Wilson Therapeutics has enrolled the first patient in the FOCuS Phase III trial to evaluate WTX101 (bis-choline tetrathiomolybdate) in comparison with standard of care (SoC) for treatment of Wilson disease.
The first patient has been enrolled at the University of Michigan, US.
FOCuS is a randomised, controlled, rater-blinded, multi-centre study that will enrol around 100 patients 18 years and older with hepatic and/or neurological symptoms, who are treatment naive, or have previously received SoC therapy.
During the trial, patients will be randomised in a 2:1 ratio to receive once-daily WTX101 or SoC.
The trial’s primary object will be copper control evaluated as the percentage change in free copper levels in blood from baseline to 48 weeks.
Additional endpoints will feature clinical such as hepatology, neurology, disease-related disability, psychiatry, and quality of life-related endpoints, as well as safety of WTX101.
Around 30 sites in the US, EU, and Israel have been selected to conduct the trial, which expects to provide top-line data next year.
The patients will complete the FOCuS trial through 48 weeks and are set to receive continued WTX101 treatment in an extension phase.
Wilson Therapeutics chief medical officer Carl Bjartmar said: “Through its novel mode of action with its high affinity and specificity to copper, WTX101 detoxifies excess copper in the liver and in the blood by forming stable tripartite complexes with copper and albumin that are then cleared through bile, the natural elimination route of copper.
“This unique approach to copper control has the potential to improve symptoms and associated disabilities in Wilson Disease patients, which was demonstrated in our successful Phase II trial.”
Wilson Disease is a rare genetic condition of impaired copper transport and excretion, which is caused by loss of function of the ATP7B copper-binding protein.