Patricia Inácio, PhD, science writer —

ASHA-624, an experimental therapy that blocks the activity of the SARM1 protein, will be developed as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS). Developer Asha Therapeutics said it will conduct preclinical studies aimed at supporting the treatment’s potential for testing in human clinical trials. “As a veteran CNS…

The U.S. Food and Drug Administration (FDA) has lifted a partial clinical hold that limited the use of certain dose levels of prosetin being tested in ProJenX’s Phase 1 trial. The PRO-101 trial (NCT05279755) is a three-part study designed to test the oral medication in healthy volunteers and…

Spanish pharmaceutical company Ferrer will co-develop and market Verge Genomics’ investigational PIKfyve inhibitor VRG50635 for the treatment of amyotrophic lateral sclerosis (ALS) in Europe, Central and South America, Southeast Asia, and Japan, under a collaboration agreement announced by the two companies. Ferrer will have exclusive co-development and commercialization…

The ALS Association awarded $400,000 to University of Missouri-Columbia School of Medicine and its NextGen Precision Health Initiative as part of its program to encourage more amyotrophic lateral sclerosis (ALS) patients living in rural areas to take part in clinical trials. The university’s 2023 Trial Capacity Award…

Axoltis Pharma‘s new treatment candidate for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases was deemed safe and well tolerated at multiple doses in healthy volunteers who participated in a Phase 1b trial. Findings from the trial and preclinical studies also indicate NX210c is working as intended, boosting…

VectorY Therapeutics has raised about $138 million in funding aiming to bring into clinical testing VTx-002, its lead treatment candidate for amyotrophic lateral sclerosis (ALS). The therapy equips nerve cells to produce antibodies against the damaging TDP-43 protein clumps that are an ALS hallmark. “The investment…

The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study. The Phase 3 trial of PrimeC is due to start…

Pasithea Therapeutics has selected a lead candidate for its PAS-003 program, which is intended to target a “velcro-like” protein to treat amyotrophic lateral sclerosis (ALS). The new therapy is a humanized monoclonal antibody that potently inhibits the alpha-5/beta-1 integrin protein. It was selected after extensive screening and characterization studies…

Treatment with Relyvrio (sodium phenylbutyrate and taurursodiol) in the CENTAUR trial significantly extended the median survival — by 10.4 months — of amyotrophic lateral sclerosis (ALS) patients compared with an historical patient control group, a post-hoc analysis of study data reported. This finding more than doubles the 4.8…

The National Institutes of Health (NIH) has awarded about $45.1 million toward an expanded access program (EAP) of CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS). The EAP, commonly referred to as compassionate use, will make CNM-Au8 available to U.S. patients who aren’t eligible for clinical…