On Sunday, Dyne Therapeutics Shared Additional Data From The Ongoing Phase 1/2 DELIVER Trial Of Zeleciment Rostudirsen (Z-rostudirsen, DYNE-251) For Duchenne Muscular Dystrophy Amenable To Exon 51 Skipping

Dyne announced the results of new analyses of cardiac and pulmonary function amongst all DELIVER participants who were randomized to z-rostudirsen treatment at baseline (any dose1) and for whom cardiac magnetic resonance imaging and/or pulmonary function data were available.

• Improvement from baseline in lung function, as measured by Forced Vital Capacity Percent Predicted (FVC%p), was observed through 24 months, as compared to the expected decline estimated in published natural history data.
• Improvement from baseline was observed through 24 months in circumferential strain, an early signal of cardiac performance, as compared to the expected worsening estimated in published natural history data.
• Improvement from baseline in left ventricular ejection fraction, a measure of how well the heart is pumping, was observed at 24 months, in contrast with the expected decline estimated in published natural history data.
• In previously reported safety and tolerability data from 86 total participants enrolled in the DELIVER trial and followed for up to 36 months, z-rostudirsen demonstrated a favorable safety profile7, and most related treatment emergent adverse events (TEAEs) were mild or moderate. The most commonly reported related TEAEs were pyrexia (fever) and headache. No related serious TEAEs were observed in the REC.

These data will be presented in a poster titled "Zeleciment rostudirsen led to trends in long-term improvement in clinical outcomes including cardiopulmonary function: Additional data from DELIVER" (poster # 476 LBT).