Backed By Two Drug Powerhouses, GenSight Alum Launches New Biotech With $40 Million

Backed By Two Drug Powerhouses, GenSight Alum Launches New Biotech With $40 Million May 4, 2017
By Alex Keown, Breaking News Staff

PARIS – French startup biotech Vivet Therapeutics snagged $41 million in Series A financing as the company launches with a 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 launches with support from investors such as Novartis Venture Fund, Roche Venture Fund, Columbus Venture Partners, HealthCap Kurma Partners and Ysios Capital.

In its coming out announcement, Vivet said its pipeline is based on novel proprietary AAV vector gene therapy technology licensed from the Fundación para la Investigación Médica Aplicada. The licenses cover use of the technology to treat metabolic diseases and certain Massachusetts Eye and Ear (MEE) patent-protected Anc80 AAV gene therapy vectors. The technology is designed to increase gene expression levels in the liver, while reducing the risk of undesired immunogenicity.

Vivet’s lead program is VTX80, a novel investigational gene therapy targeting Wilson Disease, a genetic disorder caused by a defective gene in liver cells encoding the ATP7B protein. The defective gene 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. There are approximately 10,000 people in the United States diagnosed with Wilson’s disease and about 15,000 patients in the European Union.

Vivet will be helmed by Jean-Philippe Combal. In a statement, he touted the investments the startup received from “these high profile life sciences investors.”

Vivet said its VTX801 program 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. The therapy’s goal is to restore copper metabolism, reduce liver damage and improve liver function. The company is eying first human trials in Wilson disease by the end of 2018.

“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,” Combal said.

Wilson’s disease is often treated through chelating agents, which directs human organs to release copper into the bloodstream and ultimately be filtered through the kidneys. One of the most common drugs used to treat Wilson’s disease is Valeant’s Cuprimine. In 2015, Valeant was excoriated for its quadrupling of the price of Cuprimine, a 55-year-old drug.

In December, U.S.-based Kadmon Holdings filed an Abbreviated New Drug Application with the U.S. Food and Drug Administration for its trientine hydrochloride formulation called KD034 for Wilson’s disease.

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