XORTX aims to file gout drug NDA within 12 months

Executive Summary

• XORTX Therapeutics provided a strategic update on its 2025 progress and 2026 goals, highlighting significant advancements in its lead gout program, XRx-026 (Xorlo), positioning it approximately 12 months from a New Drug Application (NDA) filing with the FDA.
• The company successfully closed a $925,000 non-brokered LIFE public offering on July 21, 2025, to fund continuing initiatives, while strengthening its leadership and intellectual property portfolio.
• Key upcoming milestones include the submission of an Investigational New Drug (IND) application, the initiation of the XRX-OXY-102 clinical trial in H2 2025, and the commencement of commercialization preparations for a potential 2026 NDA filing.

Key Details

• Strategic Focus: Pivoted to accelerate the XRx-026 gout program (Xorlo), an oral oxypurinol formulation with increased bioavailability, targeting patients intolerant to existing treatments like allopurinol.
• Market Opportunity: Targeting an estimated $700 million (U.S.) per year market opportunity for the gout therapy.
• Regulatory Progress:
  • Held a Type B meeting with the FDA on March 31, 2025, to clarify the regulatory path for an NDA via the 505(b)(2) pathway.
  • Received FDA responses on April 30, 2025, confirming key steps for Xorlo approval.
  • Conducted the XRX-OXY-101 pharmacokinetics clinical trial to support NDA preparation.
• Intellectual Property:
  • Submitted a Patent Cooperation Treaty (PCT) application on December 19, 2024, for international protection.
  • Received a patent grant from the European Patent Office on April 28, 2025, for "Formulations of Xanthine Oxidase Inhibitors" for renal and related diseases.
• Financing: Closed a $925,000 (U.S.) non-brokered LIFE public offering on July 21, 2025.
• Leadership Changes:
  • Appointed Michael Bumby (biotech/pharma veteran, 14 years at Eli Lilly) to leadership on December 19, 2024.
  • Welcomed Abigail Jenkins to the Board of Directors on April 8, 2024.
• 2025/2026 Action Plan:
  • IND Submission: Prepare and submit an IND application incorporating novel formulation data, pharmacology, toxicology, and clinical results from XRX-OXY-101.
  • Clinical Trial: Initiate the XRX-OXY-102 clinical trial in the second half of 2025 to study Xorlo pharmacokinetics in fed and fasted states for population-based modeling.
  • CMC: Produce clinical drug supplies under the IND, scale up commercial supplies, and conduct validation/stability testing under GMP standards.
  • Commercialization: Conduct studies on pricing, reimbursement, and branding; commence product launch planning and brand name selection.
  • European Strategy: Engage with the EMA to define the regulatory path for EU approval, including necessary clinical studies and reimbursement conditions.
  • Funding Strategy: Pursue non-dilutive and dilutive funding, including partnerships with major pharmaceutical/biotech companies.

Notable Quotes

• "We are confident that our strategic decisions and operational advancements in 2025 position the company for a truly transformative 2026 and beyond. XORTX remains steadfast in its commitment to developing innovative therapies that make a meaningful difference for patients while delivering significant returns for shareholders." — Dr. Allen Davidoff, CEO