Monopar’s Phase 2 ALXN1840 Study Shows 96% Necrosis Stabilization and 67% Fibrosis Improvement
MNPR•Monopar’s open-label Phase 2 trial of ALXN1840 in 29 heavily pre-treated Wilson disease patients (median 13.8 years prior therapy) showed 96% stabilized or improved hepatocyte necrosis and 67% improved fibrosis stage at Week 48. Neurological, Clinical Global Impressions and EuroQoL quality-of-life scores improved (p<0.05) with mostly Grade 1–2 adverse events.
1. Trial Design
Monopar enrolled 29 Wilson disease patients with a median 13.8 years of prior standard therapy into an open-label, multicenter, pathologist-blinded Phase 2 trial of ALXN1840 monotherapy over 48 weeks, with an optional 48-week extension. Liver biopsies at baseline and Week 48 assessed hepatic copper concentration, fibrosis stage, steatosis grade and inflammation.
2. Histologic Outcomes
Among 24 patients with paired biopsies, 96% showed stabilization or improvement in hepatocyte necrosis and 88% improved steatosis grade. Lobular inflammation improved in 79%, portal inflammation in 71%, NAFLD Activity Score in 71%, hepatocellular ballooning in 75% and fibrosis stage in 67%.
3. Clinical and Safety Results
Patients experienced significant improvements in UWDRS Part III, Clinical Global Impressions and EQ-5D quality-of-life scores (p<0.05 versus baseline). ALXN1840 was generally well tolerated, with most treatment-emergent adverse events graded 1–2 and no new safety signals emerging through Week 48.
