Novartis halts ALS drug after mid-stage trial failure

  • Novartis discontinued VHB937 development after its ALS mid-stage study missed primary and secondary goals.
  • 251 patients were enrolled in the early-stage ALS trial within two years of symptom onset.
  • Novartis continues recruiting Alzheimer’s patients for a separate mid-stage trial of the drug.

Novartis discontinued development of VHB937, also known as lifonebart, after the experimental drug failed to meet its main and secondary goals in a mid-stage trial involving patients with amyotrophic lateral sclerosis (ALS), a progressive disease that impairs nerve-to-muscle communication. The setback adds to investor concerns about Novartis’ pipeline after an earlier heart-drug setback, last week’s late-stage failure of a muscle-wasting drug and the company’s halt of eight of 10 studies for its rap-cel cell therapy this month following three patient deaths. The ALS study enrolled 251 people with symptoms that began within two years and tested VHB937’s efficacy and safety. The drug is designed to stabilize TREM2, a protein involved in immune responses, inflammation and waste clearance in the brain. Novartis continues to study VHB937 in Alzheimer’s disease, with clinicaltrials.gov showing recruitment for a mid-stage trial. The company’s U.S.-listed shares were marginally higher.

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