Xorlo (oxypurinol) / Xortx Therap - LARVOL DELTA

XORTX Announces Update for Discussion with the FDA (GlobeNewswire) - "XORTX Therapeutics Inc...is pleased to provide an update regarding communications with the US Food and Drug Administration (the 'FDA'). At the request of the FDA a type B meeting package will be provided by the Company during the next week, and accompanying FDA communications are expected by April 26, 2025. The Company has prepared a broad Type B meeting review at the request of the FDA, including review of chemistry, manufacturing, pharmacology, toxicology and clinical evidence regarding the Company’s XRx-026 program for the treatment of gout. Drug Development of XORLO, the Company’s proprietary drug formulation of oxypurinol, has advanced substantially to a state where a Type B meeting and discussion with the FDA to confirm the developmental state of each element of the program is warranted."

FDA event • Gout

XORTX Commences Gout Program NDA Discussions with the FDA (GlobeNewswire) - "XORTX Therapeutics Inc...announces that it has submitted a Type C meeting request with the US Food and Drug Administration (the 'FDA') regarding the Company’s XRx-026 program for the treatment of gout. Development of XORLO, the Company’s proprietary drug formulation of oxypurinol, has advanced to the point where a Type C meeting and discussion with the FDA is warranted. The purpose of this meeting is to review the XRx-026 program and its readiness for submission of a New Drug Application ('NDA') to gain marketing approval for XORLO in the US using the FDA’s 505(b)2 development pathway."

FDA event • Gout

Xanthine Oxidase in Rats, Mice, and Humans with Polycystic Kidney Disease (KIDNEY WEEK 2024) - "In summary, in rodents with PKD, there was no correlation between increased serum or kidney XO activity and cyst index. In PKD patients there was a trend towards higher serum XO and lower GFR. In conclusion, serum XO needs to be measured in a larger group of patients from the HALT-PKD study."

Preclinical • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • PKD1 • PRKD1

Raising serum uric acid with a uricase inhibitor worsens PKD in rat and mouse models. (PubMed, Am J Physiol Renal Physiol) - "In summary, increasing serum uric acid by inhibiting uricase with oxonic acid results in an increase in kidney weight and cyst index in PCK rats and Pkd1RC/RC mice. The effect is independent of inflammasome activation or crystal deposition in the kidney."

Journal • Preclinical • Genetic Disorders • Gout • Hepatology • Inflammatory Arthritis • Nephrology • Polycystic Kidney Disease • Renal Disease • Rheumatology • PKD1 • PKHD1 • PRKD1

FDA Confirms Eligibility of XORLO for Accelerated Approval (GlobeNewswire) - "XORTX Therapeutics Inc...announce completion of a positive and constructive Type D meeting with the U.S. Food and Drug Administration ('FDA') which resulted in the identification of additional clinical endpoints potentially available for accelerated approval and further understanding of the FDA expectations for the accelerated approval of XORLO™ for the treatment of autosomal dominant polycystic kidney disease ('ADPKD')....XORTX also intends to initiate and pursue a Special Protocol Assessment (SPA) with the FDA for the XRX-OXY-301 program to further de-risk the development program for XORLO™ for the treatment of ADPKD....We now have the optimal information needed to conduct the XRX-OXY-301 phase 3 clinical trial of XORLO™ to treat individuals diagnosed with ADPKD."

FDA event • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Polycystic Kidney Disease

XORTX Announces Submission of Orphan Drug Designation Request for XRx-008 Program to Treat Autosomal Dominant Kidney Disease (GlobeNewswire) - “XORTX Therapeutics Inc…is pleased to announce it submitted an Orphan Drug Designation (‘ODD’) Request to the US Food and Drug Administration (‘FDA’) for the XRx-008 program for the treatment of Autosomal Dominant Polycystic Kidney Disease (‘ADPKD’). XORTX anticipates updates regarding the ODD application and will provide announcements as information becomes available.”

Orphan drug • Autosomal Dominant Polycystic Kidney Disease • Polycystic Kidney Disease

XORTX Therapeutics Inc. (BIO 2022) - "is a pharmaceutical company with two clinically advanced products in development – XRx-008 for Autosomal Dominant Polycystic Kidney Disease (ADPKD), XRx-101 for Coronavirus / COVID-19 infection and XRx-225 is a pre-clinical stage program for Type 2 Diabetic Nephropathy (T2DN). At XORTX Therapeutics, we are dedicated to developing medications to improve the quality of life and future health of patients. Additional information on XORTX Therapeutics is available at www.xortx.com."

Autosomal Dominant Polycystic Kidney Disease • Diabetes • Diabetic Nephropathy • Genetic Disorders • Infectious Disease • Nephrology • Novel Coronavirus Disease • Polycystic Kidney Disease • Renal Disease • Type 2 Diabetes Mellitus

XORTX to Present at BIO CEO and Investor Conference (GlobeNewswire) - "Dr. Allen Davidoff, XORTX’s President and CEO will provide an overview of the Company’s upcoming, first-in-class kidney disease programs that include XRx-008 for Autosomal Dominant Polycystic Kidney Disease (ADPKD)…Presentation is scheduled for viewing February 16-18, 2021 on the BIO-CEO conference site."

Clinical • Autosomal Dominant Polycystic Kidney Disease

XORTX increases financing to up to $5-million (Stockwatch) - "Under the revised terms of the private placement, the company plans to raise between $2-million and $5-million through the issuance of between eight million and 20 million common share units of the company at a price of 25 cents per unit....The proceeds from the sale of the units will be utilized by the company for preclinical trials, IND (investigative new drug) filing for XRx-008 for polycystic kidney disease (a phase 3 ready program), R&D (research and development), and for general corporate purposes."

Financing • Autosomal Dominant Polycystic Kidney Disease

XORTX Provides Corporate Update - "Complete orphan drug designation filing for the XRx-008 ADPKD program...Negotiating the special protocol assessment (SPA) for ADPKD phase 3 registration clinical trial...Preparing and filing additional provisional patent applications in both XRx-008 and XRx-101 programs; and...Grant of pending patent applications in US and Europe."

Orphan drug • Patent • Special Protocol Assessment • Autosomal Dominant Polycystic Kidney Disease

XORTX Announces Private Placement (Xortx Therapeutics Press Release) - "XORTX Therapeutics...announces a non-brokered private placement to raise between CDN $2,000,000 and CDN $3,000,000 through the issuance of between 8,000,000 and 12,000,000 common share units of the Company at a price of CDN $0.25 per unit (the 'Private Placement')….The proceeds from the sale of the Units will be utilized by the Company for pre-clinical trials, IND (Investigative New Drug) filing for XRx-008 for polycystic kidney disease (a phase 3 ready program), R&D and for general corporate purposes."

Financing • Autosomal Dominant Polycystic Kidney Disease

XORTX Announces Grant of European Patent (GlobeNewswire) - "XORTX Therapeutics Inc....is pleased to announce receipt of notification that the patent 'Formulations of Xanthine Oxidase Inhibitors' will be granted by the European Patent Office. The patent covers compositions and methods of using XORTX’s proprietary formulations of xanthine oxidase for, renal and other diseases where aberrant purine metabolism has been implicated in disease progression....Grant of this patent provides the protection to expand our clinical trials, commercialization and partnering opportunities throughout Europe....XORTX’s XRx-008, for ADPKD, is a proprietary combination of uric acid lowering agents and other excipients."

Patent • Autosomal Dominant Polycystic Kidney Disease • Renal Disease

XORTX Participation in Virtual Conferences (GlobeNewswire) - "XORTX Therapeutics Inc....is pleased to announce its participation in two upcoming virtual conferences in November 2020....This presentation is associated with XORTX’s ADPKD program and XRx-008 and is entitled 'Orphan Drug Development Using the 505(b)2 Pathway'."

Clinical • Autosomal Dominant Polycystic Kidney Disease

XORTX provides program update regarding XRx-008 for ADPKD and XRx-101 for coronavirus / COVID-19 programs (GlobeNewswire) - "XORTX Therapeutics Inc....is pleased to provide the following update on its clinical stage programs to treat chronic autosomal dominant polycystic kidney disease (ADPKD) - XRx-008...XORTX continues to seek non-dilutive funding for XRx-008 and XRx-101 programs with several granting agencies....The Company’s activities in the past month to advance both the XRx-008, for ADPKD and XRx-101 for acute kidney injury due to COVID-19 have accelerated as we focus on applications for non-dilutive funding opportunities for these programs."

Financing • Autosomal Dominant Polycystic Kidney Disease

Biopharma Co. reports 1st step to advance clinical COVID treatment trials (StockHouse) - "Biopharmaceutical Company XORTX Therapeutics Inc....recently announced that it had granted a manufacturing contract to Lonza Pharma & Biotech...Responding to this urgent call for therapies, XORTX has initiated the manufacturing of...XRx-008 for its program to treat autosomal dominant polycystic kidney disease (ADPKD). Lonza has been granted access to develop this active pharmaceutical ingredient....The Company added that the manufacture of the drug and its trial process are expected to begin later this month as it engages global regulatory agencies in clinical development discussions."

Clinical • Licensing / partnership • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Polycystic Kidney Disease

XORTX Provides Overview and Update of Key Activities (GlobeNewswire, XORTX Therapeutics Inc.) - "XRx-008 for Autosomal Dominant Polycystic Kidney Disease (ADPKD): Growing outreach to key polycystic disease physicians and patient advocacy groups to advance the XRx-008 program toward a phase 3 registration trial; Advanced Orphan Drug Designation ('ODD') documents for completion of filing with the FDA for XRx-008 for the treatment of progressive kidney disease due to ADPKD."

New P3 trial • Orphan drug • Autosomal Dominant Polycystic Kidney Disease • Infectious Disease

XORTX launches XRx-101 following the filing of a provisional patent to potentially treat respiratory and kidney disease injury due to coronavirus infection (GlobeNewswire, XORTX Therapeutics Inc.) - "XORTX Therapeutics Inc...is re-issuing its earlier press release with a clarified title pursuant to the Company’s announcement that it is exploring the potential of using XRx-101 (a new formulation of Oxypurinol) as a novel treatment for acute kidney and lung injury accompanying Coronavirus infection and specifically for the COVID-19 infection...XORTX believes that XRx-101 (a formulation of Oxypurinol) could be relevant and important for decreasing kidney and lung associated health consequences of the Coronavirus infection....XORTX is preparing to manufacture and submit final investigative new drug (IND) applications for XRx-008 for the Company’s planned phase 3 clinical trial in Autosomal Dominant Polycystic Kidney Disease (ADPKD)."

Clinical • Commercial • IND

XORTX announces re-pricing of private placement (GlobeNewswire) - "'...the Company is also continuing its efforts to advance clinical development of XRx-008 for polycystic kidney disease (a phase 3 ready program)...The net proceeds of the offering will be used to advance XORTX’s development of XRx-008...for polycystic kidney disease (a phase 3 ready program)...including operations and ongoing licensing discussions."

Financing • Trial status

XORTX provides overview and announces $5 million financing to advance clinical trials (GlobeNewswire) - "Preparation and filing Pre-IND documents with the FDA related to the development of XRx-008...for the treatment of autosomal dominant polycystic kidney disease ('ADPKD'). Filing of Orphan Drug Designation ('ODD') documents with the FDA for XRx-008 for the treatment of ADPKD and FDA clarification of additional information required to obtain ODD status for XRx-008. Meeting and discussion with the FDA regarding XRx-008 Clinical Development Plan resulting in an accelerated clinical development plan composed of a bioavailability study of XRx-008 in man, then a single phase 3 registration clinical trial (under a special protocol assessment (SPA)). Thereafter submission of a New Drug Application (NDA) for marketing approval."

FDA event • IND • NDA • New P3 trial • New trial • Orphan drug