Update from Design Therapeutics
First individual with FA dosed in Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2
Design Therapeutics announced today, June 4, 2025, that the first individual with FA was dosed in Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2, in Australia. Design reported that DT-216P2 has been administered in one patient to date with no adverse events reported. DT-216P2 is a small molecule that was designed to increase expression of the frataxin gene.
Design Therapeutics also shared that they filed an Investigational New Drug (IND) application for DT-216P2 with the FDA in the U.S. to expand clinical development; however, the FDA has responded with a clinical hold notice. Further details will be provided to Design in an official letter from FDA.
FARA will remain in close contact with Design to better understand the FDA’s feedback and support efforts to resume U.S. clinical development as quickly as possible.
