Biogen Enhances Commitment to SMA Research with Data Presented at Annual SMA Conference

• Research includes clinical trial data across broad SMA patient populations, expanding scientific understanding of SMA and SPINRAZA® (nusinersen) 
   
• Study presented examines neurofilament (pNF-H) as a potential biomarker for SMA, which could provide benefits for future research and deeper understanding of the disease

CAMBRIDGE, Mass., June 13, 2018 (GLOBE NEWSWIRE) -- Biogen (Nasdaq:BIIB) today 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 (June 14-17, 2018).

"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 ongoing experience 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 indicator of SMA disease activity that, through further research, could help provide a better understanding of the disease. Neurofilaments have been associated with disease activity across a range of neurological diseases.

Additional presentations include interim analyses from the SHINE and NURTURE studies, which assess SPINRAZA's safety and efficacy among those with infantile-onset SMA. A separate 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 Nusinersen Clinical 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, 10:00 a.m. CT