STOCKHOLM, April 16, 2015 /PRNewswire/ — Wilson Therapeutics AB, a development stage biopharmaceutical company, today announced that a poster of the company’s ongoing Phase 2 clinical trial for WTX101-201 will be presented at the upcoming annual meeting of the European Association for the Study of the Liver (EASL), or the 50 th International Liver Congress 2015, taking place in Vienna April 22-26. The poster, titled “WTX101-201: Phase 2 Study of Bis-choline Tetrathiomolybdate in Newly Diagnosed Wilson Disease Patients,” will be presented in the Clinical Trials in Progress session on April 25.
WTX101-201 is a Phase 2 clinical trial evaluating the efficacy and safety of WTX101 using an individualized dosing regimen in up to 30 newly-diagnosed patients with Wilson Disease. The study is being conducted at sites in the U.S. and Europe, and will follow patients on WTX101 for six months.
“We are very excited to be involved in the WTX101-201 study, the first clinical trial to evaluate a new therapy for Wilson Disease in Europe for more than a decade,” said Karl-Heinz Weiss, MD, Associate Professor, Liver Cancer Center, University of Heidelberg. “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 is available for study participants.”
WTX101 is the proprietary bis-choline salt of tetrathiomolybdate, which has been evaluated for various indications in clinical studies involving over 500 patients, including 120 patients with Wilson Disease. Through its unique mechanism of action, tetrathiomolybdate has been shown to rapidly lower and control copper levels in both Wilson Disease and other patients. Previous data also suggest that tetrathiomolybdate may stabilize neurological function and reduce the risk of neurological deterioration after initiation of treatment in Wilson Disease patients with neurological involvement. WTX101 has been shown to lower and maintain copper levels with once or twice daily oral dosing. WTX101 has received orphan drug designation in both the United States and the European Union.
About Wilson Disease
Wilsons Disease is a rare autosomal recessive disorder caused by mutations in the ATP7B gene resulting in deficient production of the copper-transporter ATPase-2. Deficient or absent production of ATPase-2 leads to impaired incorporation of Cu into ceruloplasmin and impairment of biliary Cu excretion which allows copper to accumulate in the body. The build-up of copper in liver, brain, kidneys and other tissues of Wilson Disease patients produces significant organ damage and dysfunction. Untreated or inadequately treated disease can result in brain damage, liver failure and death. The overall prevalence of Wilson Disease is estimated to be 1-2 in 30,000.
About Wilson Therapeutics
Wilson Therapeutics is a privately-held biopharmaceutical company focused on improving the lives of patients with Wilson Disease through development of new treatment options and support for increased awareness and education about Wilson Disease. The Company’s lead product candidate, WTX101, is currently in clinical development as a treatment for Wilson Disease. Investors in Wilson Therapeutics include Abingworth, HealthCap, MVM Life Science Partners and Neomed. Visit www.wilsontherapeutics.com for more information.
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SOURCE Wilson Therapeutics