ProQR Therapeutics (PRQR) investor relations material

Company overview and business model

• Clinical-stage biotechnology company focused on developing RNA editing therapies for diseases with significant unmet medical needs, leveraging the proprietary Axiomer RNA-editing platform targeting adenosine-to-inosine edits in RNA.

• Pipeline includes programs for cholestatic diseases, Hurler syndrome, metabolic dysfunction-associated steatohepatitis (MASH), and Rett syndrome, with lead candidate AX-0810 in Phase 2 for biliary atresia.

• Strategic partnerships with Lilly (expanded to 10 targets, option for 15) and Ginkgo Bioworks (AI-enabled discovery), and funding from patient organizations and government grants.

• Focus on retaining rights for select programs while seeking partnerships for broader indications and geographies.

Financial performance and metrics

• As of March 31, 2026: €81.1 million in cash and cash equivalents, €15.7 million in total debt, and €37.6 million in equity.

• Raised €570 million in gross proceeds from public offerings and private placements to date.

• Recent capital raises include $50 million from a registered direct offering and $9.2 million from a private placement to Lilly in June 2026.

Use of proceeds and capital allocation

• No proceeds will be received from the resale of shares by the selling shareholder; all proceeds go to the selling shareholder.

• Company bears registration expenses except for selling shareholder’s legal and brokerage fees.