Autosomal dominant polycystic kidney disease: As of April 2020, XORTX Therapeutics advanced documents related to orphan drug designation for oxipurinol to complete filing with the US FDA for the treatment of progressive kidney disease due to autosomal dominant polycystic kidney disease  .
In November 2018, XORTX Therapeutics received a response letter from the US FDA regarding an orphan drug designation application filed by the company for oxipurinol for the treatment of autosomal dominant polycystic kidney disease  . In September 2018, XORTX Therapeutics submitted an application for the Orphan Drug Designation status to the US FDA  .
As of April 2018, XORTX Therapeutics is conducting a phase II/III trial for autosomal dominant polycystic kidney disease under a special protocol assessment (XORTX Therapeutics pipeline, April 2019)   .
In June 2018, XORTX Therapeutics in collaboration with Cato Research (also known as Cato Clinical Research) filed a pre-IND meeting request letter with the US FDA for the phase II trial of oxipurinol for the treatment of ADPKD. The request, supported by pre-IND documents, initiated a process for communication and discussion with the FDA regarding the phase II trial, defining the path for the clinical development and marketing approval of oxipurinol  . In August 2018, XORTX Therapeutics and Cato Clinical Research announced that the companies have submitted all the necessary pre-IND meeting documents with the FDA for the scheduled pre-IND meeting with US FDA on September 20th, 2018  . In October 2018, the company reported a major revision in the development plan of oxipurinol which included manufacturing, formulation, non-clinical study plans and development strategy comprising of separate phase II and phase III clinical trials, followed by marketing application. The discussions with the FDA also suggested a substantially shorter path for marketing approval. The outcome of the pre-IND meeting with the US FDA included filing of an IND application, to characterise the bioavailability and pharmacokinetics of oxipurinol in advancing clinical studies in humans, to complete the orphan drug designation process and to conduct a single pivotal phase II/III clinical trial. The company intends to seek a special protocol assessment (SPA) from the US FDA to help in a design, endpoints and statistical analysis of a pivotal phase II/III trial to support the marketing approval  .
Earlier, XORTX Therapeutics had reported that phase II development of oxipurinol (XORLO) for the treatment of autosomal dominant polycystic kidney disease was planned for 2016  . The company will investigate the first licensing or acquisition opportunity upon completion of the phase II development.
Several investigator-sponsored phase II trials showed that decreasing and maintaining SUA levels can suppress weight gain and decrease blood pressure, slow progression of blood vessel and kidney injury in patients with high SUA.
XORTX had planned to conduct a phase II trial of the product for the treatment of hereditary hypouricaemia in 2016.
Prior to February 2016, XORTX Pharma initiated a phase I study to evaluate oxipurinol for the treatment of patients with autosomal dominant polycystic kidney disease in the US (XORTX Pharma pipeline, February 2016 and June 2017).
Acute kidney injury, Acute lung injury and COVID-2019 infections
In May 2020, XORTX submitted Coronavirus Treatment Accelerated Program (CTAP), a development and data plan regarding a COVID-19 package and received guidance to submit a pre-IND package for review  .
XORTX has also initiated discussions with the US Medical Countermeasures Group associated with the U.S. Government’s Biomedical Advanced Research and Development Authority (BARDA) and separately with the National Institute of Health (NIH). These communications with BARDA and NIH are expected to further aid development of XORTX’s XRx-101 program  .
In March 2020, XORTX Therapeutics announced that it is developing the use of XRx 101 (a new formulation of oxypurinol) for the treatment of acute kidney injury, acute lung injury and COVID-2019 infections. The company believes that XRx 101 has the potential to treat COVID-2019 infections based on its anti-viral properties. The rationale was made based on various in vivo studies showing that xanthine oxidase inhibition may confer suppression of viral infection and decrease symptoms of coronavirus COVID-19 infection and importantly improve survivability, and most importantly to protect the kidneys from AKI. The company also announced that it has received an approval letter from the US FDA to accelerate the development of oxypurinol for the treatment of acute kidney injury, acute lung injury and COVID-2019 infections   .
In January 2020, XORTX Therapeutics announced the re-pricing of private placements previously announced in April 2019. The company intends to raise approximately $US5 million and the proceeds will be used to advance development of oxipurinol for polycystic kidney disease and XRx 221 [see Adis Insight Drug profile800045135] type 2 diabetic nephropathy (a phase IIb ready programme) including operations and ongoing licensing discussions   .
XORTX raised $US1.5 million in Series A financing with proceeds allocated for the management of phase II development of XORLO.