AstraZeneca acquires oral PCSK9 inhibitor programme from Dogma Therapeutics

AstraZeneca has entered into an agreement with Dogma Therapeutics to acquire its preclinical oral PCSK9 inhibitor programme. The Company aims to take the programme 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 programme. This agreement with AstraZeneca meets our strategic goal to accelerate access to patients unable to meet target LDL cholesterol.”

AstraZeneca anticipates entering clinical development in 2021.

Dyslipidaemia and familial hypercholesterolemia

Dyslipidaemia is defined as 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.1,2 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.3 Patients with familial hypercholesterolaemia are at high risk of premature coronary heart disease.4


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.5


AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, CVRM, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information, please visit and follow the Company on Twitter @AstraZeneca.


For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here.


1. WHO. Raised cholesterol. Last accessed 14 August 2020

2. Prescriber. Targeting dyslipidaemia to prevent cardiovascular disease. Last accessed 14 August 2020

3. Medline Plus. Familial hypercholesterolemia.,of%20LDL%20in%20the%20blood. Last accessed 14 August 2020

4. BNF. Dyslipidaemias. Last accessed 14 August 2020

5. Pokhrel B, Yuet WC and Levine SN. PCSK9 Inhibitors. StatPearls Publishing. 2020.


  • Corporate and financial