Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in Friedreich Ataxia
Design Therapeutics reported positive Phase 1/2 data for DT-216P2 in Friedreich Ataxia, showing dose-dependent clinical improvements and biomarker activity after four weeks of IV dosing. At the 1 mpk dose, patients saw a 6.4-point improvement in mFARS and a 2.7-point improvement in Upright Stability Score, alongside >5-point reductions in fatigue. Biomarker data confirmed mechanistic activity, with whole blood FXN mRNA rising 65% and muscle FXN mRNA rising 42% from baseline.
Key figures
- Drug Name
- DT-216P2
- Phase Of Trial
- Phase 1/2
- Patients completed
- 16
- Fatigue improvement
- >5 points
- M FARS improvement 1mpk
- 6.4
- Fxn mrna increase blood
- 65%
- Fxn mrna increase muscle
- 42%
- Fxn protein increase blood
- 22-27%
- Upright stability improvement 1mpk
- 2.7
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