• 5 December 2016
    Wilson Therapeutics Announces that WTX101 Meets the Primary Endpoint in Phase 2 Study in Wilson Disease

    Wilson Therapeutics AB (publ), announced today that the Phase 2 study of WTX101 (bis-choline tetrathiomolybdate; Decuprate®), an investigational first in class copper modulating agent with a unique mode of action for the treatment of patients with Wilson Disease, met its primary endpoint. In the ITT population, 79% of patients achieved or maintained normalized levels of free copper, or experienced a reduction of at least 25% in free copper in blood from baseline, after 24 weeks of treatment with WTX101.

    The Phase 2 study WTX101-201 was a 24-week open-label multicenter study evaluating the efficacy and safety of WTX101 monotherapy in 28 newly-diagnosed patients with Wilson Disease who had received either no prior treatment for Wilson Disease or a standard of care agent for up to two years. All patients who successfully completed the 24-week study period elected to stay on WTX101 in an extension phase of the study.

    In the ITT population, WTX101 monotherapy reduced mean serum free copper by 77% (p<0.0001) at week 24 compared to baseline. WTX101 treatment also resulted in improvements in neurological status (p<0.0001) and disability (p<0.001) measured as a change from baseline in Unified Wilson Disease Rating Scale (UWDRS) Part 3 and Part 2 respectively. In addition, liver status, as measured by the Modified Nazer Score, was stabilized or improved in the majority of patients.

    Treatment with WTX101 was generally well tolerated with most reported adverse events being mild (grade 1) to moderate (grade 2). Reversible liver test elevations were observed in 39% of patients and these elevations were generally mild to moderate, asymptomatic and normalized with dose adjustments. No initial drug-induced neurological worsening was observed upon treatment initiation with WTX101. In total, six patients discontinued the study, including three related to adverse events. Detailed data from the WTX101-201 study will be presented at upcoming medical meetings.

    “Final data from the Phase 2 study demonstrate that WTX101 has the potential to rapidly lower and control free copper, and improve or stabilize neurological and liver status in patients with Wilson Disease. We are also very pleased the treatment improved the patients’ self-reported disability and that we have not observed any case of initial neurological worsening upon initiation of WTX101. Additionally, the data continue to show that WTX101 is generally well-tolerated with few patients reporting GI intolerance or skin reactions. We believe that this therapeutic profile, with the convenience of once-daily dosing, could translate into improved treatment adherence and outcomes”, commented Carl Bjartmar, MD, PhD, Chief Medical Officer of Wilson Therapeutics.

    “We are extremely pleased with the results from this Phase 2 study. We believe that WTX101 may have the potential to improve treatment options for patients with Wilson Disease, a life-long disease for which there has been no innovation for decades. We will now focus on agreeing the final Phase 3 program with relevant regulatory authorities, and expect to initiate a pivotal Phase 3 study in 2017”, said Jonas Hansson, Chief Executive Officer of Wilson Therapeutics.