Dyne Therapeutics (DYN) Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary

Industry context and recent developments

• Recent large acquisition in the neuromuscular space is seen as validating for therapeutic areas like DMD, DM1, and FSHD, and for the TfR1 receptor approach.

• The company positions itself as a pure-play neuromuscular player, participating in key disease areas highlighted by recent industry moves.

DMD program updates and outlook

• Top-line data from the DMD registrational expansion cohort is expected in December, with high confidence in dystrophin expression and functional improvement.

• Dystrophin levels achieved are significantly higher than previous therapies, supporting potential for accelerated approval.

• Safety is monitored in real time, with no material changes since the last public update; next update is due in December.

• Functional endpoints like SV95C and NSAA have shown early and clinically meaningful improvements, especially in the 20 mg/kg cohort.

• Commercial opportunity includes rapid conversion from existing exon 51 skippers and potential to expand the market to previously untreated patients.

DM1 program progress and challenges

• Enrollment for the DM1 registrational cohort was delayed from Q4 to Q2 next year due to site capacity issues, especially in Europe.

• Additional sites have been added in Europe and the US to address capacity and operational delays.

• The study is about 50% enrolled, with no set requirement for US patient numbers; inclusion/exclusion criteria are consistent globally.

• Baseline VHOT is expected to be similar to previous cohorts, maintaining discipline in patient selection.

• MDHI, a patient-reported outcome, has shown significant improvement, including CNS-related subscales, and may be further explored in phase three.