PRQRNasdaqPharmaceutical PreparationsUS GAAP · USD

ProQR Therapeutics N.V.

ProQR Therapeutics N.V. is a clinical-stage biotechnology company focused on discovering and developing RNA-based therapeutics. The company is organized around a single reportable segment and advances drug candidates for diseases with significant unmet medical need, including cholestatic and other genetic conditions.

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Revenue15.9m USD
−15.9 %y/y
Operating profit (EBIT)-41.2m USD
−53.4 %y/y
EBIT margin-259.2%
−117.0 ppy/y
Net income-42.2m USD
−51.9 %y/y
Free cash flow-53.8m USD
−42.3 %y/y

Fiscal year 2025 · change from previous year

Development 2022–2025

-60-40-20020m USD2022: 3.6 m USD2022: EBIT -59 m USD20222023: 6.5 m USD2023: EBIT -27 m USD20232024: 19 m USD2024: EBIT -27 m USD20242025: 16 m USD2025: EBIT -41 m USD202516-41
RevenueOperating profit (EBIT)
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Business

Share of revenue
RNA therapeutics100 %

Discovery and development of RNA-based medicines aimed at disease-causing genetic pathways.

Customers

ProQR does not sell a broad commercial product portfolio; its primary counterparties are clinical trial sites, investigators, regulators, and development partners that support its drug programs. The eventual end users are patients with rare, genetic, or cholestatic diseases, while the economic value of the programs is typically realized through partnering, licensing, or future commercialization.

Clinical development ecosystem

Investigators, sites, CROs, and regulators involved in advancing clinical programs and obtaining approvals.

Pharmaceutical partners

Potential collaborators or licensees that may fund, develop, or commercialize RNA programs.

Patients with rare diseases

Patients in cholestatic and genetic disease indications targeted by the company’s pipeline.

Strategy

ProQR’s strategy is to build value through a focused RNA-therapeutics pipeline, with emphasis on cholestatic disease programs and other genetically defined opportunities. The company’s approach relies on generating human proof-of-concept data, progressing clinical milestones, and creating optionality for partnering or future commercialization.

Advance AX-0810 through early clinical developmentShort term

Human data are the main value inflection points for a clinical-stage biotech.

Focus on cholestatic disease opportunitiesMedium term

Concentrating on a defined therapeutic area can improve scientific focus and development efficiency.

Expand value beyond a single lead assetMedium term

Pipeline breadth can reduce dependence on one clinical outcome and improve partnering leverage.

Geography

ProQR is headquartered in the United States and operates as an international biotech organization, with reportable activities centered on research and clinical development. The excerpts specifically reference a European CTA approval for AX-0810, indicating that Europe is an important geography for clinical execution and regulatory interaction. As a development-stage company, geography matters mainly through trial locations, regulatory pathways, and access to specialized clinical sites rather than manufacturing footprint.

Risks

As a clinical-stage biotechnology company, ProQR faces high development risk because its value depends on successful clinical results, regulatory approvals, and eventual partnering or commercialization. The company also carries going-concern and funding risk typical of pre-revenue drug developers, while broader biotech risks include trial delays, safety findings, and uncertainty in translating early data into later-stage success.

Sorted by severity
LowMediumHighLowMediumHighImpact →Likelihood →Clinical development failure1Safety or tolerability issues2Going-concern and financing risk3Regulatory timing risk4
Impact is the risk’s severity and likelihood how probable it is over the next few years, both assessed from the company’s reports. Numbers match the list.
  1. Clinical development failureImpact: CriticalLikelihood: Medium

    Pipeline value depends on proving that RNA candidates work in humans.

    Exposure: Lead cholestatic and genetic disease programs

  2. Safety or tolerability issuesImpact: HighLikelihood: Medium

    Dose escalation and continuation depend on acceptable safety findings.

    Exposure: AX-0810 and other early-stage studies

  3. Going-concern and financing riskImpact: HighLikelihood: Medium

    Development-stage biotech companies consume cash before meaningful product revenue.

    Exposure: Corporate funding and clinical runway

  4. Regulatory timing riskImpact: MediumLikelihood: Medium

    Trial approvals and data readouts can shift based on agency review and protocol execution.

    Exposure: European CTA and clinical milestones

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Accounting

Judgemental items
Clinical trial accruals

Costs are recognized based on the status and timing of services received, which can be estimated.

Impact: Can cause quarterly expense true-ups

Going concern

Management evaluates whether existing funding supports operations for at least one year.

Impact: Important for solvency and disclosure assessment

Research and development expense

Most company spending is tied to clinical and preclinical programs and is expensed as incurred.

Impact: Main driver of reported loss and cash burn

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Last updated: 28/09/2026

ProQR Therapeutics N.V. (Nasdaq) – Pharmaceutical Preparations | Financial Analysis – Clarifo