XORTX Therapeutics (NasdaqCM:XRTX) Update / Briefing Transcript

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]