Biogen Inc. (BIIB) has announced an important breakthrough on Wednesday, revealing positive top-line data from Part B of the Phase 2/3 DEVOTE study. This study evaluates a higher dosage regimen of nusinersen in infants suffering from spinal muscular atrophy (SMA).
The study’s primary endpoint was successfully achieved at the six-month mark, demonstrating a statistically significant improvement in motor function among infants who received the higher dosage regimen compared to untreated sham controls from the Phase 3 ENDEAR study. The ENDEAR study is one of the crucial trials that contributed to the regulatory approval of nusinersen, branded as Spinraza, which is currently marketed for the treatment of SMA in a 12 mg dose.
The advanced dosage regimen of nusinersen features an accelerated loading phase, consisting of two 50 mg doses administered 14 days apart, followed by a higher maintenance dosage of 28 mg every four months, a significant increase from the approved 12 mg Spinraza regimen.
"The encouraging top-line results from the DEVOTE study indicate that the higher dosage regimen can more rapidly slow neurodegeneration, as evidenced by substantial reductions in neurofilament levels by day 64 relative to the approved dose. Over time, this higher dosage regimen has provided meaningful clinical benefits in infants with symptomatic SMA," stated Stephanie Fradette, Head of the Neuromuscular Development Unit at Biogen.
