Design Therapeutics Inc., a clinical-stage biotechnology firm focused on treating degenerative genetic diseases, announced the beginning of patient dosing in its RESTORE-FA Phase 1/2 multiple-ascending dose trial of DT-216P2 for Friedreich ataxia outside the United States. Initial data from a Phase 1 single-ascending dose trial in healthy volunteers indicated favorable safety and pharmacokinetics for DT-216P2. Design Therapeutics has submitted an investigational new drug $(IND.AU)$ application to the U.S. FDA to expand the trial to U.S. sites. However, the FDA issued a clinical hold on the IND application, citing nonclinical deficiencies. The company intends to address these issues promptly to continue their development efforts in the U.S. The trial is currently open for enrollment in Australia, with data anticipated in 2026.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.