GKT831 recorded high statistical significance following six weeks of treatment, said the French biotech company.
The 24-week placebo-controlled trial is being held across multiple countries in 111 PBC patients having inadequate response to ursodeoxycholic acid (UDCA).
Divided into three groups, the patients were either given UDCA plus placebo, UDCA plus GKT831 400mg once a day (OD) or UDCA plus 400mg twice a day (BID).
The primary efficacy endpoint of the mid-stage trial is change in gamma glutamyl transpeptidase (GGT) at week 24. Genkyotex said that GKT831 delivered even greater GGT reductions, of 29%, in patients with higher baseline GGT.
The French firm revealed that the progressive reductions from baseline to week 2 and to week 6 indicate that further improvements can be reached with continued treatment with GKT831.
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.”
Some of the main secondary endpoints included in the trial are additional markers of liver and bile duct injury, markers of inflammation, non-invasive markers of liver fibrosis, the Enhanced Liver Fibrosis (ELF) score, and circulating collagen fragments.
Genkyotex claimed that the data that has come out so far establish GKT831 as an attractive treatment option in a broad PBC population. Further, the data also supports its development in multiple fibrotic diseases such as NASH and IPF, said the French biotech company.
Genkyotex CEO Elias Papatheodorou said: “The clinical data further establish GKT831 as a novel anti-fibrotic candidate and confirm the therapeutic value of NOX inhibition for patients. NOX inhibitors are an emerging therapeutic class able to address many human diseases with unmet medical need.
“Following these interim results we are investigating options in non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC).”