ProQR Therapeutics Reports Q2 2025 Results, Submits CTA for AX-0810, and Receives $2M Milestone Payment from Lilly Collaboration.

ProQR Therapeutics reported a strong cash position of €119.8 million, extending its financial runway into mid-2027. The company submitted a Clinical Trial Application for its lead program AX-0810 targeting NTCP for cholestatic diseases, with initial Phase 1 data expected in Q4 2025. ProQR also received a $2.0 million milestone payment from its collaboration with Eli Lilly, with potential for an additional $50 million payment as the partnership expands.

ProQR Therapeutics (Nasdaq: PRQR) reported robust financial results for the second quarter of 2025, highlighting significant progress in its RNA editing pipeline. The company maintained a strong cash position of €119.8 million, providing a financial runway into mid-2027. This substantial cash reserve is crucial for the company's ongoing clinical trials and research and development activities.

One of the key highlights of ProQR's Q2 2025 report is the submission of a Clinical Trial Application (CTA) for its lead program AX-0810, targeting NTCP for cholestatic diseases. Initial Phase 1 data from this trial is expected in the fourth quarter of 2025. This milestone marks a significant step forward for ProQR as it transitions into a clinical-stage organization, potentially providing a near-term catalyst for investor interest.

Additionally, ProQR received a milestone payment of $2.0 million from its collaboration with Eli Lilly. This payment, amounting to approximately €1.8 million, is part of a broader agreement that includes an option for Lilly to expand to 15 targets, which could trigger an additional $50 million payment to ProQR. The collaboration with Eli Lilly represents a strategic partnership that could bring substantial financial benefits and expand ProQR's therapeutic reach.

ProQR's financial position remains solid, despite increased research and development expenses. R&D costs for the first half of 2025 were €23.7 million, compared to €16.3 million in the same period last year. This increase reflects the company's intensified development activities across its pipeline. The company reported a net loss of €22.3 million for H1 2025, up from €10.4 million in the prior year period. This net loss is primarily attributed to the increased R&D expenditures.

Beyond AX-0810, ProQR is advancing other pipeline programs, including AX-2402 targeting MECP2 for Rett Syndrome and AX-2911 targeting MASH. These programs expand the company's therapeutic reach into CNS disorders and cardiovascular diseases, respectively. 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.

ProQR's strong cash position and recent milestone payments position the company well for continued clinical progress and expansion of its therapeutic pipeline. The upcoming events and data releases will be closely watched by investors for further insights into ProQR's progress and potential future catalysts.

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