Novartis ends ALS drug development after mid-stage trial failure

• The drug failed to stabilize TREM2 protein in early-stage ALS patients within two years of symptom onset, adding to a pattern of TREM2 drug candidate failures across the industry
• Novartis continues to study the same drug for Alzheimer's disease, with mid-stage patient recruitment ongoing despite the ALS trial failure
• The failure intensifies investor concerns following multiple recent setbacks, including three patient deaths that led to halting eight of 10 studies for the company's rap-cel cell therapy

Novartis Halts ALS Drug Development After Mid-Stage Trial Failure

Novartis has discontinued development of its experimental amyotrophic lateral sclerosis (ALS) drug, lifonebart (VHB937), following failure to meet primary and secondary endpoints in a mid-stage clinical trial. The study, which enrolled 251 patients with early-stage ALS (within two years of symptom onset), evaluated the drug's efficacy and safety.

Key Details:

• The drug aimed to stabilize TREM2, a protein regulating immune responses, inflammation, and waste clearance in the brain
• This marks the latest in a series of setbacks for the Swiss pharmaceutical company in 2026
• Recent failures include a heart drug disappointment, late-stage failure of a muscle-wasting treatment, and the halt of eight out of ten studies for its rap-cel cell therapy following three patient deaths

Market Impact:

Following the announcement on Wednesday, September 16, Novartis's U.S.-listed shares declined. The continuous stream of clinical failures has intensified investor scrutiny of the company's drug pipeline.

Broader Industry Context:

TREM2-targeted drug development has proven challenging across the pharmaceutical industry. Competitor Alector also reported a mid-stage trial failure for its TREM2 candidate in 2024, highlighting the difficulty of this therapeutic approach.

Ongoing Development:

Novartis continues to study VHB937 in Alzheimer's disease patients, with mid-stage trials currently in the recruitment phase according to clinicaltrials.gov. However, the ALS indication has been completely abandoned.

ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative condition affecting nerve-muscle communication, leading to worsening muscle weakness over time.