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ProQR Therapeutics (PRQR) investor relations material
ProQR Therapeutics Study Update summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Study Update summary3 Nov, 2025
Strategic Overview and Platform Highlights
The Axiomer RNA editing platform enables precise, reversible RNA edits to modulate protein function, offering new therapeutic possibilities for genetic and acquired diseases.
The pipeline includes AX-0810 for cholestatic diseases, AX-2402 for Rett syndrome, and additional programs for cardiovascular and metabolic diseases.
Strategic partnerships, notably with Eli Lilly, provide validation and resources, while financial runway extends into mid-2027.
The company continues to invest in its patent estate and collaborative partnerships, underpinning leadership in RNA editing therapies.
Clinical Trial Authorization and Study Design
Authorization granted to begin Phase 1 clinical trial of AX-0810 in healthy volunteers in the Netherlands.
The first-in-human study will enroll 33 healthy volunteers, using a multiple ascending dose design to assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics.
Biomarker assessments, including plasma bile acids and TUDCA challenge, are standardized to robustly capture drug-induced changes and confirm NTCP specificity.
Initial safety, tolerability, and PK data are expected by Q4 2025, with target engagement data in the first half of 2026.
A virtual investor and analyst event is scheduled for November 3, 2025, to discuss study details and biomarker roadmap.
Scientific Rationale and Disease Context
AX-0810 targets NTCP to reduce toxic bile acid accumulation in the liver, addressing a core driver of cholestatic diseases such as primary sclerosing cholangitis (PSC) and biliary atresia (BA), both with high unmet need and no approved disease-modifying therapies.
NTCP modulation is supported by human genetics, preclinical, and clinical data, showing reduced bile acid uptake and improved liver biomarkers.
NTCP deficiency in humans leads to reduced bile acid uptake without liver dysfunction, supporting the safety of this mechanism.
The approach aims to reduce bile acid toxicity, inflammation, and fibrosis, potentially modifying disease progression.
NTCP vs IBAT: efficacy in severe cholestasis?
Patient cohort: rationale for PSC selection?
What plasma bile acid increase is target in HV?
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Next ProQR Therapeutics earnings date
Q3 202521 Nov, 2025
Next ProQR Therapeutics earnings date
Q3 202521 Nov, 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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