Monopar to Announce Long-Term ALXN1840 Data in Wilson Disease at ANA 2025

09/14/2025 – 08:00 AM

WILMETTE, Ill. – Monopar Therapeutics Inc. (Nasdaq: MNPR), a biopharmaceutical company focused on developing treatments for unmet medical needs, announced today that new long-term data regarding the neurological efficacy and safety of its investigational therapy, ALXN1840 (tiomolybdate choline), for Wilson disease will be presented at the 150th American Neurological Association (ANA) Annual Meeting on September 14-15, 2025. The presentation, encompassing both a poster session and an oral presentation, will be delivered by Matthew Lorincz, M.D., Ph.D., Professor of Neurology and Co-Director of the Wilson Disease Center of Excellence at the University of Michigan. The poster presentation is available on Monopar’s website, with the oral presentation slated for release concurrently with Dr. Lorincz’s presentation on September 15, 2025.

The pooled analysis incorporates efficacy outcomes from three independent clinical trials (n=255) and safety data from a fourth independent clinical trial (n=266). Participants in both the efficacy and safety analyses had a median treatment duration of approximately 2.6 years with ALXN1840.

These findings build upon the recently presented long-term hepatic and systemic efficacy and safety data shared at the European Association for the Study of the Liver (EASL) International Liver Congress 2025. Collectively, the data suggests ALXN1840 holds significant promise for addressing both the neurological and hepatic manifestations of Wilson disease, a rare genetic disorder characterized by copper accumulation in various organs, primarily the liver and brain.

Key data points to be presented at ANA include:

• Sustained Neurological Improvement: Clinically meaningful and statistically significant neurological improvement from baseline was observed and sustained over six years, as measured by the Unified Wilson Disease Rating Scale (UWDRS) Part II (patient-reported symptoms) and Part III (clinician-reported symptoms). The UWDRS is a validated tool used to assess the severity of neurological impairment in Wilson disease.
• Crossover Benefit: Patients initially treated with standard of care (SoC) who subsequently transitioned to ALXN1840 exhibited additional neurological improvement. Notably, a significant portion of patients who had experienced neurological worsening while on SoC demonstrated a reversal of this decline after switching to ALXN1840. This suggests a potential benefit even for those who do not respond adequately to traditional therapies.
• Psychiatric Outcomes: Statistically significant psychiatric improvement from baseline, as assessed using the Brief Psychiatric Rating Scale (BPRS), was also sustained over multiple years. This is particularly significant considering the often-overlooked psychiatric manifestations of Wilson disease.
• Consistency Across Trials: The neurological benefits of ALXN1840 were consistently observed across multiple independent clinical studies, reinforcing the robustness of the findings.
• Favorable Safety Profile: Over 645 patient-years of exposure to ALXN1840, less than 1% of patients experienced a drug-related neurological serious adverse event (SAE), highlighting the drug’s safety profile.

“These results are very encouraging for Wilson disease patients, including for those already on standard of care treatment,” stated Dr. Matthew Lorincz, emphasizing the potential for ALXN1840 to improve patient outcomes and quality of life.

This long-term data provides further validation of ALXN1840’s potential as a disease-modifying agent in Wilson disease. While standard therapies primarily focus on chelating copper, ALXN1840 offers a novel mechanism of action by directly binding copper, potentially preventing its accumulation and subsequent damage to tissues. Further studies are warranted to fully elucidate the long-term impact of ALXN1840 on disease progression and patient survival.

About Monopar Therapeutics Inc.

Monopar Therapeutics is a clinical-stage biopharmaceutical company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs including Phase 1-stage MNPR-101-Zr for imaging advanced cancers, and Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers. For more information, visit: www.monopartx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Examples of these forward-looking statements include statements concerning: that these findings underscore the potential of ALXN1840 for both the neurological and hepatic manifestations of Wilson disease; and that these results are very encouraging for Wilson disease patients, including for those already on standard of care treatment. The forward-looking statements involve risks and uncertainties including, but not limited to: uncertainties related to the regulatory process that Monopar intends to initiate related to ALXN1840 and the outcome thereof; the rate of market acceptance and competitiveness in terms of pricing, efficacy and safety, of any products for which Monopar receives marketing approval, and Monopar’s ability to competitively market any such products as compared to larger pharmaceutical firms; Monopar’s ability to raise sufficient funds in order for the Company to support continued preclinical, clinical, regulatory, precommercial and commercial development of its programs and to make contractual milestone payments, as well as its ability to further raise additional funds in the future to support any existing or future product candidate programs through completion of clinical trials, the approval processes and, if applicable, commercialization; and the significant general risks and uncertainties surrounding the research, development, regulatory approval, and commercialization of imaging agents and therapeutics. Actual results may differ materially from those expressed or implied by such forward-looking statements. Risks are described more fully in Monopar’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Monopar undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Any forward-looking statements contained in this press release represent Monopar’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

FAQ

When will Monopar (MNPR) present ALXN1840 data at the ANA Annual Meeting 2025?

Monopar will present the data on September 14-15, 2025, with Dr. Matthew Lorincz delivering both poster and oral presentations.

What are the key efficacy results for ALXN1840 in Wilson disease?

The data showed statistically significant neurologic improvements sustained over 6 years on UWDRS scales, with additional benefits observed in patients who switched from standard of care.

How many patients were included in the ALXN1840 clinical trials analysis?

The analysis included 255 patients for efficacy outcomes from three trials, and 266 patients for safety data including a fourth trial, with a median treatment duration of 2.6 years.

What is the safety profile of ALXN1840 in Wilson disease patients?

ALXN1840 showed a favorable safety profile with less than 1% of patients experiencing drug-related neurological serious adverse events across more than 645 patient-years.

Where can investors access Monopar’s ALXN1840 presentation materials?

The poster presentation is available at monopartx.com/ALXN1840-ANA-2025-Poster-14-Sep-2025, and the oral presentation will be available at monopartx.com on September 15, 2025.