Drugmaker Eli Lilly recently reported promising early results for an experimental gene-editing therapy that may reduce low-density lipoprotein (LDL), or “bad,” cholesterol levels with a one-time treatment.

The treatment, called VERVE-102, is designed to permanently turn off the gene PCSK9 in the liver. PCSK9 helps regulate LDL cholesterol levels, and reducing its activity can lower cholesterol and potentially reduce the risk of cardiovascular diseases such as heart attack and stroke.

A One-Time Treatment That Could Lower LDL Cholesterol Dramatically

The early trial included 35 adults with premature coronary artery disease (CAD). LDL cholesterol levels fell by an average of 51% among participants who received the lowest dose and by 88% among those who received the highest dose.

According to the researchers, cholesterol reductions were still observed in a subset of patients up to 18 months after treatment. 

“These are really exciting interim results,” said Riyaz S. Patel, MD, an author of the study and a professor of cardiology at University College London. “The treatment effect is as good as the best available therapies and, importantly, appears durable at least out to 18 months. This opens the door to a one-and-done future treatment paradigm.” 

In the trial, the drug was given as an intravenous (IV) infusion and was well tolerated across all dose levels, with no serious adverse events reported. The most common side effects were infusion-related reactions and fatigue.

“Many patients with elevated LDL-C struggle to achieve sustained control despite ongoing efforts with the medicines available today, putting them at significant risk for cardiovascular events. With coronary artery disease still one of the leading causes of death worldwide, the need for new approaches is real,” Patel said.

The latest VERVE-102 results come from a phase 1 trial, and Eli Lilly plans to begin a phase 2 study by the end of the year.

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Could This Be an Alternative to Daily Cholesterol Medications?

If future studies confirm the treatment’s safety and long-term benefits, VERVE-102 could represent a major shift in how high cholesterol is managed. Instead of taking daily cholesterol-lowering medications or receiving regular injections, some patients may eventually be able to lower their LDL cholesterol with a single treatment that provides years of benefit.

Roger Blumenthal, MD, a volunteer expert at the American Heart Association (AHA) and director of the Johns Hopkins Ciccarone Center for the Prevention of Cardiovascular Disease, said the concept makes sense especially for higher-risk people because “the lower you get your LDL-C, the more protection against cardiovascular disease you have.” 

“More data on VERVE-102 has to be accrued for safety and efficacy,” he added, “But the results so far are exciting, especially for people with really high LDL cholesterol levels.”

According to the AHA, about one in four adults has LDL cholesterol levels that are too high. Earlier this year, the AHA and the American College of Cardiology released updated cholesterol management guidelines that recommend earlier LDL cholesterol screening and, for the first time, tailor LDL cholesterol targets to a person’s individual cardiovascular risk.

The FDA has granted VERVE-102 Fast Track designation, which is intended to speed the development and review of promising treatments. Based on the typical clinical trial timeline, Blumenthal estimated the therapy could be ready for FDA review in about three to four years.

If approved, the treatment could appeal to people whose LDL cholesterol is well controlled with medications yet would prefer a long-lasting alternative to daily pills or regular injections, Blumenthal said. However, it won’t change the need for “healthy lifestyles including a healthy diet and regular exercise because those have implications for other health conditions, including helping to prevent cancer and cognitive decline,” he added.