Nyrada cholesterol-lowering drug studies validated in peer reviewed medical journal

Nyrada blood cholesterol lowering drug studies ASX NYR protein PCSK9
Nyrada has identified a small molecule that inhibits the production of the blood protein PCSK9 and lowers blood cholesterol levels in the body.

Drug discoverer and early stage developer Nyrada (ASX: NYR) has had a significant advance made in the pre-clinical studies of its cholesterol-lowering drug program published in a medical journal.

The international peer-reviewed journal Bioorganic and Medicinal Chemistry published a paper that demonstrates Nyrada has successfully identified a small molecule that inhibits the production of PCSK9 in-vitro and lowers total cholesterol levels in the blood plasma in an in-vivo model (in this case, mice).

According to Nyrada, it has been a long-standing pharmaceutical challenge to design a small molecule that can bind to and inhibit the function of PCSK9, a blood protein involved in regulating the amount of cholesterol in the bloodstream by reducing the body’s ability to remove low-density lipoprotein (LDL) cholesterol. This is often referred to as “bad” cholesterol and is known to raise the risk of heart disease and stroke.

The biotech company said the advance marks strong progress in its strategy to develop an oral cholesterol-lowering drug for patients to assist the estimated 40% of individuals who do not respond adequately to statin therapy.

The published paper, entitled A small molecule inhibitor of PCSK9 that antagonizes LDL receptor binding via interaction with a cryptic PCSK9 binding groove, forms the basis of Nyrada’s intellectual property patent application currently under consideration by the US Patent Office.

“It is a tremendous achievement to have our PCSK9i science validated by our peers,” Nyrada Scientific Advisory Board member, PCSK9 pioneer and publication co-author Professor Gilles Lambert said.

“The paper confirms that Nyrada PCSK9i compounds lower blood cholesterol levels in an in-vivo model and restore LDL cholesterol capture by human cells. This provides proof-of-concept that a small molecule inhibitor against PCSK9 is therapeutically viable,” he said.

“Overcoming this binding challenge with our PCSK9i drug candidate and having the results validated by the scientific community is a vital early step in protecting the commercial value of this asset,” Nyrada chief executive officer James Bonnar said.

Combination therapy

The standard treatment for lowering cholesterol is statin therapy, with global market sales of statins currently estimated at US$20 billion in 2020.

However, statin drugs that cause the body to make less cholesterol lead the body to offset this effect by increasing PCSK9 levels.

According to Nyrada, these statin-associated rises in PCSK9 levels are considered responsible for the instances where statins alone fail to deliver target LDL-cholesterol levels in individuals.

“A small molecule PCSK9 inhibitor that can be combined with a statin opens the potential for a convenient and cost-effective single pill therapy for high LDL cholesterol,” the company stated.

In addition, Nyrada said the the oral combination therapy is expected to have broader patient acceptance over injectable PCSK9i options that currently are available.

Next steps

Since submitting the paper for publication, Nyrada said it has identified further small molecules with “even greater potency” to the compound described in the paper.

The next step in the PCSK9i program is to confirm the lead candidate that will be taken through to the clinic.

The PCSK9i program is one of Nyrada’s two main drug programs, with the other concerning a drug to treat traumatic brain injury and stroke.

Nyrada made its debut on the ASX last month after successfully raising $8.5 million in an IPO.

The IPO funds were planned to accelerate the development of Nyrada’s leading research and development programs with the aim to have at least one drug candidate ready to enter a first-in-human phase I safety, tolerability and pharmacokinetic study by the end of 2021.

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