Biogen highlights ‘promising’ results for higher-dose nusinersen in SMA trials

Biogen (BIIB) announced new data that it says reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy. These latest findings from Part C of the DEVOTE trial evaluating a higher dose regimen of nusinersen and the NURTURE trial which evaluated the approved 12 mg regimen — SPINRAZA — in clinically presymptomatic SMA were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. Biogen’s applications for the higher dose regimen of nusinersen are currently under review in the U.S., Europe, Japan and other global markets. 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 four months. Detailed results from Part C of the DEVOTE study highlight the potential clinical benefits of an investigational higher dose of nusinersen in a broad range of individuals who had been previously treated with nusinersen at the approved 12 mg dose for approximately four years. Participants were 4 to 65 years of age and half were ambulatory. Participants in Part C received one loading dose and two maintenance doses of open-label higher dose nusinersen during the study period. Most participants experienced improvements on the Hammersmith Functional Motor Scale – Expanded, Revised Upper Limb Module, and/or Clinical Global Impression of Change after transitioning to the higher dose regimen. These improvements were observed across phenotypes, functional status and age. For example, non-ambulatory participants improved by +2.5 on average on the HFMSE, and ambulatory participants improved by +1.1. At the study conclusion, all children who participated in NURTURE were alive and experienced continued clinical benefits over the course of the study. No participants required permanent ventilation, and the majority went without any ventilatory support throughout the study. Ninety-two percent of participants achieved the ability to walk independently, many within normal developmental timeframes. Participants with elevated levels of neurofilament light chain at baseline experienced rapid and sustained reductions in NfL after initiation of nusinersen, reinforcing the potential utility of NfL as an objective biomarker of disease activity and treatment response in SMA.