pipeline

Internal Programs

Silence’s lead internal RNAi therapeutic candidate, Atu027, is currently in a Phase I clinical trial.  Atu027 is a liposomal siRNA formulation targeting PKN3 for the treatment of advanced solid cancer. 

PKN3 is a key regulator during angiogenesis and lymphangiogeneis, while also acting as a major regulator of metastasis and motility during pathological processes.  Accordingly, Silence believes that inhibition of PKN3 with Atu027 may lead to a reduction in nutrient and oxygen supply to solid tumors, as well as interfering with tumor formation, endothelial cell motility and metastasis.

In preclinical studies, Atu027 has demonstrated anticancer activity against a broad range of tumor types including gastrointestinal (including pancreatic), non-small cell lung, prostate, melanoma, liver and others.  These animal studies also showed no indications of genotoxicity in both rodents and non-human primates, while demonstrating no indications of cardiovascular or pulmonary function impairment in Cynomolgus monkeys.