Genkyotex has reported positive interim efficacy results from the Phase II trial of GKT831 for the treatment of primary biliary cholangitis (PBC).

The trial met both its primary and secondary interim efficacy endpoints after six weeks of treatment.

The trial’s primary efficacy endpoint is a change in gamma glutamyl transferase (GGT) at week 24, while secondary endpoints include additional markers of liver and bile duct injury, markers of inflammation, and non-invasive markers of liver fibrosis, including liver stiffness evaluated by transient elastography.

Results showed that GKT831 reduced the level of GGT in patients with higher baseline GGT, indicating its potential for use in patients with advanced disease, as well as patients with the highest medical need and limited therapeutic options.

Progressive reductions from baseline to weeks two and six also imply that further improvements can be achieved with continued treatment with GKT831.

The 400mg twice daily dose (BID) of GKT831 reduced 15% of GGT at week two and 23% at week six, while alkaline phosphatase (ALP) reductions were reported to be 12% at week two and 17% at week six.

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By GlobalData
“The significant effect on the primary endpoint GGT provides clinical confirmation of GKT831’s mechanism of action.”

This data is based on an interim analysis conducted in 92 patients after six weeks of treatment.

Final study results from the trial are expected to be unveiled next year after 24 weeks of treatment have been completed.

The final results will feature additional endpoints data such as evaluating quality of life (fatigue and pruritus), fibrosis and cholestasis.

Genkyotex chief medical officer Philippe Wiesel said: “The significant effect on the primary endpoint GGT, a marker of inflammatory and cholestatic liver injury, provides clinical confirmation of GKT831’s mechanism of action.

“We are very impressed by the highly significant effect on ALP, which indicates cholangiocyte protection. Importantly, ALP is a key endpoint used globally for regulatory approval.

“We are looking forward to the launch of the NIH funded IPF Phase II trial scheduled in H1 2019, the continuation of the JDRF funded DKD Phase II trial, and of course, seeing the final data of the PBC Phase II trial at week 24 in spring 2019.”

The Phase II trial is a 24-week placebo-controlled study being conducted in 62 centres across the US, Canada, Belgium, Germany, Greece, Israel, Italy, Spain, and the UK. It has enrolled 111 patients.