Clinical TrialJun 25, 2026, 06:41 AM
ProQR AX-0810 Phase 1 Data Positive, Validates Axiomer RNA Platform
AI Summary
ProQR Therapeutics N.V. announced positive Phase 1 target engagement data for AX-0810 in healthy volunteers, validating its Axiomer RNA editing platform. The study showed dose-dependent increases in total bile acids up to 8-fold, along with a favorable safety profile and sustained target engagement. These results support the advancement of the NTCP franchise, including the next-generation candidate AX-0811, and plans for Phase 2 development in biliary atresia.
Key Highlights
- AX-0810 Phase 1 data showed dose-dependent target engagement in 3 mg/kg and 6 mg/kg cohorts.
- AX-0810 demonstrated up to 8-fold change in total bile acids in serum, exceeding the 2-fold threshold.
- Favorable safety and tolerability profile observed with no serious adverse events or pruritus.
- Pharmacokinetic findings support sustained target engagement, with a half-life of eight weeks.
- Next-generation candidate AX-0811 projected to have 4-fold higher editing and half-life over three months.
- ProQR expects to submit a CTA for AX-0811 in mid-2026, with initial human data by year-end 2026.
- Initial clinical data from an investigator-initiated trial (IIT) for biliary atresia expected H1 2027.
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