Biogen and Ionis report positive trial data for spinal muscular atrophy drug

Biogen and Ionis Pharmaceuticals have reported positive clinical data supporting the safety and efficacy of their new spinal muscular atrophy drug nusinersen.

The investigational treatment met its primary endpoint in the phase III ENDEAR trial, which evaluated nusinersen in infantile-onset spinal muscular atrophy and showed that infants receiving the drug experienced a statistically significant improvement in the achievement of motor milestones.

Based on the results of this interim analysis, the ENDEAR study will be stopped and participants will be able to transition into the SHINE open-label study, in which all patients receive nusinersen.

In response to this positive outcome, Biogen has exercised its option to develop and commercialise nusinersen globally and has paid Ionis a $75 million (57.05 million pounds) licence fee.

Biogen will now be responsible for all future development, regulatory and commercialisation activities and costs for this drug after Ionis completes the current phase III studies.

Dr Alfred Sandrock, executive vice-president and chief medical officer of Biogen, said: "We remain committed to understanding the potential of nusinersen in the broader spinal muscular atrophy population and will continue to focus on the rapid completion of our ongoing studies."