Novartis has stopped development of VHB937, also known as lifonebart, for amyotrophic lateral sclerosis after the experimental treatment missed the primary and secondary goals of a mid-stage trial. The company confirmed the decision to Reuters on 16 September. ALS affects communication between motor neurons and muscle and leads to progressive weakness; treatment options remain limited.

The compound targeted TREM2, a protein involved in regulating immune responses in the brain. The hope was that modifying this pathway might slow damage. The required benefit over placebo was not shown, and the ALS programme is ending. Novartis continues to assess the same compound in Alzheimer’s disease — a separate programme, not a continuation of the ALS study.

A failed trial does not prove the biological target is useless in every setting. It shows the distance between a laboratory mechanism and a medicine that changes the course of disease. People with ALS should not change treatment because of this report; decisions remain with their care team.

The US institute NINDS describes ALS as a neurodegenerative disease without a current cure. The mid-stage failure adds to a long series of drug attempts that have not moved from a signal to a clear clinical benefit.

Photo: a researcher at a microscope, an illustrative image, not a VHB937 procedure. Via Wikimedia Commons.

Source consulted: Reuters — Novartis halts ALS drug development; NINDS — amyotrophic lateral sclerosis.