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1
PDSB
Metastatic Prostate Cancer
Phase 1
2023-10-11 00:00:00
First in human safety and immune data presented at Cytokines reported a decrease in PSA levels, and that all doses of the combination were well-tolerated with one patient experiencing Grade 4 neutropenia, noted October 11, 2023.
1
FBIO
B-cell Non-Hodgkin Lymphoma & Chronic Lymphocytic Leukemia (CLL)
Phase 1/2
2023-08-16 00:00:00
Initial Phase 1/2 data reported clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy, noted August 16, 2023.
1
CAPR
Duchenne Muscular Dystrophy (DMD)
Phase 2
2023-10-10 00:00:00
Phase 2 data presented at the World Muscle Society reported that the mean PUL 2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7-point decline on average observed over 24-months in the placebo, noted October 10, 2023.
1
BLRX
Stem-cell mobilization for autologous transplantation
Approved
2023-09-11 00:00:00
FDA Approved on September 11, 2023.
1
AGIO
Glioma
Phase 1
2018-06-01 00:00:00
Phase 1 data presented at ASCO June 1, 2018. 1 minor response + 1 partial response out of 93 patients.
1
RHHBY
Hepatocellular Carcinoma (HCC), Solid tumors
Phase 1
2023-04-17 00:00:00
Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.
1
GSK
Severe eosinophilic asthma (6-11 yrs)
Approved
2019-09-12 00:00:00
FDA approval announced September 12, 2019.
1
PRQR
Autosomal dominant retinitis pigmentosa (adRP)
Phase 1/2
2021-11-18 00:00:00
Phase 1/2 data reported 3 cases of cataract worsening, with maximum benefit in BCVA observed after 5 weeks and declined thereafter. A mean BCVA benefit of +1.4 letters was reported across all subjects. In doses 75 - 300µg, the mean BCVA benefit was +5 letters, maximum benefit observed was +7 letters, noted November 18, 2021.
1
SLS
HER2 3+ breast cancer
Phase 2b
2021-11-30 00:00:00
Phase 2b trial noted DFS rate of 75.2% reduction in relative risk of recurrence or death, noted November 2021.
1
DVAX
Hepatitis B
Approved
2017-11-09 00:00:00
Final approval announced November 9, 2017
1
SRPT
Duchenne muscular dystrophy
Phase 2
2021-05-03 00:00:00
Phase 2 30 mg/kg arm data presented May 3, 2021 - mean exon skipping of 10.79% and mean dystrophin expression of 6.55%.
1
INCY
Follicular lymphoma
Phase 2
2020-12-07 00:00:00
Phase 2 data due released at ASH December 2020 - ORR 73%.
0
ARGX
Chronic inflammatory demyelinating polyneuropathy
Phase 2
2023-07-17 00:00:00
Phase 2 top-line data met primary endpoint; VYVGART Hytrulo demonstrated 61% reduction in risk of relapse versus placebo
1
RHHBY
Babies with pre-symptomatic spinal muscular atrophy (SMA)
Phase 3
2023-10-04 00:00:00
Phase 3 OLE met its primary endpoint with 80% of babies sitting without support for at least 5 seconds after 1 year of Evrysdi, noted October 4, 2023.
0
ZLAB
Immune thrombocytopenia (ITP)
Phase 3
2022-05-05 00:00:00
Phase 3 top-line data reported that trial met primary endpoint, noted May 5, 2022.
0
PFE
HIV
Approved
2021-01-21 00:00:00
FDA approval announced January 21, 2021.
1
CLNN
Parkinson's Disease
Phase 2
2021-08-05 00:00:00
Phase 2 trial met primary endpoint - August 5, 2021.
0
ERAS
Non-small-cell Lung Cancer
Phase 1b
2023-06-05 00:00:00
Deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023.
1
BHVN
Amyotrophic lateral sclerosis (ALS)
Phase 3
2022-09-29 00:00:00
Phase 3 trial did not meet its primary endpoint, noted September 29, 2022.
1
ACOR
OFF episodes of Parkinson’s disease (PD)
Approved
2018-12-24 00:00:00
FDA Approval announced December 22, 2018.
1
CTXR
CTCL non-Hodgkin lymphoma
CRL
2023-07-29 00:00:00
CRL received on July 29, 2023.
0
PTGX
Polycythemia Vera (PV)
Phase 2
2023-06-11 00:00:00
Phase 2 data presented at EHA showed that PK-efficacy model adequately described the rusfertide plasma concentration-time profile and the time course of Hb, noted June 9, 2023.
1
XNCR
Relapsed or refractory diffuse large B cell lymphoma
Approved
2020-07-31 00:00:00
FDA Approval announced July 31, 2020.
1
PFE
Atopic dermatitis
Phase 3
2021-08-30 00:00:00
Phase 3 trial met its co-primary and key secondary efficacy endpoints.
0
VSTM
KRAS mutant non-small cell lung cancer (NSCLC)
Phase 2
2022-10-04 00:00:00
Phase 2 part A data did not meet the pre-defined criteria to continue the trial, noted October 4, 2022.
1
RARE
Ornithine Transcarbamylase (OTC) Deficiency
Phase 1/2
2022-05-19 00:00:00
Phase 1/2 trial safety and efficacy data reported that in the prophylactic steroid cohort, one of two patients demonstrated a complete response, noted May 19, 2022.
1
VOR
Acute myeloid leukemia (AML)
Phase 1/2
2023-06-09 00:00:00
Phase 1/2a additional data presented at EHA showed that patients transplanted with trem-cel achieved primary neutrophil engraftment and high levels of myeloid donor chimerism, noted June 9, 2023.
1
NKTR
Urothelial cancer
Phase 2
2022-04-14 00:00:00
Phase 2 trial did not meet the efficacy threshold in its final ORR analysis, noted April 14, 2022.
1
RHHBY
Relapsed or refractory multiple myeloma
Phase 3
2023-09-29 00:00:00
Phase 3 data reported that it showed improvement in median PFS of 9.9 months compared to 5.8 months with the combination of study comparator pomalidomide and dexamethasone; however, the results did not reach statistical significance, noted September 29, 2023.
1
REGN
Wet AMD
CRL
2018-08-13 00:00:00
CRL issued August 13, 2018 - ongoing labeling discussions.
0
RARE
Glycogen Storage Disease Type Ia (GSDIa)
Phase 1/2
2022-05-19 00:00:00
Phase 1/2 week 52 clinical efficacy and safety data reported that patients reached a mean total daily reduction of cornstarch intake of 70%, noted May 19, 2022.
1
IRWD
Irritable Bowel Syndrome with Constipation (IBS-C)
Phase 3
2019-06-18 00:00:00
Phase 3b data met all endpoints - June 18, 2019.
0
ZLAB
Metastatic breast cancer
Approved
2021-09-07 00:00:00
Final overall survival data did not demonstrate MARGENZA plus chemotherapy had a statistically significant advantage compared to trastuzumab plus chemotherapy; September 7, 2021.
1
RHHBY
Prurigo nodularis
Phase 2a
2020-04-22 00:00:00
Phase 2a trial met primary endpoint - April 22, 2020.
0
IMPL
Migraine
Approved
2021-09-03 00:00:00
Approved September 3, 2021.
1
VBIV
Covid-19
Phase 1
2023-09-27 00:00:00
Phase 1 interim data reported a 25% reduction in GMT against Wuhan after 5 months vs. peak responses, noted September 27, 2023.
1
MGNX
Squamous cell carcinoma of the head and neck (SCCHN)
Phase 2
2022-07-08 00:00:00
Phase 2 trial discontinued following an internal review of safety data, which included the occurrence of seven fatalities potentially associated with hemorrhagic events in both arms of the study, noted July 8, 2022.
-1
INFI
Solid tumors
Phase 1
2018-11-10 00:00:00
Further data due at SITC November 2018 noted 2/44 PRs.
0
ALNY
Alzheimer's Disease and Cerebral Amyloid Angiopathy
Phase 1
2023-10-25 00:00:00
Phase 1 achieve sustained pharmacodynamic activity up to 10 months after administration, noted October 25, 2023.
1
ARDS
Cystic Fibrosis
Phase 2a
2023-03-13 00:00:00
Phase 2a trial met primary and secondary endpoints, noted March 13, 2023.
0
LGVN
Aging Frailty
Phase 2b
2021-09-29 00:00:00
Phase 2b biomarker data showed Lomecel-B had statistically significant reduction in serum levels of soluble TIE-2 in a dose-dependent fashion at Day 270 compared to placebo. The levels fell by -243.13 ± 1073.21 pg/mL compared to an increase of 356.03 ± 1018.95 pg/mL in the placebo group, noted September 29, 2021.
1
IMRA
Sickle cell disease
Phase 2a
2021-12-13 00:00:00
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
1
IDYA
Metastatic uveal melanoma (MUM)
Phase 1
2021-12-07 00:00:00
Phase 1 data showed 100% Disease Control Rate (DCR) with (16/16) evaluable patients with >1 post-baseline scan showed tumor shrinkage as determined by target lesion size reduction , 31% ORR, 46% of patients (6/13) with > 2 post-baseline scans observed >30% tumor reduction, including one patient with an unconfirmed PR, December 7, 2021.
1
ADCT
Diffuse Large B-Cell (DLBCL) or Mantle Cell Lymphoma (MCL)
Phase 2
2021-12-13 00:00:00
Phase 2 interim data reported an ORR of 57.1% in the overall DLBCL cohort, and 45.5% and 76.9% in the non-GCB DLBCL and GCB DLBCL cohorts, respectively. Median duration of response in the overall DLBCL cohort was 5.49 months and was not reached in the non-GCB DLBCL or GCB DLBCL cohorts, noted December 13, 2021.
1
IONS
Huntington's Disease
Phase 3
2021-03-22 00:00:00
Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.
-1
XLO
Solid Tumors
Phase 1/2
2023-11-03 00:00:00
Phase 1/2 trial data presented at SITC showed initial evidence of dose-dependent disease control rate with 50% disease control rate at higher doses (≥2.8 mg/kg) and 31% disease control rate across all dose levels in a range of solid tumor types, including cold tumors, noted November 3, 2023.
1
NLSP
Narcolepsy
Phase 3
2023-03-27 00:00:00
OLE trial data reported that the mean ESS score for these patients declined by approximately 10.2 (SD=5.83) points from their DB study baseline to month 6 in the OLE, noted March 27, 2023.
0
HCM
Colorectal cancer
Phase 3
2022-09-08 00:00:00
Phase 3 data demonstrated that treatment reduced the risk of death by 34% in metastatic colorectal cancer , and Increased disease control with risk of disease progression or death reduced by 68%, noted September 8, 2022
1
CCXI
Focal Segmental Glomerulosclerosis
Phase 2
2020-05-18 00:00:00
Phase 2 trial did not meet primary endpoint - May 18, 2020.
1
MRK
First-line, lower-risk Myelodysplastic syndromes (MDS)
Approved
2023-08-28 00:00:00
Approved August 28, 2023.
1
PCRX
Nerve blocker in lower extremity surgeries
Phase 3
2022-09-06 00:00:00
Phase 3 trial met primary endpoint, noted September 7, 2022.
0
QLGN
Lactose intolerance
Phase 3
2019-09-12 00:00:00
Phase 3 data failed to meet primary endpoint.
1
RHHBY
First line unfit AML
Approved
2018-11-21 00:00:00
FDA approval announced November 21, 2018.
1
VRTX
Children (6-11 years old) who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive
Approved
2021-06-09 00:00:00
FDA approval announced June 9, 2021.
1
BHVN
Glioblastoma (GBM)
Phase 2/3
2023-10-31 00:00:00
Phase 2/3 data reported that it did not perform better than the current standards of care in glioblastoma, noted October 31, 2023.
0
HCM
Gastric Cancer
Phase 2
2022-11-14 00:00:00
Phase 3 trial met primary endpoint regarding progression-free survival. The trial did not meet the the overall survival primary endpoint, noted November 14, 2022.
1
MRK
Non-muscle invasive bladder cancer (NMIBC)
Phase 1
2023-07-28 00:00:00
Phase 1 dosing initiated, noted July 28, 2023.
0
ORTX
Mucopolysaccharidosis type I (MPS-I)
Phase 1
2023-10-26 00:00:00
PoC data presented at ESGCT resulted in improvement 62.5% of patients or stabilization 37.5% of patients of corneal clouding at the time of last follow-up ranging from 3.14-4.58 years compared to baseline measured prior to administration with OTL-203, noted October 26, 2023.
1
TENX
Septic shock
Phase 2b
2016-10-05 00:00:00
Phase 2b trial did not meet primary endpoint.
1
HARP
Multiple myeloma
Phase 1
2023-11-02 00:00:00
Phase 1 data from ASH abstract reported that among the 12mg and 24mg cohorts, 55% (22/40) of efficacy-evaluable pts currently have had a confirmed response (PR or better) and among pts with a response, 73% (16/22) have had a confirmed response of VGPR or better, noted November 2, 2023.
1
CYDY
Non-alcoholic steatohepatitis (NASH)
Phase 2
2022-01-05 00:00:00
Phase 2 trial met primary and secondary endpoint, noted January 5, 2022.
0
AZN
Asthma
Approved
2021-12-17 00:00:00
Approved December 17, 2021.
1
MREO
COVID-19
Phase 1/2
2021-12-22 00:00:00
Phase 1b/2 data reported no safety signals were observed and 62.5% of (5/8) patients had a 2-point decrease in the WHO Disease Severity score by Day 5, compared to 28.5% (2/7) patients in the placebo arm, noted December 22, 2021.
1
ZYNE
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
ABVC
Attention-Deficit Hyperactivity Disorder (ADHD)
Phase 2
2023-04-26 00:00:00
Phase 2 study opened new sites, clinical trial continues to demonstrate that medicines derived from plants have significant therapeutic benefits with few, noted April 26, 2023.
1
CRSP
Sickle cell disease
Phase 1/2
2022-06-13 00:00:00
Data presented at EHA reported that all 31 patients with severe SCD were free of VOCs after exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months, noted June 13, 2022.
1
MRK
Cutaneous squamous cell carcinoma (cSCC)
Approved
2021-07-06 00:00:00
FDA approval announced July 6, 2021.
1
YS
Rabies
Phase 3
2023-09-26 00:00:00
Phase 3 trial initiated, noted September 26, 2023.
0
REGN
Genetic hearing loss
Phase 1/2
2023-10-26 00:00:00
Phase 1/2 data reported that one patient experienced improvements in auditory responses through week 6 compared to baseline, noted October 26, 2023.
1
AVXL
Rett syndrome
Phase 3
2022-02-01 00:00:00
Phase 3 data reported that trial met primary and all secondary efficacy and safety endpoints, noted February 1, 2022.
0
CORT
Cushing's Syndrome
Approved
2012-02-17 00:00:00
Approved February 17, 2012.
1
BMY
Relapsed/refractory multiple myeloma
Approved
2018-11-06 00:00:00
FDA approval announced November 6, 2018.
1
CLDX
Chronic inducible urticaria (CIndU), cold urticaria (ColdU) and symptomatic dermographism (SD)
Phase 1
2023-06-10 00:00:00
Phase 1 data reported at EAACI 2023 include safety data through 12 weeks were 56% patients achieved a complete response (negative test) with PCE provocation testing with just one dose of barzolvolimab and most responses remained durable through to week 12, noted June 10, 2023.
1
LRMR
Friedreich’s ataxia (FA)
Phase 2
2023-07-25 00:00:00
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.
0
GLMD
Non-Alcoholic Steatohepatitis (NASH)
Phase 3
2022-05-17 00:00:00
Open label portion of study met objective, noted May 17, 2022.
0
ABBV
HCV - genotype 1
Approved
2014-12-19 00:00:00
Approved December 19, 2014.
1
RHHBY
Diffuse Large B-Cell Lymphoma
Approved
2023-04-19 00:00:00
FDA Approved on April 19, 2023.
1
BMY
Plaque psoriasis
Approved
2022-09-10 00:00:00
FDA Approval on September 10, 2022.
1
APVO
Relapsed or refractory peripheral T-cell lymphoma
Phase 2
2018-11-14 00:00:00
Development terminated - noted November 14, 2018.
-1
MRSN
Ovarian Cancer
Phase 3
2023-06-15 00:00:00
A clinical hold placed due to bleeding events, noted June 15, 2023.
0
CHRS
Non-small cell lung cancer (NSCLC)
Phase 3
2023-06-06 00:00:00
Phase 3 final overall survival and biomarker analyses presented at ASCO reported a significant improvement in OS was observed for the toripalimab arm over the placebo arm: median OS 23.8 vs 17.0 months, noted June 6, 2023.
1
LIAN
Obstructive hypertrophic cardiomyopathy (oHCM)
Phase 3
2023-08-28 00:00:00
Phase 3 data presented at ESC reported that treatment demonstrated improvement in Valsalva LVOT peak gradient, LVOT obstruction, clinical symptoms, health status, cardiac biomarkers, and cardiac structure in Chinese oHCM patients, noted August 28, 2023.
1
APTX
Parkinson’s disease cognitive impairment, healthy volunteers
Phase 2
2023-02-27 00:00:00
Phase 2 study did not demonstrate sufficient efficacy to support further development, noted February 27, 2023.
1
PFE
Endometriosis-associated pain
Approved
2022-08-05 00:00:00
Approved August 5, 2022.
1
GNLX
Platinum-Resistant or Refractory Ovarian Cancer
Phase 2
2023-05-25 00:00:00
Phase 2 topline data reported that trial met primary endpoint, noted May 25, 2023.
0
NXTC
Solid tumors
Phase 1/2
2021-11-13 00:00:00
Combined Phase 1 and Phase 2 data reported one complete response (CR) and one partial response (PR), with disease control rate across all tumors in both studies was 37% and a median progression-free survival (PFS) of 5.0 months, noted November 13, 2021.
0
APLS
COVID-19 / Acute respiratory distress syndrome (ARDS)
Phase 1/2
2021-03-04 00:00:00
Phase 1/2 trial to be discontinued following Independent data monitoring committee (DMC) review which found no meaningful reduction in the overall mortality rate.
1
AADI
Open-angle glaucoma (OAG)
Phase 2
2020-12-11 00:00:00
Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.
0
BIIB
Major Depressive Disorder (MDD)
Phase 3
2022-02-16 00:00:00
Phase 3 trial met its primary and key secondary endpoints, comparing a 50 mg co-initiated with standard of care antidepressant vs. standard of care co-initiated with placebo. At the Day 3 primary endpoint, dosing with a standard of care antidepressant showed a statistically significant reduction in depressive symptoms.
1
CKPT
Non-small cell lung cancer (NSCLC)
Phase 1/2
2018-09-25 00:00:00
Phase 1/2 preliminary data released September 24, 2018.
0
AZN
Lung cancer
Phase 3
2019-08-21 00:00:00
Phase 3 data released August 21, 2019 did not meet primary endpoint.
1
BHC
Dry Eye Disease Associated With Meibomian Gland Dysfunction
Approved
2023-06-29 00:00:00
Approved on May 18, 2023.
1
BNOX
Post-Traumatic Stress Disorder (PTSD)
Phase 2b
2023-09-28 00:00:00
Phase 2b topline data met its primary endpoint showing BNC210 treatment led to a statistically significant reduction in total PTSD symptom severity at 12 weeks. Statistically significant secondary endpoints showed improvements in depressive symptoms and sleep, noted September 28, 2023.
1
TAK
Multiple myeloma
Phase 3
2020-03-10 00:00:00
Phase 3 trial did not meet primary endpoint - March 10, 2020.
1
VRTX
Cystic fibrosis
Approved
2020-12-21 00:00:00
FDA approval announced December 21, 2020.
1
PRFX
Bunionectomy
Phase 3
2023-06-08 00:00:00
Phase 3 clinical trial Company's supplier of the API (active pharmaceutical ingredient) has received a deficiency notice from the FDA related to its Drug Master File (DMF). As a result, the second part of the Phase 3 trial is expected to commence once the supplier has provided the required information to the FDA and the deficiency notice has been resolved, noted June 8, 2023.
0
MORF
Ulcerative colitis
Phase 2a
2023-09-23 00:00:00
Phase 2a trial achieved the primary endpoint and demonstrated clinically meaningful improvements across secondary and exploratory measures, noted September 22, 2023.
1
BHVN
Migraine - preventative
Approved
2021-05-27 00:00:00
FDA approval announced May 27, 2021.
1
IMUX
Progressive Multiple Sclerosis
Phase 2
2023-10-09 00:00:00
Phase 2 24-Week data from first half of patients shows improvements in biomarker NfL, Consistent throughout the overall population as well as all subtypes, noted October 9, 2023.
1
KZR
Lupus nephritis (LN)
Phase 2
2022-11-03 00:00:00
Phase 2 complete results reported that zetomipzomib added to stable background therapy reduced proteinuria by 50% or greater (ORR) in 11 of 17 patients (64.7%) reaching end of treatment at Week 25, noted November 3, 2022.
1