Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) has announced the dosing of the first participant with Duchenne muscular dystrophy (DMD) in the Phase 1b clinical trial of SAT-3247. This marks a significant step in evaluating the drug's potential as a treatment for DMD. The company has also completed the enrollment of healthy volunteers in the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 trial.
Phase 1b Trial Details
The Phase 1b trial is designed to assess the safety and pharmacokinetics (PK) of SAT-3247 in up to 10 adult participants with genetically confirmed DMD. The trial is an open-label, single-dose cohort study that will monitor patients over 28 days. Researchers will also explore potential pharmacodynamic markers to understand how the drug affects the disease.
Frank Gleeson, Satellos Co-founder and CEO, stated, "The dosing of our first DMD participant is a major milestone in evaluating SAT-3247’s potential benefit for DMD patients. We are very pleased to have safely completed the full enrollment and dosing of all five cohorts in the single ascending dose ("SAD") study and the first two multiple ascending dose ("MAD") cohorts. This reinforces our view that SAT-3247 has potential to be a safe and tolerable, once-daily, oral treatment option for DMD patients. We look forward to continuing to advance SAT-3247 as a potential disease modifying therapy for degenerative muscle conditions."
Phase 1 Trial in Healthy Volunteers
Prior to the Phase 1b trial, Satellos enrolled 72 healthy adult volunteers in a blinded, randomized, placebo-controlled study. This part of the trial aimed to evaluate the safety and pharmacokinetic properties of SAT-3247. The volunteers were divided into five SAD cohorts, four MAD cohorts, and one food effect dose cohort.
Safety and Tolerability
According to Satellos, healthy volunteers who received SAT-3247 in the SAD and the first MAD cohorts experienced no drug-related adverse events. The drug was reported to be well-tolerated, with no abnormal findings in clinical labs, vital signs, ECGs, or physical exams. Furthermore, the PK profile of SAT-3247 in healthy volunteers translated effectively from preclinical models to human subjects.
SAT-3247: A Novel Approach to DMD Treatment
SAT-3247 is an orally administered small molecule drug designed to restore skeletal muscle regeneration in degenerative or injury conditions by correcting muscle stem cell polarity. Satellos is developing SAT-3247 as a potential disease-modifying treatment for DMD, independent of dystrophin and regardless of exon mutation status. Preclinical evidence suggests that SAT-3247 could repair and strengthen degenerated or damaged muscles.
Future Plans
Satellos is on track to present Phase 1 data at a major medical conference in Q1 2025. The company also plans to submit a Phase 2 investigational new drug (IND) filing by the end of Q1 2025. Satellos is also exploring the potential of SAT-3247 in other degenerative muscle diseases or injury conditions through its MyoReGenX discovery platform.
