Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
Arialys Therapeutics Inc. launched this month with $58 million in seed funding, an experimental compound it is developing for autoimmune encephalitis and autoimmune psychosis, and high aspirations for its field. “Yes, I want to treat these patients, I want these patients to have a better life. But I also want drug discovery and development folks to think differently about discovering new drugs for the CNS,” Jay Lichter told BioWorld.
Acurastem Inc. said on Sept. 25 that it struck an out-licensing deal potentially worth $580 million with Takeda Pharmaceutical Co. Ltd. to develop drugs for amyotrophic lateral sclerosis (ALS) and other PIKfyve gene-targeting therapeutics. Under the terms, Tokyo-headquartered Takeda obtains exclusive worldwide rights to Acurastem’s PIKfyve-targeting therapeutics, including Acurastem’s lead AS-202 asset, an antisense oligonucleotide therapy to treat ALS.
Thanks to a raft of new approvals by Japan’s Ministry of Health, Labor and Welfare (MHLW), patients in Japan will soon have access to Biogen Inc./Eisai Co. Ltd.’s Leqembi (lecanemab), an amyloid-beta binder, for slowing progression of mild cognitive impairment and mild dementia due to Alzheimer's disease.
Acurastem Inc. has entered into a license agreement with Takeda Pharmaceutical Company Ltd. to develop and commercialize Acurastem’s PIKFYVE-targeted therapeutics, including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of amyotrophic lateral sclerosis (ALS).
Orexia Therapeutics Ltd. has identified bicyclic-heterocycle derivatives acting as orexin OX2 receptor agonists. As such, they are reported to be useful for the treatment of narcolepsy, circadian rhythm disorder, genetic disorders, coma, metabolic syndrome, neurodegeneration, osteoporosis and sleep apnea, among others.
How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.
An artificial intelligence (AI) tool developed by Annalise.ai Pty Ltd. was able to improve radiologists’ accuracy in detecting abnormalities on non-contrast computed tomography brain (CTB) scans. According to a study published in European Radiology, the Annalise Enterprise CTB module improved radiologists' accuracy by 32% and reduced their overall reading time by 11%.
Light therapy during sleep helps increase resistance to Alzheimer’s disease (AD) progression, a study in Frontiers of Optoelectronics suggests. The researchers found that photobiomodulation (PBM) during deep sleep improved the ability of the lymphatic system to flush beta-amyloid from the brains of mice, demonstrating the importance of sleep in fending off the neurodegenerative disease and opening a possible new therapy for prevention and treatment of Alzheimer’s.
Scientists at the University of Chicago have discovered a signaling pathway guided by nicotinic acetylcholine receptors (nAChRs) that could relieve pain even when tolerance to opioids was present. This mechanism did not produce dependence after treatment and could be explored to develop new drugs.
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