Dyne Therapeutics (DYN) Corporate presentation summary

Mission and platform overview

• Aims to deliver functional improvement for people with genetically driven neuromuscular diseases using the FORCE platform, which targets transferrin receptor 1 (TfR1) for precise delivery to muscle and CNS.

• Pipeline includes clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), with additional candidates for FSHD and Pompe disease.

• FORCE platform enables modular payload selection (ASO, PMO, siRNA, enzyme) to match disease biology, offering targeted, redosable, and durable therapies.

• Platform design minimizes systemic exposure and manufacturing costs by using a single Fab across programs.

Clinical pipeline and milestones

• Zeleciment rostudirsen (z-rostudirsen) for DMD and zeleciment basivarsen (z-basivarsen) for DM1 are in late-stage clinical development, with positive topline results and ongoing registrational cohorts.

• First potential commercial launch expected in Q1 2027, with U.S. Accelerated Approval submissions planned for both lead programs.

• Pipeline expansion includes additional DMD exons, FSHD (DYNE-302), and Pompe disease (DYNE-401), leveraging the same platform technology.

• Cash position of ~$1.1 billion as of 12/31/25, supporting operations into Q1 2028.

DMD program (z-rostudirsen)

• DELIVER trial met primary endpoint with a statistically significant, robust increase in dystrophin at 6 months (p<0.0001), and functional improvements across multiple measures up to 24 months.

• Demonstrated improvements in TTR velocity, 10MWR, NSAA, SV95C, PUL2.0, and FVC % predicted, with sustained benefit and favorable safety profile.

• Safety data show most adverse events were mild or moderate, with no persistent related anemia or thrombocytopenia at the registrational dose.

• Positioned for commercial launch in a well-characterized rare disease market with established reimbursement and treatment centers.