AX-0811

September 09, 2026: ProQR Therapeutics N.V. (NASDAQ: PRQR) announced first dosing in a Phase 1 trial of AX-0811 in healthy volunteers. AX-0811 is an investigational RNA editing oligonucleotide designed to modulate NTCP and is being developed for cholestatic liver diseases, including biliary atresia. The Phase 1 study will assess safety, tolerability, pharmacokinetics, and pharmacodynamic biomarkers.

The company said preclinical data showed AX-0811 had greater potency and a longer half-life than AX-0810. Initial Phase 1 target engagement data from the first two cohorts are expected in early January 2027.

Separately, ProQR will host a virtual investor and analyst educational event, “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing,” on September 30, 2026 at 11 a.m. ET. The event will include ProQR management and Gideon Hirschfield of the Toronto Centre for Liver Disease, and will cover bile acids and NTCP modulation in cholestatic liver disease, review recently reported AX-0810 Phase 1 target engagement data, and discuss the rationale and development strategy for AX-0811 in biliary atresia.