Design Therapeutics reports positive Phase 1/2 data for DT-216P2 in Friedreich ataxia

Design Therapeutics, Inc.

After 4 weeks at 1 mpk, mFARS improved 6.4 points, Upright Stability Score 2.7 points, fatigue >5 points (PROMIS).
Whole blood FXN mRNA increased 65% (p<0.001); muscle FXN mRNA up 42% (p=0.015); FXN protein increased 22-27%.
DT-216P2 well-tolerated; no serious adverse events or discontinuations; mild-moderate transient ALT elevations in 3 patients.
Company plans to pursue registrational path; update expected in Q4 2026.

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