XORTX Receives No Objection Letter from Health Canada

Rhea-AI Summary

XORTX Therapeutics Inc. announced the receipt of a no objection letter from Health Canada for its XRX-OXY-101 clinical bridging pharmacokinetics study. The study aims to identify the optimal formulation for oxypurinol and assess food effect, safety, and pharmacokinetics. This information is critical for advancing toward a phase 3 trial in Autosomal Dominant Polycystic Kidney Disease (ADPKD). Dr. Allen Davidoff emphasized that this milestone accelerates the development of the XRx-008 program towards potential marketing approvals for treating progressive kidney disease.

Positive

• Early receipt of no objection letter from Health Canada for XRX-OXY-101 study.
• Study designed to optimize oxypurinol formulations which may enhance treatment outcomes.
• Important step towards phase 3 trial and potential marketing approval for ADPKD treatment.

Negative

• None.

Bridging Pharmacokinetics Study Autosomal Dominant Polycystic Kidney Disease – XRx-008 Program

CALGARY, Alberta, April 12, 2022 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce receipt of a no objection letter (NOL) from Health Canada regarding the Company’s upcoming XRX-OXY-101 clinical bridging pharmacokinetics study.

XRX-OXY-101 Bridging Pharmacokinetics Study. XRX-OXY-101 has been designed with three important objectives: 1) to determine which of XORTX’s novel formulations results in the best circulating oxypurinol concentrations; 2) to determine the effect of food on the bioavailability of this formulation; and 3) to determine the safety and pharmacokinetics of multiple doses of this selected formulation. Knowledge gained during the conduct of this trial will provide guidance regarding the future oral dosing of oxypurinol formulations in support of the Company’s planned phase 3 registration trial in Autosomal Dominant Polycystic Kidney Disease (“ADPKD”). Additionally, this study will provide data to support future NDA (New Drug Application) marketing submissions to the United States Food and Drug Administration and the European Medicines Agency.

Dr. Allen Davidoff, CEO of XORTX stated, “We are pleased to receive the NOL from Health Canada earlier than expected. This important milestone provides regulatory approval for dosing of subjects with our novel proprietary product candidates. The XRX-OXY-101 study is designed to characterize the enhanced bioavailability of our novel proprietary xanthine oxidase formulation and is an important step forward in the development of XRx-008 program through the US FDA 505(b)2 development, our future phase 3 registration trial and eventually toward marketing approval for the treatment of individuals with progressive kidney disease due to ADPKD.”

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 and dietary restrictions.⁴ New therapies to slow decline of kidney function in ADPKD are needed.

About XORTX Therapeutics Inc.

XORTX is a pharmaceutical company with two clinically advanced products in development: 1) our lead program in XRx-008 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.

About Clinical Trial Applications

https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/applications-submissions/guidance-documents/clinical-trials/applications.html

For further 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

The TSX Venture Exchange and Nasdaq have neither approved nor disapproved the contents of this news release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein.

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

Forward Looking Statements

This press release may contain express or implied forward-looking statements pursuant to Canadian and U.S. Federal securities laws. These forward-looking statements and their implications are based on the current reasonable 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 such forward-looking statements. Except as otherwise required by law, XORTX undertakes no obligation to publicly release any revisions or updates 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 in the Company’s most recently filed Annual Information Form and the Management Discussion and Analysis for its most recent financial reporting period filed on the Company’s SEDAR profile (www.sedar.com) and under the heading “Risk Factors” in XORTX’s Registration Statement on Form F-1 filed with the Securities and Exchange Commission (“SEC”) available on the SEC's website, www.sec.gov.

FAQ

What is the purpose of the XRX-OXY-101 study by XORTX?

The XRX-OXY-101 study aims to determine the optimal formulation of oxypurinol, assess food effects on bioavailability, and evaluate safety and pharmacokinetics for treating ADPKD.

When did XORTX receive the no objection letter from Health Canada?

XORTX received the no objection letter on April 12, 2022.

What is Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

ADPKD is a rare disease affecting over 10 million individuals worldwide, characterized by the expansion of cysts in the kidneys leading to loss of kidney function.

How does XORTX plan to advance the XRx-008 program?

XORTX plans to advance the XRx-008 program through a phase 3 registration trial and aims for future marketing approvals.

What are the main objectives of the XRX-OXY-101 study?

The main objectives are to identify the best formulation for circulating oxypurinol concentrations, assess the effect of food on bioavailability, and determine the safety and pharmacokinetics of the selected formulation.