Satellos Bioscience (MSCL) investor relations material

Key insights and company focus

• Focused on regenerating muscle in Duchenne muscular dystrophy (DMD) and other muscle degeneration diseases using a paradigm-shifting technology platform.

• Identified a unique aspect of DMD: the inability to repair and regenerate muscle, which is not addressed by current treatments.

• DMD affects 1 in 5,000 males globally, with about 12,000 cases in the U.S.; current treatments have significant limitations and side effects.

• The company’s approach is dystrophin-independent, targeting satellite stem cell dysfunction via a small molecule drug.

Clinical development and trial updates

• Two phase II trials underway: BASECAMP (ages 7–9) and TRAILHEAD (ages 16+), aiming for NDA filing in 2027.

• BASECAMP is a 12-week, placebo-controlled, double-blind study in ambulatory children, with 51 participants across up to 21 sites in 8 countries.

• TRAILHEAD is a 12-month study in adults, enrolling up to 30 individuals, with FDA clearance and multiple safety and efficacy endpoints.

• Clinical assessments include grip strength, MRI, muscle biopsies, NSAA, and a range of biomarkers; studies are inclusive of patients on other therapies.

• Full enrollment for BASECAMP expected by end of Q3, with top-line data by year-end; ongoing progress and updates planned for both trials.

Preclinical and clinical results

• Preclinical canine model showed transformative muscle regeneration and up to 4x strength gains; clinical studies in adults showed up to 2x grip strength improvement.

• Drug demonstrated a clean safety profile, rapid clearance, and no concerning side effects in phase Ia trials.

• Significant reduction in established DMD damage markers observed in treated adults; grip strength gains sustained over one year.

• Data suggest the drug reverses the typical decline seen in DMD, with improvements correlating to drug exposure and baseline muscle mass.