Lindsey Shapiro, PhD, science writer —

A Phase 1/2 clinical trial is investigating the long-term safety and tolerability of BIIB105, Biogen‘s investigational treatment targeting the ataxin-2 protein, in people with amyotrophic lateral sclerosis (ALS). Called ALSpire (NCT04494256), the trial is currently recruiting adults with ALS at 12 sites in the U.S., Canada, and…

The investigational cell-based therapy NurOwn may slow disease progression in people with amyotrophic lateral sclerosis (ALS) who have less severe disease, according to analyses from a Phase 3 trial. Researchers’ analyses excluded patients with the lowest scores on the ALS Functional Rating Scale–Revised (ALSFRS-R) or in individual…

Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…

Proposed updates to the National ALS Registry’s case-identification algorithm and classifications may help provide a better estimate of the prevalence of amyotrophic lateral sclerosis (ALS) in the U.S., a study reported. Under the revision, individuals previously classified as “possible ALS” cases and excluded from the registry would now…

Dazucorilant, Corcept Therapeutics‘ investigational cortisol modulator for people with amyotrophic lateral sclerosis (ALS), is being evaluated in a Phase 2 clinical trial. The DAZALS Phase 2 study (NCT05407324) aims to determine dazucorilant’s safety and efficacy against a placebo in about 198 adults with ALS. It will be…

The U.S. Food and Drug Administration (FDA) is extending by three months its review of tofersen, Biogen’s investigational treatment for forms of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. Earlier this summer, the FDA granted the application priority review, with a decision expected no…

An extended-release formulation of PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS), demonstrated a favorable pharmacokinetic profile in healthy adults in a Phase 1 trial, helping with an ongoing Phase 2b study and work into a potential pivotal trial in ALS patients. A pharmacokinetic (PK) study aims…

NurOwn, Brainstorm Cell Therapeutics’ investigational cell-based therapy, leads to meaningful disease-related biomarker changes regardless of amyotrophic lateral sclerosis (ALS) severity at the start of treatment, according to data from a Phase 3 trial. Specifically, the therapy increased neuroprotection biomarker levels and anti-inflammatory molecules, and reduced those of neurodegeneration…

The COURAGE-ALS Phase 3 trial of Cytokinetic’s experimental therapy reldesemtiv in people with amyotrophic lateral sclerosis (ALS) will continue as planned following its first planned interim analysis. A data monitoring committee met to review the trial’s data so far with the goal of assessing futility, or whether the…

Biohaven‘s therapy candidate verdiperstat did not significantly slow functional decline among people with amyotrophic lateral sclerosis (ALS) in the HEALEY ALS platform trial, failing to meet its primary goal, data show. Key secondary efficacy goals, such as survival and changes in lung function and muscle strength, also were…