Design Therapeutics provides corporate highlights, anticipated milestones

Friedreich Ataxia: Design has initiated dosing in a Phase 1 clinical trial in healthy volunteers in Australia to evaluate the safety and pharmacokinetics of single ascending doses of DT-216P2 via multiple routes of administration. A Phase 1/2 multiple ascending dose clinical trial to assess safety, PK and pharmacodynamics in FA patients is anticipated to begin in mid-2025. Data based on twelve weeks of DT-216P2 dosing in patients is anticipated in 2026. Fuchs Endothelial Corneal Dystrophy: Design has completed dosing in a Phase 1 MAD clinical trial of DT-168 in healthy volunteers and expects to report data in the first half of 2025. The company achieved its enrollment goal for the observational study by recruiting and completing baseline assessments on approximately 250 FECD patients. Based on the baseline characteristics data, Design has chosen approximately 100 patients for future follow-up visits. Pipeline programs: Design continues to advance preclinical work toward the selection of a development candidate for myotonic dystrophy type-1 later in 2025. Preclinical characterization of several candidate molecules also continues in Huntington’s disease.