Our HoFH Therapy
Unmet Clinical Need
Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic disorder characterized by abnormally high levels of bad cholesterol (LDL) levels in the blood. There is an accelerated deposition of bad cholesterol in the walls of coronary arteries of HoFH patients that results in pre-mature atherosclerosis. In other words, HoFH patients develop heart disease and associated events such as heart attacks and stroke at a much younger age than would be expected in the general population.
HDL Therapy Platform
HDL therapy is widely considered to be the next frontier in the fight against heart disease in HoFH patients. HDL counteracts the effects of LDL. HDL removes excess lipids from vessel walls and carries it to the liver where they are eliminated. A build up in excess lipids results in coronary plaque formation that if gone untreated can result in heart attack or stroke.
Pre-Beta HDL Role
HDL is found in the human body in two forms, pre-beta HDL and alpha HDL. Pre-beta HDL acts like an empty dump truck removing lipids from vessel walls. Once the empty dump truck is filled, HDL changes form to the alpha HDL, the full dump truck, where the lipids are taken to the liver where excess lipids are eliminated from the body. The pre-beta HDL, or the empty dump truck, is the critical component in removing excess lipids.
The problem is that, in humans, only 5% of the HDL comes in the pre-beta form. In other words, there is not enough naturally occurring “empty dump trucks” to get enough lipids out. More pre-beta HDL is required to get the job done. There is currently no available therapy to increase the level of pre-beta HDL.
Our Therapeutic SolutionWe have developed the first innovative acute care therapeutic device called PDS-2™ System to address this unmet need. Our patented PDS-2™ System uses a patient’s own plasma to transform the majority of the circulating alpha HDL (full dump trucks) into pre-beta HDL particles (empty dump trucks).
The pre-beta enriched plasma is re-infused back to the patient to accelerate removal of lipids from atherosclerotic plaque, wherever it may occur in the cardiovascular circulation.
Treatment with the PDS-2™ System will complement treatment with other lipid-modifying therapies.