XORTX Adds Late Stage Gout Program to Pipeline
Rhea-AI Summary
XORTX Therapeutics (NASDAQ: XRTX) has announced the launch of a new late-stage program, XRx-026, focused on treating individuals with gout who are intolerant to allopurinol. The company plans to initiate discussions with the FDA in the first half of 2025 regarding a New Drug Application (NDA) for this program.
The company will seek FDA orphan drug designation (ODD) for XRx-026, noting that oxypurinol was previously granted ODD for allopurinol intolerant gout. Clinical studies have demonstrated that oxypurinol is safe and effective in treating individuals with gout who cannot tolerate allopurinol, addressing an important unmet medical need for patients and physicians.
Positive
- Launch of new late-stage drug development program XRx-026
- Previous orphan drug designation for oxypurinol in allopurinol intolerant gout
- Demonstrated safety and efficacy in clinical studies for target population
Negative
- None.
Insights
The clinical significance lies in addressing a critical gap in gout treatment for patients who cannot tolerate the first-line therapy allopurinol. Oxypurinol, the active compound, has demonstrated both safety and efficacy in previous clinical studies. The prior Orphan Drug Designation (ODD) for oxypurinol in this indication provides regulatory advantages including potential market exclusivity and tax incentives.
The company's timing for FDA discussions in H1 2025 regarding NDA filing suggests confidence in their data package. For a micro-cap company with a market cap of just
A simplified explanation: Think of this like developing a specialized key (XRx-026) for people who are allergic to the standard key (allopurinol) used to unlock and solve their gout problem. Having previous regulatory recognition (ODD) makes the path to approval potentially smoother.
The regulatory strategy here is particularly noteworthy. XORTX's approach of leveraging a previous Orphan Drug Designation while pursuing a new NDA demonstrates sophisticated regulatory navigation. The planned FDA consultation in H1 2025 suggests they're positioning for a 505(b)(2) pathway, which could expedite approval by utilizing existing safety and efficacy data for oxypurinol.
The company's dual-track approach - maintaining development of XORLOTM while advancing XRx-026 - indicates efficient resource allocation and regulatory planning. The potential for a new ODD designation could provide 7 years of market exclusivity upon approval, creating significant market protection for a micro-cap company.
In simpler terms: They're taking a smart shortcut by using previously proven data about the drug's safety, potentially saving years of development time and millions in costs. The special designation they're seeking is like getting a 'fast pass' at an amusement park - it could help them get to market faster and with certain advantages.
XORTX to focus on late stage allopurinol intolerant gout program; discussion with FDA planned for first half 2025 regarding NDA filing
CALGARY, Alberta, Jan. 06, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces the launch of a new late stage program to treat gout. The new drug development program - XRx-026 - will focus on the treatment of individuals who have gout and are intolerant to allopurinol.
With the clinical development of XORLOTM 1 having advanced sufficiently, including completion of a pivotal program, XORTX is initiating discussions for the XRx-026 program with the US Food and Drug Administration (“FDA”) regarding preparation of a New Drug Application (“NDA”). Pending FDA feedback, the Company will also seek FDA orphan drug designation (“ODD”) and NDA marketing approval for the XRx-026 program. Previously, oxypurinol was granted ODD for allopurinol intolerant gout.
Dr. Allen Davidoff commented, “Oxypurinol has been demonstrated to be safe and effective in clinical studies focused on the treatment of individuals with gout who are intolerant to allopurinol. Intolerance to allopurinol remains an important issue for many patients and physicians and the XRx-026 program has demonstrated the potential to address this unmet medical need. XORTX will consult with the FDA during the first half of 2025, regarding requirements to file a NDA.”
About Hyperuricemia, Gout and Health Consequences
The breakdown of nucleotides in the blood occurs through purine metabolism and results in the formation of uric acid by the xanthine oxidase enzyme. However, chronically high blood uric acid concentrations (hyperuricemia) have been associated with health consequences including gout, kidney stones, diabetes, cardiovascular disease, and renal failure. Worldwide, approximately
Addressable Gout Market Opportunity
In North America, approximately 3.5 million people suffer from gout due to elevated uric acid levels in blood. The therapeutic options to lower uric acid levels include three major classes of drugs: (i) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; (ii) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and (iii) oral xanthine oxidase inhibitors (“XOIs”) that are used to inhibit the production of uric acid. XOIs are the preferred first-line treatment for gout. Allopurinol is the most commonly prescribed XOI, with approximately 3 million prescriptions written per year in North America, however 3 to
About XORTX Therapeutics Inc.
XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our lead, XRx-008 program for ADPKD; 2) our XRx-026 program for the treatment of allopurinol intolerant gout; and 3) our secondary program in XRx-101 for acute kidney and other acute organ injury associated with Coronavirus / COVID-19 infection. In addition, XRx-225 is a pre-clinical stage program for Type 2 Diabetic Nephropathy. XORTX is working to advance its clinical development stage products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, we are dedicated to developing medications to improve the quality of life and health of kidney disease patients and individuals with gout. Additional information on XORTX is available at www.xortx.com.
For more information, please contact:
| Allen Davidoff, CEO | Nick Rigopulos, Director of Communications |
| adavidoff@xortx.com or +1 403 455 7727 | nick@alpineequityadv.com or +1 617 901 0785 |
Neither the TSX Venture Exchange nor Nasdaq has approved or disapproved the contents of this news release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein.
Forward Looking Statements
This press release contains express or implied forward-looking statements pursuant to applicable securities laws. These forward-looking statements include, but are not limited to, the Company's beliefs, plans, goals, objectives, expectations, assumptions, estimates, intentions, future performance, other statements that are not historical facts and statements identified by words such as "expects", "anticipates", "intends", "plans", "believes", "seeks", "estimates" or words of similar meaning. These forward-looking statements and their implications are based on the current expectations of the management of XORTX only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks, uncertainties, and other factors include, but are not limited to, our ability to obtain additional financing; the accuracy of our estimates regarding expenses, future revenues and capital requirements; the success and timing of our preclinical studies and clinical trials; the performance of third-party manufacturers and contract research organizations; our plans to develop and commercialize our product candidates; our plans to advance research in other kidney disease applications; and, our ability to obtain and maintain intellectual property protection for our product candidates. Except as otherwise required by applicable law and stock exchange rules, XORTX undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting XORTX is contained under the heading “Risk Factors” in XORTX’s Annual Report on Form 20-F filed with the SEC, which is available on the SEC's website, www.sec.gov (including any documents forming a part thereof or incorporated by reference therein), as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada, which are available on www.sedarplus.ca.
1 XORLOTM is XORTX’s proprietary formulation of oxypurinol, that has granted US and EU patents.
2 Source: Takeda Pharmaceutical Company 2018 Annual Report.
FAQ
What is XORTX's new XRx-026 program targeting?
When will XORTX (XRTX) discuss the NDA filing with FDA for XRx-026?
Has XRTX's oxypurinol previously received FDA orphan drug designation?
What clinical evidence supports XRTX's XRx-026 program?
