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  • 23 October 2017
    Wilson Therapeutics reaches agreement with the FDA and EMA to initiate pivotal Phase 3 FOCuS study with WTX101 in Wilson Disease

    Wilson Therapeutics (publ) today announced that the discussions with the U.S Food and Drug Administration (FDA) and the European Medicines Agency (EMA), regarding the study protocol for the single pivotal Phase 3 FOCuS trial for WTX101 in Wilson Disease, have been concluded successfully. Wilson Therapeutics will now complete the preparatory work to start the trial and expects to enroll the first patient in early 2018. WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper-protein-binding agent with a unique mechanism of action, under investigation as a novel therapy for Wilson Disease.  

    The Phase 3 FOCuS trial will be a randomized, controlled, rater-blinded, multi-center study assessing the efficacy and safety of WTX101 monotherapy administered once daily for 48 weeks, compared to standard of care (SoC), in patients with Wilson Disease aged 18 years and older. The study will enroll approximately 100 patients with hepatic and/or neurological symptoms, who are treatment naïve or have previously received SoC therapy. The primary endpoint will be copper control, assessed as the percentage change in free blood copper levels from baseline to 48 weeks. Consistent with the successful Phase 2 study, copper control will be assessed using NCCcorrected. Additional endpoints include clinical (hepatology, neurology, psychiatry, disability) and quality of life related endpoints, and safety of WTX101. The study will be conducted at approximately 30 sites in the US, EU and Israel, including sites involved in the completed Phase 2 study. Patients completing the FOCuS study through 48 weeks will be offered continued WTX101 treatment in an extension phase.

    In line with Wilson Therapeutics’ commitment to improve the life of patients with Wilson Disease, the company will continue to conduct exploratory research in parallel with the FOCuS study to further deepen the understanding of the disease, the mode of action of WTX101 and its impact on copper control.

    “We are very excited to move WTX101 into the pivotal phase of development and initiate the first global, randomized, controlled, multi-center study ever conducted in Wilson Disease. The FDA and the EMA have been very supportive and collaborative and have provided valuable discussions and input on the design of the Phase 3 study. We are confident that we have a robust trial design with the potential to repeat and confirm the excellent data observed in our Phase 2 trial, and support approval”, said Jonas Hansson, CEO of Wilson Therapeutics.

    “Having cared for patients with Wilson Disease for more than 30 years, I am extremely excited to be part of the WTX101 development program. Today, people with Wilson Disease are routinely treated with drugs that were first introduced in the 1950s and 1960s and there is a significant need for improved therapies. We are excited to offer WTX101 in the context of a clinical trial. The data generated in the Phase 2 study were very promising, particularly the rapid and clinically meaningful improvement of disease-related symptoms achieved with once-daily dosing of WTX101. In addition, the possibility of using WTX101 to avoid the early paradoxical neurological worsening, unfortunately seen in some patients started on the currently approved treatments penicillamine and trientine, is encouraging. Also, the once-daily dosing of WTX101 has the potential to minimize the treatment burden, leading to better long-term compliance and treatment outcomes. I am very hopeful we will be able to replicate the positive Phase 2 results in Phase 3”, said Frederick K. Askari, MD, PhD, Associate Professor and Director of the Wilson Disease program at the University of Michigan.