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  • 6 October 2017
    Phase 2 Clinical Trial Data for WTX101 Published in The Lancet Gastroenterology & Hepatology

    Wilson Therapeutics (publ.) today announced that data from a Phase 2 trial of WTX101 (bischoline tetrathiomolybdate), an investigational oral first-in-class copper-protein-binding agent for the treatment of patients with Wilson Disease, has been published online in The Lancet Gastroenterology & Hepatology and will be available in print in the December issue. The results show that treatment with WTX101 induces rapid copper control, significantly reducing free copper levels by 72% from baseline after approximately 3 months, and is associated with significant early improvements in neurological symptoms and disability. The study data have previously been reported at medical conferences but this is the first time the complete study results are available in a peer-reviewed scientific journal.

    “This is the first global prospective multi-center trial conducted in Wilson Disease and I am especially encouraged by the rapid and clinically meaningful improvement of disease related symptoms, and the apparent lack of paradoxical neurological worsening after WTX101 treatment initiation, an adverse effect that unfortunately is seen in some patients when starting chelators. Furthermore, the once-daily dosing of WTX101 can possibly minimize the treatment burden, leading to better long-term compliance and treatment outcomes. All Wilson Disease patients need life-long therapy but current treatment options have significant limitations. WTX101 has the potential to bring new hope to patients and their caregivers”, said Prof. Karl Heinz Weiss, University Hospital of Heidelberg, Germany, a principal investigator in the trial and lead author of the publication.

    “Patients with Wilson Disease can be severely affected by hepatic, neurologic and psychiatric symptoms. The main treatment goal is to quickly remove and detoxify excess copper in patients without worsening their disease, and then to maintain copper control. The rapid biochemical and clinical improvements seen with WTX101 are possibly related to its novel, copper-specific mechanism of action which acts directly in the liver and lowers toxic free copper levels in the blood. I am very excited about the results achieved so far as it provides further evidence of the potential of WTX101 as a novel and effective treatment for patients with Wilson Disease”, said Michael Schilsky, MD, Professor of Medicine and Surgery at Yale University, USA, also a principal investigator in the trial and lead author of the publication.