New results from NUTURE study of Spinraza

Biogen announced new results from the NURTURE study adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with Spinraza in comparison to the natural history of this disease.

These new data also showed that patients treated with Spinraza had continuous improvement with the overwhelming majority of patients achieving motor milestones in a normal timeframe. These data are being presented at the Cure SMA Annual SMA Conference in Anaheim, CA (June 28-July 1, 2019) and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway (June 29-July 2, 2019).

Results from NURTURE, an ongoing, Phase II, open-label study of 25 pre-symptomatic patients with SMA (most likely to develop SMA Type 1 or 2) who received their first dose of Spinraza before six weeks old, demonstrated unparalleled results in comparison to the natural history of SMA. As of March 2019: 100 percent were alive without a need for permanent ventilation. The median age of the study participants was nearly three years old. The majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation.100 percent of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance. 88 percent of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently. Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function– 63.4 for patients with 3 SMN 2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15), demonstrating the powerful impact of early Spinraza treatment.

Spinraza demonstrated longer term efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function. Spinraza was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.

“A few years ago, SMA patients had no treatment options and faced significant care challenges,” said Kenneth Hobby, President of Cure SMA, a patient advocacy organization dedicated to the treatment and cure of SMA. “However, the future of SMA has changed and especially with early treatment patients now have a very strong chance to reach age appropriate developmental milestones. These new data demonstrate the dramatic impact where children are now walking independently at four years of age, when the usual lifespan would be under two if untreated. This study provides additional evidence on the maintenance of these improvements. It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types.”