Vivet Therapeutics y Pfizer Inc reciben autorización de la FDA para iniciar GATEWAY, ensayo clínico de fase 1/2 con VTX-801, la terapia génica de Vivet para la enfermedad de Wilson

PARIS, Francia y NUEVA YORK, NY — 18 de noviembre de 2020— Vivet Therapeutics (“Vivet”), empresa privada de biotecnología dedicada al desarrollo de terapia génica para enfermedades hepáticas hereditarias  y Pfizer Inc (NYSE: PFE ), anunciaron que la Administración de Alimentos y Medicamentos de los EE. UU. (FDA) aprobó la solicitud de Nuevo Medicamento en Investigación (IND) de Vivet para el estudio GATEWAY, un ensayo clínico de fase 1/2 que evalúa el vector de terapia génica patentado por Vivet, VTX-801, para el tratamiento de la enfermedad de Wilson (WD), una enfermedad hepática poco común y potencialmente mortal. Se espera que el ensayo comience a principios de 2021.

"Nos complace anunciar la primera autorización de un IND de Vivet por parte de la FDA para GATEWAY, nuestro ensayo clínico de Fase 1/2 con VTX-801 ", dijo Jean-Philippe Combal, director ejecutivo y cofundador de Vivet. “Este es un hito muy importante para la comunidad de pacientes con la enfermedad de Wilson para quienes VTX-801 puede ofrecer un importante beneficio terapéutico. VTX-801 tiene como objetivo restaurar el correcto metabolismo del cobre. El ensayo clínico GATEWAY analizará biomarcadores relevantes para evaluar la restauración fisiológica de la eliminación y el transporte de cobre en los pacientes. Esperamos que VTX-801 llegue a la clínica a principios de 2021 ".

VTX-801 es un nuevo vector de terapia génica basado en virus adenoasociados (AAV) diseñado para administrar una versión miniaturizada la proteína ATP7B la cual se ha demostrado que restaura la homeostasis del cobre, revierte la patología hepática y reduce la acumulación de cobre en hígado y en cerebro en un modelo de ratón de enfermedad de Wilson. El serotipo de rAAV que se ha empleado en la producción del vector terapéutico VTX-801 se seleccionó en función de su capacidad para la transducción de células hepáticas humanas.

En marzo de 2019, se anunció que Pfizer obtuvo una participación minoritaria en Vivet y se había asegurado una opción exclusiva para adquirir todas las acciones en circulación. En septiembre de 2020, Vivet y Pfizer anunciaron la firma de un acuerdo para la producción por parte de Pfizer del vector VTX-801 para el ensayo clínico GATEWAY.

"La aprobación de la FDA del IND de Vivet marca un hito importante para el programa VTX-801, que creemos tiene el potencial de convertirse en una terapia transformadora para las personas con la enfermedad de Wilson", dijo Seng Cheng, director científico de la Unidad de Investigación de Enfermedades Raras de Pfizer. "Pfizer ha comenzado a fabricar material clínico para el estudio GATEWAY y espera con interés el inicio del estudio".

“Este IND es un reconocimiento a la experiencia del equipo de investigación de Vivet dirigido por nuestra CSO y cofundadora, la Dra. Gloria González-Aseguinolaza, gracias a las colaboraciones de mantenidas con la Fundación para la Investigación Médica Aplicada (Cima Universidad de Navarra), y un equipo de desarrollo experimentado de Vivet. Creemos que nuestra experiencia en desarrollo global, junto con nuestra colaboración con Pfizer, nos coloca en una posición sólida para ejecutar rápidamente y acercar esta terapia potencialmente transformadora a pacientes con necesidades médicas no satisfechas”, agregó Jean-Philippe Combal.

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About GATEWAY - Phase 1/2 Clinical Trial of VTX-801 in Wilson disease

The GATEWAY trial is a multi-center, non-randomized, open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background WD therapy withdrawal.

 Six leading centers in the United States and Europe are expected to participate in the GATEWAY Phase 1/2 trial. The trial is expected to enroll up to sixteen adult patients with Wilson disease and will evaluate up to three doses of VTX-801. Patients will participate in a pre-dosing observational period and will be administered a prophylactic steroid regimen.

The primary endpoint of the GATEWAY trial is to assess the safety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal.

Vivet Therapeutics expects to enroll the first patient in early 2021.

More details on:

https://clinicaltrials.gov/ct2/show/NCT04537377?term=VIVET&draw=2&rank=1

About Vivet Therapeutics

Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

Vivet’s lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

Vivet’s second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

Please visit us on www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice

The information contained in this release is as of November 18, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about Vivet Therapeutics’ (Vivet) investigational gene therapy, VTX-801, and Pfizer’s collaboration with Vivet on the development of VTX-801, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, risks related to the ability to realize the anticipated benefits of the collaboration, including the possibility that the expected benefits from the collaboration will not be realized or will not be realized in the expected time; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when any applications may be filed in any jurisdiction for VTX-801; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether VTX-801 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of VTX-801; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.