Dyne Therapeutics Releases Promising ACHIEVE and DELIVER Trial Data

Dyne Therapeutics, a clinical-stage muscle disease company, has announced promising results from its Phase 1/2 trials of DYNE-101 and DYNE-251, aimed at treating myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), respectively. Both trials showcased significant advancements in key disease biomarkers, functional improvements, and favorable safety profiles.

The ACHIEVE trial for DYNE-101 involved 40 adult patients with DM1. DYNE-101 displayed robust muscle delivery and dose-dependent splicing correction, leading to enhancements in muscle strength, functional performance, and patient-reported outcomes. In the 5.4 mg/kg cohort, a 27% mean splicing correction was observed within three months. Improvements in myotonia, muscle strength, and timed function tests were also noted. Furthermore, patient-reported outcomes indicated progress across all 17 subscales of the Myotonic Dystrophy Health Index (MDHI) and the DM1-ACTIVc, which assesses daily living activities.

From a safety perspective, DYNE-101 was well-tolerated across 56 patients, with most adverse events being mild or moderate in severity. No serious treatment-related adverse events were detected, and the trial saw complete enrollment through the 6.8 mg/kg cohort.

The DELIVER trial for DYNE-251 included eight male patients with DMD amenable to exon 51 skipping. The study revealed that DYNE-251, administered at 10 mg/kg monthly, achieved a mean unadjusted dystrophin level of 3.22% of normal at six months, significantly higher than the levels reported for weekly-administered standard treatments for DMD. Trends in functional improvement were noted in various assessments, such as the North Star Ambulatory Assessment (NSAA) and 10-Meter Walk/Run Time.

DYNE-251 also demonstrated a favorable safety profile among 48 patients, with most adverse events being mild or moderate. No serious treatment-related adverse events were reported, and enrollment was completed through the 40 mg/kg cohort.

John Cox, President and CEO of Dyne, expressed enthusiasm about the data, highlighting the transformative potential of DYNE-101 and DYNE-251. Both trials are designed to be registrational, with Dyne actively engaging with global regulatory authorities to pursue expedited approval pathways.

Dr. Wildon Farwell, Dyne's Chief Medical Officer, noted that the data reflects the potential of Dyne's FORCE platform to address rare muscle diseases. Dyne plans to provide an update on the path to registration for both DYNE-101 and DYNE-251 by the end of 2024.

In conclusion, Dyne Therapeutics is making notable progress in the development of therapies for DM1 and DMD. The promising clinical data from the ACHIEVE and DELIVER trials underscore the potential of DYNE-101 and DYNE-251 to significantly improve patient outcomes, with both treatments demonstrating substantial efficacy and safety. Dyne remains committed to advancing these therapies to address the urgent unmet medical needs of these patient communities.