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Design Therapeutics Highlights Upcoming Milestones and Reports Second Quarter 2022 Financial Results
Initial Data from Friedreich Ataxia Phase 1 Trial of DT-216 Expected in the Fourth Quarter of 2022 Strong Financial Position with $359.4 Million in Cash and
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Initial Data from Friedreich Ataxia Phase 1 Trial of DT-216 Expected in the Fourth Quarter of 2022 Strong Financial Position with $359.4 Million in Cash and Securities to Support Multi-Year Operating Runway CARLSBAD, Calif., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported second quarter 2022 financial results. “Throughout the first half of 2022, we have successfully advanced our pipeline of novel GeneTAC™ small molecules designed to address the root cause of disease, without the need for irreversible gene therapy or gene editing. We believe this approach paves the way for disease-modifying treatments for many more patients with inherited genetic diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “Our Phase 1 clinical trial evaluating DT-216, our lead GeneTAC™ molecule, as a treatment for patients with Friedreich ataxia (FA) is progressing well and we look forward to sharing initial data in the fourth quarter of this year. In parallel, we’ve advanced our GeneTAC™ program for myotonic dystrophy type-1 (DM1), announced compelling preclinical data in Fuchs endothelial corneal dystrophy (FECD), and made meaningful strides across multiple earlier-stage research programs, all of which represent potentially transformative opportunities. With a financial runway to support our current multi-year operating plan, we continue to be well-positioned to execute our milestones.” Upcoming Pipeline Milestones Initial Data from Single-Ascending Dose Portion of Phase 1 trial for DT-216 Expected in the Fourth Quarter of 2022: DT-216, Design’s lead GeneTAC™ molecule, is designed to treat FA by specifically targeting the GAA repeat expansion mutation, the underlying cause of disease, and restore frataxin (FXN) gene expression. DT-216 is being evaluated in a Phase 1 clinical trial in adult patients with FA. The company plans to report initial data, including safety, tolerability, pharmacokinetics and FXN levels from the single-ascending dose portion of the trial in the fourth quarter of 2022. Design expects to initiate dosing of DT-216...