The US Food and Drug Administration (FDA) has accepted French biopharmaceutical firm Genkyotex's investigational new drug (IND) application, allowing the company to initiate a Phase II trial of its GKT831 for the treatment of primary biliary cholangitis (PBC).

GKT831 is an inhibitor of the NOX1 and NOX4 enzymes.

The international, double-blind, placebo-controlled, multicentre Phase II trial is designed to assess the safety and efficacy of GKT831 in 102 PBC patients who have shown an inadequate response to ursodeoxycholic acid.

Set to be conducted at more than 50 clinical sites in North America and Europe, the 24-week trial will evaluate 400mg once a day and a 400mg twice a day dose of GKT831.

Genkyotex chief executive officer Elias Papatheodorou said: "FDA clearance of our IND application is an important milestone for Genkyotex.

"Demonstrating the clinical efficacy of GKT831 in fibrotic liver disease is our primary objective in this study.

"FDA clearance of our IND application is an important milestone for Genkyotex."

"Importantly, the extended treatment duration and large study size will allow us to assess the effects of GKT831 on liver inflammation and fibrosis, which are important unaddressed therapeutic targets in PBC patients.”

The trial's primary objective is the demonstration of therapeutic activity through a decrease in the liver injury marker known as gamma glutamyl transpeptidase.

The secondary efficacy endpoints include liver inflammation markers such as CK-18, hs-CRP, ALT and non-invasive liver fibrosis markers such as liver fibrosis score, transient elastography and circulating collagen fragments.

The interim top-line results are expected to be available in the first half of next year, and the full results available in the second half.

Image: Micrograph of PBC. Photo: courtesy of Nephron.