Febuxostat

XORTX to focus on late stage allopurinol intolerant gout program; discussion with FDA planned for first half 2025 regarding NDA filingCALGARY, Alberta, Jan. 06, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces the launch of 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 XORLOTM 1 having advanced sufficiently, including completion of a pivotal program, XORTX is initiating discussions for the XRx-026 program with the US 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 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% of individuals have hyperuricemia and an estimated 1 to 2% 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 3 million prescriptions written per year in North America, however 3 to 5% of patients cannot tolerate allopurinol. An alternative XOI, Febuxostat, launched in the US in 2009 with the hope of treating allopurinol intolerant patients, however while Febuxostat achieved peak sales of approximately US$450 million2, 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) our lead, XRx-008 program for ADPKD; 2) our XRx-026 program for the treatment of allopurinol intolerant gout; and 3) our 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 pre-clinical 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, we are dedicated to developing medications to improve the quality of life and health of kidney disease patients and individuals with gout. Additional information on XORTX is available at www.xortx.com. For more information, please contact: Allen Davidoff, CEONick Rigopulos, Director of Communicationsadavidoff@xortx.com or +1 403 455 7727nick@alpineequityadv.com or +1 617 901 0785 Neither the TSX Venture Exchange nor Nasdaq has approved or disapproved the contents of this news release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein. Forward Looking Statements This press release contains express or implied forward-looking statements pursuant to applicable securities laws. These forward-looking statements include, but are not limited to, the Company's beliefs, plans, goals, objectives, expectations, assumptions, estimates, intentions, future performance, other statements that are not historical facts and statements identified by words such as "expects", "anticipates", "intends", "plans", "believes", "seeks", "estimates" or words of similar meaning. These forward-looking statements and their implications are based on the current expectations of the management of XORTX only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks, uncertainties, and other factors include, but are not limited to, our ability to obtain additional financing; the accuracy of our estimates regarding expenses, future revenues and capital requirements; the success and timing of our preclinical studies and clinical trials; the performance of third-party manufacturers and contract research organizations; our plans to develop and commercialize our product candidates; our plans to advance research in other kidney disease applications; and, our ability to obtain and maintain intellectual property protection for our product candidates. Except as otherwise required by applicable law and stock exchange rules, XORTX undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting XORTX is contained under the heading “Risk Factors” in XORTX’s Annual Report on Form 20-F filed with the SEC, which is available on the SEC's website, www.sec.gov (including any documents forming a part thereof or incorporated by reference therein), as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada, which are available on www.sedarplus.ca. 1 XORLOTM is XORTX’s proprietary formulation of oxypurinol, that has granted US and EU patents. 2 Source: Takeda Pharmaceutical Company 2018 Annual Report.

ZHEJIANG, China--( BUSINESS WIRE )--Atom Therapeutics Co. Ltd (Atom Therapeutics), a clinical stage biotechnology company developing best-in-class treatments for inflammatory and metabolic diseases, announced today the company entered into an exclusive commercialization collaboration agreement with a subsidiary of China Medical System Holdings Ltd (CMS) for lingdolinurad (ABP-671), a novel oral small molecule URAT1 inhibitor for the treatment of chronic gout and hyperuricemia, in mainland China, Hong Kong Special Administrative Region and Macao Special Administrative Region. Atom Therapeutics and CMS will execute a Contract Sales Organization (CSO) business model. Under the agreement, Atom Therapeutics will receive a substantial sum from CMS through upfront and milestone payments, and will pay marketing and sales service fees to CMS. Lingdolinurad is expected to generate substantial income and revenue from the sales collaborated with CMS. This event marks a significant advancement for Atom Therapeutics in the commercialization of ABP-671. Atom Therapeutics is currently conducting multi-center Phase 2b/3 clinical trials of lingdolinurad worldwide, including in China and the United States. Lingolinurad reduces renal re-absorption of uric acid by inhibiting URAT1 (urate anion transporter 1). Results from multiple clinical trials have demonstrated that lingdolinurad is safe and effective at multiple dose levels (from 1 mg to 12 mg). The 2 mg once-daily tablet is expected to be as effective as benzbromarone (80 mg) and febuxostat (80 mg). The efficacy of lingdolinurad is maintained for over 24 hours without significant safety concerns, positioning it as a potentially safer and more effective treatment option for patients with chronic gout and hyperuricemia. CMS is a platform company linking pharmaceutical innovation and commercialization with a strong product lifecycle management capability, outstanding market promotion capabilities, and extensive commercialization experience. Through this collaboration, Atom Therapeutics will leverage the broad market channels and rich commercialization experience of CMS to achieve rapid promotion and broad clinical application of ABP-671 following its launch. Atom Therapeutics CEO, Chairman and Founder William Dongfang Shi, Ph.D., commented, “This collaboration represents a deep integration of our two companies' business operations and strategic initiatives. We look forward to advancing the implementation and application of ABP-671 through this partnership, delivering more effective treatment options for patients with gout and hyperuricemia as soon as possible.” About Atom Therapeutics Atom Therapeutics Co., Ltd (originally named Jiangsu Atom Bioscience and Pharmaceutical Co., Ltd) officially changed its name on October 8, 2024. Atom Therapeutics is a fast-growing innovative drug company focused on development of best-in-class small molecule therapeutics for treatment of inflammatory and metabolic diseases. The company’s lead product, lingdolinurad, is in late-stage clinical development for treatment of chronic gout. Another small molecule ABP-745, for anti-inflammatory and autoimmune conditions, has completed Phase 1 clinical trials in the US and demonstrated good pharmacokinetics and safety, and will enter Phase 2 clinical trials soon. For more information, please visit: https://www.atombp.com or atombp.us . About CMS CMS is a platform company linking pharmaceutical innovation and commercialization with strong product lifecycle management capability, dedicated to providing competitive products and services to meet unmet medical needs. CMS focuses on the global first-in-class and best-in-class innovative products, and efficiently promotes the clinical research, development and commercialization of innovative products, enabling the continuous transformation of scientific research into clinical practices to benefit patients. CMS deeply engages in several specialty therapeutic fields, and has developed proven commercialization capabilities, extensive networks and expert resources, resulting in leading academic and market positions for its major marketed products. CMS continues to promote the in-depth development of its advantageous specialty fields and expand business boundaries. While strengthening the competitiveness of the cardio-cerebrovascular/gastroenterology business, CMS independently operates its dermatology and medical aesthetics business, and ophthalmology business, aiming to gain leading positions in specialty therapeutic fields, whilst enhancing the scale and efficiency. At the same time, CMS has expanded its business territory to the Southeast Asian market, striving to become a "bridgehead" for global pharmaceutical companies to enter the Southeast Asian market, further escorting the sustainable and healthy development of the Group. For more information, please visit: https:/veb.cms.net.cn.