XORTX Therapeutics Inc. - XRTX STOCK NEWS

XORTX Therapeutics has received Orphan Drug Designation from the FDA for oxypurinol to treat autosomal dominant polycystic kidney disease (ADPKD). This designation is a significant milestone in the development of XORLO^TM, which may slow progression of kidney damage associated with ADPKD. While not an approval for patient use yet, it positions XORTX favorably for marketing approval.

The Orphan Drug Act provides benefits like seven years of exclusive marketing rights, 25% tax credits on clinical testing costs, and regulatory assistance, all essential for the drug's development. A planned FDA meeting on May 1, 2023, will further discuss phase 3 clinical trials. XORTX has demonstrated positive results in preclinical animal models, showing potential effectiveness in treating ADPKD.

XORTX Therapeutics has submitted a Type D meeting request to the FDA for its clinical program concerning XRX-OXY-301, aiming to expedite the approval process for XORLO^TM, its innovative treatment for autosomal dominant polycystic kidney disease (ADPKD). A virtual meeting is set for May 1, 2023. The revised protocol aims to gather data necessary for accelerated approval of the drug based on total kidney volume metrics. The trial will target patients with stages 2-4 ADPKD and high uric acid levels and is expected to commence in the latter half of 2023.

XORTX Therapeutics reported significant progress in 2022, setting a robust foundation for its 2023 goals focused on advancing technology and clinical trials for treating progressive kidney disease. Key milestones included the completion of the XRX-OXY-101 clinical study and successful regulatory submissions to the FDA and Health Canada for the XRx-008 program targeting autosomal dominant polycystic kidney disease (ADPKD). In 2023, XORTX aims to initiate a Phase 3 trial for XORLO™ to gain 'Accelerated Approval' and secure Orphan Drug Designation. Financially, the company completed a $5 million public offering. XORTX seeks strategic partnerships to advance its ambitious agenda.

XORTX Therapeutics submitted an Orphan Drug Designation request to the FDA for its XRx-008 program aimed at treating Autosomal Dominant Polycystic Kidney Disease (ADPKD). This designation, intended for rare diseases affecting fewer than 200,000 individuals in the US, offers several advantages such as seven years of post-approval exclusivity and tax credits on clinical testing expenses. The XRx-008 program utilizes XORLO, a proprietary formulation of oxypurinol, which showed positive results in animal models, potentially slowing the decline of kidney function in ADPKD patients. The company will provide updates on the ODD application as they become available.

XORTX Therapeutics announced positive topline results from the XRX-OXY-101 trial, which evaluates the pharmacokinetics of XORLO™. This oral formulation of oxypurinol showed significant improvements in bioavailability and safety across 88 subjects. Key findings include:

• Over 100% increase in bioavailability compared to non-formulated oxypurinol.
• 40% higher exposure when taken with food.
• Consistent target therapeutic values achieved in multiple dosing.
• Confirmed innovations from newly granted US and EU patents.

This data will inform dosing in the upcoming Phase 3 trial for patients with autosomal dominant polycystic kidney disease (ADPKD).

XORTX Therapeutics (NASDAQ: XRTX), a late-stage pharmaceutical company, has submitted a Patent Cooperation Treaty (PCT) application for a patent titled “Compositions and Methods for Diagnosis, Treatment and Prevention of Kidney Disease”. This application stems from findings indicating a diagnostic and therapeutic opportunity related to polycystic kidney disease (PKD). The patent proposes methods to identify patients at risk of accelerated kidney disease progression through aberrant purine metabolism and hyperuricemia, aiming to personalize treatment approaches.

XORTX Therapeutics has completed dosing for its XRX-OXY-101 pharmacokinetics bridging study, part of the development for its proprietary oral oxypurinol formulation, XORLO^TM. This study aims to assess the pharmacokinetics in healthy individuals and informs the upcoming phase 3 trial for treating autosomal dominant polycystic kidney disease (ADPKD). The study comprises four parts, with topline results from parts 3 and 4 expected soon. Company CEO Allen Davidoff highlighted that results will guide dosing and support FDA submissions. Consolidated results are anticipated in January 2023.

XORTX Therapeutics has announced promising results from an independent study indicating that its proprietary formulation, XORLO™, effectively inhibits kidney expansion in a mouse model of Autosomal Dominant Polycystic Kidney Disease (ADPKD). Conducted at the University of Colorado, the study supports prior data presented at the American Society of Nephrology meeting. Key findings include a significant correlation between increased uric acid levels and kidney volume, with XORLO™ mitigating these harmful effects. CEO Allen Davidoff highlighted the relevance of these findings for future clinical trials.

XORTX Therapeutics (NASDAQ: XRTX) has announced the successful enrollment of participants in the XRX-OXY-101 Bridging Pharmacokinetics Study, aimed at optimizing the formulation and dosage of oxypurinol for treating ADPKD. The trial's completion and topline results are expected soon, which will inform the dosing for the upcoming Phase 3 XRX-OXY-301 trial. Additionally, the company granted 70,000 stock options to certain officers at an exercise price of $1.38 for five years, indicating ongoing engagement with its leadership team.