Australian chemists at Nyrada Inc (ASX: NYR) have gained international recognition in developing a protein to lower blood cholesterol levels – a market expected to exceed US$20 billion in 2020.
Nyrada has made a significant breakthrough with its work in developing a small molecule PCSK9 inhibitor (PCSK9i), as published in the international peer-reviewed journal, Bioorganic and Medicinal Chemistry.
As published, Nyrada has successfully identified a small molecule that inhibits the production of PCSK9 in vitro and lowers total blood cholesterol levels in an animal model.
The journal noted that the success by the Australian chemists in identifying a binding site for a small molecule on the PCSK9 protein, where major pharma companies had failed.
It is a tremendous achievement to have our PCSK9i science validated by our peers. The paper confirms that Nyrada PCSK9i compounds lower blood cholesterol levels in an in vivo model and restore LDL- cholesterol capture by human cells,” said publication co-author, Nyrada Scientific Advisory Board member and PCSK9 pioneer, Prof Gilles Lambert of Laboratoire Inserm, France.
“This provides proof-of-concept that a small molecule inhibitor against PCSK9 is therapeutically viable.”
Nyrada CEO James Bonnar said that overcoming the binding challenge with the 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.
“The advance marks strong progress in the company’s strategy to develop an oral cholesterol lowering drug for patients that can be taken to assist the estimated 40% of individuals who do not respond adequately to statins.
Statins are the standard-of-care cholesterol-lowering treatment with a global market expected to exceed US$20 billion in 2020.
“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 oral combination therapy is expected to have broader patient acceptance over injectable PCSK9i options that currently are available.”
The paper is entitled ‘A small molecule inhibitor of PCSK9 that antagonises LDL receptor binding via interaction with a cryptic PCSK9 binding groove.’ This work forms the basis of the company’s intellectual property patent application currently under consideration by the US Patent Office.
Mr Bonnar said Since submitting the paper for publication, Nyrada has identified further small molecules with even greater potency to the compound described in the paper.
The science described in the publication, along with the new, more potent compounds, are the subject of a series of patent applications currently under examination.
The next step in the PCSK9i programme is to confirm the lead candidate based on PCSK9-binding potency and oral bioavailability. That will be the candidate to be taken through to the clinic.