Clinical Read2026-06-30

AX-0810

ProQR Therapeutics N.V.PRQR
Indication
Healthy volunteers, deficiency of sodium taurocholate cotransporting polypeptide (NTCP), cholestatic diseases
Stage
phase1
Event
Interim Data
Details

May 12, 2026: ProQR Therapeutics N.V. (NASDAQ: PRQR) reported Q1 2026 results and provided R&D/catalyst updates for its Axiomer RNA editing pipeline. Lead program AX-0810 (NTCP) remains on track to report Phase 1 target engagement data in healthy volunteers in Q2 2026. ProQR selected biliary atresia as the initial Phase 2 indication and plans an investigator-initiated trial (IIT) in pediatric biliary atresia patients in China (instead of a previously planned adult PSC cohort within Phase 1); initial clinical data are targeted for H1 2027, while PSC remains a potential future indication.

Pipeline catalysts:

  • AX-0811 (next-generation NTCP; cholestatic diseases): CTA filing expected mid-2026; initial healthy-volunteer clinical data anticipated by year-end 2026.
  • AX-0422 (IDUA; Hurler syndrome): CTA filing expected early 2027; initial patient data targeted for H1 2027.
  • AX-2911 (PNPLA3; MASH): first-in-human IIT in China anticipated H1 2027.

Other events: multiple May conference presentations (TIDES USA, ASGCT, RNA Society, EASL); strategic partnership with Ginkgo Bioworks for high-throughput data generation; AI Advisory Board formed; AGM scheduled June 2, 2026 (board nomination). Cash and equivalents were €81.1M at March 31, 2026 (runway into mid-2027).