Vivet Therapeutics (‘Vivet’), an emerging biotechnology company developing novel gene therapies for rare, inherited metabolic diseases, announced it has raised €37.5 million in a Series A financing round. 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.
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.”
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.
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.”