Overviewing Positive Early Data from ACHIEVE Trial of Zeleciment Basivarsen in DM1: Doug Kerr, MD, PhD

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"Patients are showing substantial and sustained improvement across measures of strength, function, and cognition. For a condition with no approved therapies, these early results offer real, measurable hope for people living with myotonic dystrophy."

Myotonic dystrophy type 1 (DM1) remains one of the most challenging neuromuscular disorders to treat, with no currently approved disease-modifying therapies. The condition, caused by a toxic RNA repeat expansion in the DMPK gene, results in widespread splicing defects that affect skeletal, cardiac, and smooth muscle as well as the central nervous system. These abnormalities manifest as progressive muscle weakness, myotonia, cognitive impairment, and multisystem dysfunction. New therapeutic approaches are focusing on RNA-targeted strategies and improved delivery platforms to address the underlying genetic mechanisms of the disease.

One such investigational agent, zeleciment basivarsen (z-basivarsen; formerly DYNE-101), is being developed by Dyne Therapeutics as part of its FORCETM platform, designed to enhance oligonucleotide delivery to muscle and CNS tissue. The agent is currently being evaluated in the phase 1/2 ACHIEVE study (NCT05481879), a global, double-blind, placebo-controlled trial assessing safety, tolerability, and efficacy in patients with DM1.

NeurologyLive^® recently sat down withDoug Kerr, MD, PhD, chief executive officer at Dyne Therapeutics, to discuss the ins and outs of the trial, and the early results backing the agent. He spoke about the consistent improvements across measures of myotonia, muscle strength, and CNS function, with sustained gains observed through 12 months. He noted that these data have positioned z-basivarsen as a potential first-in-class therapy for DM1, with phase 3 plans slated for 2026.