Dyne Therapeutics (NasdaqGS:DYN) Earnings Call Presentation

Z-Rostudirsen Clinical Trial Results - The DELIVER trial of z-rostudirsen in exon 51 DMD met its primary endpoint with a statistically significant increase in dystrophin at 6 months (p<0.0001)[9] - The Registrational Expansion Cohort (REC) replicated a ~7-fold increase in muscle content-adjusted dystrophin from baseline at 6 months with z-rostudirsen 20 mg/kg Q4W (p<0.0001)[32] - Z-Rostudirsen achieved ~10-fold higher level of unadjusted dystrophin relative to the level reported in Phase 3 trial of SoC[37] - The trial showed sustained functional improvement from baseline across multiple clinical endpoints out to 24 months[9] - At 6 months, TTR velocity improved exceeding MCID relative to placebo with nominal p<0.05[42] and 10MWR velocity improved relative to baseline and placebo with nominal p<0.05[45] Safety and Tolerability - Z-rostudirsen has a favorable safety and tolerability profile up to 36 months[59] - 1,062 doses of z-rostudirsen at 20 mg/kg dose level have been administered to date[25] - No participants have persistent related anemia or thrombocytopenia at 20 mg/kg[68] Regulatory and Commercial Strategy - The company plans to submit a BLA for U S Accelerated Approval in Q2 2026[7] - Potential U S launch is anticipated in Q1 2027, assuming FDA grants Priority Review[7] FORCE Platform Validation - The DELIVER trial data validates the potential of the FORCE platform for targeted drug delivery[67]