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Injectable cholesterol-lowering drug developed with RNAi technology shows promise

UMass Chan researcher explains noteworthy trial results in NEJM Perspective Column

  Anastasia Khvorova

Anastasia Khvorova, PhD

Statin pills are the gold standard for treating high cholesterol, but  research published in the New England Journal of Medicine this month is generating excitement about a new class of cholesterol-lowering therapies based on RNAi technology.

In an accompanying editorial, Anastasia Khvorova, PhD, professor of molecular medicine and biochemistry & molecular pharmacology, explains the promise of oligonucleotide therapeutics, including the results of a Phase I clinical trial of a new injectable drug.

“This is noteworthy as the result describes an unprecedented duration of effect for the RNAi drug, and that’s why we are so enthusiastic about the data,” said Dr. Khvorova, who was not involved in the study.

The drug, inclisiran, was developed by Alnylam Pharmaceuticals, a company co-founded by Phillip Zamore, PhD, Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences and professor of biochemistry & molecular pharmacology.

Researchers tested inclisiran and found it cut low-density lipoprotein LDL, or bad cholesterol, by half or more. The drug prompts the liver to flush more LDL cholesterol out of the bloodstream by blocking a protein called PCSK9. According to the early data, inclisiran significantly reduced levels of PCSK9 and LDL cholesterol for six months following one single injection.

“The first generation of PCSK9 inhibitors required patients to received up to 24 injection year, which is expensive and inconvenient for the patient,” said Khvorova.

Researchers estimate patients would only need an injection of Inclisiran two to three times a year to control their cholesterol.

While the results are early, Khvorova said, “We are on the right path to changing the future of how we treat bad cholesterol.”