Novartis has discontinued development of VHB937 (lifonebart) for amyotrophic lateral sclerosis (ALS) after its Phase 2 ASTRALS study failed both primary and secondary endpoints. The 251-patient trial enrolled people with early-stage ALS.
VHB937 is an antibody intended to stabilize and activate TREM2 on microglia—immune cells in the brain and spinal cord—with the aim of slowing neurodegeneration. Reported endpoints included a composite of survival/ventilation-free status and ALS Functional Rating Scale–Revised change, plus respiratory-function and neurofilament-light measures.
The company’s ALS decision does not necessarily end the asset altogether:
a Phase 2 Alzheimer’s study of lifonebart was reported to be continuing, where the biological rationale for TREM2 is viewed as stronger.
The setback adds to a difficult recent stretch for Novartis R&D. Reports cite late-stage misses for pelacarsen, its Lp(a)-lowering cardiovascular drug, and del-desiran, a treatment for a muscle-wasting disease, as well as pauses in autoimmune CAR-T trials after three deaths.
Novartis said it would apply lessons from ASTRALS to further neurodegenerative-disease research. Detailed results from the ALS trial had not yet been publicly released in the reporting reviewed.
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