ProQR Therapeutics BV ( (PRQR) ) has released its Q4 earnings. Here is a breakdown of the information ProQR Therapeutics BV presented to its investors.
ProQR Therapeutics is a biotechnology company focused on developing transformative RNA therapies using its proprietary Axiomer RNA editing technology, primarily targeting rare and prevalent diseases with unmet needs.
In its latest earnings report, ProQR Therapeutics highlighted significant advancements in its Axiomer RNA editing pipeline, particularly in liver and central nervous system (CNS) programs. The company is on track to file a Clinical Trial Application (CTA) for its lead program AX-0810 in the second quarter of 2025, targeting NTCP for cholestatic diseases. ProQR anticipates up to four clinical data readouts in 2025 and 2026, with the first clinical data for AX-0810 expected in the fourth quarter of 2025.
Financially, ProQR ended 2024 with €149.4 million in cash and cash equivalents, providing a financial runway into mid-2027. The company reported a net loss of €27.8 million for the year, slightly higher than the €27.7 million loss in 2023. Research and development expenses increased to €36.4 million, reflecting the company’s investment in advancing its pipeline, while general and administrative costs decreased to €13.7 million.
Strategically, ProQR expanded its collaboration with the Rett Syndrome Research Trust and secured additional funding to advance its AX-2402 therapy into clinical trials. The company also strengthened its leadership team by appointing Dr. Peter A. Beal as Chief ADAR Scientist. ProQR’s partnership with Eli Lilly remains a cornerstone, with potential milestone income and expansion opportunities.
Looking ahead, ProQR is poised for significant growth as it progresses its RNA editing programs and explores new partnerships. The company’s strong financial position and strategic collaborations position it well to achieve key milestones and continue its mission of developing innovative RNA therapies for patients in need.