Design Therapeutics reported new biomarker and clinical data from its Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with Friedreich ataxia.
Sixteen patients completed four weeks of weekly intravenous DT-216P2 treatment. In the 1 mg/kg dose cohort, patients showed mean improvements of 6.4 points on the modified Friedreich's Ataxia Rating Scale.
DT-216P2 was well-tolerated with no serious adverse events. The company plans to provide an update on its development plans in the fourth quarter of 2026.












