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News
Novartis has discontinued development of lifonebart (VHB937), its experimental ALS drug, after it failed to hit both primary and secondary endpoints in a mid-stage trial of 251 patients. The setback is the latest in a rough stretch for the Swiss pharma giant, which has also seen failures in a heart drug, a muscle-wasting drug, and a cell therapy program. The TREM2-targeting drug class continues to struggle across the industry.
Novartis is closing the door on its ALS drug candidate lifonebart (VHB937) after the experimental therapy failed to meet both primary and secondary goals in a mid-stage clinical trial. The study enrolled 251 patients with early-stage ALS — the progressive neurodegenerative disease also known as Lou Gehrig's disease — within two years of symptom onset. The drug was designed to stabilize TREM2, a protein involved in regulating immune responses, inflammation, and waste clearance in the brain.
The failure adds to a difficult 2026 for Novartis, which has faced a string of pipeline setbacks including a late-stage heart drug failure, a muscle-wasting drug disappointment, and the suspension of eight out of ten studies for its rap-cel cell therapy following three patient deaths. The mounting setbacks have drawn sharp scrutiny from investors, with at least one major shareholder calling for a board shake-up.
Key Takeaways:
Why it matters: ALS remains a disease with very limited treatment options, and each pipeline failure narrows the field of hope for patients. The repeated stumbles of TREM2-targeting drugs suggest this mechanism may face fundamental challenges — a signal worth watching for researchers and clinicians alike.
