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.
Thanks to positive phase III data from Phathom Pharmaceuticals Inc.’s Phalcon-EE study of patients with erosive esophagitis, the company is prepping to file an NDA with the FDA for its lead compound, vonoprazan.
The FDA’s Vaccines and Related Biological Products Advisory Committee unanimously agreed to recommend a booster dose of Janssen Biotech Inc.’s COVID-19 vaccine for those age 18 years and older at least two months following the first dose. In the end, the vote swung on numbers from Janssen showing a booster significantly improved efficacy among study participants. Janssen’s data leaped to 94% protection after a booster dose, up from 70% efficacy following the first dose. That higher efficacy rate nearly matches that of the mRNA vaccines from Moderna Inc. and Pfizer Inc.-Biontech SE.
Jonathan Moore, who was employee No. 36 when he came to Vertex Inc. in 1990 and ended up spending the next 28 years at the company, realized he wasn’t done with his work in ATP-binding cassette (ABC) transporters and has now formed Rectify Pharmaceuticals Inc. The new company just closed on a $100 million series A financing to fund development of a pipeline of therapies to restore ABC transporter function for treating genetic diseases.
Biogen Inc. opened the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting with data showing those treated with its multiple sclerosis (MS) therapies had an effective antibody response to COVID-19 vaccination.
Bumetanide, a diuretic pill commonly used for treating fluid retention for those with heart, kidney or liver disease, showed a significantly lower prevalence of Alzheimer’s disease in its users than those not taking it.
Pacira Biosciences Inc. will add to its core non-opioid-based therapies business by buying Flexion Therapeutics Inc. for $427 million. That brings into the company’s fold Zilretta (triamcinolone acetonide extended-release injectable suspension) for treating osteoarthritis knee pain, an injectable non-opioid that’s already FDA approved in addition to being in a phase II study for treating shoulder osteoarthritis.
A lack of toxicity and the shrinking of tumors were at the heart of new interim data produced from a two-stage phase II study by Relay Therapeutics Inc. The company reported that the oral small molecule RLY-4008, designed to elicit responses across a broad spectrum of resistance mutations and in multiple tumor types, selectively inhibited fibroblast growth factor receptor (FGFR) 2 in ways that are not limited by off-target toxicities of hyperphosphatemia (FGFR1) and diarrhea (FGFR4).
Data from two phase I studies from Prelude Therapeutics Inc. helped open the American Association for Cancer Research virtual International Conference on Molecular Targets and Cancer Therapeutics on Oct. 7 but tugged the company’s stock sharply downward.
Privately held Retrotope Inc., the only company currently in the clinic studying the ultra-rare and fatal neurological disorder infantile neuroaxonal dystrophy, has new data it hopes to present to the FDA early next year.
