Clinical Efficacy And SafetyPositive late‑stage trial results showing meaningful copper mobilization and sustained neurological improvement alongside a favorable safety profile could increase the probability of regulatory approval and clinician interest.
Differentiation And Commercial OpportunityOnce‑daily oral dosing and a mechanism distinct from traditional chelators, combined with a multi‑billion dollar addressable market for Wilson disease, could drive significant commercial upside if regulatory acceptance and physician uptake occur.
Regulatory Data And AnalysisA comprehensive re‑analysis demonstrating net‑negative copper balance through increased fecal excretion, supported by new animal data, directly addresses earlier methodological concerns and strengthens the case for an NDA submission.