XORTX aims to file gout drug NDA within 12 months

Dr. Allen Davidoff reports XORTX PROVIDES CORPORATE UPDATE AND PLANNED ACTIVITIES FOR 2025 / 2026 XORTX Therapeutics Inc. has provided a comprehensive update on its 2025 progress and strategic goals for 2026. The first half of 2025 has been marked by intense focus and significant advancement, solidifying a robust plan to accelerate the lead gout program toward a NDA (new drug application) filing, aiming to propel technology toward revenue generation and foster substantial shareholder value. Strategic acceleration of the gout program: XRx-026 and Xorlo In the first quarter of the year, XORTX pivoted to prioritize and accelerate its gout program, XRx-026, leveraging significant progress in formulation development and clinical validation of its proprietary Xorlo formulation. Xorlo, an oral oxypurinol formulation, shows increased oral bioavailability, a key differentiator for improved patient outcomes. XORTX is committed to developing a needed gout therapy for patients who are intolerant to existing treatments. The XRx-026 program is approximately 12 months from filing a NDA (new drug application) with the United States Food and Drug Administration (the FDA) for Xorlo marketing approval, targeting an estimated $700-million-(U.S.)-per-year market opportunity. 2025 achievements This year, the company has made significant strides in advancing our mission to deliver high-value therapies: Strategic focus on XRx-026 gout program: XRx-026 program advanced to address gout in patients who are intolerant to allopurinol, targeting a $700-million (U.S.) per year market opportunity. Engaged with the FDA through a Type B meeting (held March 31, 2025) to clarify the regulatory path for a NDA via the 505(b)(2) pathway, with responses received on April 30, 2025, confirming key steps for Xorlo approval. Conducted the XRX-OXY-101 pharmacokinetics clinical trial to support NDA preparation. Intellectual property advancements: On Dec. 19, 2024, the company submitted a patent co-operation treaty (PCT) application for international protection, leveraging clinical data linking aberrant purine metabolism to kidney disease progression. On April 28, 2025, the European Patent Office granted a patent, "Formulations of Xanthine Oxidase Inhibitors" for renal and related diseases, thus strengthening our portfolio for Xorlo in gout and other conditions. Corporate enhancements: Strengthened the company's leadership with the appointment of Michael Bumby, a biotech/pharma veteran with over 20 years of experience, including 14 years at Eli Lilly, on Dec. 19, 2024; Welcomed Abigail Jenkins to the company's board of directors on April 8, 2024, adding strategic expertise; Closed a $925,000 (U.S.) non-brokered LIFE public offering on July 21, 2025, to support continuing initiatives. Clinical and manufacturing progress: Validated Xorlo formulation and advanced the company's XRx-026 (gout) and XRx-008 (autosomal dominant polycystic kidney disease) programs. 2025/2026 goals and action plan Looking ahead, XORTX is focused on advancing Xorlo within the XRx-026 program toward a NDA filing and market approval within approximately 12 months. The company's key objectives include: Investigational new drug (IND) application: The company will prepare and submit an IND (investigative new drug) application to the FDA, incorporating novel formulation data, pharmacology, toxicology and clinical results from the XRX-OXY-101 trial to support further clinical studies. XRX-OXY-102 clinical trial: The company plans to initiate a clinical trial in the second half of 2025 to study Xorlo pharmacokinetics in fed and fasted states. This trial will provide critical data for population-based pharmacokinetic modelling and support future FDA and European Medicines Agency (EMA) submissions. Chemistry, manufacturing and controls (CMC): In parallel with items 1 and 2, the company will produce clinical drug supplies under the IND, scale-up commercial supplies, and conduct validation and stability testing for Xorlo, adhering to GMP standards to support the company's planned NDA filing. Commercialization preparations: ;To prepare for a potential 2026 NDA filing under the FDA's 505(b)(2) pathway, the company will conduct commercialization studies, including interviews with nephrologists, patients and payers to analyze pricing, reimbursement, and branding strategies. Product launch planning, including brand name selection, will also commence. European market strategy: The company will engage with the EMA to define the regulatory path for Xorlo approval in the European Union, including necessary clinical studies and reimbursement conditions, with activities continuing through 2025/2026. To achieve these goals, XORTX will strategically pursue non-dilutive and dilutive funding, expanding discussions for partnerships with major pharmaceutical/biotech companies with global reach. Such partnerships are critical accelerants for commercialization. XORTX also plans to increase financial and healthcare conference participation to strengthen and expand its investor base. Items 2 through 5 are subject to available funding. Dr. Allen Davidoff, chief executive officer of XORTX, stated: "We are confident that our strategic decisions and operational advancements in 2025 position the company for a truly transformative 2026 and beyond. XORTX remains steadfast in its commitment to developing innovative therapies that make a meaningful difference for patients while delivering significant returns for shareholders. We thank shareholders for the continued support and look forward to sharing further updates as we progress toward these milestones." About XORTX Therapeutics Inc. XORTX is a pharmaceutical company with three clinically advanced products in development: 1) the company's 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, the company is dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases. We seek Safe Harbor.