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
IPHA	Hematological malignancies	Phase 1/2	10/16/23	Phase 1/2 data from ESMO abstract reported that 3/8 patients achieved complete remission (CR), noted October 16, 2023.	6
MRK	Metastatic solid tumors	Phase 2	10/23/23	Additional Phase 2 data presented at ESMO reported that 10 out of 18 evaluable NSCLC patients (56%) had achieved a partial response as their best overall response and 3 out of 6 evaluable SCCHN patients had achieved partial response as their best overall response, noted October 23, 2023.	6
CYTO	COVID-19	Phase 1	1/24/23	Phase 1 topline data reported that trial did not meet primary endpoint, noted January 24, 2023.	6
PSTV	Leptomeningeal metastases (LM)	Phase 1/2	11/3/23	FDA granted Orphan Drug Designation (ODD), noted November 3, 2023.	5
PFE	Early stage breast cancer	Phase 3	10/9/20	Phase 3 trial did not meet primary endpoint - October 9, 2020.	4
SYBX	Enteric Hyperoxaluria	Phase 1b	12/15/22	Phase 1b study results and proof of concept data reported a 38% reduction in urinary oxalate compared to placebo, noted December 15, 2022.	6
BMY	Stroke prevention (SSP)	Phase 2	8/28/22	Phase 2 trial results reported that a a dose response was not observed and a relative risk reduction of approximately 30% in symptomatic ischemic stroke, noted August 28, 2022.	6
MRNS	PCDH19-related epilepsy	Phase 2	3/9/21	Phase 2 trial showed a median 61.5% reduction in seizure frequency for ganaxolone compared to 24.0% for placebo (p=0.17).	6
JANX	Metastatic castration-resistant prostate cancer (mCPRC)	Phase 1	7/17/23	Phase 1 interim clinical data showed tumor-activated T cell engagement in patients with prostate cancer representing the first-in-human data for the TRACTr platform and providing confidence for continuing clinical development of JANX007 and advancing future programs from the platform, noted July 17, 2023.	6
SUPN	Epilepsy	Approved	10/22/12	Approved October 22, 2012.	6
ALZN	Dementia related to Alzheimer's	Phase 2a	6/22/23	Phase 2a topline results identified a safe & appropriate dose to explore the potential to distribute more lithium to the brain but at lower systemic exposure, resulting in an improved safety profile compared to currently marketed lithium, noted June 22, 2023	6
GBT	Sickle cell disease (SCD)	Phase 2/3	11/2/23	Phase 2/3 data from ASH abstract reported that safety data showed GBT021601 was well tolerated. For 27 patients with VOC at baseline, the baseline annualized VOC rate was 2.30, noted November 2, 2023.	6
NVAX	Respiratory Syncytial Virus (RSV) in healthy pregnant women - protect infants via maternal immunization	Phase 3	2/28/19	Phase 3 top-line data released March 28, 2019 - primary endpoint not met.	4
PFE	Amyotrophic lateral sclerosis (ALS)	CRL	7/19/19	CRL issued July 19, 2019.	5
EWTX	dystrophinopathies including Duchenne Muscular Dystrophy in children and adolescent boys	Phase 2	10/26/23	Phase 2 initiated, noted October 26, 2023.	5
AUTL	T-cell lymphoma	Phase 1	6/16/23	Additional data presented at the ICML showed that all 4 patients at the highest dose in the study achieved a response and 2/4 remained in a complete metabolic response beyond 12 months, noted June 16, 2024.	6
NVTA	Early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE)	Phase 3	10/2/23	Phase 3 results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients, noted October 2, 2023.	6
AMGN	Cardiovascular disease	Approved	9/24/21	Approved for pediatric patients 10 years and older September 24, 2021.	6
RHHBY	Neoadjuvant triple negative breast cancer	Phase 3	6/18/20	Phase 3 trial met primary endpoint - June 18, 2020.	6
PFE	Respiratory syncytial virus (RSV) vaccine	Approved	6/1/23	Approved June 1, 2023.	6
ASMB	Hepatitis B virus (HBV)	Phase 2	11/1/22	Phase 2 preliminary data from 65 patients randomized to receive AB-729+VBR+NA (n=32), AB-729+NA (n=17) or VBR+NA (n=16) for 48 weeks showed that adding VBR to AB-729+NA does not result in greater on-treatment improvements in markers of HBV infection as compared to AB-729+NA alone, noted November 1, 2022.	6
NVS	Asthma	Phase 3	12/16/19	Phase 3 trial did not meet primary endpoint - December 16, 2019.	4
TCRT	Solid tumors	Phase 1/2	8/14/23	Program wind down due to prioritization. Phase 1/2 interim data reported a 83% disease control rate, noted August 14, 2023.	5
ARAV	Growth hormone deficiency, or GHD - pediatric	Phase 3	9/21/17	Phase 3 top-line data released September 21, 2017 - primary endpoint not met.	4
GILD	Refractory Large B-cell Lymphoma (R-LBCL)	Phase 2	6/6/22	Phase 2 data presented at ASCO reported a CR rate of 65% and ORR of 88%. With a median follow-up of 17 mo, 65% of the patients had ongoing response, with 57% ongoing in CR, noted June 6, 2022.	6
GLTO	Idiopathic pulmonary fibrosis (IPF)	Phase 2b	8/15/23	Phase 2b trial did not meet its primary endpoint, noted August 15, 2023.	6
MREO	COVID-19	Phase 1/2	12/22/21	Phase 1b/2 data reported no safety signals were observed and 62.5% of (5/8) patients had a 2-point decrease in the WHO Disease Severity score by Day 5, compared to 28.5% (2/7) patients in the placebo arm, noted December 22, 2021.	6
BMY	Myelofibrosis	Phase 1/2	12/12/22	Phase 1/2 results presented at ASH reported that most TRAEs were grade 1/2 in severity, while grade 3 TRAEs were successfully managed with dose delays or modifications without leading to treatment discontinuation, noted December 12, 2022.	6
INAB	Allogeneic hematopoietic stem cell transplantation (HSCT)	Phase 1	11/2/23	Phase 1 data from ASH abstract reported that treatment emergent AE's included transient WBC and ANC decreased (100% each), platelet count decreased and anemia (88.9% each) and maculopapular rash, hypomagnesemia and blood creatine increased (55.6% each), noted November 2, 2023.	6
FDMT	Wet Age-Related Macular Degeneration (wet AMD)	Phase 2	9/7/23	Phase 2 enrollment commenced, noted September 7, 2023.	5
BHVN	Amyotrophic lateral sclerosis (ALS)	Phase 3	9/29/22	Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.	6
RHHBY	1L Extensive-Stage Small Cell Lung Cancer	Phase 3	6/7/22	Phase 3 results noted that the combination did not provide benefit in patients with untreated ES-SCLC, noted June 7, 2022.	6
DCPH	Solid tumors	Phase 1/2	11/30/21	Program discontinued, noted November 30, 2021.	4
LIPO	Hemorrhagic cystitis	Phase 2a	4/30/23	Phase 2a results presented at AUA reported that treatment was tolerated without report of product related serious adverse events, noted April 30, 2023.	6
BGNE	Ovarian Cancers or Triple-Negative Breast Cancer	Phase 1/2	9/17/20	Phase 1/2 data to be presented at ESMO September 2020. ORR 64.6% and 31.6% in cohorts 1 and 2, respectively.	5
PHGE	P. aeruginosa in patients with Cystic Fibrosis (CF)	Phase 1/2	9/10/23	Phase 1b/2 Part 1 data presented at ERS reported that treatment was well-tolerated with no adverse events related to study drug, noted September 10, 2023.	6
JNJ	Type 2 diabetes	Approved	10/30/18	FDA Approval announced October 30, 2018.	6
ORMP	Non-alcoholic steatohepatitis (NASH)	Phase 2	6/15/20	Phase 2 interim data presented at ADA 2020 Scientific Sessions - mean 6.9% reduction in liver fat content.	6
LLY	Atopic Dermatitis	CRL	11/2/23	CRL received in the US, noted November 2, 2023.	5
ALLO	Clear cell renal cell carcinoma (ccRCC)	Phase 1	4/17/23	Phase 1 presentation at AACR reported early anti-tumor activity with deepening responses over time, noted April 17, 2023.	6
BGNE	Stage II-IIIA non-small cell lung cancer (NSCLC)	Phase 3	10/23/23	Phase 3 data presented at ESMO reported that MPR & pCR rates were significantly improved with TIS + CT vs CT, noted October 23, 2023.	6
VRCA	Common warts	Phase 2	6/26/19	Phase 2 data noted 51% of patients noted complete clearance of warts - June 26, 2019.	5
GRTS	COVID-19 booster (60+)	Phase 1	10/11/23	Additional Phase 1 data reported at IDweek noted that durable binding and high neutralizing antibodies were induced and sustained for up to at least 12 months, noted October 11, 2023.	5
RVNC	Upper limb spasticity	Phase 2	2/22/21	Phase 2 top-line data released February 22, 2021. One of two co-primary endpoints met (one missed due to reduced enrollment).	6
CCXI	C3 glomerulopathy (C3G)	Phase 2	12/21/20	Phase 2 trial did not meet primary endpoint - December 21, 2020.	4
AXSM	Treatment resistant depression	Phase 2	8/9/21	Phase 2 trial met primary and key secondary endpoints - August 9, 2021.	6
YS	Rabies	Phase 3	9/26/23	Phase 3 trial initiated, noted September 26, 2023.	5
CTIC	COVID-19	Phase 3	10/1/21	Phase 3 trial did not demonstrate a clinical benefit, trial will no longer continue.	4
CGTX	Early Alzheimer's disease	Phase 2	10/27/23	Phase 2 data points to a role for the sigma-2 (σ-2) receptor in regulating key "housekeeping" processes such as autophagy that are impaired in Alzheimer's and other neurodegenerative diseases, noted October 27, 2023.	5
INO	COVID-19 vaccine	Phase 2	5/10/21	Phase 2 data released May 10, 2021 - generally safe and well-tolerated.	6
MRUS	Solid tumors - NRG1	Phase 1/2	10/23/23	Phase 1/2 data update presented at ESMO showed a 37% ORR and 14.9 months median DOR in 78 evaluable NRG1+ NSCLC patients and 42% ORR and 9.1 months median DOR in 33 evaluable NRG1+ PDAC patients. Sufficient clinical data expected in 1H 2024 to support potential BLA submissions, noted October 23, 2023.	6
ROIV	Atopic dermatitis (AD)	Phase 3	5/16/23	Phase 3 data reported that the trial met its primary endpoint, noted May 16, 2023.	5
MRK	HIV-1	Phase 3	10/25/21	Phase 3 data met safety and efficacy endpoints, noted October 25, 2021.	6
MRK	First and Second line locally advanced or metastatic urothelial cancer - bladder cancer	Approved	5/18/17	Approved May 18, 2017.	6
BLCM	HER2+ Solid Tumors	Phase 1/2	12/6/21	Phase 1/2 initial data reported no dose-limiting toxicities with other Grade 3+ treatment-emergent adverse events being neutropenia, leukopenia, and anemia, noted December 6, 2021.	6
MRK	Solid tumors - TMB-H ≥10 mutations/megabase	Approved	6/17/20	FDA Approval June 17, 2020.	6
BPTH	Solid tumors	Phase 1	7/17/23	Phase 1/1b first cohort completed, noted July 17, 2023	5
INKT	Acute respiratory distress (ARDS) secondary to COVID-19	Phase 1	5/21/23	Phase 1 data update reported a 75% survival rate in patients with severe respiratory distress treated with agenT-797, noted May 21, 2023.	6
ORTX	Mucopolysaccharidosis type I (MPS-I)	Phase 1	10/26/23	PoC data presented at ESGCT resulted in improvement 62.5% of patients or stabilization 37.5% of patients of corneal clouding at the time of last follow-up ranging from 3.14-4.58 years compared to baseline measured prior to administration with OTL-203, noted October 26, 2023.	6
ABBV	Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).	Approved	4/21/20	FDA Approval announced April 21, 2020.	6
ALZN	Mild to Moderate Dementia of the Alzheimer's Type	Phase 1/2	3/28/22	Phase 1 results reported that treatment was safe and well-tolerated and salicylate exposures were within safe limits, noted March 28, 2022.	6
COGT	Gastrointestinal Stromal Tumors (GIST)	Phase 3	6/5/23	Phase 3 efficacy data presented at ASCO reported a 55% Disease Control Rate (DCR) in heavily pre-treated GIST patients, including 100% DCR and 17% overall response rate (ORR) in efficacy evaluable 2nd-line GIST patients, noted June 5, 2023.	6
AZN	Chronic rhinosinusitis with nasal polyps (CRSwNP)	CRL	3/14/22	CRL announced March 14, 2022.	5
AXSM	Major Depressive Disorder	Approved	8/19/22	FDA Approved on August 19, 2022.	6
GSK	Prevention of chemotherapy induced nausea and vomiting, or CINV	Approved	9/2/16	Approved September 2, 2015.	6
PBYI	Rb-deficient head and neck squamous cell cancer	Phase 1/2	10/16/23	Phase 1/2 data reported that the median PFS was 1.4 months, and the median overall survival (OS) was 13.5 months, noted October 16, 2023.	5
BTAI	Neuroendocrine Prostate Cancer (NEPC)	Phase 2a	1/11/23	Phase 2a data reported that combination with pembrolizumab has demonstrated an encouraging response rate, noted January 11, 2023.	6
PSTV	Glioma	Phase 1/2	6/29/23	Phase 1/2 data presented at the Society of Nuclear Medicine & Molecular Imaging's (SNMMI) Annual Meeting demonstrated efficacy signals in a prognostically unfavorable patient population. The median overall survival (OS) in all 21 patients was 11 months or a 38% increase in OS versus a median OS of approximately 8 months for standard of care in rGBM. Median OS in patients receiving >100 Gy of absorbed radiation dose was 76 weeks versus 22 weeks for those receiving <100 Gy (p=0.0002), noted June 29, 2023.	6
RGNX	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
MRK	Cytomegalovirus (CMV) Infection	Approved	11/9/17	Approval announced November 9, 2017.	6
ACOR	Post stroke deficits	Phase 3	11/21/16	Phase 3 data released November 21, 2016 - failed to show efficacy, development to be discontinued.	4
BGNE	Waldenström’s macroglobulinemia	Approved	9/1/21	FDA Approval announced September 1, 2021.	6
JNJ	HIV in Women	Phase 2b	8/31/21	Phase 2b data did not meet endpoints, study will not continue - August 31, 2021.	4
ASMB	Hepatitis B virus (HBV)	Phase 2	11/12/21	Phase 2 data reported that treatment led to a deeper level of viral suppression as measured by pgRNA and HBV DNA levels, noted November 12, 2021.	6
SNY	Dengue	Approved	5/1/19	FDA approval announced May 1, 2019.	6
RHHBY	First-line BRAF wild-type metastatic or unresectable locally advanced melanoma	Phase 3	6/20/19	Phase 3 data did not meet primary endpoint - June 20, 2019.	4
RLYB	Fetal and neonatal alloimmune thrombocytopenia (FNAIT)	Phase 1/2	12/1/21	Phase 1/2 data reported that treatment accelerated the elimination of HPA-1ab-positive platelets through 7 days following administration compared with placebo, noted December 1, 2021.	6
AMRX	Parkinson's disease	CRL	7/3/23	CRL issued by the FDA on July 3, 2023.	5
INCY	Alopecia Areata	Approved	6/13/22	Approval announced June 13, 2022.	6
ARCT	COVID-19 variant vaccine	Phase 1/2	8/18/22	Additional Phase 1/2 data reported broad neutralizing antibody response against Omicron variants of concern, including BA.5, lasting for up to at least six months after administration of low-dose (5 mcg) ARCT-154 booster, noted August 18, 2022.	5
LNTH	Pheochromocytoma	Approved	7/30/18	FDA approval announced July 30, 2018.	6
MYOV	Advanced prostate cancer	Approved	12/18/20	FDA approval announced Decmber 18, 2020.	6
JNJ	Plaque psoriasis	Phase 3	9/18/22	Phase 3b data reported that patients treated with guselkumab ≤2 years after disease onset (versus >2 years) are more likely to achieve super-respondera status, noted September 18, 2022.	6
ADPT	Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL)	Approved	5/19/23	Approved May 19, 2023.	6
PBYI	Glioblastoma (GBM)	Phase 2	11/20/21	Phase 2 results reported that in the intent-to-treat population, there was no significant improvement of PFS and OS. Median PFS with neratinib was 6.0 months versus 4.7 months and median OS was 13.8 months 14.7 months, noted November 20, 2021.	4
SLS	Malignant pleural mesothelioma (MPM)	Phase 1	6/28/23	Phase 1 topline data reported that the median overall survival for patients who entered the study as Stage IV patients was 62.3 weeks, noted June 28, 2023.	5
BDTX	Glioblastoma multiforme (GBM) and NSCLC	Phase 1	10/14/23	Phase 1 clinical data presented at AACR-NCI-EORTC meeting reported that five of the 13 patients with either intrinsic driver, acquired resistance or complex mutations had a confirmed partial response (PR), noted October 14, 2023.	6
VERA	Kidney transplant	Phase 2	6/4/22	Phase 2 data results showed that the antiviral effect was higher in the dosed group than the placebo group. Further, the treatment was well tolerated, with a comparable frequency of adverse events and serious adverse events between groups	6
CRNX	Acromegaly	Phase 2	6/15/23	Phase 2 2-year data presented at ENDO 2023 showed that treatment was well-tolerated, associated with stable IGF-I and symptom control relative to that achieved by iSRLs, and was preferred over injected therapy, noted June 15, 2023.	6
MRK	Resectable stage II, IIIA or IIIB non-small cell lung cancer (NSCLC)	Phase 3	10/20/23	Phase 3 data presented at ESMO 23 demonstrated statistically significant improvement in overall survival versus neoadjuvant placebo plus chemotherapy, noted October 20, 2023.	6
ALGS	HBeAg-positive chronic hepatitis B (CHB)	Phase 1b	6/21/23	Additional Phase 1b data presented at EASL demonstrated multi-log reductions in HBsAg, DNA, and RNA levels in subjects dosed for up to 36 weeks, noted June 21, 2023.	6
EYPT	Non-infectious uveitis	Approved	10/15/18	FDA approval announced October 15, 2018.	6
GSK	Meningococcal disease	Approved	10/17/22	Approved October 17, 2022.	6
LIAN	Cholangiocarcinoma	Approved	6/1/21	FDA approval announced June 1, 2021.	6
SGEN	Non-muscle invasive bladder cancer (NMIBC)	Phase 1	6/3/23	Phase 1 interim data shared at ASCO reported that of 4 pts receiving 125mg of intravesical EV, 3 achieved CR and continue in response, noted June 3, 2023.	6
NVO	Type 2 diabetes	Phase 3	6/3/22	Phase 3a trial met primary endpoint, noted June 3, 2022.	5
RHHBY	Prurigo nodularis	Phase 2a	4/22/20	Phase 2a trial met primary endpoint - April 22, 2020.	6
ASND	Solid tumors	Phase 1/2	10/26/23	Phase 1/2 updated data presented at ESMO resulted in clinical responses demonstrated as monotherapy or in combination with checkpoint inhibitor, noted October 26, 2023.	6
AMGN	Herceptin biosimilar	Approved	6/13/19	FDA approval announced June 13, 2019.	6
HCM	Gastric Cancer	Phase 2	11/14/22	Phase 3 trial met primary endpoint regarding progression-free survival. The trial did not meet the the overall survival primary endpoint, noted November 14, 2022.	6

IPHA

Hematological malignancies

Phase 1/2

10/16/23

Phase 1/2 data from ESMO abstract reported that 3/8 patients achieved complete remission (CR), noted October 16, 2023.

6

MRK

Metastatic solid tumors

Phase 2

10/23/23

Additional Phase 2 data presented at ESMO reported that 10 out of 18 evaluable NSCLC patients (56%) had achieved a partial response as their best overall response and 3 out of 6 evaluable SCCHN patients had achieved partial response as their best overall response, noted October 23, 2023.

6

CYTO

COVID-19

Phase 1

1/24/23

Phase 1 topline data reported that trial did not meet primary endpoint, noted January 24, 2023.

6

PSTV

Leptomeningeal metastases (LM)

Phase 1/2

11/3/23

FDA granted Orphan Drug Designation (ODD), noted November 3, 2023.

5

PFE

Early stage breast cancer

Phase 3

10/9/20

Phase 3 trial did not meet primary endpoint - October 9, 2020.

4

SYBX

Enteric Hyperoxaluria

Phase 1b

12/15/22

Phase 1b study results and proof of concept data reported a 38% reduction in urinary oxalate compared to placebo, noted December 15, 2022.

6

BMY

Stroke prevention (SSP)

Phase 2

8/28/22

Phase 2 trial results reported that a a dose response was not observed and a relative risk reduction of approximately 30% in symptomatic ischemic stroke, noted August 28, 2022.

6

MRNS

PCDH19-related epilepsy

Phase 2

3/9/21

Phase 2 trial showed a median 61.5% reduction in seizure frequency for ganaxolone compared to 24.0% for placebo (p=0.17).

6

JANX

Metastatic castration-resistant prostate cancer (mCPRC)

Phase 1

7/17/23

Phase 1 interim clinical data showed tumor-activated T cell engagement in patients with prostate cancer representing the first-in-human data for the TRACTr platform and providing confidence for continuing clinical development of JANX007 and advancing future programs from the platform, noted July 17, 2023.

6

SUPN

Epilepsy

Approved

10/22/12

Approved October 22, 2012.

6

ALZN

Dementia related to Alzheimer's

Phase 2a

6/22/23

Phase 2a topline results identified a safe & appropriate dose to explore the potential to distribute more lithium to the brain but at lower systemic exposure, resulting in an improved safety profile compared to currently marketed lithium, noted June 22, 2023

6

GBT

Sickle cell disease (SCD)

Phase 2/3

11/2/23

Phase 2/3 data from ASH abstract reported that safety data showed GBT021601 was well tolerated. For 27 patients with VOC at baseline, the baseline annualized VOC rate was 2.30, noted November 2, 2023.

6

NVAX

Respiratory Syncytial Virus (RSV) in healthy pregnant women - protect infants via maternal immunization

Phase 3

2/28/19

Phase 3 top-line data released March 28, 2019 - primary endpoint not met.

4

PFE

Amyotrophic lateral sclerosis (ALS)

CRL

7/19/19

CRL issued July 19, 2019.

5

EWTX

dystrophinopathies including Duchenne Muscular Dystrophy in children and adolescent boys

Phase 2

10/26/23

Phase 2 initiated, noted October 26, 2023.

5

AUTL

T-cell lymphoma

Phase 1

6/16/23

Additional data presented at the ICML showed that all 4 patients at the highest dose in the study achieved a response and 2/4 remained in a complete metabolic response beyond 12 months, noted June 16, 2024.

6

NVTA

Early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE)

Phase 3

10/2/23

Phase 3 results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients, noted October 2, 2023.

6

AMGN

Cardiovascular disease

Approved

9/24/21

Approved for pediatric patients 10 years and older September 24, 2021.

6

RHHBY

Neoadjuvant triple negative breast cancer

Phase 3

6/18/20

Phase 3 trial met primary endpoint - June 18, 2020.

6

PFE

Respiratory syncytial virus (RSV) vaccine

Approved

6/1/23

Approved June 1, 2023.

6

ASMB

Hepatitis B virus (HBV)

Phase 2

11/1/22

Phase 2 preliminary data from 65 patients randomized to receive AB-729+VBR+NA (n=32), AB-729+NA (n=17) or VBR+NA (n=16) for 48 weeks showed that adding VBR to AB-729+NA does not result in greater on-treatment improvements in markers of HBV infection as compared to AB-729+NA alone, noted November 1, 2022.

6

NVS

Asthma

Phase 3

12/16/19

Phase 3 trial did not meet primary endpoint - December 16, 2019.

4

TCRT

Solid tumors

Phase 1/2

8/14/23

Program wind down due to prioritization. Phase 1/2 interim data reported a 83% disease control rate, noted August 14, 2023.

5

ARAV

Growth hormone deficiency, or GHD - pediatric

Phase 3

9/21/17

Phase 3 top-line data released September 21, 2017 - primary endpoint not met.

4

GILD

Refractory Large B-cell Lymphoma (R-LBCL)

Phase 2

6/6/22

Phase 2 data presented at ASCO reported a CR rate of 65% and ORR of 88%. With a median follow-up of 17 mo, 65% of the patients had ongoing response, with 57% ongoing in CR, noted June 6, 2022.

6

GLTO

Idiopathic pulmonary fibrosis (IPF)

Phase 2b

8/15/23

Phase 2b trial did not meet its primary endpoint, noted August 15, 2023.

6

MREO

COVID-19

Phase 1/2

12/22/21

Phase 1b/2 data reported no safety signals were observed and 62.5% of (5/8) patients had a 2-point decrease in the WHO Disease Severity score by Day 5, compared to 28.5% (2/7) patients in the placebo arm, noted December 22, 2021.

6

BMY

Myelofibrosis

Phase 1/2

12/12/22

Phase 1/2 results presented at ASH reported that most TRAEs were grade 1/2 in severity, while grade 3 TRAEs were successfully managed with dose delays or modifications without leading to treatment discontinuation, noted December 12, 2022.

6

INAB

Allogeneic hematopoietic stem cell transplantation (HSCT)

Phase 1

11/2/23

Phase 1 data from ASH abstract reported that treatment emergent AE's included transient WBC and ANC decreased (100% each), platelet count decreased and anemia (88.9% each) and maculopapular rash, hypomagnesemia and blood creatine increased (55.6% each), noted November 2, 2023.

6

FDMT

Wet Age-Related Macular Degeneration (wet AMD)

Phase 2

9/7/23

Phase 2 enrollment commenced, noted September 7, 2023.

5

BHVN

Amyotrophic lateral sclerosis (ALS)

Phase 3

9/29/22

Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.

6

RHHBY

1L Extensive-Stage Small Cell Lung Cancer

Phase 3

6/7/22

Phase 3 results noted that the combination did not provide benefit in patients with untreated ES-SCLC, noted June 7, 2022.

6

DCPH

Solid tumors

Phase 1/2

11/30/21

Program discontinued, noted November 30, 2021.

4

LIPO

Hemorrhagic cystitis

Phase 2a

4/30/23

Phase 2a results presented at AUA reported that treatment was tolerated without report of product related serious adverse events, noted April 30, 2023.

6

BGNE

Ovarian Cancers or Triple-Negative Breast Cancer

Phase 1/2

9/17/20

Phase 1/2 data to be presented at ESMO September 2020. ORR 64.6% and 31.6% in cohorts 1 and 2, respectively.

5

PHGE

P. aeruginosa in patients with Cystic Fibrosis (CF)

Phase 1/2

9/10/23

Phase 1b/2 Part 1 data presented at ERS reported that treatment was well-tolerated with no adverse events related to study drug, noted September 10, 2023.

6

JNJ

Type 2 diabetes

Approved

10/30/18

FDA Approval announced October 30, 2018.

6

ORMP

Non-alcoholic steatohepatitis (NASH)

Phase 2

6/15/20

Phase 2 interim data presented at ADA 2020 Scientific Sessions - mean 6.9% reduction in liver fat content.

6

LLY

Atopic Dermatitis

CRL

11/2/23

CRL received in the US, noted November 2, 2023.

5

ALLO

Clear cell renal cell carcinoma (ccRCC)

Phase 1

4/17/23

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

6

BGNE

Stage II-IIIA non-small cell lung cancer (NSCLC)

Phase 3

10/23/23

Phase 3 data presented at ESMO reported that MPR & pCR rates were significantly improved with TIS + CT vs CT, noted October 23, 2023.

6

VRCA

Common warts

Phase 2

6/26/19

Phase 2 data noted 51% of patients noted complete clearance of warts - June 26, 2019.

5

GRTS

COVID-19 booster (60+)

Phase 1

10/11/23

Additional Phase 1 data reported at IDweek noted that durable binding and high neutralizing antibodies were induced and sustained for up to at least 12 months, noted October 11, 2023.

5

RVNC

Upper limb spasticity

Phase 2

2/22/21

Phase 2 top-line data released February 22, 2021. One of two co-primary endpoints met (one missed due to reduced enrollment).

6

CCXI

C3 glomerulopathy (C3G)

Phase 2

12/21/20

Phase 2 trial did not meet primary endpoint - December 21, 2020.

4

AXSM

Treatment resistant depression

Phase 2

8/9/21

Phase 2 trial met primary and key secondary endpoints - August 9, 2021.

6

YS

Rabies

Phase 3

9/26/23

Phase 3 trial initiated, noted September 26, 2023.

5

CTIC

COVID-19

Phase 3

10/1/21

Phase 3 trial did not demonstrate a clinical benefit, trial will no longer continue.

4

CGTX

Early Alzheimer's disease

Phase 2

10/27/23

Phase 2 data points to a role for the sigma-2 (σ-2) receptor in regulating key "housekeeping" processes such as autophagy that are impaired in Alzheimer's and other neurodegenerative diseases, noted October 27, 2023.

5

INO

COVID-19 vaccine

Phase 2

5/10/21

Phase 2 data released May 10, 2021 - generally safe and well-tolerated.

6

MRUS

Solid tumors - NRG1

Phase 1/2

10/23/23

Phase 1/2 data update presented at ESMO showed a 37% ORR and 14.9 months median DOR in 78 evaluable NRG1+ NSCLC patients and 42% ORR and 9.1 months median DOR in 33 evaluable NRG1+ PDAC patients. Sufficient clinical data expected in 1H 2024 to support potential BLA submissions, noted October 23, 2023.

6

ROIV

Atopic dermatitis (AD)

Phase 3

5/16/23

Phase 3 data reported that the trial met its primary endpoint, noted May 16, 2023.

5

MRK

HIV-1

Phase 3

10/25/21

Phase 3 data met safety and efficacy endpoints, noted October 25, 2021.

6

MRK

First and Second line locally advanced or metastatic urothelial cancer - bladder cancer

Approved

5/18/17

Approved May 18, 2017.

6

BLCM

HER2+ Solid Tumors

Phase 1/2

12/6/21

Phase 1/2 initial data reported no dose-limiting toxicities with other Grade 3+ treatment-emergent adverse events being neutropenia, leukopenia, and anemia, noted December 6, 2021.

6

MRK

Solid tumors - TMB-H ≥10 mutations/megabase

Approved

6/17/20

FDA Approval June 17, 2020.

6

BPTH

Solid tumors

Phase 1

7/17/23

Phase 1/1b first cohort completed, noted July 17, 2023

5

INKT

Acute respiratory distress (ARDS) secondary to COVID-19

Phase 1

5/21/23

Phase 1 data update reported a 75% survival rate in patients with severe respiratory distress treated with agenT-797, noted May 21, 2023.

6

ORTX

Mucopolysaccharidosis type I (MPS-I)

Phase 1

10/26/23

PoC data presented at ESGCT resulted in improvement 62.5% of patients or stabilization 37.5% of patients of corneal clouding at the time of last follow-up ranging from 3.14-4.58 years compared to baseline measured prior to administration with OTL-203, noted October 26, 2023.

6

ABBV

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

Approved

4/21/20

FDA Approval announced April 21, 2020.

6

ALZN

Mild to Moderate Dementia of the Alzheimer's Type

Phase 1/2

3/28/22

Phase 1 results reported that treatment was safe and well-tolerated and salicylate exposures were within safe limits, noted March 28, 2022.

6

COGT

Gastrointestinal Stromal Tumors (GIST)

Phase 3

6/5/23

Phase 3 efficacy data presented at ASCO reported a 55% Disease Control Rate (DCR) in heavily pre-treated GIST patients, including 100% DCR and 17% overall response rate (ORR) in efficacy evaluable 2nd-line GIST patients, noted June 5, 2023.

6

AZN

Chronic rhinosinusitis with nasal polyps (CRSwNP)

CRL

3/14/22

CRL announced March 14, 2022.

5

AXSM

Major Depressive Disorder

Approved

8/19/22

FDA Approved on August 19, 2022.

6

GSK

Prevention of chemotherapy induced nausea and vomiting, or CINV

Approved

9/2/16

Approved September 2, 2015.

6

PBYI

Rb-deficient head and neck squamous cell cancer

Phase 1/2

10/16/23

Phase 1/2 data reported that the median PFS was 1.4 months, and the median overall survival (OS) was 13.5 months, noted October 16, 2023.

5

BTAI

Neuroendocrine Prostate Cancer (NEPC)

Phase 2a

1/11/23

Phase 2a data reported that combination with pembrolizumab has demonstrated an encouraging response rate, noted January 11, 2023.

6

PSTV

Glioma

Phase 1/2

6/29/23

Phase 1/2 data presented at the Society of Nuclear Medicine & Molecular Imaging's (SNMMI) Annual Meeting demonstrated efficacy signals in a prognostically unfavorable patient population. The median overall survival (OS) in all 21 patients was 11 months or a 38% increase in OS versus a median OS of approximately 8 months for standard of care in rGBM. Median OS in patients receiving >100 Gy of absorbed radiation dose was 76 weeks versus 22 weeks for those receiving <100 Gy (p=0.0002), noted June 29, 2023.

6

RGNX

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

MRK

Cytomegalovirus (CMV) Infection

Approved

11/9/17

Approval announced November 9, 2017.

6

ACOR

Post stroke deficits

Phase 3

11/21/16

Phase 3 data released November 21, 2016 - failed to show efficacy, development to be discontinued.

4

BGNE

Waldenström’s macroglobulinemia

Approved

9/1/21

FDA Approval announced September 1, 2021.

6

JNJ

HIV in Women

Phase 2b

8/31/21

Phase 2b data did not meet endpoints, study will not continue - August 31, 2021.

4

ASMB

Hepatitis B virus (HBV)

Phase 2

11/12/21

Phase 2 data reported that treatment led to a deeper level of viral suppression as measured by pgRNA and HBV DNA levels, noted November 12, 2021.

6

SNY

Dengue

Approved

5/1/19

FDA approval announced May 1, 2019.

6

RHHBY

First-line BRAF wild-type metastatic or unresectable locally advanced melanoma

Phase 3

6/20/19

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

4

RLYB

Fetal and neonatal alloimmune thrombocytopenia (FNAIT)

Phase 1/2

12/1/21

Phase 1/2 data reported that treatment accelerated the elimination of HPA-1ab-positive platelets through 7 days following administration compared with placebo, noted December 1, 2021.

6

AMRX

Parkinson's disease

CRL

7/3/23

CRL issued by the FDA on July 3, 2023.

5

INCY

Alopecia Areata

Approved

6/13/22

Approval announced June 13, 2022.

6

ARCT

COVID-19 variant vaccine

Phase 1/2

8/18/22

Additional Phase 1/2 data reported broad neutralizing antibody response against Omicron variants of concern, including BA.5, lasting for up to at least six months after administration of low-dose (5 mcg) ARCT-154 booster, noted August 18, 2022.

5

LNTH

Pheochromocytoma

Approved

7/30/18

FDA approval announced July 30, 2018.

6

MYOV

Advanced prostate cancer

Approved

12/18/20

FDA approval announced Decmber 18, 2020.

6

JNJ

Plaque psoriasis

Phase 3

9/18/22

Phase 3b data reported that patients treated with guselkumab ≤2 years after disease onset (versus >2 years) are more likely to achieve super-respondera status, noted September 18, 2022.

6

ADPT

Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

Approved

5/19/23

Approved May 19, 2023.

6

PBYI

Glioblastoma (GBM)

Phase 2

11/20/21

Phase 2 results reported that in the intent-to-treat population, there was no significant improvement of PFS and OS. Median PFS with neratinib was 6.0 months versus 4.7 months and median OS was 13.8 months 14.7 months, noted November 20, 2021.

4

SLS

Malignant pleural mesothelioma (MPM)

Phase 1

6/28/23

Phase 1 topline data reported that the median overall survival for patients who entered the study as Stage IV patients was 62.3 weeks, noted June 28, 2023.

5

BDTX

Glioblastoma multiforme (GBM) and NSCLC

Phase 1

10/14/23

Phase 1 clinical data presented at AACR-NCI-EORTC meeting reported that five of the 13 patients with either intrinsic driver, acquired resistance or complex mutations had a confirmed partial response (PR), noted October 14, 2023.

6

VERA

Kidney transplant

Phase 2

6/4/22

Phase 2 data results showed that the antiviral effect was higher in the dosed group than the placebo group. Further, the treatment was well tolerated, with a comparable frequency of adverse events and serious adverse events between groups

6

CRNX

Acromegaly

Phase 2

6/15/23

Phase 2 2-year data presented at ENDO 2023 showed that treatment was well-tolerated, associated with stable IGF-I and symptom control relative to that achieved by iSRLs, and was preferred over injected therapy, noted June 15, 2023.

6

MRK

Resectable stage II, IIIA or IIIB non-small cell lung cancer (NSCLC)

Phase 3

10/20/23

Phase 3 data presented at ESMO 23 demonstrated statistically significant improvement in overall survival versus neoadjuvant placebo plus chemotherapy, noted October 20, 2023.

6

ALGS

HBeAg-positive chronic hepatitis B (CHB)

Phase 1b

6/21/23

Additional Phase 1b data presented at EASL demonstrated multi-log reductions in HBsAg, DNA, and RNA levels in subjects dosed for up to 36 weeks, noted June 21, 2023.

6

EYPT

Non-infectious uveitis

Approved

10/15/18

FDA approval announced October 15, 2018.

6

GSK

Meningococcal disease

Approved

10/17/22

Approved October 17, 2022.

6

LIAN

Cholangiocarcinoma

Approved

6/1/21

FDA approval announced June 1, 2021.

6

SGEN

Non-muscle invasive bladder cancer (NMIBC)

Phase 1

6/3/23

Phase 1 interim data shared at ASCO reported that of 4 pts receiving 125mg of intravesical EV, 3 achieved CR and continue in response, noted June 3, 2023.

6

NVO

Type 2 diabetes

Phase 3

6/3/22

Phase 3a trial met primary endpoint, noted June 3, 2022.

5

RHHBY

Prurigo nodularis

Phase 2a

4/22/20

Phase 2a trial met primary endpoint - April 22, 2020.

6

ASND

Solid tumors

Phase 1/2

10/26/23

Phase 1/2 updated data presented at ESMO resulted in clinical responses demonstrated as monotherapy or in combination with checkpoint inhibitor, noted October 26, 2023.

6

AMGN

Herceptin biosimilar

Approved

6/13/19

FDA approval announced June 13, 2019.

6

HCM

Gastric Cancer

Phase 2

11/14/22

Phase 3 trial met primary endpoint regarding progression-free survival. The trial did not meet the the overall survival primary endpoint, noted November 14, 2022.

6