ProQR submits lead program AX-0810, advances AX-2402, and extends runway to mid-2027.

• ProQR Therapeutics submitted AX-0810 for cholestatic diseases • AX-2402 advancing toward clinical candidate selection • €119.8 million cash, runway into mid-2027 • Fall Analyst and Investor Event to feature AX-0810 Phase 1 trial design • Updates on liver and CNS pipeline expected • Lilly partnership potential milestones not included in runway estimate

ProQR Therapeutics (Nasdaq: PRQR) has reported its Q2 2025 financial and operational results, highlighting significant progress in its RNA editing pipeline. The company has submitted a Clinical Trial Application (CTA) for AX-0810, its lead program targeting NTCP for cholestatic diseases, with initial Phase 1 data expected in Q4 2025 [1].

The company maintains a strong financial position with €119.8 million in cash, providing a runway into mid-2027. This extended cash runway doesn't include potential milestone payments from its Eli Lilly partnership, which delivered $2.0 million (~ €1.8 million) in milestone achievements during H1 2025 [1].

ProQR's Q2 2025 results demonstrate significant clinical progress with their proprietary Axiomer RNA editing technology platform. The submission of a Clinical Trial Application (CTA) for lead program AX-0810 targeting NTCP for cholestatic diseases represents a crucial inflection point as the company transitions to a clinical-stage organization. Initial data from this first-in-human study is expected in Q4 2025, providing a near-term catalyst [1].

The company is also advancing AX-2402 targeting MECP2 for Rett Syndrome with clinical candidate selection expected in 2025. This program expands their platform into CNS disorders, potentially broadening the applicability of their RNA editing approach. Additional pipeline programs targeting MASH (AX-2911) and cardiovascular disease (AX-1412) further diversify their therapeutic reach [1].

The upcoming Analyst and Investor Event this fall will provide detailed insights into the AX-0810 Phase 1 trial design and set expectations for the 2025 data readout, serving as another potential catalyst for investor interest [1].

ProQR's financial position remains solid with €119.8 million in cash, providing runway into mid-2027. This extended cash runway doesn't include potential milestone payments from their Eli Lilly partnership, which delivered $2.0 million (~ €1.8 million) in milestone achievements during H1 2025. The partnership contains an option for Lilly to expand to 15 targets, which would trigger a substantial $50 million payment to ProQR [1].

R&D expenses increased to €23.7 million for H1 2025 (vs €16.3 million in H1 2024), reflecting intensified development activities across the pipeline. This contributed to a wider net loss of €22.3 million ( €0.21 per share) compared to €10.4 million ( €0.13 per share) in the prior year period [1].

References:
[1] https://www.stocktitan.net/news/PRQR/pro-qr-announces-second-quarter-2025-operating-and-financial-3dney9j6qc9i.html