Dyne Therapeutics Announces Additional One-Year Clinical Data from Phase 1/2 ACHIEVE Trial of Z-Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1 (DM1)
Globe Newswire•29/09/2026•16:05 ET
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Key Highlights
- ➤12-month data showed functional improvement across multiple measures versus baseline and matched natural history.
- ➤vHOT improved 3.2 seconds at six months versus 0.4-second placebo worsening.
- ➤Q1 2027 topline ACHIEVE REC data remain expected for potential Q3 2027 U.S. Accelerated Approval submission.
- ➤HARMONIA Phase 3 trial continues recruiting at over 35 active global sites.
- ➤No related serious treatment-emergent adverse events identified through April 20, 2026.
Expert Statements
Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne Therapeutics
“This week we are presenting data at 6 and 12 months from a larger pooled dose group of participants from ACHIEVE, which further support our confidence in the results we have previously presented from smaller individual dose cohorts.”
Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne Therapeutics
“In addition, for the first time, we are showing functional improvement across multiple measures compared to a matched natural history cohort, not just compared to placebo and compared to baseline.”
Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne Therapeutics
“We attribute these results to the differentiated properties of our FORCE platform and z-basivarsen, which is designed to correct the core underlying splicing abnormalities in DM1 by delivering a targeted therapeutic payload to the nucleus in both muscle and the CNS.”
Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne Therapeutics
“We aim to confirm these findings through both the ACHIEVE REC and the Phase 3 HARMONIA study, which is currently recruiting participants at over 35 active global sites.”
- Data from a pooled dose group of participants initially enrolled in the multiple ascending dose (MAD) portion of ACHIEVE showed functional improvement versus baseline, versus placebo and versus a matched natural history cohort across multiple measures at 12 months -
- Data continued to support improvement at 6 months in video hand opening time (vHOT) as an early indicator of clinical benefit with z-basivarsen in DM1 -
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