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An analysis of 26 participants in the phase I/II ACHIEVE trial found sustained improvement from baseline in measures of hand myotonia, function and strength after up to 12 months of z-basivarsen treatment. The findings are preliminary; a separate registrational expansion cohort is expected to report topline results in the first quarter of 2027.
An analysis of 26 people with myotonic dystrophy type 1 (DM1) in the phase I/II ACHIEVE trial found sustained improvement from baseline in measures of hand myotonia, function and strength during up to 12 months of treatment with investigational z-basivarsen. The findings, presented by Dyne Therapeutics clinical development executive Shauna Andersson at a medical meeting in Orlando, are early clinical evidence, not proof of benefit; results from a separate registrational cohort are still pending.
The analysis pooled participants from the ACHIEVE trial’s multiple ascending dose portion. Most received doses below the company’s selected 6.8 mg/kg dose given once every eight weeks. Researchers assessed whether improvement trends previously observed in a smaller group of six participants who received that dose also appeared in the larger group. The analysis followed the pooled-dose participants for up to 12 months.
Hand myotonia was measured using video hand-opening time (vHOT). The pooled-dose group’s mean starting score was 8.2 seconds, and the measure had decreased by 3.2 seconds at six months. The report said this was a 3.6-second improvement relative to the placebo group in the multiple ascending dose portion, whose vHOT increased by 0.4 seconds over the same period. These figures describe the six-month comparison; the report also said improvement from baseline was sustained at one year.
The group also showed sustained improvement from baseline on the five-times sit-to-stand test (5xSTS) and quantitative muscle testing (QMT) total scores, which assess function and strength. Participants reported improvement in disease burden, including on the Myotonic Dystrophy Health Index. Andersson also described better functional and strength outcomes compared with a natural-history cohort. The source report does not provide numerical effect sizes for those comparisons.
Potential Gains Across DM1 Measures
DM1 is a progressive disorder, and the source report says there are no disease-modifying therapies currently available; care is focused on managing symptoms. Improvement across measures of hand myotonia, everyday function and muscle strength could matter to patients if it is confirmed in larger and more definitive studies. The findings also offer a signal that the treatment’s effects may extend beyond a single test of muscle stiffness.
However, the results should be read as preliminary. The analysis included 26 participants from a dose-escalation part of a phase I/II trial, and most received less than the selected registrational dose. Comparisons with untreated natural-history participants can add context but do not have the same evidentiary weight as a randomized, controlled comparison. The registrational cohort and phase III study are intended to provide further evidence.
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ACHIEVE and the Next Trial Tests
Z-basivarsen, also known as DYNE-101, is an investigational antisense oligonucleotide linked to an antigen-binding fragment that binds to transferrin receptor 1. It is designed to reduce toxic RNA produced by the DMPK gene, which is implicated in DM1, and thereby support more normal RNA processing. The proposed mechanism is a treatment rationale, not evidence by itself that the drug improves patients’ health.
ACHIEVE includes a registrational expansion cohort of 71 participants. Its primary endpoint is change from baseline in middle-finger myotonia, measured by vHOT at six months against placebo. The cohort’s mean baseline vHOT was 8.3 seconds. The company plans to report topline results in the first quarter of 2027. Z-basivarsen is also being studied in the phase III HARMONIA trial, which the source described as recruiting; its primary endpoint is change from baseline in 5xSTS at 12 months.
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Questions Before Efficacy Is Established
The analysis does not establish whether z-basivarsen causes the reported improvements or how large and durable any benefit may be across a broader DM1 population. The supplied report gives a six-month vHOT comparison with placebo, but does not provide detailed numerical results for the 12-month functional and strength measures or the natural-history comparisons. It also does not specify the full dose distribution within the pooled group.
Safety findings remain limited to the trial data described so far. Reported treatment-related adverse events included infusion-related reaction, nasopharyngitis, diarrhea, headache, procedural pain, influenza and back pain. The report said no serious treatment-emergent adverse events related to the study drug were identified, but it did not give event rates or longer-term safety information. Whether benefits and risks hold up in larger, longer studies remains unknown.
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Registrational Results Due in 2027
The next major data point is topline reporting from the 71-person ACHIEVE registrational expansion cohort, planned for the first quarter of 2027. Its placebo-controlled six-month vHOT endpoint will test whether the hand-myotonia signal appears at the selected dose in a dedicated cohort. Those results may inform potential regulatory submissions, including a possible accelerated approval request in the United States, according to the source report; no approval has been granted.
The phase III HARMONIA trial is also recruiting, with 5xSTS change at 12 months as its primary endpoint. Its results will be needed to assess the treatment in a larger study. Until those data are available, the current analysis remains an encouraging but preliminary report rather than a settled finding about z-basivarsen’s effectiveness.
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Key Questions
What did the ACHIEVE analysis find?
Among 26 participants in a pooled-dose analysis, researchers reported sustained improvement from baseline in measures of hand myotonia, function and strength during up to 12 months of z-basivarsen treatment. The findings are preliminary and do not establish that the drug caused the changes.
How was hand myotonia measured?
Researchers used video hand-opening time (vHOT). The pooled group’s mean baseline was 8.2 seconds; at six months it had decreased by 3.2 seconds. The report said this was a 3.6-second improvement relative to the placebo group, whose measure increased by 0.4 seconds over that period.
Is z-basivarsen approved for DM1?
No. Z-basivarsen is investigational and is being studied in clinical trials. The results described here do not mean the treatment is approved or proven effective.
When are the next results expected?
Topline results 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 did not give a results date for that study.
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