The U.S. Food and Drug Administration (FDA) has granted Wilson Therapeutics’ WTX101 Orphan Drug Designation to treat some patients with Amyotrophic Lateral Sclerosis (ALS).
WTX101 (bis-choline tetrathiomolybdate) is a copper-protein binding agent.
ALS, a neurodegenerative disease, sees 7% of patients with a mutation in the copper-dependent enzyme, superoxide dismutase 1 (SOD1) that has anti-oxidant properties. In animal models, tetrathiomolybdate can delay the severity and onset of ALS symptoms.
“ALS is a devastating disease with very limited treatment options. The Orphan Drug Designation is an important milestone for us and it recognizes the promise of WTX101 as a potential new treatment for ALS,” said Jonas Hansson, Chief Executive Officer of Wilson Therapeutics via news release
. “The disease is notoriously hard to treat but pre-clinical data for WTX101 are promising so we are currently exploring the possibility of developing WTX101 for the treatment of the subset of ALS patients that suffer from SOD1-mutated ALS.”
Additionally WTX101 is being studied in Wilson’s disease, a rare genetic disorder in which copper accumulates in various organs leading to a plethora of symptoms.
Data from a Phase 2 study was recently presented at the International Congress of Parkinson’s Disease and Movement Disorders in Vancouver, BC, 4-8 June, 2017. That study met its primary endpoint of copper control (P
< .001) and significant improvements from baseline to week 24 were observed for both patient-reported disability (P
< .001) and rater-assessed neurological status (P
Bega D, Ala A, Askari F, et al. WTX101 – A Novel Copper Modulating Agent for Wilson Disease Demonstrates Efficacy and Safety in a Phase 2, Multi-Center, Open Label Study. Presented at the International Congress of Parkinson’s Disease and Movement Disorders; Vancouver, BC; June 4-8, 2017.