Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases
As an early investor in Dyne therapeutics, we are pleased to share that Dyne released positive data today from their Phase 1/2 DELIVER clinical trial in Duchenne muscular dystrophy
The exon-skipping therapy, Dyne-251 is for exon 51 skip-amenable patients and aims to re-frame their mutations to produce a slightly smaller, functional dystrophin protein. Their FORCE platform enhances the targeting and delivery of exon skipping therapeutics to skeletal, cardiac, and smooth muscle to improve efficacy and reduce dosing frequency.
Treatment with DYNE-251 at 5 mg/kg once a month surpassed the level of exon skipping and dystrophin production reported for Eteplirsen, the standard of care for DMD exon 51.
This is important clinical proof of concept data supporting Dyne’s FORCE delivery platform. Moreover, DYNE-251 demonstrated a positive safety profile and is already being tested at a higher dose of 20 mg/kg, with the goal of achieving even higher dystrophin levels. That data is expected to be available later this year.
CureDuchenne is proud to have made a significant investment in the company early on, to encourage and support their Duchenne program. This is a great example of how CureDuchenne’s support of projects can act as a catalyst to accelerate the development of the most promising therapeutic approaches into clinical trials.
Dyne also reported positive clinical data in their myotonic dystrophy type 1 (DM1) trial today. Both of Dyne’s clinical trials in DMD exon 51 and in DM1 are designed to be registration trials, which means if the data obtained later this year continues to be positive, Dyne could submit to the FDA for marketing approval.
To access the full press release, which includes more information on DYNE-251, the DELIVER clinical trial and Dyne Therapeutics, click this link.