MNPR News & Events

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.

Monopar Therapeutics announced new analyses from the randomized controlled Phase 3 FoCus trial of ALXN1840 showing greater neurologic benefit versus standard of care in Wilson disease patients with neurologic symptoms at baseline. The data will be presented today at the American Academy of Neurology, AAN, Annual Meeting 2026, taking place April 18-22, 2026. In a late-breaker oral and poster presentation titled "Greater clinical benefit with tiomolibdate choline versus standard-of-care in neurologic Wilson disease patients in the Phase 3 FoCus Trial," Dr. Peter Hedera, MD, PhD, Department of Neurology, University of Louisville School of Medicine, will present results showing that ALXN1840 provided greater neurologic improvement and significantly less worsening than standard of care through Week 48, with durable neurologic benefit observed over multiple years of treatment.

Monopar Therapeutics (MNPR) announced the appointment of Susan Rodriguez as Chief Commercial and Strategy Officer, effective immediately. In this newly created executive role, Ms. Rodriguez will lead the Company's commercial strategy and infrastructure build-out as Monopar prepares for the planned submission of a New Drug Application to the U.S. Food and Drug Administration in the first half of 2026 for ALXN1840, its late-stage investigational therapy for Wilson disease. She most recently served as COO of Avadel Pharmaceuticals (AVDL).