Industry News: AstraZeneca acquires oral PCSK9 inhibitor program from Dogma Therapeutics

AstraZeneca anticipates entering clinical development in 2021

18 Sep 2020


AstraZeneca has entered into an agreement with Dogma Therapeutics to acquire its preclinical oral PCSK9 inhibitor program. The Company aims to take the program forward into clinical development for dyslipidaemia, or abnormal amount of lipids in the blood, and familial hypercholesterolemia, a common genetic condition that causes high cholesterol.

PCSK9 is a protein that regulates the level of low-density lipoprotein (LDL), or ‘bad’  cholesterol in the blood. Increased activity of PCSK9 is associated with high LDL cholesterol. The acquired PCSK9 inhibitors are small molecules that bind directly to a novel part of PCSK9 and have shown to block its activity and lower LDL cholesterol in preclinical models.

There are currently no oral PCSK9 inhibitors available to patients or in clinical development.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “Raised LDL cholesterol is a key risk factor for cardiovascular disease and is estimated to cause 2.6 million deaths worldwide every year. Whilst PCSK9 is a well-validated target for lowering LDL cholesterol it has been a hugely challenging target to inhibit with small molecules. 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.”

Brian Hubbard, Chief Executive Officer, Dogma Therapeutics, said: “We have built a robust data package that highlights the cholesterol-lowering and safety potential of our oral PCSK9 program. This agreement with AstraZeneca meets our strategic goal to accelerate access to patients unable to meet target LDL cholesterol.”

Dyslipidaemia and familial hypercholesterolemia

Dyslipidaemia is defined as an abnormal amount of lipids, such as LDL cholesterol, in the blood. Raised LDL cholesterol is a key risk factor for cardiovascular disease and is estimated to cause 2.6 million deaths worldwide every year. Causes may be primary (genetic) or secondary (lifestyle and other factors). Familial hypercholesterolaemia, a primary dyslipidaemia, is a genetic condition that results in the body being unable to remove LDL cholesterol from the blood. Patients with familial hypercholesterolaemia are at high risk of premature coronary heart disease.

PCSK9

PCSK9 is an important regulator of cholesterol metabolism. Increased activity of the protein is associated with higher levels of LDL cholesterol. It is believed that inhibiting PCSK9 activity lowers LDL cholesterol and reduces the risk of cardiovascular disease.

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