Monopar Therapeutics Reports Promising Long-Term Data for ALXN1840 in Wilson Disease

Monopar Therapeutics presents encouraging long-term efficacy and safety results for ALXN1840 in treating Wilson disease at EASL 2025.

Key Findings from Recent Trials

At the European Association for the Study of the Liver International Liver Congress 2025, Monopar Therapeutics Inc. revealed new findings regarding its drug candidate ALXN1840 (tiomolybdate choline) for Wilson disease. This rare genetic disorder involves the accumulation of excess copper in the body. Data from three clinical trials demonstrate ALXN1840’s potential long-term efficacy and safety. Notable outcomes include significant improvements in both patient-reported and clinician-assessed symptoms, enhanced copper mobilization, and increased patient-reported convenience over standard treatments. Additionally, serious adverse events were low, highlighting its safety profile. Dr. Karl Weiss, the lead author, expressed optimism about ALXN1840’s prospects for patient care.

Advantages of ALXN1840

• The presentation of long-term data at a significant medical conference bolsters Monopar Therapeutics’ credibility in the biopharmaceutical field.
• Results indicate sustained symptom improvements for patients, suggesting ALXN1840’s potential effectiveness as a treatment option.
• The reduced frequency of serious adverse events compared to standard care enhances the drug’s safety profile, supporting its acceptance and potential regulatory approval.

Challenges and Concerns

• Monopar’s reliance on forward-looking statements introduces uncertainties regarding ALXN1840’s actual outcomes, which could affect investor confidence.
• The necessity for funding to support ongoing development and marketing raises concerns about financial stability and operational sustainability.
• Regulatory approval and market acceptance remain uncertain, representing significant risks that may impact future profitability in the competitive biopharmaceutical market.

Frequently Asked Questions

What is ALXN1840 used for?

ALXN1840 is under development as a treatment for Wilson disease, a genetic condition linked to copper metabolism issues.

Where was the latest data on ALXN1840 presented?

The recent findings were disclosed at the European Association for the Study of the Liver (EASL) International Liver Congress 2025.

What were the key findings about ALXN1840’s efficacy?

Data indicated sustained improvements in symptoms associated with Wilson disease and enhanced patient convenience compared to existing care options.

How long was the treatment duration in the studies?

The median treatment duration for patients was approximately 961 days for efficacy evaluations and 943.5 days for safety assessments.

What safety concerns are associated with ALXN1840?

Fewer than 5% of patients reported serious drug-related adverse events, and there were no incidents of renal or urinary system complications.

Disclaimer: This summary reflects a press release distributed by GlobeNewswire, which may contain errors. For complete details, please refer to the full release.

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Full Release

WILMETTE, Ill., May 07, 2025 — Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on innovative treatments for unmet medical needs, is sharing data on the long-term efficacy and safety of its drug candidate ALXN1840 (tiomolybdate choline) for Wilson disease at the European Association for the Study of the Liver (“EASL”) International Liver Congress 2025. This influential conference addresses liver disease globally. Monopar’s late-breaking poster presentation can be accessed here.

The poster highlights ALXN1840’s potential as a therapeutic option for Wilson disease, a progressive genetic disorder impeding excess copper removal, resulting in toxic accumulation in organs like the liver and brain. Efficacy data were analyzed from three clinical trials: Phase 2 WTX101-201, Phase 2 ALXN1840-WD-205, and Phase 3 WTX101-301 (n=255). Safety analysis included data from the Phase 2 ALXN1840-WD-204 trial (n=266). The median treatment duration was 961 days (approximately 2.63 years) for efficacy and 943.5 days (approximately 2.58 years) for safety assessments. Presented data included:

• Sustained improvements in the Unified Wilson Disease Rating Scale (“UWDRS”) Part II (patient-reported symptoms) and Part III (clinician-assessed symptoms).
• Increased copper mobilization shown by sustained dNCC (directly measured non-ceruloplasmin-bound copper) increases.
• Notable improvements on the Clinical Global Impression – Improvement (“CGI-I”) scale for ALXN1840 compared to standard care.
• Enhanced performance in the New Wilson Index (based on bilirubin, AST, INR, leukocytes, and albumin) for patients treated with ALXN1840.
• Higher patient-reported convenience and effectiveness compared to standard treatments, including those transitioning from standard care to ALXN1840 during the Phase 3 clinical trial extension.
• Fewer than 5% of patients experienced serious drug-related adverse events, with no reports of renal or urinary system issues.