Monopar’s ALXN1840 Achieves Neurologic Improvement (p=0.006) and Global Benefit (p<0.001) in Wilson Disease Trial
MNPR•ALXN1840 achieved significant neurologic improvement (UWDRS Part III p=0.006) and greater global clinical benefit (CGI-I p<0.001) versus standard therapy in 207 Wilson disease patients over 48 weeks in the Phase 3 FoCus trial. Safety data from 266 patients (median 2.58-year exposure) showed 4.9% SAEs, under 1% neurologic SAEs, no deaths.
1. FoCus Trial Analyses at EAN 2026
Monopar will present new analyses of the Phase 3 FoCus randomized controlled trial of ALXN1840 (tiomolibdate choline) at the European Academy of Neurology congress. The subset of 207 Wilson disease patients with neurologic symptoms at baseline was randomized 2:1 to ALXN1840 versus standard of care over 48 weeks.
2. Neurologic and Global Efficacy
ALXN1840 demonstrated significant neurologic improvement on the rater-blinded Unified Wilson Disease Rating Scale Part III (p=0.006) that continued over time versus p=0.435 for standard therapy. Global clinical improvement on the Clinical Global Impressions–Improvement scale at Week 48 was significantly greater with ALXN1840 (p<0.001).
3. Psychiatric, Hepatic and Safety Outcomes
ALXN1840 produced similar or superior improvements on psychiatric and hepatic measures at Week 48. Safety was evaluated in 266 patients (median 2.58-year treatment, up to 8 years exposure), with 4.9% drug-related SAEs, under 1% neurologic SAEs and no treatment-related deaths.
4. Next Steps Toward Approval
These results support Monopar’s planned New Drug Application submission for ALXN1840 to the U.S. FDA in mid-2026, advancing the therapy toward potential regulatory approval for neurologic Wilson disease patients.
