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XORTX Announces Presentation at the Rare and Genetic Disease Summit

XORTX Announces Presentation at the Rare and Genetic Disease Summit

XORTX宣佈將在稀有和遺傳疾病峯會上進行演講
GlobeNewswire ·  12/12 09:17

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."

加拿大卡爾加里,2024年12月12日(環球新聞)—— XORTX Therapeutics Inc.("XORTX"或"公司")(納斯達克:XRTX | TSXV:XRTX | 法蘭克福:ANU)是一家專注於開發創新療法以治療進行性腎病的晚期臨床藥品公司,樂於宣佈由Allen Davidoff博士在美國馬薩諸塞州波士頓舉行的罕見和遺傳性腎病峯會上進行的報告,時間爲2024年12月12日星期四上午10:30(東部時間)。該報告題爲「常染色體顯性多囊腎病 - 遺傳與環境因素→ 港面代謝異常作爲決定疾病進展的第二hit的證據。」

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.

該報告強調了XORTX在常染色體顯性多囊腎病("ADPKD")領域中的最新開創性發現,以及最新的同行評審、獨立發佈的研報,確定了影響黃嘌呤氧化酶("XO")過度表達的遺傳因素,並在多種疾病中發揮作用,包括腎病。這些突破性發現表明,影響異常嘌呤代謝的遺傳因素可能會影響ADPKD的進展速度。

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首席執行官Allen Davidoff博士表示:「最近識別出的遺傳因素增加了XO的表達和/或導致慢性高尿酸血癥,支持了「第二hit」的概念——當出現時加速疾病進展速率的因素。這些新發現是我們理解爲何ADPKD的進展甚至在家族成員中可能差異顯著的重要第一步。這些發現突顯了爲那些因獨特的遺傳因素而易感ADPKD的個人制定個性化治療方案的機會,以及爲了治療那些有風險個體而需要抑制XO。我們相信XORTX在開發XO抑制劑方面的專業知識,受益於一個預見到這一機會的專利組合,加上我們的治療平台,理想地定位於爲個人提供針對性的療法。我們計劃在ADPKD患者中進行的臨床試驗將爲進一步了解這些新識別的遺傳因素在進行性腎病個體中的作用提供機會。」

About Xanthine Oxidase

關於黃嘌呤氧化酶

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.

迄今爲止尚未報道人類多囊腎疾病(PKD)中XO的過表達證據,儘管王等人的研究表明遺傳因素與PKD1相關。最近,新興發現將遺傳因素與特定人群相關聯,並顯示XO的高表達與多種狀態相關,包括高尿酸血癥2、膿毒症、器官衰竭及與膿毒症相關的急性呼吸窘迫綜合症(ARDS)3,4、腎功能障礙3,4、糖尿病5、多囊腎病1,5和腎衰竭6,7。從機制的角度來看,這些研究倡導一種精準醫學的方法,其中遺傳風險變異會指導治療決策1。

References:

參考文獻:

  1. Korsmo HW, Emerging roles of xanthine oxidoreductase in chronic kidney disease, Antioxidants, June 2024
  2. Major TJ, et all, Evaluation of the diet wide contribution to serum urate levels: Met-analysis of population-based cohorts, BMJ, 363, k3952, 2018
  3. Gao, Li et al., Xanthine oxidoreductase gene polymorphism are associated with high risk of sepsis and organ failure, Respir. Res, 24, 177_2023
  4. Liu H, et al., Genetic variants in XDH are associated with prognosis off gastric cancer in a Chines population, 663, 196, 2013
  5. 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.
  6. 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
  7. 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
  1. Korsmo HW, 黃嘌呤氧化還原酶在慢性腎病中的新興角色,抗氧化劑,2024年6月
  2. Major TJ等人,飲食對血清尿酸水平的廣泛貢獻評估:基於人群的隊列的薈萃分析,BMJ,363,k3952,2018
  3. Gao,Li等人,黃嘌呤氧化還原酶基因多態性與膿毒症和器官衰竭的高風險相關,呼吸研究,24,177_2023
  4. 劉H等人,XDH中的遺傳變異與中國人群胃癌預後相關,663,196,2013
  5. 王等人,糖尿病腎病的遺傳易感性與XOR的啓動子變異相關。"作者發現了黃嘌呤脫氫酶或黃嘌呤氧化還原酶(XO)的順式作用調控區域中的表達定量性狀位點(QTL),這是C/EBPβ的結合位點,與雄性小鼠的糖尿病引起的足細胞喪失相關。他們得出結論,控制黃嘌呤氧化酶表達的基因的某些類型等位基因可在慢性腎病、糖尿病腎病和多囊腎病中過度表達。
  6. Kudo m等人對Xanthine Oxidase基因啓動子區域中發現的遺傳多態性進行了功能評估,藥物代謝和藥動學,25,599,2010
  7. Boban m等人,循環中嘌呤化合物、尿酸與原發性高血壓和終末期腎病中的黃嘌呤氧化酶/脫氫酶關係,腎衰竭,36,613,2014

About XORTX Therapeutics Inc.

關於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是一家藥品公司,正在開發兩款臨床先進產品:1)我們主要的XRx-008項目針對多囊腎病(ADPKD);2)我們次要的XRx-101項目針對與冠狀病毒/COVID-19感染相關的急性腎臟和其他急性器官損傷。此外,XRx-225是一個針對2型糖尿病腎病的臨床前階段項目。XORTX正在努力推進其臨床開發階段的產品,目標是針對異常的嘌呤代謝和黃嘌呤氧化酶,以減少或抑制尿酸的生成。在XORTX,我們致力於開發改善腎病患者生活質量和健康的藥物。有關XORTX的更多信息,請訪問。

For more information, please contact:

如需更多信息,請聯繫:

Allen Davidoff, CEO
adavidoff@xortx.com or +1 403 455 7727
Nick Rigopulos, Director of Communications
nick@alpineequityadv.com or +1 617 901 0785
艾倫·達維多夫,首席執行官
adavidoff@xortx.com 或 +1 403 455 7727
通信-半導體主任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.

無論是TSX創業公司交易所還是納斯達克都未批准或不批准本新聞稿的內容。沒有任何股票交易所、證券委員會或其他監管機構批准或不批准此處包含的信息。

Forward Looking Statements

前瞻性聲明

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 .

本新聞稿包含根據適用證券法的明確或隱含的前瞻性聲明。這些前瞻性聲明包括但不限於公司對其信念、計劃、目標、預期、假設、估計、意圖、未來表現以及其他非歷史事實的陳述,以及由"預期"、"期待"、"打算"、"計劃"、"相信"、"尋求"、"估計"或具有相似含義的詞所標識的聲明。這些前瞻性聲明及其含義僅基於XORTX管理層的當前預期,並且受多種因素和不確定性的影響,這些因素可能導致實際結果與前瞻性聲明中描述的結果存在重大差異。此類風險、不確定性和其他因素包括但不限於我們獲得額外融資的能力;我們對費用、未來收入和資本需求的估計準確性;我們臨床前研究和臨床試驗的成功和時機;第三方製造商和醫藥外包概念的表現;我們開發和商業化產品候選者的計劃;我們在其他腎臟疾病應用中推進研究的計劃;以及,我們獲得和維持產品候選者的知識產權保護的能力。除非適用法律和證券交易所規則另有要求,否則XORTX沒有義務公開發布任何對這些前瞻性聲明的修訂,以反映本日期之後的事件或情況,或反映意外事件的發生。關於影響XORTX的風險和不確定性的更詳細信息包含在XORTX向SEC提交的20-F表格的「風險因素」標題下,該文件可以在SEC的網站www.sec.gov(包括其任何形成部分或引用其中的文件)獲得,以及我們在加拿大的報告、公開披露文件和其他向證券委員會及其他監管機構提交的備案材料,均可在該網站上獲得。


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