Key findings presented at EAN 2026:

In the subset of patients with neurologic symptoms at baseline from the 2:1 randomized Phase 3 FoCus clinical trial (NCT03403205; n=207), ALXN1840 demonstrated improved outcomes compared to SoC across multiple clinical measures:

  • Neurologic improvement on the rater-blinded, physician-assessed Unified Wilson Disease Rating Scale (UWDRS) Part III was significant and continued over time with ALXN1840 (p=0.006) but not with SoC (p=0.435).
  • Global clinical improvement as assessed by the Clinical Global Impressions – Improvement (CGI-I) scale at Week 48 was significantly greater with ALXN1840 than with SoC (p<0.001).
  • A greater proportion of patients treated with ALXN1840 achieved improvement on the rater-blinded UWDRS Part III at Week 48 compared with SoC, with consistent results observed across multiple improvement thresholds.
  • ALXN1840 also produced similar or greater improvement than SoC at Week 48 across psychiatric and hepatic measures.



     

Across Phase 2 and Phase 3 studies, ALXN1840 has demonstrated a well-characterized and favorable safety profile in 266 patients, with a median treatment duration of 2.58 years and maximum exposure of more than 8 years. Drug-related serious adverse events (SAEs) occurred in 4.9% of patients, including neurologic SAEs in less than 1% and no treatment-related deaths.

"For Wilson disease patients with neurologic symptoms, meaningful improvement can be difficult to achieve with existing therapies, and some patients experience severe paradoxical worsening," said Dr. Poujois. "These new analyses from the Phase 3 FoCus trial are encouraging because they show that ALXN1840 treatment was associated with continued neurologic improvement over time and greater global clinical benefit compared with standard of care."