Monopar Therapeutics Presents Phase 2 ALXN1840 Data at EASL 2026

Monopar Therapeutics announced the presentation of Phase 2 ALXN1840-WD-205 data at the European Association for the Study of the Liver, or EASL, Congress 2026. In a presentation, the company presented results from the open-label, multicenter Phase 2 trial evaluating the effects of ALXN1840 on liver pathology and clinical outcomes in heavily pre-treated patients with Wilson disease. The open-label, multicenter, pathologist-blinded Phase 2 trial evaluated ALXN1840 monotherapy over 48 weeks in 29 treatment-experienced Wilson disease patients, with an optional 48-week extension period. The study population had extensive prior treatment, with a median duration of 13.8 years. Liver biopsy was performed at baseline and Week 48 to assess hepatic copper concentration, stage of fibrosis, and grade of steatosis. Neurologic, clinical, and quality-of-life outcomes were also assessed at baseline and Week 48, with patients continuing in the extension period assessed again at Week 96. By Week 48, among the 24 patients with paired biopsies, the preponderance demonstrated stabilization or improvement across histologic measures assessed, including hepatocyte necrosis, steatosis grade, lobular inflammation, portal inflammation, NAFLD Activity Score total, hepatocellular ballooning and fibrosis stage. No statistically significant change in hepatic copper concentration after 48 weeks, consistent with published Wilson disease studies of standard of care therapies showing hepatic copper remains stable or increases even after years on treatment. Significant improvements in the Unified Wilson Disease Rating Scale Part III score were observed at Week 48. Significant improvements in the Clinical Global Impressions scale were observed at Week 48. Significant improvements in patient-reported quality of life, as measured by the EuroQoL 5-Dimensions UK Health Index, were observed at Week 48. ALXN1840 was generally well tolerated; most treatment-emergent adverse events were nonserious and Grade 1 or 2 in severity. A safety analysis of the extension period showed a consistent treatment-emergent adverse event profile. These results, in a heavily pre-treated Wilson disease population, demonstrate that ALXN1840 can stabilize liver disease and provide clinically meaningful improvements in neurologic symptoms and quality of life. The neurologic and quality of life findings from this study complement the increased copper mobilization and clinical improvement shown in the completed Phase 3 pivotal trial.