XORTX Initiates Precision Medicine Program
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU) has initiated a precision medicine program following recent research linking genetic factors to xanthine oxidase (XO) over-expression in various diseases, including kidney disease. This initiative aims to combine genetic diagnostics with XO inhibition to target individuals most in need.
The company plans to develop diagnostic tools to identify specific genetic factors, which, combined with XORTX's expertise in developing unique formulations of uric acid lowering agents and XO inhibitors, will allow for tailored treatments for subpopulations with common susceptibilities or similar drug responses.
XORTX will begin evaluating individuals in their planned registration clinical trial for autosomal dominant polycystic kidney disease (ADPKD) patients, providing an opportunity to better understand the role of genetic factors in progressive kidney disease.
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU) ha iniziato un programma di medicina di precisione a seguito di recenti ricerche che collegano fattori genetici all'iperespressione della xantina ossidasi (XO) in varie malattie, comprese quelle renali. Questa iniziativa mira a combinare diagnosi genetiche con inibizione di XO per mirare agli individui più bisognosi.
L'azienda prevede di sviluppare strumenti diagnostici per identificare specifici fattori genetici, che, combinati con l'esperienza di XORTX nello sviluppo di formulazioni uniche di agenti riduttori dell'acido urico e inibitori di XO, permetteranno trattamenti su misura per sottopopolazioni con comuni suscettibilità o simili risposte ai farmaci.
XORTX inizierà a valutare gli individui nel loro trial clinico di registrazione pianificato per i pazienti con malattia renale policistica autosomica dominante (ADPKD), offrendo l'opportunità di comprendere meglio il ruolo dei fattori genetici nella malattia renale progressiva.
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU) ha iniciado un programa de medicina de precisión después de investigaciones recientes que vinculan factores genéticos con la sobreexpresión de xantina oxidasa (XO) en diversas enfermedades, incluyendo enfermedades renales. Esta iniciativa tiene como objetivo combinar diagnósticos genéticos con la inhibición de XO para dirigirse a los individuos más necesitados.
La compañía planea desarrollar herramientas diagnósticas para identificar factores genéticos específicos, que, combinados con la experiencia de XORTX en el desarrollo de formulaciones únicas de agentes reductores de ácido úrico e inhibidores de XO, permitirán tratamientos personalizados para subpoblaciones con susceptibilidades comunes o respuestas similares a medicamentos.
XORTX comenzará a evaluar a los individuos en su ensayo clínico de registro planificado para pacientes con enfermedad renal poliquística autosómica dominante (ADPKD), proporcionando una oportunidad para comprender mejor el papel de los factores genéticos en la enfermedad renal progresiva.
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU)는 정밀 의학 프로그램을 시작했습니다. 이는 최근 연구에서 다양한 질병, 특히 신장 질환에서 xanthine oxidase (XO) 과발현과 유전적 요인 간의 연관성을 발견했기 때문입니다. 이 이니셔티브는 유전자 진단과 XO 억제를 결합하여 가장 도움이 필요한 개인을 목표로 하는 것을 목적으로 하고 있습니다.
회사는 특정 유전적 요인을 식별하기 위한 진단 도구를 개발할 계획입니다, 이 도구는 XORTX의 독창적인 요산 감소제 및 XO 억제제의 독특한 제형 개발 전문성과 결합하여 특정 치료법을 제공할 것입니다. 이는 공통된 민감성이나 유사한 약물 반응을 가진 하위 집단을 위해 적용될 수 있습니다.
XORTX는 자식 우성 다낭신장병(ADPKD) 환자를 위한 계획된 등록 임상 시험에서 개인을 평가하기 시작할 것이며, 이는 진행성 신장 질환에서 유전적 요인의 역할을 더 잘 이해할 기회를 제공합니다.
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU) a lancé un programme de médecine de précision suite à des recherches récentes établissant un lien entre des facteurs génétiques et la surexpression de la xanthine oxydase (XO) dans diverses maladies, y compris les maladies rénales. Cette initiative vise à combiner le diagnostic génétique avec l'inhibition de XO pour cibler les individus qui en ont le plus besoin.
L'entreprise prévoit de développer des outils diagnostiques pour identifier des facteurs génétiques spécifiques, qui, associés à l'expertise de XORTX dans le développement de formulations uniques d'agents réduisant l'acide urique et d'inhibiteurs de XO, permettront de proposer des traitements personnalisés pour des sous-populations ayant des susceptibilités communes ou des réponses médicamenteuses similaires.
XORTX commencera à évaluer les individus dans son essai clinique d'enregistrement prévu pour les patients atteints de maladie polykystique rénale autosomique dominante (ADPKD), offrant une occasion de mieux comprendre le rôle des facteurs génétiques dans la maladie rénale progressive.
XORTX Therapeutics Inc. (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU) hat ein Präzisionsmedizin-Programm initiiert, nachdem kürzliche Forschungen genetische Faktoren mit einer Überexpression der Xanthinoxidase (XO) in verschiedenen Erkrankungen, einschließlich Nierenerkrankungen, in Verbindung gebracht haben. Diese Initiative zielt darauf ab, genetische Diagnostik mit XO-Hemmung zu kombinieren, um Personen zu identifizieren, die am dringendsten Hilfe benötigen.
Das Unternehmen plant, diagnostische Werkzeuge zu entwickeln, um spezifische genetische Faktoren zu identifizieren, die in Kombination mit XORTXs Expertise in der Entwicklung einzigartiger Formulierungen von Harnsäuresenkern und XO-Hemmern maßgeschneiderte Behandlungen für Subpopulationen mit gemeinsamen Anfälligkeiten oder ähnlichen Arzneimittelreaktionen ermöglichen.
XORTX wird beginnen, Individuen in ihrer geplanten Registrierung klinischen Studie für Patienten mit autosomal dominanter zystischer Nierenerkrankung (ADPKD) zu bewerten, was die Möglichkeit bietet, die Rolle genetischer Faktoren bei fortschreitenden Nierenerkrankungen besser zu verstehen.
- Initiation of a precision medicine program targeting genetic factors linked to xanthine oxidase over-expression
- Potential expansion of company programs by combining genetic diagnostics with XO inhibition
- Opportunity to develop tailored treatments for specific subpopulations
- Planned evaluation of genetic factors in upcoming ADPKD clinical trial
- None.
Insights
This announcement of XORTX's precision medicine program represents a significant shift in the company's approach to treating kidney diseases. By integrating genetic diagnostics with their existing expertise in xanthine oxidase inhibitors, XORTX is positioning itself at the forefront of personalized medicine for kidney disorders.
The potential impact of this program is substantial:
- It could lead to more effective treatments for subpopulations of patients with specific genetic profiles
- It may improve patient outcomes by tailoring treatments to individual genetic factors
- It opens up new avenues for drug development and could expand XORTX's pipeline
However, investors should note that precision medicine programs often require significant investment in research and development. While promising, the timeline for realizing commercial benefits from this initiative may be extended. The integration of genetic testing into the planned ADPKD clinical trial is a positive step, potentially providing valuable data to support this new direction.
Overall, this move aligns XORTX with current trends in biotechnology and could enhance its competitive position in the kidney disease treatment market.
While XORTX's new precision medicine program is scientifically intriguing, its financial implications are less clear in the short term. For a company with a market cap of just
Key financial considerations include:
- Increased R&D expenses are likely as the company develops genetic diagnostic tools
- Potential for new partnerships or licensing agreements in the genetic testing space
- Possible expansion of addressable market by targeting specific genetic subpopulations
- Long-term potential for higher-priced, tailored therapies if successful
Investors should be prepared for increased cash burn in the near term as XORTX invests in this new program. However, if successful, this could lead to more valuable and differentiated products in the future, potentially justifying a higher valuation multiple.
The integration of genetic testing into the planned ADPKD trial is a cost-effective way to gather initial data. This approach could provide early validation of the concept without significant additional expense, which is important for a company of XORTX's size.
Pioneering Research Indicating a Role for Genetic Regulation of Xanthine Oxidase and Therapeutic Targeting of Aberrant Purine Metabolism
CALGARY, Alberta, Oct. 09, 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 the Company has initiated a precision medicine program. On August 29, 2024, XORTX announced that independent peer-reviewed research reported that genetic factors are linked to the over-expression of xanthine oxidase (“XO”) and play a role in several diseases, including kidney disease. These ground-breaking findings provide an opportunity to expand the Company’s programs and approach by combining genetic diagnostics focused on treating kidney and diseases such as sepsis by inhibiting XO with xanthine oxidase inhibition targeting individuals most in need.
Recent pioneering discoveries provide XORTX with the opportunity to develop diagnostics that identify specific genetic factors. These diagnostic tools alongside XORTX’s expertise at developing unique formulations of uric acid lowering agents and XO inhibitors will permit XORTX to tailor treatments to subpopulations of individuals that have common susceptibility or similar response to a particular drug.
Dr. Allen Davidoff, PhD., Chief Executive Officer of XORTX commented, “The application of genetic diagnostic tools and recent pioneering discoveries in autosomal dominant polycystic kidney disease (“ADPKD”), diabetic kidney and non-diabetic kidney disease provide a unique opportunity that XORTX is ideally positioned to address. The new opportunity to specifically identify a series of genetic factors, then targeting XO has enormous therapeutic potential for treating the health consequences associated with these alleles. The Company will begin evaluating individuals as early as our planned registration clinical trial in patients with ADPKD providing XORTX with an opportunity to better understand the role these genetic factors play in progressive kidney disease.”
About Xanthine Oxidase:
Xanthine oxidase is an essential enzyme within the uric acid metabolic pathway and is required for the breakdown of purine nucleotides. The breakdown products of XO, uric acid (UA) and reactive oxygen species (ROS), are released during the enzymatic reaction and may play a detrimental role in the circulatory system and within tissue during disease. XORTX sponsored discoveries in rodent models of polycystic kidney disease (“PKD”) implicate over-expression or over-activity of XO as a potentially important target in treating this disease.
Recent evidence for the over expression of Xanthine Oxidase in Disease:
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 PKD(1). Recently, new emerging discoveries link genetic factors to specific populations and show that higher XO expression is associated with a variety of conditions including hyperuricemia(2), sepsis, organ failure and sepsis associated 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).
References:
- 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
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, 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
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