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Design Therapeutics Highlights Pipeline Progress and Reports Fourth Quarter and Full Year 2022 Financial Results
Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Ongoing with Data Expected Mid-Year Continued Progress Across Pipeline of GeneTAC™ Small
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Ongoing with Data Expected Mid-Year Continued Progress Across Pipeline of GeneTAC™ Small Molecules with Three Programs Expected to be in Clinical Development in the Next Two Years Strong Financial Position with $330 Million in Cash and Securities Expected to Support Operating Runway through 2025 CARLSBAD, Calif., March 14, 2023 (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 fourth quarter and full year 2022 financial results. “We are very proud of the important progress made last year with our novel GeneTAC™ small molecules, which represent a potential new class of therapies for patients suffering from devastating genetic diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “The data from the Phase 1 single-ascending dose clinical trial of DT-216 in patients with Friedreich ataxia (FA) showed, for the first time, that our GeneTAC™ small molecule DT-216 could be dosed safely and is capable of overcoming the transcription block for frataxin in individuals with FA. This underscores the potential for DT-216 to address the root cause of the disease. We’re underway with our Phase 1 multiple-ascending dose trial and look forward to evaluating the effects of three doses of DT-216 in people with FA.” Dr. Siffert continued, “Beyond FA, we further advanced and expanded our earlier-stage pipeline, selecting DT-168 as the development candidate for our second program in FECD and progressing our DM1 program through lead optimization and advancing our discovery portfolio, with the aim of bringing two additional programs into the clinic in the next two years. With a solid cash position to support operations through a steady cadence of milestones ahead, an expert team, and diligent execution, we are well-positioned to deliver on our near- and long-term goals.” Pipeline Progress and Anticipated Upcoming Milestones Data from Phase 1 Multiple-Ascending Dose (MAD) Trial of DT-216 for Friedreich Ataxia (FA) On-track for Mid-2023: Design is evaluating its lead ...