XORTX to prepare new drug filing for XRx-026

Executive Summary

• XORTX Therapeutics has initiated the preparation of an Investigational New Drug (IND) application for its lead gout program, XRx-026 (XORLO), with a targeted submission in the second half of 2025.
• The company engaged Allucent, a global contract research organization, to support regulatory and clinical development efforts.
• Concurrently, the company reported the issuance of 73,871 common shares under its At-The-Market (ATM) offering, generating approximately $113,547 in gross proceeds during the quarter ended March 31, 2025.

Key Details

• IND Preparation: XORTX initiated IND preparation for XRx-026, focusing on a comprehensive review of non-clinical, pharmacologic, toxicological, and regulatory progress.
• Clinical Plan: The IND will incorporate a clinical development plan and protocol for a pharmacologic characterization study of XORLO (proprietary oxypurinol formulation) in both fed and fasted states.
• Regulatory Timeline: The company anticipates submitting the IND in the second half of 2025.
• FDA Guidance: The IND preparation follows a Type B meeting held in April 2025, where the FDA outlined four critical requirements for a New Drug Application (NDA):
  1. Filing of an IND.
  2. Preparation of clinical and commercial drug supply with supporting stability data.
  3. A pharmacologic study characterizing absorption of XORLO in fasted versus fed individuals.
  4. Compilation of data from steps 2 and 3, followed by NDA submission.
• Financing Activity: Issuance of 73,871 common shares under the ATM offering announced on November 30, 2023.
• Share Price: $1.54 (U.S.) / $2.21 (Canadian) per share.
• Financial Proceeds:
  • Gross Proceeds: $113,547.11 (U.S.) / $163,178.55 (Canadian).
  • Net Proceeds: $109,665.93 (U.S.) / $157,600.91 (Canadian).
• Regulatory Filing Context: The ATM offering is made pursuant to a registration statement on Form F-3 (File No. 333-269429), declared effective February 3, 2023, with a prospectus supplement dated November 29, 2023.

Notable Quotes

• Dr. Allen Davidoff, CEO: "The initiation of IND preparation marks a pivotal step toward regulatory submission and ultimately bringing XORLO to individuals with gout... Partnering with Allucent ensures we have the depth of regulatory expertise needed to deliver a high-quality submission and advance this important program with rigour and speed."
• Dr. Stephen Haworth, CMO: "Gout continues to impose a substantial burden on patients worldwide, and the limitations of current therapies leave many individuals undertreated. 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."