Company Product Description Indication Status
Phase I
Arvinas Inc., of New Haven, Conn. ARV-471 Protein degrader Locally advanced or metastatic ER+/HER2-negative breast cancer Demonstrated evidence of antitumor activity and estrogen receptor degradation profile and robust efficacy signals in a heavily pretreated patient population; efficacy signals include 1 RECIST confirmed partial response, 2 patients with unconfirmed PRs and a clinical benefit rate of 42%
Arvinas Inc., of New Haven, Conn. ARV-110  Protein degrader Metastatic castration-resistant prostate cancer Ongoing dose-escalation portion of phase I/II study provided additional evidence of antitumor activity, including PSA reduction of more than 50% rate of 40% in a molecularly defined patient population
Freeline Therapeutics Holdings plc, of London FLT-180a AAV gene therapy  Severe hemophilia B Updated data from phase I/II B-Amaze trial show durable factor IX (FIX) activity up to a period of nearly 3 years, with no bleeds reported requiring FIX supplementation, and support selection of a dose and immune management regimen that has the potential to deliver FIX activity in the normal range
Kintor Pharmaceutical Ltd., of Suzhou, China Proxalutamide Androgen receptor antagonist COVID-19 Preliminary outcome showed hospitalization rate was 0.8% for proxalutamide arm, compared to 27% for control arm; percentage of mechanical ventilation usage was 0% for proxalutamide vs, 9% for control arm; percentage of death was 0% for proxalutamide arm vs. 2% for control arm; both co-primary efficacy endpoints were achieved; final study report is expected in January 2021
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 COVID-19 spike glycoprotein modulator COVID-19 infection Analysis of 37 participants vaccinated in ongoing phase I/II study in Germany showed all had newly generated spike protein-specific CD4+ T cell responses and about 92% showed CD8+ T-cell responses
Synlogic Inc., of Cambridge, Mass. SYNB-1891 (SYN-STING) STING protein stimulator Solid tumors; lymphoma Ongoing trial advanced to combination therapy stage 
TCR2 Therapeutics Inc., of Cambridge, Mass. TC-210 (gavocabtagene autoleucel) Mesothelin modulator Solid tumors Interim data from ongoing phase I portion of phase I/II trial in mesothelin-expressing solid tumors showed 3 partial responses according to RECIST 1.1 in first 8 participants; disease control rate was 100%, with all showing tumor regression and median decrease in sum of diameters of target lesions of 43% (range, 5% to 75%)
Phase II
Aerpio Pharmaceuticals Inc., of Cincinnati Razuprotafib (formerly AKB-9778) Protein tyrosine phosphatase beta inhibitor Glaucoma Primary endpoint of decline from baseline in diurnal mean intraocular pressure achieved with twice-daily dose group at day 28 vs. latanoprost control (2-sided p=0.0130); once-daily dose did not show statistically significant improvement at day 28
Bellus Health Inc., of Laval, Quebec BLU-5937 P2X3 purinoceptor antagonist Chronic pruritus First participant dosed in Blueprint trial in people with atopic dermatitis; primary efficacy endpoint is change from baseline in weekly mean Worst Itch-Numeric Rating Scale score at week 4, with top-line data expected in fourth quarter of 2021
Biophytis SA, of Paris Sarconeos (BIO-101; 20‐hydroxyecdysone) Proto-oncogene Mas agonist Sarcopenia; sarcopenic obesity Observational study showed rapid deterioration of mobility in participants, measured by 400-meter walk test and 6-minute walk test, due to natural disease progression, validating inclusion criteria for phase IIb Sara-Int trial
Caladrius Biosciences Inc., of Basking Ridge, N.J. CLBS-16 Peripheral blood derived autologous CD34-positive cell therapy Coronary microvascular dysfunction Ongoing phase IIb Freedom trial targeted to complete enrollment of 105 participants by year-end 2021, with top-line data in third quarter of 2022
Cytokinetics Inc., of South San Francisco Reldesemtiv Fast skeletal muscle troponin activator Amyotrophic lateral sclerosis Post-hoc analyses from Fortitude-ALS showed middle and fastest progressing tertiles, combined, treated with study drug showed 27% difference at 12 weeks vs. placebo (1.15 ALSFRS-R points, p=0.011) compared to 18% (0.4 points; p=0.43) in slowest progressing tertile, providing rationale for planned phase III trial design
Curevac NV, of Tübingen, Germany CVnCoV (SARS-CoV-2 mRNA vaccine) COVID-19 spike glycoprotein modulator COVID-19 infection First of > 35,000 participants enrolled in pivotal phase IIb/III study; co-primary efficacy measures include effectiveness in preventing first episodes of confirmed cases of infection of any severity and in preventing moderate to severe confirmed infection; participants will be monitored in 1-year extension study
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. Datopotamab deruxtecan (DS-1062) Trop-2 calcium signal transducer modulator Non-small-cell lung cancer Tropion-Lung05 trial initiated in people with advanced or metastatic disease with actionable genomic alterations previously treated with kinase inhibitor and platinum-based chemotherapy with or without immunotherapy; primary trial endpoint is overall response rate assessed by blinded independent central review
Innovent Biologics Inc., of Suzhou, China IBI-310 Anti-CTLA4 monoclonal antibody Advanced cervical cancer First of 174 participants enrolled and dosed in combination trial with Tyvyt (sintilimab, Eli Lilly and Co./Innovent) in second-line or above disease
Poxel SA, of Lyon, France PXL-770 AMP activator Nonalcoholic steatohepatitis Additional phase IIa data from Stamp-NAFLD trial showed therapy associated with -27% mean relative reduction in liver fat content at 500 mg once daily (p=0.004) vs. baseline in participants with coexisting type 2 diabetes (T2D); other clinically meaningful improvements (p<0.05) in T2D seen in alanine and aspartate transaminase, fasting glucose and hemoglobin A1c; 52-week phase IIb trial will assess up to 2 doses of study drug in up to 120 people per study arm with biopsy-proven NASH and pre-diabetes or T2D, with primary endpoint of NASH resolution with no worsening of fibrosis
Phase III
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor COVID-19 infection CardioLink-9 study drug prompted 25% within-group reduction in high-sensitivity C-reactive protein (p=0.011) and within-group reduction in D-dimer (p=0.048) as well as 52% reduction of influenza patient-reported outcome prevalence score vs. 24% reduction for usual care (p=0.003 between groups)
Cytokinetics Inc., of South San Francisco Reldesemtiv Fast skeletal muscle troponin activator Amyotrophic lateral sclerosis Courage-ALS trial expected to enroll about 555 people with ALS within first 2 years of initial muscle weakness plus vital capacity of ≥65% predicted and screening ALS Functional Rating Scale – Revised ≤44; participants will be randomized 2-to-1 to 300 mg of study drug or placebo twice daily for 24 weeks followed by 24 weeks of study drug for all; primary efficacy endpoint is change from baseline to 24 weeks in ALSFRS-R; trial will include 2 unblinded interim analyses by DMC
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. Datopotamab deruxtecan (DS-1062) Trop-2 calcium signal transducer modulator Non-small-cell lung cancer Pivotal Tropion-Lung01 study vs. docetaxel initiated in about 590 participants with advanced or metastatic disease without actionable genomic alterations who previously received platinum-based chemotherapy and immunotherapy; primary endpoints are progression-free and overall survival
Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Willington, Del. Baricitinib Monoclonal antibody targeting SARS-CoV-2 COVID-19 prophylaxis Data from the ACTT-2 study published in The New England Journal of Medicine showed treatment with baricitinib shortened the median time to recovery from 18 days to 10 days
Junshi Biosciences Co. Ltd., of Shanghai Toripalimab Monoclonal antibody targeting PD-1 Non-small-cell lung cancer In an interim analysis, the Choice-01 study met its primary endpoint of improvement in progression-free survival for toripalimab in combination with standard first-line chemotherapy compared to chemotherapy alone
Mitsubishi Tanabe Pharma Corp., of Osaka, Japan Edaravone (MT-1186) Neuroprotectant Amyotrophic lateral sclerosis Started the 380-patient MT-1186-A02 study of the oral formulation to meet the postmarketing commitment following the FDA approval of Radicava (edaravone); study will compare daily medication to an on/off dosing regimen during each 28-day cycle; primary is endpoint change in ALS Functional Rating Scale-Revised score from baseline to week 48
Novartis AG, of Basel, Switzerland Ruxolitinib JAK1/2 inhibitor COVID-19 Study didn't meet its primary endpoint of reducing the composite endpoint of proportion of patients who die, develop respiratory failure or require admission to the intensive care unit by day 29
Odonate Therapeutics Inc., of San Diego Tesetaxel Taxane chemotherapy Metastatic breast cancer In the Contessa study, median progression-free survival was 9.8 months for tesetaxel plus a reduced dose of capecitabine compared to 6.9 months for the full dose of capecitabine (p=0.003)
Oxford University, of Oxford, U.K. Azithromycin Macrolide-type antibiotic COVID-19 In the Recovery study, mortality rate on day 28 was 19% for azithromycin vs. 19% standard of care (p=0.99)
Polyphor AG, of Allschwil, Switzerland Balixafortide CXCR4 chemokine antagonist HER2-negative, locally recurrent or metastatic breast cancer After the third interim review of data from 401 patients, the independent data safety monitoring committee recommended continuing the study without modifications
Puma Biotechnology Inc., of Los Angeles Neratinib EGFR antagonist; ERBB2/ERBB4 tyrosine kinase receptor inhibitor HER2-positive metastatic breast cancer In the Nala study, progression-free survival was 8.8 months for neratinib plus capecitabine compared to 6.6 months for Tykerb (lapatinib, Glaxosmithkline plc) plus capecitabine (p=0.0059); no difference in overall survival between the 2 therapies (p=0.2098)
Russian Direct Investment Fund, and the National Research Center for Epidemiology and Microbiology, both of Moscow Sputnik V vaccine  Adenoviral-based vaccine COVID-19 prophylaxis Vaccine had a 91.4% efficacy rate with 62 cases of COVID-19 for participants who received placebo and 16 cases for those who received the vaccine
Sanofi SA, of Paris Fitusiran RNAi therapeutic Hemophilia A or B Resuming treatment of U.S. adolescent and adult patients after a pause due to reports of nonfatal thrombotic events; plans to implement amended protocols with an adjusted dose and dosing regimen; pediatric study and studies outside the U.S. remain paused


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