Date: 2015-12-10
Type of information: Treatment of the first patient
phase: 3
Announcement: treatment of the first patient
Company: Cerenis Therapeutics (France)
Product: CER-001
Action
mechanism: protein./HDL-mimetic. CER-001 is a complex of recombinant human ApoA-I and charged phospholipids. It is designed to mimic pre-beta HDL, the \"good\" cholesterol, to promote the removal of excess cholesterol and other lipids from artery walls and enhance reverse lipid transport.
Disease: familial primary hypoalphalipoproteinemia (FPHA)
Therapeutic area: Cardiovascular diseases - Metabolic diseases - Genetic diseases - Rare diseases
Country: Canada, Europe, USA
Trial
details: The TANGO trial is a multicenter, randomized, 48-week, double-blind, parallel-group, placebo-controlled study involving thirty patients from several sites across Europe, Canada, the United States and other countries based on availability of patients with familial primary hypoalphalipoproteinemia (FPHA).
Latest
news: * On December 10, 2015, Cerenis Therapeutics announced that the first patient has been enrolled into the Phase III TANGO trial that is designed to assess both the efficacy of CER-001 to regress atherosclerosis and its safety in patients with FPHA, who are characterized by ABCA1 or apoA-I genetic mutation and receiving background optimized lipid therapy. Inherited defects in the apoA-I or ABCA1 genes can act in a dominant manner to cause FPHA, a rare syndrome characterized by the absence or severe deficiency of HDL particles in the circulation. This means that the the body’s only natural mechanism for the elimination of cholesterol is compromised. These patients experience a rapid accumulation of cholesterol, particularly in blood vessels, which often results in accelerated atherosclerosis and premature cardiovascular disease. The LOCATION and SAMBA clinical studies have indicated that CER-001 preferentially targets atherosclerotic plaques and stimulates
The TANGO trial, which began in the 4th quarter of 2015, is on schedule and results are expected in Q3 2017. The trial is headed by Professor Erik Stroes, Professor of Medicine, Head of the Department of Vascular Medicine at the Academic Medical Center in Amsterdam. It Steering Committee includes also Prof. Jacques Genest, MD, Department of Medicine, Division of Cardiology, Faculty of Medicine, McGill University, Montréal, Canada; Prof. Henry Ginsberg, MD, Irving Professor of Medicine at Columbia University College of Physicians and Surgeons, New York, NY, USA; Prof. Eran Leitersdorf, Director, Center for Research, Prevention and Treatment of Atherosclerosis, Professor of Medicine Dorothy & Maurice Bucksbaum Chair in Molecular Genetics and Dean of the Hebrew University of Hadassah School of Medicine, Israel; Prof. Arnold von Eckardstein, Chair of the Institute of Clinical Chemistry at the University of Zurich, Switzerland, Professor of Clinical Chemistry at the University of Zurich and Director of the Institute of Clinical Chemistry of the University Hospital of Zurich, Chairman of the European Lipoprotein Club and Chairman of the Executive Committee of the Swiss Working Group on Lipids and Atherosclerosis.
cholesterol removal by emulating all the steps of the reverse cholesterol transport pathway. This increased lipid removal
has been accompanied by marked reductions in atherosclerosis as measured by the reduction in the vessel wall
dimensions of atherosclerotic arteries in patients with genetically-determined low HDL cholesterol. The primary objective of the TANGO trial is to evaluate the effect of 24 weeks treatment with CER-001 on carotid vessel wall area as compared to placebo using magnetic resonance imaging (MRI).
Cerenis Therapeutics has received two Orphan Drug Designations from the European Medicines Agency (EMA) for the use of CER-001 in the treatment of patients with apoA-I and ABCA1 deficiencies, the two patient populations who will be recruited into the TANGO trial.
Prior data supporting the orphan designation applications were obtained from the SAMBA Phase II trial of CER-001 in patients with FPHA which showed that CER-001 reconstituted the reverse lipid transport (RLT) pathway in individuals who have defects in the natural HDL pathway, facilitating elimination of cholesterol from the body.
