• 12 April 2018
    Promising preliminary long-term data for WTX101 in Wilson Disease presented at EASL Annual Meeting

    Wilson Therapeutics AB (publ), announced today that preliminary long-term efficacy and safety data from the ongoing extension phase of the company’s Phase 2 trial of WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper-protein-binding agent under investigation as a novel therapy for Wilson Disease, will be presented today during a Late Breaker poster session at The International Liver Congress™ 2018, the Annual Meeting of the European Association for the Study of the Liver (EASL), in Paris.

    Carl Bjartmar, Chief Medical Officer, Wilson Therapeutics AB commented: “Data from the Phase 2 study have previously demonstrated that once-daily treatment with WTX101 significantly reduces free copper levels in patients with Wilson Disease. The 72-week extension data indicate that the control of copper is maintained long term. Importantly, this translates into further improvements or stabilization of liver status, as well as continuous improvements in neurological status and patient reported disability over the whole 72-week study period. This is very promising, especially for patients with persistent neurological problems despite years of therapy with currently available drugs. WTX101 also appears to be generally well tolerated with few adverse events reported during the extension period. All in all, these long-term data are highly encouraging and we look forward to further evaluating the profile of WTX101 in our ongoing Phase 3 FOCuS study”.

    All 22 patients who completed the 24-week open-label, single-arm, Phase 2 study opted to continue once-daily WTX101 treatment in the extension phase. 20 patients completed week 72; one patient discontinued treatment due to her wish to conceive and one patient was unable to comply with study procedures due to a progressive disease course despite ongoing treatment.

    As previously announced when the abstract was published on March 28, 2018, the initial improvements in free copper levels, hepatic status, neurological status and disability observed at week 24 and 48 were preserved or further improved after once-daily treatment for 72 weeks with WTX101.

    WTX101 was generally well tolerated over 72 weeks of treatment. Reversible ALT elevations requiring dose adjustment (observed in about one-third of patients in the core period of the Phase 2 trial) have not been observed in the extension phase. No cases of drug-induced neurological worsening have been observed during the study.