Core Insights - ProQR Therapeutics reported its financial and operational results for Q3 2025, highlighting significant advancements in its RNA editing technology platform, Axiomer, and the initiation of a first-in-human study for its lead program AX-0810 [1][2]. Financial Highlights - As of September 30, 2025, ProQR held cash and cash equivalents of approximately €106.9 million, a decrease from €149.4 million at the end of 2024 [4]. - The net cash used in operating activities for the nine-month period ended September 30, 2025, was €39.4 million, compared to €27.0 million for the same period in 2024 [4][6]. - The net loss for the nine-month period was €33.3 million, or €0.32 per diluted share, compared to a net loss of €18.5 million, or €0.23 per diluted share, for the same period last year [7][16]. Recent Progress and Upcoming Events - The company received CTA authorization for the Phase 1 trial of AX-0810, targeting NTCP for cholestatic diseases, and is initiating the study in healthy volunteers [4][5]. - Initial safety, tolerability, and pharmacokinetic (PK) data from Cohort 1 are expected by year-end 2025, with target engagement data anticipated in the first half of 2026 [5]. - ProQR's Axiomer pipeline includes programs targeting various diseases, such as AX-2402 for Rett syndrome and AX-2911 for fatty liver disease, with updates expected in the coming months [5]. Collaboration and Milestones - The company achieved certain milestones in its collaboration with Eli Lilly, amounting to $2.0 million (~€1.8 million) [6]. - ProQR continues to execute its partnership with Eli Lilly, with potential data updates and milestone income from the ongoing collaboration [5]. Research and Development Costs - Research and development costs for the nine-month period ended September 30, 2025, were €34.8 million, compared to €25.7 million for the same period last year [6]. - General and administrative costs were €11.2 million for the same period, up from €9.7 million in 2024 [6].
ProQR Announces Third Quarter 2025 Operating and Financial Results