ProQR announces initial safety, PK data from Phase 1 trial of AX-0810

ProQR Therapeutics (PRQR) announced encouraging initial safety and PK data from the first cohort of healthy volunteers in its ongoing Phase 1 trial of AX-0810 and provided an update on broader pipeline progress and anticipated 2026 milestones. “The initial human data from AX-0810 mark an important early milestone for ProQR, providing safety and pharmacokinetic observations in healthy volunteers,” said Cristina Lopez Lopez, MD, PhD, Chief Medical Officer of ProQR. “These data support continued dosing and position us well for the upcoming target engagement readout in the first half of 2026.” Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort. Development Candidates selected for pipeline programs AX-2402 for Rett syndrome and AX-2911 for MASH. Strategic collaboration with Eli Lilly (LLY) achieved $4.5 million in milestones in 2025, contributing to strong financial position with runway into mid-2027