XORTX Highlights Genetic Regulation of Xanthine Oxidase and Purine Metabolism Therapy

XORTX Therapeutics Inc., a clinical-stage pharmaceutical company specializing in advanced treatments for progressive kidney disease, recently announced pivotal findings from independent research. This research, which has been peer-reviewed and published, underscores the importance of genetic factors in the over-expression of the enzyme xanthine oxidase (XO) and its role in various diseases, including chronic kidney disease, diabetic kidney disease, and polycystic kidney disease (PKD). These insights align with XORTX's strategy of targeting XO to treat these conditions.

Xanthine oxidase is a critical enzyme in the metabolic pathway of uric acid, essential for breaking down purine nucleotides. During this process, the enzyme produces uric acid (UA) and reactive oxygen species (ROS), which can have harmful effects on the circulatory system and tissues during disease progression. Research funded by XORTX in rodent models of PKD has identified the over-expression or hyperactivity of XO as a significant target for potential treatments.

Although over-expression of XO in human PKD has not yet been documented, studies by Wang et al. have indicated a genetic link to PKD1. New research further connects genetic factors to specific populations and conditions, showing that elevated XO expression is associated with hyperuricemia, sepsis, organ failure, sepsis-related acute respiratory distress syndrome (ARDS), kidney dysfunction, diabetes, and kidney failure. These findings suggest that a precision-medicine approach, guided by genetic risk variants, could be effective in tailoring treatments for these conditions.

Allen Davidoff, Ph.D., CEO of XORTX, commented on these developments, highlighting the combination of groundbreaking research in autosomal dominant polycystic kidney disease (ADPKD) and the company's own studies. He emphasized that targeting XO pharmacologically offers substantial therapeutic potential, particularly in non-diabetic and diabetic kidney diseases where increased XO activity is prevalent. These discoveries provide an opportunity to create personalized treatments for individuals genetically predisposed to these diseases, underlining the necessity of xanthine oxidase inhibition.

Davidoff pointed out that XORTX is well-positioned to deliver targeted therapeutics, thanks to its expertise in developing XO inhibitors and its robust patent portfolio. The company's therapeutic platform aims to advance its clinical trial for patients with ADPKD, testing XORLO™, a proprietary formulation of oxypurinol. This trial will also help further understand the role of newly identified genetic factors in individuals with PKD.

XORTX Therapeutics Inc. currently develops two main clinical products: XRx-008 for ADPKD and XRx-101 for acute kidney and other organ injuries associated with COVID-19. Additionally, XRx-225 is in the pre-clinical stage for Type 2 Diabetic Nephropathy. The company's focus is on advancing clinical development-stage products that target aberrant purine metabolism and xanthine oxidase to reduce or inhibit uric acid production. XORTX is committed to improving patients' quality of life and health outcomes through innovative medications.

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