Dyne Therapeutics Achieves 5.46% Dystrophin Expression, Holds $1.1B Cash for Q2 BLA Plan
Dyne Therapeutics reported a Phase 1/2 trial of z-rostudirsen achieved 5.46% dystrophin expression (p<0.0001) at six months, with TTR and 10MWR velocity improvements and preserved lung function versus placebo. With $1.1 billion cash and runway into Q1 2028, the company plans a Q2 2026 BLA submission for a Q1 2027 launch.
1. Financial Position and Runway
Dyne closed 2025 with $1.1 billion in cash, providing funding through Q1 2028 to support ongoing trials and pipeline advancement without immediate financing needs.
2. Phase 1/2 z-rostudirsen Trial Results
In the 32-patient registrational expansion cohort of the DELIVER trial, z-rostudirsen delivered a 7-fold increase in dystrophin to 5.46% of normal at six months (p<0.0001), improved Time to Rise and 10-Meter Walk/Run velocities (nominal p<0.05), preserved FVC% predicted, and maintained a favorable safety profile.
3. z-rostudirsen Regulatory Pathway
Dyne intends to submit a BLA for U.S. Accelerated Approval of z-rostudirsen in Q2 2026, initiate a global confirmatory Phase 3 trial in the same quarter, and, pending Priority Review and approval, target a launch in Q1 2027.
4. z-basivarsen and Pipeline Expansion
Enrollment in the 60-patient ACHIEVE registrational expansion cohort for z-basivarsen is expected to complete in Q2 2026, with Phase 3 trial design scheduled for presentation and a potential U.S. launch in Q1 2028; additionally, four exon-skipping candidates are advancing into IND-enabling studies.