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Sep 30, 2024
Dyne Therapeutics Q3 2024 Earnings Report
Dyne Therapeutics reported third quarter results and provided a corporate update.
Key Takeaways
Dyne Therapeutics reported positive progress in their ACHIEVE and DELIVER trials, with IND clearance for DYNE-101. The company is enrolling a registrational cohort of DYNE-251 DELIVER trial in DMD and anticipates reporting new data from the ACHIEVE trial in early January 2025. The company's cash position is expected to fund operations into the second half of 2026.
FDA cleared the IND application for DYNE-101 for DM1.
New clinical data from DYNE-101 ACHIEVE trial is expected in early January 2025.
Enrollment of registrational cohort of DYNE-251 DELIVER trial in DMD is underway.
Cash, cash equivalents and marketable securities were $723.7 million as of September 30, 2024, anticipated to fund operations at least into the second half of 2026.
Dyne Therapeutics
Dyne Therapeutics
Forward Guidance
Dyne Therapeutics 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. Based on the encouraging biomarker and functional data from the DELIVER trial of DYNE-251 in DMD, they are enrolling patients in a registrational cohort at 20 mg/kg. The Company continues to pursue expedited approval pathways for DYNE-101 and DYNE-251.
Positive Outlook
- New data from the ACHIEVE trial is expected in early January 2025.
- 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.
- 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.
Challenges Ahead
- Uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials.
- Uncertainties as to the availability and timing of results from preclinical studies and clinical trials.
- The timing of and Dyne’s ability to enroll patients in clinical trials.
- Whether results from preclinical studies and initial data from early clinical trials will be predictive of the final results of the clinical trials or future trials.
- Uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and the regulatory approval process.