PRQR · ProQR Therapeutics N.V.

ProQR Therapeutics detailed significant advancements in its Axiomer RNA editing platform during its Investor & Analyst Event, emphasizing the lead program AX-0810 targeting NTCP for cholestatic diseases. The ongoing Phase 1 trial in healthy volunteers shows no safety signals to date, with pharmacokinetics aligning with preclinical models, and target engagement data measured by bile acid biomarkers on track for H1 2026 readout. Biliary atresia was selected as the initial Phase 2 indication due to its severe pediatric profile, lack of approved therapies, strong biological rationale via NTCP modulation to reduce bile acid toxicity, and favorable regulatory path with orphan potential. Pipeline expansion includes AX-0811, an AI-optimized next-generation candidate with ~3x higher editing efficiency than AX-0810, advancing to CTA filing mid-2026 and Phase 1 data by year-end 2026. AX-0422 addresses Hurler syndrome by editing the IDUA W402X mutation, with CTA early 2027 and initial liver data H1 2027, potentially expanding to CNS. AX-2911 targets PNPLA3 for MASH with FIH in H1 2027. AI-enabled discovery, trained on proprietary data and partnered with Ginkgo Bioworks, accelerates EON design by ~90%, enabling rapid iteration and better performance. These catalysts position ProQR for clinical validation across three programs within its cash runway to mid-2027, building a leading NTCP franchise and addressing high unmet needs in liver and rare diseases.