Bis-choline tetrathiomolybdate, or WTX101, is a salt of tetrathiomolybdate (TTM, MoS42−) and choline currently under investigation as a therapy against Wilson's disease, a rare and potentially fatal disease in which the body cannot regulate copper. Wilson disease is an autosomal recessive genetic disorder that is manifested by serious hepatic, neurologic or psychiatric symptoms. The disease is fatal if left undiagnosed and untreated. It is estimated that approximately 1 individual in every 15,000 worldwide have Wilson's disease, corresponding to approximately 30,000 individuals in the European Union and approximately 20,000 in the United States.
WTX101-201 is a Phase 2 clinical trial evaluating the efficacy and safety of WTX101 using an individualized dosing regimen in up to 30 newly-diagnosed patients with Wilson Disease. The study is being conducted at sites in the U.S. and Europe, and will follow patients on WTX101 for six months.
"We are very excited to be involved in the WTX101-201 study, the first clinical trial to evaluate a new therapy for Wilson Disease in Europe for more than a decade," said Karl-Heinz Weiss, MD, Associate Professor, Liver Cancer Center, University of Heidelberg. "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 is available for study participants."