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  • 17 November 2014
    Wilson Therapeutics announces start of Phase 2 study to evaluate the efficacy and safety of WTX101 in newly diagnosed Wilson Disease patients

    Wilson Therapeutics AB, a development stage biopharmaceutical company, today announced the start of a Phase 2 clinical study to evaluate efficacy and safety of its lead product candidate WTX101 in Wilson Disease, a rare genetic disorder and designated orphan disease, in which the body is unable to process copper.

    "We are delighted to proceed with this important clinical study in Wilson Disease patients as a next step in the development program for WTX101", said David Clark, MD, Chief Medical Officer at Wilson Therapeutics. "The data from this study will provide additional insights in the optimal dosing of WTX101, and further inform our planning of the Phase 3 program, which is currently in the final stages of clinical design".

    The phase 2 clinical trial will evaluate the efficacy and safety of WTX101 using an individualized dosing regimen in up to 30 newly-diagnosed patients with Wilson Disease. The study will be conducted at sites in the U.S. and Europe, and will follow patients on WTX101 for six months.

    "We are very excited to offer people with Wilson Disease the opportunity to participate in the WTX101-201 study, the first clinical trial to evaluate a new therapy for Wilson Disease in this decade," said Frederick Askari, M.D, Ph.D. Associate Professor and Director of the Wilson Disease Program and Wilson Disease Center of Excellence, Department of Internal Medicine, Division of Gastroenterology, at the University of Michigan. "Based on earlier clinical studies with tetrathiomolybdate in people with neurologic Wilson Disease, WTX101 holds great promise to mitigate the neurologic damage from Wilson Disease, so we are delighted it will be available for study participants as soon as possible."

    Mary Graper, President of the Wilson Disease Association continued: "Although Wilson Disease has been recognized for more than one hundred years, it is still widely misunderstood. Because of this, many patients have a delayed diagnosis or a misdiagnosis causing disease progression, potentially irreversible organ damage, and even death. While there are currently several available treatments, there is a significant need for new treatment options such as WTX101 that could be of great benefit to patients with Wilson Disease."