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XORTX Announces Presentation at American Society of Nephrology – Kidney Week 2024

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XORTX Therapeutics Inc. (NASDAQ: XRTX) announced the acceptance of an abstract for presentation at the American Society of Nephrology's Kidney Week 2024. The abstract, titled 'Xanthine oxidase in rats, mice and humans with polycystic kidney disease', focuses on the role of xanthine oxidase (XO) in polycystic kidney disease (PKD). The study, conducted at the University of Colorado and sponsored by XORTX, aimed to understand serum xanthine oxidase activity (XOa) in PKD and its relation to total kidney volume and glomerular filtration rate (GFR) decline.

The research investigated XO activity in PCK1 rats, PKD1RC/RC mice, and 34 patients from the HALT-PKD Clinical study. Results provide insights into how aberrant purine metabolism in PKD tissue may contribute to circulating uric acid levels, kidney expansion rate, and GFR decline. This study builds on previous findings suggesting XO overexpression in PKD kidney tissue. XORTX plans to provide a detailed update on the study results in early November.

XORTX Therapeutics Inc. (NASDAQ: XRTX) ha annunciato l'accettazione di un abstract per la presentazione alla Kidney Week 2024 della American Society of Nephrology. L'abstract, intitolato 'L'enzima xantina ossidasi nei ratti, nei topi e negli esseri umani con malattia renale policistica', si concentra sul ruolo della xantina ossidasi (XO) nella malattia renale policistica (PKD). Lo studio, condotto presso l'Università del Colorado e sponsorizzato da XORTX, mirava a comprendere l'attività della xantina ossidasi nel siero (XOa) nella PKD e la sua relazione con il volume totale dei reni e il declino del tasso di filtrazione glomerulare (GFR).

La ricerca ha investigato l'attività di XO nei ratti PCK1, nei topi PKD1RC/RC e in 34 pazienti dello studio clinico HALT-PKD. I risultati forniscono indicazioni su come un metabolismo aberrante delle purine nel tessuto PKD possa contribuire ai livelli di acido urico circolante, al tasso di espansione renale e al declino del GFR. Questo studio si basa sui risultati precedenti che suggeriscono un'iperespressione di XO nel tessuto renale della PKD. XORTX prevede di fornire un aggiornamento dettagliato sui risultati dello studio all'inizio di novembre.

XORTX Therapeutics Inc. (NASDAQ: XRTX) anunció la aceptación de un resumen para su presentación en la Kidney Week 2024 de la American Society of Nephrology. El resumen, titulado 'Xantina oxidasa en ratas, ratones y humanos con enfermedad renal poliquística', se centra en el papel de la xantina oxidasa (XO) en la enfermedad renal poliquística (PKD). El estudio, realizado en la Universidad de Colorado y patrocinado por XORTX, tuvo como objetivo entender la actividad de la xantina oxidasa en suero (XOa) en la PKD y su relación con el volumen renal total y el declive del índice de filtración glomerular (GFR).

La investigación investigó la actividad de XO en ratas PCK1, ratones PKD1RC/RC y 34 pacientes del estudio clínico HALT-PKD. Los resultados ofrecen perspectivas sobre cómo el metabolismo aberrante de las purinas en el tejido PKD puede contribuir a los niveles circulantes de ácido úrico, la tasa de expansión renal y el declive del GFR. Este estudio se basa en hallazgos anteriores que sugieren una sobreexpresión de XO en el tejido renal de PKD. XORTX planea proporcionar una actualización detallada sobre los resultados del estudio a principios de noviembre.

XORTX Therapeutics Inc. (NASDAQ: XRTX)는 미국 신장학회(Kidney Week 2024)에서 발표를 위한 초록이 수락되었다고 발표했습니다. 이 초록은 '다낭성 신장 질환을 가진 쥐, 마우스 및 인간에서의 자이산 산화효소'라는 제목을 가지고 있으며, 다낭성 신장 질환(PKD)에서 자이산 산화효소(XO)의 역할에 집중하고 있습니다. 콜로라도 대학교에서 진행된 이 연구는 XORTX가 후원하였으며, PKD에서의 혈청 자이산 산화효소 활성(XOa)와 총 신장 용적 및 사구체 여과율(GFR) 감소와의 관계를 이해하는 것을 목표로 하였습니다.

이 연구는 PCK1 쥐, PKD1RC/RC 마우스 및 HALT-PKD 임상 연구의 34명의 환자에서 XO 활성을 조사하였습니다. 결과는 PKD 조직에서 비정상적인 퓨린 대사가 어떻게 순환하는 요산 수치, 신장 확장률 및 GFR 감소에 기여할 수 있는지를 보여줍니다. 이 연구는 PKD 신장 조직에서 XO의 과발현을 시사하는 이전 연구 결과를 기반으로 합니다. XORTX는 11월 초에 연구 결과에 대한 상세한 업데이트를 제공할 계획입니다.

XORTX Therapeutics Inc. (NASDAQ: XRTX) a annoncé l'acceptation d'un résumé pour présentation lors de la Kidney Week 2024 de l'American Society of Nephrology. Le résumé, intitulé 'Oxydase de xanthine chez les rats, les souris et les humains atteints de maladie polykystique des reins', se concentre sur le rôle de l'oxydase de xanthine (XO) dans la maladie polykystique des reins (PKD). L'étude, menée à l'université du Colorado et parrainée par XORTX, visait à comprendre l'activité de l'oxydase de xanthine dans le sérum (XOa) dans la PKD et sa relation avec le volume rénal total et le déclin du taux de filtration glomérulaire (GFR).

La recherche a étudié l'activité de XO chez des rats PCK1, des souris PKD1RC/RC et 34 patients issus de l'étude clinique HALT-PKD. Les résultats offrent des aperçus sur la manière dont le métabolisme aberrant des purines dans le tissu PKD peut contribuer aux niveaux d'acide urique circulants, au taux d'expansion rénale et au déclin du GFR. Cette étude s'appuie sur des résultats antérieurs suggérant une surexpression de XO dans le tissu rénal PKD. XORTX prévoit de fournir une mise à jour détaillée des résultats de l'étude début novembre.

XORTX Therapeutics Inc. (NASDAQ: XRTX) gab die Annahme eines Abstracts für die Präsentation auf der Kidney Week 2024 der American Society of Nephrology bekannt. Der Abstract mit dem Titel 'Xanthinoxidase bei Ratten, Mäusen und Menschen mit zystischer Nierenerkrankung' konzentriert sich auf die Rolle der Xanthinoxidase (XO) bei der zystischen Nierenerkrankung (PKD). Die Studie, die an der Universität von Colorado durchgeführt und von XORTX gesponsert wurde, zielte darauf ab, die Serum-Xanthinoxidase-Aktivität (XOa) bei PKD und deren Zusammenhang mit dem Gesamtvolumen der Nieren und dem Rückgang der glomerulären Filtrationsrate (GFR) zu verstehen.

Die Forschung untersuchte die XO-Aktivität bei PCK1-Ratten, PKD1RC/RC-Mäusen und 34 Patienten aus der HALT-PKD-Studie. Die Ergebnisse bieten Einblicke, wie abnormer Purinstoffwechsel im PKD-Gewebe zu den zirkulierenden Harnsäurespiegeln, der Nierenexpansionsrate und dem Rückgang der GFR beitragen kann. Diese Studie baut auf früheren Erkenntnissen auf, die eine Überexpression von XO im PKD-Nierengewebe nahelegen. XORTX plant, Anfang November ein detailliertes Update zu den Studienergebnissen zu geben.

Positive
  • Acceptance of abstract for presentation at a major nephrology conference, indicating scientific recognition
  • Study provides insights into the role of xanthine oxidase in PKD progression, potentially supporting XORTX's therapeutic approach
  • Research encompasses multiple species (rats, mice, humans), strengthening the validity of findings
Negative
  • Full study results are not yet available, limiting immediate assessment of potential impact on XORTX's drug development
  • No direct mention of how this research translates to XORTX's current drug candidates or clinical trials

Insights

This abstract presentation at the ASN Kidney Week 2024 provides valuable insights into the role of xanthine oxidase (XO) in polycystic kidney disease (PKD). The study's findings, linking XO activity to cyst growth and disease progression, could have significant implications for XORTX's XRx-008 program.

Key points to consider:

  • The research spans mouse, rat and human studies, enhancing its translational value.
  • It explores the relationship between XO activity, total kidney volume and GFR decline, which are important markers in PKD progression.
  • The study may provide a scientific basis for XORTX's approach in targeting XO for PKD treatment.

While this news doesn't provide immediate financial impact, it strengthens XORTX's scientific position and could influence future clinical trials and drug development strategies. Investors should monitor how these findings translate into the company's pipeline progress and potential partnerships in the nephrology space.

This abstract acceptance represents a moderate positive development for XORTX, albeit without immediate financial implications. Here's why it matters:

  • Scientific validation: Acceptance by ASN, a prestigious nephrology society, lends credibility to XORTX's research direction.
  • Competitive advantage: Novel insights into XO's role in PKD could differentiate XORTX's approach in a competitive market.
  • Potential catalyst: Positive results could attract investor attention and possibly lead to partnerships or funding opportunities.

However, investors should temper expectations as:

  • Abstract acceptance doesn't guarantee groundbreaking results.
  • Translating preclinical findings to successful human trials remains challenging.
  • The full impact on XORTX's pipeline and valuation will only become clear after the full presentation in November.

Overall, this news reinforces XORTX's position in PKD research but doesn't significantly alter the company's near-term prospects.

Health consequences of over active xanthine oxidase may accelerated PKD progression

CALGARY, Alberta, Aug. 20, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the acceptance of an abstract submitted to the American Society of Nephrology (the “ASN”).  The abstract entitled "Xanthine oxidase in rats, mice and humans with polycystic kidney disease" was reviewed by the ASN review panel for scientific merit and novel discoveries.  The study was conducted at the University of Colorado in the independent laboratory of Dr. Charles Edelstein and was sponsored by XORTX and will be presented during the Session Title: Genetic Diseases: Cystic - Therapeutic Investigations and Prognosis.

About this study

The xanthine oxidase (“XO”) enzyme is an essential enzyme within the uric acid pathway, and is required for the breakdown of purine nucleotides. Uric acid as well as reactive oxygen species released during the enzymatic reaction may also play a detrimental role in the circulatory system and within tissue during disease. Recent pioneering discoveries in rodent models of polycystic kidney disease (“PKD”) implicate over expression or over activity of XO. It is currently unknown if XO over expression or over activity in humans is associated with PKD or more rapid progression of disease.   The aim of the study was to gain insight into whether increased XO activity results in cyst growth, XO activity was measured in PCK1 rats, PKD1RC/RC (RC) mice and 34 patients from the HALT-PKD Clinical study.

The abstract outlines study results from mouse, rat and human studies of PKD. The purpose of the study was to gain an understanding of serum xanthine oxidase activity (XOa) in PKD during varied stages of disease and further to relate that activity to total kidney volume, and decline of glomerular filtration rate (GFR).  The results of the study provide understanding of where aberrant purine metabolism in PKD tissue due to sources XO enzyme may contribute to circulating uric acid levels, expansion rate of kidney and cyst and functional GFR decline.  Prior study results suggested over expression of XO in PKD kidney tissue may be a feature of cystic disease. XORTX will provide a further update on the results of the study during the first week of November.

Dr. Allen Davidoff, CEO of XORTX, stated, “We are pleased to once again be presenting pioneering studies in PKD due to ADPKD at the American Society of Nephrology annual meeting during Kidney Week 2024 with this poster presentation. Most importantly, results of this study deepen our understanding of how increased serum uric acid or aberrant kidney tissue expression of XO contribute to accelerate injury using data from mouse, rat and human studies of PKD. The XRx-008 program continues to pioneer our understanding of how too much or too active xanthine oxidase may result in a health consequence in PKD.”

About the American Society of Nephrology – Kidney Week

ASN represents more than 21,000 kidney health professionals working to help people with kidney diseases and their families. (Source: https://www.asn-online.org/)

The Kidney Week Conference is attended by approximately 10,000 other kidney professionals from across the globe at Kidney Week 2024 in Orlando, Florida. The world's premier nephrology meeting, Kidney Week provides participants with exciting and challenging opportunities to exchange knowledge, learn the latest scientific and medical advances, and listen to engaging and provocative discussions with leading experts in the field. (Source: https://www.asn-online.org/education/kidneyweek/American Society of Nephrology - Program and Abstracts)

The Kidney Week program is available on the ASN website. Abstracts will be available on the ASN website by October 14, 2024.

About ADPKD

ADPKD is a rare disease that affects more that 10 million individuals worldwide.1,2 ADPKD is typically diagnosed based upon expansion of fluid-filled cysts in the kidneys. Over time, the increasing number and size of cysts can contribute to structural and functional changes to kidneys and is frequently accompanied by chronic pain which is a common problem for patients with ADPKD.3 Expansion of cysts is thought to compress healthy functioning tissue surrounding the cysts and contribute to further loss of kidney function, fibrosis, impaired nutrient exchange and impaired kidney function, accompanied later by end-stage renal disease.1 Health consequences of high uric acid have been reported to be increased in ADPKD individuals, including increased incidence of kidney stones5 and gout.6,7 For individuals with progressing ADPKD, treatment recommendations include anti-hypertensive treatment, dietary restrictions, and, for a limited percentage of suitable patients, pharmacotherapy.4 New, more broadly applicable therapies to effectively slow decline of kidney function in ADPKD are needed.

References:

  1. Wiley C., Kamat S., Stelhorn R., Blais J., Analysis of nationwide date to determine the incidence and diagnosis of autosomal dominant polycystic kidney disease in the USA, Kidney Disease, 5(2): 107-117, 2019
  2. Bergmann C., Guay-Woodford L.M., Harris P.C., Horie S., Peters D.J., Torres V.E., Polycystic Kidney Disease, Nat Rev Dis Primers. 4(1): 50, 2018
  3. https://pkdcure.org/living-with-pkd/chronic-pain-management
  4. Gimpel C., Bergmann C., Bockenhauer D., et al., International consensus statement of the diagnosis and management of autosomal dominant polycystic kidney disease in children and young people, Nat Rev Nephrol 15(11):713-726, 2019
  5. Torres VE, et al, The association of nephrolithiasis and autosomal dominant polycystic kidney disease, Am J Kidney Dis, 1988, vol 11, 318-325
  6. Newcombe, DS. Letter Gouty Arthritis and polycystic kidney disease, Ann Intern Med, 1973 vol 79, pg 605
  7. Rivera JV Martinez, et al, Association of hyperuricemia and polycystic kidney disease, Bol Asoc Med P R, 1965 vol 7 251-263

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 future health of patients. Additional information on XORTX is available at www.xortx.com.

For more information, please contact: 
  
Allen Davidoff, CEONick Rigopulos, Director of Communications
adavidoff@xortx.com or +1 403 455 7727nick@alpineequityadv.com or +1 617 901 0785
  
Kim Golodetz, LHA Investor Relations 
kgolodetz@lhai.com or +1 212 838 3777 
  

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 is the main focus of XORTX's research presented at ASN Kidney Week 2024?

XORTX's research focuses on the role of xanthine oxidase (XO) in polycystic kidney disease (PKD), studying its activity in rats, mice, and humans to understand its relationship with kidney volume and glomerular filtration rate decline.

How might the findings of XORTX's study impact PKD treatment?

The study's findings could provide insights into how increased serum uric acid or aberrant kidney tissue expression of XO contributes to accelerated injury in PKD, potentially leading to new therapeutic approaches.

When will XORTX (NASDAQ: XRTX) release the full results of their PKD study?

XORTX plans to provide a detailed update on the study results during the first week of November 2024, following the presentation at ASN Kidney Week.

What is the significance of xanthine oxidase in XORTX's research on PKD?

Xanthine oxidase is an essential enzyme in the uric acid pathway, and its overexpression or overactivity may play a detrimental role in PKD progression, making it a potential target for XORTX's therapeutic development.

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