Dyne hopes to bring new treatment for DM1 to US market in 2028
Company is targeting a mid-2027 application for FDA accelerated approval
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Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen (z-basivarsen), an experimental treatment for myotonic dystrophy type 1 (DM1).
The company announced that enrollment is now complete for this key group of patients. If the results are positive, Dyne plans to apply for accelerated approval from the U.S. Food and Drug Administration (FDA) by mid-2027, aiming to launch the therapy in the first half of 2028. The company also intends to seek approval in other countries.
Accelerated approval goals
“Completing enrollment in the registrational expansion cohort of ACHIEVE is a critical milestone as we advance a potentially best-in-class therapy designed to address the multi-system nature of DM1,” Doug Kerr, MD, PhD, chief medical officer of Dyne, said in a company press release. “With data from this cohort expected in the first quarter of next year, we believe we are well positioned to show a significant improvement in myotonia [where muscles cannot relax properly after contracting] and trends on important functional outcomes to support a potential submission for U.S. Accelerated Approval.”
Accelerated approval is a regulatory mechanism in which the FDA can allow a therapy to be brought to market based on early data indicating that it is likely effective. If z-basivarsen ultimately gets accelerated approval, Dyne will be required to run additional testing to prove benefit, with long-term approval of the drug contingent on those results.
Dyne is already running a Phase 3 trial, dubbed HARMONIA (NCT07486934), that aims to demonstrate clinical benefit and support the transition to full approval. That study plans to enroll about 150 people with DM1, 16 and older. Participants will be randomly assigned to receive z-basivarsen or a placebo for a little less than one year, with the main goal of testing if the experimental therapy improves a measure of motor function called the five-times sit-to-stand test. Recruitment is ongoing at sites in the U.S. and Japan.
“We expect that the strong interest we have seen in z-basivarsen will support enrollment in the ongoing confirmatory Phase 3 HARMONIA trial,” Kerr said.
DM1 is a genetic disease marked by symptoms such as muscle wasting and myotonia. DM1 is caused by mutations in the DMPK gene, which leads to the production of a toxic RNA molecule that forms clumps in muscle cells. These clumps disrupt RNA splicing, a process that cells normally use to regulate the activity of many genes. Z-basivarsen, previously known as DYNE-101, is designed to clear the toxic RNA clumps to normalize splicing.
The first part of the Phase 1/2 ACHIEVE trial (NCT05481879) tested various doses of z-basivarsen to identify an optimal dose. The now fully-enrolled registrational cohort is testing that dose (6.8 mg/kg of z-basivarsen administered every eight weeks) against a placebo in 71 participants. The registrational cohort’s main goal is to see if z-basivarsen improves video hand opening time (vHOT) test, an assessment of hand myotonia.
Previous data from the first part of ACHIEVE indicated that z-basivarsen improved vHOT as well as a range of other functional and cognitive measures.
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