Design Therapeutics updates RESTORE-FA trial design, reports Q2 2026 results

Design Therapeutics said on August 3, 2026 that it is modifying its ongoing RESTORE-FA trial cohorts for DT-216P2 in Friedreich ataxia following four-week data reported in May 2026. In that earlier readout, DT-216P2 was generally well-tolerated and showed dose-dependent increases in endogenous frataxin mRNA and protein levels, along with improvements across multiple clinical measures after four weeks of intravenous dosing in Friedreich ataxia patients.¹

Based on those results, the study will continue to evaluate 1 mpk as the planned go-forward dose, with plans to enroll 10 patients in the 12-week cohort.¹ The company is also designating percent change from baseline in endogenous blood FXN protein as the primary efficacy endpoint, and it plans to explore a dose level above 1 mpk.¹ On timing, Design said it expects to give an update on its registrational plans in the fourth quarter of 2026, with 12-week treatment data anticipated in the first quarter of 2027.¹

Elsewhere in the pipeline, Design has begun dosing patients with myotonic dystrophy type 1 in a Phase 1 multiple-ascending dose trial of DT-818, and expects to report data from that study in 2027.¹ In Fuchs endothelial corneal dystrophy, a Phase 2 biomarker trial of DT-168 is ongoing, but data is now expected in 2027 due to a delay in the anticipated supply of DT-168 blow-fill-seal eye droppers.¹ The company also said it continues to advance preclinical characterization of several candidate molecules for its Huntington's disease program.¹

On finances, cash, cash equivalents and investment securities totaled $207.4 million as of June 30, 2026.¹ Net loss for the quarter was $20.2 million.¹