Novartis halts ALS drug VHB937 after phase 2 trial fails to meet endpoints

Novartis announced it is stopping development of its ALS candidate VHB937 after the phase 2 Astrals trial did not meet its primary or secondary endpoints.

The study enrolled 251 patients with early‑stage ALS and compared the TREM2 stabilizer/activator to placebo, but the results showed no clinical benefit.

The same molecule, also called lifonebart, continues in a separate phase 2 trial for Alzheimer’s disease, where target validation for TREM2 is considered stronger.

Analysts said the failure adds to a string of disappointing outcomes for TREM2‑targeting drugs, underscoring the challenges in neurodegeneration research.