ProQR Therapeutics (PRQR) Conference presentation summary

Clinical pipeline advancement

• Multiple RNA editing candidates in development, including AX-0810 and AX-0811 for cholestatic diseases, AX-0422 for Hurler syndrome, and AX-2911 for MASH, with additional partnered programs and a €81.1M cash runway into mid-2027.

• AX-0810 Phase 1 study in healthy volunteers showed favorable safety, no serious adverse events, and dose-dependent target engagement across three biomarkers, confirming NTCP modulation without affecting hormone or drug transport.

• AX-0811 demonstrated 4-fold greater potency than AX-0810 in preclinical models, achieving up to 65% editing in human PK projections and significant reduction in cholestasis in animal studies.

• Both AX-0810 and AX-0811 are designed to selectively modulate NTCP's bile acid transport function, preserving hormone and drug transport, supporting safer long-term therapy.

• Phase 2 trials in biliary atresia are planned for mid-2027, with initial data from investigator-initiated trials in pediatric patients expected in H1 2027.

Key clinical and preclinical results

• AX-0810 Phase 1 showed up to 8-fold increase in serum bile acids, confirming target engagement and NTCP specificity, with hepatotoxic bile acids redirected to urine.

• No clinically significant changes in liver enzymes, bilirubin, or hormone levels were observed, and pharmacokinetics matched expectations with an 8-week half-life.

• AX-0811 preclinical data showed robust biomarker response, 60% editing in vivo, and 67% reduction in cholestasis in animal models, with a projected 3+ month half-life in humans.

• Both candidates utilize GalNAc conjugation for targeted delivery and are administered subcutaneously.

Upcoming milestones and strategic priorities

• AX-0810 Phase 1 Cohort 3 and 12-week follow-up data expected by year-end 2026; AX-0811 initial Phase 1 data in healthy volunteers also expected by year-end 2026.

• Investigator-initiated trial in pediatric biliary atresia to report initial data in H1 2027; Phase 2 trial in biliary atresia to start mid-2027 with interim analysis by mid-2028.

• Additional clinical updates anticipated for AX-0422 in Hurler syndrome and AX-2911 in MASH in H1 2027.

• Ongoing partnerships with Eli Lilly and Ginkgo Bioworks to advance the Axiomer platform and expand the pipeline.