Satellos Begins Dosing Duchenne Patient in Phase 1b SAT-3247 Trial

Satellos Bioscience Inc., a biotech company listed on TSX and OTCQB, is pioneering new small molecule therapies aimed at enhancing the treatment of muscle diseases. Recently, they announced a significant development in their ongoing research for Duchenne muscular dystrophy (DMD) by dosing the first participant in a Phase 1b trial. This trial focuses on evaluating the safety and pharmacokinetics (PK) of their investigational drug, SAT-3247, in DMD patients.

Frank Gleeson, Co-founder and CEO of Satellos, expressed enthusiasm about this milestone, noting it as a crucial step in assessing SAT-3247 for its potential benefits for DMD patients. He highlighted the successful completion of participant enrollment in both the single ascending dose (SAD) and the initial cohorts of the multiple ascending dose (MAD) stages of the trial, emphasizing the drug’s promise as a safe, once-daily oral treatment for those affected by DMD. Gleeson is optimistic about advancing SAT-3247 as a potential therapy that can modify the disease progression in muscle degeneration conditions.

The trial aims to enroll up to ten adult participants with confirmed genetic diagnoses of DMD. These participants will undergo a 28-day, open-label study, receiving a single dose to evaluate safety and PK properties. The study also seeks to explore potential pharmacodynamic markers that could provide further insights into the drug's impact.

The results from the initial SAD and MAD cohorts involving healthy volunteers have been promising. Participants did not experience any drug-related adverse side effects, and SAT-3247 was well tolerated, showing no abnormalities in clinical labs, vital signs, ECG readings, or physical exams. Additionally, the PK profile of SAT-3247 observed in pre-clinical models effectively translated into human subjects, further supporting the drug's potential.

The Phase 1 clinical trial is structured into two main components. The first involves 72 healthy adult volunteers participating in a blinded, randomized, placebo-controlled study to assess the safety and PK properties of SAT-3247. This segment encompasses five SAD cohorts, four MAD cohorts, and one food effect dose cohort. The second part, currently ongoing, involves the Phase 1b trial with DMD patients, focusing on similar safety and PK assessments along with exploring pharmacodynamic markers.

Satellos Bioscience, dedicated to developing innovative medicines for degenerative muscle diseases, has created SAT-3247 as a pioneering small molecule drug. It is designed to restore skeletal muscle regeneration, with an initial focus on DMD. The company has illustrated through preclinical studies that correcting muscle stem cell polarity with SAT-3247 has the potential to repair and strengthen damaged muscles. As the lead drug candidate, SAT-3247 is advancing through clinical development as a potential disease-modifying treatment for DMD.

Beyond DMD, Satellos is leveraging its MyoReGenX™ platform to identify other degenerative muscle diseases that could benefit from therapeutic intervention. The company aims to extend its research and development efforts to other areas where muscle regeneration deficits are present, exploring new avenues for clinical development and potential treatments for a broader range of muscle-related conditions.