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  • 8 June 2017
    Wilson Therapeutics presents Phase 2 Data for WTX101 at MDS Meeting

    Wilson Therapeutics AB (publ), announced today a poster presentation at the International Congress of Parkinson’s Disease and Movement Disorders in Vancouver, BC, 4-8 June, 2017. The presentation highlighted results from the recently completed Phase 2 trial of WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper-protein binding agent with a unique mechanism of action for the treatment of patients with Wilson Disease. The poster was presented by Danny Bega, MD, Assistant Professor of Neurology at Northwestern University.

    WTX101-201 was a 24-week open-label Phase 2 study evaluating the efficacy and safety of WTX101 monotherapy in 28 newly-diagnosed adult 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. As previously reported, the Phase 2 study met its primary endpoint of copper control (p< 0.001) and significant improvements from baseline to week 24 were observed for both patient-reported disability (p< 0.001) and rater-assessed neurological status (p< 0.0001), measured using the Unified Wilson Disease Rating Scale (UWDRS) Part 2 and 3 respectively.

    Treatment with WTX101 was generally well-tolerated with most reported adverse events being mild (grade 1) to moderate (grade 2). Importantly, no early neurological worsening upon initiation of WTX101 was observed.

    “It was very exciting to participate in this study which was the first clinical trial with a new investigational drug for Wilson Disease in several decades. We are very encouraged by the results as we saw a fast and strong effect on neurological symptoms without any cases of definitive drug-induced worsening of neurological symptoms, which is a potentially devastating side effect that may occur during the initial treatment phase with the existing therapies,” said Danny Bega, MD, Assistant Professor of Neurology at Northwestern University.

    “Furthermore, WTX101 can be dosed once daily and was generally well-tolerated so it clearly has the potential to become a very valuable treatment option for patients with Wilson Disease”.