공시 • May 21
Monopar Therapeutics Inc Announces Publication of Phase 2 Study Demonstrating ALXN1840 Significantly Improves Copper Balance in Patients with Wilson Disease Monopar Therapeutics Inc. announced that Hepatology Communications has published a peer-reviewed manuscript entitled “Effect of Tiomolibdate Choline on Copper Balance in Patients with Wilson Disease: an Open-label Phase 2 Trial.” The publication reports results from the Phase 2 ALXN1840-WD-204 study (NCT04573309) and demonstrates that ALXN1840 (tiomolibdate choline) produces a rapid, statistically significant, and sustained improvement in daily copper balance in patients with Wilson disease, driven by increased fecal copper excretion. Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised, leading to damage from toxic copper build-up in organs such as the liver and brain. The open-label, single-arm Phase 2 trial evaluated daily dosing of ALXN1840 in nine patients with Wilson disease across two centers in the United Kingdom and New Zealand. Patients were admitted to a clinical research unit and initiated on a copper-controlled diet, with all copper intake and output collected during a pre-treatment baseline period and after initiation of daily ALXN1840 over multiple weeks. The publication builds on a recently published peer-reviewed Journal of Hepatology Letter to the Editor, which highlighted the importance of comparing outcomes to a pre-treatment baseline to accurately assess the effect of a potential Wilson disease treatment on copper balance. Statistically significant reduction in daily copper balance from baseline, due to increased fecal copper excretion. Cumulative mean decrease from baseline in copper balance of -6.08 mg over 21 days (95% CI: -10.18 mg to -1.98 mg). Mean daily copper balance change from baseline of -0.37 mg (p=0.005) during the 15 mg/day treatment period and -0.29 mg (p=0.023) through the overall study period. Approximately 50% increase in the daily fecal copper output-to-intake ratio compared to baseline (p=0.041). Immediate increases in plasma total copper and directly measured non-ceruloplasmin-bound copper (dNCC), consistent with copper mobilization and formation of stable albumin tripartite complexes (ATCs) consisting of copper, ALXN1840, and albumin. ALXN1840 was generally well tolerated; no serious adverse events were reported. The observed improvements in copper balance and copper mobilization occurred in a Wilson disease patient population with a mean prior current standard of care treatment duration of 16 years, suggesting that despite years of treatment with currently available therapies, patients present with a considerable amount of residual copper in the body that ALXN1840 is able to mobilize and eliminate. This finding is consistent with data from the completed 48-week Phase 3 trial, in which ALXN1840 demonstrated superior copper mobilization compared to standard of care even in patients with a mean prior standard of care treatment duration of 11 years. 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. 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. 공시 • May 04
Monopar Therapeutics Inc., Annual General Meeting, Jun 22, 2026 Monopar Therapeutics Inc., Annual General Meeting, Jun 22, 2026. Location: at 1000 skokie blvd., wilmette, il 60091, United States 공시 • 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. Recent Insider Transactions Derivative • Apr 07
Co-Founder exercised options and sold US$108k worth of stock On the 31st of March, Chandler Robinson exercised options to acquire 2k shares at no cost and sold these for an average price of US$54.79 per share. This trade did not impact their existing holding. For the year to December 2019, Chandler's total compensation was 53% salary and 47% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2025, Chandler's direct individual holding has increased from 73.47k shares to 153.29k. Company insiders have collectively sold US$3.1m more than they bought, via options and on-market transactions in the last 12 months. 공시 • Mar 03
Monopar Therapeutics Inc. Appoints Susan Rodriguez as Chief Commercial and Strategy Officer, Effective from March 2, 2026 Monopar Therapeutics Inc. announced the appointment of Susan Rodriguez as Chief Commercial and Strategy Officer, effective March 2, 2026. 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. Susan Rodriguez brings more than 30 years of biopharmaceutical leadership experience. She most recently served as Chief Operating Officer of Avadel Pharmaceuticals, where she led the company’s commercial strategy and execution, contributing to the recently completed acquisition of Avadel by Alkermes for over $2 billion. Prior to Avadel, Ms. Rodriguez was Chief Commercial Officer of Ardelyx, where she established the company’s first commercial organization and successfully transitioned Ardelyx from a development-stage company to a commercial biopharmaceutical enterprise. She led the launches of two key therapeutics, building market access, sales, marketing, and patient services capabilities and delivering strong early adoption in highly competitive areas. Before that, she served as Chief Executive Officer of Tolmar Pharmaceuticals, a specialty oncology company, where she established a new U.S. commercial entity, secured a market-leading position for its primary oncology therapy, advanced its pipeline, and successfully launched a new rare disease therapy. Earlier, she held various commercial leadership positions across multiple divisions at Abbott as well as the Abbott–Takeda joint venture, TAP Pharmaceuticals. She holds an M.S. and a B.S. in psychology, with a minor in chemistry, from the University of Pennsylvania. 
