Chronic Kidney Diseases

CALGARY, Alberta, March 19, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to provide a summary of Company’s achievements in 2023 and objectives planned for 2024. Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals. Key milestones include: 1/ the grant of U.S. Orphan Drug Designation for the XRx-008 program for Autosomal Dominant Polycystic Kidney Disease (“ADPKD”) that is being developed under the US FDA 505(b)2 rules further de-risking this program; 2/ ongoing discussions with the US FDA have aligned our endpoints and other Phase 3 clinical trial elements to make XORLO™ the Company’s proprietary oxypurinol formulation, eligible for accelerated approval. These key advances on the XRx-008 program during 2023 were made possible by the exceptional efforts of our Board of Directors, employees, consultants, and vendors. We believe the goals set for 2024 will advance our lead program XRx-008 for ADPKD ever closer to conducting a registration clinical trial leading to marketing approval and value creation for XORTX and its shareholders.” In 2023, the Company made steady progress advancing its strategic plan in key areas, including chemistry, formulation, manufacturing, and non-clinical studies using XORLO™ to attenuate polycystic kidney disease (“PKD”) progression in animal models, and topline results from the XRX-OXY-101 bridging clinical study of XORLO™. Each of these milestones permit the next step in the Company’s clinical development plan, being a “registration” clinical trial – XRX-OXY-201 in pursuit of accelerated approval and support of the Company’s lead program XRx-008 program for ADPKD. Synopsis of 2023 Achievements Chemistry and Manufacturing, Clinical and Pre-Clinical Highlights – Produced drug substance for oxypurinol production and produced GMP drug substance; confirmed XORLO™ formulation, produced enhanced bioavailability and produced clinical supply of tablets for clinical trials. Regulatory Submissions to US Food and Drug Administration (“FDA”) and European Medicines Agency (“EMA”) Supporting Clinical Trial Conduct April 21, 2023 – FDA granted Orphan Drug Designation for XRx-008 program for ADKPD, following review of February 1st submission of a comprehensive scientific review package within an Orphan Drug Designation application for XRx-008 for treatment of progressing kidney disease due to ADPKD with the US FDA.May 5, 2023 – FDA confirmed the eligibility of XORLO™ for Accelerated Approval following review of March 14th submission of type D meeting request with US FDA to discuss the clinical development plan (XRX-OXY-201 clinical trial design).August 29, 2023 – XORTX submitted an Orphan Drug Designation application to the EMA for the treatment of ADPKD. Following discussion and guidance from EMA, XORTX will expand data package and resubmit to gain EMA Orphan Drug Designation. Technology and Patent Advancements January 3, 2023 – XORTX submitted a new Provisional Patent Application seeking broadened and lengthened future patent protection, in a patent entitled “Compositions and Methods for Diagnosis, Treatment and Prevention of Kidney Disease.November 2, 2023 – XORTX sponsored study results were presented at American Society of Nephrology (“ASN”) under Session Title: “Genetic Diseases of the Kidneys”, by Dr. Charles Edelstein of the University of Colorado. Results of these studies suggest that management of xanthine oxidase activity in PKD may be more important than previously appreciated and further that previously unrecognized factors related to diet, genetic factors or prescribed drugs that increase uric acid levels could potentially aggravate the progression of PKD. Organizational Highlights In addition to substantial technological advancement, the Company continued to bolster the XORTX team with the following appointments: June 26, 2023 - James Fairbairn was appointed Chief Financial Officer of XORTX Therapeutics. Mr. Fairbairn has more than 20 years of experience with publicly-traded companies. He is a Chartered Professional Accountant, and an Institute-certified Director.December 31, 2023 – Patrick Treanor was appointed as a member of the XORTX Board of Directors. Patrick Treanor is a seasoned pharmaceutical industry executive with over 25 years experience. He is the current Chief Operating Officer of Pathalys Pharma, Inc., a private company specializing in advanced therapeutics for late-stage chronic kidney disease management. Mr. Treanor earned a BS in Management from Bryant University and an MBA from Rensselaer Polytechnic Institute. On November 29, 2023, XORTX returned to NASDAQ compliance following a reverse split to increase share price to greater than $1.00. XRx-008 Program Highlights – Independent Commercial Assessment In support of ongoing pharmaceutical partnership discussions, XORTX initiated an independent commercial assessment of the XRx-008 program for ADPKD with Bluestar BioAdvisors. This evaluation included interviews with 30 Nephrologists and 10 “Payers” with Large national Plans that cover greater than 290 million lives. Outcome of this assessment suggests that the XRx-008 program for ADPKD worldwide peak net sales per year that may exceed $1B, with a total product life estimated to surpass 7 to 10 years.2024 Corporate Objectives March 4, 2024 – XORTX completed an oversubscribed financing of $2.7 million.The Company will provide guidance, in the near future, regarding 2024 Corporate Objectives including announcements regarding clinical and regulatory submissions in support of the XRX-OXY-201 clinical “registration” trial designed to demonstrate the benefit of XORLO™ in slowing the progression of declining filtering capacity in ADPKD. 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 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 U.S. Federal securities laws. 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. Except as otherwise required by law, 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 Registration Statement on Form F-1 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.

Pictured: Bayer's office in Berlin, Germany/iStock, Elenasfotos Bayer reported another Phase III win for its menopause drug candidate Tuesday, linking the molecule to improved outcomes in a long-term study and setting the stage for approval filings. The clinical trial evaluated the safety and efficacy of elinzanetant, a dual neurokinin-1,3 (NK-1,3) receptor antagonist, in the treatment of hot flashes associated with menopause over 52 weeks. Bayer reported a pair of 12-week Phase III studies met their coprimary endpoints in January 2024, but still needed a hit on the third trial to show the long-term effects of the molecule. Bayer’s topline readout revealed that the trial met its primary endpoint, which tracked the mean change in the frequency of moderate to severe hot flashes after 12 weeks of oral daily dosing with elinzanetant or placebo. The company also said the safety pro across 52 weeks of dosing was consistent with the results of previously published studies including its Phase 2b dose-finding trial. The company plans to include data from all three clinical trials in applications for approval. If Bayer wins approval, it will face competition from Astellas’ rival NK3 receptor antagonist Veozah (fezolinetant). The FDA approved Veozah for the treatment of vasomotor symptoms, the medical term for hot flashes, due to menopause in May 2023. Bayer is keeping back data from all three trials to present at upcoming scientific congresses, making it impossible to assess how elinzanetant compares to Veozah. Astellas reported 3.6 billion yen ($24 million) of Veozah sales over the first nine months of its financial year and has run ads during the Super Bowl and the Oscars to promote the product. Elinzanetant may have an advantage over Veozah because it targets NK1 and NK3, rather than just the latter receptor. Some studies have suggested NK1 antagonists may improve sleep. Bayer’s long-term trial looked at changes in sleep disturbances, which are frequently associated with hot flashes, but the company has yet to release the data. The details of the readout will shape the prospects of an important molecule for Bayer. The company has 10 programs in Phase III development but CEO Bill Anderson has warned the “late-stage pipeline is thin relative to the patent losses we have in the next years,” telling the Financial Times that the problems stem from historical under-investment. Having stopped a Phase III trial of the investigational factor XIa inhibitor asundexian in November 2023, Bayer is looking to elinzanetant and late-phase prostate cancer and chronic kidney disease prospects to drive growth and offset the upcoming loss of patent protection for Xarelto (rivaroxaban). Nick Paul Taylor is a freelance pharmaceutical and biotech writer based in London. He can be reached on LinkedIn.

It's been safer and healthier for chronic kidney disease patients TAICHUNG, March 18, 2024 /PRNewswire/ -- Chronic Kidney Disease has become a new "national disease" in Taiwan, and the number of patients requiring hemodialysis is increasing. Traditionally, hemodialysis patients and accompanies require at least an hour spent on commuting, and that maybe even more time consuming for those who live in suburbs. Moreover, patients almost feel tired or fatigue and need rest half a day following hemodialysis. Since 2019 in Taiwan, China Medical University Hospital (CMUH) has initiated and implemented AI monitoring system for hemodialysis patients. The unique AI system receives the messages sent by the dialysis machine to keep track of any risk message with incorporated AI system, ensuring patient safety and collecting information for prescription adaptation. Accredited with CMUH's AI technology, those who require hemodialysis are not necessary to visit a healthcare facility for this routine. Dr. Huey-Liang Kuo, Director of Hemodialysis Center at CMUH, stated that patients have been heathier and safer to do hemodialysis at home. Continue Reading Home hemodialysis is associated with the best outcomes. The patient Mr. Lin now has better quality of life and no dietary restrictions. Hospital visits for dialysis is no longer a burden for him. Mr. Lin, a 62-year-old residing in a remote area of Taiwan, has been undergoing hemodialysis for thirty years. With reduced mobility, he faced the challenge of commuting for two hours every other day to receive his routine four-hour hemodialysis treatment. This rigorous schedule often left him feeling fatigued after each session. However, five years ago, Mr. Lin's situation took a positive turn when he enrolled in the Home Hemodialysis (HHD) training program at China Medical University Hospital (CMUH). Since committing to HHD in 2019, Mr. Lin has experienced a significant improvement in his quality of life. Today, he not only enjoys more time with his family but also remains energetic enough to successfully manage his own business. According to Dr. Kuo, HHD has been a choice of renal replacement therapy more than fifty years in some countries, but in Taiwan the general public has very limited knowledge about it. Dr. Kuo further explained, "patients and their family are concerned about the safety of doing it at home. It is worth to notice that patients may be given the option of HHD to avoid fatigue from traditional hemodialysis which has side effects on their work and daily life. "Approximately 12% of the Taiwanese, or about 2.86 million people, have Chronic Kidney Disease, and more than 80 thousand of these patients have progressed to end-stage kidney failure that require routine hemodialysis or peritoneal dialysis, with 90% selecting the former" stated Professor Chiu-Ching Huang of Nephrology Center at CMUH. With HHD, patients may be able to return to their jobs the following day, thereby enhancing their quality of life and minimizing the disruptions caused by hemodialysis schedules. Professor Huang further explained, "The tiredness following hemodialysis comes from ultrafiltration and osmotic imbalance, as hemodialysis in medical facilities takes up 4 hours and requires rapid dehydration to remove the excess water as well as uremic toxins accumulated in a patient's body, leading to drastic change in osmotic pressure. Patients usually experience serious fatigue and require considerable rest." Prof. Huang pointed out that HHD takes a longer time (8 hours), however, water and waste are removed in a slow but steady rate that comes with a less drastic change in osmotic pressure and less stress on patient's heart. Patients don't feel tired after hemodialysis and can continue with their work." As for the safety of HHD, patients need to complete a training program lasting 8-12 weeks and encompassing the establishment of vascular access puncture, operation and troubleshooting of hemodialysis machine, blood pressure and pulse monitoring, recording a nursing report, emergency evacuation, medical waste management, and the maintaining of dialysis RO water quality. They also need to pass a final test of independent operation before they can do it at home. Media Contact: Carolyn Chen [email protected] SOURCE China Medical University Hospital