Satellos' Phase 1 Trial for SAT-3247 Gets Regulatory Nod

Satellos Bioscience Inc., a biotech company based in Toronto, Canada, announced a significant milestone on August 19, 2024. The company has received approval for a clinical research proposal from the Human Research Ethics Committee (HREC) in Australia. This approval allows Satellos to proceed with a first-in-human Phase 1 clinical trial of SAT-3247 under the Therapeutic Goods Administration’s (TGA’s) Clinical Trial Notification (CTN) scheme. Satellos is focused on developing small molecule therapies aimed at improving the treatment of muscle diseases and disorders.

Frank Gleeson, the CEO and Co-founder of Satellos, highlighted the importance of this milestone, stating that it marks a pivotal point in the company’s journey to develop an oral medication that could potentially modify the course of Duchenne muscular dystrophy (DMD). He expressed excitement about advancing SAT-3247 into human trials, emphasizing the drug's potential to restore the muscle regeneration and repair process, which is compromised in individuals with Duchenne.

The Phase 1 clinical trial will be divided into two parts. The first part will involve 72 healthy volunteers in a blinded, randomized, placebo-controlled design. The aim is to evaluate the safety and pharmacokinetic properties of SAT-3247. Volunteers will be divided into five single-ascending dose (SAD) cohorts, four multiple-ascending dose (MAD) cohorts, and one cohort to assess the impact of food on the drug's absorption (food effect, FE).

The second component of the study will enroll 10 adult volunteers who have genetically confirmed DMD. These participants will be part of a 28-day open-label dose cohort. The goal is to compare safety and pharmacokinetic data with the results from healthy volunteers and to explore pharmacodynamic markers that could give insights into the drug's effectiveness.

SAT-3247 is a proprietary, oral small molecule drug developed by Satellos specifically to regenerate skeletal muscle lost in Duchenne muscular dystrophy and other muscle-degenerative conditions. This drug aims to provide a new treatment avenue independent of the dystrophin protein and the specific exon mutation status of the patient. By regulating a pathway that does not depend on dystrophin, SAT-3247 seeks to enhance muscle function and repair.

Duchenne muscular dystrophy is a genetic disorder caused by mutations in the dystrophin gene, leading to insufficient functioning of the dystrophin protein. This results in impaired muscle repair and regeneration. Satellos has designed SAT-3247 to address this dysfunction by regulating a dystrophin-independent pathway, with the objective of improving muscle function. The treatment is intended to be effective regardless of the patient’s genetic mutation or their ability to walk. This approach could potentially complement treatments aimed at restoring dystrophin production.

Satellos Bioscience Inc. is dedicated to developing innovative medicines for degenerative muscle diseases. The company utilizes a proprietary discovery platform, MyoReGenX™, which focuses on muscle stem cell polarity to identify diseases that can be treated by promoting muscle regeneration. This platform has enabled Satellos to build a pipeline of therapeutics designed to correct deficits in muscle stem cell polarity and enhance the body's natural muscle repair processes. SAT-3247 is the lead candidate in their program, representing a potential disease-modifying treatment for Duchenne muscular dystrophy. Satellos continues to operate from its headquarters in Toronto, Ontario.