VTX-801 / Vivet Therapeutics, Pfizer - LARVOL DELTA

Collaborating Across Borders to Advance Patient Care (ASGCT 2025) - P1/2 | "It evaluates VTX-801 in adult patients with Wilson's Disease (NCT04537377)...The second case focuses on the development of varnimcabtagene autoleucel (Var-Cel) , a CAR-T cell therapy initially created within an academic hospital. Var-Cel is designed to treat certain types of relapsed or refractory B-cell malignancies, such as acute lymphoblastic leukemia and non-Hodgkin lymphoma. This groundbreaking, hospital-originated CAR-T therapy is now reaching patients at a fraction of the traditional cost through a national network, with international expansion already underway."

Clinical • Acute Lymphocytic Leukemia • Genetic Disorders • Hematological Malignancies • Hepatology • Leukemia • Lymphoma • Metabolic Disorders • Movement Disorders • Non-Hodgkin’s Lymphoma • Oncology • ATP7B

GATEWAY: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (clinicaltrials.gov) - P1/2 | N=4 | Active, not recruiting | Sponsor: Vivet Therapeutics SAS | Recruiting ➔ Active, not recruiting | N=16 ➔ 4 | Trial completion date: Mar 2029 ➔ Jun 2029

Enrollment change • Enrollment closed • Trial completion date • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders

Interim safety results of the ongoing international phase I/II GATEWAY gene therapy trial with VTX-801 conducted in adult patients with Wilson disease (EASL-ILC 2024) - P1/2 | "Treatment of patients of the GATEWAY Cohort 1 (low dose) has been completed. No serious adverse events have been reported. Follow-up of treated patients and enrolment of Cohort 2 (intermediate dose) are underway."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • Pain • ATP7B

GATEWAY: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (clinicaltrials.gov) - P1/2 | N=16 | Recruiting | Sponsor: Vivet Therapeutics SAS | Trial primary completion date: Mar 2024 ➔ Mar 2025

Trial primary completion date • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders

Therapeutic Efficacy of the Combination of Long-Term Penicillamine Treatment with a Single Administration of VTX-801 in Wilson's Disease Mice (ASGCT 2023) - "Current medical management relies on life-long oral treatment with copper chelators such as D-penicillamine (DPA) or zinc salts. Altogether these results indicate that treatment with a copper chelator contributes to stabilization of liver disease progression in WD mice. DPA treatment significantly improved the therapeutic efficacy of VTX-801 when administered at 20 weeks of age resulting in full restoration of copper metabolism in the absence of adverse events."

Preclinical • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Inflammation • Metabolic Disorders • Movement Disorders • Psychiatry • Rare Diseases • ATP7B

Therapeutic Efficacy of VTX-801 in Advanced Stage of Wilson's Disease in Mice (ASGCT 2023) - "Epub 2019 Mar 20. PMID: 30706949."

Metastases • Preclinical • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Liver Failure • Metabolic Disorders • Movement Disorders • Psychiatry • Rare Diseases • ATP7B

GATEWAY: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (clinicaltrials.gov) - P1/2 | N=16 | Recruiting | Sponsor: Vivet Therapeutics SAS | Trial completion date: Jul 2027 ➔ Mar 2029 | Trial primary completion date: Jul 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders

High value of Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease. (PubMed, Mol Ther Methods Clin Dev) - "VTX-801 treatment in WD mice resulted in a significant reduction of hepatic Cu accumulation, the restoration of fecal Cu excretion, and the correction of Cu BK. This study showed that VTX-801 restores physiological copper metabolism in WD mice, confirming the mechanism of action of VTX-801, and demonstrated the translational potential of [Cu]CuCl-PET to explore VTX-801 pharmacodynamics in a minimally invasive and sensitive manner in WD patients."

Journal • Gene Therapies • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • ATP7B

Safety and Biodistribution of VTX-801, an AAV3B Gene Therapy Vector, in Healthy Cynomolgus Monkeys (ASGCT 2022) - "CONCLUSION This study of a single intravenous administration of VTX-801 in juvenile cynomolgus monkeys demonstrated safety of the doses evaluated with no toxicity, an excellent liver tropism with limited off-target biodistribution, no immune response, and sustained expression of the gene therapy product in vector-treated animals. This study has supported the clinical development of VTX-801 for a first-in-human clinical trial in Wilson’s patients (GATEWAY)."

Clinical • Cardiovascular • Gene Therapies • Hematological Disorders • Hepatology • Metabolic Disorders • Movement Disorders

GATEWAY: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: Vivet Therapeutics SAS; Initiation date: Apr 2021 ➔ Aug 2021

Clinical • Trial initiation date • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • CP