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Trade names | Decuprate |
Synonyms | Choline tetrathiomolybdate; ATN-224; WTX101 |
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Chemical and physical data | |
Formula | C10H28MoN2O2S4 |
Molar mass | 432.542 g/mol |
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Bis-choline tetrathiomolybdate (or tiomolibdate USAN, trade name Decuprate), is a salt of (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's 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 30,000 worldwide have Wilson's disease, corresponding to approximately 15,000 individuals in the European Union and approximately 11,000 in the United States.
Bis-choline tetrathiomolybdate has been evaluated in clinical trials in patients with various forms of cancer and has received orphan designation in the US and EU as a potential therapy against Wilson's disease.
Bis-choline tetrathiomolybdate is a de-coppering therapy in clinical development against Wilson's disease under the code name WTX101 by Wilson Therapeutics AB. Wilson Therapeutics was founded by HealthCap in 2012.
Tetrathiomolybdate has a unique mechanism of action through which it selectively forms highly stable complexes with copper and proteins. These complexes are then believed to be primarily excreted via the bile, restoring the normal excretion route of copper that is impaired in patients with Wilson's disease.
The binding and excretion mechanism of WTX101 is stable; whereas many de-coppering agents form unstable complexes that are excreted via urine.