Monday, Design Therapeutics, Inc. (DSGN) announced positive biomarker and clinical data from the ongoing Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with Friedreich ataxia.
The study observed both dose-dependent increases in FXN levels and dose-dependent improvements across multiple clinical measures, including mFARS, upright stability score and patient-reported fatigue.
After 4 weeks, patients treated with DT-216P2 demonstrated mean improvements from baseline of 6.4 points in the modified Friedreich's Ataxia Rating Scale and 2.7 points in the Upright Stability Score.
Based on these data, the company intends to pursue a registrational path and provide an update on its plans in the fourth quarter of 2026.
In the pre-market hours, DSGN is trading at $15.70, up 8.99 percent on the Nasdaq.
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Business News
May 15, 2026 15:25 ET Apart from the confirmation of Kevin Warsh as the next Fed chair, the main news on the economics front this week included key price data from the U.S. and the first quarter economic growth figures from major economies. Both consumer prices and producer costs have started to reflect the effect of supply shocks due to the Middle East conflict. In Europe, GDP data was in focus, while inflation data from China dominated the news flow in Asia.