Drugs

Dyne Reports Strong Dystrophin Gains in Duchenne Study

Dyne Therapeutics is reporting positive topline results from its Phase 1/2 DELIVER trial of z-rostudirsen (DYNE-251), saying the investigational exon-skipping therapy produced large increases in dystrophin and measurable functional benefits in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. It says it remains on track to file for accelerated approval in the second quarter of 2026, setting up a potential launch as early as early 2027 if the FDA grants a priority review.

In the Registrational Expansion Cohort (REC) of 32 participants, those receiving 20 mg/kg of z-rostudirsen every four weeks achieved a mean muscle-content–adjusted dystrophin level of 5.46% of normal at six months, a result the company described as both statistically significant and consistent with the seven-fold increase observed previously at the same dose in the dose-escalation phase.

The company says unadjusted for muscle content, dystrophin levels averaged 2.87% of normal, roughly ten times higher than levels reported for eteplirsen, the current weekly standard-of-care exon 51 therapy.

Dyne also says the z-rostudirsen data showed improvement relative to placebo across all six pre-specified functional endpoints over six months, including:

Time to Rise velocity
10-meter walk/run velocity
North Star Ambulatory Assessment
Stride Velocity 95th Centile
Performance of Upper Limb

Importantly, Dyne says that lung function was preserved in treated patients at six months, contrasting with declines in the pooled placebo group. Preservation of respiratory capacity is considered a key clinical need in DMD, where progressive pulmonary decline is a major cause of mortality.

No treatment-related serious adverse events were reported in the REC, the company says. Two participants in the extension cohort experienced related serious events (malaise and fever) since the last update, but both recovered fully and continued treatment.

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