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Last update 15 May 2025

Allopurinol

Last update 15 May 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

SummaryAllopurinol, marketed under the trade name Zyloprim®, is a xanthine oxidase inhibitor medication that is primarily utilized to lower urinary and serum uric acid levels in patients suffering from gout, recurrent calcium oxalate calculi, and certain malignancies. The mechanism of action of this medication involves inhibiting the enzyme xanthine oxidase. Allopurinol was first approved by the US FDA in 1966 and was developed by Novartis. Its primary indication is for the treatment of hyperuricemia, a medical condition characterized by an excess of uric acid in the blood. Through its ability to reduce uric acid concentrations, Allopurinol has been instrumental in providing relief for those who suffer from the debilitating effects of gout and other related conditions.

Drug Type

Small molecule drug

Synonyms

1,5-Dihydro-4H-pyrazolo(3,4-d)pyrimidin-4-one, 1,5-Dihydro-4H-pyrazolo(3,4-d)pyrimidine-4-one, 1H-Pyrazolo(3,4-d)pyrimidin-4-ol

+ [23]

Target

Action

inhibitors

Mechanism

XO inhibitors(Xanthine dehydrogenase inhibitors)

Therapeutic Areas

Immune System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [3]

Active Indication

GoutNephrolithiasisHyperuricemia

Inactive Indication

Acute myocardial infarctionChronic Kidney DiseasesDiabetes Mellitus+ [2]

Originator Organization

Sebela Ireland Ltd.

Active Organization

GlaxoSmithKline KK

Sumitomo Pharma Co., Ltd.Jiangsu Meitong Pharmaceutical Co. Ltd.

+ [1]

Inactive Organization

AstraZeneca PLC

Ardea Biosciences, Inc.

Advancell Advanced In Vitro Cell Technologies SL

License Organization

AstraZeneca PLC

Ironwood Pharmaceuticals, Inc.

Drug Highest PhaseApproved

First Approval Date

United States (19 Aug 1966),

Hyperuricemia

Regulation-

Structure/Sequence

Molecular FormulaC5H4N4O

InChIKeyOFCNXPDARWKPPY-UHFFFAOYSA-N

CAS Registry315-30-0

272

Clinical Trials associated with Allopurinol

IRCT20110807007244N11

/ SuspendedPhase 2/3IIT

Comparison of the effect of erythropoietin and allopurinol on Prooxidant antioxidant balance, interleukin 6, 8 and 1 in infants with hypoxic ischemic encephalopathy

Start Date01 Oct 2025

Sponsor / Collaborator

Mashhad University of Medical Sciences

IRCT20170609034406N12

/ RecruitingPhase 3IIT

The effect of allopurinol in preventing liver fibrosis in non-alcoholic fatty liver disease: A Randomized Placebo-Controlled Clinical Trial

Start Date21 Nov 2024

Sponsor / Collaborator

Tabriz University of Medical Sciences

NCT05888233

/ RecruitingPhase 2IIT

Allopurinol Improves Diastolic Function in African Americans With Resistant Hypertension

African American adults in the United States have the highest prevalence rate of high blood pressure (hypertension) and heart failure in the world. African Americans with treatment resistant hypertension have higher levels of the enzyme - xanthine oxidase compared to Caucasians. This trial will test if administration of the xanthine oxidase inhibitor - Allopurinol (commonly used in the treatment of gout), given over a period of 8 weeks, will improve heart function, exercise ability and quality of life in African American Veterans with resistant hypertension.

Start Date30 Sep 2024

Sponsor / Collaborator

VA Office of Research and Development

100 Clinical Results associated with Allopurinol

100 Translational Medicine associated with Allopurinol

100 Patents (Medical) associated with Allopurinol

11,705

Literatures (Medical) associated with Allopurinol

31 Dec 2025·RENAL FAILURE

Comparative efficacy and safety of febuxostat and allopurinol in chronic kidney disease stage 3–5 patients with asymptomatic hyperuricemia: a network meta-analysis

Review

Author: Chen, Lu ; Zhang, Yanyun ; Wang, Yinglin ; Chen, Jiaojiao

OBJECTIVE:

This study evaluates and compares the effectiveness and safety of febuxostat and allopurinol in chronic kidney disease (CKD) stages 3-5 patients with asymptomatic hyperuricemia using a network meta-analysis.

METHODS:

A systematic review and network meta-analysis were conducted, adhering to PRISMA-NMA guidelines. Searches included PubMed, Embase, Cochrane Library, and Chinese databases up to June 2024. Randomized controlled trials (RCTs) and cohort studies were assessed for methodological rigor using GRADE.

RESULTS:

A total of 12 RCTs and 4 cohort studies (n = 2,423 participants) were included. Febuxostat was associated with greater improvements in estimated glomerular filtration rate compared to allopurinol (MD, 4.99 mL/min/1.73 m²; 95%CI -0.65 to 10.78; certainty: low) and placebo (MD, 4.72 mL/min/1.73 m²; 95%CI 0.67 to 8.82; low). Serum uric acid reduction was also more pronounced with febuxostat (MD, -0.61 mg/dL; 95%CI -1.15 to -0.05; moderate). Safety outcomes, including major cardiovascular events and adverse events, showed no significant differences between febuxostat and allopurinol. Subgroup analyses revealed enhanced effectiveness of febuxostat at six months of treatment.

CONCLUSIONS:

This analysis provides robust evidence that febuxostat might offers greater improvements in kidney function and uric acid levels compared to allopurinol or placebo in asymptomatic hyperuricemia with CKD stage 3-5 patients, without compromising safety. These findings can guide clinical decision-making and treatment optimization.

01 Oct 2025·JOINT BONE SPINE

Addition of allopurinol to traditional Vietnamese medicine shows major improvement of 100 gout patients in a single center one-year prospective study

Article

Author: Nguyen, Quang Huy ; Tran, Khoi ; Dalbeth, Nicola ; Bausson, Valérie ; Tran, Chuc ; Huynh, Dai ; Richette, Pascal ; Resche-Rigon, Matthieu ; Bardin, Thomas ; Do, Minh ; Nguyen, Quang Dinh

OBJECTIVES:

Gout is frequently severe in Vietnam, where urate-lowering drugs (ULDs) are seldom used and many patients are treated only with traditional herbal medicine. We assessed the addition of Western medicine on severe gout in Vietnamese people.

METHODS:

One hundred Vietnamese, ULD-free, crystal-proven gout patients with a GFR>60mL/min, were prospectively followed for 1year after allopurinol initiation. The treatment protocol included allopurinol given according to the 2016 EULAR recommendations, flare prophylaxis with colchicine during the first months, and traditional herbal medicine. At each visit, gout flares were counted by a daily diary, digital foot photographs were taken for semi-quantitative tophus scoring, serum urate (SU) was measured. Ultrasound (US) scan was performed at baseline, 6 and 12months for double contour (DC) and index tophus measurement. Quality of life (Gout Impact Score [GIS]) and function were recorded at inclusion and after 12months. Foot gout radiographic erosion scores were obtained at baseline, 6 and 12months. Outcomes were compared in patients who had reached SU targets at 3months and those who did not.

RESULTS:

Patients' median age and disease duration were 47 and 8years respectively, 91 had clinical tophi and 70 foot gouty erosions. Eighty-four patients were seen at 6months and 68 at 12months. Allopurinol dosage was progressively increased to a median of 600mg/d. Significant improvement of flare rates at M6 and M12, and of GIS and function at M12 were noted and did not associate with SU targets. Tophi (assessed by photograph and US measurements) and DC sign significantly decreased from M6 in association with achievement of<300μmol/L SU target. Foot erosion scores significantly decreased with no association with SU targets. Mild skin rash to allopurinol developed in 7 patients.

CONCLUSION:

This one-year open study tested a global gout care delivery model in which treat to uricemia target allopurinol, Western medicine self-treatment of flares, and patient education were added to a background of traditional herbal medicine in a Vietnamese population where gout is traditionally treated with herbal medicine. This shift in practice resulted in dramatic gout improvement.

01 Aug 2025·Current Drug Safety

Carbamazepine-induced Stevens-Johnson Syndrome: A Case Report with Review of the Literature

Article

Author: S, Nirenjen ; T., Tamilanban ; Manavalan, Mohankumar ; Raja, Sangeetha ; Subramanian, Arunkumar ; Haitharali, Rajamohamed ; Gnanasekaran, Sabariakilesh ; Dhanasekaran, Sivaraman

Background::

Stevens-Johnson Syndrome (SJS) is an infrequent yet severe mucocutaneous reaction that involves less than 10% of the Body Surface Area (BSA). It is predominantly induced by certain medications, including anticonvulsants (e.g., Lamotrigine, Carbamazepine, Phenytoin, Phenobarbitone), Allopurinol at doses above 100 mg per day, and sulphonamides (e.g., Cotrimoxazole, Sulfasalazine). Genetic predispositions, particularly the presence of the HLA-B*1502 allele, significantly increase the risk of developing SJS. This case report discusses a unique presentation of SJS in a young female patient, emphasizing the critical need for genetic screening and careful monitoring when prescribing Carbamazepine, especially in populations at higher genetic risk.

Case Presentation::

A 19-year-old female patient, who had been on Phenytoin and Sodium Valproate for epilepsy management over the past year, was newly prescribed Carbamazepine. Within a week of initiating Carbamazepine, the patient experienced a seizure, followed by the sudden onset of fever, painful sores, and blisters covering the upper body, along with mucous discharge from both eyes. These symptoms rapidly worsened. Based on clinical presentations and the extent of epidermal detachment, the patient was diagnosed with SJS. The severity and mortality risks were assessed using the SCORTEN score. Therapeutic interventions included intravenous Ranitidine, Ondansetron, Paracetamol, Midazolam, Levetiracetam, and Dexamethasone, along with oral Fluconazole, Chlorpheniramine tablets, and Ciprofloxacin eye drops. The patient showed significant improvement and was discharged after fourteen days with followup advice.

Conclusion::

This case underscores the critical importance of performing genetic testing for the HLA-B*1502 allele and conducting baseline blood tests before initiating Carbamazepine therapy. Such precautionary measures can significantly mitigate the risk of severe adverse reactions like SJS. This report adds to the scientific literature by highlighting the potential dangers associated with anticonvulsant therapies and the necessity for personalized medicine approaches in preventing life-threatening conditions. The main takeaway is the pivotal role of genetic screening and vigilant monitoring in the management of patients requiring anticonvulsant medications to prevent serious adverse reactions.

News (Medical) associated with Allopurinol

30 Apr 2025

·www.globenewswire.com

XORTX Provides Update on FDA Type B Meeting Request

● 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.

Drug ApprovalNDA

28 Apr 2025

·www.globenewswire.com

XORTX Announces Grant of European Patent

Patent Supporting Gout and Autosomal Dominant Polycystic Kidney Disease ProgramsCALGARY, Alberta, April 28, 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 receipt of notification that the patent “Xanthine Oxidase Inhibitor Formulations” will be granted by the European Patent Office. The patent covers compositions and methods of formulating using XORTX’s proprietary formulations of xanthine oxidase inhibitors (“XOI”) for the treatment of health consequences of chronically high uric acid, gout, renal, cardiovascular and other diseases where aberrant purine metabolism has been implicated in disease progression. Dr. Allen Davidoff, CEO of XORTX stated, “This patent covers compositions of formulations key to XORTX’s platform technology and this issuance strengthens our intellectual property portfolio in the European Union (“EU”). Importantly, this European patent broadens protection of our first-in-class program for gout as well as supporting our autosomal dominant polycystic kidney disease (“ADPKD”) program. This patent focuses on the health consequences that arise from uric acid crystal formation due to hyperuricemia in conditions including gout, kidney stone formation, cardiovascular disease and covers novel therapeutic solutions developed specifically to address these problems. The granting of this patent provides additional breadth of patent to XORTX for commercialization and partnering opportunities throughout Europe. Including this newly granted patent, XORTX now has five granted patents in the United States (“US”) and/or EU covering compositions and uses of uric acid lowering agents to treat gout, the health consequences of hyperuricemia, and progressive kidney disease.” About Hyperuricemia, Gout and Health Consequences It is estimated that 14% of the adult population has hyperuricemia – i.e. serum uric acid levels greater than the normal range. Approximately 3% of the US population suffer from gout as a result of their hyperuricemia, though not all individuals who have hyperuricemia develop gout. The factors that contribute to hyperuricemia include genetic factors(1), dietary factors and lifestyle choices. Nucleotides are normally broken down in the blood by the xanthine oxidase (“XO”) enzyme through the purine metabolic pathway. This results in the formation of uric acid. However, if an individual experiences chronically high blood uric acid concentrations (hyperuricemia), they often experience health consequences including gout, kidney stones, diabetes, cardiovascular disease, and renal failure. Evidence that unique health consequences may arise from hyperuricemia, independent of the health consequences of gout, suggest that aberrant purine metabolism, hyperuricemia, merits an increased therapeutic focus. Lowering blood levels of uric acid in gout patients is strongly correlated with improved health outcomes. Recent groundbreaking studies by Wang et al. suggests linkage of genetic factors to the overexpression of XO(1). Recently, emerging discoveries link genetic factors to specific populations and show that higher XO expression is associated with a variety of conditions including hyperuricemia(2), general sepsis, organ failure and sepsis associated with acute respiratory distress syndrome (ARDS)(3,4), kidney dysfunction(3,4), diabetes(5), polycystic kidney disease(1,5) and kidney failure(6,7). From a mechanistic standpoint, these studies advocate for a precision medicine approach in which genetic risk variants would guide treatment decisions(1). 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: 1) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; 2) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and 3) oral XOIs that are used to inhibit the production of uric acid by the purine metabolic pathway. 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, was launched in the US in 2009 with the hope of treating allopurinol intolerant patients. While Febuxostat achieved peak sales of approximately US$450 million1, 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. References: Korsmo HW, Emerging roles of xanthine oxidoreductase in chronic kidney disease, Antioxidants, June 2024Major TJ, et all, Evaluation of the diet wide contribution to serum urate levels: Met-analysis of population based cohorts, BMJ, 363, k3952, 2018Gao, Li et al., Xanthine oxidoreductase gene polymorphism are associated with high risk of sepsis and organ failure, Respir. Res, 24, 177_2023Liu H, et al., Genetic variants in XDH are associated with prognosis off gastric cancer in a Chines population, 663, 196, 2013Wang et al., Genetic susceptibility to diabetic kidney disease is linked to promoter variants of XOR,” The authors identified an expression quantitative trait loci (QTL) in the cis-acting regulatory region of the xanthine dehydrogenase, or xanthine oxidoreductase (XO), a binding site for C/EBPβ, to be associated with diabetes-induced podocyte loss in diabetic kidney disease in male mice. They concluded that certain types of alleles of a gene that controls the expression of xanthine oxidase can be over expressed in CKD, diabetic kidney disease and polycystic kidney disease.Kudo M et al., Functional Characterization of Genetic Polymorphisms Identified in the Promotor Region of the Xanthine Oxidase Gene, Drug Metab. Pharmacokinet., 25, 599, 2010Boban M, et al., Circulating purine compound, uric acid, and xanthine oxidase/dehydrogenate relationship in essential hypertension and end stage renal disease., Ren. Fail., 36, 613, 2014 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.

28 Mar 2025

·www.prnewswire.com

Shanton Reveals Topline Data from Phase 2b Study in Refractory Gout Patients

SAP-001 is Shanton's lead investigational compound with First- and Best-in-Class potential in uncontrolled gout SINGAPORE and PRINCETON, N.J., March 28, 2025 /PRNewswire/ -- Shanton Pharma, a clinical-stage biotech company developing a novel treatment for gout, today announced topline data of a Phase 2b study in refractory gout patients with its investigational drug SAP-001 that uses a First-in-Class Mechanism of Action targeting a distinct kidney transporter. "We are excited and very pleased with the outcome of our Phase 2b clinical study in this difficult-to-treat population of refractory gout patients" says Dr. Bing Li, Shanton's CEO. "By the end of month 3 in our study, nearly 100% of patients that were confirmed exposed to daily SAP-001 30 and 60 mg dosages on top of conventional therapy reached the study's therapeutical target (serum uric acid levels below 6mg/dL), compared to only about 10% of patients on conventional treatment who reached that goal. The majority of treatment effects persisted until the end of treatment at month 6 of the study. More than half of those patients confirmed exposed to a daily 60 mg dose reached serum uric acid levels below 3mg/dL. Importantly, the study demonstrated an excellent safety profile in this population." Shanton's gout program already showed excellent, best-in-class efficacy compared to other Urate Lowering Therapies in prior Phase 1 and 2a studies in regular gout patients, an outcome that has now been repeated in the challenging target population of refractory patients that often have a more severe form of gout with limited treatment options. Dr. Wenfeng Miao, Shanton's CMO adds: "This is the first successful clinical study with an oral gout drug to treat these refractory patients that normally need to be treated with an intravenous uricase. The efficacy of SAP-001 in this population is very impressive in all dosage groups at all assessed time points across the six months of treatment. We believe that the excellent efficacy and safety outcomes in this study show the exciting potential of our First-in-Class compound with its novel mechanism of action (MOA) that selectively targets a distinct kidney transporter for urate lowering treatment. The medical community has long been looking for this new mechanism in gout to address the shortcomings with conventional MOAs, especially where it comes to treating refractory and tophaceous patients. Our goal is to proceed into pivotal studies with our program later this year to eventually offer a paradigm-shifting gout treatment to our patients and make a big impact in their lives." About Gout Gout is the most common form of inflammatory arthritis and the second most prevalent metabolic disease. It is caused by too much uric acid in the bloodstream, with over 12 million adult patients diagnosed in the US alone.1 Hyperuricemia, elevated serum uric acid levels, can over time result in gout when urate crystals are deposited in joints and other body tissues, causing inflammatory responses and painful gout attacks (flares). Recurrent gout flares are debilitating, and chronic gouty arthritis can over time lead to joint destruction and joint disfigurement. About 29% of diagnosed gout patients in the US are treated with urate lowering therapies (ULTs), but only half of those patients sufficiently respond to or can tolerate current treatment options. It is estimated that up to 20% of US gout patients on standard-of-care allopurinol or febuxostat are physiologically refractory to these treatments. 2,3 About Shanton's Phase 2b Gout Study Shanton's Phase 2b study is a six-month, multi-center, randomized, double-blind, placebo-controlled, dose-ranging clinical study in refractory gout patients with or without tophi, to evaluate the efficacy and safety of a 10, 30, or 60 mg tablet dose of SAP-001. The study enrolled 87 patients from 21 US sites. The Primary objective is to assess the effectiveness of SAP-001 in lowering serum urate levels in gout patients that are refractory to standard-of-care Xanthine Oxidase Inhibitor (XOI) therapy. Secondary study objectives include safety and tolerability assessments and frequency of gout flares among others. About Shanton Pharma Shanton Pharma is a privately held, clinical-stage biotech founded in 2016 by experienced pharma entrepreneurs, with a research focus on unmet needs associated with hyperuricemia and gout. The company is headquartered in Singapore with research and development activities in the US, China, and Singapore. SAP-001 is Shanton's lead investigational compound for once-a-day oral urate-lowering therapy that targets refractory gout. SAP-001's urate lowering properties are based on a unique mechanism-of-action and the product has shown unparalleled efficacy and safety in Phase 1 and Phase 2a clinical studies in gout patients with hyperuricemia, and now also has shown Best-in-Class efficacy and safety in gout patients with hyperuricemia refractory to Standard of Care Xanthine Oxidase Inhibitor therapy. To learn more about Shanton Pharma, go to . References Yokose C, McCormick N, Lu N, et al. Trends in Prevalence of Gout Among US Asian Adults, 2011-2018. JAMA Netw Open. 2023. Juraschek SP, Kovell LC, Miller ER 3rd, Gelber AC. Gout, urate-lowering therapy, and uric acid levels among adults in the United States. Arthritis Care Res (Hoboken). 2015. Clarivate Incidence and Prevalence Data Base Media Contact Pieter de Ridder VP of Business Development [email protected] Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical ResultPhase 1

100 Deals associated with Allopurinol

External Link

KEGG	Wiki	ATC	Drug Bank
D00224	Allopurinol	M04AA01	DB00437

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Gout	China	Jiangsu Meitong Pharmaceutical Co. Ltd.	01 Jan 1985
Nephrolithiasis	China	Jiangsu Meitong Pharmaceutical Co. Ltd.	01 Jan 1985
Hyperuricemia	United States	Casper Pharma LLC	19 Aug 1966

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Acute myocardial infarction	Phase 3	United States	AstraZeneca PLC	01 Jul 2005
Diabetes Mellitus	Phase 3	United States	AstraZeneca PLC	01 Jul 2005
Heart failure with normal ejection fraction	Phase 2	United States	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Argentina	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Australia	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Austria	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Bulgaria	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Canada	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Germany	AstraZeneca PLC	19 May 2020
Heart failure with normal ejection fraction	Phase 2	Mexico	AstraZeneca PLC	19 May 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


UEGW2024	Not Applicable	Inflammatory Bowel Diseases 6-TGN \| 6-MMP \| FC (fecal calprotectin)	237	Low-dose azathioprine plus allopurinol	vyjwxftkzw(kfrtsmssfi): OR = 2.46 (95% CI, 1.43 - 4.25), P-Value = 0.001	Positive	13 Oct 2024
				Azathioprine monotherapy
ESC2024	Not Applicable	-	-	Allopurinol + hydration	kfceygxplg(pjwoptscvy) = yrtfyhepui irykjdgsjx (elckowlpni )	-	31 Aug 2024
				allopurinol (Hydration only)	kfceygxplg(pjwoptscvy) = bqsfvgplda irykjdgsjx (elckowlpni )
NCT04327024 (AMETHYST, Pubmed \| JAMA Cardiol) Manual	Phase 2	Heart failure with normal ejection fraction	159	Verinurad plus Allopurinol	wqvrjqiecy(rciqsjsbif) = nsetbokeor quqnqhqsmy (afnkdndeke, -0.56 to 1.10) View more	Negative	14 Aug 2024
				Allopurinol	wqvrjqiecy(rciqsjsbif) = bpporudmcs quqnqhqsmy (afnkdndeke, -1.03 to 0.69) View more
EULAR2024	Not Applicable	fractional excretion of uric acid (FEUA)	1,465	Allopurinol	hffkyipluv(ydumjoinli) = zkreecdaeg zjsumblbqy (uczzlumfdy ) View more	Positive	05 Jun 2024
				Febuxostat	hffkyipluv(ydumjoinli) = jmjclesuvx zjsumblbqy (uczzlumfdy )
ISN WCN2024	Not Applicable	Chronic Kidney Diseases serum uric acid levels ≥6 mg/dl	5,000	Allopurinol 100 mg/day	fjrmlgpldw(rnnyefajwl) = wkkjovwyph rpopujloqn (wipffokuau ) View more	Positive	01 Apr 2024
ACR2023	Not Applicable	-	-	Allopurinol (Asian patients)	tqsfpwnrzw(fqmbucvatt) = yafuxgvrhc xoaksdgnwb (jerhepotmp )	-	12 Nov 2023
				Allopurinol (Black patients)	tqsfpwnrzw(fqmbucvatt) = lrzfcwxwgr xoaksdgnwb (jerhepotmp )
NCT03648996 (CTgov)	Phase 2	Hyperuricemia \| Diabetes Mellitus, Type 2 \| Arteriosclerosis	34	Allopurinol (Allopurinol)	pcdxajviqb(gpxsbpijwk) = ypadjwvhli cemkqnjsfr (deojgysdxm, 1.47) View more	-	03 Jul 2023
				Placebo (Placebo)	pcdxajviqb(gpxsbpijwk) = yzbxwrxomf cemkqnjsfr (deojgysdxm, 1.04) View more
NCT04327024 (AMETHYST, CTgov)	Phase 2	Heart failure with normal ejection fraction	159	Placebo for verinurad	vonjrtwjgj(mnlvwggdci) = lgjznkiukh slbgcyjool (gofesqldjs, uwsvifcbhn - outsdroled) View more	-	29 Jun 2023
FAST (ESH2023)	Not Applicable	HYPERURICEMIA CHRONIC	298	Allopurinol	wilwdbodxr(pioymmxvnq) = rtrokgwafq jjfasczefl (bkjwcjjjpa, -1.76 to 1.56)	Negative	01 Jun 2023
				Febuxostat	wilwdbodxr(pioymmxvnq) = vkkaapzlui jjfasczefl (bkjwcjjjpa, -4.20 to 2.67)
ESH2023	Not Applicable	Diabetes Mellitus, Type 2	194	Allopurinol users	baehjrxxva(uzrtcagcjp) = bgpdzhtxmi uuoopwwjne (dfleyrfufl, 6.20 - 7.80)	Positive	01 Jun 2023
				ALLOPURINOL (Control group)	baehjrxxva(uzrtcagcjp) = vowemrevxk uuoopwwjne (dfleyrfufl, 6.60 - 8.40)

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