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ProQR Therapeutics (PRQR) investor relations material
ProQR Therapeutics Q2 2025 earnings summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Q2 2025 earnings summary7 Aug, 2025
Executive summary
Submitted Clinical Trial Application for AX-0810, targeting NTCP in cholestatic diseases, with first-in-human Phase 1 trial expected to start in the Netherlands and initial data anticipated in Q4 2025.
Advancing AX-2402 program for Rett Syndrome toward clinical candidate selection in 2025; additional pipeline programs progressing, including AX-2911 for MASH and AX-1412 for cardiovascular disease.
Hosted and participated in key scientific conferences, highlighting advances in Axiomer RNA editing platform, and announced new CFO and CMO appointments to support clinical stage transition.
Financial highlights
Cash and cash equivalents stood at €119.8 million as of June 30, 2025, down from €149.4 million at December 31, 2024.
Net cash used in operating activities was €27.2 million for the first half of 2025, compared to €21.4 million in the same period last year.
R&D costs increased to €23.7 million for the six months ended June 30, 2025, from €16.3 million year-over-year; G&A costs rose to €8.1 million from €6.5 million.
Net loss for the six-month period was €22.3 million (€0.21 per diluted share), compared to €10.4 million (€0.13 per diluted share) in the prior year.
Achieved $2.0 million (~€1.8 million) in milestone payments from Eli Lilly collaboration in H1 2025.
Outlook and guidance
Initial Phase 1 data for AX-0810 expected in Q4 2025; clinical candidate selection for AX-2402 and AX-2911 anticipated in 2025.
Virtual Analyst and Investor Event planned for fall 2025 to provide updates on AX-0810 trial design, data expectations, and broader pipeline progress.
Cash runway projected into mid-2027, excluding potential additional milestones from the Lilly partnership.
Explain the increase in operating cash burn.
Lilly partnership: Status of additional target option?
AX-0810: Outline registrational pathway strategy.
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H.C. Wainwright Liver Disease Virtual Conference21 Oct, 2025
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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.
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