XORTX Therapeutics (XRTX) Investor Presentation summary

Investment highlights and strategic focus

• Prioritizing development of oral therapy for gout, with potential revenue within two years and NDA filing projected within 12 months.

• Large unmet medical need in gout and kidney diseases, with peak sales potential estimated at $0.4–1.5B for gout and $1–6B for ADPKD in the US.

• Advancing ADPKD program in parallel, leveraging orphan designation and accelerated FDA approval pathways.

• Board and management have a track record of successful exits, including Cynapsus Therapeutics ($624M) and Trillium Therapeutics ($2.2B).

• Three patent families protect proprietary pipeline-in-a-product technology.

Product pipeline and technology

• Lead drug oxypurinol, a xanthine oxidase inhibitor, is formulated for maximum uptake and once-daily oral dosing.

• Pipeline targets gout, ADPKD, acute kidney injury, and type 2 diabetic nephropathy, with US, EU, and other national rights secured.

• XRX-026 (gout) and XRX-008 (ADPKD) are both advancing via 505(b)(2) regulatory pathways, with XRX-026 NDA-ready and XRX-008 preparing for registration trials.

• Clinical data show >90% inhibition of xanthine oxidase and robust safety/efficacy in allopurinol-intolerant patients.

• XRX-026 demonstrates a superior safety profile compared to allopurinol, with lower incidence of skin and liver side effects.

Market opportunity and competitive landscape

• Gout prevalence has doubled in 20 years, with 6.7 million symptomatic US patients and 120,000 allopurinol-intolerant cases.

• Projected US peak sales for XRX-026 are $0.4–1.5B/year, with a launch price of $6,000–8,000/year and 7-year orphan designation.

• XRX-026 is positioned as a safer alternative to allopurinol and febuxostat, the latter of which carries a black box warning.

• ADPKD US market includes 160,000 diagnosed patients, with 38% experiencing hyperuricemia; projected peak sales for XRX-008 are $1–6B/year.

• Tolvaptan, the only FDA-approved ADPKD drug, has low usage due to tolerability issues; XRX-008 is preparing for Phase 3 trials.