Infants benefit from Nusinersen/Spinraza in Spinal Muscular Atrophy

– Initial Phase 3 Safety Data Presented in Infantile-onset Spinal Muscular Atrophy Demonstrated Favorable Safety Profile with no Treatment Related Adverse Events

CAMBRIDGE, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–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) in the late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. The presentations included safety results from the interim analysis of the Phase 3 ENDEAR study in infantile-onset SMA (most likely to develop Type 1), encouraging preliminary results from NURTURE, a Phase 2 open-label study in pre-symptomatic infants, and a recent analysis of the ongoing Phase 2 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.”

Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen. Submission of the Marketing Authorization Application to the European Medicines Agency is planned in the next week. Biogen will initiate regulatory filings in other countries in the coming months.

Interim Data from Phase 3 ENDEAR Trial
In the interim analysis of the controlled, Phase 3 ENDEAR study in infantile-onset SMA, infants treated with nusinersen demonstrated a statistically and clinically significant improvement in the primary endpoint (p<0.0001), defined as the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). A responder was defined as a patient who improved in more motor milestone categories (kicking, head control, rolling, sitting, crawling, standing, walking) than worsened.

Nusinersen was generally well-tolerated, with an acceptable safety profile. No adverse events (AEs) were considered related to treatment. Based on the results of the positive interim analysis, patients who elect to do so will be transitioned to SHINE, an open-label extension study, in which they will be able to receive nusinersen. Following the transition of patients, the ENDEAR study will close. Detailed efficacy data will be presented at a future medical conference once participants complete their final study visit in ENDEAR.

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