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.
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.
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).
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.
“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”
Akava Therapeutics Inc. has announced FDA clearance of its IND application for AKV-9 (formerly NU-9) for the treatment of amyotrophic lateral sclerosis (ALS). The company is planning a first-in-human phase I study in healthy subjects.
Researchers from Oregon Health and Science University presented data from a study that aimed to identify and validate potential new therapeutic targets for the treatment of amyotrophic lateral sclerosis (ALS). Systems immunology analysis utilizing mass cytometry (CyTOF) was used to assess α5 integrin expression in 78,293 single cells, including 21,250 CD45+/CD11b+ myeloid cells from CNS of superoxide dismutase-1 (SOD) G93A mouse model of ALS (SOD1G93A).
Insilicotrials Technologies SpA has entered into a collaborative partnership with Axoltis Pharma SA in the field of central nervous system (CNS) diseases, with the aim of optimizing the clinical development plan for Axoltis’ drug candidate, NX-210c.
When a group of British scientists studied which proteins might be in the wrong place of the cell in amyotrophic lateral sclerosis (ALS) patients, they found hundreds of them mislocalized. Other studies had shown that TDP-43 protein was mislocalized. But it was not known that the phenomenon was widespread, and affected mRNA as well as proteins. “Our study revealed that these mislocalized proteins were heavily involved in RNA binding functions and exhibited high binding affinities to RNAs,” Rickie Patani told BioWorld.
Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.
