Design Therapeutics announced positive Phase 1 trial data for DT-168, an eye drop formulated to treat Fuchs endothelial corneal dystrophy (FECD) by targeting the mutant TCF4 gene. The trial in healthy volunteers showed the drug was well-tolerated with no serious or ocular adverse events. A Phase 2 biomarker trial in FECD patients is planned for the second half of 2025.
This development is potentially groundbreaking for millions of FECD patients currently without disease-modifying treatment options. The positive safety profile and convenient eye drop formulation of DT-168 position it as a promising alternative to corneal transplant, the current standard of care for advanced FECD. Furthermore, the identification of corneal endothelium RNA biomarkers could revolutionize clinical trials for FECD by providing a quantifiable measure of drug efficacy.
The Phase 1 trial involved 24 healthy volunteers receiving either placebo or escalating doses of DT-168 twice daily for seven days. No drug-related adverse events were reported, and systemic drug exposure remained below quantifiable levels. Concurrent reference range studies confirmed distinct splicing differences in corneal endothelium between healthy individuals and FECD patients, validating the use of RNA biomarkers in the upcoming Phase 2 trial. This trial will enroll FECD patients awaiting corneal transplants, administering DT-168 for approximately four weeks prior to surgery. Post-surgery tissue analysis will assess the impact of DT-168 on corneal endothelium RNA biomarkers.
The positive Phase 1 data and upcoming Phase 2 trial represent significant milestones in the development of a much-needed FECD treatment. If the Phase 2 trial confirms the drug’s efficacy in modifying disease biomarkers, DT-168 could become the first disease-modifying therapy for FECD, transforming the treatment landscape and significantly improving the quality of life for patients suffering from this debilitating disease.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
