Community update from Biogen
We are happy to share the latest Community Statement issued by Biogen about the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a higher dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA).
Today Biogen announced topline results from Part B of DEVOTE:
• The study met its primary endpoint, achieving a statistically significant improvement in motor function, assessed using the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), from baseline to six months for the higher dose cohort as compared to a matched untreated control group from the Phase 3 ENDEAR study;
• Results favored the higher dose regimen relative to the untreated control group across secondary endpoints and trended in favor of the higher dose regimen over the currently available 12 mg regimen among key biomarker and efficacy measures; and
• The higher dose regimen was generally well tolerated with reported adverse events generally consistent with SMA and the known safety profile of nusinersen.