WTX101 is being investigated as a novel first-in-class copper-protein-binding agent with a unique mode of action, which, unlike current treatments for Wilson Disease, is designed to:
- provide an alternative copper-protein transport mechanism
- rapidly form copper-protein complexes with very high specificity for copper
- quickly de-toxify free copper both in the liver and blood, and
- promote biliary excretion of copper (the body’s natural route of elimination) to reduce copper overload.
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 has the 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. In addition, WTX101 has shown potential as a treatment for several other medical conditions including Amyotrophic Lateral Sclerosis (ALS). WTX101 has received US orphan drug designation for the treatment of ALS.