Lepodisiran is an experimental small interfering RNA (siRNA) therapy developed by Eli Lilly and Company, designed to significantly lower lipoprotein(a) [Lp(a)] levels—a genetically inherited risk factor for cardiovascular diseases such as heart attacks and strokes.
In a Phase II clinical trial, lepodisiran demonstrated remarkable efficacy. Participants administered a 400 mg dose experienced an average reduction in Lp(a) levels by 93.9% over a six-month period. Notably, the effects were durable, with reductions maintaining at 91% after one year and 74.2% after 1.5 years post-treatment. The study also reported that lepodisiran was well-tolerated, with no serious adverse events related to the drug.
These findings offer hope for individuals with elevated Lp(a) levels, a condition affecting approximately 20% of the global population, for whom current treatment options are limited. Eli Lilly is advancing lepodisiran into late-stage clinical trials to further evaluate its impact on cardiovascular outcomes.