Novel Duchenne therapy shows promise for halting disease progression
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A next-generation exon skipping therapy led to significant dystrophin expression and functional improvement in patients with Duchenne muscular dystrophy amenable to exon 51 skipping, according to new data from a phase 1/2 clinical trial.
DYNE-251 is a phosphorodiamidate morpholino oligomer conjugated to a fragment antibody that binds to the transferrin receptor 1, manufacturer Dyne Therapeutics Inc. said in a company release. Developed to positively target muscle tissue and promote exon skipping in the nucleus, the treatment’s mechanism of action permits muscle cells to create dystrophin proteins that either reverse or halt disease progression.
The novel therapeutic is currently being examined in the DELIVER clinical trial, a global, randomized, placebo-controlled study that includes children and adolescents aged 4 to 16 years with the rare neuromuscular condition.
According to data cited in the release, those given 20 mg/kg of DYNE-251 every 4 weeks recorded a mean absolute dystrophin expression of 3.71% of normal, more than 10-fold higher than the 0.3% reported in a clinical trial of eteplirsen, the standard treatment for patients with Duchenne.
Additionally, study data showed meaningful improvements in multiple functional endpoints in groups given 10 mg/kg and 20 mg/kg every 4 weeks, including on North Star Ambulatory Assessment, Stride Velocity 95th Centile, 10-Meter Walk/Run Time as well as Time to Rise from Floor metrics. Researchers also found those in the 10 mg/kg cohort logged consistent improvement across all relevant metrics from 6 months to 12 months.
Further, Dyne reported that among 54 participants, the drug demonstrated a favorable safety profile with most treatment emergent adverse events being mild or moderate.
“We believe these data reinforce the opportunity to transform the treatment paradigm for individuals living with Duchenne,” Wildon Farwell, MD, MPH, chief medical officer of Dyne, said in the release. “With these exciting data, we are moving quickly to initiate registrational cohorts in DELIVER, and we continue to pursue expedited approval pathways and plan to provide an update on our path to registration by the end of this year.”
Editor's Note: This story was corrected on Sept. 6, 2024 to reflect that DYNE-251 is an exon-skipping therapy. Healio regrets the error.