Dyne Therapeutics Announces Positive Initial Clinical Data From ACHIEVE And DELIVER Trials

(RTTNews) - Muscle disease company Dyne Therapeutics, Inc. (DYN) announced Wednesday positive initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

The initial efficacy assessment of the DYNE-101 ACHIEVE trial is based on data from 32 adult DM1 patients enrolled in the randomized, placebo-controlled multiple ascending dose (MAD) portion of the trial, including 6-month data from the 1.8 mg/kg cohort and 3-month data from the 3.4 mg/kg Q4W cohort.

In each of these cohorts, participants were randomized to receive either DYNE-101 or placebo once every four weeks or participants in the recovery arm received two doses of DYNE-101 followed by placebo for the remainder of the MAD portion of the trial.

In the ACHIEVE trial, DYNE-101 demonstrated a dose-dependent splicing correction and increase in muscle delivery and DMPK knockdown while also showing functional improvement in myotonia.

Dyne anticipates providing its next clinical data update from the ACHIEVE trial in the second half of 2024.