Design Therapeutics, Inc.
For the period ended Aug 3, 2026. Filed Aug 3, 2026.
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Aug 3, 2026 · Filed Aug 3, 2026
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EX-99.1 · RESTORE-FA Progress
RESTORE-FA Progress
Positive RESTORE-FA Four-Week Data Support Advancement of DT-216P2. As reported in May 2026, DT-216P2 was generally well-tolerated and demonstrated dose-dependent increases in endogenous frataxin mRNA and protein levels, together with improvements across multiple clinical measures following four weeks of intravenous dosing in patients with Friedreich ataxia.
Modifications to RESTORE-FA. Based on the four-week data, Design is modifying the ongoing cohorts in the RESTORE-FA trial to support the next stage of clinical development. The study will continue to evaluate 1 mpk as the planned go-forward dose, with the intention of enrolling 10 patients in the 12-week cohort. In addition, modifications include specifying endogenous blood FXN protein percent change from baseline as the primary efficacy endpoint and exploring a dose level above 1 mpk.
Next Steps and Expected Milestones: Design expects to provide an update on its registrational plans in the fourth quarter of 2026, with data following 12 weeks of treatment expected in the first quarter of 2027.
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