Satellos Shows Muscle Repair from SAT-3247 in Canine DMD Model

15 July 2024
Satellos Bioscience Inc., a Toronto-based biotech company, has announced promising preliminary findings from a single-case study on SAT-3247 treatment in a canine model of Duchenne muscular dystrophy (DMD). SAT-3247 is being developed as a novel regenerative medicine to address this severe form of dystrophy, which closely mirrors the disease progression seen in humans with DMD. The recent data were shared at the Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference.

Frank Gleeson, Co-founder and CEO of Satellos Bioscience, expressed optimism over the initial data showing that SAT-3247 improved muscle regeneration and muscle force in the canine model. He noted that the results from this large animal study validate the improvements in muscle repair that have been observed in previous mouse studies. He emphasized that the pilot study supports the potential of SAT-3247 to restore muscle repair and regeneration, which are impaired in individuals with Duchenne.

The study involved treating dystrophic animals with SAT-3247 for four months, with a daily oral dose. Following the treatment, an increase in the Regenerative Index (RI) was observed. RI measures the number of newly regenerated muscle fibers in comparison to damaged and dying ones, indicating that muscle repair and regeneration are taking place.

Dr. Phil Lambert, Chief Scientific Officer of Satellos Bioscience, commented that the increases in the Regenerative Index suggest muscle repair and regeneration are occurring. He highlighted that these preliminary results enhance the understanding of SAT-3247's unique mechanism of action. The company is making diligent efforts to advance this small molecule drug candidate to a first-in-human clinical trial slated for the next quarter.

The pilot study data were drawn from one of two dystrophic animals treated with SAT-3247. The single case study results showed a substantial improvement in the Regenerative Index of skeletal muscle, with an approximate 450% increase after four months of treatment. Additionally, there was up to a 100% increase in muscle force after just two months of treatment.

SAT-3247 is an oral small molecule drug designed to be taken once daily. It targets the underlying cause of muscle loss in degenerative diseases, initially focusing on Duchenne muscular dystrophy. This drug introduces a novel mechanism of action aimed at restoring impaired muscle regeneration due to the lack of functional dystrophin.

Duchenne muscular dystrophy is an inherited condition caused by mutations in the dystrophin gene, resulting in the malfunction of the dystrophin protein. This impairment hinders muscle repair and regeneration. SAT-3247 aims to restore the muscle repair process by regulating a dystrophin-independent pathway, consequently enhancing muscle function. It is intended to be a standalone therapeutic, irrespective of a patient's genetic mutation or ambulatory status, and has the potential to complement other approaches that aim to restore dystrophin production.

Satellos Bioscience Inc. is dedicated to developing innovative treatments for degenerative muscle diseases. Utilizing breakthrough research in muscle stem cell polarity, the company has created a proprietary discovery platform named MyoReGenX™. This platform helps identify muscle diseases where deficits in muscle regeneration can be therapeutically targeted. Satellos is building a pipeline of new therapeutics to correct muscle stem cell polarity and promote the body's natural muscle repair processes. The company's leading program involves developing an oral small molecule drug candidate as a potential disease-modifying treatment for Duchenne muscular dystrophy.

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