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
RHHBY	Hemophilia A	Phase 1/2	12/12/22	Phase 1/2 results reported a 92% reduction in ABR for all bleeds, with all-bleed ABR of 0.98 post-infusion compared to 11.62, noted December 12, 2022.	6
EIGR	Hepatitis Delta Virus (HDV) infection	Phase 3	9/12/23	Phase 3 LIMT-2 trial discontinued due to DSMB recommendation, noted September 12, 2023.	4
MRK	Hospital-acquired bacterial pneumonia (HABP)	Approved	6/3/19	FDA approval announced June 3, 2019.	6
INCY	COVID-19	Approved	5/11/22	FDA approval on May 11, 2022.	6
MRK	Heart Failure	Approved	1/20/21	FDA approval announced January 20, 2021.	6
ABBV	Biosimilar candidate to Avastin (bevacizumab)	Approved	9/14/17	Approved September 14, 2017.	6
AQST	Erectile dysfunction	CRL	11/16/18	CRL issued November 16, 2018.	5
LEGN	Relapsed/Refractory Multiple Myeloma (R/R MM)	Phase 2	12/12/22	Phase 2 26-month data reported that median PFS and OS were NR; 24-month PFS and OS rates were 52.6% and 74.2%, respectively, noted December 12, 2022.	5
BOLT	Solid Tumors	Phase 1/2	10/17/23	Phase 1/2 initiated, noted October 17, 2023.	5
EYPT	Non-proliferative diabetic retinopathy (NPDR)	Phase 2	9/11/23	Phase 2 interim analysis of masked data showed that EYP-1901 is well tolerated with no reported drug-related ocular or systemic serious adverse events, noted September 11, 2023.	6
EXEL	Advanced Neuroendocrine Tumors	Phase 3	10/23/23	Phase 3 data from ESMO 23 reported that the trial met the primary objective for each cohort, demonstrating that cabozantinib provided dramatic improvements in median progression-free survival (PFS) for the patients in the pNET and epNET cohorts, noted October 23, 2023.	6
AZN	Severe, uncontrolled asthma	Approved	11/14/17	Approval announced November 14, 2017.	6
BIIB	Chronic Kidney Disease	Phase 2	5/10/23	Program discontinued, noted May 10, 2023.	4
CTIC	B-cell non-Hodgkin lymphoma	Phase 3	7/9/18	Phase 3 data released July 10. 2018. Primary endpoint not met.	4
BMY	Multiple Myeloma	Phase 3	3/9/20	Phase 3 trial did not meet progression-free survival primary endpoint.	4
VNDA	Bipolar Disorder	Phase 3	12/19/22	Phase 3 trial results reported that treated patients showed a larger improvement than placebo treated patients, and this difference was highly statistically significant, noted December 19, 2022.	6
CGTX	Mild-to-Moderate Alzheimer's Disease	Phase 2	6/28/23	Phase 2 study met its primary endpoints for safety and tolerability and showed positive effects for CT1812-treated participants as measured via quantitative electroencephalogram (qEEG), noted June 28, 2023.	6
ADAP	Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS)	Phase 1	5/26/22	Phase 1 data released May 26, 2022. The overall response rate was 36% in heavily pre-treated patients across both types of sarcomas (41% in synovial sarcoma and 10% for MRCLS), with a median duration of response of 52 weeks. As reported last year, the pivotal trial SPEARHEAD-1 met its primary endpoint for efficacy and the benefit:risk profile of afami-cel has been favorable with mainly low-grade cytokine release syndrome and tolerable/reversible hematologic toxicities.	6
NEXI	Multiple Myeloma	Phase 1/2	12/12/21	Phase 1/2 data reported that treatment is well-tolerated without dose-limiting toxicities, noted December 12, 2021.	6
NVO	Hemophilia A	CRL	5/4/23	CRL received in 2Q 2023.	5
ABUS	Healthy volunteers	Phase 1	9/11/23	Phase 1 discontinued, noted September 11, 2023.	4
REGN	Prurigo nodularis (PN)	Approved	10/27/22	Approved in 3Q 2022.	6
KMDA	Prophylaxis of rabies disease	Approved	8/25/17	Approval announced August 25, 2017.	6
GRCL	Diffuse large B-cell lymphoma (DLBCL)	Phase 1	9/27/23	Additional Phase 1 data presented at IMS reported an 100% overall response rate (ORR) and a 100% MRD- sCR rate among 19 transplant-eligible, high-risk NDMM patients, noted September 27, 2023.	6
MRK	Triple negative breast cancer (TNBC)	Phase 3	9/19/21	Phase 3 overall survival data showed risk of death reduced by 27%, and an increase of 6.9 months in median OS with KEYTRUDA plus chemotherapy. OS with treatment was 23 months, versus 16.1 months with chemotherapy, September 19, 2021.	6
NKTR	Solid tumors	Phase 1/2	11/12/21	Phase 1b data from 1b/2 trial reported 1 partial response (PR) and 5 stable disease (SD). Treatment was noted to be well tolerated, November 12, 2021.	6
INCY	Myelfibrosis	Phase 2	12/12/22	Phase 2 data reported that 28.1% (9), 3.1% (1) and 0% of patients who received daily followed by weekly dosing experienced ≥10%, ≥25% and ≥35% reduction in spleen volume, respectively, noted December 12, 2022,	6
AMAM	HER2-positive metastatic breast cancer	Phase 3	3/1/23	Phase 3 trial met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater progression-free survival benefit compared to the control, noted March 1, 2023.	6
BCTX	Breast cancer	Phase 1/2	2/23/23	Additional Phase 1/2 data reported that 9 of 11 (82%) patients remain alive from 2021/2022 dosing and 7 of 11 (64%) patients showed either disease control or progression-free survival, noted February 23, 2023.	6
CMRX	Allogeneic hematopoietic stem cell transplant (HCT) recipients with adenovirus (AdV)	Phase 2	5/9/19	Phase 2 trial discontinued - May 9, 2019.	4
CADL	Glioma	Phase 1	5/19/23	Additional Phase 1 data presented at ASGCT reported a median overall survival rate at 11.8 months after a single injection, noted May 19, 2023.	6
CRDF	KRAS-Mutated Colorectal Cancer	Phase 1/2	9/10/22	Phase 1b/2 data presented at ESMO showed durable responses to treatment, with a median duration of response (mDoR) of 11.7 months, noted September 10, 2022.	6
CERS	Chronic anemia in thalassemia patients	Phase 3	1/23/18	Phase 3 trial met endpoints - January 23, 2018. CE mark submission due 2H 2018.	6
EIGR	Hutchinson-Gilford Progeria Syndrome (HGPS)	Approved	11/20/20	FDA approval announced November 20, 2020.	6
ITCI	Agitation in patients with dementia	Phase 2	12/18/18	Phase 3 interim analysis December 18, 2018 noted trial to stop due to futility.	4
PCSA	Gastroparesis	Phase 2a	5/6/23	Phase 2a data reported at DDW noted that treatment improved both gastroparesis symptom scores and gastric emptying rate, noted May 6, 2023.	6
PCRX	Postsurgical pain	Approved	10/31/11	Approved October 31, 2011.	6
JNCE	Second-line non-small cell lung cancer (NSCLC) patients	Phase 2	8/30/22	Phase 2 trial did not meet its primary endpoint, noted August 30, 2022.	6
MVIR	Advanced hepatocellular carcinoma (HCC)	Phase 2a	10/5/23	Phase 2a data reported that 3 patients achieved Partial Response and 10 patients Stable Disease, providing 17% Overall Response Rate (ORR) and 72% Disease Control Rate (DCR), noted October 5, 2023.	6
BMY	Non-Small Cell Lung Cancer (NSCLC)	Approved	4/11/22	Approved April 11, 2022.	6
RYTM	Alstrom Syndrome	CRL	6/16/22	CRL issued for sNDA application for Alstrom Syndrome issued June 16, 2022.	5
PTCT	Spinal Muscular Atrophy (SMA) in babies	Approved	5/31/22	Approved May 31, 2022.	6
RHHBY	B-Cell Non-Hodgkin's Lymphoma	Approved	6/16/23	FDA Approved on June 16, 2023.	6
OMER	Hematopoietic stem cell-associated TMA (HSCT-TMA)	CRL	10/18/21	CRL announced October 18, 2021.	5
ABBV	Jawline definition	Approved	8/3/22	Approved August 3, 2022.	6
AZN	Venous thromboembolism (VTE) Prevention	Approved	6/23/17	FDA Approval noted June 23, 2017.	6
BMRN	Children under age 5 with Achondroplasia	Approved	10/23/23	Approved October 23, 2023.	6
TAK	Complex Crohn's Perianal Fistulas (CPF)	Phase 3	10/18/23	Phase 3 trial did not meet its primary endpoint, noted October 18, 2023.	6
KALV	Diabetic macular edema (DME)	Phase 2	12/9/19	Phase 2 data did not meet primary endpoint - December 9, 2019.	4
AGEN	Solid tumors	Phase 1/2	2/9/21	Phase 1/2 data announced February 9, 2021 - six confirmed responses.	5
LLY	Advanced solid tumors	Phase 1b	4/18/23	Phase 1b data presented at AACR showed that dosing was well-tolerated with favorable pharmacokinetics and no apparent drug-drug interactions, noted April 18, 2023.	6
BIVI	Parkinson's disease	Phase 2a	8/28/23	Phase 2a results reported that in comparison of Day 1 and Day 14 of treatment, PK parameters demonstrated that NE3107 administration did not affect the PK profile of levodopa, noted August 28, 2023.	6
AUPH	Lupus	Approved	1/22/21	FDA Approval announced January 22, 2021.	6
MRK	Metastatic non-small cell lung cancer (NSCLC)	Phase 1b	6/5/23	Phase 1b presented at ASCO demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts, noted June 6, 2023	6
KMDA	AATD - Alpha-1 Antitrypsin deficiency	Phase 2	8/30/16	Top-line data released August 30, 2016. Primary endpoint met	6
AKBA	Hyperphosphatemia	Approved	9/5/14	Approved Sept 5, 2014.	6
ALXO	2nd line or greater gastric/gastroesophageal junction cancer	Phase 2	10/3/23	Phase 2 data reported a confirmed overall response rate for evorpacept combination treatment was 52% compared to 22% for control treatment, noted October 3, 2023.	6
TAK	Psoriasis	Phase 2b	9/11/23	Additional Phase 2b data reported that the safety and tolerability profile of TAK-279 in the Phase 2b trial was consistent with previous TAK-279 clinical trials, noted September 11, 2023.	6
MRK	Beta-thalassemia	Approved	11/8/19	FDA Approval announced November 8, 2019.	6
PFE	Hemophilia B	Phase 3	12/29/22	Phase 3 trial met primary endpoint, noted December 29, 2022.	5
GSK	Endometrial Cancer	Approved	7/31/23	FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.	6
FGEN	Anaemia in Chronic Kidney Disease	CRL	8/11/21	CRL announced August 11, 2021.	5
SWTX	Multiple Myeloma	Phase 1/2	6/9/23	Phase 1/2 data presented at EHA Data from 34 patients with low-dose and 37 patients with monotherapy are presented. Patients had a median (range) age of 68 (48–81) years and a median (range) of 5 (3–14) prior lines of therapy. As of the data cutoff (Dec 9, 2022), patients received a median (range) of 4 (1–20) cycles of the combination and 3 (1–9) monotherapy cycles. For the combination and monotherapy arms, respectively, ORR was 29% (95% CI 15.1, 47.5) and 38% (22.5, 55.2) (Table). Incidence of Grade ≥3 adverse events was 76% and 65%, noted June 9, 2023.	6
HALO	Rheumatoid arthritis (RA)	Approved	10/14/13	Approved Oct 14, 2013.	6
ZLDPF	Pediatric Congenital hyperinsulinism (CHI)	Phase 3	9/19/22	Phase 3 trial additional data showed that treatment significantly reduced the requirement for intravenous (IV) glucose to maintain glycemia in neonates and infants with CHI and reduced glucose requirements to levels that potentially allow for discontinuation of IV glucose support, noted September 19, 2022.	6
NVO	Prostate cancer (CRPC)	Phase 1	10/7/21	Phase 1 initial preliminary results reported include data as of Sept. 1, 2021, from eight men enrolled in the trial. The dose was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. (3/8) patients remain on study; (5/8) patients left the study (4 due to disease progression and 1 withdrawal of consent).	5
ABBV	Rheumatoid arthritis	Approved	8/16/19	FDA Approval announced August 16, 2019.	6
PRTA	AL Amyloidosis	Phase 3	12/12/22	Phase 3 VITAL data reported a significant improvement in time to all-cause mortality, noted December 12, 2022.	6
PFE	ALK-positive Anaplastic Large Cell Lymphoma	Approved	1/14/21	FDA approval announced January 14, 2021.	6
CARA	Stage 3-4 Chronic Kidney Disease-Associated Pruritus	Phase 2	12/3/19	Phase 2 data met primary endpoint but secondary missed - December 3, 2019.	5
ACHV	Smoking cessation	Phase 3	3/3/23	Additionnal Phase 3 data presented at the Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting confirmed that successful abstinence was observed in subgroups of smokers who received cytisinicline. Subjects who received either 6 or 12-weeks of cytisinicline treatment experienced consistently higher rates of abstinence, noted March 3, 2023.	6
BLRX	First-line pancreatic ductal adenocarcinoma (PDAC)	Phase 2	9/28/23	Phase 2 trial data presented at the AACR Special pancreatic cancer conference reported that 6 patients (55%) experienced a partial response (PR) of which 4 (36%) were confirmed PRs with one patient experiencing resolution of the hepatic (liver) metastatic lesion, noted September 28, 2023.	6
SAGE	Major Depressive Disorder (MDD)	Phase 3	8/7/23	Phase 3 additional data reported that there were no new safety signals identified, noted August 7, 2023.	5
ANAB	Endometrial Cancer	Approved	7/31/23	FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.	6
ELTX	Acute lung injury in patients with COVID-19	Phase 3	6/29/21	Phase 2 trial did not meet primary or secondary efficacy endpoints - June 29, 2021.	4
ACHV	Smoking cessation of cigarettes	Phase 3	5/23/23	Phase 3 topline results reported that subjects that received treatment had 2.85 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p=0.0008). The smoking cessation rate during weeks 3 through 6 was 14.8% for cytisinicline compared to 6% for placebo, noted May 23, 2023.	6
INBX	Alpha-1 Antitrypsin Deficiency (AATD)	Phase 1	11/2/22	Phase 1 data presented at ACoP noted that treatment demonstrated the potential to achieve and maintain normal levels of functional AAT. The 120 mg/kg Q4W regimen is predicted to achieve average trough values >21.1 µM, noted November 2, 2022.	6
MRK	Complicated urinary tract infections (cUTI) and Complicated intra-abdominal infections (cIAI)	Approved	7/17/19	FDA Approval announced July 17, 2019.	6
RHHBY	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3	12/12/22	Phase 3 results reported that trial met primary endpoints, noted December 12, 2022.	5
NBIX	Parkinson's disease	Approved	4/27/20	FDA Approval announced April 27, 2020.	6
VERU	COVID-19 / Severe Acute Respiratory Syndrome, SARS-CoV-2, Influenza A and B, Respiratory Syncytial Virus (RSV) and other viruses	Phase 3	9/26/23	Phase 3 new study authorized by FDA, with an FDA meeting in September 2023 resulted in agreement with FDA on New Phase 3 Clinical Trial for Sabizabulin for Broader Indication, noted September 26, 2023.	6
TAK	Post-transplant Hodgkin lymphoma (HL) cancer	Approved	8/17/15	Approved August 17, 2015 under priority review.	6
PFE	BRAF mutant melanoma cancer	Approved	6/27/18	Approval announced June 27, 2018.	6
IMCR	Solid tumors	Phase 1	12/8/22	Phase 1 initial data reported that one patient had a durable Partial Response (PR), with a duration of 12.7 months, one patient who had a Stable Disease (SD) converted to an unconfirmed PR after the poster data cutoff date and is still ongoing, and 5 had SD, noted December 8, 2022.	5
HRMY	Fragile X syndrome	Phase 2/3	5/13/22	Phase 2/3 long-term data reported that sustained improvement in ABC-CFXS Social Avoidance from baseline, noted May 13, 2022.	6
ELOX	Cystic fibrosis	Phase 2	11/17/21	Phase 2 monotherapy dosing data at 1.5mg/kg/day demonstrated a statistically significant 5.4mmol/L mean sweat chloride reduction, an established surrogate for restoration of CFTR biological activity, noted November 17, 2021.	6
SYRS	Pancreatic Cancer	Phase 1	11/14/22	Phase 1 safety data reported that treatment was generally safe, noted November 14, 2022.	6
ENDP	Men diagnosed with hypogonadism	Approved	3/6/14	Approved March 6, 2014.	6
SCPH	Chronic heart failure	Phase 2	7/12/22	Phase 2 trial data reported a 37% reduction in the risk of a heart failure hospitalization relative to patients randomized to "treatment as usual" at day 30, noted July 12, 2022.	6
REGN	Various cancers	Phase 1	11/4/23	Phase 1 safety data reported that a total of 11 adverse events (including one serious adverse event not considered related to SNS-101) was reported in five patients, with no dose-limiting toxicities observed, noted November 4, 2023.	6
BLPH	COVID-19	Phase 3	11/23/20	Phase 3 trial placed on clinical hold - November 23, 2020.	4
VIR	HIV T cell vaccine	Phase 1	5/8/23	Phase 1 initial data reported that no safety signals and no vector shedding or viremia were reported, noted June 28, 2023.	5
GSK	Chronic rhinosinusitis with nasal polyps	Approved	7/29/21	FDA approval announced July 29, 2021.	6
IOVA	Cervical cancer	Phase 2	5/31/19	Data released May 31, 2019 noted ORR 44%; CR 11%.	5
CORT	Pancreatic cancer	Phase 3	6/22/21	Phase 3 initial data released June 22, 2021. 2/31 (6%) partial responses. Enrollment to be stopped. Level of benefit does not justify further study.	4
MRK	Non-small cell lung cancer (NSCLC)	Approved	4/11/19	FDA Approval announced April 11, 2019.	6
IMGO	Myelofibrosis	Phase 2	12/12/22	Phase 2 data reported that 65% (17/26) of patients showed a decrease in Total Symptom Score (TSS), 19% (5/26) showed a ≥ 50% decrease in TSS, 66% (33/50) showed spleen volume reductions from baseline, and 28% (14/50) showed a ≥ 20% spleen volume reduction, noted December 12, 2022.	6
SONN	Healthy volunteers	Phase 1	11/2/23	Phase 1 interim analysis presented at CTAD reported that in single doses up to 300 ng/kg and in patients with advanced cancer in repeated doses up to at least 540 ng/kg. Incorporation of a desensitizing first dose at 150-300 ng/kg and dosing every 3 weeks has been implemented to capitalize on the potential benefits of tachyphylaxis with rhIL-12, noted November 2, 2023.	6
RLMD	Major depressive disorder (MDD)	Phase 3	12/7/22	Phase 3 trial did not meet primary endpoint, noted December 7, 2022.	6
BMRN	Phenylketonuria (PKU)	Approved	5/24/18	Approval announced May 24, 2018.	6

RHHBY

Hemophilia A

Phase 1/2

12/12/22

Phase 1/2 results reported a 92% reduction in ABR for all bleeds, with all-bleed ABR of 0.98 post-infusion compared to 11.62, noted December 12, 2022.

6

EIGR

Hepatitis Delta Virus (HDV) infection

Phase 3

9/12/23

Phase 3 LIMT-2 trial discontinued due to DSMB recommendation, noted September 12, 2023.

4

MRK

Hospital-acquired bacterial pneumonia (HABP)

Approved

6/3/19

FDA approval announced June 3, 2019.

6

INCY

COVID-19

Approved

5/11/22

FDA approval on May 11, 2022.

6

MRK

Heart Failure

Approved

1/20/21

FDA approval announced January 20, 2021.

6

ABBV

Biosimilar candidate to Avastin (bevacizumab)

Approved

9/14/17

Approved September 14, 2017.

6

AQST

Erectile dysfunction

CRL

11/16/18

CRL issued November 16, 2018.

5

LEGN

Relapsed/Refractory Multiple Myeloma (R/R MM)

Phase 2

12/12/22

Phase 2 26-month data reported that median PFS and OS were NR; 24-month PFS and OS rates were 52.6% and 74.2%, respectively, noted December 12, 2022.

5

BOLT

Solid Tumors

Phase 1/2

10/17/23

Phase 1/2 initiated, noted October 17, 2023.

5

EYPT

Non-proliferative diabetic retinopathy (NPDR)

Phase 2

9/11/23

Phase 2 interim analysis of masked data showed that EYP-1901 is well tolerated with no reported drug-related ocular or systemic serious adverse events, noted September 11, 2023.

6

EXEL

Advanced Neuroendocrine Tumors

Phase 3

10/23/23

Phase 3 data from ESMO 23 reported that the trial met the primary objective for each cohort, demonstrating that cabozantinib provided dramatic improvements in median progression-free survival (PFS) for the patients in the pNET and epNET cohorts, noted October 23, 2023.

6

AZN

Severe, uncontrolled asthma

Approved

11/14/17

Approval announced November 14, 2017.

6

BIIB

Chronic Kidney Disease

Phase 2

5/10/23

Program discontinued, noted May 10, 2023.

4

CTIC

B-cell non-Hodgkin lymphoma

Phase 3

7/9/18

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

4

BMY

Multiple Myeloma

Phase 3

3/9/20

Phase 3 trial did not meet progression-free survival primary endpoint.

4

VNDA

Bipolar Disorder

Phase 3

12/19/22

Phase 3 trial results reported that treated patients showed a larger improvement than placebo treated patients, and this difference was highly statistically significant, noted December 19, 2022.

6

CGTX

Mild-to-Moderate Alzheimer's Disease

Phase 2

6/28/23

Phase 2 study met its primary endpoints for safety and tolerability and showed positive effects for CT1812-treated participants as measured via quantitative electroencephalogram (qEEG), noted June 28, 2023.

6

ADAP

Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS)

Phase 1

5/26/22

Phase 1 data released May 26, 2022. The overall response rate was 36% in heavily pre-treated patients across both types of sarcomas (41% in synovial sarcoma and 10% for MRCLS), with a median duration of response of 52 weeks. As reported last year, the pivotal trial SPEARHEAD-1 met its primary endpoint for efficacy and the benefit:risk profile of afami-cel has been favorable with mainly low-grade cytokine release syndrome and tolerable/reversible hematologic toxicities.

6

NEXI

Multiple Myeloma

Phase 1/2

12/12/21

Phase 1/2 data reported that treatment is well-tolerated without dose-limiting toxicities, noted December 12, 2021.

6

NVO

Hemophilia A

CRL

5/4/23

CRL received in 2Q 2023.

5

ABUS

Healthy volunteers

Phase 1

9/11/23

Phase 1 discontinued, noted September 11, 2023.

4

REGN

Prurigo nodularis (PN)

Approved

10/27/22

Approved in 3Q 2022.

6

KMDA

Prophylaxis of rabies disease

Approved

8/25/17

Approval announced August 25, 2017.

6

GRCL

Diffuse large B-cell lymphoma (DLBCL)

Phase 1

9/27/23

Additional Phase 1 data presented at IMS reported an 100% overall response rate (ORR) and a 100% MRD- sCR rate among 19 transplant-eligible, high-risk NDMM patients, noted September 27, 2023.

6

MRK

Triple negative breast cancer (TNBC)

Phase 3

9/19/21

Phase 3 overall survival data showed risk of death reduced by 27%, and an increase of 6.9 months in median OS with KEYTRUDA plus chemotherapy. OS with treatment was 23 months, versus 16.1 months with chemotherapy, September 19, 2021.

6

NKTR

Solid tumors

Phase 1/2

11/12/21

Phase 1b data from 1b/2 trial reported 1 partial response (PR) and 5 stable disease (SD). Treatment was noted to be well tolerated, November 12, 2021.

6

INCY

Myelfibrosis

Phase 2

12/12/22

Phase 2 data reported that 28.1% (9), 3.1% (1) and 0% of patients who received daily followed by weekly dosing experienced ≥10%, ≥25% and ≥35% reduction in spleen volume, respectively, noted December 12, 2022,

6

AMAM

HER2-positive metastatic breast cancer

Phase 3

3/1/23

Phase 3 trial met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater progression-free survival benefit compared to the control, noted March 1, 2023.

6

BCTX

Breast cancer

Phase 1/2

2/23/23

Additional Phase 1/2 data reported that 9 of 11 (82%) patients remain alive from 2021/2022 dosing and 7 of 11 (64%) patients showed either disease control or progression-free survival, noted February 23, 2023.

6

CMRX

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

Phase 2

5/9/19

Phase 2 trial discontinued - May 9, 2019.

4

CADL

Glioma

Phase 1

5/19/23

Additional Phase 1 data presented at ASGCT reported a median overall survival rate at 11.8 months after a single injection, noted May 19, 2023.

6

CRDF

KRAS-Mutated Colorectal Cancer

Phase 1/2

9/10/22

Phase 1b/2 data presented at ESMO showed durable responses to treatment, with a median duration of response (mDoR) of 11.7 months, noted September 10, 2022.

6

CERS

Chronic anemia in thalassemia patients

Phase 3

1/23/18

Phase 3 trial met endpoints - January 23, 2018. CE mark submission due 2H 2018.

6

EIGR

Hutchinson-Gilford Progeria Syndrome (HGPS)

Approved

11/20/20

FDA approval announced November 20, 2020.

6

ITCI

Agitation in patients with dementia

Phase 2

12/18/18

Phase 3 interim analysis December 18, 2018 noted trial to stop due to futility.

4

PCSA

Gastroparesis

Phase 2a

5/6/23

Phase 2a data reported at DDW noted that treatment improved both gastroparesis symptom scores and gastric emptying rate, noted May 6, 2023.

6

PCRX

Postsurgical pain

Approved

10/31/11

Approved October 31, 2011.

6

JNCE

Second-line non-small cell lung cancer (NSCLC) patients

Phase 2

8/30/22

Phase 2 trial did not meet its primary endpoint, noted August 30, 2022.

6

MVIR

Advanced hepatocellular carcinoma (HCC)

Phase 2a

10/5/23

Phase 2a data reported that 3 patients achieved Partial Response and 10 patients Stable Disease, providing 17% Overall Response Rate (ORR) and 72% Disease Control Rate (DCR), noted October 5, 2023.

6

BMY

Non-Small Cell Lung Cancer (NSCLC)

Approved

4/11/22

Approved April 11, 2022.

6

RYTM

Alstrom Syndrome

CRL

6/16/22

CRL issued for sNDA application for Alstrom Syndrome issued June 16, 2022.

5

PTCT

Spinal Muscular Atrophy (SMA) in babies

Approved

5/31/22

Approved May 31, 2022.

6

RHHBY

B-Cell Non-Hodgkin's Lymphoma

Approved

6/16/23

FDA Approved on June 16, 2023.

6

OMER

Hematopoietic stem cell-associated TMA (HSCT-TMA)

CRL

10/18/21

CRL announced October 18, 2021.

5

ABBV

Jawline definition

Approved

8/3/22

Approved August 3, 2022.

6

AZN

Venous thromboembolism (VTE) Prevention

Approved

6/23/17

FDA Approval noted June 23, 2017.

6

BMRN

Children under age 5 with Achondroplasia

Approved

10/23/23

Approved October 23, 2023.

6

TAK

Complex Crohn's Perianal Fistulas (CPF)

Phase 3

10/18/23

Phase 3 trial did not meet its primary endpoint, noted October 18, 2023.

6

KALV

Diabetic macular edema (DME)

Phase 2

12/9/19

Phase 2 data did not meet primary endpoint - December 9, 2019.

4

AGEN

Solid tumors

Phase 1/2

2/9/21

Phase 1/2 data announced February 9, 2021 - six confirmed responses.

5

LLY

Advanced solid tumors

Phase 1b

4/18/23

Phase 1b data presented at AACR showed that dosing was well-tolerated with favorable pharmacokinetics and no apparent drug-drug interactions, noted April 18, 2023.

6

BIVI

Parkinson's disease

Phase 2a

8/28/23

Phase 2a results reported that in comparison of Day 1 and Day 14 of treatment, PK parameters demonstrated that NE3107 administration did not affect the PK profile of levodopa, noted August 28, 2023.

6

AUPH

Lupus

Approved

1/22/21

FDA Approval announced January 22, 2021.

6

MRK

Metastatic non-small cell lung cancer (NSCLC)

Phase 1b

6/5/23

Phase 1b presented at ASCO demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts, noted June 6, 2023

6

KMDA

AATD - Alpha-1 Antitrypsin deficiency

Phase 2

8/30/16

Top-line data released August 30, 2016. Primary endpoint met

6

AKBA

Hyperphosphatemia

Approved

9/5/14

Approved Sept 5, 2014.

6

ALXO

2nd line or greater gastric/gastroesophageal junction cancer

Phase 2

10/3/23

Phase 2 data reported a confirmed overall response rate for evorpacept combination treatment was 52% compared to 22% for control treatment, noted October 3, 2023.

6

TAK

Psoriasis

Phase 2b

9/11/23

Additional Phase 2b data reported that the safety and tolerability profile of TAK-279 in the Phase 2b trial was consistent with previous TAK-279 clinical trials, noted September 11, 2023.

6

MRK

Beta-thalassemia

Approved

11/8/19

FDA Approval announced November 8, 2019.

6

PFE

Hemophilia B

Phase 3

12/29/22

Phase 3 trial met primary endpoint, noted December 29, 2022.

5

GSK

Endometrial Cancer

Approved

7/31/23

FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.

6

FGEN

Anaemia in Chronic Kidney Disease

CRL

8/11/21

CRL announced August 11, 2021.

5

SWTX

Multiple Myeloma

Phase 1/2

6/9/23

Phase 1/2 data presented at EHA Data from 34 patients with low-dose and 37 patients with monotherapy are presented. Patients had a median (range) age of 68 (48–81) years and a median (range) of 5 (3–14) prior lines of therapy. As of the data cutoff (Dec 9, 2022), patients received a median (range) of 4 (1–20) cycles of the combination and 3 (1–9) monotherapy cycles. For the combination and monotherapy arms, respectively, ORR was 29% (95% CI 15.1, 47.5) and 38% (22.5, 55.2) (Table). Incidence of Grade ≥3 adverse events was 76% and 65%, noted June 9, 2023.

6

HALO

Rheumatoid arthritis (RA)

Approved

10/14/13

Approved Oct 14, 2013.

6

ZLDPF

Pediatric Congenital hyperinsulinism (CHI)

Phase 3

9/19/22

Phase 3 trial additional data showed that treatment significantly reduced the requirement for intravenous (IV) glucose to maintain glycemia in neonates and infants with CHI and reduced glucose requirements to levels that potentially allow for discontinuation of IV glucose support, noted September 19, 2022.

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NVO

Prostate cancer (CRPC)

Phase 1

10/7/21

Phase 1 initial preliminary results reported include data as of Sept. 1, 2021, from eight men enrolled in the trial. The dose was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. (3/8) patients remain on study; (5/8) patients left the study (4 due to disease progression and 1 withdrawal of consent).

5

ABBV

Rheumatoid arthritis

Approved

8/16/19

FDA Approval announced August 16, 2019.

6

PRTA

AL Amyloidosis

Phase 3

12/12/22

Phase 3 VITAL data reported a significant improvement in time to all-cause mortality, noted December 12, 2022.

6

PFE

ALK-positive Anaplastic Large Cell Lymphoma

Approved

1/14/21

FDA approval announced January 14, 2021.

6

CARA

Stage 3-4 Chronic Kidney Disease-Associated Pruritus

Phase 2

12/3/19

Phase 2 data met primary endpoint but secondary missed - December 3, 2019.

5

ACHV

Smoking cessation

Phase 3

3/3/23

Additionnal Phase 3 data presented at the Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting confirmed that successful abstinence was observed in subgroups of smokers who received cytisinicline. Subjects who received either 6 or 12-weeks of cytisinicline treatment experienced consistently higher rates of abstinence, noted March 3, 2023.

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BLRX

First-line pancreatic ductal adenocarcinoma (PDAC)

Phase 2

9/28/23

Phase 2 trial data presented at the AACR Special pancreatic cancer conference reported that 6 patients (55%) experienced a partial response (PR) of which 4 (36%) were confirmed PRs with one patient experiencing resolution of the hepatic (liver) metastatic lesion, noted September 28, 2023.

6

SAGE

Major Depressive Disorder (MDD)

Phase 3

8/7/23

Phase 3 additional data reported that there were no new safety signals identified, noted August 7, 2023.

5

ANAB

Endometrial Cancer

Approved

7/31/23

FDA approved on July 31, 2023. Ahead of PDUFA on Sept. 23, 2023.

6

ELTX

Acute lung injury in patients with COVID-19

Phase 3

6/29/21

Phase 2 trial did not meet primary or secondary efficacy endpoints - June 29, 2021.

4

ACHV

Smoking cessation of cigarettes

Phase 3

5/23/23

Phase 3 topline results reported that subjects that received treatment had 2.85 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p=0.0008). The smoking cessation rate during weeks 3 through 6 was 14.8% for cytisinicline compared to 6% for placebo, noted May 23, 2023.

6

INBX

Alpha-1 Antitrypsin Deficiency (AATD)

Phase 1

11/2/22

Phase 1 data presented at ACoP noted that treatment demonstrated the potential to achieve and maintain normal levels of functional AAT. The 120 mg/kg Q4W regimen is predicted to achieve average trough values >21.1 µM, noted November 2, 2022.

6

MRK

Complicated urinary tract infections (cUTI) and Complicated intra-abdominal infections (cIAI)

Approved

7/17/19

FDA Approval announced July 17, 2019.

6

RHHBY

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3

12/12/22

Phase 3 results reported that trial met primary endpoints, noted December 12, 2022.

5

NBIX

Parkinson's disease

Approved

4/27/20

FDA Approval announced April 27, 2020.

6

VERU

COVID-19 / Severe Acute Respiratory Syndrome, SARS-CoV-2, Influenza A and B, Respiratory Syncytial Virus (RSV) and other viruses

Phase 3

9/26/23

Phase 3 new study authorized by FDA, with an FDA meeting in September 2023 resulted in agreement with FDA on New Phase 3 Clinical Trial for Sabizabulin for Broader Indication, noted September 26, 2023.

6

TAK

Post-transplant Hodgkin lymphoma (HL) cancer

Approved

8/17/15

Approved August 17, 2015 under priority review.

6

PFE

BRAF mutant melanoma cancer

Approved

6/27/18

Approval announced June 27, 2018.

6

IMCR

Solid tumors

Phase 1

12/8/22

Phase 1 initial data reported that one patient had a durable Partial Response (PR), with a duration of 12.7 months, one patient who had a Stable Disease (SD) converted to an unconfirmed PR after the poster data cutoff date and is still ongoing, and 5 had SD, noted December 8, 2022.

5

HRMY

Fragile X syndrome

Phase 2/3

5/13/22

Phase 2/3 long-term data reported that sustained improvement in ABC-CFXS Social Avoidance from baseline, noted May 13, 2022.

6

ELOX

Cystic fibrosis

Phase 2

11/17/21

Phase 2 monotherapy dosing data at 1.5mg/kg/day demonstrated a statistically significant 5.4mmol/L mean sweat chloride reduction, an established surrogate for restoration of CFTR biological activity, noted November 17, 2021.

6

SYRS

Pancreatic Cancer

Phase 1

11/14/22

Phase 1 safety data reported that treatment was generally safe, noted November 14, 2022.

6

ENDP

Men diagnosed with hypogonadism

Approved

3/6/14

Approved March 6, 2014.

6

SCPH

Chronic heart failure

Phase 2

7/12/22

Phase 2 trial data reported a 37% reduction in the risk of a heart failure hospitalization relative to patients randomized to "treatment as usual" at day 30, noted July 12, 2022.

6

REGN

Various cancers

Phase 1

11/4/23

Phase 1 safety data reported that a total of 11 adverse events (including one serious adverse event not considered related to SNS-101) was reported in five patients, with no dose-limiting toxicities observed, noted November 4, 2023.

6

BLPH

COVID-19

Phase 3

11/23/20

Phase 3 trial placed on clinical hold - November 23, 2020.

4

VIR

HIV T cell vaccine

Phase 1

5/8/23

Phase 1 initial data reported that no safety signals and no vector shedding or viremia were reported, noted June 28, 2023.

5

GSK

Chronic rhinosinusitis with nasal polyps

Approved

7/29/21

FDA approval announced July 29, 2021.

6

IOVA

Cervical cancer

Phase 2

5/31/19

Data released May 31, 2019 noted ORR 44%; CR 11%.

5

CORT

Pancreatic cancer

Phase 3

6/22/21

Phase 3 initial data released June 22, 2021. 2/31 (6%) partial responses. Enrollment to be stopped. Level of benefit does not justify further study.

4

MRK

Non-small cell lung cancer (NSCLC)

Approved

4/11/19

FDA Approval announced April 11, 2019.

6

IMGO

Myelofibrosis

Phase 2

12/12/22

Phase 2 data reported that 65% (17/26) of patients showed a decrease in Total Symptom Score (TSS), 19% (5/26) showed a ≥ 50% decrease in TSS, 66% (33/50) showed spleen volume reductions from baseline, and 28% (14/50) showed a ≥ 20% spleen volume reduction, noted December 12, 2022.

6

SONN

Healthy volunteers

Phase 1

11/2/23

Phase 1 interim analysis presented at CTAD reported that in single doses up to 300 ng/kg and in patients with advanced cancer in repeated doses up to at least 540 ng/kg. Incorporation of a desensitizing first dose at 150-300 ng/kg and dosing every 3 weeks has been implemented to capitalize on the potential benefits of tachyphylaxis with rhIL-12, noted November 2, 2023.

6

RLMD

Major depressive disorder (MDD)

Phase 3

12/7/22

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

6

BMRN

Phenylketonuria (PKU)

Approved

5/24/18

Approval announced May 24, 2018.

6