WTX101

WTX101 is a first in class copper modulating agent with a unique mechanism of action, under investigation as a novel therapy for Wilson Disease. WTX101, unlike current treatments for Wilson Disease, exhibits a specific copper buffering activity and acts in the liver where it removes copper from the overloaded copper buffer. WTX101 also rapidly neutralizes toxic free copper in tissue and blood by forming complexes with excess copper and albumin. The excess copper is excreted via the bile, the body’s natural route for excess copper elimination.

The active ingredient of WTX101, tetrathiomolybdate, has been tested in several clinical studies in Wilson Disease patients and the data from these studies, as well as data from the Company’s WTX101-201 study, 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 may have 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.