Monopar Therapeutics Presents New Data on ALXN1840 for Wilson Disease at ANA Meeting

Strategic Insights -

Data from three independent clinical trials and a fourth independent clinical trial show sustained neurological improvement, consistency across trials, and a favorable safety profile for ALXN1840 in patients with Wilson disease. The new data presented at the American Neurological Association (ANA) meeting highlight the long-term neurological benefit of ALXN1840 and underscore its potential for both the neurological and hepatic manifestations of the disease.

Key Takeaways:

  • Statistically significant neurological 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 in patients treated with ALXN1840.
  • 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.
  • Statistically significant psychiatric improvement from baseline was sustained over multiple years, as measured by the Brief Psychiatric Rating Scale (BPRS).
  • Neurological benefit was observed consistently across multiple independent studies, with favorable safety data showing less than 1% of patients experiencing a drug-related neurological serious adverse event (SAE) over more than 645 patient-years.
  • The analysis pooled efficacy outcomes from three independent clinical trials (n=255), while safety data included a fourth independent clinical trial (n=266).

Statistics:

  • 255 patients were included in the efficacy analysis, with a median treatment duration of approximately 2.6 years.
  • 266 patients were included in the safety analysis, with a median treatment duration of approximately 2.6 years.
  • Less than 1% of patients experienced a drug-related neurological serious adverse event (SAE) over more than 645 patient-years on ALXN1840.
  • Statistically significant neurological improvement was sustained over 6 years in patients treated with ALXN1840.

Sources:

  • Globe Newswire, "Monopar Therapeutics Presents New Data on ALXN1840 for Wilson Disease at ANA Meeting" (September 14, 2025)
  • Monopar Therapeutics, Inc. website, "About Monopar Therapeutics" (no date provided)