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
PFE	Lyme disease vaccine for outdoor recreationists	Phase 3	2/17/23	Phase 3 study discontinued about 50% of patients enrolled following violations of Good Clinical Practice (GCP) at certain clinical trial sites run by a third-party clinical trial site operator, noted February 17, 2023.	4
BXRX	Acute pain following bunionectomy surgery	Approved	2/20/20	FDA Approval announced February 20, 2020.	6
KALV	Hereditary angioedema (HAE)	Phase 1	6/9/23	Phase 1 data presented at EAACI showed that pharmacokinetic and pharmacodynamic data support globalization of the phase 3 clinical trial and use in short-term prophylaxis, noted June 9, 2023.	6
SLRX	Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)	Phase 1/2	12/12/22	Phase 1/2 data presented at ASH reported an ORR of 50% among eight myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), noted December 12, 2022.	6
INCY	Solid Tumors	Phase 1/2	6/3/23	Phase 1/2 data from ASCO reported that the best overall response in part 1 was stable disease, and in part 2 was a confirmed and durable partial response in 1 pt, noted June 3, 2023.	5
GOVX	Covid-19	Phase 2	9/11/23	Phase 2 target enrollment met, noted September 11, 2023.	5
AZN	Hyperkalaemia	Approved	5/18/18	Approval announced May 18, 2018.	6
OPK	Mild-to-moderate COVID-19	Phase 2	12/23/21	Phase 2 top-line data reported that mean serum 25D levels increased with treatment and remained elevated for the duration of the trial, with 88% of subjects attaining the targeted level. Three symptoms related to respiratory function resolved more quickly when serum 25D was elevated at Days 7 and 14, with resolution of chest congestion occurring 3.4 days sooner, noted December 23, 2021.	6
CPRX	Congenital Myasthenic Syndromes (CMS)	Phase 3	10/30/19	Phase 3 trial did not meet primary endpoint - October 30, 2019.	4
VALN	Booster shot COVID-19 vaccine	Phase 1/2	1/19/22	Additional data reported that all 30 samples (100%) presented neutralizing antibodies against the ancestral virus and Delta variant, and 26 samples (87%) presented neutralizing antibodies against the Omicron variant, noted January 19, 2022.	6
MESO	End-Stage Heart Failure with LVADs	Phase 2b	11/11/18	Phase 2b trial did not meet primary endpoint - November 11, 2018.	4
RAIN	MDM2-amplified advanced solid tumors	Phase 2	5/30/23	Phase 2 enrollment suspended, noted May 30, 2023.	4
MRK	Metastatic castration-resistant prostate cancer (mCRPC)	Phase 3	8/3/22	Phase 3 trial results were not statistically significant, noted August 3, 2022.	5
IOBT	Non-muscle invasive bladder cancer (NMIBC)	Phase 1	7/28/23	Phase 1 dosing initiated, noted July 28, 2023.	5
BIIB	Lupus	Phase 2	12/3/19	Phase 2 data met primary endpoint - December 3, 2019.	6
IMAB	Growth hormone deficiency	Phase 3	8/17/23	Phase 3 trial met its primary endpoint, noted August 17, 2023.	5
PFE	HIV	Approved	12/21/21	Approved December 21, 2021.	6
PFE	Lyme disease	Phase 2	9/7/23	Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.	6
AGIO	Sickle cell disease	Phase 2	6/26/23	Phase 2 portion met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms, noted June 26, 2023	5
ENTX	Osteoporosis	Phase 2	10/14/23	Phase 2 data presented at ASBMR showed that an increase in plasma ionized calcium should result in decreased secretion and plasma concentrations of endogenous PTH(1-84), noted October 14, 2023	6
ZYME	3L+ HER2-positive, HR-positive breast cancer	Phase 2	12/9/22	Phase 2 data reported a confirmed objective response rate (cORR) of 33%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 9.6 months, noted December 9, 2022.	5
ALKS	Recurrent or metastatic head and neck squamous cell carcinoma	Phase 2	11/13/21	Phase 2 data showed that combination therapy was generally well tolerated (n=14), 8 had no prior response to dosing and 6 had previous best response of SD or PR. Mean (SD±) age was 62±12; AE were consistent with those observed with intravenous studies. Peripheral immune cell expansion profiles are comparable to that observed with the same regimen.	6
SRPT	Limb-girdle muscular dystrophy type 2E (LGMD2E)	Phase 1/2	10/11/21	Results from most recent study found that treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the NSAA compared to a matched natural history cohort 3 years after treatment- noted October 11, 2021.	6
GSK	Covid-19 vaccine, omicron variant	Phase 1	1/30/23	Phase 1 immunogenicity data reported that on day 29 at the 12µg dose level, CV0501 generated a ratio of post-boost to pre-boost serum neutralizing titers against BA.1 of 13.3, noted January 30, 2023.	5
HCM	Gastric cancer (GC) and gastroesophageal junction adenocarcinomas (GEJ)	Phase 2	4/19/23	Phase 2 data reported a confirmed ORR by IRC was 45%, and reached 50% in 16 patients with MET GCN (high) while only 1 PR was observed in 4 patients with MET GCN, noted April 19, 2023.	6
BCTX	Breast cancer	Phase 1/2	12/9/21	Phase 1/2a data reported that the control rate was 25% and 30% for the monotherapy studies and 33% for the combination study, noted December 9, 2021.	5
UTHR	Pulmonary hypertension associated with interstitial lung disease (PH-ILD) (WHO Group 3)	Approved	4/1/21	FDA approval announced April 1, 2021.	6
GSK	HIV for adolescents 12 years and older	Approved	3/29/22	Approved March 29, 2022.	6
GMDA	Severe aplastic anemia	Phase 2	12/6/20	Phase 2 data presented at ASH 2020 - sustained early engraftment shown.	6
EXEL	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
TXMD	Moderate-to-severe vaginal pain	Approved	5/30/18	CRL announced May 8, 2017. NDA resubmitted with approval announced May 30, 2018.	6
MCRB	Mild-to-Moderate Ulcerative Colitis	Phase 2b	12/16/21	Phase 2b microbiome analyses reported engraftment and anticipated changes in disease-relevant metabolites post-administration were not observed, noted December 16, 2021.	6
AUTL	Adult Acute lymphoblastic leukemia (ALL) / Chronic Lymphocytic Leukemia (CLL) / B-cell non-Hodgkin Lymphoma (B-NHL)	Phase 1/2	12/12/22	Longer term extension trial data reported that 35% of adult relapsed/refractory B-ALL patients treated with obe-cel in sustained complete remissions between 24 and 47 months without any need for additional anti-leukemia therapy, noted December 12, 2022.	6
MRNA	Bivalent booster COVID-19 vaccine	Phase 2/3	6/22/22	Phase 2/3 trial data reported that neutralizing titers against BA.4/BA.5 were approximately 3-fold lower than previously reported neutralizing titers against BA.1. One month after an mRNA-1273.214 booster, neutralizing geometric mean titers (GMT) against BA.4/BA.5 were 941 (95% CI: 826, 1071) in all participants, and 727 (95% CI: 633, 836) in seronegative participants, noted June 22, 2022.	5
RVNC	Forehead lines	Phase 2	6/30/20	Phase 2 data noted 100% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group - June 30, 2020.	6
PFE	Tick-borne encephalitis (TBE) vaccine	Approved	8/13/21	FDA Approval announced August 13, 2021	6
TSHA	Rett syndrome	Phase 1/2	8/24/23	FDA granted Fast Track Designation (FTD), noted August 24, 2023.	5
BIIB	Alport Syndrome	CRL	2/25/22	CRL issued February 25, 2022.	5
BPMC	Fourth-line GIST	CRL	5/15/20	CRL issued May 15, 2020.	5
BDRX	Children with Newly Diagnosed Diffuse Midline Gliomas (DMGs)	Phase 1	7/10/23	Phase 1 enrollment completed, noted July 10, 2023.	5
BMY	First-line Hepatocellular carcinoma (HCC) - cancer	Phase 3	6/24/19	Phase 3 trial did not meet primary endpoint.	4
RGLS	Alport Syndrome	Phase 2	7/15/22	Phase 2 trial terminated after failing to meet pre-defined futility criteria, noted July 15, 2022.	6
RHHBY	Hemophilia	Approved	10/4/18	FDA Approval announced October 4, 2018.	6
INVA	Hereditary Hemochromatosis	Phase 2	6/6/19	Phase 2 data met primary endpoint - June 6, 2019.	4
BMY	Ovarian Cancer - First-line maintenance treatment	Phase 3	6/6/22	Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.	6
ACIU	Autosomal Dominant Alzheimer's Disease (ADAD	Phase 3	8/2/22	Additional Phase 3 data reported that cognitive test scores of API ADAD composite 22.9%, FCSRT 19.9% and RBANS total score 43.8%, noted August 2, 2022.	5
NVS	Breast cancer	Phase 3	9/9/22	Phase 3 overall survival data reported a median OS of approximately five years, noted September 9, 2022.	5
CLDX	Chronic spontaneous urticaria (CSU)	Phase 2	7/11/23	Phase 2 data ahead of schedule, enrollment completed, noted July 11, 2023.	5
REPL	Liver and kidney transplant recipients with recurrent Cutaneous squamous cell carcinoma	Phase 1/2	3/30/22	Phase 1b/2 data reported that two of six patients (33%) achieved a response after treatment, with one complete response and one partial response, noted March 30, 2022.	6
BDRX	Recurrent Glioblastoma (GBM)	Phase 1	10/3/23	Phase 1 Cohort A enrollment completed, noted October 3, 2023.	5
PBYI	Small Cell Lung Cancer (SCLC)	Phase 1a	8/8/23	FDA IND Clearance on August 8, 2023.	5
MGNX	Gastric cancer	Phase 2/3	9/16/21	Phase 2/3 results had 4 patients with confirmed complete responses and 17 confirmed partial responses, September 16, 2021.	5
PPBT	2L metastatic pancreatic cancer (PDAC)	Phase 1/2	10/6/23	Phase 1 biomarker data demonstrated that CM24 treatment significantly reduced the level of NET marker in patients' serum, noted October 6, 2023.	6
CNTX	Ovarian cancer	Phase 2	6/6/22	Phase 2 data presented at ASCO reported that a 12-month PFS rate of 20.1% and a CBR of 35.7% in patients with GCT. No objective responses were observed, noted June 6, 2022.	6
NBIX	Tourette syndrome - adults	Phase 2	1/18/17	Phase 2 data released January 18, 2017 did not meet primary endpoint.	4
INCY	Atopic dermatitis	Approved	9/21/21	Approved September 21, 2021.	6
AGIO	Glioma	Phase 1	6/1/18	Phase 1 data presented at ASCO June 1, 2018. 1 minor response + 1 partial response out of 93 patients.	5
GSK	Mild to moderate COVID-19	Phase 3	3/3/21	Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.	4
LLY	Cluster headache	Approved	6/4/19	FDA Approval announced June 4, 2019.	6
JNJ	COVID-19 vaccine - two-dose regimen	Approved	10/20/21	EUA for second dose for adults aged 18 and older at least two months following primary vaccination with single-shot Johnson & Johnson COVID-19 vaccine; and for eligible individuals who received a different authorized or approved COVID-19 vaccine, noted October 20, 2021.	6
FNCH	Clostridioides difficile infection	Phase 2	6/1/22	Phase 2 data presented at DDW reported that 88.2% of participants were without CDI recurrence through 8 weeks in this post-hoc analysis, noted June 1, 2022.	6
ATAI	Treatment Resistant Depression	Phase 1	10/2/23	Phase 1 completed, noted October 2, 2023.	5
PMVP	Solid tumors	Phase 1/2	10/12/23	Phase 1/2 updated results presented at AACR-NCI-EORTC reported that the overall response rate (ORR) was 38% (6/16 evaluable patients) at the Recommended Phase 2 Dose (RP2D) of 2000 mg daily and 34% (13/38) in the efficacious dose range, noted October 12, 2023.	6
MOR	Diffuse Large B-Cell Lymphoma (DLBCL)	Phase 1b	12/12/22	Phase 1b data reported that ORR at EoT visit and best response across all visits were higher in Arm T/L, as were 18-month DoR, PFS, and OS rates, noted December 12, 2022.	5
BMY	Stage IIB or IIC melanoma	Approved	10/16/23	Approved October 13, 2023.	6
VRTX	Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations	Approved	3/17/15	Approved March 17, 2015.	6
ETON	Nutritional requirements for patients with liver disease	Approved	4/11/22	Approved April 11, 2022.	6
VIR	Influenza	Phase 2	7/20/23	Phase 2 trial did not meet its primary endpoint, noted July 20, 2023.	6
RCKT	Danon disease	Phase 1	5/18/23	Phase 1 additional results reported that efficacy results continue to demonstrate sustained improvement or stabilization in all patients with preserved left ventricular systolic function at time of treatment, noted May 18, 2023.	6
LPTX	Endometrial Cancer	Phase 2	3/22/21	Phase 2 data presented at Society of Gynecologic Oncology 2021 Annual Meeting, March 19-25, 2021. PFS 5.4 months.	5
GOSS	Pulmonary arterial hypertension (PAH)	Phase 2	7/25/23	Phase 2 OLE data reported that 9 of 11 patients continued to improve, with a mean improvement of 9 PVR dual responders of 39%, and 3 patients reached a PVR below 200 dyne*s/cm5n, noted July 25, 2023.	6
BMY	Newly Diagnosed Multiple Myeloma	Phase 2a	12/10/22	Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.	6
ORMP	COVID-19 vaccine	Phase 1	10/7/22	Phase 1 preliminary results reported a significant antibody response (2-6 fold over baseline) and no safety issues observed, noted October 7, 2022.	6
RGLS	Autosomal Dominant Polycystic Kidney Disease (ADPKD)	Phase 1	9/20/23	Phase 1b data showed that it was well tolerated by all 9 subjects receiving active drug with no safety findings. Increases in both PC1 and PC2 biomarkers were observed. Statistically significant increases in mean PC1 levels were observed at Day 85 and Day 86 (n=9) compared to baseline (36%-41%). Numeric increases in PC2 were observed during the treatment period, although not statistically significant, noted September 20, 2023.	6
PBYI	Metastatic triple negative breast cancer with a HER2 mutation	Phase 2	10/27/22	Phase 2 basket trial interim efficacy results showed that the objective response rate (ORR) was 35% overall, 30% in patients pretreated with TKIs, and 50% in patients not pretreated with TKIs. Response or stable disease lasting for ≥ 48 weeks was observed in 7 patients (6 PR, 1 SD), noted October 27, 2022.	6
ZYNE	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
RGNX	Duchenne Muscular Dystrophy (DMD)	Phase 1/2	10/3/23	Phase 1/2 additional data shared at the World Muscle Society Congress reported that in the patient aged 4.4 years old, RGX-202 microdystrophin expression was measured to be 38.8% compared to control, and in the patient aged 10.6 years old, RGX-202 microdystrophin expression was measured to be 11.1% compared to control, noted October 3, 2023.	5
SAGE	Postpartum Depression - moderate	Approved	3/19/19	FDA approval announced March 19, 2018.	6
MRK	HIV	Approved	9/20/19	FDA Approval for sNDA announced September 20, 2019.	6
MRK	HIV-1	Phase 2b	10/27/21	Phase 2b data through 144 weeks demonstrated that dosing continued to maintain viral suppression, as measured by the number of study participants achieving HIV-1 RNA levels <50 copies/mL, similar to DOR/3TC/TDF, noted October 27, 2021.	6
GSK	Chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype	CRL	9/7/18	CRL issued September 7, 2018.	5
BMY	Plaque psoriasis	Phase 3	11/3/20	Phase 3 trial met both co-primary endpoints - November 3, 2020.	6
ARWR	Cystic Fibrosis	Phase 1/2	7/2/21	Phase 1/2 trial paused following a trial in rats that contained unexpected signals of local lung inflammation - July 2, 2021.	4
KPTI	Quadruple Refractory Multiple Myeloma	Approved	7/3/19	FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).	6
EXEL	BRAF V600 Mutation-Positive Advanced Melanoma - Cancer	Approved	11/10/15	Approved November 10, 2015.	6
ROIV	Plaque psoriasis	Approved	5/24/22	Approved May 24, 2022.	6
PFE	Atopic Dermatitis	Approved	1/14/22	Approved January 14, 2022.	6
GERN	Myelodysplastic syndromes	Phase 3	6/2/23	Phase 3 data presented at ASCO showed that its primary endpoint of 8-week transfusion independence (TI) continues significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders, noted June 2, 2023	6
REGN	Osteoarthritis pain of the hip or knee	Phase 3	8/5/20	Phase 3 data achieved the co-primary endpoints for fasinumab 1 mg monthly. Fasinumab every two months did not reach statistical significance - August 5, 2020.	5
SGEN	Diffuse large B-cell lymphoma (DLBCL)	Approved	6/10/19	FDA approval announced June 10, 2019.	6
SNY	Asthma - 6-11 year-olds	Approved	10/20/21	Approved October 20, 2021.	6
MOR	Myelofibrosis	Phase 2	6/10/22	Phase 2 efficacy and safety data reported that over two-thirds of JAK inhibitor-naïve patients treated with the combination achieved at least a 35% reduction in spleen volume (SVR35) from baseline at week 24. Notably, 80% of patients achieved SVR35 at any time on study, noted June 10, 2022.	6
BMY	Triple-negative breast cancer	Approved	3/8/19	FDA Approval announced March 8, 2019.	6
ALDX	Dry eye disease	Phase 3	12/20/21	Phase 3 trial did not meet the primary endpoint, secondary endpoint was achieved, December 20, 2021.	6
PRTA	Alzheimer’s disease	Phase 1	7/17/23	Phase 1 SAD data presented at AAIC met key pharmacokinetic (PK) and immunogenicity secondary endpoints, noted July 17, 2023	6
ADVM	Diabetic macular edema (DME)	Phase 2	10/9/21	Phase 2 data from trial (June 22, 2021, cutoff date, n=34) at ASRS demonstrated that dose and disease state appear to have an impact in determining the therapeutic window which balances efficacy and safety. Dose-dependent inflammation observed may be in part related to the study population which included patients with severe vascular and renal disease. Further drug development will focus on wet AMD and low doses (2 x 10^11 vg/eye and lower) - noted October 9, 2021	5
VTGN	Premenstrual dysphoric disorder (PMDD)	Phase 2a	9/12/23	Phase 2a exploratory analysis demonstrated statistically and clinically significant improvement versus placebo, noted Septemeber 12, 2023.	6
PYPD	Abdominal (soft tissue) sternal surgical site infections	Phase 3	9/2/22	Phase 3 trial did not meet primary endpoint, noted September 2, 2022.	6
ZLAB	Non-Hodgkin Lymphoma (NHL)	Phase 2	12/12/22	Phase 2 data presented at ASH showed a 49% ORR in heavily pre-treated patients who were naïve to prior CAR-T, with 31% achieving a complete response (CR), noted December 12, 2022.	6
RHHBY	BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma	Approved	7/30/20	FDA approval announced July 30, 2020.	6

PFE

Lyme disease vaccine for outdoor recreationists

Phase 3

2/17/23

Phase 3 study discontinued about 50% of patients enrolled following violations of Good Clinical Practice (GCP) at certain clinical trial sites run by a third-party clinical trial site operator, noted February 17, 2023.

4

BXRX

Acute pain following bunionectomy surgery

Approved

2/20/20

FDA Approval announced February 20, 2020.

6

KALV

Hereditary angioedema (HAE)

Phase 1

6/9/23

Phase 1 data presented at EAACI showed that pharmacokinetic and pharmacodynamic data support globalization of the phase 3 clinical trial and use in short-term prophylaxis, noted June 9, 2023.

6

SLRX

Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Phase 1/2

12/12/22

Phase 1/2 data presented at ASH reported an ORR of 50% among eight myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), noted December 12, 2022.

6

INCY

Solid Tumors

Phase 1/2

6/3/23

Phase 1/2 data from ASCO reported that the best overall response in part 1 was stable disease, and in part 2 was a confirmed and durable partial response in 1 pt, noted June 3, 2023.

5

GOVX

Covid-19

Phase 2

9/11/23

Phase 2 target enrollment met, noted September 11, 2023.

5

AZN

Hyperkalaemia

Approved

5/18/18

Approval announced May 18, 2018.

6

OPK

Mild-to-moderate COVID-19

Phase 2

12/23/21

Phase 2 top-line data reported that mean serum 25D levels increased with treatment and remained elevated for the duration of the trial, with 88% of subjects attaining the targeted level. Three symptoms related to respiratory function resolved more quickly when serum 25D was elevated at Days 7 and 14, with resolution of chest congestion occurring 3.4 days sooner, noted December 23, 2021.

6

CPRX

Congenital Myasthenic Syndromes (CMS)

Phase 3

10/30/19

Phase 3 trial did not meet primary endpoint - October 30, 2019.

4

VALN

Booster shot COVID-19 vaccine

Phase 1/2

1/19/22

Additional data reported that all 30 samples (100%) presented neutralizing antibodies against the ancestral virus and Delta variant, and 26 samples (87%) presented neutralizing antibodies against the Omicron variant, noted January 19, 2022.

6

MESO

End-Stage Heart Failure with LVADs

Phase 2b

11/11/18

Phase 2b trial did not meet primary endpoint - November 11, 2018.

4

RAIN

MDM2-amplified advanced solid tumors

Phase 2

5/30/23

Phase 2 enrollment suspended, noted May 30, 2023.

4

MRK

Metastatic castration-resistant prostate cancer (mCRPC)

Phase 3

8/3/22

Phase 3 trial results were not statistically significant, noted August 3, 2022.

5

IOBT

Non-muscle invasive bladder cancer (NMIBC)

Phase 1

7/28/23

Phase 1 dosing initiated, noted July 28, 2023.

5

BIIB

Lupus

Phase 2

12/3/19

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

6

IMAB

Growth hormone deficiency

Phase 3

8/17/23

Phase 3 trial met its primary endpoint, noted August 17, 2023.

5

PFE

HIV

Approved

12/21/21

Approved December 21, 2021.

6

PFE

Lyme disease

Phase 2

9/7/23

Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.

6

AGIO

Sickle cell disease

Phase 2

6/26/23

Phase 2 portion met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms, noted June 26, 2023

5

ENTX

Osteoporosis

Phase 2

10/14/23

Phase 2 data presented at ASBMR showed that an increase in plasma ionized calcium should result in decreased secretion and plasma concentrations of endogenous PTH(1-84), noted October 14, 2023

6

ZYME

3L+ HER2-positive, HR-positive breast cancer

Phase 2

12/9/22

Phase 2 data reported a confirmed objective response rate (cORR) of 33%, disease control rate (DCR) of 92%, and median progression-free survival (mPFS) of 9.6 months, noted December 9, 2022.

5

ALKS

Recurrent or metastatic head and neck squamous cell carcinoma

Phase 2

11/13/21

Phase 2 data showed that combination therapy was generally well tolerated (n=14), 8 had no prior response to dosing and 6 had previous best response of SD or PR. Mean (SD±) age was 62±12; AE were consistent with those observed with intravenous studies. Peripheral immune cell expansion profiles are comparable to that observed with the same regimen.

6

SRPT

Limb-girdle muscular dystrophy type 2E (LGMD2E)

Phase 1/2

10/11/21

Results from most recent study found that treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the NSAA compared to a matched natural history cohort 3 years after treatment- noted October 11, 2021.

6

GSK

Covid-19 vaccine, omicron variant

Phase 1

1/30/23

Phase 1 immunogenicity data reported that on day 29 at the 12µg dose level, CV0501 generated a ratio of post-boost to pre-boost serum neutralizing titers against BA.1 of 13.3, noted January 30, 2023.

5

HCM

Gastric cancer (GC) and gastroesophageal junction adenocarcinomas (GEJ)

Phase 2

4/19/23

Phase 2 data reported a confirmed ORR by IRC was 45%, and reached 50% in 16 patients with MET GCN (high) while only 1 PR was observed in 4 patients with MET GCN, noted April 19, 2023.

6

BCTX

Breast cancer

Phase 1/2

12/9/21

Phase 1/2a data reported that the control rate was 25% and 30% for the monotherapy studies and 33% for the combination study, noted December 9, 2021.

5

UTHR

Pulmonary hypertension associated with interstitial lung disease (PH-ILD) (WHO Group 3)

Approved

4/1/21

FDA approval announced April 1, 2021.

6

GSK

HIV for adolescents 12 years and older

Approved

3/29/22

Approved March 29, 2022.

6

GMDA

Severe aplastic anemia

Phase 2

12/6/20

Phase 2 data presented at ASH 2020 - sustained early engraftment shown.

6

EXEL

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

TXMD

Moderate-to-severe vaginal pain

Approved

5/30/18

CRL announced May 8, 2017. NDA resubmitted with approval announced May 30, 2018.

6

MCRB

Mild-to-Moderate Ulcerative Colitis

Phase 2b

12/16/21

Phase 2b microbiome analyses reported engraftment and anticipated changes in disease-relevant metabolites post-administration were not observed, noted December 16, 2021.

6

AUTL

Adult Acute lymphoblastic leukemia (ALL) / Chronic Lymphocytic Leukemia (CLL) / B-cell non-Hodgkin Lymphoma (B-NHL)

Phase 1/2

12/12/22

Longer term extension trial data reported that 35% of adult relapsed/refractory B-ALL patients treated with obe-cel in sustained complete remissions between 24 and 47 months without any need for additional anti-leukemia therapy, noted December 12, 2022.

6

MRNA

Bivalent booster COVID-19 vaccine

Phase 2/3

6/22/22

Phase 2/3 trial data reported that neutralizing titers against BA.4/BA.5 were approximately 3-fold lower than previously reported neutralizing titers against BA.1. One month after an mRNA-1273.214 booster, neutralizing geometric mean titers (GMT) against BA.4/BA.5 were 941 (95% CI: 826, 1071) in all participants, and 727 (95% CI: 633, 836) in seronegative participants, noted June 22, 2022.

5

RVNC

Forehead lines

Phase 2

6/30/20

Phase 2 data noted 100% of subjects achieved a score of none or mild wrinkle or line severity at Week 4 in at least one treatment group - June 30, 2020.

6

PFE

Tick-borne encephalitis (TBE) vaccine

Approved

8/13/21

FDA Approval announced August 13, 2021

6

TSHA

Rett syndrome

Phase 1/2

8/24/23

FDA granted Fast Track Designation (FTD), noted August 24, 2023.

5

BIIB

Alport Syndrome

CRL

2/25/22

CRL issued February 25, 2022.

5

BPMC

Fourth-line GIST

CRL

5/15/20

CRL issued May 15, 2020.

5

BDRX

Children with Newly Diagnosed Diffuse Midline Gliomas (DMGs)

Phase 1

7/10/23

Phase 1 enrollment completed, noted July 10, 2023.

5

BMY

First-line Hepatocellular carcinoma (HCC) - cancer

Phase 3

6/24/19

Phase 3 trial did not meet primary endpoint.

4

RGLS

Alport Syndrome

Phase 2

7/15/22

Phase 2 trial terminated after failing to meet pre-defined futility criteria, noted July 15, 2022.

6

RHHBY

Hemophilia

Approved

10/4/18

FDA Approval announced October 4, 2018.

6

INVA

Hereditary Hemochromatosis

Phase 2

6/6/19

Phase 2 data met primary endpoint - June 6, 2019.

4

BMY

Ovarian Cancer - First-line maintenance treatment

Phase 3

6/6/22

Additional Phase 3 data reported that median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months, noted June 6, 2022.

6

ACIU

Autosomal Dominant Alzheimer's Disease (ADAD

Phase 3

8/2/22

Additional Phase 3 data reported that cognitive test scores of API ADAD composite 22.9%, FCSRT 19.9% and RBANS total score 43.8%, noted August 2, 2022.

5

NVS

Breast cancer

Phase 3

9/9/22

Phase 3 overall survival data reported a median OS of approximately five years, noted September 9, 2022.

5

CLDX

Chronic spontaneous urticaria (CSU)

Phase 2

7/11/23

Phase 2 data ahead of schedule, enrollment completed, noted July 11, 2023.

5

REPL

Liver and kidney transplant recipients with recurrent Cutaneous squamous cell carcinoma

Phase 1/2

3/30/22

Phase 1b/2 data reported that two of six patients (33%) achieved a response after treatment, with one complete response and one partial response, noted March 30, 2022.

6

BDRX

Recurrent Glioblastoma (GBM)

Phase 1

10/3/23

Phase 1 Cohort A enrollment completed, noted October 3, 2023.

5

PBYI

Small Cell Lung Cancer (SCLC)

Phase 1a

8/8/23

FDA IND Clearance on August 8, 2023.

5

MGNX

Gastric cancer

Phase 2/3

9/16/21

Phase 2/3 results had 4 patients with confirmed complete responses and 17 confirmed partial responses, September 16, 2021.

5

PPBT

2L metastatic pancreatic cancer (PDAC)

Phase 1/2

10/6/23

Phase 1 biomarker data demonstrated that CM24 treatment significantly reduced the level of NET marker in patients' serum, noted October 6, 2023.

6

CNTX

Ovarian cancer

Phase 2

6/6/22

Phase 2 data presented at ASCO reported that a 12-month PFS rate of 20.1% and a CBR of 35.7% in patients with GCT. No objective responses were observed, noted June 6, 2022.

6

NBIX

Tourette syndrome - adults

Phase 2

1/18/17

Phase 2 data released January 18, 2017 did not meet primary endpoint.

4

INCY

Atopic dermatitis

Approved

9/21/21

Approved September 21, 2021.

6

AGIO

Glioma

Phase 1

6/1/18

Phase 1 data presented at ASCO June 1, 2018. 1 minor response + 1 partial response out of 93 patients.

5

GSK

Mild to moderate COVID-19

Phase 3

3/3/21

Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.

4

LLY

Cluster headache

Approved

6/4/19

FDA Approval announced June 4, 2019.

6

JNJ

COVID-19 vaccine - two-dose regimen

Approved

10/20/21

EUA for second dose for adults aged 18 and older at least two months following primary vaccination with single-shot Johnson & Johnson COVID-19 vaccine; and for eligible individuals who received a different authorized or approved COVID-19 vaccine, noted October 20, 2021.

6

FNCH

Clostridioides difficile infection

Phase 2

6/1/22

Phase 2 data presented at DDW reported that 88.2% of participants were without CDI recurrence through 8 weeks in this post-hoc analysis, noted June 1, 2022.

6

ATAI

Treatment Resistant Depression

Phase 1

10/2/23

Phase 1 completed, noted October 2, 2023.

5

PMVP

Solid tumors

Phase 1/2

10/12/23

Phase 1/2 updated results presented at AACR-NCI-EORTC reported that the overall response rate (ORR) was 38% (6/16 evaluable patients) at the Recommended Phase 2 Dose (RP2D) of 2000 mg daily and 34% (13/38) in the efficacious dose range, noted October 12, 2023.

6

MOR

Diffuse Large B-Cell Lymphoma (DLBCL)

Phase 1b

12/12/22

Phase 1b data reported that ORR at EoT visit and best response across all visits were higher in Arm T/L, as were 18-month DoR, PFS, and OS rates, noted December 12, 2022.

5

BMY

Stage IIB or IIC melanoma

Approved

10/16/23

Approved October 13, 2023.

6

VRTX

Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations

Approved

3/17/15

Approved March 17, 2015.

6

ETON

Nutritional requirements for patients with liver disease

Approved

4/11/22

Approved April 11, 2022.

6

VIR

Influenza

Phase 2

7/20/23

Phase 2 trial did not meet its primary endpoint, noted July 20, 2023.

6

RCKT

Danon disease

Phase 1

5/18/23

Phase 1 additional results reported that efficacy results continue to demonstrate sustained improvement or stabilization in all patients with preserved left ventricular systolic function at time of treatment, noted May 18, 2023.

6

LPTX

Endometrial Cancer

Phase 2

3/22/21

Phase 2 data presented at Society of Gynecologic Oncology 2021 Annual Meeting, March 19-25, 2021. PFS 5.4 months.

5

GOSS

Pulmonary arterial hypertension (PAH)

Phase 2

7/25/23

Phase 2 OLE data reported that 9 of 11 patients continued to improve, with a mean improvement of 9 PVR dual responders of 39%, and 3 patients reached a PVR below 200 dyne*s/cm5n, noted July 25, 2023.

6

BMY

Newly Diagnosed Multiple Myeloma

Phase 2a

12/10/22

Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.

6

ORMP

COVID-19 vaccine

Phase 1

10/7/22

Phase 1 preliminary results reported a significant antibody response (2-6 fold over baseline) and no safety issues observed, noted October 7, 2022.

6

RGLS

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Phase 1

9/20/23

Phase 1b data showed that it was well tolerated by all 9 subjects receiving active drug with no safety findings. Increases in both PC1 and PC2 biomarkers were observed. Statistically significant increases in mean PC1 levels were observed at Day 85 and Day 86 (n=9) compared to baseline (36%-41%). Numeric increases in PC2 were observed during the treatment period, although not statistically significant, noted September 20, 2023.

6

PBYI

Metastatic triple negative breast cancer with a HER2 mutation

Phase 2

10/27/22

Phase 2 basket trial interim efficacy results showed that the objective response rate (ORR) was 35% overall, 30% in patients pretreated with TKIs, and 50% in patients not pretreated with TKIs. Response or stable disease lasting for ≥ 48 weeks was observed in 7 patients (6 PR, 1 SD), noted October 27, 2022.

6

ZYNE

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

RGNX

Duchenne Muscular Dystrophy (DMD)

Phase 1/2

10/3/23

Phase 1/2 additional data shared at the World Muscle Society Congress reported that in the patient aged 4.4 years old, RGX-202 microdystrophin expression was measured to be 38.8% compared to control, and in the patient aged 10.6 years old, RGX-202 microdystrophin expression was measured to be 11.1% compared to control, noted October 3, 2023.

5

SAGE

Postpartum Depression - moderate

Approved

3/19/19

FDA approval announced March 19, 2018.

6

MRK

HIV

Approved

9/20/19

FDA Approval for sNDA announced September 20, 2019.

6

MRK

HIV-1

Phase 2b

10/27/21

Phase 2b data through 144 weeks demonstrated that dosing continued to maintain viral suppression, as measured by the number of study participants achieving HIV-1 RNA levels <50 copies/mL, similar to DOR/3TC/TDF, noted October 27, 2021.

6

GSK

Chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype

CRL

9/7/18

CRL issued September 7, 2018.

5

BMY

Plaque psoriasis

Phase 3

11/3/20

Phase 3 trial met both co-primary endpoints - November 3, 2020.

6

ARWR

Cystic Fibrosis

Phase 1/2

7/2/21

Phase 1/2 trial paused following a trial in rats that contained unexpected signals of local lung inflammation - July 2, 2021.

4

KPTI

Quadruple Refractory Multiple Myeloma

Approved

7/3/19

FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).

6

EXEL

BRAF V600 Mutation-Positive Advanced Melanoma - Cancer

Approved

11/10/15

Approved November 10, 2015.

6

ROIV

Plaque psoriasis

Approved

5/24/22

Approved May 24, 2022.

6

PFE

Atopic Dermatitis

Approved

1/14/22

Approved January 14, 2022.

6

GERN

Myelodysplastic syndromes

Phase 3

6/2/23

Phase 3 data presented at ASCO showed that its primary endpoint of 8-week transfusion independence (TI) continues significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders, noted June 2, 2023

6

REGN

Osteoarthritis pain of the hip or knee

Phase 3

8/5/20

Phase 3 data achieved the co-primary endpoints for fasinumab 1 mg monthly. Fasinumab every two months did not reach statistical significance - August 5, 2020.

5

SGEN

Diffuse large B-cell lymphoma (DLBCL)

Approved

6/10/19

FDA approval announced June 10, 2019.

6

SNY

Asthma - 6-11 year-olds

Approved

10/20/21

Approved October 20, 2021.

6

MOR

Myelofibrosis

Phase 2

6/10/22

Phase 2 efficacy and safety data reported that over two-thirds of JAK inhibitor-naïve patients treated with the combination achieved at least a 35% reduction in spleen volume (SVR35) from baseline at week 24. Notably, 80% of patients achieved SVR35 at any time on study, noted June 10, 2022.

6

BMY

Triple-negative breast cancer

Approved

3/8/19

FDA Approval announced March 8, 2019.

6

ALDX

Dry eye disease

Phase 3

12/20/21

Phase 3 trial did not meet the primary endpoint, secondary endpoint was achieved, December 20, 2021.

6

PRTA

Alzheimer’s disease

Phase 1

7/17/23

Phase 1 SAD data presented at AAIC met key pharmacokinetic (PK) and immunogenicity secondary endpoints, noted July 17, 2023

6

ADVM

Diabetic macular edema (DME)

Phase 2

10/9/21

Phase 2 data from trial (June 22, 2021, cutoff date, n=34) at ASRS demonstrated that dose and disease state appear to have an impact in determining the therapeutic window which balances efficacy and safety. Dose-dependent inflammation observed may be in part related to the study population which included patients with severe vascular and renal disease. Further drug development will focus on wet AMD and low doses (2 x 10^11 vg/eye and lower) - noted October 9, 2021

5

VTGN

Premenstrual dysphoric disorder (PMDD)

Phase 2a

9/12/23

Phase 2a exploratory analysis demonstrated statistically and clinically significant improvement versus placebo, noted Septemeber 12, 2023.

6

PYPD

Abdominal (soft tissue) sternal surgical site infections

Phase 3

9/2/22

Phase 3 trial did not meet primary endpoint, noted September 2, 2022.

6

ZLAB

Non-Hodgkin Lymphoma (NHL)

Phase 2

12/12/22

Phase 2 data presented at ASH showed a 49% ORR in heavily pre-treated patients who were naïve to prior CAR-T, with 31% achieving a complete response (CR), noted December 12, 2022.

6

RHHBY

BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma

Approved

7/30/20

FDA approval announced July 30, 2020.

6