Novartis Discontinues ALS Drug VHB937 After Failing Mid-Stage Trial
Novartis Faces Setbacks in Drug Development Pipeline
By Sriparna Roy
ALS Drug VHB937 Development Halted
Sept 16 (Reuters) - Novartis 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.
Recent Drug Pipeline Challenges
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.
Confirmation and Reporting of Discontinuation
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.
About VHB937 and ALS
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.
Mid-Stage Trial Results
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.
Mechanism of Action: TREM2 Stabilization
The drug aims to stabilize TREM2, a protein that helps regulate immune responses, inflammation and waste clearance in the brain.
Industry-Wide Challenges with TREM2 Drugs
Companies have been trying to develop TREM2 drug candidates but have faced failures. In 2024, Alector said its experimental drug failed in a mid-stage trial.
Ongoing Research and Future Prospects
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.
(Reporting by Anusha Shah and Sriparna Roy in Bengaluru; Editing by Vijay Kishore)