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Biogen to Advance Investigational Spinal Muscular Atrophy Asset to Registrational Studies Based on Positive Interim Phase 1 Results
Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular
About this update from Biogen Inc.
[{"type":"text","content":"Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular atrophy (SMA)Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersenBased on these encouraging Phase 1 data, Biogen is engaging with regulators to advance salanersen to registrational stage studies, building on extensive experience in SMA CAMBRIDGE, Mass., June 25, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for the treatment of spinal muscular atrophy (SMA). Leveraging the same mechanism of action as SPINRAZA (nusinersen) but designed to achieve greater potency, salanersen has the potential to achieve high efficacy and enable once yearly dosing. An interim analysis of the Phase 1 study in participants with SMA who were previously treated with gene therapy was conducted to inform the decision on whether to move salanersen forward into registrational studies. Both dose levels tested, 40 mg and 80 mg, given once a year, were generally well-tolerated and led to substantial slowing of neurodegeneration, as shown by reductions in neurofilament. Exploratory clinical outcome data shows clinically meaningful improvements in function and attainment of new World Health Organization (WHO) milestones over 1 year. These data will be presented today at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. “Of the data generated, to me, it is neurofilament and the WHO milestones that are most easily interpretable, given these children had previously received gene therapy. To see a child dosed with gene therapy at one year of age and still unable to sit without support at age five then gain the ability to sit independently just 3 months after initiating salanersen, that is unexpected,” said Valeria A. Sansone, M.D., Ph.D., Clinical and Scientific Director at the Clinical Center NeMO in Milan, Professor of Neurology, University of Milan, and a principal investigator for the salanersen Phase 1 trial. “Given these are early data from a relatively small cohort, I ...