The study enrolled 320 patients, dividing them into three dosing groups receiving a single dose of either 16mg, 96mg, or 400mg at the point of baseline measurement. Credit: Shutterstock / Dmytro Zinkevych.
Eli Lilly has reported promising Phase II results for lepodisiran, its experimental therapy aimed at reducing lipoprotein(a) \[Lp(a)\], a genetically inherited risk factor for heart disease.
The study demonstrated that the therapy significantly reduced Lp(a) by an average of 93.9%, with the highest tested dose of 400mg for 60 to 180 days, meeting the primary endpoint.
The findings were presented as part of the American College of Cardiology 2025 Scientific Sessions in Chicago, US.
Lp(a) is a form of lipoprotein capable of carrying cholesterol through a patient’s blood, high levels of which can be genetically inherited and is a common risk factor in conditions such as heart disease. At the same time, high levels of Lp(a) can double or even triple the risk of a heart attack according to research published by Harvard Medical School.
Ruth Gimeno, vice president of diabetes, obesity and cardiometabolic research at Eli Lilly, said: “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.
“This data underscores 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 III cardiovascular outcomes trial.”
In the Phase II ALPACA trial, treatment-emergent adverse events related to lepodisiran occurred in 14% of the 400mg group and 12% of the 16mg group. A single death was observed in the 16mg dose group due to complications of chronic coronary disease.
Research by GlobalData estimates that if the therapy goes to market, it could generate sales of up to $25m by the end of 2028, with that figure expected to grow to $100m by the end of 2031.
GlobalData is the parent company of _Clinical Trials Arena._