Dyne Therapeutics (DYN) investor relations material

Recent progress and strategic focus

• 2025 marked a year of data de-risking, while 2026 is focused on execution and commercial launch preparation for DMD and DM1 therapies.

• The FORCE platform underpins a deep pipeline, including DMD, DM1, FSHD, and additional exon targets.

• Breakthrough Therapy designation for both lead products enables consistent, high-level FDA engagement.

• Pre-BLA meeting for DMD aligned on dystrophin as the endpoint, with BLA submission on track for this quarter.

• Priority Review is assumed in timelines due to Breakthrough Therapy status.

Clinical development and regulatory strategy

• DELIVER confirmatory trial for DMD initiated, enrolling 90 patients, with rise from floor velocity as the primary endpoint over 72 weeks.

• Dystrophin remains accepted as a surrogate endpoint for Accelerated Approval, supported by statistically significant data.

• Functional improvements demonstrated in DELIVER, with robust 24-month durability data.

• Other exons (53, 45, 44) are in development, leveraging the same platform and potentially tripling the addressable market.

• Regulatory strategy for additional exons will be determined post-approval of exon 51.

Commercialization and market positioning

• Established pricing and reimbursement landscape for DMD, with strong patient advocacy and motivated patient base.

• Monthly dosing offers a frequency advantage over competitors' weekly regimens.

• Commercial infrastructure and manufacturing will be leveraged for future launches, including FSHD.