ProQR Therapeutics Reports Positive Phase 1 Data for AX-0810

ProQR Therapeutics announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810, the company's first investigational Axiomer RNA editing oligonucleotide, or EON, in healthy volunteers. Axiomer is the company's proprietary RNA editing technology platform. AX-0810 is an Axiomer RNA EON which 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. 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 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 has been 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 the advancement of next-generation candidate AX-0811 and future clinical studies in biliary atresia. ProQR expects to submit a Clinical Trial Application in mid-2026 for AX-0811 and anticipates initial human clinical data in healthy volunteers by year-end 2026. The company has selected biliary atresia as the initial indication for Phase 2 development of AX-0810 or AX-0811. The company expects to report initial clinical data from the trial in the first half of 2027.