Satellos gets FDA clearance for forazapadin IND in FSHD

Satellos Bioscience Inc. said on September 24, 2026 that the U.S. Food and Drug Administration has cleared its Investigational New Drug application for forazapadin for the treatment of facioscapulohumeral muscular dystrophy (FSHD).¹ The company plans to initiate a Phase 2 clinical study in FSHD in the fourth quarter of 2026.¹

Alongside the clearance, the FSHD Canada Foundation has agreed to provide up to US$5 million in non-dilutive financing toward the clinical development of forazapadin in FSHD.¹ Under the terms disclosed, the Foundation will contribute up to $5 million in milestone payments spread over the next five quarters, in return for a capped share of future revenue tied to FSHD-related proceeds.¹

The planned trial is described as a randomized, double-blind, placebo-controlled proof-of-concept study. It is designed to assess the safety, tolerability, pharmacokinetics and potential efficacy of oral forazapadin at 60 mg and 120 mg doses in FSHD patients aged 18 and older, and Satellos expects to begin it in the fourth quarter of 2026.¹

This marks the drug's second disease indication. The FSHD IND clearance follows Satellos's ongoing evaluation of forazapadin in Duchenne muscular dystrophy (DMD), where early results from a Phase 2 trial in adults reportedly showed an acceptable safety profile along with a decrease in muscle fat fraction on MRI and a rise in total effort after six months on the 60 mg dose.¹ Satellos said these findings could be consistent with muscle regeneration.¹

FSHD affects an estimated 800,000 people worldwide and results from abnormal activation of the DUX4 gene, which damages muscle tissue and drives progressive weakness.¹ There are currently no approved disease-modifying therapies for the condition.¹

Forazapadin targets a protein called AAK1, which Satellos believes plays a role in muscle repair, and the drug is intended to inhibit AAK1 to restore a signaling pathway linked to muscle regeneration.¹