HONG KONG – Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted approval for Biocryst Pharmaceuticals Inc.’s Orladeyo (berotralstat), the first and only drug for prophylactic treatment of hereditary angioedema (HAE) approved in the country. Biocryst’ partner, Torii Pharmaceutical Co. Ltd., will launch the drug after completing pricing negotiations with the Japanese National Health Insurance System (NHI). Tokyo-based Orphanpacific Inc., Biocryst’s representative in Japan, holds the marketing authorization.
Almost three years after announcing it would quit development of its humanized immunoglobulin G1 candidate, birtamimab, in light chain (AL) amyloidosis, Prothena Corp. plc is revving the program back up with a new focus, sending shares (NASADQ:PRTA) 30.5% higher to close at $14.35 on Feb. 2.
As solid efficacy data are reported for at least four more front-runner vaccines, and while biopharma companies join arms with combination antibodies, SARS-CoV-2 variants continue to spread and countries are racing to vaccinate. None of the research is slowing, and governments are determined to make sure another COVID-19 pandemic never again takes the world by surprise. Global deaths are up to 2.2 million, with 102.9 million confirmed cases, according to the World Health Organization. At least 865 therapeutics and vaccines to fight the virus have entered development in the last year.
With the FDA approval of Keytruda (pembrolizumab, Merck & Co. Inc.) in the front-line setting of metastatic or unresectable, recurrent head and neck cancer squamous cell carcinoma, the space has become a race to develop drugs using a variety of mechanisms of action to improve the efficacy of the anti-PD-1 monoclonal antibody.
When Cassava Sciences Inc.’s president and CEO, Remi Barbier, opened up the interim analysis of the company’s open-label study for Alzheimer’s disease candidate simufilam, he was more than pleased with the results but found himself an audience of one. His researchers had been confident about the results before the data were tallied. “I was ecstatic,” he told Bioworld. “No one’s seen this type of improvement in Alzheimer’s. But our scientists said, ‘See, I told you so.’ It was not my expectation.” The new data showed six months’ treatment with the oral, small molecule not just stabilized but improved cognition and behavior scores in AD patients.
Less than a month after informing the market of a slowdown caused by pandemic-related site closures in phase II studies with neonatal Fc receptor (FcRn)-targeting IMVT-1401, Immunovant Inc. said it’s voluntarily pausing dosing with the compound in thyroid eye disease (TED) in the study called Ascend Go-2 because of a worrisome physiological signal. The New York-based firm plans to investigate and, after seeking the FDA’s go-ahead on tweaks to the program, forge onward with development.
LONDON –The Russian COVID-19 vaccine Sputnik V now has validation from the Western science establishment, after The Lancet published full interim results of the phase III trial on Feb 2. The peer-reviewed paper confirms the 91%-plus efficacy that the vaccine’s developer, Gamaleya National Center of Epidemiology and Microbiology, claimed in its own announcement of the results in December.
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