GenKyoTex

Genkyotex operates as a clinical-stage biopharmaceutical company focused on developing therapies that target NADPH oxidase (NOX) enzymes. Founded in 2006 by an international team of scientists from Switzerland, the USA, and Japan, the company was established to capitalize on the discovery of the role NOX enzymes play in a variety of diseases. This foundational science revealed that overactive NOX enzymes produce an excess of reactive oxygen species (ROS), which can trigger pathological processes like fibrosis and inflammation across different organs.

The company's core business revolves around its platform of small-molecule NOX inhibitors, designed to modulate and normalize the overproduction of ROS. This therapeutic approach targets the underlying disease mechanisms rather than just the symptoms. Genkyotex's business model is centered on the research, clinical development, and eventual commercialization of these first-in-class drug candidates. Revenue generation is anticipated through partnerships and the successful launch of its proprietary drugs. A significant milestone was achieved in 2020 when Swedish rare disease specialist Calliditas Therapeutics acquired a controlling interest in Genkyotex, eventually taking full ownership. This acquisition, valued at approximately €32 million plus up to €55 million in potential milestone payments, integrated Genkyotex's pipeline into Calliditas's broader strategy for treating orphan diseases.

The lead product candidate is setanaxib (formerly GKT831), an orally available inhibitor of NOX1 and NOX4 enzymes. Setanaxib has demonstrated anti-fibrotic and anti-inflammatory activity in clinical trials, positioning it as a treatment for various conditions. Its primary development focus has been on Primary Biliary Cholangitis (PBC), a chronic orphan liver disease. Positive results from a Phase II trial in PBC showed that setanaxib was well-tolerated and led to reductions in markers of liver inflammation and fibrosis. Beyond PBC, setanaxib is being explored for other fibrotic diseases, including Type 1 Diabetes and Kidney Disease (DKD) and Idiopathic Pulmonary Fibrosis (IPF), with a Phase II trial in IPF being funded by an $8.9 million grant from the U.S. National Institutes of Health (NIH). The recognition of NOX inhibitors as a new therapeutic class by the World Health Organization, which assigned the stem "naxib" to the class with setanaxib as its first representative, underscores the novelty of Genkyotex's approach.

Keywords: NOX inhibitors, setanaxib, Calliditas Therapeutics, fibrosis, primary biliary cholangitis, anti-fibrotic, reactive oxygen species, orphan diseases, NADPH oxidase, liver disease, GKT831, inflammation, clinical trials, biopharmaceutical, drug development, idiopathic pulmonary fibrosis, diabetic kidney disease, small molecules, enzyme inhibition, orphan drug