• 20 October 2017
    Promising preliminary long-term data for WTX101 in Wilson Disease highlighted at The Liver Meeting®

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

    The poster will be presented by Professor Michael Schilsky, Director of the Center for Excellence for Wilson Disease at Yale University Medical Center, tomorrow October 21 at 17:30-19:30 ET.

    Professor Michael Schilsky commented: “The data from the Phase 2 extension study are impressive. I am very excited to see that the positive effects of WTX101 are sustained or further improved over time, particularly its meaningful impact on patient-reported disability and neurological status. Wilson Disease is a serious life-long disorder and it is vital that new therapies that are effective, safe and easy to use are developed – it is highly encouraging to see such a profile emerging with WTX101.”

    Jonas Hansson, Chief Executive Officer of Wilson Therapeutics added: “These promising preliminary results build on the strong data we have observed in the 24-week core study, indicating that WTX101 has the potential to provide both fast and sustained copper control as well as long term improvements of symptoms. In addition, WTX101 has generally been well tolerated and can be dosed once daily. All in all, this further supports our belief that WTX101 has the potential to be an important novel drug for patients with Wilson Disease.”

    As previously announced when the abstract was published October 1 2017, patients taking WTX101 for up to 48 weeks continued to experience improvements in key aspects of their disease including neurological function and disability. Additionally, copper control and hepatic function were maintained or further improved with longer-term treatment.

    All 22 patients who completed the 24-week open-label, single arm, Phase 2 study opted to continue once-daily WTX101 treatment in the ongoing 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 at 24 weeks to 0.5 µM at 48 weeks.

    Liver function parameters (INR, MELD score etc.) 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 disability measured by the Unified Wilson Disease Rating Scale (UWDRS) Part II further improved from mean 4.1 to 2.2 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 core period of the Phase 2 trial were not observed in the extension period.