Acute Kidney Injury

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.

Podium presentation will include clinical updates from the Company's Phase 2 clinical trials investigating azeliragon, in combination with radiation therapy with or without temozolomide, in newly diagnosed glioblastoma WESTON, Fla., March 19, 2024 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, announced today that the Company's CEO, Stephen Marcus, M.D., will be an expert speaker at 5th Annual Glioblastoma Drug Development Summit, which will be held March 26-28 at the Hilton Boston Logan Airport. In a podium presentation titled, "Delving into a Clinical Update on Azeliragon for GBM - Implementable Lessons for Small Molecule Success," Dr. Marcus will update the Company's progress with its two ongoing azeliragon Phase 2 clinical trials. In those clinical trials, azeliragon is being studied in combination with radiation therapy with or without temozolomide in patients with newly diagnosed glioblastoma (GBM). In addition, Dr. Marcus will also highlight azeliragon's potential effect on cerebral edema and its potential ability to reduce dexamethasone dosing in GBM treatment. GBM is a highly malignant primary brain tumor for which current therapeutic options provide a limited life extension benefit. In 2023, Cantex received Food and Drug Administration Orphan Drug Designation for azeliragon for the treatment of glioblastoma. FDA Orphan Drug Designation provides Cantex with seven years of azeliragon marketing exclusivity from the time of product launch for the orphan indication, and several other important benefits, including assistance in the drug development process, tax credits for clinical costs, and exemptions from certain FDA fees. "We look forward to meeting with esteemed colleagues and researchers to discuss the many important advances being made to combat this challenging and devastating form of cancer and azeliragon's progress in our two Phase 2 GBM clinical trials," said Dr. Marcus. "We are excited to discuss this novel clinical candidate and its unique mechanism of action, which we believe has the potential to impact several devastating cancers that continue to challenge researchers, oncologists, and patients." Details of the event are as follows: During the conference, Dr. Marcus will conduct one-on-one meetings to review the Company's business and clinical development strategy, recent corporate achievements, and upcoming milestones. About Azeliragon Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer's disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from which Cantex licensed worldwide rights to azeliragon. Clinical safety data from these trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated. Cantex has ongoing Phase 2 clinical trials in glioblastoma, brain metastasis, pancreatic cancer, breast cancer, and a Phase 3 trial in hospitalized patients with pneumonia to prevent acute kidney injury. These trials are based on azeliragon's robust preclinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials. About Cantex Pharmaceuticals, Inc. Cantex Pharmaceuticals, Inc. is a privately held, clinical stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed. For more information, please visit . About vTv Therapeutics Inc. vTv Therapeutics Inc. is a clinical stage biopharmaceutical company focused on developing oral, small molecule drug candidates, led by cadisegliatin (TTP399), a potentially transformative treatment for the reduction of hypoglycemic episodes in type 1 diabetes patients. To learn more please visit vtvtherapeutics.com. Contact Information: Tiberend Strategic Advisors, Inc. Investors Daniel Kontoh-Boateng +1 862-213-1398 dboateng@tiberend.com Media Casey McDonald +1 646-577-8520 cmcdonald@tiberend.com Cantex Pharmaceuticals, Inc. Stephen G. Marcus, M.D. +1 954-315-3660 info@cantex.com Juan F. Rodriguez +1 954-315-3660 info@cantex.com Company Codes: NASDAQ-NMS:VTVT

Trial met the primary efficacy endpoint demonstrating non-inferiority of the incidence of acute kidney injury for INTERCEPT Red Blood Cells compared to conventional red blood cells CONCORD, Calif.--(BUSINESS WIRE)-- Cerus Corporation (Nasdaq: CERS) today announced positive topline results for ReCePI, a pivotal Phase 3 clinical trial of pathogen reduced INTERCEPT Red Blood Cells (INTERCEPT RBCs) transfused to complex cardiac surgery patients. The trial met its primary efficacy endpoint, demonstrating non-inferiority for INTERCEPT RBCs compared to conventional RBCs as measured by the incidence of acute kidney injury (AKI) following transfusion of study RBCs. AKI is a sensitive transfusion efficacy indicator of RBC tissue oxygen delivery. In transfused subjects, by modified intent to treat (mITT pre-specified primary endpoint population) the incidence of AKI was 29.3% (46/157) for INTERCEPT RBC recipients compared to 28.0% (45/161) for conventional RBC recipients, demonstrating non-inferiority of INTERCEPT RBCs compared to conventional RBCs with an upper limit of the 95% confidence interval of 10.4% compared with a non-inferiority margin of 14.0% (p = 0.001). The safety endpoint of the proportion of patients with any related treatment-emergent adverse events (TEAEs) within 28 days of last transfusion was not significantly different for INTERCEPT RBCs (2.5%) compared to conventional RBCs (0.6%) (p = 0.130). There was no clinical significance related to treatment-emergent RBC antibodies observed in five patients receiving INTERCEPT RBCs. The trial’s independent Data Safety and Monitoring Board (DSMB) evaluated these patients and was supportive of continued transfusion, having found no adverse health effects from trial transfusions. This safety endpoint is also being explored in the Company’s ongoing RedeS Phase 3 clinical trial of INTERCEPT RBCs. Cerus plans to include data from both the ReCePI and RedeS clinical trials in an integrated safety analysis as part of its planned modular premarket approval (PMA) submission. “We are extremely pleased by the positive topline results of the ReCePI clinical trial,” stated Richard Benjamin, M.D., Ph.D., Cerus’ chief medical officer and principal investigator for the ReCePI clinical trial. “The ReCePI clinical trial is a pioneering trial in the field of transfusion medicine, providing critical information about pathogen reduced INTERCEPT RBCs compared to conventional RBCs. We continue to look forward to completing the RedeS clinical trial, Cerus’ ongoing pivotal Phase 3 clinical trial for INTERCEPT RBCs enrolling a broader patient population requiring RBC transfusion for acute and chronic anemia.” “Today’s news from the ReCePI clinical trial is an important milestone for the INTERCEPT RBC program,” stated William “Obi” Greenman, Cerus’ president and chief executive officer. “We are now one meaningful step closer to potentially making pathogen reduction available for RBCs, the most commonly transfused blood component in the U.S., with approximately 11 million transfused units annually. If approved, pathogen reduced RBCs would complement our existing commercial portfolio of INTERCEPT Systems for Platelets, Plasma, and Fibrinogen Complex, providing clinicians and patients access to the benefits of pathogen inactivation across all transfused components. We are grateful to all the patients, blood centers, and physicians who participated in the ReCePI clinical trial.” Cerus anticipates initiating a modular PMA application to the U.S. Food and Drug Administration (FDA) in the second half of 2025, with the final PMA module submission planned for the second half of 2026, upon the anticipated completion of the RedeS clinical trial. Data from preclinical studies, manufacturing information, previously reported Phase 1 and Phase 2 clinical trials in the U.S., and the successfully completed Phase 3 clinical trials in the EU (STARS and SPARC) will be included in the modular PMA submission. Cerus expects the data from the ReCePI clinical trial to be presented at upcoming medical conferences as well as to be submitted for peer reviewed publication. Webcast The Company will host a webcast at 9:00 A.M. EDT this morning, during which management will discuss the results of the ReCePI clinical trial. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at A replay will be available on Cerus’ website approximately three hours after the call through April 2, 2024. ABOUT ReCePI The ReCePI Phase 3 clinical trial was a prospective, multicenter, randomized, double-blinded, controlled, non-inferiority design trial (ClinicalTrials.gov: NCT03459287) to evaluate the efficacy and safety of RBCs prepared with the INTERCEPT RBC System in patients requiring transfusion for acute blood loss during complex cardiac surgery. Adult subjects at high risk of need for RBC transfusion received either conventional (control) or INTERCEPT RBCs during and for 7 days after complex cardiovascular surgery (CVS), including thoracic aorta surgery. The primary efficacy endpoint was the proportion of transfused patients with acute kidney injury (AKI) defined by increased serum creatinine (≥0.3 mg/dL or 26.5 µmol/L) from pre-transfusion baseline, within 48 hours of surgery. Safety endpoints were defined as related TEAEs through 28 days after enrollment; or antibodies with confirmed specificity to INTERCEPT RBCs through 75 days after enrollment. The ReCePI trial enrolled and randomized a total of 581 patients across 18 clinical study sites. The mITT population included 321 patients requiring RBC transfusions in the trial. Not all enrolled patients required RBC transfusion. INTERCEPT RBCs used in the ReCePI trial were produced by several blood centers in the U.S. The development program of the INTERCEPT Red Blood Cell System has been funded in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; BARDA, under contract number HHSO100201600009C. ABOUT THE INTERCEPT BLOOD SYSTEM FOR RED BLOOD CELLS The INTERCEPT Blood System for Red Blood Cells is designed to inactivate blood-borne pathogens and donor leukocytes in RBCs intended for transfusion. The RBC system is designed to prevent pathogen replication by using a small molecule compound added to RBC concentrates to form bonds within nucleic acids in pathogens and leukocytes that may be present in donated RBC collections. The nucleic acid targeted mechanism of action is designed to preserve the therapeutic qualities of the RBCs. RBCs, platelets, and plasma do not require nucleic acid replication for therapeutic efficacy. The RBC system is under development and is not yet approved anywhere in the world. Cerus has filed for a CE Mark for the RBC system under the Medical Device Regulation and anticipates initiating a modular PMA application submission to the FDA in the second half of 2025. ABOUT CERUS Cerus Corporation is dedicated solely to safeguarding the world’s blood supply and aims to become the preeminent global blood products company. Headquartered in Concord, California, the company develops and supplies vital technologies and pathogen-protected blood components to blood centers, hospitals, and ultimately patients who rely on safe blood. The INTERCEPT Blood System for platelets and plasma is available globally and remains the only pathogen reduction system with both CE mark and FDA approval for these two blood components. The INTERCEPT red blood cell system is under regulatory review in Europe, and in late-stage clinical development in the US. Also in the US, the INTERCEPT Blood System for Cryoprecipitation is approved for the production of Pathogen Reduced Cryoprecipitated Fibrinogen Complex (commonly referred to as INTERCEPT Fibrinogen Complex), a therapeutic product for the treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency. For more information about Cerus, visit and follow us on LinkedIn. INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation. Forward Looking Statements Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus’ products, prospects and expected results, including statements relating to: the therapeutic and commercial potential of INTERCEPT RBCs, including Cerus potentially making pathogen reduction available for RBCs; Cerus’ planned modular premarket approval (PMA) submission and the anticipated timing thereof, including Cerus’ plan to include data from both the ReCePI and RedeS clinical trials in an integrated safety analysis as part of its planned modular PMA submission; the anticipated completion of the RedeS clinical trial and the anticipated timing thereof; and other statements that are not historical fact. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation: risks associated with the uncertain and time-consuming development and regulatory process, including the risks that (a) the topline results reported herein and related findings and conclusions are subject to change following a more comprehensive review of the data from the ReCePI clinical trial, (b) Cerus’ planned modular PMA submission for INTERCEPT RBCs may be delayed or may not occur for a variety reasons, including those outside of the control of Cerus, (c) Cerus may be unable to report data from its RedeS clinical trial in a timely manner or at all and that even if reported, such data may not be supportive of Cerus’ planned modular PMA submission for INTERCEPT RBCs, (d) even if submitted, Cerus’ planned modular PMA submission for INTERCEPT RBCs may not be accepted by the FDA for review in a timely manner or at all or if accepted for review, may not be approved by the FDA in a timely manner or at all, (e) Cerus’ expectation that, if Cerus’ planned modular PMA submission for INTERCEPT RBCs is approved, the FDA would require a post marketing study, (f) Cerus may be unable to obtain CE Mark approval of INTERCEPT RBCs in a timely manner or at all, and (g) Cerus may otherwise be unable to obtain the requisite regulatory approvals to advance its pipeline programs and bring them to market in a timely manner or at all; risks related to product safety, including the occurrence of antibody reactivity to INTERCEPT RBCs; Cerus’ ability to maintain an effective, secure manufacturing supply chain, including the risks that (a) Cerus’ supply chain could be negatively impacted as a result of the evolving impact of macroeconomic developments, including the ongoing military conflicts in Ukraine and Israel, rising interest rates, inflation and the evolving effects of the COVID-19 pandemic, (b) Cerus’ manufacturers could be unable to comply with extensive FDA and foreign regulatory agency requirements, and (c) Cerus may be unable to maintain its supply agreements with its third party suppliers; risks related to Cerus’ ability to demonstrate to the transfusion medicine community and other health care constituencies that pathogen reduction is safe, effective and economical; risks associated with Cerus’ need for additional funding; risks associated with macroeconomic developments, including ongoing military conflicts in Ukraine and Israel and the COVID-19 pandemic and resulting global economic and financial disruptions, and the current and potential future negative impacts to Cerus’ business operations; and other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including under the heading “Risk Factors” in Cerus’ Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC March 5, 2024. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.