New compounds from the lab of medicinal chemist Longqin Hu hold promise as a much-needed treatment for people with cystinuria.
This series of novel compounds with the potential to revolutionize the treatment of cystinuria has been licensed by Rutgers to PharmaKrysto Ltd, a U.K. biopharmaceutical company, for continuing preclinical development, with the goal of entering clinical studies as soon as possible. The compounds, known as L-cystine diamides, emerged from the cystinuria research project in the laboratory of Dr. Hu, professor and chair in the Department of Medicinal Chemistry, in collaboration with colleagues in genetics at the School of Arts and Sciences. Among these L-cystine compounds is the highly effective inhibitor LH708.
Cystinuria is a rare genetic condition affecting children and adults and causing large amino acid crystals to form in the kidneys. These crystals are responsible for repeated episodes of severe, debilitating pain and, ultimately, kidney damage. Available treatments are ineffective in many patients and can cause significant side effects; if untreated, cystinuria can lead to kidney failure.
L-cystine diamides act as molecular “imposters,” preventing amino acid crystals from forming in the kidneys and thus represent a potentially revolutionary treatment for people with this chronic, lifelong condition.
The exclusive global licensing deal with PharmaKrysto Ltd, a Scotland-based start-up company, was announced in July. In recognition of the rarity and seriousness of cystinuria, the leading candidate, PK10, has been granted an orphan drug designation by both the U.S. Food and Drug Administration and the European Medicines Agency.
Dr. Hu and his colleague, Rutgers geneticist Amrik Sahota, are co-principal investigators of a five-year, $2.8-million NIH grant to develop effective, small molecule drugs that can prevent recurrent stone formation in patients with cystinuria.