Novartis halts ALS drug development after mid-stage trial failure

By Sriparna Roy

Sept 16 (Reuters) - Novartis NOVN.S has halted development of an experimental drug for amyotrophic lateral sclerosis after it failed to meet goals in a mid-stage study, marking the latest disappointment for the Swiss drugmaker this year.

This follows an earlier setback for a closely watched heart drug, last week's late-stage failure of a muscle-wasting drug, and the halt of eight of 10 studies for its rap-cel cell therapy this month after three patient deaths. These developments have intensified investor scrutiny of its pipeline.

Endpoints News and Bloomberg News reported the news earlier on Wednesday, which the company later confirmed in an emailed statement to Reuters, saying it was discontinuing development of the drug VHB937.

The drug, also called lifonebart, was being studied in patients with ALS, also called Lou Gehrig's disease. It is a neurodegenerative condition that affects how nerve cells communicate with muscles and leads to muscle weakness that worsens over time.

The company's U.S.-listed shares were marginally up.

The drug failed to meet both main and secondary goals in the mid-stage trial, which was evaluating the efficacy and safety of VHB937 in participants with early-stage ALS within two years of symptom onset.

The study had enrolled 251 patients, according to clinicaltrials.gov.

The drug aims to stabilize TREM2, a protein that helps regulate immune responses, inflammation and waste clearance in the brain.

Companies have been trying to develop TREM2 drug candidates but have faced failures. In 2024, Alector ALEC.O said its experimental drug failed in a mid-stage trial.

Novartis is also studying the drug in patients with Alzheimer's disease, a progressive brain disorder that slowly robs memory.

The clinical trials website shows the company is recruiting patients for the mid-stage trial.