Genkyotex (Euronext Paris & Brussels: FR0013399474 – GKTX) announced today the final results of its Phase 2 trial of GKT831 in primary biliary cholangitis (PBC). These data include pre-determined secondary efficacy analyses that were not previously available, as well as full safety data.
The efficacy results demonstrate that GKT831 at the 400mg twice a day (BID) dose achieved statistically significant reductions in gamma glutamyl transpeptidase (GGT) (p<0.002) and alkaline phosphatase (ALP) (p<0.001) compared to placebo over the 24-week treatment period.
GKT831 at 400mg BID achieved a 22% reduction in liver stiffness as compared to a 4% increase for placebo (p=0.038) in a pre-defined patient population with an estimated liver fibrosis stage of F3 or higher. This anti-fibrotic effect of GKT831 was previously reported in the top-line results.
The final Quality of Life (QoL) metrics as measured by the PBC-40 questionnaire show improvements in all QoL domain scores at weeks 12 and 24 for the 400mg BID dose. Importantly, improving QoL, in particular fatigue, is a significant unmet need in PBC.
GKT831 was well tolerated at all doses, with 119 adverse events (AEs) in the 400mg once a day (OD) and 100 AEs in the 400mg BID compared to 120 AEs in the placebo group. Two serious adverse events (SAEs) were reported, both deemed unrelated to study, one case of urinary tract infection requiring hospitalization in the placebo group, and the other multiple bone fractures related to a traffic accident in the 400mg BID group.
A review of safety laboratory results, vital signs, physical examination, and ECG did not identify any safety signals associated with GKT831 at 400mg OD or 400mg BID.
Philippe Wiesel, Chief Medical Officer of Genkyotex, said: “The full efficacy and safety data from our Phase 2 PBC trial highlight the potential of GKT831 as a possible treatment for multiple complex and difficult to treat fibrotic disorders, including NASH and PSC. We look forward to complete the evaluation of GKT831 in late-stage clinical trials.”