Dyne Therapeutics (DYN) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic vision and platform advancement

• Advancing a clinically validated platform for rare neuromuscular diseases, with two late-stage assets in DMD and DM1 and near-term value drivers including first BLA submission in 2026 and commercial launches in 2027 and 2028.

• Proprietary Fab-linker and FORCE platforms enable targeted delivery of diverse payloads to muscle, CNS, cardiac, and diaphragm tissues, supporting a broad pipeline including DMD, DM1, FSHD, and Pompe.

• Proprietary Fab against TfR1 allows robust tissue distribution without interfering with iron homeostasis.

• Over $1.1 billion in cash and wholly owned assets as of 12/31/25, supporting long-term value creation and expected runway into Q1 2028.

• Management and commercial teams in place, leveraging rare disease launch experience and established manufacturing partnerships.

Clinical pipeline and data highlights

• DYNE-251 (z-rostudirsen) in DMD demonstrated a seven-fold increase in dystrophin and 10x standard of care at six months, with statistically significant and robust results.

• Functional improvements observed in time to rise, 10-meter walk, NSAA, stride velocity, upper limb function, and lung capacity.

• Favorable safety profile for DYNE-251 up to 36 months, with most adverse events mild or moderate and no persistent related anemia or thrombocytopenia.

• DYNE-101 (z-basivarsen) in DM1 showed splicing correction, functional, and patient-reported improvements through 12 months, with no serious related adverse events.

• Functional gains in five-time sit-to-stand, 10-meter walk, and quantitative muscle testing (10% at 6 months, 20% at 12 months); early CNS impact observed in MDHI subscales.

Regulatory and commercialization plans

• Planned U.S. Accelerated Approval submission for DYNE-251 in DMD in Q2 2026, with potential launch in Q1 2027; DYNE-101 in DM1 targeting Accelerated Approval submission in early Q3 2027.

• Breakthrough designation for both programs enables ongoing FDA dialogue and accelerated approval pathway.

• Confirmatory phase 3 trials to use clinically meaningful functional endpoints, with protocols aligned globally.

• Manufacturing and supply chain scaled and validated for commercial readiness; competitive gross margins expected.

• Well-characterized patient populations and concentrated treatment centers support efficient market entry.