TORONTO, Canada — Satellos Bioscience Inc. (NASDAQ: MSLE, TSX: MSCL), a clinical-stage drug development company developing potentially life-improving medicines to treat degenerative muscle diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for forazapadin for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The company plans to initiate a Phase 2 clinical study in FSHD in the fourth quarter of 2026. Satellos also announced that 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.
“We believe the biology targeted by forazapadin has the potential to address significant unmet needs in degenerative muscle diseases, and our expansion into FSHD reflects the broad potential of our muscle regeneration approach,” said Frank Gleeson, co-founder and chief executive officer of Satellos. “Progress in medicine happens when researchers, clinicians, industry partners and advocacy organizations come together around a common goal, and we are grateful to the FSHD Canada Foundation for its partnership and confidence in our work. This support is expected to enable us to advance forazapadin into clinical development in FSHD and extend our muscle regeneration strategy to a second patient community.”
“People living with FSHD, like me, are eager to find treatments that can stop our muscles from getting weaker. But we would also like to get some of those muscles back,” said Neil Camarta, co-founder of the FSHD Canada Foundation. “That is what makes this announcement so meaningful. Seeing forazapadin advance into clinical trials to evaluate the potential for muscle regeneration in FSHD is an important step for our community. While we know there is still a long road ahead, it is encouraging to see innovative approaches like this moving into the clinic. FSHD Canada appreciates the support we received from our friends at Solve FSHD, the FSHD Society and FSHD Global in helping make this possible. Time is muscle!”
The collaboration between Satellos and the FSHD Canada Foundation provides non-dilutive capital to advance forazapadin’s clinical development in FSHD. Under the agreement, the Foundation has agreed to contribute up to US$5 million in milestone payments over the next five quarters in exchange for a capped revenue-sharing interest in future FSHD-related proceeds. The funds are expected to support the IND-cleared Phase 2 randomized, double-blind, placebo-controlled proof-of-concept clinical study designed to evaluate the safety, tolerability, pharmacokinetics and potential efficacy of orally administered forazapadin at 60 mg and 120 mg doses in adults aged 18 and older living with FSHD, which we expect to initiate in the fourth quarter of 2026.
Wildon Farwell, M.D, chief medical officer of Satellos added, “We are excited to receive FDA clearance of our IND application for forazapadin in a second disease indication, one for which there are currently no approved therapies. FSHD is a genetic disease in which muscle regeneration appears to be compromised. We look forward to working with the FSHD community to evaluate the potential of forazapadin to impact muscle regeneration and benefit people living with FSHD. In particular, we are delighted that the clearance included 60 mg and 120 mg dose levels of forazapadin, enabling evaluation of two doses of our small molecule drug candidate.”
FSHD is one of the most common forms of muscular dystrophy, affecting an estimated 800,000 individuals worldwide. It is caused by abnormal activation of the DUX4 gene, which damages muscle and contributes to progressive muscle weakness. Symptoms often begin in the muscles of the face, shoulders and upper arms before progressing to other parts of the body. The severity and rate of progression vary from person to person, and there are currently no approved disease-modifying therapies.
The clearance of this IND represents the second clinical indication for which forazapadin is being developed. Forazapadin is currently being evaluated for Duchenne muscular dystrophy (DMD), where preliminary data from an ongoing Phase 2 clinical trial in adults living with DMD showed a favorable safety profile, reduced muscle fat fraction as measured by MRI, and increased total effort observed after six months of treatment at 60 mg. The company believes these findings may be consistent with muscle regeneration.
ABOUT FORAZAPADIN
Forazapadin is a proprietary, oral, small molecule drug candidate being developed by Satellos as a novel approach to regenerating skeletal muscle lost in degenerative muscle diseases or injury conditions. Forazapadin targets AAK1, a key protein identified by Satellos as believed to be capable of helping restore the body’s natural muscle repair and regeneration biology, a fundamental process that is disrupted in DMD, FSHD and other degenerative conditions. By inhibiting AAK1, forazapadin treatment aims to re-establish a biochemical signal believed to be involved in supporting muscle regeneration. Satellos is advancing forazapadin as a potential treatment for DMD that is independent of dystrophin and applicable regardless of exon mutation status as either a stand-alone or adjunctive therapy, with ongoing Phase 2 clinical studies including BASECAMP, a global, randomized, placebo-controlled study in pediatric participants, and TRAILHEAD, an open-label study in adult participants. A Phase 2 clinical study to evaluate the safety, efficacy and tolerability of forazapadin in adults with FSHD is expected to begin in the fourth quarter of 2026.
The company previously referred to the program as SAT-3247 and expects to transition to broader use of the program’s International Nonproprietary Name, forazapadin, in future scientific, regulatory and corporate communications.
ABOUT SATELLOS BIOSCIENCE INC.
Satellos is a clinical-stage drug development company focused on restoring natural muscle repair and regeneration in degenerative muscle diseases. Through its research, Satellos has developed forazapadin, an orally administered small molecule AAK1 inhibitor designed to address deficits in muscle repair and regeneration. Forazapadin is being evaluated as a potential disease-modifying treatment for Duchenne muscular dystrophy (DMD) in two Phase 2 clinical trials, BASECAMP in pediatric participants with DMD and TRAILHEAD in adults living with DMD. The FDA also cleared an Investigational New Drug (IND) application for the clinical evaluation of forazapadin for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The company has identified additional muscle diseases and injury conditions where restoring muscle repair and regeneration may have therapeutic benefit and plans to pursue these opportunities in future clinical development. For more information, visit www.satellos.com and connect with Satellos on X, LinkedIn, Facebook and Instagram.
ABOUT THE FSHD CANADA FOUNDATION
The FSHD Canada Foundation is a Calgary-based charitable organization dedicated to finding a cure for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy affecting adults and children. Founded by Neil Camarta and Craig Kelley, the Foundation funds and partners on research, natural-history studies, biomarker development, and clinical programs aimed at advancing treatments for the FSHD community in Canada and worldwide. For more information, visit fshd.ca.
Investors Contact
Caitlin Lowie
Vice President, Investor Relations & Communications
Media Contact
Emily Williams
Senior Director, Communications