swabs was reduced to zero within 3-5 days post treatment in patients with positive titers at baseline, noted December 6, 2021.

5

APTO

Acute myeloid leukemia (AML)

Phase 1/2

10/30/23

Phase 1/2 data demonstrated 44% ORR with TUS/VEN in Difficult-to-Treat VEN Failure R/R AML Patients, noted October 30, 2023.

6

APLT

Diabetic cardiomyopathy (DbCM)

Phase 3

6/22/23

Phase 3 baseline data to be presented at ADA showed a strong statistical correlation between elevations in the cardiac stress biomarker, NT-proBNP, reduced cardiac functional capacity and physical activity, underscoring the importance of early diagnosis via NT-proBNP and impact of DbCM on physical function and quality of life, noted June 22, 2023

6

MRK

Cervical cancer

Phase 3

7/19/23

Phase 3 data reported that trial met the PFS primary endpoint, noted July 19, 2023.

5

AZN

Relapsed or Refractory Mantle Cell Lymphoma

Approved

10/31/17

Priority Review announced August 2, 2017. Approval announced October 31, 2017.

6

PTCT

Mitochondrial Epilepsy

Phase 2/3

6/29/23

Phase 2/3 data reported that trial did not meet the primary endpoint, noted June 29, 2023.

6

AXSM

Narcolepsy

Phase 2

12/3/19

Phase 2 data met primary endpoint.

6

XCUR

Merkel cell carcinoma / Cutaneous squamous cell carcinoma

Phase 1/2

8/5/21

Phase 1b/2 interim data noted overall response rate (ORR) of 21% in all evaluable MCC patients - August 5, 2021.

5

ZVRA

Urea cycle disorder (UCD)

Approved

12/23/22

Approved December 23, 2022.

6

AEZS

Endometrial cancer

Phase 3

5/1/17

Phase 3 trial did not meet primary endpoint - May 1, 2017.

4

HCM

Myeloid hematological malignancies (HMs)

Phase 1

6/9/23

Phase 1 data reported that the best of response was 1CR in 100 mg cohort; 9 CRs and 1 PR in 150 mg QD; 1 CR in 200 mg QD; and 3 CRs in 250 mg QD; with an ORR of 33.3% (1/3), 40.0%(10/25), 14.3% (1/7) and 42.9% (3/7), respectively, noted June 9, 2023.

5

AADI

PEComa

Approved

11/23/21

Approved November 23, 2021.

6

ARAV

Pancreatic Adenocarcinoma

Phase 1b

5/25/23

Phase 1b data from ASCO reported that the median overall survival (OS) for patients with trough levels above the minimal batiraxcept efficacious concentration is greater than 15 months, and one patient demonstrated a complete response from 10 months to 20 months and is still on study, noted May 25, 2023.

6

QSAM

Bone cancer

Phase 1

8/16/23

Initial Phase 1 data reported that treatment was well-tolerated with no serious adverse events, a 56% - 64% decrease in SUV in patient one, and that the first two patients experienced significant pain relief and improved mobility within a week after treatment, noted August 16, 2023.

6

IRWD

Irritable Bowel Syndrome with Constipation (IBS-C)

Phase 3

6/18/19

Phase 3b data met all endpoints - June 18, 2019.

6

PFE

Squamous cell carcinoma of the head and neck (SCCHN)

Phase 2

4/17/23

Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.

6

AZN

Endometrial cancer (EC)

Phase 3

10/23/23

Phase 3 results presented at ESMO reported statistically significant and clinically meaningful PFS benefit vs CP in the ITT population, with the interim OS data being immature (27.7%) yet with a trend towards benefit, noted October 21, 2023.

6

MRK

Non-transfusion-dependent beta-thalassemia

Phase 2

6/11/21

Phase 2 data presented at EHA meeting June 11, 2021. 77.7% of patients achieved a hemoglobin increase compared to 0% in the placebo arm.

6

AMGN

Osteoporosis

Approved

4/9/19

FDA Approval announced April 9, 2019.

6

RHHBY

Duchenne muscular dystrophy

Phase 3

10/30/23

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

6

CNTA

Kidney Disease

Phase 3

12/14/21

Phase 3 data reported that ALT elevation peaked between 1.8x and 3.5x ULN and did not return to below ULN until 23 to 140 days after tolvaptan use was discontinued. Four patients were successfully titrated to a maintenance dose of lixivaptan of either 100 mg BID or 200 mg BID, noted December 14, 2021.

6

EXEL

Solid Tumors

Phase 1/2

1/18/22

Phase 1/2 cohort results showed that of the 36 patients enrolled in cohort 2 of the CAMILLA trial, 29 were evaluable for the efficacy analysis. The primary outcome of investigator-assessed ORR per modified RECIST version 1.1 was 27.6%. The confirmed PR rate was 20.7%, and the DCR was 86.2%. Median PFS was 3.8 months, with a 6-month PFS of 34.5%. Median OS was 9.1 months, noted January 18, 2022.

6

EFTR

Solid Tumors RTK BC and KRAS NSCLC

Phase 1/2

6/6/22

Phase 1/2 presentation at ASCO reported 2 partial responses, noted June 6, 2022.

6

CMRX

Adenovirus Infection

Phase 3

2/22/17

Phase 3 interim analysis released May 2016. Final data released February 22, 2017.

5

BFRI

Actinic Keratosis on face & scalp

Phase 1

8/28/23

Phase 1 data reported that treatment was generally well tolerated and all TEAEs were transient, noted August 28, 2023.

6

DRUG

Dravet Syndrome

Phase 1/2

8/8/23

Additional Phase 1/2 cohort 4 data reported an increase in central delta power and robust reduction in central alpha and beta power in active group, as previously reported for Anti-Epileptic Drugs (AEDs) in healthy individuals, noted August 8, 2023.

6

ELDN

Kidney transplant rejection

Phase 2

11/2/23

Phase 2 data from 11 participants demonstrates tegoprubart successfully prevented kidney transplant rejection and was generally safe and well-tolerated, noted November 2, 2023.

6

AZN

Asthma

Phase 3

5/16/22

Phase 3 results reported that treatment reduced the risk of a severe exacerbation by 27%, noted May 16, 2022.

6

ZNTL

Ovarian cancer

Phase 1b

6/5/23

Phase 1b dose escalation data presented at ASCO reported that confirmed ORR of 50.0% and mPFS of 7.4 months in combination with paclitaxel, and a confirmed ORR of 35.7% and mPFS of 10.4 months in combination with carboplatin, noted June 5, 2023.

6

RVNC

Lateral Canthal (Crow’s Feet) Lines

Phase 2

6/30/20

Phase 2 top-line data released June 30, 2020. 88% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group.

6

OPK

Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) - Stage 5

Phase 2

11/2/23

Phase 2 data presented at ASH indicated that early, sustained and effective treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression CKD in pre-dialysis patients, noted November 2, 2023.

6

BMY

(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC)

Approved

8/1/17

Approval announced August 1, 2017.

6

ATNXQ

Angiosarcoma

Phase 2

12/9/20

Phase 2 poster at SABCS December 9, 2020 - Complete response (CR) 43% (3/7 pts), partial response (PR) 14% (1/7 pts).

5

ACAD

Adjunctive Treatment in Patients With Major Depressive Disorder

Phase 3

7/20/20

Phase 3 trial did not meet primary endpoint - July 20, 2020.

4

BIIB

Alzheimer’s disease

Phase 3

9/13/19

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

4

GPCR

Type 2 Diabetes

Phase 1b

9/29/23

Phase 1b results reported that GSBR-1290 demonstrated significant weight loss supporting once-daily (QD) dosing, noted September 29, 2023.

6

MRK

Triple negative breast cancer (TNBC)

Phase 3

5/20/19

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

4

AMGN

Wild-Type RAS Metastatic Colorectal Cancer

Approved

6/29/17

sBLA approval announced June 29, 2017.

6

JNJ

metastatic hormone-naïve prostate cancer (mHNPC)

Approved

2/8/18

sNDA approval announced February 8, 2018.

6

PDSB

Human papillomavirus (HPV) associated cancers

Phase 2

12/28/22

Phase 2 data reported a median OS of 21 months, with 75% remain alive at a median follow-up of 27 months, noted December 28, 2022.

5

ARQT

Plaque psoriasis in children

Approved

10/6/23

Approved for treatment of children aged 6 - 11 on October 6, 2023.

6

INO

HPV 6 and HPV 11-associated Recurrent respiratory papillomatosis

Phase 1/2

10/10/23

Phase 1/2 received feedback from the FDA that data could support INOVIO's submission of a BLA for review under the FDA's accelerated approval program. The FDA also advised that the company's previously planned Phase 3 trial would not be required to support this submission, noted October 10, 2023.

6

ALNY

Primary Hyperoxaluria Type 1

Phase 3

9/30/20

Clinically meaningful reduction in spot urinary oxalate:creatinine ratio.

6