Satellos Bioscience (MSCL) H.C. Wainwright 28th Annual Global Investment Conference summary

Novel therapeutic approach for DMD

• Forazapadin targets muscle stem cell regeneration by inhibiting AAK1, offering a non-genetic, oral therapy distinct from dystrophin restoration strategies.

• The approach may be used alone or in combination with existing therapies, potentially benefiting all DMD patients regardless of genetic status.

• Resetting the regenerative cycle in muscle stem cells could slow disease progression and improve muscle function.

Clinical development and trial updates

• Phase I studies showed no safety concerns and demonstrated early biologic activity, including improved biomarkers and respiratory function.

• TRAILHEAD study participants exhibited a decline in muscle fat fraction and improved upper extremity function, contrary to natural disease progression.

• BASECAMP, a placebo-controlled trial in boys aged 7–10, is on track to complete enrollment, with interim data expected this year and full results early next year.

• Early improvements in strength and function may be observed within the first month of treatment, supporting potential for accelerated approval discussions.

Regulatory and future plans

• Data packages for regulatory submission will focus on strength (dynamometry), MRI fat fraction, and functional endpoints, aligning with FDA guidance for DMD.

• Ongoing and upcoming data from TRAILHEAD and BASECAMP will inform conversations with regulatory agencies about accelerated approval pathways.