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Dyne Therapeutics Announces Initiation of Phase 1/2 ACHIEVE Clinical Trial of DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
- First Patient Expected to be Dosed in September - - Data from Global, Multiple Ascending Dose ACHIEVE Trial Anticipated in the Second Half of 2023 - - Dyne
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- First Patient Expected to be Dosed in September - - Data from Global, Multiple Ascending Dose ACHIEVE Trial Anticipated in the Second Half of 2023 - - Dyne to Host “Spotlight on the Clinic” Virtual Event on September 12 - WALTHAM, Mass., Sept. 06, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced the initiation of its ACHIEVE Phase 1/2 clinical trial evaluating DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). “Advancing DYNE-101 into the clinic is an exciting moment for Dyne and for the DM1 community as there are no approved therapeutic options available for people living with this devastating disease. We are grateful to the teams at New Zealand Clinical Research and University of Auckland for their partnership in being our first ACHIEVE clinical trial site and to the other sites we are working to activate across the globe,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “DM1 is widely recognized as a spliceopathy by the global community. We have generated comprehensive preclinical data supporting DYNE-101 across multiple validated models, including demonstrating robust toxic DMPK knockdown leading to the correction of splicing, giving us a great deal of confidence as we advance to the clinic. In the second half of 2023, we anticipate sharing meaningful clinical data, including splicing in the MAD portion of our ACHIEVE trial that will advance our understanding of the potential for DYNE-101 to transform the lives of people living with DM1.” The ACHIEVE trial is a Phase 1/2 global clinical trial evaluating DYNE-101, consisting of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 64 adult patients with DM1 who are 18 to 49 years of age. The primary endpoints are safety and tolerability; secondary endpoints include pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function. Dyne anticipates reporting data from the MAD placebo-controlled portion of the ACHIEVE trial o...