ProQR Therapeutics Announces Positive Target Engagement Data From Ongoing Phase 1 AX-0810 Study

(RTTNews) - Thursday, ProQR Therapeutics N.V. (PRQR) announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810, an investigational Axiomer RNA editing oligonucleotide which aims to reduce toxic bile acid accumulation in the liver.

The study enrolled 33 healthy volunteers, including 24 participants receiving AX-0810 and nine participants receiving placebo across the 3 mg/kg, 6 mg/kg, and 9 mg/kg cohorts.

The findings showed that administration of AX-0810 across the evaluable 3 mg/kg and 6 mg/kg cohorts resulted in dose-dependent increases in total bile acids in serum of up to 8-fold, which was higher than the company's predefined target engagement threshold of 2-fold as a meaningful indication of NTCP modulation.

Meanwhile, administration of AX-0810 resulted in increased hepatotoxic conjugated bile acids in circulation, keeping them from accumulating in the liver.

Together, the company believes the consistent dose-response observed across these independent biomarker measures provides convergent evidence of NTCP modulation in humans following administration of AX-0810.

Moreover, increased levels of conjugated bile acids in serum circulation led to dose-dependent and statistically significantly increased urinary excretion of conjugated bile acids, indicating that NTCP modulation may allow the body to lower concentration of toxic bile acids.

The company stated that the participants of the Phase 1 study of AX-0810 will continue to be followed through the planned 12-week follow-up period across all cohorts.

Further, the company plans to present full data from the Phase 1 study at a medical or scientific conference later this year.

ProQR also added that the biomarker findings established in the AX-0810 Phase 1 study support continued advancement of ProQR's NTCP franchise, including AX-0811, a next-generation NTCP-targeting RNA editing oligonucleotide.

Notably, the company intends to submit a Clinical Trial Application in mid-2026 for AX-0811 and anticipates initial human clinical data in healthy volunteers by year-end 2026.

In the pre-market hours, PRQR is trading at $1.81, up 0.18 percent on the Nasdaq.