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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.

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Eli Lilly logo is shown on one of the company's offices in San Diego, California, U.S., September 17, 2020.
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Overview

  • 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.