US drugmaker Eli Lilly has reported promising results for a new gene therapy called VERVE-102 that could 'permanently' lower LDL, or 'bad', cholesterol.
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In an early-stage trial, one infusion of the therapy resulted in reduced PCSK9 levels by up to 88% and LDL cholesterol by up to 62% in patients at high risk of heart disease. The effects remained strong for up to 18 months after treatment, according to a press release shared by the company on Monday.
How VERVE-102 Works?
Explaining the mechanism, the company said: “VERVE-102 is designed to mimic the protective effect of naturally occurring loss-of-function variants in PCSK9, which are associated with markedly lower lifetime risk of coronary heart disease.”
It explained that VERVE-102 is a gene-editing therapy made of messenger RNA that encodes an adenine base editor along with a guide RNA targeting the PCSK9 gene. These components are packaged inside lipid nanoparticles (LNPs). The treatment is given as a single intravenous infusion lasting about four hours.
If the results hold true, the therapy could become a one-time treatment for high cholesterol and help prevent cardiovascular disease. Eli Lilly plans to begin Phase 2 testing later this year.
Details Of Early Study:
In the Phase 1b Heart-2 trial, Eli Lilly's VERVE-102 showed strong cholesterol-lowering effects after a single intravenous infusion. The study involved 35 adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD).
“These data were presented as a late-breaking oral presentation at the European Atherosclerosis Society (EAS) Congress and simultaneously published in The New England Journal of Medicine,” it said.
In the study, a single infusion of VERVE-102 led to PCSK9 levels dropping between 51% and 88%, while LDL-C reductions ranged from 9% to 62% depending on dose, the statement added.
"These early data give us encouraging evidence that in vivo base editing of PCSK9 may offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment," Riyaz S. Patel, M.D., cardiologist at Barts Health NHS Trust and professor of cardiology at University College London, was cited in Lily's press release.
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According to the company, VERVE-102 was generally well tolerated at all tested doses in the study. This means that no serious treatment-related side effects or dose-limiting toxicities were reported. Some participants experienced mild infusion-related reactions and fatigue. However, all participants were able to complete the full planned dose, and nobody withdrew from the trial due to side effects.
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