• 1 October 2017
    Promising preliminary long-term data from WTX101 Phase 2 extension study to be presented at AASLD Annual Meeting

    Wilson Therapeutics (publ) announced today that preliminary data from the ongoing extension part of the Phase 2 trial of WTX101 (bis-choline tetrathiomolybdate), an investigational oral first-in-class copper-protein binding agent for the treatment of patients with Wilson Disease, have been accepted for poster presentation at The Liver Meeting®, the annual meeting of the American Association for the Study of Liver Diseases (AASLD) in Washington DC, October 20-24, 2017.

    The abstract, published online today, highlights that the initial improvements seen after 24 weeks were continued. Free copper levels, hepatic function and neurologic status were maintained or further improved after treatment for 48 weeks with WTX101 during the extension study.

    All 22 patients who completed the 24-week open-label, single-arm, Phase 2 study opted to continue once-daily WTX101 treatment in the extension phase. The results at 48 weeks showed that copper levels remained controlled. Mean levels of non-ceruloplasmin bound copper (NCCcorrected) were further reduced from 0.9 µM to 0.5 µM between 24 to 48 weeks.

    Liver function parameters remained unchanged or improved at week 48 compared to week 24, indicating stability of liver function.

    Patients showed continued improvements in both patient-reported disability and neurological status. Disease related patient-reported disability measured by the Unified Wilson Disease Rating Scale (UWDRS) Part II further improved from mean 4.1 to 2.1 between week 24 and week 48 and neurological status assessed by UWDRS Part III further improved from mean 16.6 to 12.5.

    WTX101 was generally well tolerated. Reversible liver enzyme elevations that were observed in about one-third of patients in the Phase 2 trial were not observed in the extension period.

    Accepted abstracts are published today on the AASLD website at