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STOCKHOLM, November 17, 2014 (PR NEWSWIRE) – 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 for 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.”

About WTX101
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 f 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 one to two 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 for more information.


Jonas Hansson
+46 70 944 18 77

Ami Knoefler
Spark BioComm LLC
+ 1 650 739 9952

Source: Wilson Therapeutics