Satellos Bioscience Inc., a public biotechnology firm dedicated to advancing treatments for muscle diseases, has announced a critical milestone in the development of its new therapeutic drug,
SAT-3247. This novel oral drug is aimed at regenerating skeletal muscle in conditions such as
Duchenne muscular dystrophy (DMD) and other muscle degenerative or injury-related disorders. On September 18, 2024, the company reported that the first participant has been dosed in a Phase 1 clinical trial for SAT-3247, signaling the company's shift into a clinical-stage entity.
Frank Gleeson, Co-founder and CEO of Satellos, expressed enthusiasm about the progress, emphasizing the significance of this advancement in their mission to develop pioneering therapies for muscle regeneration. The Phase 1 clinical trial will proceed in two stages. The initial part involves 72 healthy volunteers in a double-blind, randomized, placebo-controlled study designed to evaluate the safety and pharmacokinetic characteristics of SAT-3247. The participants will be divided into various cohorts to examine single-ascending doses (SAD), multiple-ascending doses (MAD), and the impact of food intake on the drug’s behavior in the body.
The second part of the trial, scheduled to commence in the first quarter of 2025, will include 10 adult patients diagnosed with DMD. This segment will be conducted as a 28-day open-label study focused on assessing the drug’s safety and pharmacokinetic profile in actual patients, along with exploring potential pharmacodynamic markers.
SAT-3247 is a proprietary drug specifically designed by Satellos to tackle the muscle degeneration seen in DMD and similar conditions. Unlike other treatments, it aims to restore muscle repair and regeneration via a
dystrophin-independent pathway, making it potentially effective regardless of the specific genetic mutation of the patient. This approach allows SAT-3247 to function as a standalone treatment and possibly complement other therapies that aim to restore dystrophin production.
Duchenne muscular dystrophy is a genetic disorder caused by defects in the dystrophin gene, leading to the improper function of the dystrophin protein. This malfunction hinders muscle repair and regeneration. SAT-3247 aims to counteract this by regulating muscle repair through alternative pathways, offering a new avenue for treatment that doesn't depend on the patient's specific genetic background or mobility status.
Satellos Bioscience Inc. was founded on groundbreaking research into muscle stem cell polarity, which is crucial for muscle regeneration. The company has developed a proprietary discovery platform named MyoReGenX to identify muscle diseases that can be targeted for therapeutic intervention. This platform is the cornerstone for creating a pipeline of innovative treatments designed to correct muscle stem cell polarity, thereby enhancing the body's natural muscle repair and regeneration mechanisms.
Headquartered in Toronto, Ontario, Satellos is committed to translating its scientific discoveries into life-changing therapies for patients suffering from
muscle degenerative diseases. The company’s primary focus is currently on developing SAT-3247 as a disease-modifying treatment for Duchenne muscular dystrophy, potentially offering new hope to those affected by this debilitating condition.
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