Biogen presented new Phase 1b data for its antisense oligonucleotide salanersen at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 8‑11, 2026. The presentation focused on children who had suboptimal clinical status after receiving the onasemnogene abeparvovec‑xioi gene therapy.
The 24‑patient study, involving children aged 6 months to 12 years, showed that salanersen was generally well‑tolerated and produced meaningful reductions in neurofilament light chain (NfL) levels—up to 75% at six months. Half of the participants (12 of 24) achieved new World Health Organization motor milestones, and the 80 mg dose, selected for Phase 3, was highlighted as the most promising.
These results are significant for patients who have not fully responded to gene therapy, offering a potential once‑yearly treatment that could improve motor function and reduce neurodegeneration. The NfL reduction and milestone gains suggest that salanersen may address unmet needs in a population with limited therapeutic options.
Biogen outlined a comprehensive Phase 3 program comprising the STELLAR‑1, STELLAR‑2, and SOLAR trials. Enrollment for SOLAR is slated to begin in Q2 2026, with STELLAR‑2 following in Q3 2026. All studies will evaluate once‑yearly 80 mg salanersen across a broad SMA population, including treatment‑naïve infants and those previously treated with gene therapy.
The data and trial design position salanersen as a potential next‑generation SMA therapy, expanding Biogen’s rare‑disease portfolio and offering a differentiated product in a competitive market that includes Spinraza, Zolgensma, and Evrysdi. The once‑yearly dosing schedule could improve adherence and reduce long‑term costs for patients and payers.
"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 Stephanie Fradette, Head of the Neuromuscular Development Unit at Biogen. "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," added Dr. Thomas Crawford, co‑director of the MDA Clinic at Johns Hopkins Medicine.
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