Solid Biosciences (SLDB) H.C. Wainwright 28th Annual Global Investment Conference summary

Company overview and strategic focus

• Precision genetic medicine platform centers on AAV gene therapy for Duchenne muscular dystrophy (DMD) and Friedreich's ataxia (FA), with additional cardiac programs deprioritized to focus resources on these two main areas.

• Capsid delivery platform enables next-generation gene therapy delivery, collaborating with academic labs to advance technology adoption.

• Upcoming quarters are pivotal, with key FDA meetings planned for both lead programs and potential registrational trial discussions.

DMD program (003) insights

• Utilizes a refined microdystrophin construct retaining R16 and R17 domains for improved protein function and muscle integrity.

• Employs a novel capsid, SLB101 (POLARIS-101), showing 3x-19x greater muscle and heart biodistribution versus AAV9, with liver detargeting observed in preclinical and clinical data.

• ALT and AST declines post-dosing serve as biomarkers for muscle health, supporting long-term clinical benefit expectations.

• Steroids-only immunosuppression protocol differentiates from competitors, reducing monitoring burden and enabling outpatient dosing.

Regulatory and clinical development

• Phase III trial for DMD ongoing, with FDA endorsement on endpoints and trial design; primary endpoint is time to rise, supported by stride velocity as a secondary, minimizing bias.

• FDA recognizes high unmet need and novelty of the microdystrophin gene therapy, supporting continued development and potential accelerated approval pathway.

• Q4 FDA meeting planned to review data and discuss regulatory path, with public data release expected late this year or early January.