BIOGEN publishes study results for high-dose nusinersen in Nature Medicine

Biogen Inc. (NASDAQ: BIIB) announced that Nature Medicine published results from the Phase 2/3 DEVOTE study evaluating a high-dose regimen of nusinersen for spinal muscular atrophy. The study tested 50 mg loading doses and 28 mg maintenance doses compared to the currently approved 12 mg regimen marketed as SPINRAZA.

The DEVOTE study enrolled 139 participants across ages and SMA types in three parts. In the pivotal cohort, treatment-naïve infants receiving the high-dose regimen showed statistically significant improvements in motor function measured by the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders compared to an untreated control group, with a mean difference of 26.19 points.

The high-dose regimen demonstrated more rapid reduction in neurofilament, a marker of neurodegeneration, compared to the 12 mg dose. In Part C of the study, 40 participants aged 4-65 who transitioned from the 12 mg regimen after a median of 3.9 years experienced improvements in motor function, with mean increases of 1.8 points on one scale and 1.2 points on another at Day 302.

The safety profile of the high-dose regimen was consistent with the known safety profile of the 12 mg dose. Common adverse events in the infantile-onset cohort included pneumonia, respiratory failure, fever, COVID-19, and upper respiratory tract infection.

The high-dose regimen is approved in the European Union and Japan. The U.S. Food and Drug Administration has set an action date of April 3 for its review under the Prescription Drug User Fee Act.

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