A SCOTTISH biopharmaceutical start-up has signed a global licensing deal with an American university after its new treatment for the kidney disease cystinuria was granted regulatory approval in both the US and Europe.
Borders-based PharmaKrysto will work with Rutgers University in New Jersey to develop patient trials for “PK10”, a “potentially revolutionary” compound to treat people with cystinuria – an inherited disease that causes large, painful and debilitating crystals of amino acid to form in the kidneys.
PharmaKrysto will also collaborate with the Institute of Genetic Medicine at Newcastle University.
The regulators overseeing drug safety and security in the US and EU, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have granted “orphan designation”, which is designed to encourage the development of treatments for rare diseases.
This follows pre-clinical data which PharmaKrysto said demonstrates that the treatment has the “potential to be effective” in patients with cystinuria.
About 145,000 adults and children in the US and EU have cystinuria, a genetic condition which causes large amino acid crystals to form in the kidneys – these crystals, which can grow to two-three centimetres, cause repeated episodes of severe pain and ultimately irreparable kidney damage.
PharmaKrysto, headed by chief executive Julian Howell, who led clinical development and drug safety at ProStrakan, the Galashiels-based pharmaceutical company now rebranded as Kyowa Kirin, said that its new compound acts as a molecular “imposter” to prevent amino acid crystals forming.
The company is also in the process of initiating an early-stage fundraising round to prepare for patient trials and, in an early indication of the potential for this technology, has already received indications of support and interest from private and regional public sources.
“This is a painful condition with a significant impact on the quality of life for people with cystinuria,” said Mr Howell. “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.”
Most people have their first kidney stone episode in their teens or early twenties, he added.
Distinguished Macmillan Professor Jay Tischfield, of the Department of Genetics at Rutgers University, said that clinical trials would start as soon as possible. “The teams of geneticists and medicinal chemists at Rutgers have collaborated on designing important molecules that have shown promising early results in treating cystinuria,” he said.
“We are very pleased to continue this work with the experienced team from PharmaKrysto with the goal of starting clinical studies as soon as possible.”
There have been no new treatments for cystinuria introduced for over 30 years, according to PharmaKrysto, with existing drugs including anti-inflammatories used to treat rheumatoid arthritis.
Professor David Goldfarb, chief of nephrology at NYU Langone Health in New York, welcomed the development. “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,” he pointed out. “It’s very encouraging that we have new ways of treating patients being developed.”
Professor John Sayer, Clinical Professor of Renal Medicine at the Institute of Genetic Medicine at Newcastle University added: “There have been no new approaches to preventing the build-up of cystine within the kidney for many years.
“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.”
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