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New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals
About this update from Biogen Inc.
[{"type":"text","content":"Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen Investigational regimen also shows more rapid slowing of neurodegeneration, as measured by neurofilamentBiogen plans to submit regulatory applications around the world for approval of the nusinersen higher dose regimen CAMBRIDGE, Mass., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced detailed results from Part B and Part C of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of an investigational higher dose regimen of nusinersen in spinal muscular atrophy (SMA), showing benefits in both individuals previously treated and treatment-naïve to nusinersen with infantile-onset or later-onset SMA. The investigational, higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA®). Data to be presented during the World Muscle Society (WMS) 2024 Congress (Oct. 8-12, 2024 in Prague) highlight the potential of this investigational higher dose regimen to help address remaining unmet need in SMA. “Strikingly, the higher dose regimen lowered neurofilament more quickly, telling us that it’s more rapidly slowing neurodegeneration. We know how critical this is in people living with SMA. Over time, we see evidence of the benefit of the higher dose regimen across SMA phenotypes,” said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. “Despite administering much more drug, the higher dose regimen appears to have a consistent safety profile with the approved 12 mg regimen.” DEVOTE is a three-part study that enrolled 145 participants across ages and SMA types. The pivotal Part B cohort (n=75) met its primary endpoint where treatment-naïve, symptomatic infants who received the higher dose regimen saw significantly greater improvements in motor function as measured by Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) compared to a prespecified matched sham (untreated) group from the Phase 3 ENDEAR study (+15.1 vs -11.1, p","length":3041,"tagName":"div"}]