Eli Lilly’s Lepodisiran Achieves 94% Reduction in Genetic Heart Disease Risk Factor in Phase 2 Trial

The siRNA-based drug shows promise as the first treatment for high lipoprotein(a) levels, with Phase 3 trials underway to assess its impact on cardiovascular events.

Lepodisiran, an experimental drug by Eli Lilly, reduced lipoprotein(a) (Lp(a)) levels by up to 94% in a Phase 2 trial involving 320 participants.
The drug works by targeting messenger RNA to inhibit the production of apolipoprotein(a), a key component of Lp(a).
High Lp(a) levels, a genetic risk factor for cardiovascular diseases, affect 1.4 billion people globally, including 64 million Americans.
No serious adverse events were reported in the trial, and the drug demonstrated sustained efficacy over a year with infrequent dosing.
Phase 3 trials are now enrolling participants to evaluate whether the drug’s Lp(a) reductions translate into fewer heart attacks and strokes.

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