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 orphan designation and accelerated approval.

• The management 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 about 280,000, with a projected annual drug price of $6,000–$8,000 per patient.

• Febuxostat, previously filling this market, saw sales drop from $700M to under $30M after a black box warning.

• Oxypurinol, the active metabolite of allopurinol, has shown a significantly lower 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 gap.

Clinical and regulatory development

• The regulatory plan targets NDA filing for XRx-026 in H1 2027, with key milestones including IND submission and PK study in 2026.

• Proprietary oxypurinol formulation enables maximum drug uptake and once-daily dosing, supported by robust clinical data.

• Over 90% inhibition of xanthine oxidase enzyme achieved, with multiple successful trials in >700 patients.