Dr. Allen Davidoff reports

XORTX PROVIDES UPDATE ON FDA TYPE B MEETING REQUEST

XORTX Therapeutics Inc. has received responses from the U.S. Food and Drug Administration and clarified key steps for a new drug application (NDA) for the company's novel proprietary formulation of oxypurinol for the treatment of gout. The Type B meeting conducted by the FDA, included review of chemistry, manufacturing, pharmacology, toxicology and clinical evidence to support the company's XRx-026 program for the treatment of gout. Responses from the FDA substantially confirmed and clarified the remaining key steps necessary prior to filing an NDA to gain marketing approval for Xorlo in the United States using the FDA 505(b)2 development pathway. With this FDA guidance, XORTX will now advance the following key steps to support the NDA and its submission for the XRx-026 program for gout:

1. Finalize meeting minutes with the FDA regarding the agency and XORTX's responses;
2. Prepare and file an investigative new drug (IND) application for the XRx-026 program;
3. Characterize pharmacokinetics of the commercial tablet formulation of Xorlo in absence and in the presence of food;
4. Manufacture and validate commercial supplies of drug accompanied by stability data in parallel with the NDA preparation;
5. Prepare and file an NDA for marketing approval of Xorlo.

Dr. Allen Davidoff, chief executive officer of XORTX, commented: "The clarity gained during this review of supporting data with the FDA will permit the further advancement of the XRx-026 program and will facilitate the timely completion of key steps necessary for filing the NDA. The company believes that the gout NDA may be filed in the H1 2026. We continue to prioritize our XRx-026 program and advance this much needed therapeutic option for individuals with gout."

The company will provide further updates following final communications with the FDA once meeting minutes are finalized and available.

About hyperuricemia and gout

In the United States, it is estimated that approximately 44 million individuals have circulating uric acid above the normal range. The prevalence of gout was 3.9 per cent, or 9.2 million individuals. Mean serum urate levels were 6.0 milligrams per decilitre among men and 4.8 milligrams per decilitre among women, with hyperuricemia prevalences of 20.2 per cent and 20.0 per cent, respectively. The prevalence of ULT (urate-lowering therapy) use among patients with gout was 33 per cent during 2007 to 2014 and remained stable over time (p for trend greater than 0.05). Gout is an inflammatory arthritis that is triggered by the crystallization of monosodium urate inside the joints and is preceded by hyperuricemia. Gout flares lead to substantial morbidity by causing severe pain, reduced quality of life, decreased physical function, increased health care costs and lost economic productivity. Furthermore, gout is strongly associated with the metabolic syndrome and may contribute to myocardial infarction, Type 2 diabetes mellitus, chronic kidney disease and premature mortality.

About the XRx-026 program and Xorlo

The XRx-026 program is developing Xorlo, a proprietary formulation of oxypurinol to treat individuals suffering from gout. At present, oral xanthine oxidase inhibitors (XOIs) are the preferred therapeutic option used to inhibit the production of uric acid and decrease chronically high uric acid in the circulation. Allopurinol is the most commonly prescribed XOI, with approximately 3.3 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, was launched in the United States in 2009 with the hope of treating individuals with gout; however, while febuxostat achieved peak sales greater than $450-million (U.S.) after its launch, a black box warning due to its associated risk of sudden cardiovascular death resulted in a decline in its use. Xorlo can address this unmet medical need and accelerating advancement of the XRx-026 program through an NDA filing is now a priority for XORTX.

About Type B meetings with the FDA

Type B meetings for each potential application (for example, IND application, NDA and biologics licence application (BLA)) or combination of closely related products developed by the same sponsor or applicant (for example, same active ingredient but different dosage forms being developed concurrently). Typically, it may be appropriate to conduct more than one of some of the Type B meetings for concurrent development of a product for unrelated claims.

About XORTX Therapeutics Inc.

XORTX is a pharmaceutical company with three clinically advanced products in development: (1) the company's lead program, XRx-026, for the treatment of gout; (2) the XRx-008 program for ADPKD (autosomal dominant polycystic kidney disease); 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, the company 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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