XORTX Highlights Achievements of 2023 and Preparation for Registration Clinical Trial

Rhea-AI Summary

XORTX Therapeutics Inc. reports significant progress in 2023, achieving key milestones in the development of therapies for kidney diseases. The Company received U.S. Orphan Drug Designation for the XRx-008 program for ADPKD, aligning endpoints for accelerated approval. XORTX made advancements in chemistry, manufacturing, and clinical studies, aiming to conduct a registration clinical trial in 2024. Notable achievements include FDA and EMA regulatory submissions, patent advancements, and organizational enhancements. The independent commercial assessment forecasts potential worldwide peak sales exceeding $1B annually for the XRx-008 program.

Positive

• XORTX achieved substantial progress in 2023, securing U.S. Orphan Drug Designation for the XRx-008 program for ADPKD.
• The Company aligned endpoints with the FDA for accelerated approval of XORLO™, its oxypurinol formulation.
• Advancements in chemistry, manufacturing, and clinical studies set the stage for a registration clinical trial in 2024.
• XORTX submitted regulatory applications to the FDA and EMA, received patent advancements, and enhanced its organizational structure.
• An independent commercial assessment projected potential worldwide peak sales exceeding $1B annually for the XRx-008 program.

Negative

• None.

Pharmaceutical Market Analyst

The strategic advancements and regulatory milestones achieved by XORTX Therapeutics Inc. in 2023, particularly the grant of U.S. Orphan Drug Designation and discussions around accelerated approval with the FDA, suggest a positive trajectory for the company's lead program XRx-008 for ADPKD. This progress is indicative of the company's potential to capture a significant market share in the treatment of this particular kidney disease. The orphan drug status not only offers benefits such as tax credits and market exclusivity upon approval but also reflects the unmet medical need in ADPKD treatment, potentially leading to a faster time-to-market for XORLO™.

From a financial perspective, the independent commercial assessment projecting over $1 billion in peak net sales underscores the substantial revenue potential for XORTX. However, investors should consider the risks inherent in the pharmaceutical industry, such as regulatory hurdles, the possibility of unsuccessful clinical trials and the challenges of market penetration against established treatments. The oversubscribed financing round demonstrates strong investor confidence, which could be a positive sign for the company's financial stability and capacity to fund ongoing trials.

Clinical Research Analyst

The details surrounding XORTX's clinical and pre-clinical progress, particularly the production of a GMP drug substance and the successful formulation and manufacturing of XORLO™ tablets, are critical for the upcoming 'registration' clinical trial. The alignment of trial endpoints with FDA requirements for accelerated approval suggests that the company has a strategic regulatory approach, potentially reducing the time required for XORLO™ to reach the market. However, the true test of the drug's efficacy and safety will be in the results of the XRX-OXY-201 trial, which investors should watch closely.

Furthermore, the presentation of sponsored study results at the ASN conference adds to the scientific credibility of the company's approach to managing xanthine oxidase activity in PKD. This could have implications for the broader understanding of PKD progression and treatment, possibly influencing future treatment guidelines and standards of care.

Biotech Patent Analyst

The submission of a new Provisional Patent Application by XORTX is a strategic move to ensure long-term competitiveness and protect intellectual property rights. The broadened and lengthened future patent protection could provide a durable competitive edge in the market for kidney disease treatments. The ability to maintain exclusivity over their formulations and methods could be a significant factor in attracting partnership and licensing deals, which are often critical for biotech companies seeking to maximize the commercial potential of their innovations.

However, the true value of these patents will depend on the successful navigation of the clinical regulatory landscape and the eventual market acceptance of XORLO™. It is essential for investors to monitor the company's patent filings and oppositions, as these will play a important role in the company's ability to safeguard its market position and revenue streams.

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.
  

Technology and Patent Advancements

• January 3, 2023 – XORTX submitted a new Provisional Patent Application seeking broadened and lengthened future patent protection, in a patent entitled “Compositions and Methods for Diagnosis, Treatment and Prevention of Kidney Disease.
  
• November 2, 2023 – XORTX sponsored study results were presented at American Society of Nephrology (“ASN”) under Session Title: “Genetic Diseases of the Kidneys”, by Dr. Charles Edelstein of the University of Colorado. Results of these studies suggest that management of xanthine oxidase activity in PKD may be more important than previously appreciated and further that previously unrecognized factors related to diet, genetic factors or prescribed drugs that increase uric acid levels could potentially aggravate the progression of PKD.

Organizational Highlights

• In addition to substantial technological advancement, the Company continued to bolster the XORTX team with the following appointments:
  
  • June 26, 2023 - James Fairbairn was appointed Chief Financial Officer of XORTX Therapeutics. Mr. Fairbairn has more than 20 years of experience with publicly-traded companies. He is a Chartered Professional Accountant, and an Institute-certified Director.
    
  • December 31, 2023 – Patrick Treanor was appointed as a member of the XORTX Board of Directors. Patrick Treanor is a seasoned pharmaceutical industry executive with over 25 years experience. He is the current Chief Operating Officer of Pathalys Pharma, Inc., a private company specializing in advanced therapeutics for late-stage chronic kidney disease management.   Mr. Treanor earned a BS in Management from Bryant University and an MBA from Rensselaer Polytechnic Institute.
    
• On November 29, 2023, XORTX returned to NASDAQ compliance following a reverse split to increase share price to greater than $1.00.
  

XRx-008 Program Highlights – Independent Commercial Assessment

In support of ongoing pharmaceutical partnership discussions, XORTX initiated an independent commercial assessment of the XRx-008 program for ADPKD with Bluestar BioAdvisors. This evaluation included interviews with 30 Nephrologists and 10 “Payers” with Large national Plans that cover greater than 290 million lives. Outcome of this assessment suggests that the XRx-008 program for ADPKD worldwide peak net sales per year that may exceed $1B, with a total product life estimated to surpass 7 to 10 years.

2024 Corporate Objectives

• March 4, 2024 – XORTX completed an oversubscribed financing of $2.7 million.
• The Company will provide guidance, in the near future, regarding 2024 Corporate Objectives including announcements regarding clinical and regulatory submissions in support of the XRX-OXY-201 clinical “registration” trial designed to demonstrate the benefit of XORLO™ in slowing the progression of declining filtering capacity in ADPKD.

About XORTX Therapeutics Inc.

XORTX is a pharmaceutical company with two clinically advanced products in development: 1) our lead, XRx-008 program for ADPKD; and 2) 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 future health of patients. 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 U.S. Federal securities laws. 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. Except as otherwise required by law, 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 Registration Statement on Form F-1 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.

What milestone did XORTX achieve in 2023 for the XRx-008 program?

XORTX received U.S. Orphan Drug Designation for the XRx-008 program for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

What key advancements were made in chemistry, manufacturing, and clinical studies by XORTX in 2023?

XORTX made progress in producing drug substance for oxypurinol, confirming XORLO™ formulation, and producing clinical supply of tablets for trials.

What regulatory submissions did XORTX make in 2023?

XORTX submitted applications to the FDA and EMA, receiving Orphan Drug Designation for XRx-008 program for ADPKD from the FDA.

Who was appointed as Chief Financial Officer of XORTX Therapeutics in June 2023?

James Fairbairn was appointed as Chief Financial Officer of XORTX Therapeutics in June 2023.

What was the outcome of the independent commercial assessment conducted by XORTX for the XRx-008 program?

The assessment suggested potential worldwide peak sales exceeding $1B annually for the XRx-008 program.