Design Therapeutics Highlights Progress Across Lead GeneTAC® Programs and Reports Fourth Quarter and Full Year 2024 Financial Results

Core Insights - Design Therapeutics, Inc. has initiated a Phase 1 single ascending dose trial of DT-216P2 for Friedreich Ataxia (FA) with patient dosing expected to begin in mid-2025 [1][6] - The company has completed dosing in the Phase 1 trial for DT-168 and anticipates data release in the first half of 2025 [1][6] - Design Therapeutics is well-capitalized with cash and securities amounting to 245.5 million, which is expected to fund operations through multiple potential clinical proof-of-concept data sets [1][4] Clinical Development Progress - The Phase 1 trial for DT-216P2 is being conducted in healthy volunteers in Australia, focusing on safety and pharmacokinetics [6] - A Phase 1/2 multiple ascending dose trial for FA patients is anticipated to start in mid-2025, with data expected in 2026 [6] - The company has achieved its enrollment target for the observational study in Fuchs Endothelial Corneal Dystrophy (FECD), recruiting approximately 250 patients [6] Financial Performance - Research and development (R&D) expenses for Q4 2024 were 12.2 million, totaling 44.4 million for the year [6][10] - General and administrative (G&A) expenses for Q4 2024 were 4.5 million, with an annual total of $18.0 million [6][10] - The net loss for Q4 2024 was$13.7 million, with a total net loss of $49.6 million for the year [6][10] Future Outlook - The company expects to select a development candidate for myotonic dystrophy type-1 (DM1) in 2025 [1][6] - Design Therapeutics aims to advance its GeneTAC platform, with multiple clinical proof-of-concept data sets anticipated over the next few years [2][6]