ProQR Therapeutics (PRQR) investor relations material

Executive summary

• Lead RNA editing program AX-0810 remains on track for target engagement data in Q2 2026, with biliary atresia selected as the initial Phase 2 indication.

• Pipeline expanded with AX-0811 (NTCP) and AX-0422 (IDUA) advancing toward clinical trials, and AX-2911 (PNPLA3) targeting MASH in first-in-human studies in 2027.

• Strategic partnership with Ginkgo Bioworks and formation of an AI Advisory Board to enhance the Axiomer platform and accelerate discovery.

• Cash and cash equivalents of €81.1 million at March 31, 2026, supporting operations into mid-2027.

• Dr. Lykke Hinsch Gylvin nominated for Board of Directors, pending shareholder approval.

Financial highlights

• Cash and cash equivalents decreased to €81.1 million at March 31, 2026, from €92.4 million at December 31, 2025.

• Net cash used in operating activities was €11.1 million in Q1 2026, down from €15.8 million in Q1 2025.

• R&D costs were €11.8 million in Q1 2026, compared to €12.3 million in Q1 2025.

• General and administrative costs rose to €3.9 million in Q1 2026 from €3.2 million in Q1 2025.

• Net loss for Q1 2026 was €13.4 million (€0.13 per diluted share), compared to €10.1 million (€0.10 per diluted share) in Q1 2025.

Outlook and guidance

• AX-0810 Phase 1 target engagement data expected in Q2 2026; initial data from pediatric biliary atresia IIT in China targeted for H1 2027.

• AX-0811 CTA filing planned for mid-2026, with initial data by year-end 2026.

• AX-0422 CTA filing expected in early 2027, with initial data in H1 2027.

• AX-2911 first-in-human IIT in China anticipated in H1 2027.

• Continued execution on Lilly collaboration with potential data updates and milestone payments.