XORTX Therapeutics (XRTX) investor relations material

Investment highlights and market opportunity

• XRx-026 for gout targets NDA submission by H2 2027, addressing a >$0.7B peak sales opportunity due to a gap left by allopurinol alternatives with black box warnings.

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

• Over 700 patients have been treated with oxypurinol, demonstrating safety and efficacy, especially for allopurinol-intolerant gout patients.

• The team has a strong track record, including previous company exits valued at $624M and $2.2B.

Gout and ADPKD disease landscape

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

• The US market for allopurinol-intolerant gout patients is estimated at $0.5–1.0B, with 280,000 patients.

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

• ADPKD affects 160,000 in the US, with limited treatments and high annual costs; 38% have hyperuricemia.

Product pipeline and clinical development

• XRx-026 is a proprietary oxypurinol formulation with once-daily dosing, showing >90% xanthine oxidase inhibition.

• Clinical and regulatory milestones for gout include IND submission (H2 2026), PK study, CMC validation, and NDA submission (H2 2027).

• XRx-008 for ADPKD targets a $1–6B peak sales opportunity with orphan designation and accelerated approval.

• Pipeline includes additional candidates for kidney disease and anti-fibrotic therapies.