ProQR Announces Year End 2025 Operating and Financial Results

Core Insights - ProQR Therapeutics reported significant clinical advancements in 2025, particularly with AX-0810 for cholestatic diseases entering Phase 1 trials, and aims to provide target engagement data in the first half of 2026 [2][3] - The company has strengthened its pipeline with new development candidates AX-2402 for Rett syndrome and AX-2911 for MASH, alongside a successful collaboration with Eli Lilly, achieving $4.5 million in milestones [2][8] Financial Highlights - As of December 31, 2025, ProQR held cash and cash equivalents of approximately €92.4 million, down from €149.4 million in 2024 [11] - The net cash used in operating activities for 2025 was €52.8 million, compared to €36.4 million in 2024 [11] - The net loss for 2025 was €42.2 million, or €0.40 per diluted share, an increase from €27.8 million, or €0.32 per diluted share, in 2024 [12] Clinical Development - AX-0810 is currently in a Phase 1 multiple-dose escalation study in healthy volunteers, with initial safety and pharmacokinetic data reported [6] - The company plans to include a PSC patient cohort in the ongoing Phase 1 trial after completing the healthy volunteer cohorts [6] - AX-2402 has shown promising preclinical results in a Rett syndrome mouse model, with plans to initiate first-in-human clinical testing in the first half of 2027 [6][12] Pipeline Expansion - AX-2402 targets the R270X mutation in MECP2 to restore normal protein function for Rett syndrome [4] - AX-2911 aims to address the I148M mutation in PNPLA3, a significant genetic risk factor for fatty liver disease, demonstrating over 80% reduction in hepatic fat content in preclinical studies [5][7] Strategic Collaborations - The collaboration with Eli Lilly is expected to continue in 2026, with potential data updates and additional milestone payments anticipated [8]