Announcement • Apr 20

Monopar Therapeutics Inc. Presents Phase 3 Data Showing Greater Neurologic Benefit With ALXN1840 Versus Standard of Care In Wilson Disease Patients With Neurologic Symptoms

Monopar Therapeutics Inc. announced new analyses from the randomized controlled Phase 3 FoCus trial of ALXN1840 (tiomolibdate choline, TMC) showing greater neurologic benefit versus standard of care (SoC) in Wilson disease patients with neurologic symptoms at baseline. 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. In the randomized FoCus trial, analysis of patients with neurologic symptoms at baseline (TMC: n=77; SoC: n=35) demonstrated that treatment with ALXN1840 resulted in both higher rates of improvement and lower rates of worsening, addressing a critical unmet need in the neurologic management of Wilson disease. Clinically meaningful neurologic worsening at Week 48 was observed in 25% of patients treated with standard of care vs 9% of ALXN1840-treated patients (p=0.038). Clinically meaningful neurologic improvement at Week 48 was observed in 45% of ALXN1840-treated patients vs 32% on standard of care. CGI-S improvement from baseline to Week 48 was greater with ALXN1840 vs standard of care (61% vs 17%; p=0.008). CGI-I improvement at Week 48 was greater with ALXN1840 vs standard of care (47% vs 19%; p=0.003). Durable neurologic benefit in the ALXN1840-treated group continued to increase during long-term follow-up on treatment and was sustained over approximately 3 years. Neurologic benefit was consistent across both treatment-naïve and treatment-experienced patients with neurologic symptoms at baseline, supporting ALXN1840’s potential as a novel treatment option for Wilson disease. ALXN1840 has demonstrated a well-characterized and favorable safety profile across Phase 2 and Phase 3 studies (266 patients; median 2.58 years on treatment; max >8 years), with drug-related serious adverse events (SAEs) limited to 4.9% of patients — including neurologic SAEs in < 1% — and no treatment-related deaths. These findings support the continued advancement of ALXN1840 toward the planned New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) in mid-2026. Wilson disease is a rare genetic disorder that affects approximately 1 in 30,000 people worldwide. It is caused by mutations in the ATP7B gene, which impairs the body's ability to excrete copper. It is characterized by toxic accumulation of copper in the liver, brain, and other organs, leading to progressive and potentially fatal outcomes if untreated. ALXN1840 (tiomolibdate choline, TMC) is a novel first-in-class Albumin Tripartite Complex (ATC) activator under investigation for the treatment of Wilson disease. ALXN1840 rapidly mobilizes and tightly sequesters excess copper in ATCs, suppressing its redox reactivity, limiting oxidative damage, and blocking transport across the blood–brain barrier. Clinical data demonstrate that ALXN1840 improves copper balance by increasing fecal copper excretion. In the Phase 3 pivotal trial, ALXN1840 demonstrated rapid and sustained copper mobilization (primary endpoint) that was significantly greater than standard of care over 48 weeks in both previously treated and untreated patients. Durable clinical improvement and a favorable safety and tolerability profile were observed across 645 patient-years of follow-up in 266 patients.

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Monopar Therapeutics Inc. Presents Phase 3 Data Showing Greater Neurologic Benefit With ALXN1840 Versus Standard of Care In Wilson Disease Patients With Neurologic Symptoms

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