Biogen Inc. (BIIB) released promising topline data from its pivotal Phase 2/3 DEVOTE study evaluating a higher dose regimen of nusinersen, a drug used to treat spinal muscular atrophy (SMA). The study focused on treatment-naive, symptomatic infants with SMA.
The investigational higher dose regimen involved a more rapid loading phase with two 50 mg doses administered 14 days apart. This was followed by a higher maintenance regimen of 28 mg every four months, compared to the currently approved 12 mg regimen (Spinraza).
The study successfully met its primary endpoint at six months. Infants receiving the higher dose regimen showed a statistically significant improvement in motor function compared to a pre-specified sham (untreated) control group from the ENDEAR study.
“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.
In addition to the primary endpoint, the results favored the higher dose regimen across secondary endpoints. They also trended positively in comparison to the 12 mg regimen on key biomarker and efficacy measures. Importantly, the higher dose regimen was generally well tolerated, with reported adverse events consistent with SMA and the known safety profile of nusinersen.
Spinraza, the currently approved nusinersen regimen, has been authorized in over 71 countries for the treatment of SMA in infants, children, and adults. More than 14,000 individuals worldwide have received Spinraza treatment.
The positive results from the DEVOTE study provide encouraging evidence that a higher dose regimen of nusinersen could offer significant benefits for patients with SMA. Further research and regulatory approval are required before the higher dose regimen can become widely available.
