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 BLA submission and product launches in 2026–2027.

• FORCE platform enables targeted delivery to muscle and CNS using proprietary Fab against TfR1, supporting multiple payloads and broad pipeline expansion.

• Human and non-human primate data show superior delivery and functional improvement, especially with DYNE-251 in DMD.

• Cash position of ~$1.1 billion as of 12/31/25, with runway into Q1 2028 and all assets fully owned.

• Platform enables capital-efficient expansion and de-risked pipeline, aiming to triple DMD market size.

Clinical pipeline and data highlights

• DELIVER trial for DYNE-251 in DMD showed a seven-fold increase in dystrophin and significant functional improvements at six months, with robust results across TTR velocity, 10MWR, NSAA, SV95C, PUL2.0, and FVC %p.

• Durable improvements maintained up to 36 months; safety profile favorable with most adverse events mild or moderate and over 1,000 doses administered.

• DYNE-101 in DM1 demonstrated 33% DMPK knockdown, 25% splicing correction, and functional improvements in vHOT, sit-to-stand, 10-meter walk, and patient-reported outcomes through 12 months.

• Quantitative muscle testing showed 10–20% strength increase; MDHI subscales indicated CNS impact at higher doses.

• No serious related adverse events for DYNE-101; most TEAEs were mild or moderate, with 93 patient years and over 1,000 doses.

Regulatory and commercialization plans

• Planned U.S. Accelerated Approval submission for DYNE-251 in DMD in Q2 2026, with potential commercial launch in Q1 2027.

• DYNE-101 in DM1 targeting Accelerated Approval submission in early Q3 2027, with Phase 3 studies planned for global approval.

• Breakthrough designation for both DMD and DM1, with ongoing FDA dialogue and alignment on accelerated approval pathways.

• Commercial infrastructure, management, and supply chain in place for 2027 launch; gross margins expected to be competitive with rare disease antibody companies.

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