Metabolic Syndrome

● XORTX will focus on key steps to advance a NDA filing for Gout indication ● CALGARY, Alberta, April 30, 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 gout and progressive kidney disease, is pleased to announce it has received responses from the US Food and Drug Administration (the “FDA”) 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 a NDA to gain marketing approval for XORLO™ in the US using the FDA 505(b)2 development pathway. With this FDA guidance, XORTX will now advance the following key steps to support the NDA application and its submission for the XRx-026 program for gout. i) Finalize meeting minutes with the FDA regarding the agency and XORTX’s responses; ii) Prepare and file an Investigative New Drug (“IND”) application for the XRx-026 program;iii) Characterize pharmacokinetics of the commercial tablet formulation of XORLO™ in absence and in the presence of food;iv) Manufacture, validate commercial supplies of drug accompanied by stability data in parallel with the NDA preparation; and v) Prepare and file a NDA for marketing approval of XORLO™.Dr. Allen Davidoff, CEO 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 US it is estimated that approximately 44 million individuals have circulating uric acid above the normal range(1). The prevalence of gout was 3.9% or 9.2 million individuals. Mean serum urate levels were 6.0 mg/dL among men and 4.8 mg/dL among women, with hyperuricemia prevalences of 20.2% and 20.0%, respectively. The prevalence of ULT use among patients with gout was 33% during 2007 to 2014 and remained stable over time (P for trend >0.05)(1). 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(2), decreased physical function(2,3), increased healthcare costs(4), and lost economic productivity(5). Furthermore, gout is strongly associated with the metabolic syndrome(5), and may contribute to myocardial infarction(6,7), type 2 diabetes mellitus(8), chronic kidney disease(9), and premature mortality(6,10,11). About the XRx-026 Program and XORLOTM 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 to 5% of patients cannot tolerate Allopurinol. An alternative XOI, Febuxostat, was launched in the US in 2009 with the hope of treating individuals with gout, however while Febuxostat achieved peak sales greater than US$450 million1 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 (e.g., investigational new drug application (IND), NDA, biologics license application (BLA)) or combination of closely related products developed by the same sponsor or applicant (e.g., 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. References: (1) Chen-Xu M_Prevalence of Gout and Hyperuricemia in the US_ArthritisRheumatol_71-6_991-999_2019_nihms-1002533(2) Singh JA. Quality of life and quality of care for patients with gout. Curr Rheumatol Rep. 2009 4;11(2):154–60. [PubMed: 19296889] (3) Burke BT, Köttgen A, Law A, Windham BG, Segev D, Baer AN, et al. Physical Function, Hyperuricemia, and Gout in Older Adults. Arthritis Care Res. 2015 12;67(12):1730–8. (4) Rai SK, Burns LC, De Vera MA, Haji A, Giustini D, Choi HK. The economic burden of gout: A systematic review. Semin Arthritis Rheum. 2015 8;45(1):75–80. [PubMed: 25912932] (5) Choi HK, Ford ES. Prevalence of the metabolic syndrome in individuals with hyperuricemia. Am J Med. 2007 5;120(5):442–7. [PubMed: 17466656] (6) Choi HK, Curhan G. Independent impact of gout on mortality and risk for coronary heart disease. Circulation. 2007 8 21;116(8):894–900. [PubMed: 17698728] (7) Liu S-C, Xia L, Zhang J, Lu X-H, Hu D-K, Zhang H-T, et al. Gout and Risk of Myocardial Infarction: A Systematic Review and Meta-Analysis of Cohort Studies. Pizzi C, editor. PLOS ONE. 2015 7 31;10(7):e0134088. [PubMed: 26230580] (8) Choi HK, De Vera MA, Krishnan E. Gout and the risk of type 2 diabetes among men with a high cardiovascular risk profile. Rheumatology. 2008 8 13;47(10):1567–70. [PubMed: 18710901] (9) Roughley MJ, Belcher J, Mallen CD, Roddy E. Gout and risk of chronic kidney disease and nephrolithiasis: meta-analysis of observational studies. Arthritis Res Ther. 2015 12;17(1). (10) Kuo C-F, See L-C, Luo S-F, Ko Y-S, Lin Y-S, Hwang J-S, et al. Gout: an independent risk factor for all-cause and cardiovascular mortality. Rheumatology. 2010 1;49(1):141–6. [PubMed: 19933595] (11) Fisher MC, Rai SK, Lu N, Zhang Y, Choi HK. The unclosing premature mortality gap in gout: a general population-based study. Ann Rheum Dis. 2017 7;76(7):1289–94. [PubMed: 28122760]About XORTX Therapeutics Inc. XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our 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 pre-clinical 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, we are dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases. 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 Source: Takeda Pharmaceutical Company 2018 Annual Report.

COPENHAGEN, Denmark, April 29, 2025 /PRNewswire/ -- The Novo Nordisk Foundation today announced the latest edition of its annual Challenge Programme, where for the first time it is inviting researchers from outside Denmark, in the European Schengen area, Ireland and the UK, to lead collaborations applying for grants to study four major themes. As part of its ongoing internationalisation, the Novo Nordisk Foundation is now expanding its Challenge Programme, the Foundation's largest open competition grant programme. The Programme supports ambitious research projects that focus on creating new knowledge on major global challenges based on annually selected themes. This year, the budget for each grant has been increased by 25% to up to DKK 75 million (€10 million) over six years, bringing the overall budget to DKK 600 million (€80 million). Mads Krogsgaard Thomsen, CEO, Novo Nordisk Foundation, says: "The Novo Nordisk Foundation is dedicated to help solving some of the major health and planetary challenges facing the world. The expansion of the Challenge Programme outside Denmark demonstrates that we are deepening our commitment to this global mission." With themes changing every year, the Challenge Programme provides substantial, long-term funding to enable scientific depth and focus and to facilitate synergy between the research partners. The four themes for the 2026 edition are: Harnessing biology for climate-resilient and healthy soils Unravelling the pathways of human invasive fungal diseases Modelling human cardiometabolic disease Biological systems under non-equilibrium conditions The Challenge Programme calls for applications in open competition. Expressions of interest are invited by 8 October 2025, with the final awards announcement expected in May 2026. The Novo Nordisk Foundation established the Challenge Programme in 2014. Since then, the Foundation has awarded close to DKK 2.8 billion (€375 million) for close to 50 different projects through the Programme. More information on the 2024 grant recipients can be found on the Novo Nordisk Foundation website: Challenge Programme 2024. About the Novo Nordisk Foundation Established in Denmark in 1924, the Novo Nordisk Foundation is an enterprise foundation with philanthropic objectives. The vision of the Foundation is to improve people's health and the sustainability of society and the planet. The Foundation's mission is to progress research and innovation in the prevention and treatment of cardiometabolic and infectious diseases as well as to advance knowledge and solutions to support a green transformation of society. In recent years, the Novo Nordisk Foundation has significantly increased its grants both in Denmark and internationally. In 2024, on its 100th anniversary, the Foundation reached a new milestone, awarding close to DKK 10.1 billion (€1.35 billion) in grants and philanthropic investments to support nearly 1,800 new projects, aimed at improving people's health and the sustainability of society and the planet. About the Challenge Programme The Programme targets ambitious research projects to give leading researchers the opportunity to assemble a strong team that can collaborate to address some of the major societal and planetary challenges. Every year, new themes are defined that comprise the framework for calls for applications in open competition within that one year. For the first time, applications are invited from researchers based outside Denmark, in the European Schengen area, Ireland and the UK, to lead collaborations applying for grants, provided a co-applicant is from Denmark. In 2026, the Novo Nordisk Foundation will award up to DKK 600 million (€80 million) through the Challenge Programme in total. Each project can receive up to DKK 75 million (€10 million) for a six-year period. SOURCE Novo Nordisk Foundation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Pictured: White House/ iStock, Danny Kronstrom Nevertheless, Albert Bourla has been lobbying the U.S. government—as high as President Donald Trump himself—to skip the pharmaceutical industry tariffs, which Trump has threatened, in part as a way to shore up U.S. national security. President Donald Trump’s national security concerns for the pharmaceutical supply chain are “legitimate,” Pfizer CEO Albert Bourla said Tuesday. The chief executive, who is also the chair of the PhRMA organization, noted that these concerns go back to Trump’s first term and continued under former President Joe Biden. “From my discussion with the highest level of the government, they’re confirming that this is their primary concern, and I think it’s legitimate,” Bourla said on a Tuesday morning earnings call. “I think no country wants to have critical medicines produced outside of the country.” Trump is probing the link between the pharmaceutical supply chain and national security via a Section 232 filing with the U.S. Department of Commerce. The result could form the basis of the administration’s tariff policy. Pfizer has accounted for about $150 million in tariff impacts for 2025, CFO Dave Denton noted. That number is included in Pfizer’s guidance already, and the company is “trending towards the top end” of that, he explained. Bourla would not provide concrete guidance on what Pfizer expects. “We have made statements in the past. We have been burned,” he said. But he promised the company has detailed contingency plans prepared that will minimize the impact. “I’m right now focusing more on the fact that we should not have tariffs, and only if we have [them] should we try to implement measures,” Bourla said. “I’m sure all companies are doing the same.” The CEO was encouraged to see pharmaceuticals omitted from the broader tariffs that went into place on April 2. He said the import taxes were placed on “everything we can see or feel or dream, except two things: pharmaceuticals and semiconductor[s].” Then, the administration moved on April 14 to conduct the Section 232 investigation. This move, according to Bourla, clarified the concern and made the potential impact more manageable. “Clearly, there is [tariff] risk going forward, but it’s a very, very different ball game,” Bourla said. The Trump administration produced a list of critical medicines during its first term that were being manufactured outside of the U.S. That has not yet been done this time around, but Bourla expressed confidence that “there is a very clear distinction as to what comes from friendly countries or what comes from adversary countries.” This assertion was challenged by Evercore ISI analyst Umer Raffat, who noted that the broader tariffs have been placed against traditional allies. Why did Bourla think the same would not happen for pharma, Raffat wondered. “It’s up to the government to decide what they want to do, but . . . the national security concerns is very clear in my discussions,” Bourla deflected. The Right Decision Elsewhere, Bourla and his executive team addressed the recent discontinuation of lead obesity asset danuglipron. The drug was cut from the pipeline two weeks ago based on a single asymptomatic incident of drug-induced liver injury. Chief Scientific Officer Chris Boshoff said the discontinuation “was the right decision for Pfizer based on totality of data.” “Although we’re not 100% sure this is due to danuglipron, the risk was high enough for us to discontinue the program while still being committed to obesity and employing our global resources for other opportunities,” Boshoff explained. The swift move was also a demonstration of the R&D program’s discipline. Boshoff took over the science organization on January 1; Bourla said he has moved swiftly to make his mark. Boshoff reiterated Pfizer’s commitment to obesity and cardiometabolic disease despite the loss of the lead program. To replace the product, the business development team will be looking for efficacy, safety and market differentiation. Andrew Baum, chief strategy and innovation officer, said Pfizer is exploring incretins and other modalities, looking for assets that can improve on scheduling, tolerability, muscle preservation and more. The company is also considering commercial differentiation, which Baum clarified as the indications that Pfizer would pursue. “The future will also become more personalized based on combinations addressing specific diseases associated with obesity and with metabolic dysfunction, including neurology, cardiovascular and respiratory,” Boshoff said.