A type of cholesterol that often evades routine tests and defies diet, exercise, and existing treatments may finally have a solution. In a groundbreaking trial, a single dose of the experimental drug lepodisiran slashed levels of lipoprotein(a), a deadly, hard-to-treat form of cholesterol linked to heart disease, for up to a year.

An estimated 64 million U.S. adults have dangerously high levels of lipoprotein(a), a form of cholesterol that can double or even triple the risk of heart attacks. Beyond fueling heart disease, elevated Lp(a) is also linked to strokes and aortic valve stenosis, a serious condition that narrows the heart valve and can lead to heart failure. Unlike other forms of cholesterol, Lp(a) is largely influenced by genetics and remains difficult to manage with lifestyle changes or existing medications.

The trial results published in the New England Journal of Medicine and presented at the American College of Cardiology meeting in Chicago reveal that even a single dose of lepodisiran, a drug from Eli Lilly, could provide long-term control over this deadly type of cholesterol.

"What we have is a drug that can lower lipoprotein(a) with very infrequent administration," said study author Dr. Steven Nissen, a long-time cardiologist at the Cleveland Clinic.

Lepodisiran works by shutting down the body's ability to produce Lp(a) at its source. It targets messenger RNA (mRNA), which carries the instructions for making this harmful cholesterol.

In the Phase 2 clinical trial funded by Lilly, 320 participants received a single injection of lepodisiran. Six months later, their Lp(a) levels had dropped by an impressive 93.9%. Even after a full year, the reduction remained substantial at 88.5%. For those who received a second dose at the six-month mark, the decline was even greater, reaching 94.8% after one year.

"Reducing the inherited cardiovascular risk for patients with high Lp(a) has long been a critically unmet need. These results offer hope for a long-term, durable treatment option," said Ruth Gimeno, vice president of diabetes, obesity, and cardiometabolic research at Eli Lilly, in a news release.

"These data underscore Lilly's commitment to advancing genetic medicine to address one of the world's most pressing healthcare challenges. We will continue to evaluate the potential benefits of lepodisiran in the ongoing Phase 3 cardiovascular outcomes trial," Gimeno added.

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