Financial Performance and Position - As of December 31, 2024, the company's cash and cash equivalents amounted to $2,473,649, a decrease of 28.2% from $3,447,665 in 2023 [661]. - The total accounts payable and accrued liabilities decreased by 48.0% from $283,428 in 2023 to $147,205 in 2024 [661]. - The lease liability increased from $11,510 in 2023 to $38,785 in 2024, indicating a significant rise in obligations [661]. - The derivative warrant liability rose from $531,000 in 2023 to $572,000 in 2024, reflecting a 7.7% increase [661]. - The company's maximum exposure to credit risk as of December 31, 2024, is the carrying value of its financial assets, which is $2,473,649 [662]. - The total financial liabilities due within three months as of December 31, 2024, is $170,329, while the total liabilities for 2023 were $294,938 [665]. - The company is exposed to a net currency risk of $573,336 as of December 31, 2024, with a potential effect of +/- 10% change in currency amounting to $57,334 [667]. - The company has implemented a planning and budgeting process to manage liquidity risk and ensure it can meet its financial obligations [663]. - The fair value of cash and cash equivalents approximates their carrying values due to their short-term nature [660]. - There have been no changes in risk management policies since December 31, 2024 [668]. Clinical Development and Programs - The Company launched a precision medicine program targeting kidney disease, linking genetic factors to the over-expression of XO [333]. - The XRx-008 program for ADPKD is in late clinical stages, with plans for a Phase 3 registration trial to support NDA submission [352]. - The Company is developing a combination therapy for AKI associated with respiratory virus infection, integrating intravenous and oral treatments [355]. - The XRx-026 program aims to decrease chronically high serum uric acid levels by inhibiting the xanthine oxidase enzyme, potentially providing a significant treatment option for gout [383]. - The XRx-008 program aims to decrease chronic kidney disease progression in patients with ADPKD, addressing symptoms like increased kidney volume and hypertension [390]. - XRx-101 is being developed for AKI associated with respiratory virus infections, with 36% of hospitalized COVID-19 patients experiencing AKI [400]. - XRx-101 aims to rapidly decrease SUA concentrations to protect kidney function during hospitalization, targeting a patient group currently without approved treatments for AKI [403]. - XRx-225 has shown potential to significantly decrease proteinuria and improve kidney function by lowering uric acid levels, even in patients receiving standard care [412]. - XRx-225 is currently in non-clinical development stages, with plans to advance to Phase 1 clinical testing pending FDA discussions [413]. Regulatory and Approval Processes - The company plans to pursue FDA approval for XRx-101 under Section 505(b)(2) of the FDCA, leveraging prior research and existing formulations [404]. - The cost of preparing and submitting a New Drug Application (NDA) is substantial, currently over $4.3 million for an NDA with clinical information [448]. - The annual program fee for an approved NDA is currently over $400,000, with fees typically increased annually [448]. - The FDA seeks to review applications for standard review drug products within ten months and priority review drugs within six months [450]. - The FDA may impose clinical holds at any time during the life of an IND due to safety concerns or non-compliance [440]. - The approval process may require substantial post-approval testing and surveillance to monitor the drug's safety or efficacy [454]. - The FDA has 60 days to determine whether an NDA will be accepted for filing, followed by an in-depth review [450]. - The FDA may issue a Complete Response Letter (CRL) detailing deficiencies in the NDA, which could require additional clinical trials [452]. - Regulatory agencies require extensive monitoring and auditing of all clinical activities and data throughout the clinical development phases [445]. - The requirement for a Risk Evaluation and Mitigation Strategy (REMS) can materially affect the potential market and profitability of the drug [454]. Market and Competitive Landscape - The competitive landscape includes major pharmaceutical and biotechnology companies, with the company facing challenges in securing market position against established therapies [429]. - The company’s commercial opportunity may be impacted if competitors develop safer or more effective therapies, or if they obtain regulatory approval more rapidly [432]. - Third-party payors may deny coverage or reimbursement if products are deemed not medically necessary, affecting sales potential [496]. - The pharmaceutical industry faces pricing pressures due to managed healthcare trends and legislative proposals, impacting reimbursement rates from third-party payors [497]. - Legislative changes at the state level may impose additional pricing constraints and affect demand for approved products [505]. Intellectual Property and Patent Strategy - The company aims to secure patent protection for its product candidates and expand its intellectual property holdings through in-licensing and new compositions [414]. - The patent portfolio includes five active patent families, with claims covering various therapeutic applications including diabetic nephropathy and insulin resistance [418]. - Owners of relevant drug patents may apply for up to a five-year patent term extension after NDA approval [462]. - The Orphan Drug Act allows for a seven-year exclusivity period for drugs approved for rare diseases affecting fewer than 200,000 individuals in the U.S. [466]. Healthcare Regulations and Compliance - The company is subject to various healthcare laws and regulations that could impact marketing and sales of approved products [506]. - The company must ensure compliance with the federal Anti-Kickback Statute and other healthcare laws to avoid significant liabilities [507]. - Compliance with evolving EU data export laws is critical, especially following the Schrems II decision, which complicates data transfers to the U.S. [495]. - Non-compliance with foreign regulatory requirements may result in fines, suspension of clinical trials, or withdrawal of regulatory approvals [493]. - The FDA and other agencies enforce laws prohibiting the promotion of off-label uses, with significant penalties for violations [475]. Research and Development Insights - Key findings from a study on Polycystic Kidney Disease indicated that elevated serum uric acid worsens kidney conditions, while XO inhibitors can reduce uric acid levels and cyst growth [335]. - Approximately 2-4% of patients treated with allopurinol develop adverse reactions, creating an unmet medical need for alternative treatments for chronic gout [367]. - Oxypurinol has the potential to benefit approximately 70-75% of allopurinol-intolerant patients, estimated to be between 120,000 to 150,000 patients in the U.S. [367]. - Clinical findings suggest that the proprietary formulation of oxypurinol, XORLOTM, may reduce adverse events and improve tolerability compared to allopurinol [386]. - Over 600 patients have been treated with oxypurinol, showing a reduced rate of adverse events compared to allopurinol, suggesting superior tolerability [395].

● XORTX will focus on key steps to advance a NDA filing for Gout indication ● CALGARY, Alberta, April 30, 2025 (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 gout and progressive kidney disease, is pleased to announce it has received responses from the US Food and Drug Administration (the “FDA”) and clarified key steps for a new drug applicati ...

Patent Supporting Gout and Autosomal Dominant Polycystic Kidney Disease Programs CALGARY, Alberta, April 28, 2025 (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 gout and progressive kidney disease , is pleased to announce receipt of notification that the patent "Xanthine Oxidase Inhibitor Formulations" will be granted by the European Patent Of ...

Core Viewpoint - XORTX Therapeutics Inc. has received notification from Nasdaq regarding non-compliance with the minimum bid price requirement, as its common shares have been below US$1.00 for 30 consecutive business days [1][2]. Group 1: Compliance Status - The notification does not currently impact the Company's listing on the Nasdaq Capital Market, and it has 180 calendar days to regain compliance [2]. - If the bid price closes at or above US$1.00 for at least 10 consecutive business days within this period, Nasdaq will notify the Company of compliance [2]. Group 2: Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on developing therapies for progressive kidney disease and gout, with three advanced products: XRx-026 for gout, XRx-008 for ADPKD, and XRx-101 for acute kidney injury [4]. - The Company is also developing XRx-225, a pre-clinical program for Type 2 diabetic nephropathy, targeting purine metabolism and xanthine oxidase to reduce uric acid production [4].

Core Points - XORTX Therapeutics Inc. has received notification from Nasdaq that it is not in compliance with the minimum bid price requirement, as its common shares have closed below US$1.00 for 30 consecutive business days [1] - The company has 180 calendar days from the notification date to regain compliance, during which its shares will continue to trade on the Nasdaq Capital Market [2] - If the bid price closes at or above US$1.00 for at least 10 consecutive business days, Nasdaq will notify the company of compliance [2] - The notification does not affect the company's listing status on the TSX Venture Exchange [3] - XORTX is evaluating options to resolve the deficiency and regain compliance with Nasdaq Rule 5550(a)(2) [3] Company Overview - XORTX Therapeutics is focused on developing therapies for progressive kidney disease and gout, with three clinically advanced products: XRx-026 for gout, XRx-008 for ADPKD, and XRx-101 for acute kidney injury [4] - The company is also developing XRx-225, a pre-clinical program for Type 2 diabetic nephropathy [4] - XORTX aims to improve the quality of life for individuals with gout and other diseases by targeting purine metabolism and xanthine oxidase to reduce uric acid production [4]

Core Insights - XORTX Therapeutics Inc. is preparing for a Type B meeting with the FDA to discuss the XRx-026 program for gout, aiming to confirm its readiness for a New Drug Application (NDA) submission [1][2] - The XRx-026 program focuses on XORLOTM, a proprietary formulation of oxypurinol, which addresses unmet medical needs in gout treatment [4] - The company anticipates that advancing the XRx-026 program will lead to a revenue-positive state for XORTX [2] Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on innovative therapies for progressive kidney disease and gout [1] - The company has three clinically advanced products: XRx-026 for gout, XRx-008 for ADPKD, and XRx-101 for acute kidney injury [8] Gout and Hyperuricemia Context - Approximately 44 million individuals in the US have elevated uric acid levels, with gout affecting about 9.2 million people [3] - Gout is linked to significant morbidity, including severe pain and reduced quality of life, and is associated with various health conditions such as metabolic syndrome and chronic kidney disease [3] XRx-026 Program Details - The XRx-026 program is developing XORLOTM, which aims to provide an alternative to existing xanthine oxidase inhibitors (XOIs) like Allopurinol and Febuxostat [4] - Allopurinol is commonly prescribed but not tolerated by 3-5% of patients, while Febuxostat faced a decline in use due to safety concerns [4] FDA Meeting Insights - Type B meetings with the FDA are designed to discuss the development status of drug applications and confirm any additional information needed for marketing approval [5]

Core Viewpoint - XORTX Therapeutics Inc. has requested a Type C meeting with the FDA to discuss the XRx-026 program for gout treatment, indicating readiness for a New Drug Application (NDA) submission for its proprietary drug formulation, XORLO [1][2]. Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on innovative therapies for progressive kidney disease and gout, with three clinically advanced products: XRx-026 for gout, XRx-008 for ADPKD, and XRx-101 for acute kidney injury [6]. XRx-026 Program Details - The XRx-026 program is developing XORLO, a proprietary formulation of oxypurinol aimed at treating gout, particularly for patients intolerant to allopurinol [4]. - The program has progressed sufficiently to warrant a review with the FDA, with the meeting expected to occur within 75 days of the request [2]. Gout Prevalence and Impact - Approximately 44 million individuals in the US have elevated uric acid levels, with about 9.2 million suffering from symptomatic gout, which is linked to severe pain and increased healthcare costs [3]. - Gout is associated with serious health conditions, including myocardial infarction and chronic kidney disease, impacting quality of life and economic productivity [3]. Current Treatment Landscape - Oral xanthine oxidase inhibitors (XOIs) are the preferred treatment for gout, with allopurinol being the most commonly prescribed, totaling around 3.3 million prescriptions annually in the US [4]. - Febuxostat, another XOI, had peak sales of approximately US$450 million but faced a decline due to safety concerns, highlighting the need for alternatives like XORLO [4].

Core Viewpoint - XORTX Therapeutics Inc. is actively engaging with the investment community through its participation in the Microcap Conference, where it aims to present its innovative therapies for gout and progressive kidney disease, specifically the XRx-026 and XRx-008 programs [1][2]. Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on developing therapies for progressive kidney disease and gout, with three clinically advanced products: XRx-008 for autosomal dominant polycystic kidney disease (ADPKD), XRx-026 for allopurinol intolerant gout, and XRx-101 for acute kidney injury related to COVID-19 [3]. - The company is dedicated to improving the quality of life for patients with kidney disease and gout by targeting purine metabolism and inhibiting uric acid production [3]. Conference Participation - The Microcap Conference, held from January 28 to 30, 2025, in Atlantic City, NJ, provides XORTX with a platform to update investors on its gout program and ADPKD clinical trial preparations [1]. - The CEO of XORTX, Dr. Allen Davidoff, emphasized the importance of one-on-one meetings with potential investors and the opportunity to present the company's plans for FDA marketing approval [2].

Core Viewpoint - XORTX Therapeutics Inc. has changed its auditor from Smythe LLP to Davidson & Company LLP effective January 16, 2025, with no reservations or reportable events noted during the transition [1][2][3]. Auditor Change - The board of directors accepted the resignation of the former auditor and appointed the successor auditor effective January 16, 2025, to serve until the next annual general meeting of shareholders [2]. - There were no reservations in the former auditor's reports for any financial period, and no reportable events occurred between the company and the former auditor [3]. Compliance and Documentation - The Notice of Change of Auditor and required letters from both auditors have been reviewed by the company's audit committee and board of directors, and filed on the company's SEDAR+ profile [4]. Company Overview - XORTX Therapeutics Inc. is focused on developing innovative therapies for progressive kidney disease and gout, with three clinically advanced products: XRx-008 for ADPKD, XRx-026 for allopurinol intolerant gout, and XRx-101 for acute kidney injury related to COVID-19 [5]. - The company also has a pre-clinical program, XRx-225, targeting Type 2 Diabetic Nephropathy, aiming to improve the quality of life for patients with kidney disease and gout [5].

Core Viewpoint - XORTX Therapeutics Inc. is launching a late-stage program, XRx-026, aimed at treating gout in patients intolerant to allopurinol, with plans to discuss New Drug Application (NDA) filing with the FDA in the first half of 2025 [1][2][3] Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on innovative therapies for progressive kidney disease, with three clinically advanced products: XRx-008 for ADPKD, XRx-026 for allopurinol intolerant gout, and XRx-101 for acute kidney injury related to COVID-19 [6] Clinical Development - The clinical development of XORLO, a proprietary formulation of oxypurinol, has progressed sufficiently, leading to the initiation of discussions with the FDA regarding the XRx-026 program and potential orphan drug designation [2][3][10] Market Opportunity - In North America, approximately 3.5 million individuals suffer from gout, with 3-5% unable to tolerate allopurinol, creating a significant market opportunity for alternative treatments [5] - Febuxostat, an alternative XOI, peaked at approximately US$450 million in sales but has seen a decline due to safety concerns, highlighting the need for a new treatment option like XRx-026 [5] Health Context - Hyperuricemia affects about 14% of the global population, with 1-2% diagnosed with gout, and lowering uric acid levels is strongly correlated with improved health outcomes [4]