Laboratoris Sanifit S.L., a clinical-stage biopharmaceutical company focused on treatments for calcification disorders, today announced successful initiation of the first clinical trial of its lead candidate, SNF472, for the treatment of the orphan disease calciphylaxis (calcific uraemic arteriolopathy, CUA).
The recently initiated Phase II study is the first clinical investigation of SNF472 in patients with newly diagnosed calciphylaxis. Designed as an open-label investigation, eligible patients will be treated over three months to assess the effect of SNF472 on wound healing and pain. The trial is led by Prof Vincent Brandenburg, University Hospital RWTH Aachen, Germany, along with a team of internationally recognized calciphylaxis experts in the US, Spain and the UK. This multi-centre study plans to enroll a total of 15 adult patients with first data expected in Q2 2017.
Calciphylaxis is a serious and rare condition characterized by vascular calcification and thrombosis leading to necrosis (cellular death) of the skin and fatty tissue. Patients with calciphylaxis experience painful skin ulcers with a high risk of severe infection and a 50% rate of death within the first year after diagnosis. The condition is seen predominantly in patients receiving dialysis therapy due to end stage renal disease (ESRD) and is related to the abnormal deposition of calcium in small blood vessels and other tissues, a process known as ectopic calcification. Approximately 1-4% of patients with ESRD experience calciphylaxis and there is currently no US Food & Drug Administration (FDA) or European Medicines Agency (EMA) approved therapy to treat this disorder.
SNF472 is administered during hemodialysis in patients with ESRD. It selectively binds to hydroxyapatite and directly inhibits the initiation and progression of ectopic calcification. Preclinical models demonstrate that SNF472 reduces the progression of calcium deposition in blood vessels and cardiac tissue. SNF472 has received orphan drug designation for the treatment of calciphylaxis from both the EMA and FDA.