XORTX Adds Late Stage Gout Program to Pipeline
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.
XORTX Therapeutics (NASDAQ: XRTX) ha annunciato il lancio di un nuovo programma avanzato, XRx-026, focalizzato sul trattamento delle persone affette da gotta che sono intolleranti all'allopurinolo. L'azienda prevede di avviare discussioni con la FDA nella prima metà del 2025 riguardo una Nuova Domanda di Medicamento (NDA) per questo programma.
L'azienda richiederà la designazione di farmaco orfano (ODD) per XRx-026, sottolineando che l'ossipurinol aveva precedentemente ricevuto ODD per la gotta intollerante all'allopurinolo. Gli studi clinici hanno dimostrato che l'ossipurinol è sicuro ed efficace nel trattare le persone con gotta che non possono tollerare l'allopurinolo, rispondendo a un'importante esigenza medica non soddisfatta per pazienti e medici.
XORTX Therapeutics (NASDAQ: XRTX) ha anunciado el lanzamiento de un nuevo programa en fase avanzada, XRx-026, centrado en tratar a personas con gota que son intolerantes a la alopurinol. La compañía planea iniciar conversaciones con la FDA en la primera mitad de 2025 sobre una Nueva Solicitud de Medicamento (NDA) para este programa.
La empresa solicitará la designación de medicamento huérfano (ODD) para XRx-026, señalando que el oxipurínol había recibido anteriormente ODD para la gota intolerante a la alopurinol. Los estudios clínicos han demostrado que el oxipurínol es seguro y efectivo para tratar a las personas con gota que no pueden tolerar el alopurinol, abordando una importante necesidad médica no satisfecha para pacientes y médicos.
XORTX Therapeutics (NASDAQ: XRTX)는 알로퓨리놀에 불내성을 가진 통풍 환자를 치료하기 위한 새로운 후기 단계 프로그램인 XRx-026의 출범을 발표했습니다. 회사는 2025년 상반기 중에 이 프로그램에 대한 신약 신청(NDA)에 대해 FDA와 논의할 계획입니다.
회사는 XRx-026에 대해 FDA의 희귀의약품 지정(ODD)을 요청할 예정이며, 이전에 알로퓨리놀에 불내성이 있는 통풍에 대해 옥시퓨리놀에게 ODD가 부여된 바 있다고 언급했습니다. 임상 연구들은 옥시퓨리놀이 알로퓨리놀을 견딜 수 없는 통풍 환자를 치료하는 데 안전하고 효과적임을 입증했으며, 이는 환자와 의사에게 중요한 의학적 요구를 충족시킵니다.
XORTX Therapeutics (NASDAQ: XRTX) a annoncé le lancement d'un nouveau programme en phase avancée, XRx-026, axé sur le traitement des personnes atteintes de goutte qui sont intolérantes à l'allopurinol. La société prévoit d'engager des discussions avec la FDA au cours de la première moitié de 2025 concernant une Demande de Nouveau Médicament (NDA) pour ce programme.
La société demandera à la FDA la désignation de médicament orphelin (ODD) pour XRx-026, notant que l'oxypurinol avait précédemment reçu l'ODD pour la goutte intolérante à l'allopurinol. Des études cliniques ont démontré que l'oxypurinol est sûr et efficace pour traiter les personnes atteintes de goutte qui ne peuvent pas tolérer l'allopurinol, répondant à un besoin médical important non satisfait pour les patients et les médecins.
XORTX Therapeutics (NASDAQ: XRTX) hat die Einführung eines neuen Spätphasensprogramms, XRx-026, angekündigt, das sich auf die Behandlung von Personen mit Gicht konzentriert, die allergisch gegen Allopurinol sind. Das Unternehmen plant, in der ersten Hälfte des Jahres 2025 Gespräche mit der FDA über einen Antrag auf Marktzulassung (NDA) für dieses Programm zu führen.
Das Unternehmen wird die FDA um die Orphan Drug Designation (ODD) für XRx-026 bitten und darauf hinweisen, dass Oxypurinol zuvor für Gicht, die intolerant gegenüber Allopurinol ist, ODD erhalten hat. Klinische Studien haben gezeigt, dass Oxypurinol sicher und wirksam in der Behandlung von Personen mit Gicht ist, die Allopurinol nicht vertragen können, was ein wichtiges unbefriedigtes medizinisches Bedürfnis für Patienten und Ärzte anspricht.
- 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
- 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?
