Wilson Therapeutics starts Phase III of WTX101 for Wilson's disease

Wilson Therapeutics AB (SSE:WTX) began the Phase III FOCuS trial of decuprate bis-choline tetrathiomolybdate (WTX101) to treat Wilson's disease in patients ages 18 and older. Top-line data from the trial are expected 2H19.

The single-blind, international trial will enroll about 100 patients. The primary endpoint is non-inferiority of WTX101 to standard of care on copper control as measured by the percentage change in free copper levels in blood from baseline to 48 weeks. If non-inferiority is met, then superiority on the endpoint will be assessed. Secondary endpoints include Model for End-Stage Liver Disease (MELD), Unified Wilson’s disease Rating Scale (UWDRS) Parts II and III, Clinical Global Impression-Improvement Scale (CGI-I) and Clinical Global Impression-Severity Scale (CGI-S) scores and safety...