Vivet, Pfizer to begin Wilson's disease gene therapy trial

By The Science Advisory Board staff writers

The U.S. Food and Drug Administration has cleared Vivet Therapeutics' investigational new drug application for the Gateway study, a phase I/II study evaluating Vivet's VTX-801, a proprietary, investigational gene therapy for the treatment of Wilson's disease.

The trial is expected to begin in early 2021. The study is a multicenter, nonrandomized, open-label, phase I/II clinical trial designed to assess the safety, tolerability, and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson's disease, prior to and following background Wilson's disease therapy withdrawal. The study will enroll up to 16 adult patients in six locations in the U.S. and Europe.

VTX-801 is a recombinant adeno-associated virus (rAAV)-based gene therapy vector designed to delivery miniaturized ATP7B transgene encoding, a functional protein shown to restore copper homeostasis, reverse liver pathology, and reduce copper accumulation in the brain of mice with Wilson's disease. The vector was selected based on its ability to transduce human liver cells.

In March 2019, Pfizer announced that it acquired a minority equity interest in Vivet and secured an exclusive option to acquire all outstanding shares. And in September 2020, Vivet and Pfizer announced a manufacturing agreement for VTX-801 vector for the Gateway study.


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