Business
Design Therapeutics Provides Pipeline Updates and Reports First Quarter 2023 Financial Results
Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Expected in the Third Quarter of 2023 Progress Across GeneTAC™ Small
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Expected in the Third Quarter of 2023 Progress Across GeneTAC™ Small Molecule Pipeline with IND Submissions for FECD and DM1 Programs On-track for the Second Half of 2023 and 2024, Respectively $315.4 Million in Cash and Securities Expected to Support Operating Runway through 2025 CARLSBAD, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today provided updates and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported first quarter 2023 financial results. “2023 is poised to be an important year for Design as we work to bring our novel GeneTAC™ small molecules closer to patients suffering from devastating genetic diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “We have meaningfully progressed DT-216 development and expect to report initial data from the Phase 1 multiple-ascending dose (MAD) trial in the third quarter of this year. As part of this update, we plan to summarize the effects of three weekly doses of DT-216 in people with Friedreich ataxia (FA), including clinical safety and levels of DT-216 in muscle, which will be important as we plan for Phase 2 development. In parallel, we are characterizing and validating FA biomarker assays in blood and skeletal muscle samples from individuals with FA, FA carriers and healthy controls, to determine if they can be used to reliably assess treatment response. Separately, we are preparing to submit a second Investigational New Drug application (IND) in the second half of this year to support development of our novel DT-168 eye drop for patients with Fuchs endothelial corneal dystrophy (FECD) and are further advancing our myotonic dystrophy type-1 (DM1) and research programs, all of which underscore the immense potential of our platform and approach. This is an exciting time for our company, and with a sharp focus on execution of near-term catalysts and a strong balance sheet, I am confident in our ability to deliver on the promise of our potentially transformational therapeutics.” Pipeline Updates and Anticipated Upcoming Milestones Initial ...