Dyne Therapeutics, Inc. (NASDAQ: DYN) has released positive clinical data from its ongoing phase 1/2 Achieve trial of Dyne-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing phase 1/2 Deliver trial of Dyne-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The company announced compelling impact on key disease biomarkers and improvement in multiple functional endpoints, along with favorable safety profiles.
In the Achieve trial, Dyne-101 demonstrated a dose-dependent 27% mean splicing correction across all patients in the 5.4 mg/kg cohort at 3 months. The treatment also showed improvement in myotonia, muscle strength, and timed function tests, along with improvement in DM1-ActivC and MDHI patient-reported outcomes.
In the Deliver trial, Dyne-251 exhibited 3.2% mean unadjusted (7.6% mean muscle adjusted) dystrophin expression at 6 months in the 10 mg/kg cohort, which is ten times higher than the level reported for the weekly administered standard of care. Additionally, Dyne-251 demonstrated trends in functional improvement in NSAA, time to rise from floor, 10-meter walk/run time, and stride velocity 95th centile at 6 months in the 10 mg/kg cohort.
The Achieve trial reported safety and tolerability data from 56 patients enrolled through the 6.8 mg/kg q8w cohort, demonstrating a favorable safety profile with the majority of treatment emergent adverse events being mild or moderate.
Similarly, the Deliver trial reported safety and tolerability data from 48 patients enrolled through the 40 mg/kg q8w cohort, also showing a favorable safety profile with the majority of treatment emergent adverse events being mild or moderate.
Dyne Therapeutics plans to continue engaging with global regulators on the Achieve and Deliver trials and anticipates providing an update on the path to registration for both Dyne-101 and Dyne-251 by the end of 2024. The company is pursuing expedited approval pathways for both programs and has confirmed the availability of accelerated approval pathways for both DM1 and DMD.
Dyne Therapeutics is hosting a live video webcast event to discuss the Achieve and Deliver data on May 20, 2024, at 8:00 a.m. ET.
The Achieve trial is a phase 1/2 global clinical trial evaluating Dyne-101, enrolling adult patients with DM1, and is designed to be registrational, with primary endpoints of safety and tolerability and secondary endpoints of pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function. As a result of these announcements, the company's shares have moved 23.3% on the market, and are now trading at a price of $34.12. Check out the company's full 8-K submission here.
