Positive interim analysis of Phase 3 studies in spinal muscular atrophy

Biogen and Ionis Pharmaceuticals announce positive results at interim analysis of two Phase 3 studies both trialling the drug nusinersen in patients with spinal muscular atrophy (SMA).

SMA is a rare, genetically inherited neuromuscular condition, affecting the lower motor neurones. It is caused by a fault in the SMN1 gene which results in a reduction in the production of SMN protein, causing the lower motor neurons in the spinal cord to deteriorate. Currently there is no cure for SMA.

Nusinersen is a drug which corrects this faulty gene expression. Two Phase 3 studies have been carried out by Biogen and Ionis Pharmaceuticals to investigate the safety and efficacy of nusinersen in patients with SMA.

The first study, ENDEAR, for which Great Ormond Street Hospital (GOSH) was the highest UK recruiter, studied nusinersen in the most severe form of SMA (SMA type 1), known as infantile-onset SMA. The findings at a pre-specified interim analysis showed that patients who received nusinersen displayed a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. These findings were replicated in the CHERISH trial, which investigated nusinersen in patients with later-onset SMA (consistent with Type 2).

Based on the results of the interim analysis, all participants from both trials can now elect to receive nusinersen by transitioning to the open label extension study, SHINE. SHINE is intended to evaluate the long-term safety and tolerability of nusinersen.

In addition to GOSH’s involvement in ENDEAR, GOSH will be involved in the expanded program in 2017.

Click here to access Biogen's website and read the full press release on the ENDEAR trial.

Click here to access Biogen's website and read the full press release on the CHERISH trial.