ProQR Therapeutics
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ProQR Therapeutics (PRQR) investor relations material

ProQR Therapeutics Q3 2026 earnings summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Q3 2026 earnings summary12 Nov, 2026

Executive summary

  • AX-0811 Phase 1 Cohorts 1 and 2 fully enrolled; initial target-engagement data expected in early Jan 2027, following positive AX-0810 Phase 1 target-engagement data reported in June 2026.

  • €103.0m cash and cash equivalents at Sep. 30, 2026, with runway expected into mid-2028.

  • Multiple pipeline milestones expected in H1 2027, including pediatric biliary atresia, AX-0422 in MPS I Hurler syndrome and AX-2911 for MASH.

Financial highlights

  • Q3 revenue €5.155m vs €2.882m YoY; 9M revenue €15.947m vs €11.218m.

  • Q3 R&D costs €14.323m vs €11.080m YoY; G&A costs €3.967m vs €3.119m.

  • Q3 net loss €12.447m vs €11.006m; 9M net loss €34.558m vs €33.264m.

  • Q3 net cash used in operating activities €13.586m vs €12.235m YoY; 9M cash used €37.001m vs €39.434m.

Outlook and guidance

  • Full AX-0810 Phase 1 dataset, including Cohort 3 and 12-week follow-up, and new AX-0811 preclinical data expected in Dec. 2026.

  • AX-0811 initial target-engagement data from healthy-volunteer Cohorts 1 and 2 expected in early Jan. 2027.

  • Pediatric biliary atresia IIT in China targeted for initiation and initial data in H1 2027; a potentially registration-enabling Phase 2 program is expected to start mid-2027, subject to regulatory interactions, with interim data by mid-2028.

  • AX-0422 CTA filing for a first-in-patient MPS I Hurler study anticipated in early 2027, with initial data in H1; AX-2911 MASH IIT in China expected in H1 2027.

  • Lilly collaboration continues; potential data updates and milestone payments anticipated.

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Frequently asked questions

ProQR Therapeutics N.V. is a biotechnology company focused on developing innovative RNA therapies for genetic diseases. Established in 2012 in Leiden, Netherlands, ProQR pioneers RNA editing technologies, specifically its proprietary platform called Axiomer. This technology enables precise RNA editing by using a cell's own ADAR enzymes to alter specific nucleotides in RNA molecules, potentially reversing mutations or modulating protein expression to address unmet medical needs in various diseases. The company's approach to drug development is particularly significant for conditions that currently lack effective treatments. Over the years, ProQR has expanded its pipeline beyond its initial focus on cystic fibrosis to include a range of rare and common diseases, with ongoing research and development efforts in fields such as inherited retinal diseases and liver diseases. The company is headquartered in Leiden, Netherlands, and its shares are listed on the NASDAQ.