ProQR Reports Positive Initial Safety Data from AX-0810 Phase 1 Trial

ProQR Therapeutics NV announced encouraging initial safety and pharmacokinetic $(PK)$ data from the first cohort of healthy volunteers in its ongoing Phase 1 trial of AX-0810. The initial results, which showed no safety signals after four weeks of dosing and PK consistent with non-clinical data, support continued dosing in the study. Enrollment and dosing of healthy volunteers are ongoing, with target engagement data expected in the first half of 2026. Additionally, ProQR reported new non-clinical functional proof-of-concept data for AX-2911, which demonstrated over 80% reduction in hepatic fat content in a humanized PNPLA3-148M mouse model, outperforming a clinical-stage PNPLA3-directed antisense therapy. Further details and analyses on AX-2911 will be presented at a later date.

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