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Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
Positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA; Biogen plans to submit for
About this update from Biogen Inc.
[{"type":"text","content":"Positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA; Biogen plans to submit for regulatory approval of this investigational dose regimenHigher dose nusinersen regimen showed statistically significant improvement compared to a prespecified matched sham control groupAmong key measures of clinical efficacy, higher dose regimen showed positive trends compared to the approved dosing regimen CAMBRIDGE, Mass., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a higher dose regimen of nusinersen in treatment-naïve, symptomatic infants with spinal muscular atrophy (SMA). The investigational higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and a higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA). The study met its primary endpoint at six months, achieving a statistically significant improvement in motor function in infants who received the higher dose regimen as compared to a prespecified matched sham (untreated) control group from the ENDEAR study. “While there has been remarkable progress in the treatment of SMA, there remains significant unmet need. Building on the well-characterized profile of SPINRAZA established over the past 10 years, we continue to explore the potential for maximizing efficacy outcomes while maintaining our commitment to safety,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. “The encouraging topline results from DEVOTE show that the higher dose regimen can slow neurodegeneration faster, as shown by greater reductions in neurofilament at day 64 relative to the approved dose. Over time, the higher dose regimen led to meaningful clinical benefit in infants with symptomatic SMA. We look forward to sharing the detailed results with the SMA community and health authorities.” DEVOTE is a three-part study that enrolled 145 patients across ages and SMA types. The pivotal Part B cohort was comprised of treatment-naïve children with infantile-onset SMA (n=75) who were randomized 2:1 to receive the investigational higher dose regimen of nusinersen or the...