Dyne Therapeutics (DYN) TD Cowen 46th Annual Health Care Conference summary

Platform overview and strategic vision

• FORCE platform enables delivery of genetic medicines to muscle and CNS tissues, validated in DMD and DM1 clinical trials.

• Eight wholly owned pipeline programs target rare muscle diseases, aiming for a decade-long growth trajectory.

• Platform leverages similar antibody, linker, and chemistry across products, providing commercial and manufacturing efficiencies.

• Clinical validation in 2025 for DMD and DM1 supports transition to commercial stage in 2026, with further launches planned through 2028.

• Broad tissue penetration and functional benefit demonstrated in preclinical and clinical data.

DMD (Duchenne Muscular Dystrophy) program progress

• Zirasatersen for exon 51 skipping shows seven-fold increase in dystrophin and significant functional improvements over placebo.

• Six functional endpoints improved at six and 24 months, with two reaching statistical significance.

• Safety profile remains strong with mild to moderate adverse events and 1,400 doses administered.

• Accelerated approval BLA filing and confirmatory phase 3 trial both on track for the current quarter.

• Commercial launch planned for early 2026, targeting a concentrated, well-identified patient population.

Commercial strategy and market expansion

• Sales force will be capital-efficient, focusing on 100 U.S. centers covering 80% of patients.

• Experienced commercial team led by rare disease experts with prior successful launches.

• DMD franchise expanding to four additional exons, tripling addressable market to 4,000–5,000 patients.

• High probability of success for new exons due to shared platform and established reimbursement.

• All assets are wholly owned, with best-in-class profiles and significant unmet need.