Monopar Therapeutics Inc. · NASDAQ:MNPR
Monopar Therapeutics — American Academy of Neurology (AAN) 2026
10 slides from Monopar Therapeutics's conference presentation, April 2026
Slide 1: Greater clinical benefit with tiomolibdate choline versus standard-of-care in neurologic WD patients
Greater clinical benefit with tiomolibdate choline versus standard-of-care in neurologic WD patients in the Phase 3 FoCus trial Peter Hedera, MD, PhD Department of Neurology University of Louisville © 2026 American Academy of Neurology
Slide 2: Disclosures
Disclosures • Travel expenses to attend and present at AAN 2026 were paid for by Monopar Therapeutics • Dr. Peter Hedera serves as a member of the DMC for Ultragenyx UX-701 for Wilson disease © 2026 American Academy of Neurology
Slide 3: Wilson Disease and Tiomolibdate Choline
Wilson Disease and Tiomolibdate Choline Wilson disease (WD) is a genetic disorder of copper (Cu) overload due to ATP7b dysfunction Impaired Cu ATP7B excretion Tiomolibdate choline (TMC) is an investigational, first-in-class albumin tripartite complex (ATC) activator that binds Cu with high selectivi
Slide 4: FoCus Phase 3 RCT in WD Patients
FoCus Phase 3 RCT in WD Patients UWDRS Wilson disease • The Phase 3 FoCus RCT enrolled 207 patients with WD onto TMC (n=137) or standard of care (SoC, n=70) for 48 13 Other Questions weeks, with optional 5-year extension on TMC 25 • Over half of enrolled patients (56% TMC; 50% SoC) Similar demonstra
Slide 5: More Patients with Neurologic Symptoms Improved and Fewer Worsened with TMC vs. SoC
More Patients with Neurologic Symptoms Improved and Fewer Worsened with TMC vs. SoC SoC TMC Worsened Improved Worsened Improved Durable neuro benefit with TMC sustained over time (shown out Week 4 to ~3 years at the right) Week 12 Week 24 Significantly more likely to Week 36 worsen on SoC vs TMC at
Slide 6: Patients with Neurologic Symptoms Achieved Greater Global Clinical Improvement on TMC vs. SoC
Patients with Neurologic Symptoms Achieved Greater Global Clinical Improvement on TMC vs. SoC CGI-S @ Week 48 CGI-I @ Week 48 p = 0.008 p = 0.003 75% 61.2% (41/67) % Improved at Week 48 50% 47.0% (31/66) 25% 17.2% (5/29) 18.5% (5/27) SoC TMC SoC TMC P-value for comparison of mean change from baselin
Slide 7: TMC Neurologic Benefit Increased Over Time
TMC Neurologic Benefit Increased Over Time UWDRS Part III Change from Baseline – Neurologic subgroup Mean UWDRS Part III change (95% CI) 10 5 MCID 0 -2.04 (n=28) Improvement MCID -4.03 (n=67) -5 -7.16 (n=56) * -10 * = p < 0.05 vs. baseline * -15 Baseline Week 48 Week 96 © 2026 American Academy of Ne
Slide 8: Consistent Neurologic Benefit with TMC Regardless of Cohort
Consistent Neurologic Benefit with TMC Regardless of Cohort UWDRS Part III Change from Baseline – Neurologic subgroup by Cohort 10 Treatment-experienced Treatment-naïve (Cohort 1) (Cohort 2) Mean UWDRS Part III change (95% CI) 6.00 (n=2) 5 MCID 0 Improvement -2.65 (n=26) -4.12 (n=16) -5 -4.00 (n=51)
Slide 9: Favorable Safety Profile Across All Treated WD Patients in Ph 2 & Ph 3 Studies
Favorable Safety Profile Across All Treated WD Patients in Ph 2 & Ph 3 Studies Serious Adverse Events (SAEs) on TMC Number of patients 266 Median time on treatment (years) 2.58 Total patient-years (PYs) 645.6 Patients with any drug-related SAE 13 (4.9%) Neurologic 2 (0.8%) Psychiatric 1 (0.4%) No de
Slide 10: Questions?
Questions? © 2026 American Academy of Neurology
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