Wilson Therapeutics announces that the ongoing Phase II study has been fully enrolled
Wilson Therapeutics AB (publ), announces that the company’s ongoing Phase II clinical trial evaluating the safety and efficacy of Decuprate® (bis-choline tetrathiomolybdate; WTX101) in Wilson Disease patients has been fully enrolled.
WTX101-201 is a Phase II clinical trial evaluating the efficacy and safety of Decuprate® monotherapy dosed once daily in newly-diagnosed patients with Wilson Disease, aged 18 years and older, who previously have been treated with a standard of care agent for up to two years,. The study is being conducted at 11 sites in the U.S. and Europe, and will follow patients on Decuprate® for 24 weeks. Patients completing the 24 weeks can elect to stay on Decuprate® in an extension phase of the study.
As of May 18, 28 patients have been enrolled in the study and six patients have reached the end of the 24-week treatment period. All six patients have elected into the extension phase. The patients recruited had various degrees of hepatic impairment at the time of enrollment and the majority of enrolled patients had neurological symptoms at study start.
"We are excited that the phase II study is now fully enrolled and that the trial is progressing according to plan,” says Carl Bjartmar, Chief Medical Officer of Wilson Therapeutics. "The preliminary data are encouraging as they seem to support the promise that Decuprate® could offer an efficacious and well tolerated treatment option with a lowered risk of an initial neurological worsening in patients with Wilson Disease.”
Michael Schilsky (MD, FAASLD), Associate Professor at Yale Medical Center and Director, Center of Excellence for Wilson Disease continues: “We are very pleased with the progression of this study. Although treatments for Wilson Disease have been available since the 1950’s, there is a clear need for novel therapies that can address the significant unmet medical needs. Currently we are seeking treatment that yields a higher initial response rate and a more benign side effect profile. The study is ongoing so the data are still preliminary, but they are promising as they indicate that Decuprate® may address these needs. With the possibility of once daily dosing that could improve patient convenience and adherence, a successful outcome for the trial should lead to significant improvements for Wilson Disease patients.”
“The full enrollment of the Phase II trial is a significant milestone in the development program,” says Jonas Hansson, Chief Executive Officer of Wilson Therapeutics. “We now look forward to the six-month data from all subjects. Shortly after we have received and analyzed the Phase II data we intend to go back to the regulatory authorities to discuss the Phase III program.”
About Decuprate® (bis-choline tetrathiomolybdate; WTX101)
Decuprate® is a first-in class de-coppering agent with a novel and unique mechanism of action. With its strong and selective binding to copper, Decuprate® has the potential to expand the body’s own liver buffering capacity for copper, which is saturated in patients with Wilson Disease. The active ingredient of Decuprate®, tetrathiomolybdate, has been tested in several clinical studies in Wilson Disease patients and the data from these studies, as well as preliminary data from the Company’s ongoing Phase II study, suggest that Decuprate® can rapidly lower and control toxic free copper levels and improve clinical symptoms in these patients. The data also suggest that Decuprate® is well-tolerated with the potential for a reduced risk of neurological worsening after initiation of therapy compared to existing therapies. Decuprate® is expected to have a once-daily dosing regimen which may potentially translate into to improved compliance in Wilson Disease patients, leading to fewer treatment failures and ultimately improved outcomes as a result. Decuprate® has received orphan drug designation for the treatment of Wilson disease in the US and EU.
About Wilson Disease
Wilson Disease is a rare genetic disease that causes serious copper poisoning. The genetic defect causes excessive copper accumulation, primarily in the liver and/or the central nervous system and the disease results in life-threatening damage to the liver and brain if left untreated. Wilson Disease affects approximately one in every 30,000 people worldwide, corresponding to a prevalence of approximately 10,000 patients in the US and 15,000 patients in the EU. The therapies currently being used in Wilson Disease were introduced in the 50’s and 60’s and since then there have been no new treatment options developed for patients with this disease.
About Wilson Therapeutics
Wilson Therapeutics is a biopharmaceutical company, based in Stockholm, Sweden, that develops novel therapies for patients with rare diseases such as Wilson Disease. Wilson Therapeutics’ lead product, Decuprate®, is initially being developed as a novel treatment for Wilson Disease and is currently being evaluated in a Phase II clinical study in Wilson Disease patients. Wilson Therapeutics is listed in the Mid Cap segment on Nasdaq Stockholm with the stock ticker WTX.
Visit www.wilsontherapeutics.com for more information.
For further information contact:
Jonas Hansson, CEO, Wilson Therapeutics AB
Telephone: +46 8 796 00 00
Wilson Therapeutics AB (publ)
Org nr 556893-0357
Västra Trädgårdsgatan 15
SE-111 53 Stockholm
The information in the press release is such that Wilson Therapeutics is required to disclose publicly in accordance with the Swedish Securities and Clearing Operations Act and/or the Swedish Financial Instruments Trading Act. The information was submitted for publication on May 18, 2016 at 8:00 a.m.