Vivet Therapeutics: FDA approved fase 1/2 study for VTX-801

PARIS / NEW  YORK. November 2020 — Vivet Therapeutics, a gene therapy biotech company dedicated to developing treatments for inherited liver disorders with high unmet   medical  need,  and Pfizer Inc.  (NYSE:  PFE)  announced today that  the  U.S. Food  and  Drug Administration (FDA) has cleared  Vivet’s Investigational New Drug (IND) application for the GATEWAY study, a Phase 1/2 clinical trial evaluating Vivet’s proprietary,  investigational gene therapy  vector, VTX-801, for the  potential  treatment of Wilson disease  (WD), a rare  and  potentially  life-threatening  liver disorder. The trial is expected to commence in early 2021.

“We are pleased to announce Vivet’s first IND clearance by the FDA, which is for our GATEWAY Phase 1/2 study for VTX-801,” said Jean-Philippe Combal, CEO and Co-Founder of Vivet. “This is a very important milestone  for  the  Wilson disease  community  for  whom  VTX-801  could  bring  significant  potential therapeutic benefit.  VTX-801 aims to restore  copper homeostasis  and the GATEWAY trial will measure relevant biomarkers to evaluate physiological restoration of copper elimination and transport in patients. We look forward to advancing VTX-801 into the clinic in early 2021.”

VTX-801 is a novel, investigational rAAV-based gene therapy  vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein  that  has been  shown to restore  copper  homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson disease. VTX-801’s rAAV serotype  was selected  based on its demonstrated tropism for transducing  human liver cells.

In March 2019, the companies announced that Pfizer had acquired a minority equity interest  in Vivet and secured  an  exclusive option  to  acquire  all outstanding  shares.  In September  2020, Vivet and  Pfizer announced the  signing of an agreement for the  manufacture by Pfizer of the  VTX-801 vector for the GATEWAY study.

“The FDA clearance  of Vivet’s IND marks an important  milestone  for the  VTX-801 program,  which we believe has the potential to become a transformational therapy for people with Wilson disease,” said Seng Cheng, Chief Scientific Officer, Rare Disease Research Unit, Pfizer. “Pfizer has begun manufacturing clinical material for the GATEWAY study and look forward to the study’s commencement.”

“This IND is a recognition of the expertise of Vivet’s research  team  led by our CSO and Co-Founder, Dr. Gloria González-Aseguinolaza, research  collaborations,  notably with la Fundación para la Investigación Médica Aplicada  (FIMA), and experienced  development team.   We believe that our global development expertise, together with our collaboration with Pfizer, places us in a strong position to rapidly execute and bring this potentially transformational therapy to patients  with high unmet medical needs,” added Jean- Philippe Combal.

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