Dyne Therapeutics (DYN) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic overview and leadership

• Preparing to launch key neuromuscular medicines for DMD in early next year and DM1 the following year, with a fully integrated biotech structure and experienced leadership team.

• Focused on capital allocation, product launch, and commercialization, with a PDUFA date in January for the first product.

DM1 program and clinical progress

• Zeleciment basivarsen targets DM1, a severe disease with no current therapies, showing DMPK knockdown, splicing correction, and functional improvements in the ACHIEVE study.

• The FORCE platform enables broad tissue delivery, including CNS, with a Fab fragment that allows higher dosing and avoids dose-limiting toxicities.

• vHOT is used as an intermediate endpoint, while five-times sit-to-stand is the primary endpoint for phase III, aligning with FDA discussions.

• Upcoming 12-month data on 25 patients from ACHIEVE will be presented at major medical meetings, with registrational cohort data expected in Q1 next year.

• HARMONIA phase III trial is enrolling globally, with strong interest due to CNS targeting and no enrollment issues.

DMD program and product differentiation

• Z-rostudirsen for DMD (exon 51 skipping) is under priority review, showing improved functional measures and broad tissue distribution, including heart and diaphragm.

• Dystrophin expression increased to 5.5% at six months, with continued improvement and stabilization of cardiac and lung function at 18 and 24 months.

• Monthly dosing offers convenience and higher dystrophin production compared to current therapies, with strong physician and patient advocacy support.

• Phase III confirmatory study uses rise from floor velocity as the primary endpoint, with additional functional and patient-reported outcomes.

• FDA granted priority review and does not expect an AdCom for the upcoming PDUFA date.