Design Therapeutics has reported data from the ongoing Phase I/II RESTORE-FA clinical trial assessing DT-216P2 in Friedreich ataxia (FA) patients.
The results show clinical improvements and increased biomarker activity following four weeks of intravenous treatment.
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DT-216P2 is a GeneTAC small-molecule therapy intended to boost frataxin (FXN) expression by targeting the guanine-adenine-adenine (GAA) repeat expansion in the FXN gene, the genetic cause of FA.
The trial is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and exploratory clinical outcomes of the therapy.
So far, 16 patients have completed four weeks of weekly intravenous treatment, with participants evenly divided among four dose groups (0.1mg, 0.3mg, 0.6mg, and 1mg per kilogram).
Clinical findings indicate patients receiving the 1mg per kg dose had a mean improvement of 6.4 points in the modified Friedreich’s Ataxia Rating Scale (mFARS) and 2.7 points in the Upright Stability Score.
Patient-reported fatigue, measured by the PROMIS Fatigue Scale, improved by more than five points, exceeding the threshold for minimal important change.
Biomarker analysis showed dose-dependent increases in FXN messenger ribonucleic acid (mRNA) and protein in whole blood and muscle.
The 1mg per kilogram group experienced a 65% rise in whole blood FXN mRNA and 42% in muscle FXN mRNA from baseline. Protein increases were 22%–27% from baseline.
DT-216P2 was generally well-tolerated. No serious adverse events or discontinuations were observed. Mild to moderate, transient alanine transaminase elevations occurred in three patients, all asymptomatic.
Design Therapeutics aims to pursue a registrational pathway for DT-216P2 with further updates expected in the fourth quarter of 2026.
Design Therapeutics CEO Pratik Shah said: “These data represent an advancement for FA treatment, demonstrating that DT-216P2 increased endogenous frataxin and translated biomarker observations into clinical improvements after only four weeks of treatment.
“We 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. Based on these findings, we believe DT-216P2 has the potential to be a best-in-disease treatment for patients with FA and look forward to advancing the programme toward registrational development.”
In May 2025, Design Therapeutics reported positive outcomes from the double-masked, randomised Phase I trial of its GeneTAC small molecule, DT-168, in healthy volunteers.
