An interim analysis of the open-label study of simufilam, Cassava Sciences Inc., of Austin, Texas, showed six months’ treatment with the oral, small molecule improved cognition and behavior scores in Alzheimer’s disease patients. Cognition scores rose 10% from baseline to the sixth month of treatment. Anxiety, delusions and agitation, all dementia-related behavior, showed a 29% mean improvement in the same period. Cassava said the positive data are enough to propel simufilam into a phase III study sometime in the second half of 2021. Share prices of company stock (NASDAQ:SAVA) have languished in the single digits during the past 12 months but the new data put an end to that. At midday, shares were up 125%.

Immunovant halts TED dosing after lipid rise, plans to revise monitoring

Shares of New York-based Immunovant Inc. (NASDAQ:IMVT) sank $18.59, or 42%, to trade midday at $24.71 on word that the company has voluntarily paused dosing in its trials with neonatal Fc receptor-targeting IMVT-1401. Researchers found elevated total cholesterol and LDL levels in IMVT-1401-treated patients in Ascend Go-2, a phase IIb experiment in thyroid eye disease (TED). Cholesterol levels were not measured in prior clinical trials testing IMVT-1401 against myasthenia gravis and in healthy subjects, the company said. “Out of an abundance of caution,” investigators decided to hold up dosing in TED as well as warm autoimmune hemolytic anemia so that patients, investigators and regulators can be informed of the latest discovery while Immunovant adjusts the monitoring program.

Prothena brings AL amyloidosis drug back to clinic with new focus

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:PTRA) 31.8% higher by midday. Under an FDA special protocol assessment, it's planning to launch a pivotal phase III trial this year that could enable registration of the therapy if it shows a statistically significant improvement on a primary endpoint of all-cause mortality in high risk, Mayo stage IV, patients.

Sputnik V vaccine efficacy confirmed, as developers tout safety, ease of distribution

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 last December. The phase III trial also used a liquid formulation of Sputnik V which can be stored at refrigerator temperatures, making it suitable for distribution through regular supply chains. It is priced at less than $10 per dose.

For head and neck cancer, it's all about making Keytruda better

With the approval of Keytruda (pembrolizumab, Merck & Co. Inc.) in the front-line setting, the head and neck cancer 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. BioWorld takes a closer look at the drugs in development for head and neck cancer, including an aspartate beta-hydroxylase modulator from Sensei Biotherapeutics Inc., a CD47 inhibitor from Alx Oncology Inc., a Toll-like receptor 9 agonist from Checkmate Pharmaceuticals Inc., an immunotherapy of HPV16 antigens from PDS Biotechnology Inc. and a SPEAR T-cell therapy targeting MAGE-A4 from Adaptimmune Therapeutics Inc.

A year since the COVID-19 wallop, research moves furiously forward

As solid efficacy data are reported for at least four more front-runner vaccines, and while biopharma companies join arms with combination antibodies, COVID-19 variants continued to emerge and spread throughout January. Countries are racing to vaccinate, circumventing supply and distribution obstacles, and none of the research is slowing. The U.S. government also is determined to make sure another pandemic never again takes the world by surprise. Global deaths are up to 2.2 million out of about 102.4 million confirmed cases, according to the World Health Organization, and at least 865 therapeutics and vaccines have entered development in the last year to fight the SARS-CoV2 virus.

Biocryst gets Japanese approval for hereditary angioedema treatment

HONG KONG – Japan’s Ministry of Health, Labor and Welfare has granted approval for Biocryst Pharmaceuticals Inc.’s Orladeyo (berotralstat), the first and only drug for prophylactic treatment of hereditary angioedema 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. Tokyo-based Orphanpacific Inc., Biocryst’s representative in Japan, holds the marketing authorization.

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