Dyne Therapeutics (DYN) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic vision and platform overview

• Aims to become a leader in neuromuscular disease with late-stage programs in DM1 and DMD, targeting potential launches as early as 2027.

• Proprietary platform uses antibody fragments to deliver oligonucleotide payloads to muscle and CNS, showing strong tissue penetration in both animal and human studies.

• Platform flexibility demonstrated by using different payloads (PMO for DMD, ASO for DM1) and plans to expand to FSHD and Pompe disease.

• Fully owns assets and maintains a strong financial position with over $600 million in cash, funding operations into the second half of 2026.

DM1 clinical development and data insights

• Completed multi-ascending dose study and selected 6.8 mg/kg as the registrational dose, guided by splicing correction and functional benefit.

• Achieved 25% improvement in CASI splicing correction at three months, correlating with significant functional and patient-reported outcome improvements at six months.

• Safety profile remains favorable with no serious related adverse events across 855 doses and 72 patient-years of follow-up.

• Registrational expansion cohort underway, aiming for full enrollment by mid-2025, with accelerated approval package including up to 100 patients.

• Phase III study planned to start in 2024, focusing on CNS, sleep, cognitive, and cardiac outcomes.

DMD program progress

• 20 mg/kg cohort showed unprecedented dystrophin levels and functional improvement at six months, exceeding clinical meaningfulness benchmarks.

• No serious related adverse events observed in 837 doses and over 65 patient-years of follow-up.

• Registrational expansion cohort of 32 patients nearly fully enrolled, with potential filing in early 2026 and launch in 2027.

• Platform success in DMD supports expansion to additional exon candidates and broader neuromuscular pipeline.