Dyne Therapeutics (DYN) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Strategic and operational updates

• Transitioning to a commercial-stage company with two major drugs targeting serious unmet needs, supported by a strengthened management team and robust clinical and regulatory execution.

• FORCE platform enables broad and deep delivery of oligonucleotides, positioning the company as a leader in nucleic acid medicines.

• Recent competitor trial failure (Novartis) in DM1 highlights differentiation in payload and delivery approach, with the company using an ASO for nuclear targeting and higher dosing flexibility.

• Ongoing phase III program uses both vHOT and five-times sit-to-stand as endpoints, providing multiple regulatory pathways and risk mitigation.

• Market for DM1 estimated at 30,000 addressable U.S. patients, with potential for significant expansion as diagnosis rates improve.

Clinical development and regulatory milestones

• Anticipates Q1 readout from the registrational expansion cohort for DM1, with interim data to be presented at upcoming conferences.

• DMD program (z-rostudirsen) targets Exon 51 skipping, showing ~10x higher dystrophin levels than current standard and functional improvements across all major measures.

• Regulatory process for z-rostudirsen progressing smoothly, with FDA priority review and a PDUFA date set for January 21.

• Commercial launch preparations underway, including supply chain, payer engagement, and patient advocacy outreach.

• Ex-U.S. expansion planned post-phase III, with Europe and Japan as key targets.

Market opportunity and pipeline expansion

• DMD Exon 51 skippable mutation market in the U.S. estimated at 1,600 patients, with pricing around $1 million per patient.

• Plans to expand to additional exons (44, 45, 53, 55) using the same platform, potentially tripling the addressable market.

• New IND candidate DYNE-302 for FSHD leverages the same platform, with dosing and tissue distribution expected to differentiate from competitors.

• Data for DYNE-302 not expected until after next year, as dosing optimization is ongoing.

• FORCE platform supports development of multiple products, with seven identified candidates leveraging shared CMC and commercial infrastructure.