Satellos Bioscience (MSCL) Corporate presentation summary

Mission and approach

• Mission: restore the body’s natural ability to regenerate muscle and improve lives affected by devastating muscle diseases.

• Forazapadin (SAT-3247) is an oral, once-daily AAK1 inhibitor designed to restore muscle stem-cell polarity and asymmetric division, supporting muscle repair and regeneration.

• Development targets Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD); forazapadin is investigational and not approved by any regulator.

DMD evidence and clinical programs

• In two DMD canines, once-daily forazapadin for four months was associated with flexion tetanic torque gains of up to 2x at two months and 4x at four months.

• Phase 1a/1b results showed favorable safety and tolerability; five adults with DMD completed 28 days of 60 mg weekday dosing, with early functional benefit and predictable, dose-proportional PK.

• In Phase 1b, grip-strength gains correlated with drug exposure and baseline creatinine, with Spearman correlations of 0.9 for the dominant hand and 0.7 for the non-dominant hand.

• Six-month TRAILHEAD findings in four adults: MRI muscle fat fraction fell in all participants (mean change -3.7%), total effort rose ~34%, and near-doubled handgrip strength was maintained.

• Over six months, no serious TEAEs were reported, treatment compliance was 100%, and mean drug exposure was 186 days; mean serum CK declined 38% from CL-101 baseline through month 6 in TRAILHEAD.

Phase 2 development

• TRAILHEAD is an open-label, long-term Phase 2 study in adults with DMD; it includes returning Phase 1b participants and forazapadin-naïve participants, with follow-up planned through 12 months.

• BASECAMP is a Phase 2 pediatric DMD study for ages 7–9, with a 12-week placebo-controlled period followed by a 36-week randomized active-treatment period; enrollment is complete at 51 participants across 18 activated sites in seven countries.

• The presentation lists a potential TRAILHEAD program update and BASECAMP topline data expected in Q1 2027.