ProQR Therapeutics N.V. (PRQR) Form 6-K — Jun 25, 2026
ProQR Therapeutics announced positive Phase 1 target engagement data for AX-0810, demonstrating a dose-dependent increase in total bile acids of up to 8-fold at the 6 mg/kg dose level. This result exceeded the company's predefined 2-fold threshold for meaningful NTCP modulation and confirms the biological rationale of its Axiomer RNA editing platform. The study reported a favorable safety and tolerability profile with no serious adverse events or pruritus, alongside pharmacokinetic findings supporting a half-life of eight weeks. These data support the advancement of the NTCP franchise, including the next-generation candidate AX-0811 and future studies in biliary atresia.
Key figures
- Ax0810 half life weeks
- 8
- Cohorts evaluated doses
- ["3 mg/kg","6 mg/kg"]
- Bile acid fold change max
- 8-fold
- Ntcp modulation threshold
- 2-fold
- Phase1 participants total
- 33
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