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  • 9 June 2017
    Wilson Therapeutics receives US Orphan Drug Designation for WTX101 for the treatment of ALS

    Wilson Therapeutics AB (publ), announced today that the U.S. Food and Drug Administration (FDA) has granted the company Orphan Drug Designation for WTX101 for the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS). WTX101 (bis-choline tetrathiomolybdate) is an investigational first-in-class copper-protein binding agent with a unique mechanism of action.

    ALS is a neurodegenerative disease, in which the nerve cells controlling the body’s muscles gradually degenerate, leading to general paralysis and respiratory failure. Median survival for an ALS patient is three to five years. Currently, the treatments available for ALS are hampered by limited efficacy so the focus of medical care is to provide symptomatic management of all patients with mild to moderate disease and palliative intervention in patients with severe or terminal disease.

    Approximately 7% of the ALS patients have a mutation in a copper-dependent enzyme, superoxide dismutase 1 (SOD1). SOD1 is an enzyme with antioxidant properties important in the protection against free radicals, a highly reactive oxygen species that can damage cellular components via oxidative stress. The active ingredient of WTX101, tetrathiomolybdate, has been tested in mice that are genetically modified with a mutant form of human SOD1 and develop ALS. These studies show that tetrathiomolybdate can delay the onset of disease as well as reduce the symptoms after disease onset in this mouse model.

    “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. 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,” said Jonas Hansson, Chief Executive Officer of Wilson Therapeutics.

    “However, our immediate development focus remains Wilson Disease, where we are expecting to initiate Phase 3 later this year,” concluded Jonas Hansson.