The Cardiovascular Program aims to deliver a single pill for the treatment of high cholesterol. This is via a PCSK9 inhibitor drug that assists statin drugs in lowering LDL-cholesterol levels in people at risk of cardiovascular disease.

NYX-330 is intended to meet identified market needs of

  • Matching the convenience of statin therapy with a daily, oral tablet
  • Match the cost-effectiveness of statin therapy

NYX-330 currently is undergoing pre-clinical studies aimed at optimising its drug-like qualities.

Incidence of Cardiovascular Disease

Cardiovascular disease is the leading cause of death globally. In the US, more than 600,000 people die each year from cardiovascular disease, accounting for 1 in 4 deaths. In 2016, there were an estimated 17.9 million deaths globally, of which 85% or 15.2 million were due to heart attack or stroke.

When the body has too much LDL (“bad”) cholesterol, the LDL cholesterol can build up on the walls of blood vessels. This build-up is called “plaque.” As blood vessels build up plaque over time, the insides of the vessels narrow, and this blocks blood flow to and from the heart and other organs. This condition is called atherosclerotic cardiovascular disease (ASCVD), sometimes referred to as “hardening of the arteries”. When blood flow to the heart is blocked, it can cause angina (chest pain) or a heart attack. When blood flow is blocked in the arteries supplying the brain, it can cause a stroke.


PCSK9 is a blood protein that serves a normal purpose in holding LDL in blood. This means that high PCSK9 levels result in high blood LDL, which causes further build up of plaques in blood vessels. Thus, it is favourable to reduce your PCSK9 protein levels as this increases the LDL receptors on the surface of liver cells and these help to clear LDL from the blood stream.


Current standard of care

Current standard of care for hypercholesterolaemia is treatment with once daily, oral statins. Statins are inexpensive and typically lower LDL cholesterol levels by between 30-55%. However, up to 50% of patients fail to achieve a 50% reduction in LDL cholesterol. In 2017, estimated global sales of statins were US$19 billion.

Another approach to lower LDL cholesterol levels is via the inhibition of PCSK9. Although several strategies have been tried for inhibiting PCSK9, the most successful approach is the injection of monoclonal antibodies (mAb) that interfere with the function of PCSK9 thereby increasing the rate at which LDL is removed from the bloodstream. There are currently two marketed mAb PCSK9 inhibitors, both launched in 2015, evolocumab (Repatha: Amgen) and alirocumab (Praluent: Sanofi and Regeneron). The use of these mAb drugs to block the ability of PCSK9 to bind to the LDL receptor has proved to be the most effective approach to date in inhibiting PCSK9 function and in reducing LDL cholesterol levels. These mAb drugs typically can reduce LDL cholesterol levels up to 60% greater than statins alone. PCSK9 mAb drugs have few adverse side-effects, and none are considered life-threatening. However, they come with a high cost (approx. US$5K per year) and the self-injecting needles are not user friendly. To date, no orally bioavailable anti-PCSK9 drug has advanced to the clinic.

Nyrada is seeking to fill this void in the market by developing an oral PCSK9 inhibitor. Being oral, it will be more user friendly than the current injectable PCSK9 inhibitors. It will be more cost effective than current monoclonal PCSK9 inhibitors, which due to their manufacturing, cannot be mass produced and have a limit to how cheaply they can be produced. As far as the company is aware, it is only one of two companies pursuing the development of an oral PCSK9 inhibitor.