Genkyotex Meets Both Primary and Secondary Interim Efficacy Endpoints in Phase 2 Trial of GKT831 in Primary Biliary Cholangitis

Genkyotex announced that its lead product candidate GKT831, a NOX1/4 inhibitor, met both the primary and secondary interim efficacy endpoints with high statistical significance after only 6 weeks of treatment. The data establish GKT831 as an attractive therapeutic option in a broad PBC population and support its development in multiple fibrotic diseases including NASH and IPF.

GKT831 achieved even greater GGT reductions (29%, p<0.01 vs placebo) in patients with higher baseline GGT (≥ 2.5 XULN, n=68), suggesting that it may also benefit patients with more advanced disease, the patient population with the highest medical need and limited therapeutic options.

In addition, the progressive reductions from baseline to week 2 and to week 6 suggest that further improvements can be achieved with continued treatment. Specifically, the 400mg twice daily dose (BID) achieved a 15% reduction in GGT at week 2 and a 23% reduction at week 6. ALP reductions were 12% at week 2 and 17% at week 6.

The interim analysis was conducted in 92 patients after 6 weeks of treatment. Final study results after 24 weeks of treatment in 111 PBC patients are expected in Spring 2019. The final results will also include additional endpoints assessing quality of life (fatigue and pruritus), fibrosis and cholestasis.

Elias Papatheodorou, Chief Executive Officer of Genkyotex, commented: “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).”

Philippe Wiesel, Chief Medical Officer of Genkyotex, commented: “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 2 trial scheduled in H1 2019, the continuation of the JDRF funded DKD Phase 2 trial, and of course, seeing the final data of the PBC Phase 2 trial at week 24 in Spring 2019.”