Biogen Reports Positive Topline Data From DEVOTE Study Of Higher Dose Regimen Of Nusinersen In SMA

(RTTNews) - Biogen Inc. (BIIB) Wednesday announced positive, topline data from the Part B of the Phase 2/3 DEVOTE study evaluating a higher dose regimen of nusinersen in infants with spinal muscular atrophy (SMA).

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 untreated sham control group from the Phase 3 ENDEAR study. ENDEAR is one of the two pivotal studies that formed the basis of regulatory approval for Nusinersen. Nusinersen 12 mg is marketed under the brand name Spinraza for the treatment of SMA.

The 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 12 mg regimen Spinraza.

"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," said Stephanie Fradette, Head of the Neuromuscular Development Unit at Biogen.