Monopar to Present New Long-Term Neurological Efficacy and Safety Data for ALXN1840 in Wilson Disease at the 150th American Neurological Association Annual Meeting
MWN-AI** Summary
Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on innovative treatments, announced that new long-term neurological efficacy and safety data for its investigational therapy ALXN1840 (tiomolybdate choline) for Wilson disease will be showcased at the 150th American Neurological Association Annual Meeting on September 14-15, 2025. The presentations, delivered by Dr. Matthew Lorincz from the University of Michigan, will highlight findings from pooled efficacy outcomes across three clinical trials involving 255 participants and safety data from a fourth trial with 266 participants, revealing a median treatment duration of approximately 2.6 years.
Significantly, the data indicate sustained neurological improvement as measured by the Unified Wilson Disease Rating Scale (UWDRS) over six years. Patients transitioning from standard of care to ALXN1840 exhibited notable additional neurological benefits, reversing some patients' previous deterioration. Additionally, psychiatric outcomes showed statistically significant and sustained improvements, measured by the Brief Psychiatric Rating Scale (BPRS). This consistency of neurological benefits across multiple trials reinforces the credibility of the findings.
A favorable safety profile was also noted, with less than 1% of patients experiencing drug-related neurological serious adverse events (SAE) throughout more than 645 patient-years on ALXN1840. Dr. Lorincz expressed optimism regarding these results, emphasizing their encouraging implications for Wilson disease patients—including those already on existing treatment regimens.
In conjunction with previous presentations of hepatic and systemic efficacy data at the European Association for the Study of the Liver International Liver Congress 2025, these findings bolster the potential of ALXN1840 for addressing both neurological and hepatic manifestations of Wilson disease.
For further details, visit Monopar's official website.
MWN-AI** Analysis
Monopar Therapeutics Inc. (Nasdaq: MNPR) is gaining momentum in the biopharmaceutical market with its investigational therapy, ALXN1840 (tiomolybdate choline), which targets Wilson disease. The upcoming presentations at the 150th American Neurological Association (ANA) Annual Meeting promise to showcase compelling long-term efficacy and safety data, which could significantly influence Market sentiment and investor outlook.
The reported sustained neurological improvements over 6 years, alongside strong psychiatric outcomes and a favorable safety profile, reinforce ALXN1840's potential to revolutionize treatment for Wilson disease. The data indicates that patients previously on standard care who transitioned to ALXN1840 experienced marked neurological benefits, suggesting that ALXN1840 could be an essential therapy for patients not responding to current treatments. The consistency of results across multiple independent clinical trials could bolster investor confidence and potentially attract institutional interest.
From a market perspective, these developments present an opportunity for investors. As Monopar continues to disseminate positive findings and seek regulatory approval, there is likely to be increased trading volume and price appreciation. With less than 1% of patients reporting serious adverse events on ALXN1840, the favorable safety profile could further enhance its appeal to healthcare providers, potentially broadening market acceptance.
However, potential investors should remain cognizant of inherent risks, such as the competitive landscape and the uncertainties surrounding regulatory approval. Monopar's need for capital to support ongoing clinical trials increases financial risk, and the landscape of competitor treatments could shift with evolving clinical data.
In conclusion, Monopar Therapeutics is positioned well for bullish sentiment leading up to and following the ANA meeting. For investors looking to capitalize on biopharma innovations, monitoring Monopar's developments could yield attractive opportunities in the near term.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
WILMETTE, Ill., Sept. 14, 2025 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. (“Monopar” or the “Company”) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, today announced that new data on the long-term 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 poster and oral presentations 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. Monopar’s poster presentation is available at the following link: https://www.monopartx.com/ALXN1840-ANA-2025-Poster-14-Sep-2025 . The oral presentation will be made available online at www.monopartx.com concurrently with Dr. Lorincz’s presentation on September 15, 2025.
The analysis pooled efficacy outcomes from three independent clinical trials (n=255), while safety data included a fourth independent clinical trial (n=266). Median treatment duration with ALXN1840 was approximately 2.6 years for both the efficacy and safety analyses.
The new data presented at ANA highlight the long-term neurological benefit of ALXN1840, and follow the recent presentation of long-term hepatic and systemic efficacy and safety data at the European Association for the Study of the Liver (EASL) International Liver Congress 2025. Together, these findings underscore the potential of ALXN1840 for both the neurological and hepatic manifestations of Wilson disease.
Key findings to be presented at ANA include:
- Sustained Neurological Improvement: Statistically significant neurologic improvement from baseline on the Unified Wilson Disease Rating Scale (“UWDRS”) Part II (patient-reported symptoms) and Part III (clinician-reported symptoms) was sustained over 6 years.
- Crossover Benefit: Patients who crossed over from standard of care (“SoC”) to ALXN1840 showed additional neurological improvement, including a majority of patients who had worsened on SoC demonstrating a reversal on ALXN1840.
- Psychiatric Outcomes: Statistically significant psychiatric improvement from baseline was sustained over multiple years, as measured by the Brief Psychiatric Rating Scale (“BPRS”).
- Consistency Across Trials: Neurological benefit was observed consistently across multiple independent studies.
- Favorable Safety Profile: Across more than 645 patient-years on ALXN1840, less than 1% of patients experienced a drug-related neurological serious adverse event (“SAE”).
“These results are very encouraging for Wilson disease patients, including for those already on standard of care treatment,” said Dr. Matthew Lorincz.
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.
Contact:
Monopar Therapeutics Inc.
Investor Relations
Quan Vu
Chief Financial Officer
vu@monopartx.com
Follow Monopar on social media for updates:
X: @MonoparTx? LinkedIn: Monopar Therapeutics
FAQ**
How does Monopar Therapeutics Inc. (MNPR) plan to leverage the long-term efficacy data of ALXN1840 in their marketing strategy for Wilson disease treatment following the ANA presentation?
Considering the favorable safety profile of ALXN1840, how will Monopar Therapeutics Inc. (MNPR) ensure that this information is effectively communicated to healthcare providers and patients post-announcement?
What steps will Monopar Therapeutics Inc. (MNPR) take to secure necessary funding for advancing ALXN1840 through the regulatory approval process, given the potential market competition in Wilson disease treatments?
How might the crossover benefits observed in patients switching from standard care to ALXN18influence Monopar Therapeutics Inc.'s (MNPR) clinical trial design and future studies?
**MWN-AI FAQ is based on asking OpenAI questions about Monopar Therapeutics Inc. (NASDAQ: MNPR).
NASDAQ: MNPR
MNPR Trading
3.73% G/L:
$58.625 Last:
74,240 Volume:
$57.82 Open:
