Polycystic Kidney, Autosomal Recessive

Top-ranked pediatric hospital system with the lowest waitlist times marks historic milestone AURORA, Colo., Jan. 8, 2025 /PRNewswire/ -- Children's Hospital Colorado (Children's Colorado) has reached a historic milestone for its Liver Transplant Program, performing a record-breaking number of transplants in 2024. With 22 liver transplants completed, Children's Colorado surpassed the program's previous record of 20 transplants in 2021. As a nationally recognized transplant program, Children's Colorado has the only pediatric-focused, multi-specialty program in the Rocky Mountain region with the lowest waitlist times in the nation. It is one of only six pediatric transplant programs nationally with significant experience performing living donor liver transplants. This achievement reflects not only the steady growth of the Liver Transplant Program over the last decade but also the team's commitment to utilizing every available option possible to get children transplanted quickly, including grafts from directed living donors (donating to a specific person), non-directed living donors (donating to a stranger) and reduced-size or split grafts from deceased donors. "When I look at this data, it's striking to see that while living donors make up roughly 15% of pediatric liver transplants nationally, they account for 40% of transplants at Children's Colorado," said Dr. Amy Feldman, Medical Director of the Liver Transplant Program at Children's Colorado. "This highlights our unique expertise and experience in performing living donor liver transplants. With the help of our adult colleagues at the University of Colorado and the altruistic community, we are able to get kids transplanted before they experience significant morbidity and mortality." In 2024, patients traveling from out of state made up 43% of liver transplants performed at Children's Colorado, underscoring the program's regional and national impact. The breadth of diagnoses of children transplanted in 2024 – such as Wilson's disease, biliary atresia, acute liver failure, Budd-Chiari syndrome, congenital hepatic fibrosis, autosomal recessive polycystic kidney disease and non-cirrhotic portal hypertension – demonstrates the team's depth of expertise and ability to address complex medical needs quickly, before patients become too sick to transplant. "Achieving this milestone required tremendous dedication and teamwork," said Dr. Michael Wachs, Surgical Director of the Abdominal Transplant Program at Children's Colorado. "We have embraced cutting-edge advances, such as plasmapheresis and intraoperative dialysis for small children, allowing us to safely take on risks while maintaining excellent outcomes." The success of the Liver Transplant Program this past year would not have been possible without the steadfast support of Children's Colorado, UCHealth University of Colorado Hospital (UCHealth) and the unwavering commitment of the entire transplant team to deliver the lowest waitlist times in the country – less than two months, compared to the national average of eight months – and the best possible patient outcomes. As the program continues to grow, it is positioned to expand its reach and impact on a larger regional and national scale to help save the lives of more children. In partnership with UCHealth, any non-directed liver donor, or a person who donates a portion of their liver without knowing the person receiving the donation, will first be offered to a patient on the Children's Colorado waitlist. To be a living donor, one needs to be a healthy adult between the ages of 18-55. The donor does not need to be related to the recipient in any way to save their life. Surgeons at UCHealth are now able to perform the donor operation with robotic assistance, allowing for donors to be out of the hospital in just a few days. To learn more about the process of becoming a donor, go to UCHealth University of Colorado Hospital Transplant Center. For more information about pediatric liver transplants, visit Children's Colorado Liver Transplant Program. ABOUT CHILDREN'S HOSPITAL COLORADO Children's Hospital Colorado is one of the nation's leading and most expansive nonprofit pediatric healthcare systems with a mission to improve the health of children through patient care, education, research and advocacy. Founded in 1908 and ranked among the best children's hospitals in the nation as recognized by U.S. News & World Report, Children's Colorado has established itself as a pioneer in the discovery of innovative and groundbreaking treatments that are shaping the future of pediatric healthcare worldwide. Children's Colorado offers a full spectrum of family-centered care at its urgent, emergency and specialty care locations throughout Colorado, including an academic medical center on the Anschutz Medical Campus in Aurora, hospitals in Colorado Springs, Highlands Ranch and Broomfield, and outreach clinics across the region. For more information, visit or connect with us on Facebook, Instagram and YouTube. Children's Hospital Colorado complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability, or sex. ATENCIÓN: si habla español, tiene a su disposición servicios gratuitos de asistencia lingüística. Llame al 1-720-777-9800. CHÚ Ý: Nếu bạn nói Tiếng Việt, có các dịch vụ hỗ trợ ngôn ngữ miễn phí dành cho bạn. Gọi số 1-720-777-9800. Media Contacts: Blayke Roznowski, Children's Hospital Colorado 24/7 media line: 303-890-8314 [email protected] SOURCE Children's Hospital Colorado WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

● Health consequences of chronically high uric acid and xanthine oxidase in polycystic kidney disease in rats, mice and humans ● CALGARY, Alberta, Oct. 24, 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 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. Selected results from the study include: In both Rat or Mouse models of Polycystic Kidney Disease (“PKD”) i) Use of a uricase inhibitor to increase uric acid resulted in increased cyst growthii) Xanthine oxidase (“XO”) inhibitor – Oxypurinol – decreased serum uric acid and cyst growthiii) Increased XO staining in kidney and liver was abundant Prospective / Retrospective Clinical Results of the Halt Clinical Trial - Group A - Early PKD patients iv) Patients with increased serum uric acid had increased total kidney volumev) Patients with increased serum uric acid had faster PKD progressionvi) Increase serum XO activity was associated with an earlier onset of high blood pressure About this Study The 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 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 rat, mice and human studies of PKD. The purpose of the study was to gain and 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. Dr. Allen Davidoff, CEO of XORTX commented, “Exploring and understanding the contribution of chronically increased serum uric acid and/or the effect of too much or too active XO enzyme in the circulation or tissue on PKD disease progression was a goal of this study. The study results presented today are another pioneering first step towards characterizing how and when to treat individuals with PKD as well as how they might benefit from the Company’s XRx-008 program, and our upcoming registration clinical trial. This study was a preliminary investigation of individuals with early stage PKD, and provided information regarding the health consequences of hyperuricemia and XO. The Company will continue to add to this exciting discovery with future exploration of later stage PKD, assessment of genetic factors contributing to aberrant purine metabolism, including XO overexpression, and this precision medicine opportunity.” 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, CEONick Rigopulos, Director of Communicationsadavidoff@xortx.com or +1 403 455 7727nick@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.

● Health consequences of chronically high uric acid and xanthine oxidase in polycystic kidney disease in rats, mice and humans ● CALGARY, Alberta, Oct. 24, 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 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. Selected results from the study include: In both Rat or Mouse models of Polycystic Kidney Disease (“PKD”) i) Use of a uricase inhibitor to increase uric acid resulted in increased cyst growth ii) Xanthine oxidase (“XO”) inhibitor – Oxypurinol – decreased serum uric acid and cyst growth iii) Increased XO staining in kidney and liver was abundant Prospective / Retrospective Clinical Results of the Halt Clinical Trial - Group A - Early PKD patients iv) Patients with increased serum uric acid had increased total kidney volume v) Patients with increased serum uric acid had faster PKD progression vi) Increase serum XO activity was associated with an earlier onset of high blood pressure About this Study The 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 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 rat, mice and human studies of PKD. The purpose of the study was to gain and 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. Dr. Allen Davidoff, CEO of XORTX commented, “Exploring and understanding the contribution of chronically increased serum uric acid and/or the effect of too much or too active XO enzyme in the circulation or tissue on PKD disease progression was a goal of this study. The study results presented today are another pioneering first step towards characterizing how and when to treat individuals with PKD as well as how they might benefit from the Company’s XRx-008 program, and our upcoming registration clinical trial. This study was a preliminary investigation of individuals with early stage PKD, and provided information regarding the health consequences of hyperuricemia and XO. The Company will continue to add to this exciting discovery with future exploration of later stage PKD, assessment of genetic factors contributing to aberrant purine metabolism, including XO overexpression, and this precision medicine opportunity.” 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. 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. Released October 24, 2024