"2025 was a productive year for Monopar, marked by multiple ALXN1840 data presentations, an important publication, a strengthened balance sheet and continued progress toward a planned New Drug Application submission for ALXN1840 in Wilson disease," said Chandler Robinson, MD, Chief Executive Officer of Monopar. "We also recently strengthened our leadership team with the addition of Susan Rodriguez as Chief Commercial and Strategy Officer as we prepare for the potential launch of ALXN1840. We are grateful to the Wilson disease patients and their families whose experiences have informed our efforts to advance ALXN1840."
Recent Program Developments
ALXN1840, NDA Submission Planned for Mid-2026 for Wilson Disease
Wilson disease is a rare genetic disorder characterized by impaired copper elimination, resulting in toxic accumulation in organs such as the liver and brain. ALXN1840 binds and mobilizes copper and has a novel mechanism of action as an albumin tripartite complex ("ATC") activator that differentiates it from currently available first-line therapies.
Based on recent interactions with the U.S. Food and Drug Administration ("FDA"), Monopar plans to submit a New Drug Application ("NDA") for ALXN1840 in mid-2026.
ALXN1840 updates:
EASL 2025: Presented pooled long-term efficacy and safety data (n=255; median treatment duration 2.63 years), with additional safety data (n=266) supporting a favorable safety profile, as a late-breaking abstract
ANA 2025: Presented data demonstrating long-term neurological benefit; the abstract was selected for oral and poster presentation and designated an "Abstract of Distinction"
Journal of Hepatology / AASLD 2025: Reported statistically significant improvement in copper balance, with sustained improvement in daily copper balance driven by increased fecal copper excretion
EL-PFDD: Attended externally led patient-focused drug development ("EL-PFDD") meeting with the FDA on January 29, 2026. During the meeting, patients and caregivers described the burden of Wilson disease, shared their experience with the currently available treatments, and highlighted the urgent need for additional treatment options
Upcoming 2026 presentations: Abstracts accepted for presentation at EASL 2026 and the American Academy of Neurology ("AAN") 2026 Annual Meeting, including:
Tiomolybdate choline stabilizes liver disease and improves neurological symptoms as well as quality of life in treatment-experienced Wilson disease patients (EASL 2026 oral presentation)
Greater clinical benefit with tiomolybdate choline versus standard-of-care in neurologic Wilson disease patients in the Phase 3 FoCus Trial (AAN 2026 late-breaking oral and poster presentation)
MNPR-101 ...
