The U.S. Food and Drug Administration has cleared Satellos Bioscience Inc.'s investigational new drug application for forazapadin, an oral small‑molecule targeting AAK1, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The approval allows the company to commence a Phase 2 trial in the fourth quarter of 2026.
The randomized, double‑blind, placebo‑controlled study will enroll adults aged 18 and older with FSHD and will evaluate safety, tolerability, pharmacokinetics and potential efficacy of two oral doses, 60 mg and 120 mg. Participants will receive the drug daily, and the trial will assess muscle‑repair biomarkers alongside clinical outcomes.
Funding for the FSHD program includes up to $5 million in non‑dilutive financing from the FSHD Canada Foundation. The foundation will provide milestone payments over the next five quarters in exchange for a capped revenue‑sharing interest in future FSHD‑related proceeds.
FSHD affects an estimated 800,000 people worldwide and currently has no approved disease‑modifying therapies. The condition is driven by abnormal activation of the DUX4 gene, leading to progressive muscle weakness.
Forazapadin is also being evaluated for Duchenne muscular dystrophy (DMD). Preliminary data from an ongoing Phase 2 DMD study showed a favorable safety profile, reduced muscle fat fraction on MRI, and increased total effort after six months of treatment at the 60 mg dose. Satellos is running two additional Phase 2 DMD studies—BASECAMP in pediatric patients and TRAILHEAD in adults.
