News

Phase 2 Trial Will Test Cortisol Modulator Dazucorilant in ALS

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…

‘Good Cholesterol’ Protein Improved Blood Vessel Cell Health

A cholesterol-related protein, called apolipoprotein A1 (ApoA1), can prevent the death of endothelial cells — those lining blood vessel walls — in a cell model of amyotrophic lateral sclerosis (ALS), a recent lab study shows. According to researchers, these results “show promise for ApoA1 as a therapeutic agent to…

FDA Extends Tofersen Review Period to April 2023

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…

Trial Planned Into Digital, Home Monitoring of ALS Progression

The nonprofit EverythingALS is teaming up with several biotech companies for a study into the feasibility of monitoring amyotrophic lateral sclerosis (ALS) progression in people via disease-specific biomarkers captured digitally at the home. The study will be open to adults with an ALS diagnosis or for whom ALS…

Biomarkers Boosted by NurOwn Regardless of Disease Severity: Data

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…

First Patients Dosed in Eye Study of Tracer for ALS Diagnosis

The first patients have been dosed in a Phase 1/2a clinical study that’s testing AMDXP-2011P for its ability to trace disease-associated toxic protein clumps in the eyes of adults with amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, which may allow earlier diagnosis. The study, dubbed PROBE (NCT05542576),…