XORTX Therapeutics Inc. has initiated preparation of an Investigational New Drug (IND) application for XORLO™, its proprietary formulation of oxypurinol designed to treat gout, marking a critical regulatory milestone for the late-stage pharmaceutical company. The Calgary-based company announced it has engaged Allucent, a global contract research organization specializing in regulatory and clinical development, to support the IND submission anticipated in the second half of 2025.
The IND preparation follows a Type B meeting held in April 2025 with the U.S. Food and Drug Administration, where the FDA provided specific guidance on the pathway toward a New Drug Application (NDA) for XORLO™. The regulatory agency outlined four critical requirements prior to NDA submission: filing of an IND, preparation of clinical and commercial drug supply with supporting stability data, a pharmacologic study characterizing absorption of XORLO™ in fasted versus fed individuals, and compilation of data from the previous steps for NDA submission.
Addressing Unmet Medical Need in Gout Treatment
The XRx-026 program represents XORTX's lead therapeutic candidate targeting a significant unmet medical need in gout management. Current standard-of-care oral xanthine oxidase inhibitors face notable limitations, with approximately 3 to 5% of patients unable to tolerate allopurinol. Additionally, febuxostat, despite previously achieving over $450 million in annual sales, now carries a boxed warning for cardiovascular risk.
"Gout continues to impose a substantial burden on patients worldwide, and the limitations of current therapies leave many individuals undertreated," stated Dr. Stephen Haworth, Chief Medical Officer of XORTX. "We believe that XORLO™ has the potential to provide a differentiated option for those who cannot tolerate or do not respond adequately to existing xanthine oxidase inhibitors."
Significant Disease Burden and Market Opportunity
The clinical need for alternative gout treatments is substantial, with approximately 44 million individuals in the United States having uric acid levels above the normal range and 9.2 million Americans living with gout. The disease is associated with severe pain, reduced quality of life, decreased physical function, increased healthcare costs, and lost economic productivity. Gout also demonstrates strong associations with metabolic syndrome, myocardial infarction, type 2 diabetes mellitus, chronic kidney disease, and premature mortality.
The global prevalence of gout is increasing, with cases expected to double over the next 25 years, highlighting the growing market opportunity for effective treatments that address current therapeutic limitations.
Comprehensive IND Preparation Strategy
The IND preparation will include a comprehensive review of non-clinical, pharmacologic, toxicological, and regulatory progress. The submission will incorporate the clinical development plan and protocol for a pharmacologic characterization study of XORLO™ in fed and fasted states, addressing one of the FDA's specific requirements for the NDA pathway.
"The initiation of IND preparation marks a pivotal step toward regulatory submission and ultimately bringing XORLO™ to individuals with gout," stated Dr. Allen Davidoff, Chief Executive Officer of XORTX. "Partnering with Allucent ensures we have the depth of regulatory expertise needed to deliver a high-quality submission and advance this important program with rigor and speed."
Company Portfolio and Strategic Focus
XORTX maintains a focused pipeline of three clinically advanced products in development: the lead XRx-026 program for gout treatment, XRx-008 program for autosomal dominant polycystic kidney disease (ADPKD), and XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections. The company is also developing XRx-225, a pre-clinical stage program for Type 2 diabetic nephropathy.
The company's therapeutic approach targets aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. Advancement of XRx-026 through NDA filing represents a strategic priority for XORTX as it works to develop medications that improve quality of life and health outcomes for individuals with gout and other important diseases.
