Company Product Description Indication Status
Phase I
Achilles Therapeutics Ltd., of London cNeT therapy Clonal neoantigen T-cell therapy Metastatic non-small-cell lung cancer Independent data safety and monitoring committee completed its first review of the ongoing phase I/IIa Chiron and Thetis trials and recommended that both continue as planned; the overall tolerability profile was generally similar to that of standard tumor-infiltrating lymphocyte products that have not been enriched for cNeT reactivities, with the lymphodepletion regimen accounting for most higher-grade adverse events, and there was encouraging evidence of cNeT engraftment
I-Mab Biopharma Co. Ltd., of Shanghai and Gaithersburg, Md. Uliledlimab (TJD-5 or TJ-004309) Humanized CD73 antibody Advanced solid tumors The first patient was dosed in a combination study with anti-PD-1 in China; top-line results from a phase I study in the U.S. demonstrate safety and clinical activity
Sanofi SA, of Paris, and Glaxosmithkline plc, of London COVID-19 vaccine candidate Vaccine COVID-19 Sanofi disclosed in fourth-quarter 2020 earnings interim results from phase I/II trial, demonstrating an insufficient response in older adults; companies will need to refine the concentration of antigen in order to provide high-level immune response across all age groups and plan a phase IIb study with an improved antigen formulation, expected to start February 2021
Spark Therapeutics Inc., of Philadelphia, a unit of Roche Holding AG SPK-8016 Gene therapy Hemophilia A Preliminary data from part 1 of the ongoing phase I/II study show all 4 participants have stable and durable factor VIII activity at greater than 52 weeks ranging from 5.9% to 21.8% with no serious adverse events and no FVIII inhibitor development; data show a 98% reduction in annualized infusion rate and 85% reduction in annualized bleed rate after a follow-up of between 15 and 18 months or 5.5 total patient years
Phase II
Covaxx, subsidiary of United Biomedical Inc., both of Hauppauge, N.Y. UB-612 Multitope peptide-based vaccine COVID-19 infection Initiated trials in Taiwan 
Sanofi SA, of Paris Venglustat  Glucosylceramide synthase inhibitor Parkinson’s disease Disclosed in fourth-quarter 2020 earnings that study in patients with GBA mutations did not meet primary endpoint; indication was halted
Spero Therapeutics Inc., of Cambridge, Mass. SPR-720 Oral antimicrobial agent Nontuberculous mycobacterial pulmonary disease FDA placed a clinical hold on its phase IIa trial, following the notification by Spero to the FDA of its decision to pause dosing as a precautionary measure following events in its ongoing animal toxicology study 
Phase III
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) Monoclonal antibody targeting PD-L1 Recurrent or metastatic head and neck cancer The Kestrel trial did not meet the primary endpoint of improving overall survival (OS) vs. the EXTREME treatment regimen (chemotherapy plus cetuximab), a standard of care; also, the combination did not indicate an OS benefit in all-comer patients, a secondary endpoint
PTC Therapeutics Inc., of South Plainfield, N.J. Translarna (ataluren)  Regulator of nonsense transcripts modulator Duchenne muscular dystrophy Study 045 fell short of statistical significance on primary endpoint of change in dystrophin expression (6.56% increase as measured by electrochemiluminescence in the intent-to-treat population; p=0.24)


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