– Presentation and Discussion with Two Leading Neuromuscular Disease Experts –
– Data from DYNE-101 ACHIEVE and DYNE-251 DELIVER Clinical Trials Anticipated in the Second Half of 2023 –
– Refined Focus on Lead Clinical Programs Extending Cash Runway Through 2024 –
WALTHAM, Mass., Sept. 12, 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 hosts a virtual event, “Spotlight on the Clinic,” highlighting its clinical programs, DYNE-101 in myotonic dystrophy type 1 (DM1) and DYNE-251 in Duchenne muscular dystrophy (DMD) from 7:30-9:00 a.m. ET. Speakers include neuromuscular disease experts, Valeria Sansone, M.D., Ph.D., Clinical and Scientific Director at Clinical Center NeMO; and Professor of Neurology, University of Milan, Richard Finkel, M.D., Director of the Center for Experimental Neurotherapeutics at St. Jude Children’s Research Hospital, as well as members of Dyne’s leadership team. The live virtual event as well as the replay and slide presentation are available at https://investors.dyne-tx.com/events/event-details/dyne-spotlight-clinic-virtual-event.
“With our DYNE-251 and DYNE-101 clinical trials now underway, this is a very exciting time for Dyne and people living with DMD and DM1. DELIVER and ACHIEVE, which are both designed to be registrational trials, are expected to report data in the second half of 2023, including evaluating important biomarkers of dystrophin in DMD and splicing in DM1,” said Joshua Brumm, president and chief executive officer of Dyne. “We are extremely grateful to Drs. Sansone and Finkel for sharing their insights today, including on the unmet needs of their patients and where new therapies have the potential to address diseases with no or limited treatment options.”
In addition to remarks and discussion with the neuromuscular disease experts, the event includes a review of Dyne’s clinical programs and pipeline:
- DELIVER Trial in DMD: Patient dosing is underway in DELIVER, a Phase 1/2 global clinical trial evaluating DYNE-251 for the treatment of DMD mutations amenable to exon 51 skipping. The DELIVER trial consists 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 46 ambulant and non-ambulant males with DMD who are ages 4 to 16 and have mutations amenable to exon 51 skipping therapy. The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping and pharmacokinetics. Data from the MAD placebo-controlled portion of the DELIVER trial on safety, tolerability and dystrophin are anticipated in the second half of 2023.
- ACHIEVE Trial in DM1 – Dyne has initiated its ACHIEVE trial evaluating DYNE-101 for the treatment of DM1. The ACHIEVE trial is a Phase 1/2 global clinical trial consisting of a 24-week 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. Data from the MAD placebo-controlled portion of the ACHIEVE trial on safety, tolerability and splicing are anticipated in the second half of 2023.
- Pipeline and Resources: Dyne is prioritizing its focus and resources on its clinical programs, DYNE-101 in DM1 and DYNE-251 in DMD. The Company remains committed to advancing its facioscapulohumeral muscular dystrophy (FSHD) program but is deferring the Investigational New Drug application submission for DYNE-301 originally targeted for the second half of 2022. As a result, Dyne expects its cash runway will be extended through 2024 and plans to provide an update on its FSHD program in 2023.
