Can Dyne Deliver And Achieve Milestones In DMD And Myotonic Dystrophy Trials?

(RTTNews) - Dyne Therapeutics Inc. (DYN), a clinical-stage company developing treatments for genetically driven neuromuscular diseases, is advancing its programs in Myotonic Dystrophy and Duchenne Muscular Dystrophy, aiming for potential commercial launches by 2027.

Myotonic dystrophy type 1 (DM1), also known as a triplet repeat disease, is caused due to a defective DMPK gene. The DMPK gene provides instructions for making a protein called myotonic dystrophy protein kinase, which plays an important role in the development of muscle, heart, and brain cells. Muscle weakness throughout the body, Myotonia, or difficulty relaxing muscles in the jaw, tongue, throat, lower arm, and hand, Respiratory issues, and Cognitive deficits are some of the symptoms associated with DM1. About 40,000 people in the U.S. and roughly 55,000 people in the EU are affected by this condition.

The company's Myotonic Dystrophy type 1 (DM1) program, DYNE-101, is an investigational therapeutic being evaluated in a phase I/II global clinical trial, dubbed ACHIEVE.

In the ACHIEVE trial, the results of which were reported last May, treatment with DYNE-101 demonstrated consistent, dose-dependent splicing correction, which led to an improvement in muscle strength, function, and patient-reported outcomes.

A Registrational Expansion Cohort of the ACHIEVE trial with video hand opening time (vHOT) as the primary endpoint, to serve as an intermediate clinical endpoint, is enrolling patients, with enrolment expected to be completed next quarter (Q4, 2025). This cohort is expected to enroll 60 participants, randomized 3:1 to receive DYNE-101 6.8 mg/kg every 8 weeks (Q8W) or placebo.

The company expects to report data from the Registrational Expansion Cohort of the ACHIEVE trial by mid-2026 to support a potential U.S. Accelerated Approval submission for DYNE-101 in Myotonic Dystrophy type 1 in late 2026.

The Duchenne Muscular Dystrophy (DMD) therapeutic under development is DYNE-251, which is being tested in a phase I/II global trial for people living with DMD who are amenable to exon 51 skipping, dubbed DELIVER.

DMD is a rare genetic muscle disease caused by a mutation in the DMD gene on the X chromosome. This gene regulates the production of dystrophin, a protein essential to healthy muscle development and function. In people with DMD, dystrophin levels are absent or nearly absent, which causes permanent damage to muscle cells.

In the phase I/II DELIVER trial, the results of which were reported last September, DYNE-251 achieved the highest level of dystrophin expression reported for an exon 51 skipping therapy and improvement in multiple functional endpoints across multiple cohorts, according to the company.

The DELIVER trial also includes a Registrational Expansion Cohort and is fully enrolled with 32 patients, with data from this cohort planned for late 2025.

If all goes according to plan, the company anticipates submitting a BLA for U.S. Accelerated Approval of DYNE-251 in DMD in early 2026.

In addition to the clinical programs, the company has a preclinical drug candidate, DYNE-302, for facioscapulohumeral muscular dystrophy.

Facioscapulohumeral muscular dystrophy (FSHD) is a rare disease characterized by progressive loss of skeletal muscle, which causes profound weakness. FSHD affects roughly 15,000 to 40,000 people in the U.S. and about 20,000 to 50,000 people in the EU.

Cash Position

Dyne ended June 30, 2025, with cash, cash equivalents, and marketable securities of $683.9 million.

Dyne's common stock began trading on the Nasdaq Global Select Market on Thursday, September 17, 2020, under the ticker symbol "DYN", priced at $19 per share.

In the last 1 year, DYN has traded in a range of $6.36 to $47.45. The stock closed yesterday's (Jul.28, 2025) trading at $9.31, down 6.43%.