ProQR Therapeutics (PRQR) said Thursday its phase 1 clinical study of healthy volunteers evaluating its liver drug candidate AX-0810 met its primary endpoint.
The drug candidate demonstrated target engagement and achieved an up to eight-fold increase in serum bile acids, the company said.
The experimental therapy showed a favorable safety profile with no serious adverse events reported among the evaluated groups, the company said.
ProQR intends to use the study findings to progress its next-generation candidate AX-0811 and prepare for future trials in biliary atresia.
Shares of the company rose more than 5% in Thursday's premarket activity.
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