XORTX Therapeutics (XRTX) Investor presentation summary

Investment highlights

• XRx-026 for gout is targeted for NDA submission by H1 2027, with demonstrated safety and efficacy in over 700 patients.

• Peak sales opportunity exceeds $0.7B due to a gap left by allopurinol alternatives with black box warnings.

• XRx-008 for ADPKD is set to begin registration trials, targeting a >$5B market with accelerated approval and orphan designation.

• Leadership team has a strong track record, including previous acquisitions valued at $624M and $2.2B.

Gout market and product positioning

• Gout prevalence has doubled in 20 years, with 7 million diagnosed in the US and 3-5% intolerant to allopurinol.

• The addressable US market for allopurinol-intolerant patients is valued at $0.5–1.0B, with a projected annual drug price of $6,000–8,000 per patient.

• Febuxostat, previously filling this market, saw sales drop after a black box warning, leaving a significant gap.

• Oxypurinol, the active metabolite of allopurinol, has shown a safer side effect profile and efficacy in allopurinol-intolerant patients.

• XRx-026 is positioned as a pre-NDA ready xanthine oxidase inhibitor, aiming to fill the current market void.

Clinical and regulatory development

• Regulatory and clinical milestones for XRx-026 include IND submission in H1 2026, PK study results in H2 2026, and NDA submission in H1 2027.

• Clinical trials have demonstrated safety and efficacy in over 700 patients, with proprietary formulation optimizing absorption and protection.

• The product targets over 90% inhibition of the xanthine oxidase enzyme, reducing uric acid levels.