Biogen today announced it will present new data focusing on SMA at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA., May 4–11.
Results from the NURTURE study demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA over three years achieved motor milestones more consistent with normal childhood development. After three years of follow-up, interim results from the ENDEAR/CHERISH/SHINE open-label extension study show that treatment with SPINRAZA, particularly when initiated earlier, leads to progressive motor milestone improvements and increased survival rates for individuals with infantile-onset SMA. Additional data suggest that phosphorylated neurofilament heavy chain (pNF-H) may emerge as a promising biomarker to predict disease activity in SMA.
Highlights of Biogen’s Platform and Poster Presentations for Spinal Muscular Atrophy:
Platform Presentations
- Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study – Platform S25.001 – Tuesday, May 7, 1:00 p.m. ET
- Interim Report on the Safety and Efficacy of Longer-Term Treatment With Nusinersen in Infantile-Onset Spinal Muscular Atrophy (SMA): Updated Results From the SHINE Study – Platform S25.004 – Tuesday, May 7, 1:33 p.m. ET
- Association of Phosphorylated Neurofilament Heavy Chain (pNF-H) Levels With Motor Function Achievement in Individuals With Spinal Muscular Atrophy (SMA) Treated With Nusinersen – Platform S27.009 – Tuesday, May 7, 2:28 p.m. ET
Poster Presentations
- Symptoms and Complications Over Three Years Among Later Childhood, Adolescent and Adult Spinal Muscular Atrophy Patients: A Natural History Study Within U.S. Hospitals – Poster P1.6-051 – Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET
- Ambulation Status, Role Participation and Caregiver Assistance Among Individuals With Spinal Muscular Atrophy Type III: Results from the Cure SMA Membership Survey – Poster P1.6-061 – Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET
- Interim Report on the Safety and Efficacy of Longer-Term Treatment With Nusinersen in Later-onset Spinal Muscular Atrophy (SMA): Results from the SHINE Study – Poster P1.6-063 – Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET
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 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
- New SPINRAZA (nusinersen) Data Unveiled at AAN Annual Meeting Show Continued Improvement in Motor Function for Broad Age Range and Survival Benefit for Infants