Sio Gene Therapies Inc. CEO Pavan Cheruvu said the company sees no “significant rate limiters to moving forward” with FDA fast-tracked AXO-AAV-GM1 for the treatment of GM1 gangliosidosis. Shares of the New York-based firm (NASDAQ:SIOX) closed at $2.36, up 21 cents, as Wall Street welcomed positive interim data from the ongoing phase I/II study with the adeno-associated viral vector 9-based gene therapy for GM1 gangliosidosis.
Stealth Biotherapeutics Corp.’s refusal to file (RTF) letter from the FDA regarding the NDA for elamipretide, a candidate that targets mitochondria in the treatment of Barth syndrome, hardly knocked the stock, as investors likely saw the bad news coming.
The Menarini Group and Radius Health Inc.’s phase III Emerald study of oral elacestrant as a monotherapy vs. standard of care in treating estrogen receptor-positive/HER2-negative advanced or metastatic breast cancer hit its two primary endpoints. The study’s top-line data, according to the companies, showed statistically significant progression-free survival in both the overall population and in patients with tumors harboring estrogen receptor 1 mutations.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Abivax, Atea, Avadel, Avrobio, Cour, Dicerna, Edesa, Eli Lilly, Entasis, Galera, Harbour, Immune, Immunitybio, Inhibikase, Iterion, Noxxon, Ono, Opthea, Paratek, Pharnext, Seal Rock, Sifi, Small, Statera, Ultragenyx, Vyne.
Based on positive phase IIb data from 2020 for avasopasem manganese (GC-4419) in treating severe oral mucositis (SOM) in patients with locally advanced head and neck cancer, Galera Therapeutics Inc. had been confident about phase III data for the selective small-molecule dismutase. However, the phase III Roman trial failed to hit its primary endpoint of reduction in the incidence of SOM. Mel Sorensen, Galera’s CEO, expressed his surprise at the results and said the company is evaluating the next steps for the program.
Baidu Inc.’s preclinical studies showed the mRNA vaccine sequences for COVID-19 designed using its Lineardesign algorithm outperformed the benchmark sequences designed by traditional algorithms in terms of stability, protein expression and immunogenicity. The firm teamed up with Stemirna Therapeutics Co. Ltd., which specializes in the R&D of mRNA vaccines and drugs, to test seven mRNA COVID-19 vaccine sequences designed using Lineardesign.
Top-line results of a phase III trial testing sulbactam-durlobactam, a combination beta-lactam antibiotic and beta-lactamase inhibitor, in patients with carbapenem-resistant Acinetobacter baumannii infections, found the novel combo to be statistically noninferior to colistin in 28-day all-cause mortality, the study's primary endpoint.
Phase II results from Atea Pharmaceuticals Inc. with AT-527 in COVID-19 treatment had pundits scrambling to stack the odds of success for the direct-acting oral antiviral against those of Merck & Co. Inc.’s molnupiravir, though cross-trial comparisons are beset by the usual hurdles, with two especially dramatic ones.
RSS
