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The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS

  • Publication includes the Phase 3 VALOR SOD1-ALS trial and its open-label-extension study, underscoring the importance of these longer-term data
  • 12-month data show earlier initiation of tofersen slowed decline across critical measures of function and strength in people diagnosed with SOD1-ALS
  • Results offer important learnings regarding the biology of SOD1-ALS and design of ALS clinical trials

Excerpt from the Press Release:

CAMBRIDGE, Mass., Sept. 21, 2022 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced that The New England Journal of Medicine (NEJM) has published detailed results from the Phase 3 VALOR study and the combined analysis of VALOR and its open label extension (OLE) study evaluating tofersen for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). There is currently no treatment targeted for SOD1-ALS.

“I see three key take home points from these data. First, tofersen clearly leads to lowering of SOD1 protein, as would be expected. Second there is substantial lowering of neurofilament levels, which I interpret as potentially slowing the underlying disease process. And third, there is a meaningful clinical benefit when looking at the later time points in the open label extension,” said Timothy Miller, M.D., Ph.D., principal investigator of VALOR and ALS Center co-Director at Washington University School of Medicine, St. Louis. “We are grateful to the dedication from participants, their families, and the sites for taking part in this important study.”

Data from the combined analysis were previously presented at the European Network to Cure ALS (ENCALS) annual meeting and included within Biogen’s New Drug Application for tofersen that was recently accepted for priority review by the U.S. Food and Drug Administration. The application was given a Prescription Drug User Fee Act action date of January 25, 2023.

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