Biogen Presents Additional Salanersen Data Showing New Motor Milestones Achieved in Children with SMA Previously Treated with Gene Therapy

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene therapy Salanersen is a novel antisense oligonucleotide and has the potential to deliver high efficacy in SMA with once-yearly dosing Biogen also debuted the design of the Phase 3 salanersen clinical trial program which is being initiated at sites around the world CAMBRIDGE, Mass., March 11, 2026 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) presented additional results from the Phase 1b study of salanersen, an investigational novel antisense oligonucleotide (ASO) given once a year for spinal muscular atrophy (SMA), at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The study evaluated salanersen in children who had suboptimal clinical status despite prior administration of gene therapy (onasemnogene abeparvovec-xioi). Salanersen was generally well-tolerated. Participants experienced a slowing of neurodegeneration and functional improvement, including achievement of new World Health Organization (WHO) motor milestones, following initiation of salanersen. These new results include a minimum of one year of follow-up for all participants, building on the interim study data presented at Cure SMA 2025. The company also presented the study designs of the Phase 3 salanersen clinical trials. “Spinal muscular atrophy has benefitted from extraordinary therapeutic progress, but across the treatment landscape there remains room for improvement. There is growing scientific and clinical enthusiasm about the advances that salanersen offers,” said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. “These additional Phase 1 data add confidence in the emerging salanersen clinical profile. We have more reason to look forward to results of the Phase 3 program." Salanersen Phase 1b Study Results In this analysis, new data are available for study participants (n=24, aged 0.5-12 years), all of whom have received at least 2 doses of salanersen (40 mg or 80 mg). The 80 mg dose will be further evaluated in the Phase 3 studies.

The release as fetched from its publisher. biogen.com ↗