Novartis Halts Development of ALS Drug Rifonebart After Mid-Stage Trial Misses Primary Endpoint

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Swiss pharmaceutical giant Novartis has discontinued development of its candidate ALS treatment VHB937, also known as rifonebart, after a mid-stage clinical trial failed to achieve its primary endpoint. Endpoints News and Bloomberg News reported the news early on the 16th, and Novartis later confirmed the reports in an email to Reuters. Rifonebart failed to meet both its primary and secondary endpoints in the mid-stage trial, which evaluated the efficacy and safety of the drug in 251 patients with early-stage ALS within two years of symptom onset. The drug is designed to stabilize TREM2, a protein that helps regulate immune responses, inflammation, and the clearance of waste products in the brain. The decision to halt development follows the failure of a trial for a muscle-wasting disease treatment announced last week, in addition to setbacks with a closely watched heart disease treatment, and the company's move this month to pause eight of ten trials of its experimental cell therapy rap-cel after three patient deaths, heightening investor scrutiny of Novartis's development pipeline. Novartis is also pursuing research into the drug in patients with Alzheimer's disease, and according to a clinical trial registry, a mid-stage trial in Alzheimer's is still recruiting participants.

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Novartis halted development of ALS drug rifonebart after it failed primary and secondary endpoints in a mid-stage trial.

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