ProQR details pipeline expansion with multiple clinical catalysts

ProQR Therapeutics (NASDAQ: PRQR) outlined pipeline developments and upcoming clinical milestones during a virtual investor event. The RNA therapy company announced target engagement data for lead program AX-0810 remains on track for the first half of 2026, with biliary atresia selected as the initial Phase 2 indication.

The company expanded its pipeline with two new programs. AX-0811, a next-generation NTCP program for cholestatic diseases, expects a Clinical Trial Application filing in mid-2026 with initial clinical data anticipated by year-end 2026. AX-0422, targeting IDUA for Hurler syndrome, plans CTA filing in early 2027 with initial clinical data expected in the first half of 2027.

ProQR also announced AX-2911, targeting PNPLA3 for metabolic dysfunction-associated steatohepatitis, with plans for a first-in-human investigator-initiated trial in China during the first half of 2027.

"We look forward to the target engagement data with AX-0810 later this quarter and are excited about additional programs approaching the clinic," said Daniel A. de Boer, founder and chief executive officer.

The company continues developing its Axiomer RNA editing technology platform through artificial intelligence-enabled discovery and high-throughput screening, supported by a partnership with Ginkgo Bioworks.

ProQR expects to deliver multiple clinical data readouts across its pipeline within its current cash runway, which extends into mid-2027. The information was provided during the company's investor and analyst event.

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