Date: 2014-06-02
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the European Atherosclerosis Society (EAS) Meetings in Madrid, Spain
Company: Cerenis Therapeutics (France)
Product: CER-001
Action
mechanism: CER-001 is a complex of recombinant human ApoA-I and 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), Homozygous Familial Hypercholesterolemia (HoFH)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Country:
Trial details:
Latest
news: * On June 2, 2014, Cerenis Therapeutics announced that two of its Phase II studies, SAMBA and MODE (Modifying Orphan Disease Evaluation), with CER-001, an engineered human apoA-I-containing pre-β HDL mimetic, met their primary clinical endpoints in patients with Familial Primary Hypoalphalipoproteinemia (FPHA) and Homozygous Familial Hypercholesterolemia (HoFH), respectively.
SAMBA clinical trial: Proof-of-Mechanism data will be presented at the EAS from the SAMBA Phase II efficacy and safety trial in patients with Familial Primary Hypoalphalipoproteinemia (FPHA), a rare syndrome of severe HDL deficiency caused by mutations in the genes responsible for HDL synthesis /maturation and characterized by accelerated atherosclerosis. This pharmacokinetic/pharmacodynamic trial, conducted by Principal Investigator, Erik S.G. Stroes, MD, PhD of the Department of Vascular Medicine at the Academic Medical Center (AMC) in Amsterdam, The Netherlands, enrolled 7 FPHA patients in an open-label single-arm active-treatment study and assessed the efficacy of infused CER-001 engineered human apoA-I-containing pre-β HDL particles in reconstituting the endogenous Reverse Lipid Transport in individuals who have defects in the natural HDL pathway and facilitate elimination of cholesterol from the body.
The data from patients receiving CER-001 treatment on top of optimized standard of care LDL-c-lowering therapy showed that CER-001 performed the four steps of the Reverse Lipid Transport pathway: CER-001 increased cholesterol mobilization and esterification in the HDL fraction, and one month of treatment with 9 doses of CER-001 resulted in a statistically significant reduction in carotid artery Mean Vessel Wall Area, as measured by Magnetic Resonance Imaging (3T-MRI). CER-001 was well-tolerated.
MODE clinical trial: Cerenis also reported that data from the MODE (Modifying Orphan Drug Evaluation) trial, a Phase II efficacy and safety study in patients with Homozygous Familial Hypercholesterolemia (HoFH), a rare disease of markedly elevated LDL-cholesterol (bad cholesterol) levels caused by genetic defects in the LDL-receptor pathway and characterized by premature atherosclerosis. Data will also be presented as a late-breaking clinical trial at the EAS. The open-label single-arm active-treatment study in 23 patients with homozygous FH met the prespecified primary clinical endpoint in the modified Intention to Treat population, demonstrating a statistically significant reduction in carotid artery Mean Vessel Wall Area, as measured by Magnetic Resonance Imaging (3T-MRI), after 6-months of bi-weekly CER-001 treatment on top of optimized LDL-c-lowering therapy, including apheresis. CER-001 was well-tolerated. (NCT01412034)
