Overview
- Design Therapeutics reported results from its Phase 1/2 RESTORE-FA study, which is testing DT-216P2 for the rare genetic disorder Friedreich’s ataxia.
- DT-216P2, a GeneTAC small molecule designed to boost activity of the frataxin gene, produced dose-based biomarker gains, including a 65% rise in FXN messenger RNA in whole blood and 22% to 27% higher frataxin protein two weeks after dosing at 1 mpk, with muscle FXN mRNA up 42%.
- Patients at the highest dose improved by 6.4 points on the modified Friedreich’s Ataxia Rating Scale after four weeks, with a 2.7-point gain in upright stability and more than five-point drops in fatigue that persisted two weeks after treatment.
- The therapy was generally well tolerated with no serious adverse events or treatment discontinuations, and all side effects were mild or moderate.
- Shares of Design Therapeutics fell about 29% to $10.22 in afternoon trading after an early surge following the data release, according to Benzinga.