Satellos Bioscience (MSCL) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company background and scientific rationale

• Founded on a discovery about dystrophin's role in muscle stem cells, challenging traditional views of Duchenne pathology.

• Identified AAK1 as an alternative pathway to restore muscle stem cell function and regeneration.

• Developed forazapadin (formerly SAT-3247) to target this pathway, aiming to improve muscle repair in Duchenne and other muscle diseases.

Clinical development and trial updates

• Two phase II trials (BASECAMP and TRAILHEAD) are ongoing, with data expected before year-end and early next year.

• BASECAMP is a placebo-controlled study in 51 boys with Duchenne, evaluating two dose levels and multiple functional and biological endpoints.

• TRAILHEAD is an open-label study in adults, showing early improvements in hand grip strength, fat fraction, and muscle function.

• Plans to expand TRAILHEAD to 30 participants and initiate a phase II trial in FSHD before year-end.

Key clinical findings and endpoints

• Phase I data showed a strong safety profile and near doubling of hand grip strength in adults with higher drug exposure.

• Improvements observed in biomarkers, fat fraction (mean 3.7% decrease), and total effort in muscle function.

• BASECAMP will assess muscle biopsy (Regenerative Index), dynamometry, MRI fat fraction, and time function tests, aiming for endpoints relevant to regulatory approval.

• Early biological changes may precede functional improvements, with long-term extension designed to capture sustained effects.