Genkyotex, a biopharmaceutical company and the leader in NOX therapies, announced that the US Food & Drug Administration (FDA) has accepted its Investigational New Drug (IND) Application, which allows Genkyotex to proceed with a phase 2 clinical trial of GKT831, its NOX1 and NOX4 inhibitor, in patients with Primary Biliary Cholangitis (PBC). Genkyotex expects to initiate this study prior to the end of the second quarter 2017, with interim top-line results anticipated in the first half of 2018, and full results anticipated in the second half of 2018.

A substantial proportion of patients currently receiving approved anti-cholestatic PBC therapies show sustained liver injury and progressive fibrosis. In multiple rodent models of liver disease, treatment with GKT831 achieved marked anti-inflammatory and anti-fibrotic effects, as well as statistically significant reductions in markers of liver injury and inflammation in patients with type 2 diabetes.

The planned phase 2 trial will be a 24-week, double-blind, placebo controlled, multi-center trial evaluating the safety and efficacy of GKT831 in patients with PBC and inadequate response to ursodeoxycholic acid. A total of 102 PBC patients will be enrolled in this international study and allocated to placebo or one of two doses of GKT831 (400mg once a day or 400mg twice a day). The primary objective of the trial will be to demonstrate therapeutic activity through a reduction of gamma glutamyl transpeptidade, a marker of liver injury, which also reflects oxidative stress. Secondary efficacy endpoints include markers of liver inflammation and injury (CK-18, hs-CRP, ALT), non-invasive markers of the liver fibrosis (Enhanced Liver Fibrosis score, transient elastography and circulating collagen fragments).

"FDA clearance of our IND application is an important milestone for Genkyotex,” said Elias Papatheodorou, Genkyotex’s chief executive officer. “We are continuing with preparations aimed at initiating our phase 2 trial at over 50 investigational centers in North America and Europe.

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