Monopar Presents ALXN1840 Late-Breaker Data at EASL 2025

Core Viewpoint - Monopar Therapeutics Inc. is presenting promising long-term efficacy and safety data for its drug candidate ALXN1840 for Wilson disease at the EASL International Liver Congress 2025, indicating its potential as a therapeutic option for patients with this rare genetic condition [1][2][3] Group 1: Efficacy and Safety Data - The efficacy data were pooled from three clinical trials involving a total of 255 patients, while safety data were derived from an additional trial with 266 patients [2] - The median treatment duration for efficacy was 961 days (approximately 2.63 years) and for safety was 943.5 days (approximately 2.58 years) [2] - Sustained improvements were observed in the Unified Wilson Disease Rating Scale (UWDRS) for both patient-reported and clinician-assessed symptoms [6] - There was a significant increase in copper mobilization, evidenced by a sustained increase in directly measured non-ceruloplasmin-bound copper (dNCC) [6] - Improvements were noted on the Clinical Global Impression – Improvement (CGI-I) scale for ALXN1840 compared to standard care [6] - The New Wilson Index showed improvement for patients treated with ALXN1840, based on various clinical parameters [6] - Patient-reported convenience and effectiveness of ALXN1840 were higher compared to standard care, especially among those transitioning from standard care to ALXN1840 [6] - Fewer than 5% of patients experienced drug-related serious adverse events, with no cases reported for renal or urinary system issues [6] Group 2: Company Overview - Monopar Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments, including ALXN1840 for Wilson disease and various radiopharmaceutical programs for advanced cancers [4]