Dr. Allen Davidoff reports

XORTX ADDS LATE STAGE GOUT PROGRAM TO PIPELINE

XORTX Therapeutics Inc . has launched a new late-stage program to treat gout. The new drug development program -- XRx-026 -- will focus on the treatment of individuals who have gout and are intolerant to allopurinol.

With the clinical development of XORLO having advanced sufficiently, including completion of a pivotal program, XORTX is initiating discussions for the XRx-026 program with the U.S. Food and Drug Administration (FDA) regarding preparation of a new drug application (NDA). Pending FDA feedback, the company will also seek FDA orphan drug designation (ODD) and NDA marketing approval for the XRx-026 program. Previously, oxypurinol was granted ODD for allopurinol-intolerant gout.

Dr. Allen Davidoff, chief executive officer, commented: "Oxypurinol has been demonstrated to be safe and effective in clinical studies focused on the treatment of individuals with gout who are intolerant to allopurinol. Intolerance to allopurinol remains an important issue for many patients and physicians and the XRx-026 program has demonstrated the potential to address this unmet medical need. XORTX will consult with the FDA during the first half of 2025 regarding requirements to file a NDA."

About hyperuricemia, gout and health consequences

The breakdown of nucleotides in the blood occurs through purine metabolism and results in the formation of uric acid by the xanthine oxidase enzyme. However, chronically high blood uric acid concentrations (hyperuricemia) have been associated with health consequences, including gout, kidney stones, diabetes, cardiovascular disease and renal failure. Worldwide, approximately 14 per cent of individuals have hyperuricemia and an estimated 1 per cent to 2 per cent have gout. Lowering blood levels of uric acid in gout patients is strongly correlated with improved health outcomes.

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: (i) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; (ii) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and (iii) oral xanthine oxidase inhibitors (XOIs) that are used to inhibit the production of uric acid. 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 per cent to 5 per cent of patients cannot tolerate allopurinol. An alternative XOI, febuxostat, launched in the United States in 2009 with the hope of treating allopurinol-intolerant patients; however, while febuxostat achieved peak sales of approximately $450-million (U.S.), it now 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) the company's lead program, XRx-008, for ADPKD (autosomal dominant polycystic kidney disease); (2) the company's XRx-026 program for the treatment of allopurinol-intolerant gout; and (3) the company's secondary program in XRx-101 for acute kidney and other acute organ injury associated with coronavirus/COVID-19 infection. In addition, XRx-225 is a preclinical-stage program for Type 2 diabetic nephropathy. XORTX is working to advance its clinical-development-stage products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, the company is dedicated to developing medications to improve the quality of life and health of kidney disease patients and individuals with gout.

We seek Safe Harbor.