Patricia Inácio, PhD,  science writer—

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…

An advisory committee of the European Medicines Agency has maintained its recommendation not to approve AMX0035 for amyotrophic lateral sclerosis (ALS). In its initial assessment issued in June, the Committee for Medicinal Products for Human Use (CHMP) said data from the CENTAUR Phase 2 trial (NCT03127514), which formed…

The Barrow Neurological Institute has received $16.7 million from the National Institutes of Health (NIH) to help coordinate a nationwide repository of clinical data of people with amyotrophic lateral sclerosis (ALS). The Access for All in ALS (ALL ALS) Consortium will serve as a repository of biological samples and clinical…

PrimeC, NeuroSense Therapeutics‘ investigational, fixed-dose combination therapy for amyotrophic lateral sclerosis (ALS), significantly boosted the survival of patient-derived motor neurons to levels similar to healthy controls in a lab study. The independent study was led by Justin Ichida, PhD, a professor of stem cell biology and regenerative…

Amyotrophic lateral sclerosis (ALS) patients not eligible to participate in clinical trials of pridopidine — a small molecule developed by Prilenia Therapeutics to slow disease progression in ALS — will soon be able to receive the experimental therapy via an expanded access program (EAP). EAPs, also known…