Design Therapeutics reports early Friedreich ataxia data for DT-216P2 at 1 mpk dose
Design Therapeutics reported biomarker and clinical data from the ongoing Phase 1/2 RESTORE-FA trial of DT-216P2 in Friedreich ataxia patients, including mean improvements at the 1 mpk dose on two rating scales and increases in frataxin levels. The company said the data are preliminary and that it intends to pursue a registrational path, with an update on its plans expected in the fourth quarter of 2026.
Key points
- As of May 17, 2026, 16 patients had completed four weeks of weekly IV DT-216P2 across 0.1, 0.3, 0.6 and 1 mpk cohorts (n=4 per cohort).
- At 1 mpk, mean improvements from baseline were 6.4 points on the modified Friedreich's Ataxia Rating Scale and 2.7 points on the Upright Stability Score.
- Whole blood FXN mRNA increased 65% from baseline after four weeks at 1 mpk (p < 0.001).
- No serious adverse events or treatment discontinuations were reported; three patients had mild to moderate, asymptomatic ALT elevations.
The filing
Written by AI on Nov 9, 2026, 11:43 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on May 18, 2026.
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