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int64
-1
1
EIGR
Hutchinson-Gilford Progeria Syndrome (HGPS)
Approved
2020-11-20 00:00:00
FDA approval announced November 20, 2020.
1
GILD
Psoriatic arthritis
Phase 2
2018-05-30 00:00:00
Phase 2 data released May 30, 2018 - primary endpoint met.
0
AGEN
Solid cancers
Phase 1
2018-06-04 00:00:00
Phase 1 presentation at ASCO 2018. 31% ORR.
1
GSK
Anaemia in chronic kidney disease for patients on dialysis
Approved
2023-02-01 00:00:00
Approved February 1, 2023.
1
ITCI
Agitation in patients with dementia
Phase 2
2018-12-18 00:00:00
Phase 3 interim analysis December 18, 2018 noted trial to stop due to futility.
-1
ABBV
Multiple myeloma
Phase 3
2021-12-13 00:00:00
Phase 3 overall survival results reported that there were 78 (40%) deaths in the treatment arm versus 36 (37%). Median OS was not reached in the treatment or placebo arm among all patients, noted December 13, 2021.
1
HGEN
Chronic myelomonocytic leukemia (CMML)
Phase 2
2023-04-14 00:00:00
Phase 2 preliminary study results reported that 6 subjects demonstrated clinical benefit, with ten grade 3/4 Serious Adverse Events, noted April 14, 2023.
1
SGEN
Frontline CD30-positive mature T-cell lymphomas - cancer
Approved
2018-11-16 00:00:00
FDA Approval announced November 16, 2018.
1
ASRT
Postherpetic neuralgia - shingles
Approved
2011-01-28 00:00:00
Approved January 28, 2011.
1
ITCI
Schizophrenia
Approved
2019-12-23 00:00:00
FDA Approval announced December 23, 2019.
1
RHHBY
Triple-negative breast cancer (TNBC)
Phase 3
2020-08-06 00:00:00
Phase 3 trial did not meet primary endpoint - October 6, 2020.
1
SQZ
Solid Tumors
Phase 1/2
2021-12-09 00:00:00
Phase 1/2 monotherapy data reported that of the five patients in this cohort, one patient showed a radiographic response and symptomatic improvement and reported a complete response, noted December 9, 2021.
1
AMGN
Rheumatoid Arthritis
Phase 2
2022-11-14 00:00:00
Phase 2 data reported that DAZ reduced DAS28-CRP and RF significantly vs. PBO at d113 in all dose regimens, noted November 14, 2022.
1
INTS
Solid tumors
Phase 1/2
2022-11-17 00:00:00
Phase 1/2 data reported at CTOS reported that patients who had 40% or more of their tumor burden treated with INT230-6, an exploratory analysis showed that overall survival was improved when compared to historical benchmarks, noted November 17, 2022.
1
MRK
Solid tumors
Phase 1/2
2023-08-09 00:00:00
Phase 1/2 sixth dose escalation cohort data and agent expansion data to showed durable anti-cancer activity observed during monotherapy dose escalation without dose-limiting toxicities. Late-stage pancreatic cancer patient achieved 80% tumor shrinkage with complete regression observed in two of three metastatic lesions in the liver in response to single-agent MDNA11, noted August 9, 2023.
1
SRPT
Duchenne muscular dystrophy
Approved
2021-02-25 00:00:00
FDA approval announced February 25, 2021.
1
MEIP
B-Cell Malignancies or Acute Myeloid Leukemia (AML)
Phase 1
2023-11-02 00:00:00
Phase 1 data from ASH abstract reported that at 200 mg, 5 of 10 AML pts (50%) had stable disease, noted November 2, 2023.
0
RHHBY
Neovascular Age-Related Macular Degeneration
Approved
2021-10-22 00:00:00
Approved October 22, 2021.
1
BLUE
β-Thalassemia
Approved
2022-08-17 00:00:00
Approved August 17, 2022.
1
MRK
Pneumococcal Vaccine in children
Approved
2022-06-22 00:00:00
Approved June 22, 2022.
1
INSM
Bronchiectasis
Phase 2
2020-02-03 00:00:00
Phase 2 trial met primary endpoint - February 3, 2020.
0
PFE
Diffuse Large B-cell Lymphoma (DLBCL), Cutaneous T-cell lymphoma (CTCL)
Phase 1
2017-12-11 00:00:00
Phase 1a/b updated data released at ASH 2017 - 5/18 objective response rate.
0
MRNS
Postpartum depression
Phase 2
2019-07-23 00:00:00
Phase 2 negative data released July 23, 2019.
0
ASMB
Hepatitis B virus (HBV)
Phase 2
2021-11-12 00:00:00
Phase 2 data reported that treatment led to a deeper level of viral suppression as measured by pgRNA and HBV DNA levels, noted November 12, 2021.
1
XERS
Endogenous Cushing’s syndrome
Approved
2021-12-30 00:00:00
Approved December 30, 2021.
1
MDWD
Basal Cell Carcinoma (BCC)
Phase 1/2
2023-07-10 00:00:00
Phase 1/2 data reported that treatment was safe and well-tolerated with eleven out of fifteen patients achieved complete clearance of their BCCs, noted July 10, 2023.
1
STTK
Solid Tumors or Lymphomas
Phase 1
2021-11-09 00:00:00
Phase 1 dose escalation data reported one confirmed partial response (PR), and a second unconfirmed PR, both in patients with PD-1/L1 inhibitor pretreated non-cutaneous melanoma, noted November 9, 2021.
1
TYRA
Achondroplasia
Phase 2
2023-08-01 00:00:00
FDA Orphan drug designation (ODD) granted on August 1, 2023.
0
RHHBY
Influenza
Approved
2018-10-24 00:00:00
FDA Approval announced October 24, 2018.
1
QNRX
Netherton Syndrome (NS)
Phase 1
2023-10-24 00:00:00
Phase 1 data from 6 subjects demonstrated well defined efficacy signals across a number of study endpoints, noted October 24, 2023.
1
PFE
Psoriatic arthritis
Approved
2017-12-14 00:00:00
Approval announced December 14, 2017.
1
ABBV
Non-radiographic axial spondyloarthritis
Approved
2022-10-21 00:00:00
FDA. Approved on October 21, 2022.
1
GSK
Mild to moderate COVID-19
Phase 3
2021-03-03 00:00:00
Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.
0
AMGN
Behçet’s Disease
Approved
2019-07-19 00:00:00
FDA Approval announced July 19, 2019.
1
RHHBY
Duchenne muscular dystrophy (DMD)
Phase 1b
2021-10-11 00:00:00
Phase 1b open label interim results from the first 11 participants in Cohort 1 (ambulatory boys aged 4-7 years) provided evidence of robust expression of micro-dystrophin and no new safety signals were identified, September 24, 2021. Functional results from study cohort 1 (n=11, ages 4-7) found participants improved 3.0 points on NSAA six months after treatment - noted October 11, 2021.
1
MRK
Pneumococcal conjugate vaccine
Phase 3
2023-07-27 00:00:00
Phase 3 trial met primary endpoint, noted July 27, 2023.
0
PFE
Ovarian cancer
Phase 3
2018-12-21 00:00:00
Phase 3 trial to be terminated due to lack of efficacy.
-1
CANF
Psoriasis
Phase 3
2023-06-29 00:00:00
Phase 3 top-line data reported that trial met primary endpoint, noted June 29, 2022. Additionally in their Phase 3 the FDA encouraged the Company to enroll adolescent patients due to the strong safety profile of the drug demonstrated over the development history and prior clinical studies, noted June 29, 2023
1
CLNN
Multiple Sclerosis
Phase 2
2023-04-25 00:00:00
Phase 2 topline data presentated at AAN reported that the primary BC-LCLA outcome LS-mean difference was 3.13 and 2 of 3 key secondary outcomes were significant, noted April 25, 2023.
1
TEVA
Migraine
Approved
2018-09-14 00:00:00
FDA Approval announced September 14, 2018.
1
MEIP
Acute Myeloid Leukemia
Phase 3
2020-07-02 00:00:00
Phase 3 trial discontinued due to lack of efficacy - July 2, 2020.
-1
PFE
HIV
Approved
2022-02-01 00:00:00
Approved February 1, 2022.
1
VALN
Chikungunya Virus Infection
Phase 3
2021-08-05 00:00:00
Phase 3 trial met primary endpoint - August 5, 2021.
0
AMGN
Osteoporosis
Approved
2019-04-09 00:00:00
FDA Approval announced April 9, 2019.
1
ETON
Allergic conjunctivitis
Approved
2020-09-25 00:00:00
FDA approval announced September 25, 2020.
1
IMMX
Solid tumors
Phase 1/2
2023-07-17 00:00:00
Phase 1b/2a additional data noted that tumor shrinkage was observed in 3 out of 4 (75%) patients; 1 out of 4 (25%) experienced tumor control as of the July 7, 2023 clinical data cutoff date. All patients had stage IV relapsed/refractory metastatic colorectal cancer and received 8 median lines of therapy prior to IMX-110 + tislelizumab, noted July 17, 2023.
1
JSPR
Fanconi Anemia (FA)
Phase 1b
2023-09-21 00:00:00
Phase 1b updated results showed that all three patients treated achieved full donor engraftment and full blood count recovery. Briquilimab was well tolerated without any complications, noted September 21, 2023.
1
MRK
First-line ovarian cancer following platinum-based chemotherapy
Approved
2018-12-19 00:00:00
FDA Approval announced December 19, 2018.
1
CORT
Antipsychotic-induced weight gain (APIWG)
Phase 2
2022-12-08 00:00:00
Phase 2 data reported that AIWG did not reverse, noted December 8, 2022.
0
HALO
Chronic inflammatory demyelinating polyneuropathy (CIDP)
Phase 2
2023-07-17 00:00:00
Phase 2 trial met primary endpoints, noted July 17, 2023.
0
RHHBY
Spinal Muscular Atrophy (SMA) Type 1
Approved
2020-08-07 00:00:00
FDA Approval announced August 7, 2020.
1
TAK
Dravet Syndrome / Lennox-Gastaut Syndrome
Phase 3
2023-04-27 00:00:00
Phase 3 data reported that 78% of patients tolerated the highest dose level well with sustained reduction in frequency of drop seizures, noted April 27, 2023.
1
MRUS
Solid tumors
Phase 1
2021-12-06 00:00:00
Phase 1 data demonstrated preliminary evidence of antitumor activity has been observed at doses ≥25 mg biweekly, median duration was approximately 6 (1–74) weeks. TEAEs occurred in 33 patients (97.1%); treatment-related TEAEs occurred in 23 patients (67.6%), most commonly fatigue and decreased neutrophil count, noted December 6, 2021.
1
XENE
Postherpetic neuralgia
Phase 2b
2017-06-27 00:00:00
Phase 2b data released June 27, 2017. Endpoints not met.
0
RDHL
COVID-19
Phase 2/3
2022-02-07 00:00:00
Phase 2/3 data reported significantly reduced mortality when given to patients who received standard-of-care (SoC), as well as a significant benefit in time to recovery, noted February 7, 2022.
1
PFE
Squamous cell carcinoma of the head and neck (SCCHN)
Phase 2
2023-04-17 00:00:00
Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.
1
XENT
Recurrent sinus obstruction
Approved
2017-12-11 00:00:00
Approval announced December 11, 2017.
1
CMPX
Biliary Tract Cancer (cholangiocarcinoma)
Phase 2
2023-01-19 00:00:00
Phase 2 data presented at ASCO GI reported a 37.5% overall response rate (ORR) in 24 patients, and an ORR of 63.6% in a sub-group analysis, noted January 19, 2023.
1
AMGN
Plaque psoriasis
Approved
2021-12-20 00:00:00
Approved December 20, 2021.
1
BMY
AML Maintenance
Approved
2020-09-01 00:00:00
FDA approval announced September 1, 2020.
1
AZN
Metastatic Breast Cancer
Phase 1/2
2022-12-09 00:00:00
Phase 1/2 presentation reported that treatment significantly reduced risk of death by 36%, noted December 9, 2022.
1
RGEN
Detection of pancreatic duct abnormalities
CRL
2012-06-22 00:00:00
CRL received June 22, 2012.
0
OTLK
Wet age-related macular degeneration (wet AMD)
Phase 3
2021-10-12 00:00:00
Phase 3 safety data showed no intraocular inflammation or vasculitis, and the frequency and incidence of adverse events and ocular adverse events were low. The most common adverse event in the study eye conjunctival hemorrhage related to injection procedure, not to ONS-5010, and there were no additional serious adverse events associated with these injections, noted October 12, 2021.
1
ARWR
Cardiovascular disease / Elevated Lipoprotein
Phase 2
2023-08-28 00:00:00
Phase 2 data reported at ESC noted that results from the off-treatment extension period show that patients previously dosed with ≥75 mg of olpasiran sustained a ~40-50% placebo-adjusted percent reduction in Lp(a) nearly a year after the last dose, noted August 28, 2023.
1
RHHBY
Non-small cell lung cancer (NSCLC)
Phase 3
2022-08-01 00:00:00
Phase 3 trial met co-primary endpoints, noted August 2, 2022. Regulatory filing planned.
0
PFE
Pneumococcal conjugate vaccine (18 yrs +)
Approved
2021-06-08 00:00:00
https://www.businesswire.com/news/home/20210608006149/en/
0
REGN
Non-Hodgkin Lymphoma (NHL)
Phase 2
2022-12-12 00:00:00
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.
1
ATNM
Acute myeloid leukemia (AML)
Phase 3
2023-06-12 00:00:00
Additional Phase 3 data at EHA presented results demonstrate unprecedented bone marrow transplant access and outcomes with Iomab-B on June 12, 2023
1
EXEL
Thyroid Carcinoma
Phase 3
2021-09-20 00:00:00
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.
1
IGXT
Migraine
CRL
2020-03-27 00:00:00
CRL announced March 27, 2020.
0
RGLS
Alport Syndrome
Phase 2
2022-07-15 00:00:00
Phase 2 trial terminated after failing to meet pre-defined futility criteria, noted July 15, 2022.
1
EXEL
Advanced Neuroendocrine Tumors
Phase 3
2023-10-23 00:00:00
Phase 3 data from ESMO 23 reported that the trial met the primary objective for each cohort, demonstrating that cabozantinib provided dramatic improvements in median progression-free survival (PFS) for the patients in the pNET and epNET cohorts, noted October 23, 2023.
1
AVRO
Cystinosis
Phase 1/2
2023-05-18 00:00:00
Phase 1/2 data presented at ASGCT reported sustained engraftment and durable reduction in leukocyte cystine levels across all patients, noted May 18, 2023.
1
NGM
NASH cirrhosis
Phase 2b
2023-05-08 00:00:00
Phase 2b top-line data demonstrated statistically significant reductions in serum 7αC4 (a marker of bile acid synthesis) and fecal bile acids versus placebo in patients with idiopathic BAM with IBS-D, noted May 8, 2023.
1
MRK
Untreated locally advanced or metastatic urothelial cancer (la/mUC)
Phase 3
2023-10-23 00:00:00
Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.
1
NOVN
Acne vulgaris
Phase 3
2017-01-27 00:00:00
Phase 3 top-line data released January 27, 2017. Met one of three co-primary endpoints in NI-AC301 trial. Met all endpoints in NI-AC302 trial.
0
BMRN
Duchenne Muscular Dystrophy (DMD)
CRL
2016-01-14 00:00:00
CRL issued January 14 2016.
0
ENLV
COVID-19
Phase 2
2021-02-03 00:00:00
Phase 2 data released February 3, 2021. 14/16 (87.5%) patients recovered and were discharged from the hospital by day-28.
0
IMVIQ
Platinum Resistant Ovarian Cancer
Phase 2
2021-12-09 00:00:00
Phase 2 data reported increases in both T and B cell immune infiltration, and more prominent antigen-specific humoral responses, noted December 9, 2021.
0
SMMT
Non-small cell lung cancer (NSCLC)
Phase 2
2023-06-05 00:00:00
Phase 2 data reported a median progression-free survival (PFS) of 11.0 months and an overall response rate (ORR) of 67%, noted June 5, 2023.
0
BHC
Psoriasis
Approved
2017-02-15 00:00:00
Approved February 15, 2017.
1
LGND
Chronic obstructive pulmonary disease (COPD)
Phase 2
2021-10-13 00:00:00
Phase 2 part B data noted when administered by hand-held pMDI over one week treatment provided clinically meaningful, statistically significant and dose-dependent bronchodilation, noted October 13, 2021.
1
JNJ
Crohn's disease
Phase 3
2023-10-16 00:00:00
Phase 3 data reported that in the combination 54% of patients reached a clinical remission, noted October 16, 2023.
1
GMAB
Follicular lymphoma (FL)
Phase 1/2
2023-06-28 00:00:00
Phase 1/2 FL cohort data reported an overall response rate (ORR) of 82% and that the median duration of response (DOR) was not reached, noted June 28, 2023.
1
PFE
BRAFV600E-mutant metastatic non-small cell lung cancer (NSCLC)
Approved
2023-10-12 00:00:00
Approved October 12, 2023.
1
ATHE
Multiple System Atrophy (MSA)
Phase 2
2023-07-26 00:00:00
Phase 2 trial recommended by DMC on July 26, 2023.
0
EIGR
Post-Bariatric Hypoglycemia (PBH)
Phase 2
2018-10-16 00:00:00
Phase 2 data released October 16, 2018 - endpoints met.
0
NVO
Hemophilia A and B
CRL
2023-05-04 00:00:00
CRL received noted May 4, 2023.
0
UNCY
Hyperphosphatemia
Phase 1
2022-12-28 00:00:00
Trial met primary endpoint, noted December 28, 2022
0
INMB
Neuroinflammation caused by Alzheimer’s disease
Phase 2
2023-10-24 00:00:00
Phase 2 presentation at CTAD reported that XPro was associated with dose-dependent improvement in neurology-related proteins, noted October 24, 2023.
1
XERS
Severe hypoglycemia
Approved
2019-09-10 00:00:00
FDA Approval issued September 10, 2019.
1
CRBU
B-Cell Non-Hodgkin Lymphoma
Phase 1
2023-07-13 00:00:00
Phase 1 update reported a 94% overall response rate (ORR), 69% complete response (CR) rate, and 44% CR rate at ≥6 months following a single dose of CB-010, with 24 months as the longer CR maintained to date, noted July 13, 2023.
0
CYTK
Heart failure with reduced ejection fraction (HFrEF)
CRL
2023-02-28 00:00:00
CRL issued February 28, 2023.
0
AGIO
IDH1m Relapsed/Refractory AML - cancer
Approved
2018-07-20 00:00:00
FDA Approval announced July 20, 2018.
1
SAGE
Essential tremor
Phase 2
2021-04-12 00:00:00
Phase 2 trial met primary endpoint (P=0.049) - April 12, 2021. 62% of patients were down-titrated to from 60mg to 45 mg or 30 mg with the high dose not well tolerated.
1
AMGN
Thyroid Eye Disease (TED)
Approved
2020-01-21 00:00:00
FDA Approval announced January 21, 2020.
1
ELOX
Cystic fibrosis
Phase 2
2023-06-14 00:00:00
Phase 2 data demonstrated clinically relevant improvement in percent predicted forced expiratory volume (ppFEV1) based on final data assessment, noted June 14, 2023.
1
PFE
COVID-19 vaccine
Approved
2022-06-17 00:00:00
EUA approval for children 4 years - 6 months, noted June 17, 2022.
1
CYCN
Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
Phase 2
2022-06-28 00:00:00
Phase 2 data reported that treatment was well tolerated, with no reports of serious adverse events (SAEs) or treatment discontinuation due to adverse events (AEs), and showed improvements across multiple mitochondrial disease-associated biomarkers, inflammatory biomarkers, cerebral blood flow, and functional connectivity between neural networks, noted June 28, 2022.
1
PGEN
Myelodysplastic Syndromes, Acute Myeloid Leukemia
Phase 1b
2022-12-13 00:00:00
Phase 1/1b data reported a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients, noted December 13, 2022.
1