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Wilson Therapeutics presents promising neurological Phase 2 data for WTX101 at AAN meeting

Apr 25, 2017

 Wilson Therapeutics AB (publ), announced today a poster presentation at the 69th American Academy of Neurology (AAN) Annual Meeting in Boston, MA, 22-28 April, 2017, highlighting the results on neurological parameters from the recently completed Phase 2 trial of WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper modulating agent with a unique mode of action for the treatment of patients with Wilson Disease.

WTX101-201 was a 24-week open-label Phase 2 study evaluating the efficacy and safety of WTX101 monotherapy in 28 newly-diagnosed adult patients with Wilson Disease, who had received either no prior treatment for Wilson Disease or a standard of care agent for up to two years. As previously reported, the Phase 2 study of WTX101 met its primary endpoint of copper control (p< 0.001).

Secondary endpoints in the trial included neurological status and disability. Neurological status was measured using the Unified Wilson Disease Rating Scale (UWDRS) Part 3 where a physician assesses the patients’ status using a comprehensive set of standardized tests. Mean UWDRS Part 3 score was 22.8 at baseline and mean reduction from baseline was 8.7 (p< 0.0001) after 24 weeks. Per protocol, a reduction of ≥4 points from baseline was defined as a clinically relevant improvement. Of the 21 patients that completed the full study period and could perform all the tests, 13 patients improved by ≥4 points, 6 patients stabilized (± 3 points of baseline) and 2 deteriorated (both by 5 points). Significant reductions in mean UWDRS 3 score were observed from week 8 and onwards.

Disease related disability was rated by the patients using UWDRS Part 2, assessing the ability to perform specific tasks related to activities of daily living. Mean UWDRS Part 2 score was 6.6 at baseline and mean reduction from baseline was 3.7 (p< 0.001) after 24 weeks. Per protocol, a reduction of ≥1 points from baseline was defined as a clinically relevant improvement. Of the 21 patients that completed the full study period and could perform all the tests, 12 patients improved by ≥1 points, 9 patients stabilized (no change from baseline) and no patient reported a deterioration.

Treatment with WTX101 was generally well-tolerated with most reported adverse events being mild (grade 1) to moderate (grade 2). Importantly, no early neurological worsening upon initiation of WTX101 was observed.

“A large proportion of patients with Wilsons Disease present with neurological symptoms, ranging from mild tremor to significant motor and speech impairments. There are significant unmet needs for these patients in terms of efficacy with higher response rates and faster reduction of clinical symptoms. Therefore, it is very encouraging that we observed a high rate of response and fast impact on both neurological status and disability that was both statistically significant and clinically relevant,” said Carl Bjartmar, Chief Medical Officer of Wilson Therapeutics.

“Furthermore, current treatment options for this group of patients are also significantly limited by the potential for paradoxical neurological worsening upon initiation of therapy. We believe the data from the Phase 2 trial with WTX101 is also highly encouraging in this area, with no reported cases of typical early drug-induced neurological worsening. We look forward to advancing WTX101 into a pivotal Phase 3 trial that we plan to initiate during the second half of 2017,” concluded Carl Bjartmar.

Poster Presentation summary details:

Title: “Efficacy and Safety of a Novel Copper Modulating Agent (WTX-101) for Wilson Disease: Results of a Phase 2, Multi-Center, Open Label Study”
Authors: D. Bega (Northwestern University), A. Ala (The Royal Surrey Country Hospital), F. Askari (University of Michigan Hospital), J. Bronstein (UCLA), A. Czlonkowska (2nd Dept of Neurology, Institute of Psychiatry and Neurology), P. Ferenci (Medical University of Vienna), D. Nicholl (City Hospital), K. Weiss (University Hospital Heidelberg), M. Schilsky (Yale University School of Medicine).
Session: Movement Disorders: Other 1
Date: Monday, 24 April

Abstracts for the AAN meeting are available on the conference website (
About WTX101 (bis-choline tetrathiomolybdate)
WTX101 (bis-choline tetrathiomolybdate) 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.

A Phase 2 study evaluating the efficacy and safety of WTX101 in Wilson Disease patients was successfully completed in 2016. In addition, the active ingredient of WTX101, tetrathiomolybdate, has been tested in several previous clinical studies in Wilson Disease patients. The data from these studies 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.

About Wilson Disease
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.

About Wilson Therapeutics
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 in development as a novel treatment for Wilson Disease. A Phase 2 clinical study has been successfully completed and preparations for a pivotal Phase 3 study are ongoing. Wilson Therapeutics is listed in the Mid Cap segment on Nasdaq Stockholm with the stock ticker WTX.

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For further information contact:
Jonas Hansson, CEO, Wilson Therapeutics AB
Telephone: +46 8 796 00 00

Wilson Therapeutics AB (publ)
Corp reg nr 556893-0357
Kungsgatan 3
SE-111 43 Stockholm

The information in the press release is information that Wilson Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 08.00 CET on 25 April, 2017.



Source: Wilson Therapeutics