New data support benefits of nusinersen in spinal muscular atrophy

Positive new data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen (Nasdaq: BIIB) and Ionis Pharmaceuticals (Nasdaq: IONS) were presented at the 2016 World Muscle Society Congress in Granada, Spain.

The presentations included safety results from the interim analysis of the Phase III ENDEAR study in infantile-onset SMA (most likely to develop Type 1), encouraging preliminary results from NURTURE, a Phase II open-label study in pre-symptomatic infants, and a recent analysis of the ongoing Phase II open-label study in patients with later-onset SMA (consistent with Types 2 or 3).

“We continue to be encouraged by the consistently positive results with nusinersen across our clinical program, including our first data in infants treated before they show signs of the disease,” said Wildon Farwell, senior director SMA clinical development at Biogen. “NURTURE is the first study to evaluate an investigational therapy in pre-symptomatic infants genetically at risk for SMA. In this analysis, infants treated for up to one year achieved motor milestones in timelines more consistent with normal development than what is observed in the natural history of patients with Type 1 SMA,” he noted.