Dyne Therapeutics have reported their initial clinical data from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
DELIVER is a Phase 1/2 global clinical trial evaluating DYNE-251, consisting of a 24-week multiple ascending dose (MAD) randomised placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is enrolling ambulant and non-ambulant males with Duchenne muscular dystrophy who are ages 4 to 16 and have mutations amenable to exon 51 skipping.
In this phase 1/2 DELIVER trial, the initial efficacy assessment of the DYNE-251 is based on 6-month data from 6 male patients with DMD amenable to exon 51 skipping enrolled in the 5 mg/kg (approximate PMO dose) cohort of the randomised, placebo-controlled MAD portion of the trial.
What is DYNE-251?
DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle.
What is the FORCETM platform?
The proprietary FORCETM platform drives Dyne Therapeutics efforts to develop targeted, modern oligonucleotide therapeutics with the potential to be life-transforming for patients with serious muscle diseases.
DYNE-251 in DMD Data Highlights
- In 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 6 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 6 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’s President and CEO, Joshua Brumm said:
“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 optimise 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”
Wildon Farwell (M.D., MPH), Dyne’s Chief Medical Officer, added – “These compelling initial data highlight the exciting opportunity we have to advance our investigational therapeutics for devastating 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 favourable 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.”
Dyne Therapeutics anticipates providing our next clinical data update from the DELIVER trial in the second half of 2024.
Read more about Dyne Therapeutics DELIVER clinical trial
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
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