Summary of XORTX Therapeutics Webinar Company Overview - **Company**: XORTX Therapeutics (NasdaqCM:XRTX) - **Focus Areas**: Development of therapies for gout and autosomal dominant polycystic kidney disease (ADPKD) [9][12] Core Points and Arguments Gout Program - **Primary Focus**: Developing a formulation of oxypurinol for gout treatment, targeting allopurinol-intolerant patients [9][11] - **Market Opportunity**: Estimated at $700 million for allopurinol-intolerant gout patients, filling a gap left by febuxostat [11][15] - **Clinical Background**: The drug has a safe and effective clinical record established in over 800 patients [12] - **Regulatory Path**: Preparing to file a New Drug Application (NDA) within the next 12 to 14 months, with a review period of 9 to 12 months [19][26] - **Patient Demographics**: Approximately 50 million individuals have high uric acid levels, with 7 to 9 million diagnosed with gout [14] - **Pricing Strategy**: Projected launch price between $6,000 and $8,000 per patient per year [15] ADPKD Program - **Market Opportunity**: Estimated at $1 to $1.5 billion, with a focus on patients with high uric acid levels [12][24] - **Regulatory Path**: A single small registration trial may suffice for FDA approval [12] - **Current Treatment Landscape**: Tolvaptan is the only approved drug, costing $156,000 per patient per year, treating only 5% of patients [23][25] - **Potential Market Size**: Estimated peak net sales could exceed $1 billion by treating around 18,000 individuals [25] Development and Manufacturing - **Key Activities**: Preparing an Investigational New Drug (IND) application and conducting pharmacokinetic studies [10][20] - **Manufacturing Plans**: A year of stability testing is required before NDA filing [21][26] - **Team Expertise**: Strong developmental team with experience in drug development and commercialization [27][28] Additional Important Information - **Patent Protections**: Recent EU patent grants cover major countries, with U.S. patents extending through 2034 and additional layers through 2041 and 2043 [34][35] - **Commercialization Strategy**: Plans to establish manufacturing and distribution contracts in preparation for the product launch [36] - **Market Capitalization**: Currently around $4 million to $5 million, with potential for significant value creation as the NDA is submitted [38][39] Conclusion - **Investment Opportunity**: XORTX Therapeutics is positioned for growth with low-risk activities planned over the next 12 to 24 months, aiming to transition from a research-focused company to a revenue-generating entity [39]

Core Insights - XORTX Therapeutics Inc. has initiated the preparation for an Investigational New Drug (IND) application for its lead program, XRx-026, aimed at treating gout, with support from Allucent, a global contract research organization [1][3] - The IND preparation will involve a thorough review of non-clinical, pharmacologic, toxicological, and regulatory progress, and is expected to be submitted in the second half of 2025 [2][3] - The FDA has outlined four critical requirements for the New Drug Application (NDA) submission for XORLO™, the proprietary formulation of oxypurinol [3][6] Company Developments - XORTX has confirmed the issuance of 73,871 common shares at US$1.54 per share, resulting in gross proceeds of approximately USD $113,547.11 during the quarter ended March 31, 2025, under its at-the-market offering [4] - The company is focused on developing innovative therapies for gout and progressive kidney disease, with three clinically advanced products in development [9] Market Context - Approximately 44 million individuals in the U.S. have elevated uric acid levels, with 9.2 million living with gout, indicating a significant market need for effective treatments [5] - The global prevalence of gout is expected to double over the next 25 years, highlighting the growing demand for new therapeutic options [5] Product Insights - The XRx-026 program is developing XORLO™, which aims to provide an alternative for patients who cannot tolerate existing xanthine oxidase inhibitors, addressing a notable gap in current treatment options [6][7] - Current standard treatments for gout have limitations, with 3-5% of patients unable to tolerate allopurinol, and febuxostat carrying cardiovascular risk warnings [6][7]

Core Insights - XORTX Therapeutics Inc. is advancing its lead gout program, XRx-026, with a focus on filing a New Drug Application (NDA) within approximately 12 months, targeting a market opportunity of USD $700 million annually [3][6][8] Group 1: Strategic Focus and Achievements - The company has prioritized the XRx-026 gout program, leveraging advancements in the proprietary XORLO™ formulation, which demonstrates increased oral bioavailability [2][4] - Significant achievements in 2025 include the validation of the XORLO™ formulation and progress in both the XRx-026 and XRx-008 programs [5][6] - Engagement with the FDA through a Type B meeting has clarified the regulatory path for NDA submission via the 505(b)(2) pathway [6] Group 2: Goals and Future Plans - Key objectives for 2025/2026 include advancing the XRx-026 program and preparing for NDA filing, alongside conducting clinical trials to support regulatory submissions [6][12] - The company plans to pursue both non-dilutive and dilutive funding, and is actively seeking partnerships with major pharmaceutical and biotech companies to accelerate commercialization [7][8] - Preparations for commercialization will include market studies and engagement with healthcare professionals to analyze pricing and branding strategies [12]

Core Viewpoint - XORTX Therapeutics Inc. is advancing its new drug application (NDA) for XORLO™, a proprietary formulation of oxypurinol aimed at treating gout, following guidance from the FDA on key steps necessary for the filing [2][4]. Company Developments - XORTX has received responses from the FDA clarifying the steps needed for the NDA submission for its XRx-026 program targeting gout [2][4]. - The company plans to finalize meeting minutes with the FDA, prepare an Investigational New Drug (IND) application, characterize pharmacokinetics, manufacture commercial supplies, and file the NDA [3][4]. - The NDA for XORLO™ is anticipated to be filed in the first half of 2026, emphasizing the company's commitment to addressing the unmet medical needs of gout patients [4]. Market Context - Approximately 44 million individuals in the US have elevated uric acid levels, with gout affecting about 9.2 million people, indicating a significant market opportunity for gout treatments [5]. - Current treatments include xanthine oxidase inhibitors (XOIs) like allopurinol, which is prescribed to around 3.3 million patients annually in North America, but some patients cannot tolerate it [6]. - XORLO™ aims to fill the gap left by existing treatments, particularly after the decline in the use of Febuxostat due to safety concerns [6].