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 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.
The data was 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).
“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 and making unprecedented gains in a previously hopeless and often fatal condition,” said Darryl De Vivo, M.D., Sidney Carter Professor of Neurology and Pediatrics, Columbia University Irving Medical Center in New York, New York. “SPINRAZA is setting patients on a path toward survival, greater mobility and independence, 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 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.”
Additional presentations at the two meetings 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.
Cure SMA Provides Seed Funding for Spinraza
From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach behind SPINRAZA. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.
For More Information
To learn more about Spinraza please visit www.curesma.org/spinraza or the links below:
- Biogen Presents Data at the AAN Annual Meeting Highlighting Spinraza and Emerging Biomarkers for SMA
- Biogen Presents New Data Regarding Adults and Infants with SMA
- Biogen Presents New Data at the Annual Congress of the World Muscle Society
- Biogen Releases Community Statement on Spinraza Access and New Data
- Biogen Announces New Interim Phase 2 Results from NURTURE