An experimental drug developed by Eli Lilly, lepodisiran, has shown promising results in reducing a genetically inherited risk factor for heart disease, according to data presented at a prominent medical meeting, Reuters reports.
The drug, which targets elevated lipoprotein(a) or Lp(a), was shown to reduce its levels by an average of 93.9% in a midstage clinical trial, compared to a placebo. The trial involved 141 patients receiving one or two 400-milligram doses of lepodisiran, with 69 patients receiving a placebo.
Lp(a) is a genetic risk factor that can significantly increase the likelihood of heart attack, stroke, narrowing of the aortic valve, and peripheral artery disease. It affects approximately 1.4 billion people worldwide, including 64 million in the United States. Unlike LDL cholesterol, which can be controlled through diet and statins, there are currently no approved treatments for high levels of Lp(a), making it a significant unmet medical need. Additionally, many individuals are unaware they have high levels of this risk factor.
Lilly’s experimental drug, lepodisiran, works by significantly lowering Lp(a) levels with infrequent doses. Study author Dr. Steven Nissen, a cardiologist at the Cleveland Clinic, noted that no serious adverse events related to the drug were reported during the trial. The results were presented at the American College of Cardiology meeting in Chicago and also published in the New England Journal of Medicine.
Despite the drug’s impressive ability to lower Lp(a), large-scale trials are still necessary to determine whether reducing Lp(a) levels will translate into a reduction in heart attacks and other cardiovascular events. Lilly has already moved lepodisiran into late-stage clinical trials, with plans for a second Phase 3 trial to specifically assess whether lowering Lp(a) reduces the risk of cardiovascular issues. Enrollment for this trial is expected to be completed later this year.
In addition to lepodisiran, other treatments targeting Lp(a) are in development, including injectable therapies from companies like Silence Therapeutics, Amgen, and Novartis. Lilly is also testing muvalaplin, the only oral treatment for Lp(a) currently in clinical trials.
