Dr. Allen Davidoff reports

XORTX ANNOUNCES GRANT OF EUROPEAN PATENT

XORTX Therapeutics Inc. has received notification that the patent Xanthine Oxidase Inhibitor (XOI) Formulations will be granted by the European Patent Office. The patent covers compositions and methods of formulating using XORTX's proprietary formulations of xanthine oxidase inhibitors for the treatment of health consequences of chronically high uric acid, gout, renal, cardiovascular and other diseases, where aberrant purine metabolism has been implicated in disease progression.

Dr. Allen Davidoff, chief executive officer of XORTX, stated: "This patent covers compositions of formulations key to XORTX's platform technology, and this issuance strengthens our intellectual property portfolio in the European Union. Importantly, this European patent broadens protection of our first-in-class program for gout, as well as supporting our autosomal-dominant polycystic kidney disease program. This patent focuses on the health consequences that arise from uric acid crystal formation due to hyperuricemia in conditions including gout, kidney stone formation and cardiovascular disease, and covers novel therapeutic solutions developed specifically to address these problems. The granting of this patent provides additional breadth of patent to XORTX for commercialization and partnering opportunities throughout Europe. Including this newly granted patent, XORTX now has five granted patents in the United States and/or EU covering compositions and uses of uric acid lowering agents to treat gout, the health consequences of hyperuricemia and progressive kidney disease."

About hyperuricemia, gout and health consequences

It is estimated that 14 per cent of the adult population has hyperuricemia -- that is, serum uric acid levels greater than the normal range. Approximately 3 per cent of the U.S. population suffer from gout as a result of their hyperuricemia, though not all individuals who have hyperuricemia develop gout. The factors that contribute to hyperuricemia include genetic factors, dietary factors and lifestyle choices. Nucleotides are normally broken down in the blood by the xanthine oxidase enzyme through the purine metabolic pathway. This results in the formation of uric acid. However, if individuals experiences chronically high blood uric acid concentrations (hyperuricemia), they often experience health consequences including gout, kidney stones, diabetes, cardiovascular disease and renal failure. Evidence that unique health consequences may arise from hyperuricemia, independent of the health consequences of gout, suggest that aberrant purine metabolism, hyperuricemia, merits an increased therapeutic focus. Lowering blood levels of uric acid in gout patients is strongly correlated with improved health outcomes.

Recent groundbreaking studies by Wang et al. suggest linkage of genetic factors to the overexpression of XO. Recently, emerging discoveries link genetic factors to specific populations and show that higher XO expression is associated with a variety of conditions including hyperuricemia, general sepsis, organ failure and sepsis associated with acute respiratory distress syndrome, kidney dysfunction, diabetes, polycystic kidney disease, and kidney failure. From a mechanistic standpoint, these studies advocate for a precision medicine approach in which genetic risk variants would guide treatment decisions.

Addressable gout market opportunity

In North America, approximately 3.5 million people suffer from gout due to elevated uric acid levels in blood. The therapeutic options to lower uric acid levels include three major classes of drugs: (1) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; (2) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and (3) oral XOIs that are used to inhibit the production of uric acid by the purine metabolic pathway. XOIs are the preferred first line treatment for gout. Allopurinol is the most commonly prescribed XOI, with approximately three million prescriptions written per year in North America; however, 3 to 5 per cent of patients cannot tolerate allopurinol. An alternative XOI, febuxostat, was launched in the United States in 2009 with the hope of treating allopurinol-intolerant patients. While febuxostat achieved peak sales of approximately $450-million (U.S.), it carries a black box warning due to its associated risk of sudden cardiovascular death and its use has declined significantly. This decline in febuxostat use has created an opportunity for a novel XOI to address the underlying unmet medical need, which the XRx-026 program aims to fill.

About XORTX Therapeutics Inc.

XORTX is a pharmaceutical company with three clinically advanced products in development: (1) its lead program XRx-026 program for the treatment of gout; (2) XRx-008 program for ADPKD; and (3) XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections. In addition, the company is developing XRx-225, a preclinical-stage program for Type 2 diabetic nephropathy. XORTX is working to advance products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, it is dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases.

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