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Design Therapeutics Highlights Pipeline Progress and Upcoming Milestones and Reports Third Quarter 2022 Financial Results
Initial Data from Single-Ascending Dose Phase 1 Trial of DT-216 for Friedreich Ataxia Expected to be Reported in December 2022 Dosing Initiated in the
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Initial Data from Single-Ascending Dose Phase 1 Trial of DT-216 for Friedreich Ataxia Expected to be Reported in December 2022 Dosing Initiated in the Multiple-Ascending Dose Phase 1 Trial of DT-216; Trial Completion Anticipated in Mid-2023 Strong Financial Position with $344.2 Million in Cash and Securities to Support Multi-Year Operating Runway and Further Advancement of GeneTAC™ Platform CARLSBAD, Calif., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted recent progress and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported third quarter 2022 financial results. “At Design, we’re dedicated to bringing our pipeline of novel GeneTAC™ small molecules to patients suffering from devastating diseases, and are making meaningful strides toward achieving that goal,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “Our Phase 1 clinical program evaluating DT-216, our lead GeneTAC™ molecule, as a treatment for patients with Friedreich ataxia (FA) is progressing well. We have enrolled patients in the final cohort of the single-ascending dose trial, and plan to assess and report initial data next month. In addition, we recently initiated dosing in the multiple-ascending dose trial, keeping us on-track to complete Phase 1 mid next year. We’ve also continued to advance our GeneTAC™ programs for myotonic dystrophy type-1 (DM1) and Fuchs endothelial corneal dystrophy (FECD), as well as multiple earlier-stage programs, all of which represent potential disease-modifying approaches to treating patients in need. With a solid cash position, expert team and strong science behind us, I’m confident in our ability to execute our milestones.” DT-216 Progress and Upcoming Milestones Initial Data from Single-Ascending Dose Phase 1 Trial for DT-216 On-track to be Reported in December 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. Design is evaluating DT-216 in a Phase 1 single-ascending dose (SAD) clinical trial in adult patients with FA, a...