DYN - Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints | Benzinga
- 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 Care1-
- 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 -
- Virtual Investor Event Today, May 20 at 8:00 a.m. ET -
WALTHAM, Mass., May 20, 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 announced positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles.
"We are excited to report new clinical data from both our ACHIEVE and DELIVER trials demonstrating meaningful impact on key biomarkers and functional improvement in multiple clinical endpoints that matter to patients. We believe these data reflect the best-in-class potential for these product candidates and reinforce the opportunity to transform the treatment of DM1 and DMD as well as the potential of the FORCE platform to address other rare muscle diseases," said John Cox, Dyne's president and chief executive officer.
"We believe the breadth and depth of these data are truly differentiating. Our robust preclinical work is translating into clinical benefit along with favorable safety profiles for both DYNE-101 and DYNE-251. In ACHIEVE, treatment with DYNE-101 demonstrated consistent, dose-dependent splicing correction, which led to an improvement in muscle strength, function, and patient reported outcomes. In DELIVER, treatment with DYNE-251 resulted in dystrophin expression that exceeded levels that have been reported for the standard of care for DMD as well as trends in functional improvement earlier than expected," said Wildon Farwell, M.D., MPH, Dyne's chief medical officer. "The DM1 and Duchenne communities have waited too long for new and better therapeutic options. Building on the strength of these encouraging data and recent regulatory interactions, we look forward to continuing to engage with global regulatory authorities throughout this year to pursue expedited approval pathways and address the urgent unmet medical needs in these communities. We are thankful to the participants and clinicians in these trials and the communities for their continued partnership."
Phase 1/2 ACHIEVE Trial of DYNE-101 in DM1
Efficacy Data
Dyne reported efficacy data from 40 adult DM1 patients enrolled in the randomized, placebo-controlled multiple ascending dose (MAD) portion of the DYNE-101 ACHIEVE trial, including 12-month data from the 1.8 mg/kg Q4W (approximate ASO dose) cohort (n=16), 6-month data from the 3.4 mg/kg Q4W cohort (n=16), and 3-month data from the 5.4 mg/kg Q8W cohort (n=8).
In the ACHIEVE trial, DYNE-101 demonstrated robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in multiple functional endpoints and patient reported outcomes.
Key findings from ACHIEVE include:
Splicing
- DYNE-101 continued to show dose dependent splicing correction as seen in earlier cohorts. Patients in the 5.4 mg/kg Q8W cohort had a 27% mean splicing correction from baseline across a broad, 22-gene panel at 3 months, with all participants demonstrating splicing correction.
Function
- Myotonia (vHOT): DYNE-101 demonstrated an improvement in myotonia as measured by video hand opening time (vHOT) in all reported cohorts. The 1.8 mg/kg Q4W group had a mean 3.1 second benefit in myotonia at 3 months that increased to 4.4 seconds at 12 months. In addition, the 5.4 mg/kg Q8W cohort had a mean 4.5 second improvement in myotonia at 3 months.
- Strength and Timed Assessments: DYNE-101 demonstrated an improvement in muscle strength as measured by Quantitative Myometry Testing (QMT), a test of muscle strength and fatigue, and early and sustained potential benefit in 10-Meter Walk/Run Test and 5 Times Sit to Stand Test.
Patient Reported Outcomes (PROs)
- Myotonic Dystrophy Health Index (MDHI): DYNE-101 demonstrated an overall improvement in the MDHI and benefit in all 17 subscales, including those that assess peripheral muscles, central nervous system, and gastrointestinal measures. These represent some of the most burdensome manifestations of DM1 and daily quality of life issues for patients and their families.
- Myotonic Dystrophy Type 1 Activity and Participation Scale (DM1-ACTIVc): Treatment with DYNE-101 resulted in an improvement in the assessment of various activities of daily living, as measured by DM1-ACTIVc.
Safety and Tolerability Data
- Dyne also reported safety and tolerability data from 56 patients enrolled through the 6.8 mg/kg Q8W cohort of the MAD portion of ...