Rare Disease Report

Copper Protein Binding Agent May Help ALS Patients

JUNE 11, 2017
James Radke
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 < .0001).


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.

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