Mr. Frank Gleeson reports
SATELLOS ANNOUNCES COMPLETION OF ENROLLMENT IN BASECAMP PHASE 2 CLINICAL TRIAL OF FORAZAPADIN FOR DUCHENNE MUSCULAR DYSTROPHY
Satellos Bioscience Inc. has completed enrolment for Basecamp, the company's phase 2 clinical trial evaluating forazapadin for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory boys seven to 10 years of age. Forazapadin is an orally administered, small molecule drug candidate designed to restore muscle regeneration in people living with DMD, facioscapulohumeral muscular dystrophy (FSHD) and potentially other degenerative muscle diseases.
"We are thrilled to announce that we have met and surpassed our enrollment target in Basecamp in less than nine months from screening our first participant, reflecting the strong execution of our clinical program," said Frank Gleeson, co-founder and chief executive officer of Satellos. "We believe Basecamp could support discussions with the FDA regarding a potential accelerated development pathway for forazapadin. Given the significance and proximity of this opportunity, we believe it is important to be thoughtful and transparent in how we develop and communicate the results from the trial. We have therefore decided to take the time necessary to compile the most complete data set possible. Accordingly, we are adjusting our previous plan for releasing data and now intend to share top-line clinical data in the first quarter of 2027."
"The enthusiasm and commitment of the Duchenne community have enabled us to exceed our enrolment target and build a robust global study," said Wildon Farwell, MD, chief medical officer of Satellos. "With multiple dose cohorts and a broad range of clinical, functional and biomarker assessments, we believe Basecamp can generate a substantial body of data that we expect to help us better understand forazapadin, its potential as a novel treatment for Duchenne and guide us in determining a potential path to approval. We are deeply grateful to the patients and families who have made this research possible."
Basecamp enrolled ambulatory boys seven to 10 years of age across 18 clinical sites in the United States, Canada, Australia, Belgium, Spain, Poland and Serbia. Participants were randomized 1:1:1 to receive either forazapadin 60 milligrams, forazapadin 120 mg or placebo. The study includes a 12-week placebo-controlled period, after which participants enter a 36-week randomized active-treatment period. Primary endpoints include safety, tolerability and the effect of forazapadin on muscle force as assessed by dynamometry. Secondary end points are intended to assess forazapadin's impact on muscle quality, function and 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 continuing 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.
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.
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