Familial primary hypoalphalipoproteinemia (FPHA), also known as familial high-density lipoprotein (HDL) deficiencies, are rare genetic disorders. HDL is a molecule that transports cholesterol and certain fats called phospholipids through the bloodstream from the body’s tissues to the liver. Once in the liver, cholesterol and phospholipids are redistributed to other tissues or removed from the body. HDL is often referred to as “good cholesterol” because high levels of this substance reduce the chances of developing heart and blood vessel (cardiovascular) disease. FPHA is caused by changes in the ABCA1 or the APOA1 genes. These genes are responsible for HDL function, synthesis, and maturation. ABCA1 and APOA1 gene mutations decrease the amount of cholesterol or phospholipids available to form HDL, resulting in low levels of HDL in the blood. A shortage of HDL is believed to increase the risk of cardiovascular disease.
CER-001 is an engineered complex of the major structural protein of HDL, in combination with key phospholipids. Administered through intravenous (IV) infusion, CER-001 mimics the structure and function of natural HDL to stimulate the removal of excess cholesterol and other lipids. There is currently limited treatment options available specifically for FPHA. If licensed, CER-001 may offer a new treatment option for FPHA by aiding in the transport and elimination of excess cholesterol and other lipids in the liver.
Sodium thiosulfate is an injectable solution that is being developed to treat calciphylaxis-associated pain in patients undergoing dialysis. It is thought that sodium thiosulfate works by attaching itself to the calcium to form a compound that is removed from the body in the urine, reducing the build-up of calcium in the arteries. If licensed, this product will provide a treatment option for patients with calciphylaxis, who currently have few effective treatments available.