Dyne Therapeutics is developing z-rostudirsen (formerly DYNE-251), an investigational therapy for Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 51 skipping23.
Recent data from the Phase 1/2 DELIVER trial show compelling, sustained functional improvements and favorable safety/tolerability34.
Participants receiving z-rostudirsen every four weeks demonstrated positive impact on key disease biomarkers, notably dystrophin protein levels, as well as multiple functional endpoints relevant to patients34.
The trial selected a registrational dose of 20 mg/kg every four weeks, with a fully enrolled expansion cohort aimed at supporting a regulatory submission, including the prospect of accelerated approval in the United States2.
Dyne has engaged global regulators and is leveraging dystrophin increases as a surrogate for clinical benefit, reinforcing the positive outlook for regulatory review34.
Safety data indicate most adverse events were mild or moderate, with no related serious events reported in over 35 patient-years of follow-up4.
These results underpin Dyne's strategy to seek expedited pathways for bringing z-rostudirsen to patients with urgent unmet medical need4.
Sources:
2. https://www.dyne-tx.com/clinical-trials/
3. https://www.neurologylive.com/view/duchenne-agent-dyne-251-demonstrates-functional-improvement-phase-1-2-deliver-trial
4. https://www.actionduchenne.org/dyne-therapeutics-announces-new-clinical-data-from-achieve-trial-of-dyne-101-in-dm1-and-deliver-trial-of-dyne-251-in-dmd/