Biogen today 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 nusinersen in comparison to the natural history of this disease. These new data also showed that patients treated with nusinersen had continuous improvement in sitting and walking independently, with the overwhelming majority of patients achieving motor milestones in a normal timeframe.1
These data are being presented at the Cure SMA Annual SMA Conference in Anaheim, USA (28 June 28–1 July) and the 5th Congress of the European Academy of Neurology (EAN; 29 June–2 July, Oslo, Norway).
“These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones,” said Darryl De Vivo, Sidney Carter professor of Neurology and Paediatrics, Columbia University Irving Medical Center in New York, USA. “Nusinersen is setting patients on a path toward survival, greater mobility and independence from the start of treatment, which is helping improve outcomes for patients of all ages.”
Results from NURTURE, an ongoing, Phase 2, 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 nusinersen before six weeks old, demonstrated results not before seen in comparison to the natural history of SMA. As of March 20191:
- 100 percent were alive without a need for permanent ventilation.1
- 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.1
- 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.1
- 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.1
- 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 impact of early treatment.1
- Nusinersen demonstrated efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function.1
- Nusinersen was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.1
“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 organisation dedicated to the treatment and cure of SMA. “However, the future of SMA has changed and especially with early treatment patients now have a chance to reach age appropriate developmental milestones. These new data demonstrate the impact where children are now walking independently at four years of age, when the usual lifespan, for those who are most severe, would be under two if untreated.1 This study provides additional evidence on the maintenance of these improvements. It is 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.”
Additional presentations at the two meetings will highlight results from the ongoing open-label SHINE extension study of children with infantile and later-onset SMA as well the CS2/CS12 analysis of older patients. Biogen also continues to explore the scientific value of phosphorylated neurofilament heavy chain (pNF-H) and will present new data on the ongoing evaluation of its potential as a biomarker in SMA.
1. Trade Media Briefing: Latest data update and research efforts in Spinal Muscular Atrophy (SMA), July 2019.