- Positive cardiopulmonary results and long-term dystrophin data from Phase 1/2 DELIVER trial of z-rostudirsen in exon 51 DMD presented at MDA conference -
- Completion of enrollment in registrational expansion cohort of Phase 1/2 ACHIEVE trial of z-basivarsen in DM1 on track for Q2 2026; global confirmatory Phase 3 HARMONIA trial initiated -
- New preclinical data to be presented at ASGCT underscore differentiated capability of clinically validated FORCETM platform to cross the blood-brain barrier -
WALTHAM, Mass., May 11, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today reported financial results for the first quarter of 2026 and recent business highlights.
"Following positive topline data late last year, we continue to ramp up activities to support a potential launch of z-rostudirsen in DMD in the first quarter of 2027," said John Cox, president and chief executive officer of Dyne. "We are grateful to the FDA for a collaborative pre-BLA meeting, where we aligned on the contents of our planned BLA submission for U.S. Accelerated Approval of z-rostudirsen, which is on track for this quarter."
"In DM1, we have reached our target of 60 participants in the registrational expansion cohort of the ACHIEVE trial of z-basivarsen. Based on a recent acceleration in the number of participants in screening, we currently expect to exceed this target when we complete enrollment later this quarter. As we look toward the important expected readout in the first quarter of 2027, we continue to be driven by our goal to deliver functional improvement for individuals living with DM1. We remain committed to disciplined execution as we advance our two lead programs and broader pipeline and aim to maximize returns for our shareholders," concluded Mr. Cox.
Zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in DMD (Duchenne muscular dystrophy)
Positive cardiopulmonary results and long-term dystrophin data from the DELIVER trial
In March 2026, at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, Dyne presented positive results of new analyses of cardiac and pulmonary function among all DELIVER participants who were randomized to z-rostudirsen treatment at baseline (any dose1) and for whom cardiac magnetic resonance imaging and/or pulmonary function data were available.
At the 2026 MDA Conference, Dyne also presented long-term data on dystrophin production from four participants in the DELIVER trial who had been dosed with 20 mg/kg Q4W for at least 12 months (67-104 weeks) at the time of an optional biopsy. Unadjusted dystrophin production in these participants reached an average of 9.48% of normal (n=4) as compared to 0.52% at baseline (n=3), and muscle content-adjusted dystrophin production reached an average of 18.33% of normal (n=4) as compared to 1.47% at baseline (n=3).
In previously reported safety and tolerability data from 86 total participants enrolled in the DELIVER trial and followed for up to 36 months, z-rostudirsen demonstrated a favorable safety profile,2 and most related treatment emergent adverse events (TEAEs) were mild or moderate. The most commonly reported related TEAEs were pyrexia (fever) and headache. No related serious TEAEs were observed in the registrational expansion cohort (REC).
Key milestones for z-rostudirsen
Dyne has completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) and remains on track to submit a Biologics License Application (BLA) for U.S. Accelerated Approval in Q2 2026.
Dyne plans to initiate a global confirmatory Phase 3 clinical trial of z-rostudirsen in Q2 2026. Dyne has aligned with the FDA on the Phase 3 trial design and protocol.
Dyne continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming the FDA grants Priority Review and approval is received on the anticipated timeline.
Dyne also continues to pursue approval pathways outside of the U.S. for z-rostudirsen in patients with DMD who are amenable to exon 51 skipping.
In addition to z-rostudirsen, Dyne is advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the potential treatment of DMD amenable to skipping of exons 53, 45, 44, and 55, respectively.
Zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in DM1 (myotonic dystrophy type 1)
Phase 3 HARMONIA trial underway
Dyne initiated the global confirmatory Phase 3 HARMONIA trial of z-basivarsen in March 2026. Dyne has aligned with the FDA on the Phase 3 trial design and protocol, which was presented at the 2026 MDA Clinical & Scientific Conference.
Key milestones for z-basivarsen
Dyne has reached its enrollment target of 60 participants in the ACHIEVE REC. Dyne plans to allow any participants currently in screening to enroll if they meet all eligibility criteria. As a result, Dyne expects to complete enrollment of more than 60 participants in Q2 2026.
Data from this cohort are planned for Q1 2027 to support a potential BLA submission for U.S. Accelerated Approval in early Q3 2027.
Dyne intends to use data from the REC and from the already enrolled participants in the multiple ascending dose (MAD) and ongoing long-term extension portions of the ACHIEVE trial to support a potential submission for Accelerated Approval in the U.S.
Dyne expects a potential U.S. launch of z-basivarsen in Q1 2028, assuming FDA grants Priority Review and approval is received on the anticipated timeline.
Dyne also continues to pursue approval pathways outside of the U.S. for z-basivarsen in DM1.
New preclinical data showing robust central nervous system (CNS) activity in nonhuman primates with FORCE platform
This week, Dyne is presenting new preclinical data highlighting the differentiated capability of the clinically validated FORCE platform to cross the blood-brain barrier. The oral presentation will take place on Wednesday, May 13, at 10:30 a.m. ET at the American Society of Gene + Cell Therapy (ASGCT) 2026 Annual Meeting being held in Boston, MA, and virtually.
First Quarter Financial Results
Cash position: Cash, cash equivalents and marketable securities were $972.2 million as of March 31, 2026. The Company continues to expect that its cash, cash equivalents and marketable securities as of March 31, 2026, will be sufficient to fund its operations into the first quarter of 2028.
Research and development (R&D) expenses: R&D expenses were ...
