Monopar Therapeutics Inc. (MNPR), a clinical-stage biopharmaceutical company, on Sunday, announced new analyses of promising results from FoCus, a pivotal Phase 3 trial evaluating ALXN1840 in treating Wilson Disease.
Wilson disease is a rare, genetic disorder caused by a mutation in the ATP7B gene, which impairs the body's ability to excrete copper. This leads to a toxic accumulation of copper in the liver, brain, and other organs. Affecting about 1 in 30,000 people worldwide, it is a progressive disease that results in liver ailments, psychiatric or extensive neurological symptoms, and can prove fatal if untreated.
ALXN1840 (tiomolibidate choline, TMC) is a novel, investigational, once-daily, oral medicine that was granted orphan drug designation in the U.S. and Europe for the treatment of Wilson disease. The first-in-class albumin tripartite complex (ATC) activator mobilizes and tightly sequesters excess copper in ATCs, suppressing redox activities, limiting oxidative damage, and blocking transport across the blood-brain-barrier.
The Phase 3 FoCus trial was a randomized, rater-blinded, multicenter study to evaluate the safety and efficacy of ALXN1840 in treating Wilson disease compared to existing standard-of-care (SOC) treatments. The drug showed rapid and sustained copper mobilization over 48 weeks, thus meeting its primary endpoint. ALXN1840 also showed a favorable safety and tolerability profile across 645 patient-years-of-follow up in 266 patients, over a median of 2.58 years.
The analyses included clinically meaningful improvement, improved clinical global impression (CGI) measures, and durable neurologic benefits observed in ALXN1840-treated patients when compared to those on SOC-treatments.
The company plans to file a new drug application (NDA) for ALXN1840 to the U.S. Food and Drug Administration (FDA) by mid-2026.
MNPR closed Friday at $56.22, down 2.56%.
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