Solid Biosciences (SLDB) Citigroup’s Biopharma Back to School Summit 2026 summary

Pipeline and upcoming milestones

• Focused on precision genetic medicines for Duchenne muscular dystrophy (DMD) and Friedreich's ataxia (FA), with multiple regulatory and data updates expected in the next six months.

• FDA meeting on Accelerated Approval for DMD scheduled for Q4, with clinical data and regulatory pathway updates to follow.

• Over 53 DMD patients dosed, with at least 12 having one-year data for FDA review; additional data will continue to accrue.

• Friedreich's ataxia program has dosed three patients, with two more planned by year-end; initial mFARS data expected in Q1 next year.

• Phase III IMPACT trial for DMD is underway, with European and additional US sites expected to open by early next year.

Regulatory strategy and trial design

• Statistical analysis plan (SAP) for DMD was updated after FDA feedback and submitted in July; endpoints include time to rise, Stride Velocity 95th Centile, four-stair climb, and 10-m walk/run.

• FDA will review a data package from at least 12 patients with one-year follow-up; judgment call on clinical benefit will be based on totality of data, including biomarkers and functional endpoints.

• If FDA requests more patient data (e.g., 24-25 patients), these are already dosed and data will be available within months.

• Confirmatory IMPACT trial enrollment will accelerate once European sites are active; trial design may be modified based on upcoming FDA discussions.

• For FA, a placebo-controlled phase III trial is planned, likely with around 50 patients, to support global approvals.

Lessons learned and platform advancements

• Regulatory flexibility and ongoing FDA communication in rare diseases are seen as positive, with lessons drawn from other companies' experiences.

• Stride Velocity 95th Centile is favored as an endpoint to minimize bias in open-label studies.

• POLARIS-101 capsid platform has over 50 licensing agreements and is positioned as a next-generation AAV for muscle and cardiac diseases.

• AI tools are being developed to analyze preclinical, clinical, and CMC data, assist in regulatory filings, and support partners using the capsid platform.

• The AI platform allows partners to leverage anonymized data across programs, aiming to streamline regulatory processes.