Satellos Bioscience Inc. (NASDAQ:MSLE) has received clearance from the U.S. Food and Drug Administration for its Investigational New Drug application to evaluate forazapadin in facioscapulohumeral muscular dystrophy, or FSHD, according to a company statement.
The clearance allows Satellos to proceed with clinical testing of forazapadin in FSHD and represents the second clinical indication being investigated for the candidate. It does not constitute FDA approval of forazapadin for the treatment of FSHD.
The Toronto-based clinical-stage company plans to begin a Phase 2 study during the fourth quarter of 2026.
Phase 2 FSHD Trial Planned for Q4
The planned study will be a randomised, double-blind, placebo-controlled trial evaluating orally administered forazapadin in adults aged 18 and older with FSHD.
The study will evaluate 60 mg and 120 mg doses and assess safety, tolerability, pharmacokinetics and potential efficacy.
FSHD is a muscle disease associated with abnormal activation of the DUX4 gene and progressive muscle weakness. According to the information supplied, the condition affects an estimated 800,000 people worldwide and currently has no approved disease-modifying therapies.
FSHD Canada Foundation to Provide Up to $5 Million
Separately, Satellos announced a financing agreement with the FSHD Canada Foundation, a Calgary-based charitable organisation.
Under the agreement, the Foundation will provide up to $5 million through milestone-based payments over five quarters.
In exchange, the Foundation will receive a capped revenue-sharing interest in future proceeds related to FSHD. Satellos described the financing arrangement as non-dilutive.
The amount ultimately received will depend on the achievement of the applicable milestones.
Forazapadin Also Being Studied in Duchenne Muscular Dystrophy
The FDA clearance expands the clinical development programme for forazapadin, which is already being evaluated in two ongoing Phase 2 trials involving Duchenne muscular dystrophy, or DMD.
Preliminary data from an adult DMD study showed what the company described as a favourable safety profile, along with a reduction in muscle fat fraction measured by MRI and an increase in total effort after six months of treatment with the 60 mg dose.
These preliminary observations are from an ongoing clinical development programme and do not establish the safety or efficacy of forazapadin.
Forazapadin, previously referred to as SAT-3247, targets AAK1, a protein that Satellos believes has a role in muscle repair and regeneration.
The candidate remains investigational and has not been approved for the treatment of FSHD or DMD.