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XORTX Announces Presentation at the Rare and Genetic Disease Summit

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XORTX Therapeutics (NASDAQ: XRTX) announced a presentation at the Rare and Genetic Kidney Disease Summit in Boston on December 12, 2024. The presentation focuses on recent discoveries in Autosomal Dominant Polycystic Kidney Disease (ADPKD) and genetic factors influencing xanthine oxidase (XO) expression in kidney disease progression.

The research highlights how genetic factors affecting purine metabolism may act as a 'second hit' that accelerates ADPKD progression. This discovery explains why disease progression varies among family members and supports the development of personalized therapeutic approaches using XO inhibitors for at-risk individuals.

XORTX Therapeutics (NASDAQ: XRTX) ha annunciato una presentazione al Summit sulle Malattie Renali Rare e Genetiche a Boston il 12 dicembre 2024. La presentazione si concentra su recenti scoperte riguardanti la Malattia Renale Policistica Autosomica Dominante (ADPKD) e sui fattori genetici che influenzano l'espressione della xantina ossidasi (XO) nella progressione della malattia renale.

La ricerca evidenzia come i fattori genetici che influenzano il metabolismo delle purine possano agire come un 'secondo colpo' che accelera la progressione dell'ADPKD. Questa scoperta spiega perché la progressione della malattia varia tra i membri della famiglia e supporta lo sviluppo di approcci terapeutici personalizzati utilizzando inibitori della XO per le persone a rischio.

XORTX Therapeutics (NASDAQ: XRTX) anunció una presentación en la Cumbre de Enfermedades Renales Raras y Genéticas en Boston el 12 de diciembre de 2024. La presentación se centra en descubrimientos recientes sobre la Enfermedad Renal Policística Autosómica Dominante (ADPKD) y en los factores genéticos que influyen en la expresión de xantina oxidasa (XO) en la progresión de la enfermedad renal.

La investigación destaca cómo los factores genéticos que afectan el metabolismo de las purinas pueden actuar como un 'segundo golpe' que acelera la progresión de la ADPKD. Este descubrimiento explica por qué la progresión de la enfermedad varía entre los miembros de la familia y apoya el desarrollo de enfoques terapéuticos personalizados utilizando inhibidores de XO para individuos en riesgo.

XORTX Therapeutics (NASDAQ: XRTX)는 2024년 12월 12일 보스턴에서 열리는 희귀 및 유전신장질환 정상에서 발표를 알렸습니다. 이번 발표는 유전자성 다낭성과 신장 질환의 진행에 영향을 미치는 xanthine oxidase (XO) 발현의 최근 발견에 초점을 맞추고 있습니다.

이 연구는 퓨린 대사에 영향을 미치는 유전적 요인이 ADPKD 진행을 가속화하는 '두 번째 타격'으로 작용할 수 있음을 강조합니다. 이 발견은 질병 진행이 가족 구성원 간에 왜 차이가 있는지를 설명하며, 위험이 있는 개인을 위한 XO 억제제를 사용하는 맞춤형 치료 접근법의 개발을 지원합니다.

XORTX Therapeutics (NASDAQ: XRTX) a annoncé une présentation lors du Sommet sur les Maladies Rénales Rares et Génétique à Boston le 12 décembre 2024. La présentation porte sur des découvertes récentes concernant la Maladie Rénale Polykystique Autosomique Dominante (ADPKD) et les facteurs génétiques influençant l'expression de la xanthine oxydase (XO) dans la progression des maladies rénales.

La recherche souligne comment les facteurs génétiques affectant le métabolisme des purines peuvent agir comme un 'deuxième coup' qui accélère la progression de l'ADPKD. Cette découverte explique pourquoi la progression de la maladie varie entre les membres de la famille et soutient le développement d'approches thérapeutiques personnalisées utilisant des inhibiteurs de XO pour les individus à risque.

XORTX Therapeutics (NASDAQ: XRTX) gab eine Präsentation beim Gipfel für seltene und genetische Nierenerkrankungen in Boston am 12. Dezember 2024 bekannt. Die Präsentation konzentriert sich auf jüngste Entdeckungen zur autosomal dominanten zystischen Nierenerkrankung (ADPKD) und den genetischen Faktoren, die den Ausdruck der Xanthin-Oxidase (XO) im Verlauf der Nierenerkrankung beeinflussen.

Die Forschung hebt hervor, wie genetische Faktoren, die den Purinstoffwechsel beeinflussen, als „zweiter Schlag“ wirken können, der die Progression der ADPKD beschleunigt. Diese Entdeckung erklärt, warum die Krankheitsprogression unter Familienmitgliedern variiert, und unterstützt die Entwicklung personalisierter therapeutischer Ansätze mit XO-Hemmern für gefährdete Personen.

Positive
  • Discovery of genetic factors influencing ADPKD progression supports company's therapeutic approach
  • Patent portfolio already covers the newly identified therapeutic opportunity
  • Positioning for personalized medicine approach in ADPKD treatment
Negative
  • Clinical trial still in planning phase, not yet initiated

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.

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.

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.”

About Xanthine Oxidase

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.

References:

  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

About 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 www.xortx.com.

For more information, please contact:

Allen Davidoff, CEO
adavidoff@xortx.com or +1 403 455 7727
Nick Rigopulos, Director of Communications
nick@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.


FAQ

What did XORTX present at the Rare and Genetic Disease Summit on December 12, 2024?

XORTX presented findings on genetic and environmental factors in ADPKD, focusing on aberrant purine metabolism as a second hit determining disease progression.

How do genetic factors affect ADPKD progression according to XORTX's research?

Genetic factors influence xanthine oxidase (XO) expression and contribute to chronic hyperuricemia, acting as a 'second hit' that can accelerate ADPKD disease progression.

What is XORTX's therapeutic approach for ADPKD treatment?

XORTX is developing XO inhibitors as targeted therapeutics for individuals with genetic factors predisposing them to ADPKD progression.

Why does ADPKD progression vary among family members according to XORTX's findings?

The variation is due to genetic factors that influence xanthine oxidase expression and purine metabolism, which can affect the rate of disease progression differently among individuals.

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