zeleciment rostudirsen (DYNE-251) / Dyne Therap - LARVOL DELTA

Topline data from Registrational Expansion Cohort of DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in DMD on track for December 2025 to support potential submission for U.S. Accelerated Approval in Q2 2026 (The Manila Times)

FDA approval • P1/2 data • Duchenne Muscular Dystrophy

Z-rostudirsen remains on track for potential launch in Q1 2027 (The Manila Times)

Launch US • Duchenne Muscular Dystrophy

Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy (GlobeNewswire) - "Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025."

Orphan drug • P1/2 data • Duchenne Muscular Dystrophy

Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD) (The Manila Times) - "Dyne Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial...Dyne has completed enrollment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial. Data from this cohort are planned for late 2025.Dyne anticipates a potential BLA submission for U.S. Accelerated Approval in early 2026."

Breakthrough therapy • Duchenne Muscular Dystrophy

DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - P1/2 | N=86 | Active, not recruiting | Sponsor: Dyne Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

TfR1-mediated delivery of oligonucleotides for the treatment of neuromuscular diseases: translating research into clinic (ASGCT 2025) - "We leveraged the FORCE platform to develop DYNE-101 and DYNE-251 as potential treatments for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), respectively. Clinical data in both programs support the potential of the FORCE platform to deliver functional improvement in serious neuromuscular disorders."

CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy • TFRC

DYNE-251 in DMD (GlobeNewswire) - "Dyne has fully enrolled the Registrational Expansion Cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025; Dyne anticipates a potential BLA submission for U.S. Accelerated Approval in early 2026; Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping."

FDA filing • P1/2 data • Duchenne Muscular Dystrophy

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy (GlobeNewswire) - "Dyne Therapeutics, Inc...today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD)....Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference....With full enrollment of the registrational expansion cohort in the DELIVER trial complete, we look forward to sharing data from this cohort in late 2025....Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026."

FDA filing • Orphan drug • P1/2 data • Duchenne Muscular Dystrophy

Key Milestones for the DELIVER Trial (GlobeNewswire) - "Dyne has fully enrolled the Registrational Expansion Cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025."

Enrollment status • P1/2 data • Duchenne Muscular Dystrophy

Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months (GlobeNewswire) - P1/2 | N=88 | DELIVER (NCT05524883) | Sponsor: Dyne Therapeutics | "...Updated results from the trial are being presented this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference....Key findings from the DELIVER Phase 1/2 trial presentation include: Function: Meaningful and sustained improvements from baseline in multiple functional endpoints were observed in both the 20 mg/kg (selected registrational dose) and 10 mg/kg DYNE-251 Q4W cohorts, through 12 and 18 months, respectively....Starting at the 6-month timepoint, the SV95C change from baseline observed in both the 10 mg/kg and 20 mg/kg cohorts of DELIVER exceeded the published proposed minimal clinically important difference (MCID)....At the 6-month time point, patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content)."

P1/2 data • Duchenne Muscular Dystrophy

Safety and Efficacy of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (AAN 2025) - P1/2 | "DYNE-251 had a favorable safety profile and resulted in early improvements across multiple functional endpoints."

Clinical • Duchenne Muscular Dystrophy • Infectious Disease

Dyne Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights (GlobeNewswire) - "Furthermore, in DMD, we expect data from the ongoing DELIVER trial of DYNE-251 in late 2025 to support a potential submission for U.S. Accelerated Approval in early 2026, giving us the transformational opportunity to launch two important therapies in 2027...plans to initiate a global placebo-controlled Registrational Expansion Cohort in the ACHIEVE trial that will include up to 48 patients with full enrollment planned for mid-2025 and data from this cohort planned for H1 2026."

Enrollment status • FDA filing • Launch US • P1/2 data • Duchenne Muscular Dystrophy • Myotonic Dystrophy

Dyne Therapeutics Announces Upcoming Presentations at the 2025 MDA Clinical & Scientific Conference (GlobeNewswire) - "Dyne Therapeutics...announced that the company will be presenting two oral and five poster presentations at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19, 2025, in Dallas, TX, and virtually. The oral presentations include data from the ongoing DELIVER clinical trial in Duchenne muscular dystrophy (DMD) as well as the recent positive results from the ongoing ACHIEVE clinical trial in myotonic dystrophy type 1 (DM1) which will include a summary of data on the use of splicing correction as a prognostic biomarker of functional outcomes in DM1."

Clinical data • Duchenne Muscular Dystrophy • Myotonic Dystrophy

Phase 1/2 DELIVER Trial of DYNE-251 in DMD (GlobeNewswire) - P1/2 | N=88 | DELIVER (NCT05524883) | Sponsor: Dyne Therapeutics | "Dyne reported today updated safety and tolerability data based on 54 participants enrolled in the DELIVER trial. DYNE-251 demonstrated a favorable safety profile, and the majority of treatment emergent adverse events were mild or moderate.2 The safety profile remains unchanged, and no new treatment-related serious adverse events have been observed since the prior update provided as of August 21, 2024. Approximately 837 doses have been administered to date in the DELIVER trial, representing over 65 patient-years of follow-up, with some patients followed for up to 2.2 years...Dyne is currently enrolling a 20 mg/kg (approximate PMO dose) Q4W Registrational Expansion Cohort of approximately 32 participants as part of the DELIVER trial. Dyne anticipates completion of enrollment in Q1 2025 with data from this cohort expected in late 2025."

Enrollment status • P1/2 data • Duchenne Muscular Dystrophy

DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - P1/2 | N=88 | Recruiting | Sponsor: Dyne Therapeutics | Trial completion date: Nov 2026 ➔ Nov 2029 | Trial primary completion date: Nov 2026 ➔ Nov 2029

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (AAN 2024) - P1/2 | "Based on these initial data, DYNE-251 had a favorable safety profile and reached levels of dystrophin expression, exon skipping, and PDPF at 6 months that exceeded levels reported at the same time point in prior clinical trials evaluating the standard of care PMO."

Late-breaking abstract • Anemia • Duchenne Muscular Dystrophy • Hematological Disorders • TFRC

DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - P1/2 | N=88 | Recruiting | Sponsor: Dyne Therapeutics | N=48 ➔ 88

Enrollment change • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (MDA 2024) - P1/2 | "No participants demonstrated treatment-emergent anemia and there were no discontinuations. Safety and tolerability data from multiple cohorts and dystrophin expression data from the 5 mg/kg cohort at 25 Weeks will be presented."

Anemia • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • TFRC

Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (MDA 2024) - No abstract available

Duchenne Muscular Dystrophy

A phase 1/2 study of DYNE-251 in males with DMD mutations amenable to exon 51 skipping: DELIVER study design (WMS 2023) - No abstract available

P1/2 data

Building a FORCETM platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping (WMS 2022) - "FORCE-M23D is a mouse-specific Fab-PMO conjugate designed to target TfR1 and skip exon 23 of the murine Dmd transcript to restore dystrophin expression in the mdx mouse model of DMD...Additionally, Dyne's clinical candidate, DYNE-251, a Fab-conjugated PMO designed to skip DMD exon 51, was evaluated in non-human primates (NHPs)...Lastly, we have begun development of FORCE conjugates for the treatment of exon 53 and exon 45 skipping amenable patients...This conjugate resulted in superior skipping of exon 53 compared to unconjugated PMO. Collectively, these data support utilizing the FORCE platform for the development of therapies for individuals with DMD mutations amenable to exon skipping."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TFRC

DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - P1/2 | N=46 | Recruiting | Sponsor: Dyne Therapeutics

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy