Dyne: Positive Initial Clinical Data from ACHIEVE Trial in DM1, DELIVER Trial in Duchenne

  In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose-Dependent Splicing Correction, Muscle Delivery and DMPK Knockdown -

- All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months -

- Improvement in Myotonia (vHOT) as well as Fatigue (MDHI) Observed in Lowest Dose ACHIEVE Cohort at 6 Months -

- In Phase 1/2 DELIVER Trial, DYNE-251 Showed 0.88% Mean Dystrophin Expression at 6 Months in 5 mg/kg Cohort Administered Monthly; Greater Than 2.5 Times Higher Dystrophin Than Reported for Weekly Administered Current Standard of Care1-

- Favorable Safety Profile for DYNE-101 and DYNE-251 Has Supported Dose Escalation; Enrollment Complete in 5.4 mg/kg Cohort of ACHIEVE Trial and 20 mg/kg Cohort of DELIVER Trial -

- Virtual Webcast Event Today, Wednesday, January 3 at 8:00 a.m. ET -

Dyne will host a live video webcast event, “Achieving the Promise of FORCE to Deliver for Patients,” to discuss these ACHIEVE and DELIVER data today, January 3, 2024, at 8:00 a.m. ET. Dyne’s leadership team will be joined by two leading neuromuscular disease experts: Valeria A. Sansone, M.D., Ph.D., and Perry Shieh, M.D., Ph.D. Dr. Sansone is Clinical and Scientific Director at the Clinical Center NeMO in Milan, Professor of Neurology, University of Milan, and a principal investigator for the ACHIEVE trial. Dr. Shieh is Professor of Neurology and Pediatrics at the David Geffen School of Medicine at UCLA, a Neurologist at the Ronald Reagan UCLA Medical Center in Los Angeles, and a principal investigator for the DELIVER trial.

The live webcast will be available in the Events & Presentations page of the Investors & Media section of Dyne’s website and a replay will be accessible for 90 days following the presentation. An accompanying slide presentation will also be available. To access the presentation, register for the live webcast and replay, please visit https://investors.dyne-tx.com/news-and-events/events-and-presentations.

https://www.globenewswire.com/news-release/2024/01/03/2803115/0/en/Dyne-Therapeutics-Announces-Positive-Initial-Clinical-Data-from-ACHIEVE-Trial-in-DM1-Patients-and-DELIVER-Trial-in-DMD-Patients-Demonstrating-Promise-of-the-FORCE-Platform-in-Devel.html