Wilson Therapeutics is a biopharmaceutical company, based in Stockholm, Sweden, that develops novel therapies for patients with rare diseases. Wilson Therapeutics’ lead product, WTX101, is initially being developed as a novel treatment for Wilson Disease and has been evaluated in a Phase 2 clinical study.
Wilson Disease is a rare genetic disorder of impaired copper metabolism that causes serious copper poisoning. The genetic defect severely affects the body´s ability to regulate copper balance, resulting in life-threatening damage to the liver, the brain and further organs if left untreated. Wilson Disease affects approximately one in every 30,000 people worldwide, corresponding to a prevalence of approximately 10,000 patients in the US and 15,000 patients in the EU. The therapies currently being used in Wilson Disease were introduced in the 1950’s and 60’s and since then there have been no new treatment options developed for patients with this disease.
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.
Wilson Therapeutics AB was founded in 2012 and is listed in the Mid Cap segment on Nasdaq Stockholm with the stock ticker WTX.