Data from ongoing Phase 1 study of AX-0810 in healthy volunteers demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohorts
AX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the 2-fold threshold identified by the Company as a meaningful indicator of NTCP modulation, along with concordant readouts of bile acid profile and TUDCA markers
Favorable safety and tolerability profile observed with AX-0810 to date, with no serious adverse events or pruritus reported; pharmacokinetic findings to date support sustained target engagement, including a half-life of eight weeks
The NTCP biomarker findings support advancement of next-generation candidate AX-0811 and future clinical studies in biliary atresia
LEIDEN, Netherlands & CAMBRIDGE, Mass., June 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR or the Company), a clinical-stage biotechnology company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810, the Company's first investigational Axiomer RNA editing oligonucleotide (EON), in healthy volunteers.
AX-0810 is a GalNAc-conjugated, subcutaneously administered Axiomer RNA EON designed to selectively modulate NTCP, a key transporter responsible for bile acid uptake from the bloodstream into the liver. Through this novel mechanism, AX-0810 aims to reduce toxic bile acid accumulation in the liver, the underlying driver of cholestatic liver diseases including biliary atresia, and has the potential to be disease modifying.
AX-0810 Phase 1 Study Overview
The ongoing multiple ascending dose (MAD) Phase 1 study enrolled 33 healthy volunteers, including 24 participants receiving AX-0810 and nine participants receiving placebo across the 3 mg/kg, 6 mg/kg, and 9 mg/kg cohorts. The data reported today include findings from 22 participants in cohorts 1 and 2, receiving 3 mg/kg and 6 mg/kg doses, respectively. Data from the 9 mg/kg cohort are not yet available.
NTCP Target Engagement Confirmed Across All Three Predefined Biomarker Measures
The data demonstrated clear evidence of NTCP target engagement consistent with the biological rationale and preclinical findings. Across the evaluable 3 mg/kg and 6 mg/kg cohorts, administration of AX-0810 resulted in dose-dependent increases in total bile acids in serum of up to 8-fold, exceeding the Company's predefined target engagement threshold of 2-fold as a meaningful indication of NTCP modulation. Concordant dose-dependent changes in conjugated bile acids and circulating TUDCA levels further support specificity of NTCP modulation.