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  • 25 July 2019
    Genkyotex announces positive Post-Hoc Analysis of PBC Phase 2 Trial and Reports Cash Position at June 30, 2019

    Genkyotex (Euronext Paris & Brussels: FR0013399474 – GKTX), a biopharmaceutical company and the leader in NOX therapies, announced today positive post-hoc analysis of PBC phase 2 trial. 

    As previously reported, setanaxib achieved clinically meaningful reductions in liver stiffness, statistically significant reduction in gamma glutamyl transpeptidase (GGT) (p<0.002) and alkaline phosphatase (ALP) (p<0.001) over the 24-week treatment period, but did not achieve statistical significance in the reduction of GGT at week 24, the predefined primary efficacy endpoint. This result was unexpected, as setanaxib had achieved statistical significance in GGT reduction (p<0.01) and ALP reduction (p<0.001) at week 6 interim analysis. Therefore, Genkyotex performed a post-hoc analysis to identify the reasons for the loss of statistical significance at week 24, and to further explore the therapeutic benefits achieved with setanaxib.

    The analysis identified a non-normal distribution of GGT values in the 400mg OD dose at week 24, as the cause for the loss of statistical significance at week 24. Statistical significance of p=0.02 is achieved for the primary endpoint for 400mg BID at week 24 when correcting for the non-normal distribution in the 400mg OD group. This correction is achieved with a standard statistical correction method (logtransformation) to minimize the impact of such non-normal data distribution.

    As previously reported, setanaxib 400mg BID achieved an important reduction (-22%) in liver stiffness in patients with advanced disease (≥9.6 kPa at baseline). New analysis shows that in these patients setanaxib also achieves clinically meaningful reductions in GGT (-32%) and ALP (-24%) at week 24. These new data indicate that setanaxib could become an important new therapeutic option for the difficult to treat patient populations with advanced liver fibrosis in PBC and other liver diseases, including advanced NASH. Following these positive results, the PBC phase 3 trial is being planned.

    In addition, Genkyotex announced in July that:

    • the World Health Organization (WHO) recognized NOX inhibitors as a new therapeutic class while approving the new stem “naxib”. The WHO recommended setanaxib as the international nonproprietary name (INN, or generic name) for GKT831.

    • the United States Food and Drug Administration (FDA) approved in July 2019 the Investigational New Drug (IND) application allowing the initiation of the Phase 2 trial with setanaxib in patients with IPF in the coming months.

    Elias Papatheodorou, CEO of Genkyotex, commented: “The full analysis of the data confirms that setanaxib could become an important therapeutic solution in multiple fibrotic indications including NASH. Setanaxib’s effect on liver stiffness and quality of life, along with the excellent safety of the product, positions setanaxib as a unique product candidate in the liver fibrosis landscape. As the leader in NOX therapeutics, we are particularly excited with the WHO’s decision to formally recognize NOX inhibitors as a novel therapeutic class.”

    Research highlights

    Genkyotex continues to explore the therapeutic value of NOX inhibition in additional therapeutic areas, including oncology. The Company expects to secure non-dilutive grant financing to support its ongoing collaborations with academic partners. During the second quarter of 2019, the Company announced the following important publications:

    • The manuscript “Activated Hepatic Stellate Cells and Portal Fibroblasts contribute to cholestatic liver fibrosis in MDR2 knockout mice” was published in May 2019 in the Journal of Hepatology. The regression of liver fibrosis observed in this model of cholestatic liver disease is consistent with the reduction in liver stiffness achieved in Phase 2 with setanaxib after just 24 weeks of treatment in PBC patients.
    • The Company also announced the publication of preclinical studies in Clinics and Research in Hepatology and Gastroenterology showing that its anti-fibrotic drug candidate setanaxib, prevents multiple complications of portal hypertension.
    • The manuscript “Inhibition of host NOX1 blocks tumor growth and enhances checkpoint inhibitor– based immunotherapy » was published in July in the journal Life Science Alliance. These preclinical results show that NOX1 inhibition represents a novel therapeutic strategy to treat colorectal cancer, particularly in combination with checkpoint inhibition.

    Financial highlights

    On June 30, 2019, Genkyotex' cash and cash equivalents totaled €4.5 million vs. €7.3 million on March 31, 2019. The Company’s cash burn was primarily driven by investments in the ongoing phase 2 trial in PBC. Genkyotex expects its current resources to support anticipated operations until April 2020. Upcoming financial meeting and publication

    • September 19, 2019. 2019 half-year results
    • October 24, 2019. Business & cash position update 3rd quarter 2019