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Vivet Therapeutics Testing Wilson Disease Gene Therapy in Second Cohort

NEW YORK – Vivet Therapeutics on Monday said it has begun dosing Wilson Disease (WD) patients with its investigational gene therapy VTX-801 in a second cohort of the Phase I/II GATEWAY trial.

Paris, France-based Vivet began dosing patients in this second cohort with permission from an independent data safety monitoring committee and after VTX-801 showed an encouraging safety, tolerability, and pharmacodynamic profile in the first cohort enrolled in GATEWAY. In this Phase I/II trial, researchers are enrolling adult WD patients across multiple clinical sites in the US, UK, Germany and Denmark. They hope to further gauge VTX-801's safety, tolerability, and pharmacological parameters in this second cohort by testing the gene therapy at a higher dose level.

Copper builds up to toxic levels in the brains and livers of patients with WD, causing neurological and hepatic problems and death. WD patients are more than four times more likely to die prematurely than the general population. Current treatments are designed to remove copper from the body but don't work in all patients and can cause side effects in some.

VTX-801 is designed to deliver a functional version of the ATP7B copper transporter gene to the liver with the goal of improving liver function and precluding the need for additional therapy.

In the first GATEWAY cohort, patients on the initial VTX-801 dose had no unexpected treatment-emergent adverse events. Further monitoring showed that patients sustained greater ceruloplasmin enzymatic activity and ATP7B minigene mRNA hepatic expression, and biopsies within a year of treatment showed improved liver histologies. However, at the initial dose level, the two patients in the first cohort were deemed "insufficient responders" based on a specific radio-labelled copper(64Cu) biomarker, Vivet said, and so they are continuing to receive standard-of-care treatments.

Still, this data gives Vivet reason to test VTX-801 at a higher dose. "Early, encouraging data from Cohort 1 has shown detectable vector transduction and transgene expression with early signs of improvement in liver function and histology," Michael Schilski, director of the Center for Excellence for Wilson Disease at Yale University and principal investigator of the GATEWAY clinical trial site there, said in a statement. "We look forward to moving this groundbreaking program further through the GATEWAY study and future clinical trials as we seek to demonstrate its transformative potential for patients with WD."