Novartis halts ALS candidate after Phase 2 miss
Novartis has discontinued development of its experimental ALS therapy VHB937/lifonebart following a Phase 2 trial failure. The decision ends work on the TREM2-targeting drug for amyotrophic lateral sclerosis, according to internal sources. The setback adds to challenges in finding effective treatments for the neurodegenerative disease.
The failure of VHB937/lifonebart in a Phase 2 trial marks another setback in the search for therapies against amyotrophic lateral sclerosis, a progressive neurodegenerative condition with few approved treatments. The drug, which targeted the TREM2 receptor—a protein involved in immune signaling in the brain—was being developed by Novartis. Its discontinuation underscores the difficulty of translating biological hypotheses into clinical benefit for ALS, where multiple mechanisms have shown promise in preclinical models but failed in human trials. The decision also reflects the high attrition rate in neurology drug development, where patient heterogeneity and unclear biomarkers complicate trial design. For researchers, the outcome adds to a growing list of late-stage failures, reinforcing the need for more precise patient selection and surrogate endpoints.
This setback may affect ALS patients and their families, who often face limited options and rely on a pipeline of experimental drugs. Each failed trial could reduce near-term hope for new treatments, though it may also sharpen focus on more promising targets or combination approaches. Investors and biotech firms could become more cautious about funding ALS research, potentially slowing innovation. However, the news may spur academic and industry collaboration to improve trial methodologies, ultimately benefiting future drug development across neurodegenerative diseases.