quant-chef/BioPharmaCatalysts · Datasets at Hugging Face

ticker stringlengths 2 5	disease stringlengths 3 196	stage stringclasses 15 values	date stringlengths 19 19	catalyst stringlengths 18 760	label int64 -1 1
PLUR	COVID-19 / Acute Respiratory Distress Syndrome (ARDS)	Phase 2	2021-12-27 00:00:00	Phase 2 data did not meet the primary efficacy endpoint of statistically significant improvement of ventilator free days (VFD) at 28 days. Taking into consideration the baseline risk factors of the ARDS patients, no differences in the safety profile were observed between PLX-PAD and placebo.	1
JNJ	Non-small cell lung cancer	Phase 1/2	2022-07-26 00:00:00	Phase 1b/2 data reported an overall response rate (ORR) of 50%, with 15 out of 20 patients remaining on treatment, noted July 26, 2022.	1
GSK	COVID-19 antibody	Approved	2021-05-26 00:00:00	FDA Emergency Use Authorization filing approval announced May 26, 2021.	1
VACC	Hepatitis B (HBV)	Phase 1/2	2023-06-21 00:00:00	Phase 1b/2a data at EASL showed that all participants who received and experienced a >0.5 log10 reduction in HBsAg had durable responses with reductions in HBsAg persisting through to the last measurement eight months post-final dose, noted June 21, 2023.	1
ARWR	Dyslipidemia	Phase 2b	2022-11-07 00:00:00	Phase 2b data presented at AHA reported that ARO-ANG3 is associated with relative reduction in liver fat fraction at Week 24, with no AEs related to LFT changes reported to date, noted November 7, 2022.	1
BMY	Progressive pulmonary fibrosis (PPF)	Phase 2	2023-10-24 00:00:00	Breakthrough Therapy Designation (BTD) granted by FDA on October 24, 2023.	0
TBPH	Asthma	Approved	2015-04-30 00:00:00	Approved April 30, 2015.	1
APVO	Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)	Phase 1b	2023-07-18 00:00:00	Phase 1b expansion trial data reported that one patient remained on study with sustained complete response for 8 cycles, noted July 18, 2023.	0
SNDX	Ovarian cancer	Phase 1/2	2019-03-07 00:00:00	Phase 2 top-line data March 7, 2019 failed to meet endpoints.	1
KALV	Hereditary angioedema (HAE)	Phase 1	2023-06-09 00:00:00	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.	1
PHAT	Erosive esophagitis / Gastroesophageal reflux disease (GERD)	Approved	2023-11-01 00:00:00	FDA Approved on November 1, 2023.	1
AXSM	Narcolepsy	Phase 2	2019-12-03 00:00:00	Phase 2 data met primary endpoint.	0
CLVS	Urothelial carcinoma	Phase 2	2019-04-12 00:00:00	Phase 2 trial to be discontinued due to lack of efficacy - April 12, 2019.	-1
NOVN	Fungal infections	Phase 2	2017-04-12 00:00:00	Phase 2 data released April 12, 2017. Primary endpoint met.	0
ENTA	Respiratory Syncytial Virus RSV-A and RSV-B	Phase 1	2023-06-20 00:00:00	Phase 1 data demonstrated favorable safety, tolerability, and PK supportive of once-daily dosing, with good exposure multiples, thereby supporting further clinical advancement of EDP-323, noted June 20, 2023.	1
ACIU	Alzheimer’s disease	Phase 3	2019-01-30 00:00:00	Phase 3 trial terminated following interim analysis indicating primary endpoint would not be met.	-1
BIIB	Lupus	Phase 2b	2018-10-23 00:00:00	Phase 2b data released October 23, 2018 - primary endpoint not met.	0
ANAB	Non-squamous non-small cell lung cancer (NSCLC)	Phase 2	2022-10-05 00:00:00	Phase 2 trial met primary endpoint, noted October 5, 2022.	0
CRDF	Pancreatic ductal adenocarcinoma (PDAC)	Phase 2	2023-09-26 00:00:00	Phase 2 additional data reported an objective response rate (ORR) of 19% and median progression-free survival (mPFS) of 5.0 months, noted September 26, 2023.	0
PRAX	Major Depressive Disorder and perimenopausal depression	Phase 2a	2021-08-16 00:00:00	Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes.	0
ABBV	Acute Myeloid Leukemia (AML)	Approved	2020-10-16 00:00:00	FDA approval announced October 16, 2020.	1
MRK	Biliary Tract Cancer	Approved	2023-11-01 00:00:00	Approved November 1, 2023.	1
LLY	Heart failure with preserved ejection fraction (HFpEF)	Phase 3	2019-12-13 00:00:00	Phase 3 data did not meet primary endpoint - December 13, 2019.	1
OTIC	Acute otitis externa	Approved	2018-03-02 00:00:00	Approval announced March 2, 2018.	1
MRTX	Non-Small Cell Lung Cancer (NSCLC)	Phase 2	2023-10-20 00:00:00	Phase 2 data presented at ESMO 2023 reported that in patients with PD-L1 TPS ≥50%, adagrasib + pembrolizumab demonstrated a higher response rate (63%) than expected with pembrolizumab monotherapy (39–45%3,4), noted October 20, 2023.	1
URGN	Low grade non-muscle invasive bladder cancer (LG-NMIBC)	Phase 3	2023-07-27 00:00:00	Phase 3 trial met primary endpoint with 79.2% rate of complete response at 3-months following the initial treatment, noted July 27, 2023.	1
RHHBY	Triple-negative breast cancer	Approved	2019-03-08 00:00:00	FDA Approval announced March 8, 2019.	1
JNJ	High-Risk Non-Muscle Invasive Bladder Cancer (NMIBC)	Phase 2b	2023-10-24 00:00:00	Phase 2b data presented at ESMO reported that centrally confirmed CR was achieved in 23/30 pts, noted October 24, 2023.	1
PSTV	Glioma	Phase 1/2	2023-06-29 00:00:00	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.	1
COLL	Opioid dependence	Approved	2014-06-06 00:00:00	Approved June 6, 2014.	1
AMGN	Rheumatoid arthritis (RA)	Approved	2022-06-06 00:00:00	Approved June 6, 2022.	1
MYOV	Uterine fibroids	Approved	2021-05-26 00:00:00	FDA approval announced May 26, 2021.	1
BIIB	Cognitive Impairment Associated With Schizophrenia (CIAS)	Phase 2b	2022-07-20 00:00:00	Phase 2b trial did not meet its primary endpoint, and program will be discontinued, noted July 20, 2022.	1
ASRT	Neutropenia	Approved	2022-09-09 00:00:00	Approved September 9, 2022.	1
REGN	Lymphoma	Phase 1/2	2022-05-26 00:00:00	Phase 1/2 data released in an abstract at ASCO reported no significant adverse effects (AEs) and clinical activity in 5 of 7 patients, noted May 26, 2022.	0
LLY	HR+, HER2-locoregionally recurrent or metastatic breast cancer (MBC)	Approved	2018-03-26 00:00:00	Approval announced February 26, 2018.	1
ARGX	Chronic inflammatory demyelinating polyneuropathy (CIDP)	Phase 2	2023-07-17 00:00:00	Phase 2 trial met primary endpoints, noted July 17, 2023.	0
EQRX	CNS metastases and EGFR-mutated non-small cell lung cancer (NSCLC)	Phase 3	2022-06-06 00:00:00	Phase 3 data reported that CNS PFS events were observed in 11 patients (38%) treated, and the confirmed CNS ORR were 82.8% and 75.0% in patients treated with Au and G, respectively, noted June 6, 2022.	1
GNFT	Non-alcoholic steatohepatitis (NASH)	Phase 3	2020-05-11 00:00:00	Phase 3 data did not meet primary endpoint - May 11, 2020.	1
INKT	Solid Tumors	Phase 1	2023-11-03 00:00:00	Phase 1 trial of agenT-797 alone or in combination showed durable clinical benefit, including: Clinical response in MSI-high 3L gastric cancer patient after failure on pembrolizumab and nivolumab/FOLFOX. Long-term disease stabilization (n=10), including in testicular cancer (SD 9 months) and anti-PD-1 relapsed/refractory non-small-cell lung cancer (SD > 10 months). Tolerable safety profile with no dose-limiting toxicities and no grade >3 neurotoxicity or cytokine release syndrome, noted November 3, 2023.	1
MNKKQ	Hemostasis	Approved	2015-04-30 00:00:00	Approved April 30, 2015.	1
ETNB	Severe hypertriglyceridemia	Phase 2	2023-06-24 00:00:00	Phase 2 data presented at EASL showed consistent and significant reductions in triglycerides, atherogenic lipoproteins and liver fat, and pegozafermin's favorable safety and tolerability profile, noted June 24, 2023.	1
CCCC	Synovial sarcoma	Phase 1	2023-11-01 00:00:00	Phase 1 deprioritized due to insufficient single agent efficacy, despite high levels of BRD9 degradation, noted November 1, 2023.	-1
SNY	Acquired thrombotic thrombocytopenic purpura (aTTP)	Approved	2019-02-06 00:00:00	FDA Approval announced February 6, 2019.	1
CCCC	Non-Small Cell Lung Cancer (NSCLC)	Phase 1a	2023-07-05 00:00:00	IND Cleared by FDA, July 5, 2023.	0
TAK	Cytomegalovirus (CMV)	Approved	2021-11-23 00:00:00	Approved November 23, 2021.	1
XERS	Hypoglycemia	Approved	2021-08-23 00:00:00	FDA approval August 23, 2021.	1
MRK	Cytomegalovirus (CMV) Infection	Approved	2017-11-09 00:00:00	Approval announced November 9, 2017.	1
PPBT	Squamous cell carcinoma of the head & neck (SCCHN)	Phase 1/2	2023-10-03 00:00:00	Phase 1/2 data showed that 2 out of 4 SCCHN patients at the highest dose cohort demonstrated a confirmed partial response, noted October 3, 2023.	1
REGN	Stage II to IV Cutaneous Squamous Cell Carcinoma (CSCC)	Phase 2	2023-10-16 00:00:00	Phase 2 data from ESMO reported a median duration of follow-up was 18.7 months, and 1 of 10 pts with MPR experienced recurrence, noted October 16, 2023.	0
JAZZ	Spasticity due multiple sclerosis	Phase 2	2017-02-07 00:00:00	Phase 2 trial did not meet primary endpoint - noted February 7, 2017.	1
HCM	Myeloid hematological malignancies (HMs)	Phase 1	2023-06-09 00:00:00	Phase 1 data reported that the best of response was 1CR in 100 mg cohort; 9 CRs and 1 PR in 150 mg QD; 1 CR in 200 mg QD; and 3 CRs in 250 mg QD; with an ORR of 33.3% (1/3), 40.0%(10/25), 14.3% (1/7) and 42.9% (3/7), respectively, noted June 9, 2023.	0
HCM	Follicular Lymphoma and Marginal Zone Lymphoma	Phase 2	2023-06-09 00:00:00	Phase 2 data from ICML presentation noted that median duration of response (DoR) and progression free survival (PFS) at median follow-up duration of 22.1 months was not reached, noted June 9, 2023.	1
OMER	IgA nephropathy	Phase 3	2023-10-16 00:00:00	Phase 3 9-month data did not meet its primary endpoint, as a result Omeros will not submit an application for approval of narsoplimab in this indication and will discontinue the ARTEMIS-IGAN clinical trial, noted October 16, 2023.	1
PCRX	Postsurgical pain	Approved	2011-10-31 00:00:00	Approved October 31, 2011.	1
MDWD	Severe burns	CRL	2021-06-29 00:00:00	CRL announced June 29, 2021.	0
MTEM	Solid tumors	Phase 1	2023-11-02 00:00:00	Phase 1 dosing initiated, noted November 2, 2023.	0
ENDP	Adhesive capsulitis (AC) of the shoulder	Phase 2	2022-07-01 00:00:00	Phase 2 trial did not report a significant difference between treatment and placebo, noted July 1, 2022.	1
ANNX	Huntington’s disease (HD)	Phase 2	2022-06-07 00:00:00	Phase 2 data reported that treatment led to complete and durable target engagement, disease progression was stabilized in the overall HD patient population for the entire nine months, and three trial discontinuations were due to treatment, noted June 7, 2022.	1
ATNXQ	Actinic keratosis	Approved	2020-12-15 00:00:00	FDA approval announced December 15, 2020.	1
BIIB	Lupus	Phase 2	2019-12-03 00:00:00	Phase 2 data met primary endpoint - December 3, 2019.	0
FGEN	Anemia in patients receiving concurrent chemotherapy treatment for non-myeloid malignancie	Phase 3	2023-05-18 00:00:00	Phase 3 trial met primary endpoint, noted May 18, 2023.	0
DCPH	Gastrointestinal Stromal Tumors (GIST) - second-line	Phase 3	2023-06-01 00:00:00	Phase 3 data reported that the mPFS (90% CI) by IRR was 6.44 (2.89, 8.31) months and ORR was 21.1%, noted June 1, 2023.	0
MRK	Beta-thalassemia	Approved	2019-11-08 00:00:00	FDA Approval announced November 8, 2019.	1
TBPH	Ulcerative Colitis	Phase 2b	2021-08-23 00:00:00	Phase 2b data did not meet primary endpoint - August 23, 2021.	1
RHHBY	Urothelial carcinoma	Phase 3	2019-08-05 00:00:00	Phase 3 PFS co-primary endpoint met - August 5, 2019.	0
LOGC	Methylmalonic Acidemia (MMA)	Phase 1/2	2022-02-02 00:00:00	Phase 1/2 trial placed on clinical hold due to 2 drug related SAEs, noted February 2, 2022.	0
IMMP	Solid tumors	Phase 1	2023-10-24 00:00:00	Phase 1 additional efficacy and tolerability data reported at ESMO showed strong 71.4% Overall Response Rate, 90.5% Disease Control Rate, 10.1-month median Progression Free Survival (PFS), and median Overall Survival (OS) that has not been reached, despite 81% of patients having low or negative PD-L1 expression, noted October 24, 2023.	1
AZN	First-line ovarian cancer following platinum-based chemotherapy	Approved	2018-12-19 00:00:00	FDA Approval announced December 19, 2018.	1
IOVA	Non-small cell lung cancer (NSCLC)	Phase 2	2021-11-12 00:00:00	Phase 2 data results showed a 21.4% overall response rate (ORR) in heavily pre-treated patients (n=28), including one complete response and five partial responses, noted November 12, 2021.	1
NBRV	Moderate to severe Community Acquired Bacterial Pneumonia (CABP)	Approved	2019-08-19 00:00:00	FDA approval announced August 19, 2019.	1
JNJ	Pediatric Chronic Graft-Versus-Host Disease	Approved	2022-08-24 00:00:00	Approved August 24, 2022.	1
TARS	Demodex Blepharitis	Phase 2/3	2021-06-21 00:00:00	Phase 2b/3 top-line data released June 21, 2021. Endpoints met.	0
AMGN	Metastatic castrate-resistant prostate cancer (mCRPC)	Phase 1b	2023-10-31 00:00:00	Phase 1b initial data reported that efficacy was greater at higher doses (doses ≥0.75 mg target dose) where PSA50 was 59% (n=44) and RECIST objective response rate was 41% (n=37). The safety profile was clinically manageable, with CRS that was generally low grade and primarily in cycle 1, noted October 31, 2023.	0
INCY	Atopic dermatitis (AD) in children (age ≥2 to <12 years)	Phase 3	2023-10-13 00:00:00	Phase 3 8- week analysis data shared at EADV in children (age ≥2 to <12 years) with atopic dermatitis (AD) achieved significant efficacy, as defined by the Investigator's Global Assessment-treatment success (IGA-TS), following eight weeks of treatment, noted October 13, 2023.	1
MOR	Relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL)	Phase 3	2019-11-18 00:00:00	Phase 3 futility analysis passed November 18, 2019	0
BLUE	non-β0/β0 transfusion-dependent thalassemia (TDT)	Phase 3	2022-12-12 00:00:00	Phase 3 long term data shared at ASH reported that no hematologic malignancies, insertional oncogenesis, vector-derived replication competent lentivirus, or clonal predominance was observed, noted December 12, 2022.	1
IPHA	Non-small cell lung cancer (NSCLC)	Phase 2	2021-09-17 00:00:00	Phase 2 trial met primary endpoint of confirmed ORR of 30%, and for durvalumab plus monalizumab 36%. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone, September 17, 2021.	0
NVS	Melanoma with BRAF V600E or V600K mutations	Approved	2018-04-30 00:00:00	Approval announced April 30, 2018.	1
ETON	Seizures/migraine	Approved	2021-11-08 00:00:00	FDA Approval November 8, 2021.	1
CYCN	Sickle Cell disease	Phase 2	2020-10-14 00:00:00	Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.	1
RHHBY	1L Non-Small Cell Lung Cancer (NSCLC) PD-L1 high	Phase 3	2023-08-23 00:00:00	Phase 3 data reported that median overall survival estimates of 22.9 months in the tiragolumab plus Tecentriq arm, and 16.7 months in the Tecentriq monotherapy arm, noted August 23, 2023.	0
ARQT	Atopic dermatitis	Phase 3	2023-09-07 00:00:00	OLE data reported that 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively, noted September 7, 2023.	1
BGNE	Hepatocellular Carcinoma (HCC)	Phase 2	2023-10-21 00:00:00	Phase 2 poster presented at ESMO reported that NV-assessed ORR was 35.5% with O+T+B vs. 37.5% with T+B, noted October 21, 2023.	1
CANF	Non-alcoholic steatohepatitis (NASH)	Phase 2	2020-04-07 00:00:00	Phase 2 endpoints met - April 7, 2020.	0
TNXP	fibromyalgia-type Long COVID	Phase 2	2023-09-05 00:00:00	Phase 2 topline data did not achieve the pre-specified primary endpoint of improving Long COVID pain intensity scores at Week 14, noted September 5, 2023.	1
RHHBY	Non-squamous non-small cell lung cancer (NSCLC)	Approved	2018-12-06 00:00:00	FDA Approval announced December 6, 2018.	1
MRK	adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors	Approved	2023-03-29 00:00:00	FDA approval on March 29, 2023.	1
BSGM	COVID-19	Phase 2	2020-10-26 00:00:00	Phase 2 trial halted - October 26, 2020. Unlikely that the trial would meet its primary safety endpoints.	1
SGEN	Multiple myeloma	Approved	2020-08-05 00:00:00	FDA approval granted August 5, 2020.	1
SLRX	Ewing's and other FET-rearranged sarcomas	Phase 1/2	2022-12-01 00:00:00	Phase 1/2 data appear to indicate that first- and second-relapse Ewing sarcoma patients treated with seclidemstat in combination with topotecan and cyclophosphamide who achieve disease control may have an increased time to tumor progression (TTP) compared with treatment of topotecan and cyclophosphamide alone. 60% Confirmed Disease Control Rate1 and 7.4 Months Median Time to Tumor Progression, noted December 1, 2022.	1
AMGN	Secondary hyperparathyroidism (SHPT)	Approved	2017-02-07 00:00:00	Approved February 7, 2017.	1
VRTX	Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation	Approved	2019-10-21 00:00:00	FDA Approval announced October 21, 2019.	1
AMGN	Thyroid Eye Disease (TED) - chronic	Phase 3	2023-04-10 00:00:00	Phase 4 topline data reported that treatment achieved a statistically significant reduction in proptosis from baseline compared to those receiving placebo, noted April 10, 2023.	1
LABP	Ulcerative Colitis	Phase 2	2021-10-04 00:00:00	Phase 2 data displayed clinical remission in 36.1% of the 880 mg group and 35.5% of the 440 mg group during the blinded maintenance phase. Both dose cohorts had 10% of patients report an AE of ulcerative colitis worsening, noted October 4, 2021.	0
ITCI	Heart failure	Phase 1/2	2020-06-29 00:00:00	Phase 1/2 top-line data released June 29, 2020 noted improved cardiac output by increasing heart contractility and decreasing vascular resistance.	1
HRMY	Developmental and Epileptic Encephalopathies (DEE)	Phase 2	2019-09-18 00:00:00	Phase 2 top-line data released September 18, 2019. 10/46 reported a serious adverse event (SAE).	0
TVTX	IgA nephropathy (IgAN)	Phase 3	2023-11-03 00:00:00	Additional Phase 3 data presented at ASN showed one of the slowest rates of kidney function decline in IgAN trials, consistent treatment effects across baseline eGFR and proteinuria, and higher rates of complete remission compared to maximally tolerated dose of irbesartan through 110 weeks of treatment, noted November 3, 2023.	1
IMAB	Ulcerative colitis	Phase 2	2021-04-26 00:00:00	Phase 2 trial met primary endpoint.	0
BMY	Newly Diagnosed Multiple Myeloma	Phase 2a	2022-12-10 00:00:00	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.	1

PLUR

COVID-19 / Acute Respiratory Distress Syndrome (ARDS)

Phase 2

2021-12-27 00:00:00

Phase 2 data did not meet the primary efficacy endpoint of statistically significant improvement of ventilator free days (VFD) at 28 days. Taking into consideration the baseline risk factors of the ARDS patients, no differences in the safety profile were observed between PLX-PAD and placebo.

1

JNJ

Non-small cell lung cancer

Phase 1/2

2022-07-26 00:00:00

Phase 1b/2 data reported an overall response rate (ORR) of 50%, with 15 out of 20 patients remaining on treatment, noted July 26, 2022.

1

GSK

COVID-19 antibody

Approved

2021-05-26 00:00:00

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

1

VACC

Hepatitis B (HBV)

Phase 1/2

2023-06-21 00:00:00

Phase 1b/2a data at EASL showed that all participants who received and experienced a >0.5 log10 reduction in HBsAg had durable responses with reductions in HBsAg persisting through to the last measurement eight months post-final dose, noted June 21, 2023.

1

ARWR

Dyslipidemia

Phase 2b

2022-11-07 00:00:00

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

1

BMY

Progressive pulmonary fibrosis (PPF)

Phase 2

2023-10-24 00:00:00

Breakthrough Therapy Designation (BTD) granted by FDA on October 24, 2023.

0

TBPH

Asthma

Approved

2015-04-30 00:00:00

Approved April 30, 2015.

1

APVO

Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)

Phase 1b

2023-07-18 00:00:00

Phase 1b expansion trial data reported that one patient remained on study with sustained complete response for 8 cycles, noted July 18, 2023.

0

SNDX

Ovarian cancer

Phase 1/2

2019-03-07 00:00:00

Phase 2 top-line data March 7, 2019 failed to meet endpoints.

1

KALV

Hereditary angioedema (HAE)

Phase 1

2023-06-09 00:00:00

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.

1

PHAT

Erosive esophagitis / Gastroesophageal reflux disease (GERD)

Approved

2023-11-01 00:00:00

FDA Approved on November 1, 2023.

1

AXSM

Narcolepsy

Phase 2

2019-12-03 00:00:00

Phase 2 data met primary endpoint.

0

CLVS

Urothelial carcinoma

Phase 2

2019-04-12 00:00:00

Phase 2 trial to be discontinued due to lack of efficacy - April 12, 2019.

-1

NOVN

Fungal infections

Phase 2

2017-04-12 00:00:00

Phase 2 data released April 12, 2017. Primary endpoint met.

0

ENTA

Respiratory Syncytial Virus RSV-A and RSV-B

Phase 1

2023-06-20 00:00:00

Phase 1 data demonstrated favorable safety, tolerability, and PK supportive of once-daily dosing, with good exposure multiples, thereby supporting further clinical advancement of EDP-323, noted June 20, 2023.

1

ACIU

Alzheimer’s disease

Phase 3

2019-01-30 00:00:00

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

-1

BIIB

Lupus

Phase 2b

2018-10-23 00:00:00

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

0

ANAB

Non-squamous non-small cell lung cancer (NSCLC)

Phase 2

2022-10-05 00:00:00

Phase 2 trial met primary endpoint, noted October 5, 2022.

0

CRDF

Pancreatic ductal adenocarcinoma (PDAC)

Phase 2

2023-09-26 00:00:00

Phase 2 additional data reported an objective response rate (ORR) of 19% and median progression-free survival (mPFS) of 5.0 months, noted September 26, 2023.

0

PRAX

Major Depressive Disorder and perimenopausal depression

Phase 2a

2021-08-16 00:00:00

Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes.

0

ABBV

Acute Myeloid Leukemia (AML)

Approved

2020-10-16 00:00:00

FDA approval announced October 16, 2020.

1

MRK

Biliary Tract Cancer

Approved

2023-11-01 00:00:00

Approved November 1, 2023.

1

LLY

Heart failure with preserved ejection fraction (HFpEF)

Phase 3

2019-12-13 00:00:00

Phase 3 data did not meet primary endpoint - December 13, 2019.

1

OTIC

Acute otitis externa

Approved

2018-03-02 00:00:00

Approval announced March 2, 2018.

1

MRTX

Non-Small Cell Lung Cancer (NSCLC)

Phase 2

2023-10-20 00:00:00

Phase 2 data presented at ESMO 2023 reported that in patients with PD-L1 TPS ≥50%, adagrasib + pembrolizumab demonstrated a higher response rate (63%) than expected with pembrolizumab monotherapy (39–45%3,4), noted October 20, 2023.

1

URGN

Low grade non-muscle invasive bladder cancer (LG-NMIBC)

Phase 3

2023-07-27 00:00:00

Phase 3 trial met primary endpoint with 79.2% rate of complete response at 3-months following the initial treatment, noted July 27, 2023.

1

RHHBY

Triple-negative breast cancer

Approved

2019-03-08 00:00:00

FDA Approval announced March 8, 2019.

1

JNJ

High-Risk Non-Muscle Invasive Bladder Cancer (NMIBC)

Phase 2b

2023-10-24 00:00:00

Phase 2b data presented at ESMO reported that centrally confirmed CR was achieved in 23/30 pts, noted October 24, 2023.

1

PSTV

Glioma

Phase 1/2

2023-06-29 00:00:00

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.

1

COLL

Opioid dependence

Approved

2014-06-06 00:00:00

Approved June 6, 2014.

1

AMGN

Rheumatoid arthritis (RA)

Approved

2022-06-06 00:00:00

Approved June 6, 2022.

1

MYOV

Uterine fibroids

Approved

2021-05-26 00:00:00

FDA approval announced May 26, 2021.

1

BIIB

Cognitive Impairment Associated With Schizophrenia (CIAS)

Phase 2b

2022-07-20 00:00:00

Phase 2b trial did not meet its primary endpoint, and program will be discontinued, noted July 20, 2022.

1

ASRT

Neutropenia

Approved

2022-09-09 00:00:00

Approved September 9, 2022.

1

REGN

Lymphoma

Phase 1/2

2022-05-26 00:00:00

Phase 1/2 data released in an abstract at ASCO reported no significant adverse effects (AEs) and clinical activity in 5 of 7 patients, noted May 26, 2022.

0

LLY

HR+, HER2-locoregionally recurrent or metastatic breast cancer (MBC)

Approved

2018-03-26 00:00:00

Approval announced February 26, 2018.

1

ARGX

Chronic inflammatory demyelinating polyneuropathy (CIDP)

Phase 2

2023-07-17 00:00:00

Phase 2 trial met primary endpoints, noted July 17, 2023.

0

EQRX

CNS metastases and EGFR-mutated non-small cell lung cancer (NSCLC)

Phase 3

2022-06-06 00:00:00

Phase 3 data reported that CNS PFS events were observed in 11 patients (38%) treated, and the confirmed CNS ORR were 82.8% and 75.0% in patients treated with Au and G, respectively, noted June 6, 2022.

1

GNFT

Non-alcoholic steatohepatitis (NASH)

Phase 3

2020-05-11 00:00:00

Phase 3 data did not meet primary endpoint - May 11, 2020.

1

INKT

Solid Tumors

Phase 1

2023-11-03 00:00:00

Phase 1 trial of agenT-797 alone or in combination showed durable clinical benefit, including: Clinical response in MSI-high 3L gastric cancer patient after failure on pembrolizumab and nivolumab/FOLFOX. Long-term disease stabilization (n=10), including in testicular cancer (SD 9 months) and anti-PD-1 relapsed/refractory non-small-cell lung cancer (SD > 10 months). Tolerable safety profile with no dose-limiting toxicities and no grade >3 neurotoxicity or cytokine release syndrome, noted November 3, 2023.

1

MNKKQ

Hemostasis

Approved

2015-04-30 00:00:00

Approved April 30, 2015.

1

ETNB

Severe hypertriglyceridemia

Phase 2

2023-06-24 00:00:00

Phase 2 data presented at EASL showed consistent and significant reductions in triglycerides, atherogenic lipoproteins and liver fat, and pegozafermin's favorable safety and tolerability profile, noted June 24, 2023.

1

CCCC

Synovial sarcoma

Phase 1

2023-11-01 00:00:00

Phase 1 deprioritized due to insufficient single agent efficacy, despite high levels of BRD9 degradation, noted November 1, 2023.

-1

SNY

Acquired thrombotic thrombocytopenic purpura (aTTP)

Approved

2019-02-06 00:00:00

FDA Approval announced February 6, 2019.

1

CCCC

Non-Small Cell Lung Cancer (NSCLC)

Phase 1a

2023-07-05 00:00:00

IND Cleared by FDA, July 5, 2023.

0

TAK

Cytomegalovirus (CMV)

Approved

2021-11-23 00:00:00

Approved November 23, 2021.

1

XERS

Hypoglycemia

Approved

2021-08-23 00:00:00

FDA approval August 23, 2021.

1

MRK

Cytomegalovirus (CMV) Infection

Approved

2017-11-09 00:00:00

Approval announced November 9, 2017.

1

PPBT

Squamous cell carcinoma of the head & neck (SCCHN)

Phase 1/2

2023-10-03 00:00:00

Phase 1/2 data showed that 2 out of 4 SCCHN patients at the highest dose cohort demonstrated a confirmed partial response, noted October 3, 2023.

1

REGN

Stage II to IV Cutaneous Squamous Cell Carcinoma (CSCC)

Phase 2

2023-10-16 00:00:00

Phase 2 data from ESMO reported a median duration of follow-up was 18.7 months, and 1 of 10 pts with MPR experienced recurrence, noted October 16, 2023.

0

JAZZ

Spasticity due multiple sclerosis

Phase 2

2017-02-07 00:00:00

Phase 2 trial did not meet primary endpoint - noted February 7, 2017.

1

HCM

Myeloid hematological malignancies (HMs)

Phase 1

2023-06-09 00:00:00

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

0

HCM

Follicular Lymphoma and Marginal Zone Lymphoma

Phase 2

2023-06-09 00:00:00

Phase 2 data from ICML presentation noted that median duration of response (DoR) and progression free survival (PFS) at median follow-up duration of 22.1 months was not reached, noted June 9, 2023.

1

OMER

IgA nephropathy

Phase 3

2023-10-16 00:00:00

Phase 3 9-month data did not meet its primary endpoint, as a result Omeros will not submit an application for approval of narsoplimab in this indication and will discontinue the ARTEMIS-IGAN clinical trial, noted October 16, 2023.

1

PCRX

Postsurgical pain

Approved

2011-10-31 00:00:00

Approved October 31, 2011.

1

MDWD

Severe burns

CRL

2021-06-29 00:00:00

CRL announced June 29, 2021.

0

MTEM

Solid tumors

Phase 1

2023-11-02 00:00:00

Phase 1 dosing initiated, noted November 2, 2023.

0

ENDP

Adhesive capsulitis (AC) of the shoulder

Phase 2

2022-07-01 00:00:00

Phase 2 trial did not report a significant difference between treatment and placebo, noted July 1, 2022.

1

ANNX

Huntington’s disease (HD)

Phase 2

2022-06-07 00:00:00

Phase 2 data reported that treatment led to complete and durable target engagement, disease progression was stabilized in the overall HD patient population for the entire nine months, and three trial discontinuations were due to treatment, noted June 7, 2022.

1

ATNXQ

Actinic keratosis

Approved

2020-12-15 00:00:00

FDA approval announced December 15, 2020.

1

BIIB

Lupus

Phase 2

2019-12-03 00:00:00

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

0

FGEN

Anemia in patients receiving concurrent chemotherapy treatment for non-myeloid malignancie

Phase 3

2023-05-18 00:00:00

Phase 3 trial met primary endpoint, noted May 18, 2023.

0

DCPH

Gastrointestinal Stromal Tumors (GIST) - second-line

Phase 3

2023-06-01 00:00:00

Phase 3 data reported that the mPFS (90% CI) by IRR was 6.44 (2.89, 8.31) months and ORR was 21.1%, noted June 1, 2023.

0

MRK

Beta-thalassemia

Approved

2019-11-08 00:00:00

FDA Approval announced November 8, 2019.

1

TBPH

Ulcerative Colitis

Phase 2b

2021-08-23 00:00:00

Phase 2b data did not meet primary endpoint - August 23, 2021.

1

RHHBY

Urothelial carcinoma

Phase 3

2019-08-05 00:00:00

Phase 3 PFS co-primary endpoint met - August 5, 2019.

0

LOGC

Methylmalonic Acidemia (MMA)

Phase 1/2

2022-02-02 00:00:00

Phase 1/2 trial placed on clinical hold due to 2 drug related SAEs, noted February 2, 2022.

0

IMMP

Solid tumors

Phase 1

2023-10-24 00:00:00

Phase 1 additional efficacy and tolerability data reported at ESMO showed strong 71.4% Overall Response Rate, 90.5% Disease Control Rate, 10.1-month median Progression Free Survival (PFS), and median Overall Survival (OS) that has not been reached, despite 81% of patients having low or negative PD-L1 expression, noted October 24, 2023.

1

AZN

First-line ovarian cancer following platinum-based chemotherapy

Approved

2018-12-19 00:00:00

FDA Approval announced December 19, 2018.

1

IOVA

Non-small cell lung cancer (NSCLC)

Phase 2

2021-11-12 00:00:00

Phase 2 data results showed a 21.4% overall response rate (ORR) in heavily pre-treated patients (n=28), including one complete response and five partial responses, noted November 12, 2021.

1

NBRV

Moderate to severe Community Acquired Bacterial Pneumonia (CABP)

Approved

2019-08-19 00:00:00

FDA approval announced August 19, 2019.

1

JNJ

Pediatric Chronic Graft-Versus-Host Disease

Approved

2022-08-24 00:00:00

Approved August 24, 2022.

1

TARS

Demodex Blepharitis

Phase 2/3

2021-06-21 00:00:00

Phase 2b/3 top-line data released June 21, 2021. Endpoints met.

0

AMGN

Metastatic castrate-resistant prostate cancer (mCRPC)

Phase 1b

2023-10-31 00:00:00

Phase 1b initial data reported that efficacy was greater at higher doses (doses ≥0.75 mg target dose) where PSA50 was 59% (n=44) and RECIST objective response rate was 41% (n=37). The safety profile was clinically manageable, with CRS that was generally low grade and primarily in cycle 1, noted October 31, 2023.

0

INCY

Atopic dermatitis (AD) in children (age ≥2 to <12 years)

Phase 3

2023-10-13 00:00:00

Phase 3 8- week analysis data shared at EADV in children (age ≥2 to <12 years) with atopic dermatitis (AD) achieved significant efficacy, as defined by the Investigator's Global Assessment-treatment success (IGA-TS), following eight weeks of treatment, noted October 13, 2023.

1

MOR

Relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL)

Phase 3

2019-11-18 00:00:00

Phase 3 futility analysis passed November 18, 2019

0

BLUE

non-β0/β0 transfusion-dependent thalassemia (TDT)

Phase 3

2022-12-12 00:00:00

Phase 3 long term data shared at ASH reported that no hematologic malignancies, insertional oncogenesis, vector-derived replication competent lentivirus, or clonal predominance was observed, noted December 12, 2022.

1

IPHA

Non-small cell lung cancer (NSCLC)

Phase 2

2021-09-17 00:00:00

Phase 2 trial met primary endpoint of confirmed ORR of 30%, and for durvalumab plus monalizumab 36%. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone, September 17, 2021.

0

NVS

Melanoma with BRAF V600E or V600K mutations

Approved

2018-04-30 00:00:00

Approval announced April 30, 2018.

1

ETON

Seizures/migraine

Approved

2021-11-08 00:00:00

FDA Approval November 8, 2021.

1

CYCN

Sickle Cell disease

Phase 2

2020-10-14 00:00:00

Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.

1

RHHBY

1L Non-Small Cell Lung Cancer (NSCLC) PD-L1 high

Phase 3

2023-08-23 00:00:00

Phase 3 data reported that median overall survival estimates of 22.9 months in the tiragolumab plus Tecentriq arm, and 16.7 months in the Tecentriq monotherapy arm, noted August 23, 2023.

0

ARQT

Atopic dermatitis

Phase 3

2023-09-07 00:00:00

OLE data reported that 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively, noted September 7, 2023.

1

BGNE

Hepatocellular Carcinoma (HCC)

Phase 2

2023-10-21 00:00:00

Phase 2 poster presented at ESMO reported that NV-assessed ORR was 35.5% with O+T+B vs. 37.5% with T+B, noted October 21, 2023.

1

CANF

Non-alcoholic steatohepatitis (NASH)

Phase 2

2020-04-07 00:00:00

Phase 2 endpoints met - April 7, 2020.

0

TNXP

fibromyalgia-type Long COVID

Phase 2

2023-09-05 00:00:00

Phase 2 topline data did not achieve the pre-specified primary endpoint of improving Long COVID pain intensity scores at Week 14, noted September 5, 2023.

1

RHHBY

Non-squamous non-small cell lung cancer (NSCLC)

Approved

2018-12-06 00:00:00

FDA Approval announced December 6, 2018.

1

MRK

adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors

Approved

2023-03-29 00:00:00

FDA approval on March 29, 2023.

1

BSGM

COVID-19

Phase 2

2020-10-26 00:00:00

Phase 2 trial halted - October 26, 2020. Unlikely that the trial would meet its primary safety endpoints.

1

SGEN

Multiple myeloma

Approved

2020-08-05 00:00:00

FDA approval granted August 5, 2020.

1

SLRX

Ewing's and other FET-rearranged sarcomas

Phase 1/2

2022-12-01 00:00:00

Phase 1/2 data appear to indicate that first- and second-relapse Ewing sarcoma patients treated with seclidemstat in combination with topotecan and cyclophosphamide who achieve disease control may have an increased time to tumor progression (TTP) compared with treatment of topotecan and cyclophosphamide alone. 60% Confirmed Disease Control Rate1 and 7.4 Months Median Time to Tumor Progression, noted December 1, 2022.

1

AMGN

Secondary hyperparathyroidism (SHPT)

Approved

2017-02-07 00:00:00

Approved February 7, 2017.

1

VRTX

Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation

Approved

2019-10-21 00:00:00

FDA Approval announced October 21, 2019.

1

AMGN

Thyroid Eye Disease (TED) - chronic

Phase 3

2023-04-10 00:00:00

Phase 4 topline data reported that treatment achieved a statistically significant reduction in proptosis from baseline compared to those receiving placebo, noted April 10, 2023.

1

LABP

Ulcerative Colitis

Phase 2

2021-10-04 00:00:00

Phase 2 data displayed clinical remission in 36.1% of the 880 mg group and 35.5% of the 440 mg group during the blinded maintenance phase. Both dose cohorts had 10% of patients report an AE of ulcerative colitis worsening, noted October 4, 2021.

0

ITCI

Heart failure

Phase 1/2

2020-06-29 00:00:00

Phase 1/2 top-line data released June 29, 2020 noted improved cardiac output by increasing heart contractility and decreasing vascular resistance.

1

HRMY

Developmental and Epileptic Encephalopathies (DEE)

Phase 2

2019-09-18 00:00:00

Phase 2 top-line data released September 18, 2019. 10/46 reported a serious adverse event (SAE).

0

TVTX

IgA nephropathy (IgAN)

Phase 3

2023-11-03 00:00:00

Additional Phase 3 data presented at ASN showed one of the slowest rates of kidney function decline in IgAN trials, consistent treatment effects across baseline eGFR and proteinuria, and higher rates of complete remission compared to maximally tolerated dose of irbesartan through 110 weeks of treatment, noted November 3, 2023.

1

IMAB

Ulcerative colitis

Phase 2

2021-04-26 00:00:00

Phase 2 trial met primary endpoint.

0

BMY

Newly Diagnosed Multiple Myeloma

Phase 2a

2022-12-10 00:00:00

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.

1