Pharma Focus Europe

Vivet Therapeutics Administers First Patient Dose in Cohort 2 of Phase 1/2 GATEWAY Clinical Trial for Wilson Disease Treatment

Tuesday, April 09, 2024

Vivet Therapeutics has achieved a significant milestone in its effort to address Wilson Disease (WD), a rare inherited liver metabolic disorder characterized by the accumulation of toxic copper levels in the liver and brain. The company has successfully administered its lead program, VTX-801, to the first patient in Cohort 2 of its GATEWAY Clinical Trial following the completion of Cohort 1 and approval from an independent Data Monitoring Committee (DMC).

WD poses serious health risks, including neurological and hepatic complications, with a mortality rate significantly higher than the general population despite existing treatments. With approximately 1 in 30,000 individuals affected globally, the need for effective therapies is paramount.

The ongoing GATEWAY trial, conducted across multiple clinical sites in the US, UK, Germany, and Denmark, aims to evaluate the safety, tolerability, and pharmacological activity of VTX-801 through a single intravenous infusion in adult WD patients.

Jean-Philippe Combal, CEO of Vivet Therapeutics, expressed optimism about the trial's progress, highlighting the potential of VTX-801 to address the unmet needs of WD patients and their families. The company is encouraged by initial data showing promising safety and pharmacodynamic effects.

Prof Michael Schilsky, Principal Investigator at Yale, emphasized the significance of VTX-801 in potentially revolutionizing WD treatment. Early results from Cohort 1 have shown promising signs of liver function improvement, offering hope for patients burdened by lifelong therapies with varying effectiveness and adverse effects.

With DMC approval, Vivet has advanced to higher dose levels in Cohort 2, building on the positive outcomes observed in Cohort 1. The absence of unexpected adverse events in Cohort 1 is reassuring, and ongoing monitoring has shown sustained improvements in hepatic function and histology.

Vivet plans to present further data from the GATEWAY trial at upcoming scientific conferences, marking a pivotal year for the company and its gene therapy platform in addressing the challenges posed by WD.



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