Biogen to Advance SMA Treatment Into Registrational Studies

Dow Jones

Jun 25, 2025

By Colin Kellaher

Biogen plans to advance an experimental treatment for the neuromuscular disease spinal muscular atrophy, or SMA, into registrational studies following encouraging results from an early-stage trial.

Biogen on Wednesday said interim data from a Phase 1 study of salanersen 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 the drug.

The Cambridge, Mass., biotechnology company said that based on the encouraging data, it is engaging with regulators to advance salanersen to registrational-stage studies.

Biogen said salanersen uses the same mechanism of action as its Spinraza drug for SMA but is designed to achieve greater potency, giving it the potential to achieve high efficacy and enable once-yearly dosing.

Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, which discovered the drug. Biogen and Ionis are also partners on Spinraza.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

June 25, 2025 07:57 ET (11:57 GMT)

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