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An investigational drug is showing real promise for a disease with no approved treatments. One-year pooled data from the ACHIEVE trial show that z-basivarsen meaningfully reduced hand myotonia and improved motor function in myotonic dystrophy type 1 (DM1) patients, with a favorable safety profile. The confirmatory phase 3 HARMONIA trial is now actively recruiting.
An investigational drug is showing real promise for a disease with no approved treatments. Myotonic dystrophy type 1 (DM1) is a rare, progressive multisystem disorder caused by a mutation in the DMPK gene that disrupts RNA splicing — and right now, patients have no disease-modifying options. Z-basivarsen (DYNE-101), an antisense oligonucleotide designed to break up toxic DMPK RNA and restore normal splicing, is aiming to change that.
One-year pooled data from the phase 1/2 ACHIEVE trial — covering 26 patients across multiple dose cohorts — showed sustained improvements in hand myotonia, motor function (5 times sit-to-stand test), and muscle strength. Patients who crossed over from placebo also showed improvement after switching, reinforcing the drug's treatment effect. The safety profile remained favorable, with no serious drug-related adverse events; mild infusion reactions like fever and headache were manageable with anti-inflammatories.
By the Numbers:
Why it matters: With no approved therapies for DM1, z-basivarsen represents a potentially transformative option. The phase 3 HARMONIA trial — now recruiting — will evaluate motor function and patient-reported outcomes including cognition, bringing the field one step closer to a first-ever disease-modifying treatment for DM1.
