Dyne Therapeutics Reveals Additional One-Year Clinical Data Demonstrating Functional Improvement From Phase 1/2 ACHIEVE Trial of Zeleciment Basivarsen For Myotonic Dystrophy Type 1; Meaningful Improvements In Overall Disease Burden Reported By Both Patients And Physicians

Author: Benzinga Newsdesk | October 06, 2025 05:03pm

WALTHAM, Mass., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced additional one-year data from its ongoing Phase 1/2 ACHIEVE clinical trial of zeleciment basivarsen (z-basivarsen, formerly known as DYNE-101), in patients with myotonic dystrophy type 1 (DM1) demonstrating clinically meaningful improvements in function and strength at the selected registrational dose. These data are being presented at the 30th Annual International Congress of the World Muscle Society (WMS), held virtually and in Vienna, Austria, October 7-11, 2025.

"This week we are presenting additional analyses from the data cut shared in June showing that the improvements in function and strength span both the upper and lower limbs, and are clearly meaningful to both patients and physicians," said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. "Z-basivarsen was designed to deliver broad functional improvement to patients, and we believe it has the unique potential to mitigate central nervous system-related manifestations of the disease such as cognitive impairment, sleep disturbances and fatigue."

"I believe the data for z-basivarsen support its potential to bring a wide range of benefits to DM1 patients, helping to improve their ability to function and carry out their daily lives in a way that will really matter to them," said Valeria Sansone, M.D., Ph.D., Clinical and Scientific Director at the Clinical Center NeMO in Milan, Professor of Neurology, University of Milan, and principal investigator in the ACHIEVE trial. "The consistency of these data across a variety of endpoints out to one-year increases my level of confidence in the potential of z-basivarsen. The patient perception of improvements are encouraging and provide initial evidence that there may be a broad and meaningful effect with z-basivarsen treatment."

The one-year data presented at WMS this week come from adults with DM1 (n=6) enrolled in the cohort assessing the selected registrational dose of 6.8 mg/kg Q8W in the randomized, placebo-controlled multiple ascending dose (MAD) portion of the ACHIEVE trial. These results are from an additional analysis of the same cohort and timepoint for which data were previously reported on June 17, 2025.

Data to be presented at WMS include previously disclosed as well as new findings:

• Myotonia: Robust and sustained improvement from baseline in hand myotonia, as measured by video hand opening time (vHOT).
  
   
• Function: Meaningful and sustained improvements from baseline in multiple functional endpoints, including data from the 10-Meter Walk/Run Test (10MWR) and the 5 Times Sit to Stand Test (5xSTS), as well as:
  
  
  • New data showing improvement in the 9-Hole Peg Test (9HPT), a measure of upper limb function focused on manual dexterity and coordination, which is frequently used across neurological conditions.
    
     
• Strength: Meaningful and sustained improvement from baseline on muscle strength as assessed by Quantitative Muscle Testing (QMT), as well as:
  • New data showing improvements in all QMT scores across both the upper and lower extremities: hand grip strength, elbow flexion, elbow extension, ankle dorsiflexion, knee flexion, and knee extension.
    
     
• Patient Reported Outcomes: Meaningful and sustained improvement from baseline in the Myotonic Dystrophy Health Index (MDHI) patient reported outcome measure, including in 6 subscales assessing central nervous system disease manifestations (cognitive impairment, sleep disturbances, fatigue, communication, emotional issues and pain), as well as:
  • New data showing improvements in other subscales including mobility, ability to do activities, and upper extremity function. These data support the clinical meaningfulness of the improvements seen on QMT, 10MWR, 5xSTS, and 9-HPT.
• Overall Disease Burden: Improvements from baseline in both patient and clinician impressions of global function, based on:
  • New data from Patient Global Impression of Change (PGI-C) and Clinician Global Impression of Change (CGI-C) scales.
    
     
• Safety and Tolerability: Previously reported safety and tolerability data from 56 patients enrolled through the 6.8 mg/kg Q8W cohort of the ACHIEVE trial. Z-basivarsen demonstrated a favorable safety profile, and no related serious treatment emergent adverse events were identified1.

These data will be presented in a poster titled, "DYNE-101 Targets the Underlying Cause of DM1 to Enable Multi-system Functional Improvement in the ACHIEVE Trial."

Additional Dyne Presentations at the 30th Annual International Congress of the World Muscle Society

Dyne will also present encore data on zeleciment rostudirsen (z-rostudirsen, formerly known as DYNE-251, an investigational medicine being evaluated for the treatment of individuals with DMD mutations amenable to exon 51 skipping) as well as preclinical Duchenne muscular dystrophy (DMD) data with its FORCE platform.

• "DYNE-251 Targets the Underlying Cause of DMD to Enable Sustained Functional Improvement in Males with DMD Mutations Amenable to Exon 51 Skipping Enrolled in the Phase 1/2 DELIVER Trial" (encore presentation)
• "The FORCE™ Platform Enables TfR1-mediated Delivery of Exon Skipping PMO to the CNS and Resolves Anxiety in a Mouse Model of DMD" (encore presentation)

All poster presentations, including the poster titled, "DYNE-101 Targets the Underlying Cause of DM1 to Enable Multi-system Functional Improvement in the ACHIEVE Trial," are now available in the Scientific Publications & Presentations section of Dyne's website.

Additionally, a symposium titled "Achieving Functional Improvement in Myotonic Dystrophy Type 1 (DM1): Defining Goals of Treatment and a Roadmap to Multidisciplinary Care" will be held on Wednesday, October 8 at 7:45 a.m. CEST. Slides from the symposium will be available in the Scientific Publications & Presentations section of Dyne's website on Wednesday, October 8 at the commencement of the symposium.

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