Update from Design Therapeutics
In a press release and investor call today, May 18, 2026, Design Therapeutics announced that four-week IV data from the RESTORE-FA Trial of DT-216P2 demonstrated clinical improvements and comprehensive biomarker activity in Friedreich Ataxia.
RESTORE-FA is a Phase 1/2 clinical trial evaluating DT-216P2 in patients with FA, designed to assess safety, pharmacokinetics, pharmacodynamics, and exploratory clinical endpoints.
Design reported that:
- -16 patients had completed treatment with weekly intravenous DT-216P2 across dose cohorts.
- -DT-216P2 demonstrated dose-dependent improvement in multiple clinical measures and increases in endogenous frataxin mRNA and protein after four weeks of intravenous dosing.
- -DT-216P2 was generally well-tolerated.
- Design intends to pursue a registrational path and provide an update on its plans in the fourth quarter of 2026
