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ProQR Therapeutics (PRQR) investor relations material
ProQR Therapeutics Evercore ISI 8th Annual HealthCONx Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Evercore ISI 8th Annual HealthCONx Conference summary4 Dec, 2025
Company overview and technology
Focuses on RNA editing medicines to treat genetic and common diseases by modifying messenger RNA, enabling the creation or correction of proteins for health benefits.
Holds foundational IP for its RNA editing technology, developed over the past decade.
Entered a $4 billion partnership with Eli Lilly in 2021, covering up to 15 targets, with $125 million upfront and potential for $250 million per target in milestones plus royalties.
Maintains a wholly owned pipeline targeting liver and CNS diseases, including rare and common indications.
Lead program: AX-0810 for cholestatic diseases
AX-0810 targets NTCP to modulate bile acid uptake, aiming to treat PSC and BA, both with high unmet medical need.
Initial trial in healthy volunteers (33 subjects, 3 cohorts) is randomized, placebo-controlled, and double-blinded, with dosing over five weeks and 12-week follow-up.
Primary endpoints include serum bile acid concentration, bile acid ratios, and response to TUDCA challenge.
Initial safety and PK data expected by year-end; full PD and biomarker data in the first half of next year.
Phase 1B in PSC patients planned for the second half of next year, with results before year-end.
Clinical development strategy and regulatory pathway
Indication selection between PSC and BA for phase 2 will be finalized alongside phase 1 results, considering recruitment feasibility and disease characteristics.
Accelerated approval is possible due to high unmet need and lack of disease-modifying therapies; biomarker and imaging data may support this path.
Success in the healthy volunteer study defined as at least a twofold increase in serum bile acid, favorable bile acid ratio shift, and improved TUDCA clearance.
Editing percentage correlates linearly with bile acid increase; 5% editing yields twofold, 10% yields fourfold increase.
No safety concerns anticipated from increased editing or bile acid levels, supported by human genetics and prior clinical data.
AX-0810 accelerated approval path
Rett program vs gene therapy value proposition
Eli Lilly partnership development progress
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Next ProQR Therapeutics earnings date
Q4 202513 Mar, 2026
Next ProQR Therapeutics earnings date
Q4 202513 Mar, 2026
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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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