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PharmaKrysto Ltd, a biopharmaceutical company based in Scotland, has completed an exclusive global licensing deal with Rutgers University, New Jersey, USA, 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 also announces that based on preclinical data demonstrating that this treatment has the potential to be effective in patients with cystinuria and, in a clear recognition of the seriousness of this medical condition as well as its rarity in the USA and Europe, PK10 has been granted Orphan Designation* by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Cystinuria is a genetic condition, affecting children and adults, which causes large amino acid crystals to form in the kidneys. These crystals are responsible for repeated episodes of severe pain and ultimately irreparable kidney damage. PharmaKrysto’s new compound, PK10, acts as a molecular ‘imposter’ to prevent amino acid crystals forming in the kidneys. PharmaKrysto will continue its preclinical development of PK10 with the goal of entering clinical studies and treating people with cystinuria as soon as possible.
The company is currently 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.
Julian Howell, CEO of PharmaKrysto, said: “These three events represent the achievement of major milestones for PharmaKrysto as we seek to develop this exciting new treatment for people with cystinuria. This is a painful condition with a significant impact on the quality of life for people with cystinuria. 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.”
About the licensing agreement, Distinguished Macmillan Professor Jay Tischfield, of the Department of Genetics at Rutgers University, said: “The teams of geneticists and medicinal chemists at Rutgers have collaborated on designing important molecules that have shown promising early results in treating cystinuria. 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.”
Prof David Goldfarb, Chief of Nephrology, NYU Langone Health, NY, US, commented: “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. It’s very encouraging that we have new ways of treating patients being developed.”
Prof John Sayer, Clinical Professor of Renal Medicine at the Institute of Genetic Medicine, Newcastle University, UK also said: “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.”