Wilson reports Phase II extension data for Wilson’s disease candidate

Wilson Therapeutics AB (SSE:WTX) reported data from 22 patients with Wilson’s disease in the extension portion of the 24-week Phase II WTX101-201 trial showing that once-daily oral Decuprate bis-choline tetrathiomolybdate (WTX101) controlled mean nonceruloplasmin-bound copper (NCC) levels through week 48 (0.9 µm at week 24 and 0.5 µm at week 28). Additionally, Decuprate improved or maintained albumin levels, alanine aminotransferase (ALT) levels and Model for End-Stage Liver Disease (MELD) scores at week 48 compared to week 24, which the company said indicates stability of liver function. WTX101 also led to continued improvements in Unified Wilson Disease Rating Scale (UWDRS) disability and neurologic scores at week 48 compared to week 24. Data were presented in an abstract released ahead of the American Association for the Study of Liver Diseases meeting in Washington, D.C., later this month.

WTX101-201 enrolled patients who received no prior Wilson’s disease treatments or who were treated for ≤24 months prior to enrollment with chelation therapy, zinc therapy or combination therapy to receive individualized dosing with Decuprate. In April, Wilson reported data from 28 patients in WTX101-201 showing that 71% of patients who received Decuprate achieved or maintained normalized NCC levels or had a ≥25% NCC reduction after 24 weeks compared to baseline, meeting the primary endpoint (p<0.001) (see BioCentury, May 9, 2016; Dec. 9, 2016 & April 27, 2017)...