Monopar Presents ALXN1840 Late-Breaker Data at EASL 2025

Rhea-AI Summary

Monopar Therapeutics (Nasdaq: MNPR) presented late-breaker data at EASL 2025 on ALXN1840, their drug candidate for Wilson disease. The analysis pooled data from three clinical trials (n=255) for efficacy and included a fourth trial for safety analysis (n=266), with median treatment durations of 2.63 and 2.58 years respectively.

Key findings showed sustained improvements in patient-reported and clinician-assessed symptoms (UWDRS), increased copper mobilization, better CGI-I scale results compared to standard care, and improvement in the New Wilson Index. The drug demonstrated strong safety with less than 5% of patients experiencing drug-related serious adverse events and no renal/urinary system SAEs. Patients reported higher convenience and effectiveness compared to standard treatment.

Positive

• Strong safety profile with less than 5% drug-related serious adverse events
• Sustained improvements in patient-reported and clinician-assessed symptoms
• Superior convenience and effectiveness compared to standard of care
• Long-term efficacy demonstrated over 2.6 years of treatment

Negative

• None.

Insights

Pharmaceutical Clinical Development Expert

Monopar's ALXN1840 shows positive long-term efficacy and safety data for Wilson disease across multiple clinical endpoints, bolstering its development potential.

The late-breaking data presentation from Monopar provides compelling evidence for ALXN1840 (tiomolybdate choline) as a potential treatment for Wilson disease. The pooled analysis represents a substantial dataset with 255 patients for efficacy and 266 for safety, with median treatment durations exceeding 2.5 years - unusually robust for a rare disease study.

The multi-faceted efficacy profile is particularly noteworthy. ALXN1840 demonstrated sustained improvements in both patient-reported symptoms (UWDRS Part II) and clinician-assessed symptoms (UWDRS Part III). This dual validation strengthens confidence in the drug's clinical benefit, as does the reported superiority over standard of care on the Clinical Global Impression scale.

Mechanistically, the sustained increase in directly measured non-ceruloplasmin-bound copper (dNCC) confirms the drug's intended biological action of mobilizing copper. For Wilson disease, where toxic copper accumulation drives pathology, this pharmacodynamic marker strongly supports the treatment approach.

The improvement in the New Wilson Index is significant as it incorporates multiple objective laboratory measures including bilirubin, AST, INR, leukocytes, and albumin - suggesting ALXN1840 may provide measurable improvements in liver function parameters.

From a safety perspective, the fewer than 5% rate of drug-related serious adverse events represents a favorable profile, particularly with no renal or urinary system SAEs reported - important for a drug targeting copper metabolism where nephrotoxicity could be a theoretical concern.

Patient-reported convenience and effectiveness exceeding standard of care is particularly meaningful for this chronic condition requiring lifelong treatment. The positive experience among patients who transitioned from standard of care to ALXN1840 in the extension study further validates its potential clinical utility.

While the press release doesn't specify regulatory timelines, this comprehensive dataset spanning multiple trials and years of treatment provides substantial support for ALXN1840's clinical profile in this rare genetic disease with significant unmet needs.

WILMETTE, Ill., May 07, 2025 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, is presenting today data on the long term efficacy and safety of its ALXN1840 (tiomolybdate choline) drug candidate for Wilson disease at the European Association for the Study of the Liver (“EASL”) International Liver Congress 2025, one of the most prominent global conferences in liver disease. Monopar’s late-breaker poster presentation is available at the following link: https://www.monopartx.com/pipeline/ALXN1840/EASL-poster-may-2025.

The poster supports the potential use of ALXN1840 as a therapeutic option for Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised, leading to damage from toxic copper build-up in tissues and organs such as the liver and brain. Efficacy data were pooled and analyzed from three clinical trials: Phase 2 WTX101-201, Phase 2 ALXN1840-WD-205, and Phase 3 WTX101-301 (n=255). For safety analysis, data from the Phase 2 ALXN1840-WD-204 trial were also included (n=266). The median treatment duration with ALXN1840 was 961 days (2.63 years) and 943.5 days (2.58 years) for the efficacy and safety datasets, respectively. The data presented highlight the following:

• Sustained improvements from baseline in the Unified Wilson Disease Rating Scale (“UWDRS”) Part II (patient-reported symptoms) and Part III (clinician-assessed symptoms);
• Increased copper mobilization as evidenced by a sustained increase in dNCC (directly measured non-ceruloplasmin-bound copper);
• Improvements on the Clinical Global Impression – Improvement (“CGI-I”) scale for ALXN1840 compared to standard of care;
• Improvement in the New Wilson Index (based on bilirubin, AST, INR, leukocytes, and albumin) for patients treated with ALXN1840;
• Higher patient-reported convenience and effectiveness of ALXN1840 compared to standard of care, including those who transitioned from standard of care to ALXN1840 in the extension portion of the Phase 3 clinical trial; and
  
• Fewer than 5% of patients experienced a drug-related serious adverse event (“SAE”), with no cases of a drug-related renal or urinary system SAE.
  

“These data show that the long-term efficacy, safety, and convenience profile of ALXN1840 are very encouraging and that ALXN1840 has the potential to provide a meaningful benefit to Wilson disease patients’ daily lives,” said Dr. Karl Weiss, Medical Director of Salem Medical Center Heidelberg, and lead author of the presentation at EASL.

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 Monopar’s poster supports the potential use of ALXN1840 as a therapy for Wilson disease; and that ALXN1840 has the potential to provide a meaningful benefit to Wilson disease patients’ daily lives. 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 

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FAQ

What are the key findings from Monopar's ALXN1840 clinical trials for Wilson disease?

The trials showed sustained symptom improvements, increased copper mobilization, better results than standard care on the CGI-I scale, improved New Wilson Index scores, and fewer than 5% of patients experiencing drug-related serious adverse events.

How long was the treatment duration in MNPR's ALXN1840 clinical trials?

The median treatment duration was 961 days (2.63 years) for efficacy data and 943.5 days (2.58 years) for safety data analysis.

What is the safety profile of Monopar's ALXN1840 drug for Wilson disease?

ALXN1840 showed a strong safety profile with less than 5% of patients experiencing drug-related serious adverse events and no cases of drug-related renal or urinary system SAEs.

How many patients were included in Monopar's ALXN1840 clinical trials?

The trials included 255 patients for efficacy analysis across three clinical trials, and 266 patients for safety analysis including an additional Phase 2 trial.

How does ALXN1840 compare to standard of care for Wilson disease treatment?

ALXN1840 showed higher patient-reported convenience and effectiveness compared to standard of care, including improvements in patients who switched from standard care to ALXN1840 in the Phase 3 trial extension.