Business
Design Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Recent Business Updates
Trials for DT-216P2 (RESTORE-FA) and DT-168 (FECD) Ongoing; DT-818 (DM1) Dosing in Patients Expected in the First Half of 2026 Cash and Securities of $219.8
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Trials for DT-216P2 (RESTORE-FA) and DT-168 (FECD) Ongoing; DT-818 (DM1) Dosing in Patients Expected in the First Half of 2026 Cash and Securities of $219.8 Million as of Year-End Supports Ongoing Clinical Execution CARLSBAD, Calif., March 09, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported fourth quarter and full year 2025 financial results and highlighted business updates and upcoming milestones across its GeneTAC® portfolio. “The fourth quarter capped a year of strong execution and meaningful progress across our GeneTAC® portfolio,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “We enter 2026 with three clinical programs advancing toward important milestones, including the planned initiation of patient dosing in our Phase 1 trial of DT-818 in DM1 in the first half of the year and anticipated data from DT-216P2 in FA and DT-168 in FECD in the second half of 2026. Together, these anticipated milestones position us for continued execution of our strategy to build a diversified set of clinical proof-of-concept opportunities for genetic diseases with significant unmet needs.” Corporate Highlights Friedreich Ataxia (FA): Design continues to dose FA patients in its RESTORE-FA Phase 1/2 MAD trial and anticipates providing an update on the effect of DT-216P2 on endogenous frataxin levels following 12 weeks of dosing in the second half of 2026. Fuchs Endothelial Corneal Dystrophy (FECD): A Phase 2 biomarker trial of DT-168 is ongoing to evaluate safety, tolerability and corneal endothelium biomarkers in FECD patients who are scheduled for corneal transplant surgery, with data anticipated in the second half of 2026. Myotonic Dystrophy Type-1 (DM1): Design expects to begin dosing DM1 patients in its Phase 1 multiple-ascending dose (MAD) trial of DT-818, a GeneTAC® small molecule designed to selectively reduce transcription of the mutant DMPK allele, in the first half of 2026. The study, with results anticipated in 2027, is expected to assess safety and correction of mis-splicing.In preclinical studies, DT-818 demonstrated a potential best-in-disease profile for investigational therapies targeting mutant DMPK. Pipeline: Design continues to advance preclinical c...