ALXN1840

Monopar Therapeutics presented Phase 2 data on ALXN1840 for Wilson disease at AASLD 2025. The study (n=8) demonstrated a significant reduction in mean daily copper balance, driven by increased fecal copper excretion, in patients treated with ALXN1840 compared to baseline. Improvement was observed during both initial and sustained dosing periods. While promising, the small sample size warrants further investigation. Presentation materials and the abstract are available on Monopar’s and AASLD’s websites, respectively.

Monopar Therapeutics will present long-term data on ALXN1840 for Wilson disease at the ANA Annual Meeting on September 14-15, 2025. The pooled analysis of clinical trials (n=255 efficacy, n=266 safety) with a median 2.6-year treatment duration showed sustained neurological and psychiatric improvements. Patients switching from standard of care to ALXN1840 also demonstrated improvement. The drug exhibited a favorable safety profile. ALXN1840 offers a novel mechanism of action by directly binding copper, potentially preventing its accumulation.