Clinical Catch-Up: December 27-31
Not surprisingly, the week between Christmas and New Year was quiet in terms of clinical trial news. However, there were some announcements. Here’s a look.
CanSino Biologics published Phase III data on its Convidecia vaccine against COVID-19. It demonstrated 96% efficacy in preventing severe disease and an overall efficacy of 63.7% 14 days after vaccination for healthy adults 18 years and older, with no vaccine-related serious adverse events.
Kiniksa Pharmaceuticals reported its Phase III trial of mavrilimumab for COVID-19 failed to hit the primary endpoint. Mavrilimumab is a fully human monoclonal antibody engineered to target granulocyte macrophage colony stimulating factor receptor alpha (GM-CSFRalpha).
Nykode Therapeutics dosed the first patient in its VB-D-01 Phase I/II trial of its T cell focused next-generation SARS-CoV-2 vaccine candidate. The vaccine is designed to prime T cells in hopes of generating a broad immune response against current and future variants.
Revive Therapeutics announced its Phase III trial of Bucillamine for mild to moderate COVID-19 has screened about 700 subjects and the Independent Data and Safety Monitoring Board supported continuing the study. Bucillamine is an oral drug with anti-inflammatory and antiviral properties.
Ocugen and its partner Bharat Biotech, announced positive data from a Phase II/III trial of its COVID-19 vaccine, Covaxin, in children from the ages of two to 18 years. The vaccine showed strong neutralizing antibody response and a good safety profile.
Faraday Pharmaceuticals published results from its Phase II trial of FDY-5301 for reperfusion injury after a ST-elevation myocardial infarction (STEMI) heart attack. The treatment was well-tolerated and showed encouraging efficacy signals in minimizing cardiac damage. FDY-5301 is an elemental reducing agent containing sodium iodide.
TRACON Pharmaceuticals announced the Independent Data Monitoring Committee (IDMC) for the Phase III ENVASARC trial recommended accrual as planned in both cohort A of single agent envafolimab and cohort B of enfavolimab with Yervoy (ipilimumab). Envafolimab is a single-domain anti-PD-L1 antibody. Yervoy is Bristol Myers Squibb’s anti-CTLA-4 checkpoint inhibitor.
BridgeBio Pharma reported topline data from Part A (Month 12) of its Phase III ATTRibute-CM study of acoramidis for symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The drug failed the study arm’s 6-minute walking distance (6MWD) test, but otherwise demonstrated biomarkers of efficacy. Acoramidis (AG10) is an oral small molecule engineered to stabilize tetrameric transthyretin. It was designed to mimic a naturally occurring variant of the TTR gene (T119M), a so-called “rescue mutation” that has been shown to prevent or minimize ATTR in people carrying disease-causing mutations in the TTR gene.
Saniona initiated a Phase IIb trial of Testomet in Prader-Willi syndrome. The drug is a fixed-dose combination of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a beta-1 selective blocker.
Salarius Pharmaceuticals announced a dosing milestone in the Phase I/II Sarcoma trial of seclidemstat for Ewing sarcoma and other FET-rearranged sarcomas. The patient arm of Ewing sarcoma patients receiving seclidemstat with chemotherapy drugs topotecan and cyclophosphamide (TC) advanced to the second lead-in safety cohort of patients receiving seclidemstat at 900 mg BID in combination with the two chemo drugs. Seclimdemstat is an oral, reversible inhibitor of LSD1.
Statera Biopharma submitted its Phase III protocol for STAT-201 in pediatric Crohn’s Disease to the FDA. STAT-201 is an immune modulator designed to restore the mucosa and intestinal barrier function with standard of care in moderate to severe Crohn’s.
Hansa Biopharma treated the first patient in its Phase III ConfideS trial of imlifidase in highly sensitized kidney transplant patients. The trial is at the Columbia University Medical Center, New York. The drug is designed to eliminate the antibodies responsible for transplant immunological incompatibilities.
Madrigal Pharmaceuticals announced an expected delay in the topline 52-week results in its MAESTRO-NAFLD-1 Phase III trial. The study is evaluating resmetirom in non-alcoholic fatty liver disease, presumed NASH. They cite unexpected staffing issues at the vendor conducting the statistical analyses.
Jazz Pharmaceuticals enrolled the first patient in its Phase II trial of JZP150 for post-traumatic stress disorder (PTSD). The drug is a highly selective inhibitor of the enzyme fatty acid amide hydrolase (FAAH).