Monopar Therapeutics Inc. · NASDAQ:MNPR
Monopar Therapeutics — EASL Congress 2026
16 slides from Monopar Therapeutics's conference presentation, May 2026
Slide 1: ALXN1840 stabilizes liver disease and improves neurological symptoms in Wilson disease
ALXN1840 (tiomolibdate choline) stabilizes liver disease and improves neurological symptoms as well as quality of life in treatment-experienced Wilson disease patients Valentina Medici MD, FAASLD | UC Davis Medical Center, Sacramento, CA, USA Aftab Ala, Karl Heinz Weiss, Edward J. Gane, Zoe Mariño,
Slide 2: Wilson Disease (WD)
Wilson Disease (WD) • Wilson disease is a rare autosomal recessive disorder of impaired copper (Cu) transport ~1 in 30,000 • Cu accumulates in the liver and brain, causing Estimated prevalence worldwide 1 organ damage ~25% • Inadequately treated or untreated, Cu accumulation Of patients with neurolo
Slide 3: Tiomolibdate Choline (TMC; ALXN1840)
Tiomolibdate Choline (TMC; ALXN1840) CU-BINDING AFFINITY (K D ) OF WD THERAPIES TMC-CU-ALBUMIN FORMS A TRIPARTITE COMPLEX ALXN1840 (TMC) Trientine D-penicillamine Cu 2.3 x 10-20 1.7 x 10-16 2.4 x 10-16 Cu Tighter Cu binding Cu TMC 10-20 10-16 TMC 10-19 10-18 10-17 Cu Tiomolibdate choline (TMC, Tiomo
Slide 4: TMC Protects the Liver by Binding and Neutralizing Hepatic Cu
TMC Protects the Liver by Binding and Neutralizing Hepatic Cu Differentiated from SoC, TMC's binding affinity is strong enough to inhibit reactivity of free Cu and remove Cu from metallothionein1 — directly protecting the liver TMC AND REACTIVE FREE CU TMC AND METALLOTHIONEIN (MT) Excess reactive TM
Slide 5: 48-week Liver Biopsy Study Methods
48-week Liver Biopsy Study Methods Open-label, multicenter, pathologist-blinded trial of TMC monotherapy in treatment-experienced (≥ 1 year) patients with WD SCREENIN G PRIMARY TREATMENT PERIOD OPT IONAL EXTENSION PERIOD < 4 weeks 48 weeks 48 weeks Eligibility TMC 15–30 mg/day TMC 15–30 mg/day Basel
Slide 6: Literature Shows Stable/Increased SoC Hepatic Cu Change
Literature Shows Stable/Increased SoC Hepatic Cu Change Serial liver biopsy studies of SoC show stable/increased hepatic Cu over years on treatment # of years between biopsies Zinc Increased hepatic Cu after > 5 yrs of treatment Penicillamine # of years between biopsies Adapted from Medici V et al.
Slide 7: Study Enrolled a Heavily Pre-treated Population
Study Enrolled a Heavily Pre-treated Population CUMULATIVE DURATION OF PRIOR WD TREATMENT PATIENT ENROLLMENT Median: 13.8 yrs Enrolled w/ Baseline Biopsy 6 n = 29 Number of patients Chelator ± zinc 4 Zinc monotherapy 6 5 5 Study discontinuations 6 2 Adverse event n=3 Subject withdrawal n=1 2 Physici
Slide 8: Baseline Demographics and Characteristics
Baseline Demographics and Characteristics Enrolled NASH CRN FIBROSIS STAGE patients Number of patients n=29 Demographics Male sex, n (%) 19 (65.5%) Age in years, median (IQR) 35.0 (17.0) Clinical characteristics Years of prior WD treatment, median (IQR) 13.8 (10.9) NAS TOTAL SCORE Number of patients
Slide 9: TMC Also Shows No Significant Change in Hepatic Cu
TMC Also Shows No Significant Change in Hepatic Cu No statistically significant difference in hepatic Cu concentration after 48 weeks of treatment, consistent with expected results in heavily pre-treated patients OBSERVED CHANGE FROM BASELINE IN HEPATIC CU CONCENTRATION ( μg/g) 1000 Hepatic copper (
Slide 10: TMC Treatment Leads to Hepatic Stabilization and Improvement
TMC Treatment Leads to Hepatic Stabilization and Improvement By Week 48, most patients demonstrated stabilization or improvement in: Fibrosis Inflammation Steatosis NAFLD Activity Score 100% 23/24 21/24 19/24 80% 18/24 17/24 17/24 16/24 60% Improved or stabilized 40% 20% 0% NASH CRN Total Hepatocyte
Slide 11: Neurological WD Symptoms Significantly Improved at Week 48
Neurological WD Symptoms Significantly Improved at Week 48 UWDRS PART III CHANGE FROM BASELINE Mean change from baseline (pts) -2.92 (n=26, p=0.002) *p < 0.05 vs baseline *
Slide 12: Global Clinical Disease Burden Significantly Improved at Week 48
Global Clinical Disease Burden Significantly Improved at Week 48 CLINICAL GLOBAL IMPROVEMENT FROM BASELINE Mean score (pts) Baseline * *p < 0.05 vs baseline
Slide 13: Patient-reported Quality of Life Significantly Improved at Week 48
Patient-reported Quality of Life Significantly Improved at Week 48 EUROQOL-5D UK HEALTH INDEX CHANGE FROM BASELINE Mean change from baseline (pts) * +0.06 (n=25, p=0.005) *p < 0.05 vs baseline
Slide 14: TMC is Well Tolerated, with Few Severe Adverse Events
TMC is Well Tolerated, with Few Severe Adverse Events TEAEs IN SAFETY POPULATION (N=31) • TMC was generally well tolerated; most treatment-emergent adverse events Primary period Extension period (TEAEs) were nonserious and Grade 1 n=31 n=25 or 2 in severity TEAEs 30 (96.8%) 19 (76.0%) Related 24 (77
Slide 15: Conclusions
Conclusions • Hepatic Cu concentration was stable after 48 weeks of TMC, consistent with published serial liver biopsy studies of WD patients treated with standard-of-care • Treatment with TMC in heavily pre-treated patients resulted in hepatic stabilization/improvement • TMC treatment significantly
Slide 16: Questions?
S0EASL CONGRESS a OCIS Questions?