Objective: The aim of this ongoing study is to evaluate the efficacy and safety of WTX101 in newly diagnosed Wilson Disease patients.

Background: WTX101 (bis-choline tetrathiomolybdate) is an investigational de-coppering agent that is being developed for the treatment of Wilson Disease. The aim of this ongoing study is to evaluate the efficacy and safety of WTX101 in newly diagnosed Wilson Disease patients.

Methods: Patients with a confirmed diagnosis of Wilson Disease naïve to treatment or treated for ≤ 28 days with chelation or zinc therapy received 30 or 60 mg WTX101 QD during the initiation phase. Dosing was individualized after 6 weeks, guided by laboratory and clinical criteria. Regular assessments included safety, status of liver disease including synthetic function and modified Nazer score, change from baseline in free copper. and neurological status using the Unified Wilson Disease Rating Scale (UWDRS).

Results: The first six patients (age 18-53 years; 2 males) were followed for 8 to 36 weeks in the study. Modified Nazer score ranged from 1-5 and 5/6 had neurological manifestations at baseline. Reversible elevated liver function tests related to per-protocol dose escalations was observed in the first 2 patents. After reducing the initiation dose from 30 mg BID (totally 60 mg/day) to 30 mg QD and dose escalation to a maximum daily dose of 60 mg, WTX101 was well tolerated with no SAEs and few AEs. Hepatic status improved or remained stable. Elevated baseline plasma NCC adjusted for molybdenum on average normalized within 3 months. The UWDRS scores improved in all patients with neurological manifestations. By 3 months, the neurological examination score (part III) was reduced from approximately 30 to <10, with continuous improvement thereafter. Daily activity status (part II) showed similar improvement.

Conclusions: WTX101 appears safe and well tolerated in patients with Wilson Disease. With improvement in clinical (hepatic and neurologic) and laboratory assessments including copper control in these initial patients, further clinical evaluation is warranted to establish the efficacy and safety of WTX101.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/an-ongoing-phase-2-multi-centre-open-label-study-of-wtx101-in-wilson-disease-patients-early-observations/