XORTX Highlights Achievements of 2023 and Preparation for Registration Clinical Trial

CALGARY, Alberta, March 19, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to provide a summary of Company’s achievements in 2023 and objectives planned for 2024.

Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals. Key milestones include: 1/ the grant of U.S. Orphan Drug Designation for the XRx-008 program for Autosomal Dominant Polycystic Kidney Disease (“ADPKD”) that is being developed under the US FDA 505(b)2 rules further de-risking this program; 2/ ongoing discussions with the US FDA have aligned our endpoints and other Phase 3 clinical trial elements to make XORLO the Company’s proprietary oxypurinol formulation, eligible for accelerated approval. These key advances on the XRx-008 program during 2023 were made possible by the exceptional efforts of our Board of Directors, employees, consultants, and vendors. We believe the goals set for 2024 will advance our lead program XRx-008 for ADPKD ever closer to conducting a registration clinical trial leading to marketing approval and value creation for XORTX and its shareholders.”

In 2023, the Company made steady progress advancing its strategic plan in key areas, including chemistry, formulation, manufacturing, and non-clinical studies using XORLO to attenuate polycystic kidney disease (“PKD”) progression in animal models, and topline results from the XRX-OXY-101 bridging clinical study of XORLO. Each of these milestones permit the next step in the Company’s clinical development plan, being a “registration” clinical trial – XRX-OXY-201 in pursuit of accelerated approval and support of the Company’s lead program XRx-008 program for ADPKD.

Synopsis of 2023 Achievements

Chemistry and Manufacturing, Clinical and Pre-Clinical Highlights – Produced drug substance for oxypurinol production and produced GMP drug substance; confirmed XORLO formulation, produced enhanced bioavailability and produced clinical supply of tablets for clinical trials.

Regulatory Submissions to US Food and Drug Administration (“FDA”) and European Medicines Agency (“EMA”) Supporting Clinical Trial Conduct

• April 21, 2023 – FDA granted Orphan Drug Designation for XRx-008 program for ADKPD, following review of February 1^st submission of a comprehensive scientific review package within an Orphan Drug Designation application for XRx-008 for treatment of progressing kidney disease due to ADPKD with the US FDA.
• May 5, 2023 – FDA confirmed the eligibility of XORLO for Accelerated Approval following review of March 14^th submission of type D meeting request with US FDA to discuss the clinical development plan (XRX-OXY-201 clinical trial design).
• August 29, 2023 – XORTX submitted an Orphan Drug Designation application to the EMA for the treatment of ADPKD. Following discussion and guidance from EMA, XORTX will expand data package and resubmit to gain EMA Orphan Drug Designation.