New cholesterol therapy shows promising results in phase I trial

Results from a phase I trial conducted by Alnylam Pharmaceuticals suggest that a new drug class may be available to help manage patients’ cholesterol levels.

In this trial, 70 patients with hypercholesterolemia received either placebo or various doses of the study medication, inclisiran, via single-dose or multiple-dose injection. A significant reduction in LDL-C (“bad cholesterol”) was observed in patients who received 300mg or more of inclisiran in both single-dose and multiple-dose regimens which lasted for at least six months; no serious adverse events with inclisiran were reported.

A phase II trial is currently planned that will compare inclisiran to a similar drug already on the market, Repatha.

How does it work?

Inclisiran belongs to a newer class of drugs called oligonucleotides, using small interfering RNA (siRNA) to prevent your cells from making proteins. The DNA in your cells contains instructions (genes) which are used to synthesize or “build” proteins. These instructions are first “converted” (a process known as transcription) to a single-stranded molecule called messenger RNA (mRNA). Your cells are then able to use the mRNA to create specific proteins (a process known as translation). Inclisiran works by preventing your cells from creating a specific protein by destroying the mRNA before it can be translated. Inclisiran binds to the mRNA and causes your cells to degrade the mRNA before the protein is synthesized.

Specifically, inclisiran targets the mRNA for the protein PCSK9. This protein is also the target of Repatha. Repatha works by inhibiting the function of PCSK9, instead of preventing PCSK9 from being synthesized like inclisiran. PCSK9 is an important target in cholesterol therapy because it prevents your body from removing LDL-C (“bad cholesterol”) from the bloodstream. By inhibiting the function of PCSK9 (Repatha) or preventing its synthesis (inclisiran), more LDL-C can be removed from the bloodstream.

About Anthony Faso

Anthony Faso works as a clinical research coordinator in the United States. He received his B.S. in Biology with a minor in Chemistry from the University of South Carolina Upstate, where he volunteered as an undergraduate research assistant studying small mammal populations. In his free time, he enjoys riding bikes, eating ice cream, and spending time with his wife and cat.

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