Page last updated: 10th September 2024

This phase 2/3 study, which enrolled 145 patients across all ages and SMA types, consists of 3 parts:

• An open-label safety evaluation cohort (part A)
• A pivotal, double-blind, active control randomised treatment cohort (part B) and
• An open-label treatment cohort (part C)

In Part B, 75 treatment-naïve children with infantile-onset SMA received either:

• the higher dose regimen of nusinersen – which comprises a more rapid loading regimen, two 50 mg doses 14 days apart and a higher maintenance regimen of 28 mg every four months – or
• the currently approved nusinersen 12 mg regimen (comprising four loading doses followed by maintenance doses every four months).

The main findings of this study were:

• Significant improvements in motor function were observed in the higher dose group, as measured by the CHOP-INTEND test, from the start to six months. These improvements were compared to a similar untreated control group from the Phase 3 ENDEAR study.
• Results favoured the higher dose regimen relative to the untreated control group from the ENDEAR study across secondary endpoints. They also showed a positive trend towards the higher dose regimen over the currently approved 12 mg regimen among key biomarker and efficacy measures;
• The higher dose regimen was generally well tolerated with reported side-effects similar to those seen in SMA and nusinersen’s known safety profile.

Detailed results from DEVOTE will be presented at upcoming medical conferences and Biogen plans to submit for regulatory approval of this investigational dose regimen in the UK.

Biogen’s Press Release >