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VERVE-102 Shows Durable LDL Reduction in Heart-2 Study, Lilly Plans Phase 2 Launch

Eli Lilly has released early clinical results for its gene-editing candidate VERVE-102, highlighting the potential of a single-dose treatment to deliver sustained reductions in LDL cholesterol among high-risk patients.

The investigational therapy targets the PCSK9 gene in the liver, aiming to permanently reduce its activity and lower circulating cholesterol levels. In the ongoing Phase 1b Heart-2 study, VERVE-102 is being evaluated in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease who require additional LDL-C lowering despite existing treatments.

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Data from 35 participants show a clear dose-response relationship. LDL cholesterol reductions varied across dose cohorts, with decreases of 9% at 0.3 mg/kg, 44% at 0.45 mg/kg, 45% at 0.6 mg/kg, 33% at 0.7 mg/kg, 51% at 0.8 mg/kg, and up to 62% at the highest dose of 1.0 mg/kg. In parallel, PCSK9 protein levels declined substantially, reaching reductions of up to 88% at higher dose levels.

Notably, these effects were not short-lived. Follow-up data indicate that cholesterol lowering has been maintained for as long as 18 months in some patients, suggesting the possibility of a durable, one-time treatment approach.

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VERVE-102 uses an in vivo gene-editing strategy designed to replicate the protective effect seen in individuals with naturally occurring PCSK9 loss-of-function mutations, which are associated with lower lifetime cardiovascular risk. The therapy is delivered via lipid nanoparticles carrying genetic instructions that selectively target liver cells.

The Heart-2 trial is continuing to assess safety and long-term outcomes, with participants monitored over extended periods. Based on the current findings, Lilly plans to initiate a Phase 2 study later this year to further evaluate the therapy’s efficacy and safety profile.