Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of data–for spinal muscular atrophy (SMA) at the Cure SMA 2018 Annual SMA Conference in Dallas, TX.
“We are proud to support Cure SMA in advancing scientific understanding of the disease to improve the lives of individuals with SMA and their families. Data presented include analyses of disease burden, SPINRAZA treatment, and phosphorylated neurofilament heavy chain (pNF-H) in plasma and SMA clinical characteristics, which could help shape the future of SMA research and treatment,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We remain dedicated to sharing our ongoingexperience with SPINRAZA, the first and only approved treatment for SMA, among patients of all ages, while also building a portfolio of treatments for this devastating disease.”
Presentations at the Annual SMA Conference –including data from Biogen’s ongoing open-label trials, hospital care and potential biomarkers –reiterate SPINRAZA’s effectiveness across broad SMA populations and expand scientific understanding of the disease and treatment options. One evaluation will feature pNF-H in plasma as a potential indicatorof SMA disease activitythat, through further research, could helpprovide a betterunderstanding of the disease. Neurofilaments have been associated with disease activity acrossa range of neurological diseases.
Additional presentations include interim analyses from the SHINE and NURTURE studies, which assessSPINRAZA’s safety and efficacy among those with infantile-onset SMA.Aseparate presentation will examine the experience and disease burden of untreated infants and children with SMA in hospitals, providing more context about the disease’s natural history.
Biogen’s SPINRAZA and SMA presentations include:
Platform presentations
- Phosphorylated Neurofilament Heavy Chain (pNF-H) As a Potential Biomarker of SMA Disease Activity: pNF-H Levels at Baseline and During Treatment in the NusinersenClinical Trial Program –Friday, June 15, 11:40 a.m. CT
- Longer-Term Assessment of the Safety and Efficacy of Nusinersen for the Treatment of Infantile-onset Spinal Muscular Atrophy (SMA): An Interim Analysis of the SHINE Study–Saturday, June 16, 10:00a.m. CT
- Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of SMA: Interim Efficacy and Safety Results From the Phase 2 NURTURE Study–Saturday, June 16, 10:20a.m.
CTPosters
- Survival and Ventilation Among Those with Type I SMA: Results From the 2017 Cure SMA Membership Survey(joint presentation between Cure SMA and Biogen)–Poster 39–Thursday, June 14, 4:30 p.m. CT
- Characterization of Infant and Early Childhood Spinal Muscular Atrophy Patients and Their Transitions of Care Within U.S. Hospitals–Poster 26–Friday, June 15, 12:30 p.m. CT
For more information about SPINRAZA and prescribing information in the United States, please visit www.SPINRAZA.com. Prescribing information in the European Union is available at http://www.ema.europa.eu/ema/.