The pharmaceutical company Pfizer Inc and the biotech business Vivet Therapeutics ("Vivet") have received authorization to initiate a clinical essay of Wilson's disease with gene therapy originating from the Cima University of Navarra.

The two institutions have announced that the U.S. Food and Drug Administration (FDA) has C Vivet's New Drug application at research (IND) for the GATEWAY study. This is a Phase 1/2 clinical essay evaluating the efficacy of Vivet's proprietary gene therapy vector, VTX-801, for the treatment of Wilson's disease, a rare and potentially fatal liver disease. VTX-801 is a drug developed at Cima with technology licensed to Vivet in 2016.

"This is a very important milestone for the Wilson's disease patient community for whom VTX-801 may offer significant therapeutic benefit. VTX-801 is goal intended to restore proper copper metabolism. The GATEWAY clinical essay will analyze relevant biomarkers to assess the physiological restoration of copper clearance and transport in patients. We expect VTX-801 to reach the clinic in early 2021," says Jean-Philippe Combal, director executive and co-founder of Vivet.

According to Dr. Gloria González Aseguinolaza, researcher at Cima and co-founder of Vivet, "VTX-801 is a new gene therapy vector based on adeno-associated virus (AAV) and designed to deliver a miniaturized version of the ATP7B protein. This protein reverses liver pathology and reduces copper accumulation in liver and brain in a model Wilson's disease mouse."

agreement of production

In March 2019, it was announced that Pfizer obtained a minority stake in Vivet and secured an exclusive option to acquire all outstanding shares. In September 2020, Vivet and Pfizer announced the signature of a agreement for Pfizer's production of the VTX-801 vector for the essay clinical GATEWAY.

"The FDA approval of Vivet's IND marks an important milestone for the VTX-801 program, which we believe has the potential to become a transformative therapy for people with Wilson's disease," said Seng Cheng, director scientist for Pfizer's research Rare Diseases Unit. "Pfizer has begun manufacturing clinical material for the GATEWAY study and looks forward to the initiation of the study."

"This IND is a recognition of the expertise of Vivet's research team led by our CSO and co-founder, Dr. Gloria Gonzalez-Aseguinolaza, thanks to the collaborations maintained with the Foundation for Applied Medical research (the holding entity of Cima University of Navarra), and an experienced development team at Vivet. "We believe that our expertise in development globally, together with our partnership with Pfizer, puts us in a strong position to rapidly execute and bring this potentially transformative therapy to patients with unmet medical needs," added Jean-Philippe Combal.