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  • 11 November 2016
    Wilson Therapeutics Presents Updated Preliminary Clinical Data on WTX101 At The AASLD Liver Meeting

    Wilson Therapeutics AB (publ), today announced that updated preliminary data from the company’s ongoing Phase 2 clinical trial for WTX101 (bis-choline tetrathiomolybdate; Decuprate®) in Wilson Disease will be presented at the Liver Meeting®, organized by the American Association for the Study of Liver Diseases (AASLD), in Boston, Massachusetts, November 11-15.

    The preliminary data continue to show that once-daily dosing with WTX101 reduces serum free copper and improves hepatic and neurologic status in patients with Wilson Disease. The poster presentation will take place today, and will be given by Michael Schilsky (MD, FAASLD), Associate Professor at Yale Medical Center and Director, Center of Excellence for Wilson Disease.

    “Wilson Disease is a rare genetic disorder of impaired copper metabolism that causes serious copper poisoning. Although treatments have been available since the 1950’s, there is a significant need for new therapies that can rapidly control free copper, yield a higher initial response rate, have a more benign safety profile and offer a simplified dosing regimen,” said Dr. Schilsky. “The study is still ongoing so the data presented today are preliminary, but we are very encouraged as they indicate that WTX101 may have the potential to address these needs. With the possibility of once daily dosing that could improve patient convenience and adherence, a successful outcome for the trial could lead to significant improvements for Wilson Disease patients.”

    WTX101-201 is an open-label Phase 2 clinical trial evaluating the efficacy and safety of once-daily WTX101 monotherapy in 28 newly-diagnosed patients with Wilson Disease, aged 18 years and older, who have received either no prior treatment for Wilson Disease or a standard of care agent for up to two years. The study is being conducted at 11 sites in the U.S. and Europe, and follows patients on WTX101 for 24 weeks. Patients completing the 24-week study period can elect to stay on WTX101 in an extension phase of the study.

    The poster presents preliminary data up to 24 weeks. As of October 5, the cut-off date for the current analysis, all 18 patients who had reached the end of the 24-week study period had elected to continue WTX101 treatment in the extension phase. Patients recruited in the study had various degrees of hepatic impairment at the time of enrollment and the majority also had neurological symptoms at study start.

    The presented preliminary data show that treatment with WTX101 leads to rapid control of free copper (Cu), with average levels reduced to and below the upper limit of normal after eight weeks. Free copper in blood was measured as non-ceruloplasmin bound copper, corrected for the amount of copper bound to tripartite tetrathiomolybdate-Cu-albumin complexes in blood.

    Preliminary data also show that liver status, measured using the Modified Nazer Score, improved or remained stable in 22 out of 27 evaluable patients at the last observation available in the 24-week study period compared to baseline. Three of the five patients who did not improve or remain stable during the 24-week period, stabilized or improved when treated for a longer period during the extension phase.

    Neurological impairment, measured as mean Unified Wilson Disease Rating Scale Part 3 score, a neurology scale specific for Wilson Disease, also improved over the course of therapy.

    Treatment with WTX101 was generally well tolerated and no drug-induced neurological worsening was observed upon treatment initiation. Most reported adverse events were mild (grade 1) to moderate (grade 2). Reversible liver test elevations were observed in approximately 1/3 of patients and these elevations were generally mild to moderate, asymptomatic and normalized with dose adjustments.

    “In clinical practice, treatment of Wilson Disease patients with neurological symptoms is a particular concern, as approximately 25% of these patients experience a significant drug-induced worsening of neurological symptoms upon initiation of treatment with current therapies,” said Carl Bjartmar, MD, PhD, Chief Medical Officer of Wilson Therapeutics. “The preliminary data from our ongoing trial is therefore very encouraging as we so far have not seen any cases of this initial drug-induced worsening.”

    “We are very pleased with the progression of this study and we look forward to reporting topline data from the study later this year”, says Jonas Hansson, Chief Executive Officer of Wilson Therapeutics. “With final Phase 2 data we expect to go back to the regulatory authorities to discuss and agree on the pivotal Phase 3 program.”