Design Therapeutics Phase 1/2 trial shows 65% frataxin mRNA rise, 6.4-point mFARS gain
Design Therapeutics reported four-week IV Phase 1/2 RESTORE-FA data: dose-dependent increases in frataxin mRNA (65% in whole blood) and protein (22–27%) at 1 mpk dose alongside mean clinical improvements of 6.4 mFARS and 2.7 upright stability score. DT-216P2 was generally well-tolerated with no serious adverse events, supporting a registrational development path.
1. RESTORE-FA Trial Design and Cohorts
The Phase 1/2 RESTORE-FA trial evaluated weekly intravenous DT-216P2 in 16 Friedreich ataxia patients across four dose cohorts (0.1, 0.3, 0.6 and 1 mpk) over four weeks. Each cohort included four patients, and assessments covered safety, pharmacokinetics, pharmacodynamics and exploratory clinical endpoints.
2. Biomarker and Clinical Outcomes
Following four weeks at the 1 mpk dose, whole blood FXN mRNA increased by 65% and FXN protein by 22–27%, while muscle FXN mRNA rose by 42%. Clinically, patients showed mean improvements of 6.4 points on mFARS, 2.7 points on upright stability and over five points on the PROMIS fatigue scale.
3. Safety and Path Forward
DT-216P2 was generally well-tolerated with no serious adverse events or discontinuations; mild to moderate transient ALT elevations occurred in three patients without bilirubin changes. Based on these results, the company plans to advance DT-216P2 toward registrational development with an update expected in Q4 2026.