Dyne Therapeutics Reports Third Quarter 2024 Financial Results and Provides Corporate Update

In This Article:

Dyne Therapeutics, Inc.
Dyne Therapeutics, Inc.

- IND Application for DYNE-101 for DM1 Cleared by FDA -

- New Clinical Data from DYNE-101 ACHIEVE Trial Expected in Early January 2025 -

- Enrolling Registrational Cohort of DYNE-251 DELIVER Trial in DMD -

WALTHAM, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the third quarter of 2024 and provided a corporate update.

“We’ve made significant progress in our ACHIEVE and DELIVER trials. We are very pleased to have received IND clearance for DYNE-101 and plan to report in early January additional data from the ACHIEVE trial in DM1, including from the 6.8 mg/kg cohort, which will inform our go-forward dose and dose regimen. Based on the encouraging biomarker and functional data from the DELIVER trial of DYNE-251 in DMD, we are enrolling patients in a registrational cohort at 20 mg/kg. We continue to pursue expedited approval pathways for both DYNE-101 and DYNE-251,” said John Cox, president and chief executive officer of Dyne. “Our strong financial foundation positions us to advance our clinical programs as well as our pipeline to address the significant unmet needs of people living with neuromuscular diseases.”

ACHIEVE Trial of DYNE-101 in DM1

  • The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for DYNE-101, which is being evaluated in the ongoing, global Phase 1/2 ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose).

  • Dyne anticipates reporting in early January 2025 new data from the ACHIEVE trial, including safety and tolerability, change from baseline in splicing, video hand opening time (vHOT) assessment, functional measures, as well as patient-reported outcomes.

    • Efficacy data will be shared from the 6.8 mg/kg cohort up to 6 months and the 5.4 mg/kg and 3.4 mg/kg cohorts up to 12 months.

    • Longer-term safety and tolerability data from all participants will be reported.

  • Dyne continues to pursue expedited approval pathways globally for DYNE-101 utilizing splicing as a surrogate endpoint.

DELIVER Trial of DYNE-251 in DMD

  • In September 2024, Dyne reported positive efficacy data and a favorable safety profile1 from the ongoing Phase 1/2 global DELIVER trial of DYNE-251 in males with Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. Based on these data, Dyne has begun enrolling a 20 mg/kg Q4W (approximate PMO dose) registrational cohort of 32 participants as part of the DELIVER trial. The Company continues to pursue expedited approval pathways for DYNE-251, including accelerated approval in the U.S. based on dystrophin as a surrogate endpoint.

Waiting for permission
Allow microphone access to enable voice search

Try again.