Dyne Therapeutics (DYN) Earnings Call Presentation

Clinical Trial & Regulatory Strategy - Dyne and the FDA have agreed on the next step toward Accelerated Approval, which involves submitting the revised protocol for the Registrational Expansion Cohort of the ACHIEVE trial with vHOT as the primary endpoint[9] - The FDA has granted Breakthrough Therapy Designation for DYNE-101 in DM1 following a Type C Meeting[9] - Data from the Registrational Expansion Cohort are planned for mid-2026 to support a potential U S Accelerated Approval submission in late 2026[9] - The company is planning to initiate a confirmatory Phase 3 clinical trial in Q1 2026[54] DYNE-101 Efficacy Data - New data from the ACHIEVE trial demonstrate robust and sustained functional improvement across multiple measures at 6 and 12 months at the registrational dose of 6 8 mg/kg Q8W[9] - Data support vHOT improvement as an early indicator of clinical benefit with DYNE-101[9, 22, 24, 27, 41, 48] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -3 0 second change from baseline in vHOT Middle Finger, a -39% change from baseline, compared to a +0 4 second change and +5% change from baseline in the placebo group[30] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -2 9 second change from baseline in vHOT Middle Finger, a -38% change from baseline[30] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a 10 3 %p change from baseline in Quantitative Muscle Testing (QMT) Total Score, a 20 1% change from baseline, compared to a -2 0 %p change and -3 9% change from baseline in the placebo group[32] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a 5 2 %p change from baseline in Quantitative Muscle Testing (QMT) Total Score, a 10 1% change from baseline[32] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -1 5 second change from baseline in the 5 Times Sit to Stand Test, a -14 5% change from baseline, compared to a +0 5 second change and +5 2% change from baseline in the placebo group[34] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -1 2 second change from baseline in the 5 Times Sit to Stand Test, a -12 4% change from baseline[34] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -0 4 second change from baseline in the 10-Meter Walk/Run Test, a -9 0% change from baseline, compared to a +0 1 second change and +4 3% change from baseline in the placebo group[34] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -0 3 second change from baseline in the 10-Meter Walk/Run Test, a -7 2% change from baseline[34] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -12 3 change from baseline in the Myotonic Dystrophy Health Index (MDHI) Total Score, a -46 5% change from baseline, compared to a +1 9 change and +10% change from baseline in the placebo group[37] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -11 7 change from baseline in the Myotonic Dystrophy Health Index (MDHI) Total Score, a -44% change from baseline[37] Safety Profile - DYNE-101 has demonstrated a continued favorable safety profile[9, 22, 23, 27, 41] - Approximately 1000 doses of the study drug have been administered to date, representing 93 patient-years of follow-up[45] Financial Position - The company has a strong financial position with $677 5 million in cash as of March 31, 2025, with an expected runway into Q4 2026[62]