The round involved a syndicate of leading international life sciences investors led by Novartis Venture Fund and Columbus Venture Partners, and including Roche Venture Fund, HealthCap, Kurma Partners and Ysios Capital.
The funds will be used by Vivet to advance a diversified pipeline of gene therapy programs targeting rare, inherited metabolic diseases, including Wilson Disease, progressive familial intrahepatic cholestasis type 2 (PFIC2), progressive familial intrahepatic cholestasis type 3 (PFIC3) and citrullinemia type I.
Vivet is building its pipeline based on novel technology 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; and Massachusetts Eye and Ear (MEE), Boston, USA.
The licenses cover exclusive use of novel proprietary AAV vector gene therapy technology to treat metabolic diseases and certain MEE patent-protected Anc80 AAV gene therapy vectors. Anc80 is a next-generation gene therapy technology designed to increase gene expression levels in the liver, while reducing the risk of undesired immunogenicity.
Vivet’s lead program, VTX801, is a novel investigational gene therapy targeting Wilson Disease. This rare genetic disorder is caused by a defective gene in liver cells encoding the ATP7B protein, which reduces the liver’s ability to regulate copper levels in the liver and other tissues causing severe hepatic and neurologic symptoms, leading to liver transplantation and potentially death.
The disease affects approximately one in 30,000 people worldwide, corresponding to a prevalence of approximately 10,000 patients in the US and 15,000 patients in the EU.
VTX801 uses a novel modified AAV vector to transport a truncated functional version of the ATP7B gene into the liver cells carrying the defective gene to treat the underlying cause of the disease; to restore copper metabolism, reduce liver damage and improve liver function.
Vivet expects to initiate first in human trials with VTX801 in Wilson disease by the end of 2018.
Vivet Therapeutics was created in Paris, France, with a wholly-owned subsidiary in Spain, in 2016 by Jens Kurth (ex Anokion, Novartis), Gloria Gonzalez Aseguinolaza (CIMA, University of Navarra) and Jean Philippe Combal (ex Gensight Biologics, Sanofi), bringing together significant experience and expertise in gene therapy, liver diseases and global drug discovery and development for rare diseases.
Florent Gros, Managing Director at Novartis Venture Fund, commented: “We have searched extensively for next generation AAV technologies and clinical applications. We are very excited by Vivet Therapeutics' clinical and commercial prospects; the company has outstanding management, assets and capabilities.”
Jean-Philippe Combal, Vivet’s Co-founder & CEO, said: “Vivet is delighted to have attracted such a substantial investment from these high profile life sciences investors. This fundraising reflects our shared excitement about the potential of our lead candidate VTX801 and our technology for generating further novel gene therapies targeting rare inherited metabolic diseases.
“Early results from preclinical studies with VTX801 are very promising, and we are now well funded to advance this candidate into the clinic, while developing our portfolio and technologies.”
Gloria Gonzalez Aseguinolaza, Vivet’s Co-founder & CSO, added: “Technologies for the development of gene therapies have advanced significantly in recent years which, along with an improved understanding of genetic diseases, offer new opportunities for patients with suboptimal therapeutic options. By collaborating with leading institutions such as CIMA in Spain and MEE in the United States, Vivet has secured superior and novel gene therapy technologies and liver disease expertise.
“We believe these capabilities, combined with the international development expertise of the management team, creates a company with very exciting prospects.”