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Dyne Therapeutics: Positive Initial Clinical Data from DMD DELIVER Trial

January 4, 2024

Dyne Therapeutics has reported initial clinical data from the DELIVER clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

What is Dyne-251?

Dyne-251 is an exon-skipping product that combines a phosphorodiamidate morpholino oligomer (PMO) to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to skeletal muscle.

Data Highlights from the Phase 1/2 DELIVER Trial of DYNE-251 in DMD:

  • In the Phase 1/2 DELIVER Trial, patients treated with DYNE-251 had a mean absolute dystrophin level of 0.88% of normal and a 0.28% change from baseline at six months. As reported for a separate clinical trial, eteplirsen reached a mean absolute dystrophin level of 0.30% of normal and a 0.06% change from baseline at six months.
  • Enrollment is complete through the 20 mg/kg cohort of the DELIVER trial (40 total patients enrolled), and approximately 275 doses have been administered to date, supporting dose escalation up to 40 mg/kg.
  • Dyne anticipates providing our next clinical data update from the DELIVER trial in the second half of 2024.

“We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases. The safety profiles for both DYNE-251 in DELIVER and DYNE-101 in our ACHIEVE clinical trial have supported dose escalation to a combined 10 cohorts and the administration of nearly 600 doses across both trials. This positions us to optimize dose and dose regimen in both trials with the goal of initiating registrational cohorts as we end 2024. We anticipate reporting data for multiple, higher dose cohorts from both trials in the second half of 2024, while continuing to pursue expedited regulatory pathways and working to help address the urgent need for therapeutics for people living with Duchenne.”

Joshua Brumm, President and CEO at Dyne Therapeutics

“These compelling initial data highlight the exciting opportunity we have to advance our investigational therapeutics for devasting diseases with no or limited treatment options. Treatment with DYNE-251 surpassed the level of dystrophin production reported for the standard of care for DMD exon 51 with a fraction of the dose. Underpinning these results is a favorable safety profile which is critical in the development of therapies for chronic diseases. We are grateful to the participants, clinicians and the community for their ongoing partnership as we collectively strive to transform the treatment of rare muscle diseases.”

Wildon Farwell, M.D., MPH, Chief Medical Pfficer at Dyne Therapeutics

The DELIVER clinical trial of DYNE-251 is currently underway in Canada at the London Health Sciences Centre (London, Ontario) and the Children’s Hospital of Eastern Ontario – CHEO (Ottawa, Ontario). Recruitment is complete through the 20mg/kg cohort, but Dyne is continuing to progress the study with more cohorts up to 40mg/kg. If you are interested in participating in this clinical trial, we encourage you to speak to your health care team.

Defeat Duchenne Canada will be hosting a webinar in collaboration with Dyne Therapeutics, focusing on this trial update. Keep an eye on your inbox for the official announcement, which will include the registration link and further information on the webinar.

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