XORTX Therapeutics (XRTX) Investor Presentation summary

Strategic focus and product pipeline

• Developing xanthine oxidase inhibitors targeting gout and autosomal dominant polycystic kidney disease (ADPKD), with XRx-026 for gout and XRx-008 for ADPKD as lead candidates.

• XRx-026 targets a $0.5B+ peak sales opportunity in the allopurinol-intolerant gout market, aiming for NDA submission by H1 2026.

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

• Proprietary oxypurinol formulation offers once-daily oral dosing and over 90% xanthine oxidase inhibition.

• Pipeline includes additional candidates XRx-101 and XRx-225.

Market opportunity and competitive landscape

• Over 7 million gout patients in the US, with 3-5% intolerant to allopurinol, representing 280,000 patients.

• Febuxostat, previously filling this gap, saw sales drop after a black box warning; no current drug addresses this segment.

• Projected annual drug price per patient is $6,000–$8,000, with targeted peak sales of $0.5–1.0B for XRx-026.

• ADPKD affects 160,000 diagnosed US patients; current treatments are limited and costly, with Tolvaptan at $156,000/year.

• Estimated US peak sales for ADPKD therapy are $1–6B/year, supported by orphan drug designation.

Clinical and regulatory development

• Over 850 patients have been treated with oxypurinol, demonstrating safety and efficacy, especially in allopurinol-intolerant gout.

• Oxypurinol shows lower incidence of side effects compared to allopurinol (skin: 26% vs 80%; liver: 4% vs 9%).

• XRx-026 is pre-NDA ready, with IND submission, PK study, and CMC validation planned for Q3 2025, and NDA submission in H1 2026.

• XRx-008 for ADPKD is positioned for registration trials with accelerated approval pathway.

• Multiple successful clinical trials and robust published data support the therapeutic benefit of the proprietary formulation.