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Novartis ALS Drug Fails Mid-Stage Study
16 Sep
Summary
- Novartis halts development of experimental ALS drug VHB937.
- The drug failed to meet primary and secondary goals in trials.
- This adds to a string of recent setbacks for Novartis' pipeline.
Novartis has halted development of its experimental drug VHB937, intended to treat amyotrophic lateral sclerosis (ALS). The decision was made after the drug failed to meet both primary and secondary objectives in a mid-stage clinical trial. This setback marks the latest in a series of pipeline challenges for the Swiss pharmaceutical company.
The VHB937 drug, also referred to as lifonebart, was being investigated for its potential to stabilize TREM2, a protein crucial for regulating brain immune responses and waste clearance. The mid-stage trial evaluated the drug's efficacy and safety in patients with early-stage ALS, specifically within two years of symptom onset. The trial enrolled 251 patients.
This development adds to recent disappointments for Novartis, including the failure of a heart drug and a muscle-wasting drug in late-stage studies. Earlier this month, the company also paused eight out of ten studies for its rap-cel cell therapy due to three patient deaths. These ongoing issues have led to increased scrutiny from investors regarding Novartis' drug pipeline.