Design Therapeutics Announces Favorable Phase 1 Data for DT-168 Supporting Advancement into Phase 2 Biomarker Trial for Patients with Fuchs Endothelial Corneal Dystrophy

Core Insights - Design Therapeutics, Inc. announced favorable data from a Phase 1 trial of DT-168, a treatment for Fuchs endothelial corneal dystrophy (FECD), which will be presented at an upcoming industry event [1][3] - DT-168 is a GeneTAC® small molecule eye drop targeting the mutant TCF4 gene responsible for FECD, a condition affecting millions with no approved disease-modifying therapies [2][3] - The company plans to initiate a Phase 2 biomarker trial later in 2025 to further evaluate DT-168's safety and efficacy in FECD patients [5] Group 1: Clinical Trial Results - The Phase 1 trial was a double-masked, placebo-controlled study involving 24 healthy volunteers who received either placebo or DT-168 eye drops twice daily for seven days [3][8] - DT-168 was well-tolerated with no serious adverse events or ocular adverse events reported, and systemic exposure was below the limit of quantitation across all participants [8] Group 2: Future Development Plans - Design Therapeutics plans to conduct a Phase 2 biomarker trial to assess DT-168's safety and corneal endothelium biomarkers in FECD patients scheduled for corneal transplant surgery [5] - The Phase 2 trial will involve administering 0.5% DT-168 eye drops twice daily for approximately four weeks before surgery, with results expected in 2026 [5] Group 3: Company Overview - Design Therapeutics is focused on developing a new class of therapies using its GeneTAC® platform, which targets disease-causing genes [6] - The company is also advancing other programs for conditions such as Friedreich ataxia, myotonic dystrophy type-1, and Huntington's disease [6]