News

FDA Committee, in Reversal, Favors AMX0035 Approval for ALS

In a reversal, an advisory committee of the U.S. Food and Drug Administration (FDA) now says that the current evidence of efficacy of Amylyx Pharmaceuticals’ AMX0035 is sufficient to support its approval for amyotrophic lateral sclerosis (ALS). The committee voted 7–2 in favor of AMX0035, Amylyx announced…

Engensis Gene Therapy Found Safe in Small Phase 2a Trial

Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…

Treg Therapy Found Safe, Effective at Slowing ALS in Small Trial

Treating patients with their own immune regulatory T-cells (Tregs), in combination with IL-2 — a protein that boosts the cells’ immunosuppressive function and survival — is generally safe and shows potential to slow disease progression in people with amyotrophic lateral sclerosis (ALS). That’s according to a small Phase…

Cholesterol Transporter May Be Strongest Trigger of Sporadic ALS

The cerebrospinal fluid of people with sporadic amyotrophic lateral sclerosis (sALS), but not of those with familial forms of the disease, promoted ALS-specific molecular, cellular, and motor abnormalities in healthy mice, a study showed. Apolipoprotein B-100 (apoB100), a protein involved in cholesterol transport, was identified as the main…

Masitinib Again Under Review in EU as Potential ALS Treatment

The European Medicines Agency (EMA) has agreed to review an application seeking the conditional approval of AB Science’s masitinib, now Alsitek, for the treatment of amyotrophic lateral sclerosis (ALS). Conditional approval is granted to a medication whose immediate availability fulfills an unmet medical need, when its preliminary benefits are…