- ProQR Therapeutics (PRQR, Financial) submits a Clinical Trial Application for AX-0810 to the European Medicines Agency.
- The trial marks the first clinical development milestone for ProQR's RNA editing platform, Axiomer™.
- Phase 1 study to assess AX-0810's safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers.
ProQR Therapeutics (PRQR), a biotechnology firm focused on transformative RNA therapies, has announced the submission of a Clinical Trial Application (CTA) for its investigational candidate AX-0810 to the European Medicines Agency. This submission marks a significant advancement for ProQR's Axiomer™ RNA editing platform, as it progresses into clinical testing for the first time.
AX-0810 is an ADAR-mediated RNA editing oligonucleotide targeting NTCP, a liver cell protein involved in bile acid transport. The upcoming Phase 1 study aims to evaluate the safety, tolerability, pharmacokinetics, and target engagement of AX-0810 in healthy volunteers. Initial data from the study is anticipated in the fourth quarter of 2025.
The study will also explore biomarkers to assess early signals of target engagement, providing insights into whether the treatment modulates NTCP function effectively. This approach aims to reduce toxic bile acid accumulation in the liver, potentially alleviating inflammation, fibrosis, and liver failure progression associated with cholestatic diseases.
Pending regulatory clearance, the trial will take place at a single site in the Netherlands. This CTA submission represents a crucial step forward in addressing the unmet medical needs of patients suffering from cholestatic liver diseases, reinforcing ProQR’s commitment to leveraging human genetics and preclinical data to advance novel therapies.
