AX-0810

May 12, 2026 — ProQR Therapeutics N.V. (Nasdaq: PRQR) reported Q1 2026 operating and financial results and provided a pipeline update for its Axiomer™ RNA editing platform. The company remains on track to report Phase 1 target engagement data for AX-0810 (NTCP) in healthy volunteers in Q2 2026 and selected biliary atresia as the initial Phase 2 indication. ProQR plans an investigator-initiated trial in pediatric biliary atresia patients in China (instead of a previously planned adult PSC cohort in Phase 1), targeting initial clinical data in H1 2027.

Pipeline milestones include: AX-0811 (next-gen NTCP) CTA filing expected mid-2026 with initial healthy-volunteer data by year-end 2026; AX-0422 (IDUA, Hurler syndrome) CTA expected early 2027 with initial patient data targeted H1 2027; and AX-2911 (PNPLA3, MASH) first-in-human IIT in China planned for H1 2027. ProQR also announced a partnership with Ginkgo Bioworks to enable high-throughput data generation and formed an AI Advisory Board.

Financially, ProQR ended Q1 2026 with €81.1M cash (down from €92.4M at 2025 year-end), net cash used in operations of €11.1M, and a net loss of €13.4M (€0.13/share), supporting a stated runway into mid-2027.