14 Lepodisiran is a silencing RNA compound that targets apo(a) messenger RNA. It is being tested in a Phase 3 cardiovascular outcomes trial (ACCLAIM-Lp(a); NCT06292013) with an anticipated study completion date of March 2029. The trial recruited both secondary prevention and high-risk primary prevention subjects. Other Lp(a)-specific lowering drugs in the pipeline: There are several additional compounds that are in development.31 These include zerlasiran, another siRNA compound against apo(a) mRNA (Phase 2 completed; up to 96% Lp(a) lowering), and a small molecule (muvalaplin) that disrupts Lp(a) formation (Phase 2 completed; up to 86% lowering) and is in a Phase 3 cardiovascular outcomes trial (NCT07157774). Gene editing approaches targeting LPA for permanent lowering of Lp(a) have shown promise in preclinical studies32,33 and CTX320, a Cas9-based gene editor in lipid nanoparticles, is being tested in Phase 1 trails.34
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