Dyne Therapeutics Inc (NASDAQ: DYN) released 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.
Phase 1/2 ACHIEVE Trial of DYNE-101 in DM1: The initial efficacy assessment of the DYNE-101 ACHIEVE trial is based on data from 32 adult DM1 patients enrolled.
- DYNE-101 demonstrated dose-dependent splicing correction, muscle delivery, and DMPK knockdown.
- The 3.4 mg/kg Q4W group had a 40% mean DMPK knockdown from baseline compared to 25% in patients in the 1.8 mg/kg Q4W group at three months.
- Evaluable patients treated with 3.4 mg/kg Q4W of DYNE-101 had a 19% mean ...