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963
label
int64
4
6
PFE
Migraine - preventative
Approved
5/27/21
FDA approval announced May 27, 2021.
6
LLY
First-Line Nonsquamous Non-small cell lung cancer (NSCLC)
Approved
6/5/18
FDA approval (label expansion) announced June 5, 2018.
6
NVS
Acute heart failure
Phase 3
3/22/17
Phase 3 data released March 22, 2017 - primary endpoints not met.
4
LLY
Type 2 Diabetes
Phase 3
12/9/20
Phase 3 data released December 9, 2020 - significantly reduced A1C and body weight.
6
GSK
Lupus Nephritis
Approved
12/17/20
FDA approval announced December 17, 2020.
6
VALN
Chikungunya vaccine
Phase 3
12/5/22
Phase 3 trial met primary endpoint, noted December 5, 2022.
5
GSK
Severe hypereosinophilic syndrome (HES)
Approved
9/25/20
FDA approval announced September 25, 2020.
6
SNDX
Chronic Graft versus host disease (cGvHD)
Phase 2
7/24/23
Phase 2 trial results met its primary endpoint across all cohorts with an overall response rate (ORR) of 74% at a dose of 0.3 mg/kg administered every two weeks, noted July 24, 2023.
5
AVDL
Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery
Approved
6/3/13
Approved June 3, 2013.
6
AMGN
Asthma
Approved
12/17/21
Approved December 17, 2021.
6
NAVB
Rheumatoid arthritis
Phase 2b
9/15/22
Phase 2b data reported that the quantitative TIL uptake in the hands and wrists of patients is proportional to the amount of macrophage involvement in an individual RA patient's joint inflammation, noted September 15, 2022.
5
OPK
Children - growth hormone deficiency
Approved
6/28/23
FDA Approved on June 28, 2023.
6
QLGN
Lactose intolerance
Phase 3
9/12/19
Phase 3 data failed to meet primary endpoint.
4
INVA
cIAI (complicated intra-abdominal infections)
Approved
8/27/18
FDA approval announced August 27, 2018.
6
EDIT
Sickle Cell Disease
Phase 1/2
6/9/23
Phase 1/2 data presented at EHA reported that all five patients treated with EDIT-301 successfully engrafted and all four RUBY patients treated are free of vaso-occlusive events since infusion, noted June 9, 2023.
6
SNY
Asthma
Approved
10/19/18
FDA approval announced October 19, 2018.
6
CANF
Rheumatoid arthritis
Phase 2
10/6/20
Phase 3 interim analysis recommended trial to be discontinued - October 6, 2020.
4
AVDL
Hypotension
Approved
5/2/16
Approved May 2, 2016.
6
PFE
First-Line Treatment of Philadelphia Chromosome Positive Chronic Myeloid Leukemia
Approved
12/19/17
Approval announced December 19, 2017.
6
ALDX
Proliferative vitreoretinopathy (PVR)
CRL
6/21/23
CRL issued on June 21, 2023. Aldeyra plans to have a Type-C meeting on 2H 2023.
6
BLUE
Relapsed/refractory multiple myeloma
Approved
3/26/21
FDA approval announced March 26, 2021.
6
ZVRA
Niemann-Pick disease Type C (NPC)
CRL
6/18/21
CRL issued June 18, 2021.
5
SNY
Chronic spontaneous urticaria (CSU)
CRL
10/20/23
CRL issued by the FDA, noted October 20, 2023.
5
BGNE
Esophageal Squamous Cell Carcinoma (ESCC)
Phase 3
6/4/21
Phase 3 poster at ASCO June 4, 2021 noted median OS of 8.6 months compared to 6.3 months in the chemotherapy arm.
6
IMVT
Graves Ophthalmopathy
Phase 2a
3/30/20
Phase 2a initial data March 30, 2020 noted 65% mean reduction in total IgG.
6
TYME
Prostate cancer
Phase 2
2/14/19
Phase 2 updated data February 14, 2019. Median PSA doubling-time improved by 34%.
6
PTGX
Plaque psoriasis
Phase 2b
7/5/23
Phase 2b trial met all primary and secondary efficacy endpoints, noted July 5, 2023.
5
TCON
Mesothelioma cancer
Phase 2
10/7/21
Phase 2 data reported that 9 patients reported stable disease as best response with Grade 3 TRAEs including lymphopenia (4) and anemia (1), noted October 7, 2021.
5
ENTA
Hepatitis B
Phase 1
11/18/21
Development discontinued, noted November 18, 2021.
4
PDSB
Head and neck cancer
Phase 2
10/23/23
Phase 2 data presented at ESMO reported that the combination of PDS0101 and KEYTRUDA resulted in increased polyfunctionality reflected in T cells expressing 5 or more cytokines, noted October 23, 2023.
6
APM
Neuroblastoma
Phase 1/2
6/22/23
Phase 1b/2a trial has been repurposed for small molecule drug , noted June 22, 2023.
5
ADAG
Solid tumors
Phase 1/2
4/18/23
Dose escalation data presented at AACR showed a compelling safety profile for ADG126, with no Grade 3 or higher TRAEs reported at repeat doses up to 20 mg/kg. Across all dose levels, the disease control rate was 37% among 27 evaluable patients, noted April 18, 2023.
6
SNY
Pompe disease
Approved
8/6/21
FDA approval announced August 6, 2021.
6
RANI
Rheumatoid arthritis
Phase 1
11/1/23
Program paused, noted November 1, 2023.
4
QURE
Hemophilia B
Approved
11/22/22
Approved November 22, 2022.
6
LRMR
Friedreich’s ataxia (FA)
Phase 2
7/25/23
FDA Phase 2 clearance for dose exploration trial and OLE trial, Initiation of open-label extension trial with 25 mg daily dosing expected in 1Q 2024, noted July 25, 2023. Phase 2 50mg cohort topline data expected 1H 2024. Phase 2 interim data expected 4Q 2024.
5
ADCT
Hodgkin lymphoma (HL)
Phase 2
6/22/21
Phase 2 interim data presented June 22, 2021. Overall response rate (ORR) was 66.3% (67/101 patients) with a complete response rate (CRR) of 27.7% and partial response rate (PRR) of 38.6%. Median duration of response has not been reached.
4
LIAN
Demodex blepharitis
Phase 3
10/30/23
Phase 3 results demonstrated statistically significant mite eradication in patients treated compared to vehicle, noted October 30, 2023.
6
PFE
Glioblastoma (GBM)
Phase 2/3
10/31/23
Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.
5
MRK
Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia (HABP/VABP)
Approved
6/4/20
FDA approval announced June 4, 2020.
6
NVS
Metastatic castration-resistant prostate cancer (mCRPC)
Phase 3
10/23/23
Phase 3 trial met its primary endpoint with a clinically meaningful and statistically significant benefit in radiographic progression-free survival, noted October 23, 2023.
5
HRTX
Postoperative nausea and vomiting (PONV)
Approved
9/16/22
Approved September 16, 2022.
6
INAB
Glioblastoma
Phase 2
6/5/23
Data presented at ASCO 2023 showed that 100% of treated patients to date have exceeded historical median progression-free survival, with two patients that received three doses remaining progression-free at 23.5 and 19.4 months, respectively noted on June 5, 2023.
6
AMGN
Secondary hyperparathyroidism (SHPT)
Approved
2/7/17
Approved February 7, 2017.
6
UTHR
Pulmonary Arterial Hypertension (PAH)
CRL
4/28/20
CRL announced April 29, 2020.
5
PFE
Colorectal cancer and gastrointestinal (GI) cancer
Phase 1/2
6/5/23
Initial Phase 1b/2 data showed a 50% response rate, including two confirmed partial responses (cPR) and one unconfirmed partial response (uPR), and 67% disease control rate in EC-naïve response evaluable patients at the highest dose of ERAS-007 tested (100 mg BID-QW), development deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023
6
AZN
Lung cancer
Phase 3
8/21/19
Phase 3 data released August 21, 2019 did not meet primary endpoint.
4
BMY
Ulcerative Colitis
Phase 2
10/7/21
Phase 2 trial did not meet primary or secondary endpoints, October 7, 2021.
4
ORGS
Acute pancreatitis
Approved
8/22/18
Approved August 22, 2018.
6
BMY
Myelofibrosis
Phase 2
12/9/19
Phase 2 data at ASH December 2019 noted primary endpoint met in 10%~32% of patients across four cohorts.
4
RARE
Angelman Syndrome
Phase 1/2
7/18/22
Phase 1/2 UK data reported that treatment displayed a meaningful improvement in clinical disease and an acceptable safety profile, noted July 18, 2022.
6
ADPT
Relapsed/Refractory Multiple Myeloma
Phase 1/2
12/12/22
Phase 1/2 final data reported that treatment significantly improved the degranulation of NK cells after 4-hour stimulation with K562 or PMA/Ionomycin, noted December 12, 2022.
6
MNOV
Idiopathic pulmonary fibrosis (IPF)
Phase 2
8/12/21
Phase 2 data released August 12, 2021 - no clinically meaningful trends.
4
GSK
Multiple Myeloma
Phase 1/2
6/9/23
Phase 1/2 data presented at EHA Data from 34 patients with low-dose and 37 patients with monotherapy are presented. Patients had a median (range) age of 68 (48–81) years and a median (range) of 5 (3–14) prior lines of therapy. As of the data cutoff (Dec 9, 2022), patients received a median (range) of 4 (1–20) cycles of the combination and 3 (1–9) monotherapy cycles. For the combination and monotherapy arms, respectively, ORR was 29% (95% CI 15.1, 47.5) and 38% (22.5, 55.2) (Table). Incidence of Grade ≥3 adverse events was 76% and 65%, noted June 9, 2023.
6
PRAX
Major Depressive Disorder
Phase 2/3
6/6/22
Phase 2/3 data did not achieve statistical significance on the primary endpoint.
6
EIGR
Hyperinsulinemic hypoglycemia after bariatric or other gastrointestinal surgeries
Phase 2b
6/13/22
Phase 2b data reported that trial met primary and secondary endpoints, noted June 13, 2022.
5
PFE
Diffuse Large B-cell Lymphoma (DLBCL), Cutaneous T-cell lymphoma (CTCL)
Phase 1
12/11/17
Phase 1a/b updated data released at ASH 2017 - 5/18 objective response rate.
4
INCY
Triple-negative breast cancer
Phase 2
12/10/21
Phase 2 data reported that treatment is tolerated, noted December 10, 2021.
6
VRNA
COPD
Phase 2
1/14/19
Phase 2 trial did not meet primary endpoint - January 13, 2019.
4
COCP
Hepatitis C
Phase 2a
1/22/19
Phase 2a initial data released January 22, 2019. SVR12 66%.
5
HEPA
Non-alcoholic steatohepatitis
Phase 2b
6/15/23
Phase 2b Data and Safety Monitoring Board (DSMB) review and issued a "study may proceed without modification" clearance, study expected to be completed by 1Q 2024, noted June 15, 2023.
5
IMMP
Melanoma
Phase 1
5/30/18
Phase 1 data released May 30, 2018 showed ORR of 61% (11/18 patients).
6
PTGX
Beta-thalassemia
Phase 2
10/11/21
Phase 2 clinical hold lifted October 11, 2021.
5
VALN
Chikungunya Vaccine
Phase 3
12/21/21
Phase 3 trial met its primary endpoint, demonstrating that three consecutively manufactured vaccine lots elicited equivalent immune responses measured by neutralizing antibody titer GMT ratios on Day 29 after vaccination.
6
TNXP
Fibromyalgia
Phase 3
6/15/23
Phase 3 met the primary endpoint on its RELIEF trial, noted June 15, 2023
5
TAK
Thyroid Carcinoma
Phase 3
9/20/21
Phase 3 data showed a median progression-free survival was 11.0 months for CABOMETYX compared with 1.9 months for placebo. Objective response rate (ORR) favored CABOMETYX at 11%, including one complete response, versus 0% for placebo. Median overall survival was 19.4 months, September 20, 2021.
6
ITCI
Bipolar depression
Phase 3
7/8/19
Phase 3 trial did not meet primary endpoint - July 8, 2019.
4
OBSV
Uterine fibroids
Phase 3
10/20/21
Phase 3 data reported once daily treatment without ABT reduced uterine volume by 39% after 24 weeks. However, co-administration of hormonal ABT after 24 weeks counteracted the uterine volume reducing effects at 52 weeks, noted October 20, 2021.
5
ESPR
Cardiovascular Diseases
Phase 3
8/26/23
Phase 3 total event data presented at ESC reported that bempedoic acid shows 20% risk reduction in MACE-4 and 17% risk reduction in MACE-3, noted August 26, 2023.
6
GRTS
COVID-19 vaccine
Phase 1
10/11/23
Data presented at IDweek reported that treatment was well tolerated in both HIV-negative participants and PLWH irrespective of SARS-CoV-2 serostatus at baseline, October 11, 2023.
6
MNKD
Pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease (PH-ILD)
Approved
5/24/22
Approved May 24, 2022.
6
AIM
Ovarian cancer
Phase 2
3/9/22
Phase 2 data reported 2 complete responses, 3 partial responses, 3 stable diseases, 5 progressive diseases and a total clinical benefit rate of 61.6%, noted March 9, 2022.
5
PFE
Spinocerebellar Ataxia (SCA)
Phase 3
7/27/23
The FDA informed Biohaven that it would not review the recently submitted NDA application for troriluzole given that the study's primary endpoint was not met and thus, would not permit a substantive review. The communication from the FDA indicated that the Company may request a Type A meeting within 30 days, noted July 27, 2023.
6
CKPT
Cutaneous squamous cell carcinoma (CSCC)
BLA Filing
1/4/23
Phase 1 pivotal top-line data reported that the trial met the primary endpoint, noted January 25, 2022. Phase 1 interim efficacy results reported an objective response rate of 54.8%, noted June 16, 2022. BLA submitted to FDA, noted January 4, 2022.
5
MRK
Renal Cell Carcinoma (RCC) - cancer
Phase 2
6/12/17
Phase 2 data released June 12, 2017 - primary endpoint not met.
4
RIGL
COVID-19
Phase 2/3
10/11/23
Phase 2/3 trial enrollment ceased based on the recommendations of DSMB, noting that here was an extremely low likelihood of fostamatinib providing benefits related to the primary outcome, noted October 11, 2023.
5
NBRV
Complicated urinary tract infections (cUTI)
CRL
6/19/20
CRL issued April 30, 2019. Further CRL received June 19, 2020.
5
AMGN
Neuromyelitis Optica Spectrum Disorder
Approved
6/11/20
FDA Approval announced June 11, 2020.
6
MOR
Rheumatoid Arthritis
Phase 2b
10/21/18
Phase 2b released October 21, 2018 did not meet primary endpoint.
4
IMRA
Sickle cell disease
Phase 2a
12/13/21
Phase 2a 12-month OLE data showed that dose was generally well-tolerated as a monotherapy as well as in combination with hydroxyurea. There were no clinically-significant changes in lab safety data, ECGs or vital signs, and no patients have discontinued the study due to adverse events
5
PRTA
Previously-treated patients with AL amyloidosis and persistent cardiac dysfunction
Phase 2b
4/23/18
Phase 2b trial did not meet primary endpoint - April 23, 2018.
4
MITO
Barth syndrome
NDA Filing
10/20/21
Refusal to File letter from FDA on October 20, 2021, due to issues with Phase 2 trial and open label extension.
4
PCRX
Nerve blocker in lower extremity surgeries
Phase 3
9/6/22
Phase 3 trial met primary endpoint, noted September 7, 2022.
5
SGEN
HER2+ Metastatic Breast Cancer (MBC)
Phase 3
8/19/23
Phase 3 trial met primary endpoint, noted August 19, 2023
5
QNCX
Alzheimer’s disease
Phase 2/3
11/11/21
Phase 2/3 data reported that the 40 mg demonstrated equivalent or better efficacy compared 80 mg, as well as a superior safety profile. Benefits were shown in ADAS-Cog11, CDR-SB, MMSE, and NPI, with increasing separation from placebo throughout the study consistent with disease modification. Benefits were not seen on ADCS-ADL at either dose, noted November 11, 2021.
6
LLY
Early Alzheimer's disease
Phase 3
6/11/18
Announced discontinuation of trial due to futility.
4
NVS
Atherosclerotic cardiovascular disease (ASCVD), increased risk of ASCVD or heterozygous familial hypercholesterolemia (HeFH)
Phase 3
8/28/23
Phase 3 data reported that eight in ten patients achieved target LDL-C threshold, noted August 28, 2023.
6
BPMC
EGFR exon 20 insertion-positive NSCLC
Phase 1/2
6/5/23
Phase 1/2 initial clinical data presented at ASCO reported a partial CNS response and a CNS complete response, noted June 5, 2023.
6
OCGN
Retinitis Pigmentosa
Phase 1/2
9/13/23
Phase 1/2 clinical study update suggested continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline, noted September 13, 2023.
6
PRQR
Retinitis pigmentosa
Phase 1/2
11/18/21
Additional Phase 1b/2 data reported a mean benefit of 13 letters after a single injection at 72 weeks, noted November 18, 2021
5
PFE
Bosimilar to Humira (adalimumab)
Approved
11/18/19
FDA Approval announced November 18, 2019.
6
CNSP
Glioblastoma (GBM) (adult)
Phase 2
8/14/23
Phase 2 interim update demonstrated its capability to be an innovative treatment in GBM that is safe and well tolerated, which has the potential to be a novel and effective therapy for this disease, noted August 14, 2023.
6
DYN
Myotonic Dystrophy Type 1 (DM1)
Phase 1/2
9/20/23
Phase 1/2 initiated Septmember 6, 2022. Phase 1/2 Orphan Drug Designation was granted on September 20, 2023.
5
TAK
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
NDA Filing
6/20/23
Phase 3 full result findings showed a clinically significant reduction in relapse rate as well as a delayed time to relapse compared to placebo when used as a maintenance therapy, noted June 20, 2023. NDA under current review by the FDA.
6
RHHBY
Non-squamous non-small cell lung cancer (NSCLC)
Approved
12/6/18
FDA Approval announced December 6, 2018.
6
VIR
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
XENE
Adult Focal Epilepsy
Phase 2b
12/4/22
Additional Phase 2b data reported that seizure freedom for ≥6-month and ≥12-month consecutive durations was achieved in 17.5% and 10.5% of patients, respectively, noted December 4, 2022.
6
ADMP
Anaphylaxis
Approved
9/27/18
FDA Approval announced September 27, 2018.
6
KNSA
Pericarditis
Approved
3/18/21
FDA approval announced March 18, 2021.
6
ABBV
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
Approved
6/11/18
Approval announced June 11, 2018.
6