Dyne Therapeutics (DYN) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Strategic vision and platform development

• Positioned as a pure-play neuromuscular company with two major programs (DMD and DM1) in registrational cohorts, aiming for commercialization in 2027.

• Built a management team skilled in capital raising, allocation, and near-term revenue generation.

• FORCE platform enables efficient delivery of genetic medicines to muscle and potentially CNS, overcoming previous technical hurdles.

• Platform supports a growing pipeline, including preclinical and IND-enabling studies, with integrated capabilities.

DM1 program progress and regulatory strategy

• ACOS study showed 40% improvement in hand function and up to 20% improvement in muscle strength at 12 months.

• Patient-reported outcomes indicated significant CNS-related benefits, supporting platform's CNS reach.

• Shifted registrational cohort primary endpoint to VHOT after FDA feedback, increasing cohort size to 60 for statistical significance.

• Received breakthrough designation for DM1; confirmatory phase 3 trial planned for Q1 next year.

• Top-line DM1 data expected mid-next year, with BLA submission targeted for early 2027.

DMD program highlights and commercial outlook

• DMD program targets Exon 51, addressing a significant unmet need with sustained functional improvement shown in early data.

• Achieved up to 8.7% dystrophin at six months, well above approval threshold; pursuing accelerated approval with breakthrough designation.

• Functional measures (10-meter walk, time to rise) improved over time, supporting clinical benefit.

• Commercial leader hired; rapid market uptake anticipated due to dissatisfaction with current therapies.

• Plans to expand to other exons, potentially covering up to 40% of DMD market.