Final Phase 2 results for WTX101 accepted as a late-breaker presentation at EASL Annual Meeting

Apr 05, 2017

Wilson Therapeutics AB (publ), announced today that the final data from the company’s Phase 2 trial of WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper modulating agent in development for patients with Wilson Disease, has been accepted as a late-breaker abstract for oral presentation at The International Liver Congress™ 2017, the Annual Meeting of the European Association for the Study of the Liver (EASL), in Amsterdam, the Netherlands, 19 – 23 April, 2017.

Oral Presentation summary details:

Abstract identifier: GS-016
Title
: “WTX101 in patients newly diagnosed with Wilson Disease: final results of a global, prospective phase 2 trial”
Presenter
: Karl Heinz Weiss, MD, Professor, University of Heidelberg, Germany
Session
: General session III and Award Ceremony II
Date
: Saturday, 22 April
Time
: 11:30 to 11:45 CET
Location
: RAI Amsterdam, Hall 5, Amsterdam, the Netherlands

Abstracts for the EASL meeting are now available on the ILC 2017 website (https://ilc-congress.eu).

About WTX101 (bis-choline tetrathiomolybdate)
WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper modulating agent with a unique mechanism of action, under investigation as a novel therapy for Wilson Disease. WTX101, unlike current treatments for Wilson Disease, exhibits a specific copper buffering activity and acts in the liver where it removes copper from the overloaded copper buffer. WTX101 also rapidly neutralizes toxic free copper in tissue and blood by forming complexes with excess copper and albumin. The excess copper is excreted via the bile, the body’s natural route for excess copper elimination.

A Phase 2 study evaluating the efficacy and safety of WTX101 in Wilson Disease patients was successfully completed in 2016. In addition, the active ingredient of WTX101, tetrathiomolybdate, has been tested in several previous clinical studies in Wilson Disease patients. The data from these studies suggest that WTX101 can rapidly lower and control toxic free copper levels and improve clinical symptoms in these patients. The data also suggest that WTX101 is generally well-tolerated and may have potential for a reduced risk of neurological worsening after initiation of therapy. WTX101 is expected to have a once-daily dosing regimen which may potentially translate into improved compliance in Wilson Disease patients, leading to fewer treatment failures and ultimately improved outcomes as a result. WTX101 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 disorder of impaired copper metabolism that causes serious copper poisoning. The genetic defect severely affects the body´s ability to regulate copper balance, resulting in life-threatening damage to the liver, the brain and further organs 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 1950’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. Wilson Therapeutics’ lead product, WTX101, is in development as a novel treatment for Wilson Disease. A Phase 2 clinical study has been successfully completed and preparations for a pivotal Phase 3 study are ongoing. 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
Email: jonas.hansson@wtx.se

Wilson Therapeutics AB (publ)
Org nr 556893-0357
Kungsgatan 3
SE-111 43 Stockholm

The information in the press release is information that Wilson Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 10:00 CET on April 5, 2017.