Core Insights - Monopar Therapeutics Inc. announced the publication of a peer-reviewed Letter to the Editor in the Journal of Hepatology, discussing the effects of ALXN1840 on copper balance in patients with Wilson disease [1][2] Group 1: Study Findings - The earlier conclusion from the Phase 2 ALXN1840-WD-204 study indicated that ALXN1840 did not promote copper excretion due to methodological limitations in the copper balance equation [2] - The new analysis shows that ALXN1840 statistically significantly improved copper balance, increasing copper excretion over the study duration [2] - Key findings include: - For the 15 mg/day treatment period, the mean daily difference in copper balance was -0.367 mg (p=0.005) - For the overall treatment period, the mean daily difference was -0.289 mg (p=0.023) - The cumulative mean change from baseline was -6.08 mg (95% CI: -10.18 mg to -1.98 mg) [5] Group 2: Company Overview - Monopar Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet medical needs, including late-stage ALXN1840 for Wilson disease [8] - The company also has radiopharmaceutical programs in various stages of development for imaging and treating advanced cancers [8]

Core Insights - Monopar Therapeutics Inc. announced the publication of a peer-reviewed Letter to the Editor in the Journal of Hepatology, discussing the effects of ALXN1840 on copper balance in patients with Wilson disease [1][2] Group 1: Study Findings - The earlier conclusion from the Phase 2 ALXN1840-WD-204 study indicated that ALXN1840 did not promote copper excretion due to methodological limitations in the copper balance equation [2] - By comparing pre- and post-treatment data, the analysis showed that ALXN1840 statistically significantly improved copper balance, leading to increased copper excretion [2] - Key findings from the study include: - For the 15 mg/day treatment period, the mean daily difference in copper balance was -0.367 mg (p=0.005) - For the overall treatment period, including dose changes, the mean daily difference was -0.289 mg (p=0.023) - The cumulative mean change from baseline was -6.08 mg (95% CI: -10.18 mg to -1.98 mg) [5] Group 2: Company Overview - Monopar Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet medical needs, including late-stage ALXN1840 for Wilson disease and various radiopharmaceutical programs [8]

Core Insights - Monopar Therapeutics Inc. announced new data on the long-term neurological efficacy and safety of its investigational therapy ALXN1840 for Wilson disease, to be presented at the 150th American Neurological Association Annual Meeting [1][3] Group 1: Efficacy and Safety Data - The analysis pooled efficacy outcomes from three independent clinical trials involving 255 patients, while safety data included a fourth independent clinical trial with 266 patients [2] - Median treatment duration with ALXN1840 was approximately 2.6 years for both efficacy and safety analyses [2] - Statistically significant neurological improvement was sustained over 6 years on the Unified Wilson Disease Rating Scale [6] - Patients who crossed over from standard of care to ALXN1840 showed additional neurological improvement, with many reversing worsened conditions [6] - Statistically significant psychiatric improvement was also sustained over multiple years, measured by the Brief Psychiatric Rating Scale [6] - Neurological benefits were consistently observed across multiple independent studies [6] - The safety profile of ALXN1840 was favorable, with less than 1% of patients experiencing drug-related neurological serious adverse events over more than 645 patient-years [6] Group 2: Company Overview - Monopar Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet medical needs, including ALXN1840 for Wilson disease and various radiopharmaceutical programs [4]

Core Insights - Monopar Therapeutics Inc. announced new data on the long-term neurological efficacy and safety of its investigational therapy ALXN1840 for Wilson disease, to be presented at the 150th American Neurological Association Annual Meeting [1][3] - The analysis pooled efficacy outcomes from three independent clinical trials involving 255 patients, while safety data included a fourth independent clinical trial with 266 patients, with a median treatment duration of approximately 2.6 years [2] Efficacy and Safety Findings - Long-term neurological benefits of ALXN1840 were highlighted, showing sustained improvement on the Unified Wilson Disease Rating Scale (UWDRS) over 6 years [6] - Patients who switched from standard of care to ALXN1840 experienced additional neurological improvements, with many reversing previous declines [6] - Statistically significant psychiatric improvements were also sustained over multiple years, measured by the Brief Psychiatric Rating Scale (BPRS) [6] - Neurological benefits were consistently observed across multiple independent studies [6] - The safety profile of ALXN1840 was favorable, with less than 1% of patients experiencing drug-related serious adverse events over more than 645 patient-years [6] Company Overview - Monopar Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet medical needs, including ALXN1840 for Wilson disease and various radiopharmaceutical programs [4]

Core Viewpoint - Monopar Therapeutics Inc. reported a productive year in 2024, highlighted by the in-licensing of ALXN1840, initiation of two Phase 1 clinical trials, and a strengthened balance sheet with over $55 million in net proceeds from financings [2][3]. Recent Program Developments - The company plans to submit a New Drug Application (NDA) for ALXN1840 targeting Wilson disease in early 2026, following a successful Phase 3 clinical trial [3]. - ALXN1840 is a potent copper binder and mobilizer, addressing a rare genetic condition that leads to toxic copper accumulation [3]. - Monopar has executed a worldwide exclusive license for ALXN1840 with Alexion, which included a cash payment of $4 million and a 9.9% equity stake in Monopar [3]. Clinical Trials - MNPR-101 is currently enrolling in Phase 1 imaging and therapeutic oncology trials, targeting aggressive cancers through its imaging agent MNPR-101-Zr and therapeutic agent MNPR-101-Lu [4]. Recent Financings - In Q4 2024, Monopar raised over $55 million through various financings, including public offerings and private placements [5]. - The company dosed its first patient with MNPR-101-Lu in December 2024 and presented promising clinical data at the EANM 2024 Annual Congress [5]. Financial Results - As of December 31, 2024, Monopar reported cash and short-term investments of $60.2 million, sufficient to fund operations through at least December 31, 2026 [6]. - The net loss for Q4 2024 was $10.9 million, compared to $1.8 million in Q4 2023, while the annual net loss for 2024 was $15.6 million, up from $8.4 million in 2023 [7]. Research and Development Expenses - R&D expenses for Q4 2024 were $9.9 million, significantly higher than $1.0 million in Q4 2023, primarily due to the in-licensing of ALXN1840 [8]. - For the year ended December 31, 2024, R&D expenses totaled $13.0 million, compared to $5.6 million in 2023, reflecting increased investment in clinical programs [9]. General and Administrative Expenses - G&A expenses for Q4 2024 were $1.2 million, up from $0.9 million in Q4 2023, attributed to increases in personnel salaries and consulting fees [10]. - For the full year 2024, G&A expenses remained stable at $3.2 million compared to 2023 [11].