XORTX Therapeutics (XRTX) Study Update summary

Program focus and market opportunity

• Developing therapies for gout and autosomal dominant polycystic kidney disease (ADPKD), with gout as the primary focus and ADPKD as a secondary target.

• Preparing to file a New Drug Application (NDA) for oxypurinol (XRx-026) targeting allopurinol-intolerant gout patients, with submission expected in the first half of 2026.

• The addressable U.S. market for allopurinol-intolerant gout patients is estimated at $700 million to $1 billion annually, with a projected annual drug price per patient of $6,000–$8,000.

• ADPKD program targets a $1–6 billion market, with potential for orphan drug adoption and accelerated approval.

• Both programs are positioned for large partnership deals, global commercialization, and long-term growth supported by additional pipeline candidates.

Clinical and regulatory progress

• Oxypurinol has a longstanding safety record, tested in over 750 patients, and is effective for 70% of allopurinol-intolerant individuals.

• FDA requested a small medical study with the commercial tablet, one year of stability data, and pharmacokinetic studies for NDA approval.

• NDA filing for gout is expected within 12–14 months or in the first half of 2026, with a 9–12 month review period.

• ADPKD program requires only a single small registration trial for approval and is advancing toward registration trials.

• The clinical program leverages robust published data and a patented, scalable oral formulation.

Safety and efficacy data

• Over 750 allopurinol-intolerant patients have been successfully treated with oxypurinol, showing a favorable safety profile compared to allopurinol.

• Oxypurinol demonstrated lower incidences of skin and liver side effects and provided symptom relief for 70% of allopurinol-intolerant patients.

• The proprietary formulation enables once-daily oral dosing and over 90% inhibition of the xanthine oxidase enzyme.

• Multiple successful trials support the therapeutic benefit of oxypurinol in gout and other indications.

• Published data reinforce oxypurinol as the most effective uric acid lowering agent available.