Clinical Trials

Date: 2017-02-16

Type of information: Recruitment of the first patient

phase: 3

Announcement: recruitment of the first patient

Company: Wilson Therapeutics (Sweden)

Product: WTX101 (bis-choline tetrathiomolybdate)

Action mechanism:

copper-protein-binding agent. WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper-protein-binding agent. It is formulated as an enteric coated tablet (15 mg strength) for oral administration. WTX101 provides an alternative copper-protein binding mechanism by forming a tripartite complex with copper and albumin. WTX101 thereby detoxifies excess copper in both liver and blood, and promotes copper clearance through biliary excretion (the body’s natural route of elimination).
A Phase 2 study evaluating the efficacy and safety of WTX101 in patients with Wilson Disease has successfully been completed. In addition, the active moiety of WTX101, tetrathiomolybdate, has been tested in several previous clinical studies in Wilson Disease patients. The data from these studies suggest that WTX101 can reduce and control free copper levels and improve symptoms and associated disabilities. The data also suggest that WTX101 is generally well tolerated with a low risk of drug-induced neurological worsening. The tolerability profile and the expected once-daily dosing regimen have the potential to improve compliance in Wilson Disease patients, leading to fewer treatment failures and ultimately improved outcomes. WTX101 has received Fast Track designation in the US and orphan drug designation for the treatment of Wilson Disease in the US and EU.

Disease: Wilson disease

Therapeutic area: Rare diseases - Metabolic diseases

Country: Austria,Czechia,France,Germany,Hungary,Israel,Italy,Poland,Spain,UK,USA

Trial details:

Wilson Disease (WD) is an autosomal recessive disorder of impaired copper (CU) transport caused by mutations in the ATP7B gene. FOCuS is a randomized, controlled, rater-blinded, multi-center study that will enroll approximately 100 Wilson Disease patients, aged 18 years or over, to receive once-daily WTX101 or standard of care. The primary endpoint will be copper control assessed as the percentage change in free copper levels in blood from baseline to 48 weeks. The purpose of this study is to evaluate the efficacy of WTX101 administered for 48 weeks compared to standard of care (SOC) in WD subjects aged 18 and older. The study will be conducted at approximately 30 sites in the US, EU and Israel. Patients completing the Phase 3 study through 48 weeks will be offered continued WTX101 treatment in an extension phase. (NCT03403205)

Latest news:

• On February 16, 2018, Wilson Therapeutics announced that the first patient has been enrolled in the pivotal Phase 3 FOCuS clinical trial evaluating WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper-protein-binding agent, for the treatment of Wilson Disease. Top-line data from the study is expected to be released H2 2019.
The first patient was enrolled at the University of Michigan.