Mon. 20 May 2024, 6:33am ET
Benzinga
Biotech, News, Health Care, General
- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose Dependent 27% Mean Splicing Correction Across All Patients in the 5.4 mg/kg Cohort at 3 Months
- DYNE-101 Showed Improvement in Myotonia, Muscle Strength, and Timed Function Tests and in DM1-ACTIVc and MDHI Patient Reported Outcomes
- In Phase 1/2 DELIVER Trial, DYNE-251 Showed 3.2% Mean Unadjusted (7.6% Mean Muscle Adjusted) Dystrophin Expression at 6 Months in 10 mg/kg Cohort Administered Monthly; 10 Times Higher Level of Dystrophin Than Reported for Weekly Administered Standard of Care.
- DYNE-251 Demonstrated Trends in Functional Improvement in NSAA, Time to Rise from Floor, 10-Meter Walk/Run Time, and Stride Velocity 95th Centile at 6 Months in 10 mg/kg Cohort
- Favorable Safety Profiles for DYNE-101 and DYNE-251 with Enrollment Complete in the 6.8 mg/kg Cohort of ACHIEVE and 40 mg/kg Cohort of DELIVER
- Based on Recent Regulatory Interactions, Continuing to Pursue Expedited Approvals for Both Programs with Update on Registrational Pathways Expected by Year-End