XORTX Submits a New Patent for the Treatment of Chronic Kidney Disease

Rhea-AI Summary

XORTX Therapeutics Inc. (XRTX) announces the submission of a new patent for the treatment of chronic kidney disease, focusing on innovative therapies to treat progressive kidney disease. The patent outlines new formulations and methods for safer and more effective use of xanthine oxidase inhibitors (XOI) in the setting of CKD, including autosomal dominant polycystic kidney disease, diabetic nephropathy, IgA nephropathy, lupus nephritis, and focal segmental glomerulosclerosis. Positive topline results from the XRX-OXY-101 bridging pharmacokinetic clinical study reported substantial increases in the bioavailability of oxypurinol with the XORLO™ formulation platform, increased dose proportionality compared to non-formulated oxypurinol, and achieved therapeutic target values. These results will facilitate precise dosing recommendations for upcoming registration trials in individuals with progressing kidney disease.

Positive

• Submission of a new patent for the treatment of chronic kidney disease
• Positive topline results from the XRX-OXY-101 bridging pharmacokinetic clinical study
• Substantial increase in the bioavailability of oxypurinol with the XORLO™ formulation platform
• Increased dose proportionality compared to non-formulated oxypurinol
• Achieved therapeutic target values

Negative

• None.

Pharmaceutical Patent Expert

The recent patent submission by XORTX Therapeutics for novel formulations and methods involving xanthine oxidase inhibitors (XOI) signifies a strategic move to secure intellectual property rights that could potentially give the company a competitive edge in the CKD treatment market. The protection of these discoveries is crucial for the company to maintain exclusivity and justify the investment in research and development. The patent's focus on varied degrees of kidney function indicates a tailored approach to drug delivery, which could lead to more personalized treatment options for patients with CKD and related diseases.

Given the specificity of the patent to address diseases like ADPKD, DN and others, there is a clear intent to address unmet needs within these patient populations. The patent could also pave the way for partnerships or licensing deals, which would be financially beneficial for XORTX. However, the patent's approval and the subsequent commercialization of these formulations will be critical factors in realizing these potential benefits.

Clinical Research Analyst

The positive results from the XRX-OXY-101 bridging pharmacokinetic clinical study are indicative of the potential effectiveness of XORTX's proprietary formulation, XORLO™. The improved bioavailability and dose proportionality of oxypurinol in this formulation could translate to better patient outcomes in CKD treatment. The study's findings on safety and tolerability are also encouraging for future registration trials, as these aspects are critical in the advancement of any pharmaceutical product.

From a clinical perspective, the enhanced early oral absorption and increased circulating concentrations of oxypurinol are significant because they suggest that XORLO™ could more effectively inhibit uric acid production, which is a key factor in the progression of CKD. The data from this study will be crucial in determining precise dosing recommendations, which is a fundamental step in the clinical development process. The success of upcoming registration trials will heavily depend on the robustness of this data and the ability to demonstrate clear clinical benefits in a diverse patient population.

Investment Analyst

The announcement by XORTX Therapeutics regarding their new patent submission holds potential implications for the company's market valuation. Investors typically respond positively to patent filings as they can lead to product exclusivity and long-term revenue generation. The specificity of the patent in targeting multiple forms of CKD could open up several market segments for XORTX, assuming successful commercialization.

However, investors should also be aware of the risks involved. The pharmaceutical industry is highly regulated and the path from patent submission to product launch is long and fraught with potential setbacks, including regulatory hurdles, competition from other pharmaceutical companies and the challenges of market acceptance. The long-term financial impact on the company will depend on the patent's approval, the success of subsequent clinical trials and the ability to effectively market and distribute the new formulations.

CALGARY, Alberta, Jan. 03, 2024 (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 submission of a new patent for the treatment of chronic kidney disease (“CKD”). This patent is designed to protect new discoveries and strategies for the treatment of individuals with varied degrees of kidney function in the setting of CKD. Importantly, this patent entitled “Oral and Sublingual Formulations of Xanthine Oxidase Inhibitors and Methods of Treating Disease” outlines new formulations and methods for safer and more effective the use of xanthine oxidase inhibitors (XOI) in the setting of CKD in particular autosomal dominant polycystic kidney disease (ADPKD), diabetic nephropathy (DN), IgA nephropathy, lupus nephritis and focal segmental glomerulosclerosis.

The positive topline results from the XRX-OXY-101 bridging pharmacokinetic clinical study reported in Q1 2023 (the “Study”) characterized the pharmacokinetics of the Company’s proprietary formulation of oral oxypurinol, XORLO™. Results from the Study showed that XORLO™ was well tolerated by the 88 subjects who received the drug. There were no safety concerns during the testing of drug across the various dosing regimens used. Overall results were positive and showed: i) a substantial increase in the bioavailability of oxypurinol with the XORLO™ formulation platform; (ii) a substantially increased dose proportionality compared to non-formulated oxypurinol; (iii) a multiple dosing regimen that achieved therapeutic target values. In simple terms, substantially increased early oral absorption of XORLO™, and increased circulating concentrations of oxypurinol necessary to inhibit production of uric acid across the desired therapeutic range and thereby slow down the advancements of CKD. Each of these results will provide key data to facilitate precise dosing recommendations for upcoming registration trials in individuals with progressing kidney disease due to ADPKD as well as other causes of CKD.

Dr. Allen Davidoff, CEO of XORTX, commented, “The Bridging Pharmacokinetic Study reported this year provided a wealth of clinical data regarding the potential substantive benefit of the novel formulations of the xanthine inhibitor class of drugs. Analysis of this data set, the use of in silico based pharmacokinetic modeling of data from the XRX-OXY-101 clinical trial, and further innovation, resulted in a deeper understanding of how to address the challenges of dosing in progressing kidney disease. This patent application is intended to claim new opportunities to enhance how the xanthine oxidase inhibitor class of drugs may be dosed in the future. Importantly, how to further improve the safe and effective administration of this class of drugs, including oxypurinol.”

About the XRx-008 program

Oxypurinol is a purine based XOI with important pharmacologic characteristics ideal for administration to individuals with ADPKD. Key pharmacologic attributes include:

1/ the ability to act in the circulation, kidney and cardiovascular tissue and inhibit the production of uric acid and so attenuate the mechanism of injury and accelerating effect of XO on progressing diseases.

2/ XORTX’s proprietary formulation of oxypurinol, XORLO™, provides substantially increased absorption of oxypurinol. Metabolism of oxypurinol is minimal and it is eliminated by the kidneys unchanged. This approach provides an effective, well tolerated drug with an extensive clinical safety experience suggesting the Company’s XRx-008 program has the capacity to provide superior XOI to slow the accelerating decline kidney function in patients ADPKD with coexistent hyperuricemia.

About ADPKD

ADPKD is a rare disease that affects more that 10 million individuals worldwide.^1,2 ADPKD is typically diagnosed based upon expansion of fluid-filled cysts in the kidneys. Over time, the increasing number and size of cysts can contribute to structural and functional changes to kidneys and is frequently accompanied by chronic pain which is a common problem for patients with ADPKD.³ Expansion of cysts is thought to compress healthy functioning tissue surrounding the cysts and contribute to further loss of kidney function, fibrosis, impaired nutrient exchange and impaired kidney function, accompanied later by end-stage renal disease.¹ For individuals with progressing ADPKD, treatment recommendations include anti-hypertensive treatment, dietary restrictions, and, for a limited percentage of suitable patients, pharmacotherapy.⁴ New, more broadly applicable therapies to effectively slow decline of kidney function in ADPKD are needed.

References:

1. Wiley C., Kamat S., Stelhorn R., Blais J., Analysis of nationwide date to determine the incidence and diagnosis of autosomal dominant polycystic kidney disease in the USA, Kidney Disease, 5(2): 107-117, 2019
2. Bergmann C., Guay-Woodford L.M., Harris P.C., Horie S., Peters D.J., Torres V.E., Polycystic Kidney Disease, Nat Rev Dis Primers. 4(1): 50, 2018
3. https://pkdcure.org/living-with-pkd/chronic-pain-management/
4. Gimpel C., Bermann C., Bockenhauer D., et al., International consensus statement of the diagnosis and management of autosomal dominant polycystic kidney disease in children and young people, Nat Rev Nephrol 15(11):713-726, 2019

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 Respiratory Viral 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 with kidney disease. 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 TSXV 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, including those relating to future sales of Shares under the ATM Offering, the offering price therefor and the use of proceeds thereof. 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 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.

What is the focus of the new patent submitted by XORTX Therapeutics Inc. (XRTX)?

The new patent focuses on developing innovative therapies to treat progressive kidney disease, specifically chronic kidney disease (CKD).

What are the positive topline results from the XRX-OXY-101 bridging pharmacokinetic clinical study?

The positive topline results reported substantial increases in the bioavailability of oxypurinol with the XORLO™ formulation platform, increased dose proportionality compared to non-formulated oxypurinol, and achieved therapeutic target values.

What are the specific diseases targeted by the new patent for the treatment of CKD?

The new patent targets autosomal dominant polycystic kidney disease, diabetic nephropathy, IgA nephropathy, lupus nephritis, and focal segmental glomerulosclerosis.

Who is the CEO of XORTX Therapeutics Inc. (XRTX)?

Dr. Allen Davidoff is the CEO of XORTX Therapeutics Inc.