Monopar Therapeutics Inc. · NASDAQ:MNPR
Monopar Therapeutics — AASLD The Liver Meeting 2026
19 slides from Monopar Therapeutics's conference presentation, Nov 8, 2026
Slide 1: Residual Hepatic, Neurologic, and Psychiatric Disease Burden in Treatment-Experienced Wilson Disease Patients
AASLD THE LIVER MEETING 2026 Residual Hepatic, Neurologic, and Psychiatric Disease Burden in Treatment-Experienced Patients with Wilson Disease Baseline Findings from the FoCus Phase 3 Trial Frederick Askari, MD, PhD (Presenter) Valentina Medici, MD, FAASLD · Thomas D. Sandahl, MD, PhD · Chandler Ro
Slide 2: Disclosures
Disclosures • Arbor Med, Advisor • Monopar, Advisor • Orphalan, Research Support and Advisor • ETON Pharmaceutical, Advisor • Alexion Pharmaceuticals, Research Support • Arcturus, Advisor • Astra Zeneca, Research Support • Ultragenyx, Research Support and Advisor • Prime Medicine, Advisor and Resear
Slide 3: Wilson Disease: A Rare, Serious Genetic Copper Disorder Requiring Lifelong Treatment
Wilson Disease: A Rare, Serious Genetic Copper Disorder That Requires Lifelong Treatment \ Overview of Wilson Disease Clinical Manifestation • Caused by mutations in the ATP7B gene Neuropsychiatric5 that lead to copper accumulation in the liver • Tremor and brain1 • Dystonia • Devastating hepatic, n
Slide 4: Patients Often Present with Multidomain Symptoms, and Diagnosis May Be Delayed
Patients Often Present with Multidomain Symptoms, and Diagnosis May Be Delayed \ Patients with Symptoms in Each Disease Domain at Diagnosis1 53% 53% have both hepatic and have liver cirrhosis at 90% neurologic/psychiatric symptoms1 diagnosis2 70% Many patients already have advanced liver 60% disease
Slide 5: Currently Available Treatments Reflect Absence of Innovation in Decades
Wilson Disease: Currently Available Treatments Reflect Absence of Innovation in Decades \ Penicillamine Trientine Zinc Approval Year 19701 19852 19973 First FDA-approved therapy; promotes Chelator, like penicillamine, that promotes Overview urinary copper excretion4 urinary copper excretion Blocks i
Slide 6: Patients Report Debilitating Side Effects of Wilson Disease Treatment
Patients Report Debilitating Side Effects They Have to Endure with Wilson Disease Treatment \ Patient Perspectives “[My siblings and I] took D-penicillamine… spent almost two years in the hospital with spasms and cramps throughout our bodies, which we could not control. We no longer walked or talked
Slide 7: FoCus, the Largest Controlled Wilson Disease Trial Ever
FoCus (ALXN1840 Phase 3 Trial), the Largest Controlled Wilson Disease Trial Ever, Included Both Treatment-Naïve and Treatment-Experienced Patients \ • Largest single controlled Wilson disease trial conducted 207 Patients1 • Designed to examine the efficacy and safety of ALXN1840 (tiomolibdate cholin
Slide 8: Bis Choline Tetrathiomolybdate ALXN1840/WTX101 - First in Class
\ Bis Choline tetrathiomolydbdate TTM ALXN1840/WTX 101- First in class SOURCE:
Slide 9: FoCus - ALXN1840 Phase 3 Trial Design
FoCus – ALXN1840 Phase 3 Trial Design Enrollment Randomization Rater-Blinded Treatment Open Label Extension (n=207) 2:1 48 Weeks (n=178) ALXN1840 (n=137) ALXN1840 Treatment-experienced (n=104) (n=137) Age ≥12 years Treatment-naïve (n=33) • ALXN1840 • 189 adult • Median follow-up • 18 adolescent Stan
Slide 10: Summary of Patient Demographics Across Treatment Groups
Summary of Patient Demographics Across Treatment Groups Overall Patient Demographics Unit 3 Treatment Treatment Experienced Naïve Variable (n = 160) (n = 47) Male, n (%) 84 (52.5) 35 (74.5) Sex Female, n (%) 76 (47.5) 12 (25.5) White, n (%) 121 (75.6) 39 (83.0) Asian, n (%) 32 (20.0) 5 (10.6) Race B
Slide 11: 77% of Patients Were Treatment Experienced with Median ~9.5 Years of Prior Treatment
77% of the Patients Were Treatment Experienced with a Median of ~9.5 Years of Prior Wilson Disease (WD) Treatment Prior WD Treatment Duration and Therapy Type Among Treatment-Experienced Patients ~9.5 years Median prior WD treatment 77% Treatment-experienced (n=160) Prior WD therapy options among tr
Slide 12: Unified Wilson's Disease Rating Scale (UWDRS)
Unified Wilson's Disease Rating Scale (UWDRS) \ The Unified Wilson's Disease Rating Scale (UWDRS) is a standardized clinical rating scale developed to assess the severity and progression of symptoms in patients with Wilson Disease Structure & Core Items The subscale spans two distinct components: a
Slide 13: Brief Psychiatric Rating Scale (BPRS)
Brief Psychiatric Rating Scale (BPRS) \ The Brief Psychiatric Rating Scale (BPRS) is a clinician-administered assessment tool used to measure the presence and severity of psychiatric symptoms, particularly in patients with schizophrenia, psychotic disorders, and severe mood disorders. Symptom Domain
Slide 14: Most Patients Symptomatic at Baseline, Multidomain Involvement in ~73%
Most Patients Were Symptomatic at Baseline, With Multidomain Involvement in ~73% Patient Distribution Across Individual and Overlapping Disease Domains (n=2071) Percent2 80% Neurologic 74% Psychiatric 37% Cirrhosis 5% Asymptomatic ~73% of patients had symptoms in two or more domains 1 For overlappin
Slide 15: Substantial Morbidity Persisted at Baseline Despite Prior Treatment
Substantial Morbidity Persisted at Baseline Despite Prior Treatment, with Rates Comparable to Treatment-Naive Patients \ Proportion of Patients with Baseline Morbidity – Treatment-Experienced vs. Treatment-Naive Patients (n=207) Treatment-Experienced (n=160) Treatment-Naïve (n=47) Uncontrolled Neuro
Slide 16: UWDRS Baseline Cut-off Thresholds
UWDRS Baseline Cut-off Thresholds \ Percent of Population Exceeding Threshold SOURCE: 15
Slide 17: Conclusion: Study Limitation Includes Trial Enrollment Bias
Conclusion: Study Limitation Includes Sudy Trial Enrollment Bias FoCus trial baseline data provided a unique opportunity to evaluate Wilson 1 Disease burden cross-sectionally across a large patient population; The majority of patients had significant hepatic, neurologic, and/or 2 psychiatric symptom
Slide 18: Forward-Looking Thoughts
Forward-Looking Thoughts Despite clinical diligence, these observations of ongoing symptomatic disease reflect the limitations of current therapies Diagnosis & Monitoring New Therapies Improved Emerging Non-invasive Pharmacologic Genetic Diagnostics Biomarkers Imaging Therapies Therapies Advances in
Slide 19: Thank You
AASLD THE LIVER MEETING 2026 Thank You With thanks to the patients, families, investigators, and study teams across 52 sites in 22 countries who made the FoCus trial possible.