Vivet Therapeutics Shares Interim Phase 1/2 GATEWAY Trial Data on Wilson Disease at EASL Congress 2024

Vivem Therapeutics has shared promising interim results from its international Phase 1/2 GATEWAY trial at the EASL Congress 2024 in Milan, Italy, focusing on the safety, pharmacodynamics, and efficacy of their lead program, VTX-801, a gene therapy for Wilson Disease (WD). The trial is the first-of-its-kind in humans and aims to evaluate the long-term impact of a single intravenous dose of VTX-801.

Wilson Disease is a genetic disorder causing excessive copper accumulation in the body, leading to severe liver and neurological damage. Current lifelong oral treatments are often associated with significant side effects, resulting in poor patient adherence and serious complications. Vivet Therapeutics is exploring a novel solution through VTX-801, a recombinant adeno-associated viral vector (rAAV) carrying a liver-specific ATP7B gene to restore copper metabolism in affected individuals.

The GATEWAY trial's interim data presented by Associate Professor Thomas Damgaard Sandahl focuses on two patients from Cohort 1 who received a single intravenous dose of VTX-801. The results showed promising safety and tolerability, with no serious adverse events reported. One patient experienced an infusion-related reaction that was managed with symptomatic treatment, and both patients were discharged the next day as planned. Transient, mild elevations in liver enzyme levels were observed but managed according to protocols used in other systemic AAV gene therapies.

Encouragingly, there was an increase in ceruloplasmin ferroxidase activity from baseline levels, indicating transgene activity in both patients. Additionally, liver biopsy results showed normalization of liver fibrosis and iron accumulation scores one year post-treatment, suggesting an improvement in liver health.

Jean-Philippe Combal, Co-Founder and CEO of Vivet Therapeutics, expressed optimism about the interim results, highlighting the potential of VTX-801 to provide a viable alternative to current WD treatments. The completion of Cohort 1 demonstrated notable safety, tolerability, and early pharmacodynamic changes. In the ongoing study, the first patient in Cohort 2 has been administered a higher dose of VTX-801, aiming for a more substantial pharmacodynamic response.

Combal emphasized that the progress of the study has been encouraging so far, and the company remains committed to developing VTX-801 as a potential disease-modifying treatment for individuals living with Wilson Disease.

Further information about the GATEWAY trial can be found on Clinical Trials under the identifier NCT04537377.

Vivet Therapeutics, founded in 2016 by Dr. Jean-Philippe Combal and Dr. Gloria Gonzalez-Aseguinolaza, is a clinical-stage biotech company dedicated to developing innovative and enduring gene therapies for rare inherited metabolic disorders, including Wilson Disease. The company's gene therapy platform employs recombinant adeno-associated viruses (rAAVs) as vectors. Vivet has initiated two clinical programs and has four pre-clinical assets to date. VTX-801, its most advanced therapy for Wilson Disease, is expected to have key clinical read-outs by the end of 2024.

Supported by international life science investors such as Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., and others, Vivet Therapeutics has garnered significant funding and recognition for its scientific expertise and innovative technology platforms. Notably, Pfizer contributed a €45 million investment in 2019 to collaborate with Vivet, acknowledging the company's advancements in gene therapy.

The European Association for the Study of the Liver (EASL), established in 1966, is dedicated to fostering excellence in liver research and clinical practice. EASL serves a global community of over 5,300 members from 112 countries, providing education and resources to those involved in hepatology.

In summary, Vivet Therapeutics' interim data from the GATEWAY trial offers a promising outlook for VTX-801 as a potential new treatment for Wilson Disease, demonstrating encouraging safety, tolerability, and early efficacy results.