XORTX Announces Presentation at the Rare and Genetic Disease Summit
XORTX Announces Presentation at the Rare and Genetic Disease Summit
CALGARY, Alberta, Dec. 12, 2024 (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, is pleased to announce a presentation by Dr. Allen Davidoff at the Rare and Genetic Kidney Disease Summit, in Boston, Massachusetts at 10:30 am ET, Thursday December 12, 2024. The presentation entitled "Autosomal Dominant Polycystic Kidney Disease - Genetic and Environmental Factors → Evidence for Aberrant Purine Metabolism as a Second Hit Determining Disease Progression."
加拿大卡尔加里,2024年12月12日(环球新闻)—— XORTX Therapeutics Inc.("XORTX"或"公司")(纳斯达克:XRTX | TSXV:XRTX | 法兰克福:ANU)是一家专注于开发创新疗法以治疗进行性肾病的晚期临床药品公司,乐于宣布由Allen Davidoff博士在美国马萨诸塞州波士顿举行的罕见和遗传性肾病峰会上进行的报告,时间为2024年12月12日星期四上午10:30(东部时间)。该报告题为“常染色体显性多囊肾病 - 遗传与环境因素→ 港面代谢异常作为决定疾病进展的第二hit的证据。”
The presentation highlights XORTX recent pioneering discoveries in the field of Autosomal Dominant Polycystic Kidney Disease ("ADPKD"), and recent peer-reviewed, independent, published research reports identifying genetic factors that influence over-expression of xanthine oxidase ("XO") and play a role in several diseases, including kidney disease. These ground-breaking findings suggest that genetic factors that influence aberrant purine metabolism may influence the rate of progression of ADPKD.
该报告强调了XORTX在常染色体显性多囊肾病("ADPKD")领域中的最新开创性发现,以及最新的同行评审、独立发布的研报,确定了影响黄嘌呤氧化酶("XO")过度表达的遗传因素,并在多种疾病中发挥作用,包括肾病。这些突破性发现表明,影响异常嘌呤代谢的遗传因素可能会影响ADPKD的进展速度。
Dr. Allen Davidoff, CEO of XORTX, stated, "The recent identification of genetic factors that increase the expression of XO, and/or contribute to chronic hyperuricemia support the concept of a "second hit" – a factor or factors that accelerate the rate of disease progression when present. These new discoveries are an important first step in our understanding of why ADPKD progression may vary substantially even amongst family members. These discoveries highlight an opportunity to develop a personalized therapeutic approach for individuals whose unique genetic factors predisposed them to ADPKD, and the need for XO inhibition to treat those individuals at risk. We believe that XORTX's expertise in developing XO inhibitors, protected by a patent portfolio that anticipated this opportunity, combined with our therapeutic platform is ideally positioned to deliver targeted therapeutics to individuals. Our planned clinical trial in patients with ADPKD will provide an opportunity to further understand the role of these newly identified genetic factors in individuals with progressive kidney disease."
XORTX首席执行官Allen Davidoff博士表示:“最近识别出的遗传因素增加了XO的表达和/或导致慢性高尿酸血症,支持了“第二hit”的概念——当出现时加速疾病进展速率的因素。这些新发现是我们理解为何ADPKD的进展甚至在家族成员中可能差异显著的重要第一步。这些发现突显了为那些因独特的遗传因素而易感ADPKD的个人制定个性化治疗方案的机会,以及为了治疗那些有风险个体而需要抑制XO。我们相信XORTX在开发XO抑制剂方面的专业知识,受益于一个预见到这一机会的专利组合,加上我们的治疗平台,理想地定位于为个人提供针对性的疗法。我们计划在ADPKD患者中进行的临床试验将为进一步了解这些新识别的遗传因素在进行性肾病个体中的作用提供机会。”
About Xanthine Oxidase
关于黄嘌呤氧化酶
Evidence for over-expression of XO in human PKD has not been reported to date, although work by Wang et al. suggests linkage of genetic factors to PKD1. Recently, new emerging discoveries link genetic factors to specific populations and show that higher XO expression is associated with a variety of conditions including hyperuricemia2, sepsis, organ failure and sepsis associated acute respiratory distress syndrome (ARDS)3,4, kidney dysfunction3,4, diabetes5, polycystic kidney disease1,5 and kidney failure6,7. From a mechanistic standpoint, these studies advocate for a precision-medicine approach in which genetic risk variants would guide treatment decisions1.
迄今为止尚未报道人类多囊肾疾病(PKD)中XO的过表达证据,尽管王等人的研究表明遗传因素与PKD1相关。最近,新兴发现将遗传因素与特定人群相关联,并显示XO的高表达与多种状态相关,包括高尿酸血症2、脓毒症、器官衰竭及与脓毒症相关的急性呼吸窘迫综合症(ARDS)3,4、肾功能障碍3,4、糖尿病5、多囊肾病1,5和肾衰竭6,7。从机制的角度来看,这些研究倡导一种精准医学的方法,其中遗传风险变异会指导治疗决策1。
References:
参考文献:
- Korsmo HW, Emerging roles of xanthine oxidoreductase in chronic kidney disease, Antioxidants, June 2024
- Major TJ, et all, Evaluation of the diet wide contribution to serum urate levels: Met-analysis of population-based cohorts, BMJ, 363, k3952, 2018
- Gao, Li et al., Xanthine oxidoreductase gene polymorphism are associated with high risk of sepsis and organ failure, Respir. Res, 24, 177_2023
- Liu H, et al., Genetic variants in XDH are associated with prognosis off gastric cancer in a Chines population, 663, 196, 2013
- Wang 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, 2010
- Boban 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
- Korsmo HW, 黄嘌呤氧化还原酶在慢性肾病中的新兴角色,抗氧化剂,2024年6月
- Major TJ等人,饮食对血清尿酸水平的广泛贡献评估:基于人群的队列的荟萃分析,BMJ,363,k3952,2018
- Gao,Li等人,黄嘌呤氧化还原酶基因多态性与脓毒症和器官衰竭的高风险相关,呼吸研究,24,177_2023
- 刘H等人,XDH中的遗传变异与中国人群胃癌预后相关,663,196,2013
- 王等人,糖尿病肾病的遗传易感性与XOR的启动子变异相关。"作者发现了黄嘌呤脱氢酶或黄嘌呤氧化还原酶(XO)的顺式作用调控区域中的表达定量性状位点(QTL),这是C/EBPβ的结合位点,与雄性小鼠的糖尿病引起的足细胞丧失相关。他们得出结论,控制黄嘌呤氧化酶表达的基因的某些类型等位基因可在慢性肾病、糖尿病肾病和多囊肾病中过度表达。
- Kudo m等人对Xanthine Oxidase基因启动子区域中发现的遗传多态性进行了功能评估,药物代谢和药动学,25,599,2010
- Boban m等人,循环中嘌呤化合物、尿酸与原发性高血压和终末期肾病中的黄嘌呤氧化酶/脱氢酶关系,肾衰竭,36,613,2014
About XORTX Therapeutics Inc.
关于XORTX Therapeutics Inc.
XORTX is a pharmaceutical company with two clinically advanced products in development: 1) our lead, XRx-008 program for ADPKD; and 2) 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. Additional information on XORTX is available at .
XORTX是一家药品公司,正在开发两款临床先进产品:1)我们主要的XRx-008项目针对多囊肾病(ADPKD);2)我们次要的XRx-101项目针对与冠状病毒/COVID-19感染相关的急性肾脏和其他急性器官损伤。此外,XRx-225是一个针对2型糖尿病肾病的临床前阶段项目。XORTX正在努力推进其临床开发阶段的产品,目标是针对异常的嘌呤代谢和黄嘌呤氧化酶,以减少或抑制尿酸的生成。在XORTX,我们致力于开发改善肾病患者生活质量和健康的药物。有关XORTX的更多信息,请访问。
For more information, please contact:
如需更多信息,请联系:
Allen Davidoff, CEO adavidoff@xortx.com or +1 403 455 7727 |
Nick Rigopulos, Director of Communications nick@alpineequityadv.com or +1 617 901 0785 |
艾伦·达维多夫,首席执行官 adavidoff@xortx.com 或 +1 403 455 7727 |
通信-半导体主任Nick Rigopulos nick@alpineequityadv.com 或 +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.
无论是TSX创业公司交易所还是纳斯达克都未批准或不批准本新闻稿的内容。没有任何股票交易所、证券委员会或其他监管机构批准或不批准此处包含的信息。
Forward Looking Statements
前瞻性声明
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 .
本新闻稿包含根据适用证券法的明确或隐含的前瞻性声明。这些前瞻性声明包括但不限于公司对其信念、计划、目标、预期、假设、估计、意图、未来表现以及其他非历史事实的陈述,以及由"预期"、"期待"、"打算"、"计划"、"相信"、"寻求"、"估计"或具有相似含义的词所标识的声明。这些前瞻性声明及其含义仅基于XORTX管理层的当前预期,并且受多种因素和不确定性的影响,这些因素可能导致实际结果与前瞻性声明中描述的结果存在重大差异。此类风险、不确定性和其他因素包括但不限于我们获得额外融资的能力;我们对费用、未来收入和资本需求的估计准确性;我们临床前研究和临床试验的成功和时机;第三方制造商和医药外包概念的表现;我们开发和商业化产品候选者的计划;我们在其他肾脏疾病应用中推进研究的计划;以及,我们获得和维持产品候选者的知识产权保护的能力。除非适用法律和证券交易所规则另有要求,否则XORTX没有义务公开发布任何对这些前瞻性声明的修订,以反映本日期之后的事件或情况,或反映意外事件的发生。关于影响XORTX的风险和不确定性的更详细信息包含在XORTX向SEC提交的20-F表格的“风险因素”标题下,该文件可以在SEC的网站www.sec.gov(包括其任何形成部分或引用其中的文件)获得,以及我们在加拿大的报告、公开披露文件和其他向证券委员会及其他监管机构提交的备案材料,均可在该网站上获得。