Clinical TrialJun 25, 2026, 06:26 AM
ProQR AX-0810 Phase 1 Data Shows 8-Fold Bile Acid Change; Favorable Safety
AI Summary
ProQR Therapeutics announced positive target engagement data from its ongoing Phase 1 study of AX-0810 in healthy volunteers. The data showed dose-dependent increases in total bile acids up to 8-fold, exceeding the company's 2-fold threshold for NTCP modulation, along with a favorable safety profile and an eight-week half-life. These findings support the advancement of the NTCP franchise, including the next-generation candidate AX-0811, which is projected to have higher editing efficiency and a longer half-life, with a CTA filing expected in mid-2026.
Key Highlights
- AX-0810 Phase 1 data showed up to 8-fold increase in total bile acids, exceeding the 2-fold target.
- AX-0810 demonstrated a favorable safety profile with no serious adverse events or pruritus.
- AX-0810 pharmacokinetic findings support an eight-week half-life.
- Next-gen AX-0811 projected to support at least 4-fold higher editing at lower doses and >3-month half-life.
- ProQR expects to file CTA for AX-0811 in mid-2026, with initial human data by year-end 2026.
- Phase 2 program for biliary atresia expected to start mid-2027, with interim data by mid-2028.
- Initial clinical data from IIT in pediatric biliary atresia in China targeted for H1 2027.
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