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Biogen’s ALS Drug Fails to Attain Primary Endpoint but Offers Hope With Secondary Outcomes

by Rajaneesh K. Gopinath
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While Biogen is grappling with the slow rollout of its Alzheimer's drug Aduhelm over skepticism, it has now unveiled mixed results for its ALS drug.

On October 17th, Biogen announced that its investigational antisense drug, tofersen (BIIB067), did not meet its primary endpoint of treating amyotrophic lateral sclerosis (ALS) in Phase 3 VALOR study. However, the Cambridge, MA-based company claimed that it observed trends favoring tofersen across multiple secondary and exploratory measures of biologic activity and clinical function.

“The wait for new options has been long and difficult for the ALS community, and we welcome this important research advancement in this difficult to treat disease space,” said Timothy Miller, ALS Center Director at Washington University School of Medicine and Principal Investigator of the study.

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