AstraZeneca is set to acquire a preclinical oral PCSK9 inhibitor programme from Dogma Therapeutics for an unspecified sum, with the aim of taking it forward into clinical development for dyslipidaemia and familial hypercholesterolemia.
Dyslipidaemia is a condition characterised by an abnormal amount of lipids, including low-density lipoprotein (LDL) cholesterol, in the blood – a key risk factor for cardiovascular disease. Familial hypercholesterolemia is a genetic condition that results in the body being unable to remove LDL cholesterol from the blood and also increases the risk of developing coronary heart disease.
So far, no oral PCSK9 inhibitors are currently available for patients or in clinical development. This type of medication could have great potential, given that increased levels of the PCSK9 protein are associated with high LDL cholesterol in the blood.
The PCSK9 inhibitors AZ has acquired from Dogma are able to bind directly to a novel part of PCSK9 and have been able to block its activity and lower LDL cholesterol levels in preclinical models.
“Raised LDL cholesterol is a key risk factor for cardiovascular disease and is estimated to cause 2.6 million deaths worldwide every year. While PCSK9 is a well validated target for lowering LDL cholesterol it has been a hugely challenging target to inhibit with small molecules,” said Mene Pangalos, executive vice president, BioPharmaceuticals R&D at AZ.
“This agreement with Dogma Therapeutics offers us the opportunity to develop the first small molecule, orally bioavailable PCSK9 inhibitor, for patients at risk of cardiovascular disease,” he added.
