Clinical TrialJun 1, 2026, 07:02 AM
Monopar ALXN1840 Phase 2 Data Shows Liver, Neurologic, QoL Improvements
AI Summary
Monopar Therapeutics announced positive Phase 2 data for ALXN1840 (tiomolibdate choline) in treatment-experienced Wilson disease patients, presented at EASL Congress 2026. The trial showed stabilization or improvement in liver pathology in a high percentage of patients, alongside significant improvements in neurological symptoms, global clinical status, and quality of life. ALXN1840 was also generally well tolerated, reinforcing its potential to address unmet medical needs in this patient population.
Key Highlights
- ALXN1840 Phase 2 data presented at EASL 2026 for Wilson disease.
- Liver pathology stabilized or improved in 67-96% of patients by Week 48.
- Significant improvements in neurological symptoms (UWDRS Part III, p<0.05).
- Significant improvements in global clinical status (CGI, p<0.05).
- Significant improvements in patient-reported quality of life (EQ-5D, p<0.05).
- ALXN1840 was generally well tolerated with mostly nonserious, Grade 1 or 2 adverse events.
- No statistically significant change in hepatic copper concentration after 48 weeks.
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