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An analysis of 26 people with myotonic dystrophy type 1 in the phase I/II ACHIEVE trial found sustained improvements across several measures after treatment with investigational z-basivarsen. The results were presented at an October 2026 medical meeting, but they are not final evidence of efficacy; topline data from a larger registrational cohort are expected in early 2027.
An analysis of 26 people with myotonic dystrophy type 1 in the phase I/II ACHIEVE trial found sustained improvements in measures of hand myotonia, physical function and strength after treatment with investigational z-basivarsen, according to data presented at an October 2026 medical meeting. The findings are encouraging but preliminary: the group received pooled doses, and a larger registrational cohort is expected to provide more evidence in 2027.
The analysis followed participants in the trial’s multiple ascending dose portion for up to 12 months. At baseline, their mean video hand-opening time (vHOT), a measure of how long it takes to open the hand, was 8.2 seconds. At six months, the measure had improved by a mean of 3.2 seconds from baseline. The report said this was a 3.6-second improvement relative to the placebo group, whose mean vHOT increased by 0.4 seconds over the same period. The 3.2-second change is from baseline; it is not a reported 12-month vHOT change.
Investigators also reported sustained improvement from baseline on the five-times sit-to-stand test, which measures functional mobility, and in total scores on quantitative muscle testing. Participants reported improvements in disease burden. The company’s clinical development vice president, Shauna Andersson, MD, PhD, also described better functional and strength outcomes compared with an untreated natural-history cohort. Those comparisons do not establish that the drug caused the differences, and the supplied report does not give detailed numerical results for each endpoint.
Andersson reported that z-basivarsen continued to show a favorable safety profile in the analysis. Treatment-related adverse events included infusion-related reactions, nasopharyngitis, diarrhea, headache, procedural pain, influenza and back pain. No serious treatment-emergent adverse events related to the study drug were identified in the reported data. This finding describes the trial population and follow-up available; it does not rule out risks that could emerge in larger or longer studies.
Signals Across DM1 Function Measures
DM1 is a progressive genetic condition, and the source report says there are currently no disease-modifying therapies; care is focused on managing symptoms. A treatment that improves hand function, mobility or strength could matter to daily activities, but these trial measures are indicators rather than proof of changes in long-term independence or quality of life.
The results add to the case for testing z-basivarsen in larger studies because the reported improvements extended beyond hand myotonia to physical function and muscle strength. However, the analysis pooled participants who received different doses, most of them lower than the selected registrational dose. It should be read as an early efficacy signal, not a finding that the drug is proven to work or is available as treatment.
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From ACHIEVE to Larger Trials
Z-basivarsen, also known as DYNE-101, is an investigational antisense oligonucleotide attached to an antibody fragment that binds transferrin receptor 1. It is designed to reduce toxic RNA produced by the DMPK gene, which is linked to disrupted RNA splicing in DM1. This is the proposed mechanism; the clinical analysis reports patient outcomes, not proof that the mechanism has been fully validated.
The phase I/II ACHIEVE trial includes both dose-escalation research and a registrational expansion cohort. The separate phase III HARMONIA trial is recruiting, with change from baseline in the five-times sit-to-stand test at 12 months designated as its primary endpoint. The October analysis drew on 26 people from the multiple ascending dose portion, while the registrational expansion cohort is intended to support potential regulatory submissions, including a possible accelerated approval application in the United States.
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Dose and Comparison Limits
The 26-person analysis was not the registrational expansion cohort, and the reported results combine participants who received different doses. The available account does not specify the full dose distribution, provide detailed 12-month numerical changes for every measure, or describe the statistical methods and uncertainty ranges for the comparisons. The placebo comparison cited for vHOT covers the six-month result; the natural-history comparison is a different type of comparator and is not the same as a randomized placebo comparison.
It is also not yet clear from these findings how durable the reported changes will be beyond the follow-up period, how benefits and risks will compare at the selected dose in a larger population, or whether regulators will consider the evidence sufficient for approval. Z-basivarsen remains investigational, and the reported safety experience cannot settle its risk profile.
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Registrational Results Due in 2027
Topline results from the ACHIEVE registrational expansion cohort are planned for the first quarter of 2027. The cohort’s primary endpoint is the change from baseline in middle-finger myotonia measured by vHOT at six months, compared with placebo. Its 71 participants had a mean baseline vHOT of 8.3 seconds, according to the source report.
Those results should offer a more direct test of the selected dose against placebo on the designated primary measure. HARMONIA is also recruiting and will assess the five-times sit-to-stand test at 12 months as its primary endpoint. Until those data are reported and reviewed, the current analysis supports further study but does not establish whether z-basivarsen will become an approved DM1 treatment.
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Key Questions
What did the ACHIEVE analysis find?
Among 26 participants in a pooled-dose analysis, researchers reported improvement from baseline in hand myotonia, sit-to-stand function and muscle-strength measures, with some improvements sustained through the reported follow-up. The vHOT measure improved by a mean of 3.2 seconds from baseline at six months.
Is z-basivarsen approved for DM1?
No. Z-basivarsen is investigational and is being studied in clinical trials. The analysis does not establish regulatory approval or availability as a treatment.
How strong is the evidence so far?
The findings are an early clinical signal from a 26-person pooled-dose analysis, not a definitive result. The reported natural-history comparison is not a randomized placebo comparison, and detailed results for all measures were not provided in the source account.
When are the next results expected?
Topline data from the ACHIEVE registrational expansion cohort are planned for the first quarter of 2027. The phase III HARMONIA trial is also recruiting, but the source report does not give a results date for that study.
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