Evinacumab is in clinical development for the treatment of homozygous familial hypercholesterolemia (HoFH). Familial hypercholestrolaemia (FH) is an inherited condition where a person’s cholesterol levels are higher than normal from birth as the liver is unable to break down or remove excess cholesterol. Specifically, FH patients have severe elevations in low density lipoprotein cholesterol (LDL-C) levels. This can lead to heart disease at a relatively young age. HoFH is a very rare and severe form of the disease which results from inheriting a faulty gene from both parents resulting in little or no LDL receptor activity. Treatment of hypercholesterolemia in patients with HoFH can be challenging with the current therapies and there is need for new treatments.
Evinacumab is a monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 is produced in the liver and regulates levels of triglycerides, LDL-C, and high-density lipoprotein cholesterol, in the blood. Evinacumab binds to and inhibits ANGPTL3 and lowers cholesterol thereby having the potential to reduce cardiovascular risk. Results from early studies have shown that evinacumab has the potential to result in clinically significant LDL-C reductions in HoFH patients.
Treosulfan in addition to fludarabine is in clinical development for paediatric non-malignant disease prior to allogeneic stem cell transplant. Treosulfan is a medicine given to patients before they have a bone marrow transplant from a donor known as ‘allogeneic haematopoietic stem cell transplantation’. It is used as a ‘conditioning’ treatment to clear the patient’s bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. Treosulfan is used together with another medicine called fludarabine for the treatment of a variety of disorders that require a bone marrow transplant.