CER-001 (Cerenis HDL)
CER-001 is an HDL mimetic and is based on natural apolipoprotein A-I (ApoA-I), the major structural protein of HDL. It is an infusion therapy targeting patients with acute coronary syndromes.
CER-001 has shown strong preclinical and clinical efficacy in mobilizing cholesterol and promoting reverse lipid transport (RLT). Cerenis expects to demonstrate that this compound can rapidly remove large amounts of atherosclerotic plaque and thereby significantly reduce the risk of future cardiac events.
A Phase I study of CER-001 for acute coronary syndrome (ACS) was completed and reported in May 2010.
This randomized, double-blind, placebo controlled, cross-over, single rising dose study of 32 healthy dyslipidemic volunteers
showed that CER-001 is safe and well tolerated at dosages up to 45 mg/kg. The study also demonstrated cholesterol mobilization at dose levels of 2 mg/kg and higher.
A Phase II trial for CER-001 was initiated in March 2011. This double-blind, randomized, placebo-controlled, safety and efficacy study will assess the ability of CER-001 to regress coronary atherosclerotic plaque as measured by intravascular ultrasound (IVUS). The study will include over 500 patients at 50 centers in the US, Canada and Europe, and will evaluate three different dose levels given in six weekly intravenous infusions.
An alternative development route, running in parallel with the ACS program, is the evaluation of CER-001 for the treatment of rare diseases in the lipid metabolism field.