ProQR Therapeutics (PRQR) investor relations material

Executive summary

• Achieved first clinical validation of Axiomer RNA editing platform via positive AX-0810 Phase 1 target engagement data in NTCP modulation, supporting advancement in cholestatic liver diseases.

• Multiple clinical milestones expected: AX-0811 Phase 1 data by year-end 2026, biliary atresia IIT and AX-0422 (IDUA) patient data in H1 2027, and AX-2911 (PNPLA3) clinical entry.

• Strengthened financial position with $59.2 million financing, ending Q2 2026 with €117.1 million in cash and equivalents, extending runway through mid-2028.

Financial highlights

• Cash and cash equivalents at June 30, 2026: €117.1 million, up from €92.4 million at December 31, 2025.

• Net loss for Q2 2026: €8.7 million (€0.08 per diluted share), improved from €12.2 million (€0.12 per share) in Q2 2025.

• Research and development costs for Q2 2026: €12.7 million, up from €11.4 million year-over-year.

• General and administrative costs for Q2 2026: €4.9 million, nearly flat year-over-year.

• Net cash used in operating activities for Q2 2026: €12.3 million, compared to €11.4 million in Q2 2025.

Outlook and guidance

• Full AX-0810 Phase 1 dataset to be presented later in 2026; AX-0811 initial Phase 1 data expected by year-end 2026, pending regulatory authorization.

• Biliary atresia IIT and initial data, AX-0422 first-in-patient study, and AX-2911 FIH IIT in China all targeted for H1 2027.

• Potentially registration-enabling Phase 2 NTCP program expected to start mid-2027, with interim analysis by mid-2028.

• Continued execution on Lilly collaboration with potential milestone payments.