PharmaKrysto announces licensing agreement with Rutgers University for new treatment for people with cystinuria
New treatment now also granted Orphan Designation* in the US and EU
PharmaKrysto Ltd, a biopharmaceutical company based in Scotland, UK, announces today that it 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.”
