Rare Disease Report

Rutgers & PharmaKrysto Partner Up to Develop New Treatment for Cystinuria

JULY 23, 2018
Krista Rossi
Characterized by painful and debilitating crystals comprised of amino acid in the kidneys, cystinuria is an inherited disease that has significant health implications. Available treatments for the disease have limited efficacy and are riddled with several side effects, making cystinuria a serious medical challenge.

However, a new licensing agreement between PharmaKrysto Ltd, a biopharmaceutical company based in Scotland, UK, and Rutgers University, New Jersey, has been made for the development of a new treatment for the debilitating disease that might change the game; the compound is referred to as PK10.

“I know from my work with people with cystinuria that treatments available to them are not always effective and the side effects can be a significant problem,” commented professor David Goldfarb, chief of nephrology, NYU Langone Health, New York, in a recent statement. “It’s very encouraging that we have new ways of treating patients being developed.”

PK10 is a “potentially revolutionary compound” backed by strong preclinical data, which demonstrates the treatment’s potential for effectiveness in patients with cystinuria. The genetic condition causes crystals to form in the kidneys. Since the cystine crystals can grow to many centimeters in size, they prevalently cause pain, blockage of urine, and permanent kidney damage that results in limited or stifled daily activities and living. To prevent amino acid crystals from forming in the kidneys, the drug acts as a molecular “imposter.”

Since the geneticist and medicinal chemist teams at Rutgers University collaborated and assisted in designing the important molecules that have shown promising early results in treating cystinuria, according to Macmillan Professor, Jay Tischfield, of the department of Genetics at Rutgers University, they are looking forward to continuing research in clinical trials as soon as possible.
 
“There have been no new approaches to preventing the build-up of cystine within the kidney for many years,” added professor John Sayer, clinical professor of Renal Medicine at the Institute of Genetic Medicine, at Newcastle University, in the United Kingdom. “This approach of disrupting crystal growth has been effective in the laboratory, and our department looks forward to collaborating with PharmaKrysto on clinical studies in cystinuria patients.”

Currently, PharmaKrysto Ltd is preparing for patient trials through its initiation of an early stage fundraising round. Private and regional public sources have already offered support and interest in an early indication for this technology’s potential. 

“This is a painful condition with a significant impact on the quality of life for people with cystinuria,” emphasized Julian Howell, CEO of PharmaKrysto.There are currently no effective treatments for many patients, so we are determined to develop this solution for those people who continue to experience severe, frequent pain and complicated kidney disease.”

PK10 was previously granted an orphan designation by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

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