Genkyotex (Euronext Paris & Brussels: FR0013399474 – GKTX), a biopharmaceutical company and the leader in NOX therapies, today announced the publication of preclinical efficacy data with GKT831 for cholestatic fibrosis. The article “Activated Hepatic Stellate Cells and Portal Fibroblasts contribute to cholestatic liver fibrosis in MDR2 knockout mice” was published in the Journal of Hepatology1 . 

These preclinical results are consistent with the reduction in liver stiffness achieved in Phase 2 with GKT831 after just 24 weeks of treatment in PBC patients. In patients with an estimated F score of F3 (severe) or greater, GKT831 achieved a 21% reduction in liver stiffness. In the 400mg BID dose, GKT831 achieved an absolute reduction of 2.7 kPa compared to an increase of 0.4 kPa for the placebo group.

Philippe WIesel, CMO of Genkyotex, commented: “The MDR2 KO mouse model is the most relevant model of advanced cholestatic fibrosis and is particularly relevant to human diseases like PBC and PSC where patients develop severe fibrosis. The data indicate that GKT831 achieves rapid fibrosis reversal which is in line with the clinical evidence of anti-fibrotic activity obtained in our 24-week PBC trial.”

In the MDR2 KO mouse model, GKT831 treatment was initiated at week 12 when advanced liver fibrosis was already established. After only 4 weeks of treatment, GKT831 was able to deactivate myofibroblasts and reverse fibrosis, indicating rapid anti-fibrotic activity.

GKT831 had already shown marked efficacy in multiple mouse models of inflammatory and fibrotic disorders.