By Mackenzie Tatananni
Eli Lilly shares were little changed after the pharmaceutical giant reported promising early data for an experimental heart-disease gene therapy.
The pharmaceutical giant said Monday that a single infusion of its gene therapy candidate, Verve-102, reduced PCSK9 protein levels by up to 88% and lowered LDL cholesterol by as much as 62%.
PCSK9 is a protein that regulates cholesterol levels in the bloodstream. Raised levels of the protein indicate that the body is clearing less cholesterol from the blood, leading to high LDL cholesterol, which is linked to heart disease and stroke.
Lilly is targeting patients with premature coronary artery disease and a genetic condition called heterozygous familial hypercholesterolemia (HeFH), which occurs when a patient inherits a faulty cholesterol-regulating gene from one parent. Having HeFH dramatically increases the risk of early-onset heart disease.
The results look promising, but Verve-102 is only in Phase 1b studies, meaning it's too early to draw conclusions about its efficacy. These studies follow initial safety testing in healthy volunteers, and are typically the first time an investigational treatment is administered to patients who actually have the targeted disease.
This could be part of the reason shares barely budged in premarket trading Tuesday. Lilly stock rose 1.4% as S&P 500 futures ticked up 0.7%.
Still, the Food and Drug Administration has already placed the treatment on a fast track for approval. Lilly says it plans to initiate a Phase 2 study by the end of the year.
The trial aligns with Lilly's push to embrace in vivo therapies, a faster and cheaper alternative to ex vivo therapies that involve removing a patient's cells, altering them in a lab, and reintroducing them to the body.
Verve-102 is a base editing medicine that switches off a specific gene in the liver by chemically altering a single nucleotide in a patient's DNA. It poses a less invasive alternative to traditional CRISPR-Cas9 therapy, which cuts both strands of the DNA double helix.
In vivo therapies have become a major priority for Lilly. Just last month, Lilly struck an agreement to acquire Kelonia Therapeutics, the maker in vivo gene therapies, for $3.25 billion upfront. Verve-102 itself was developed by Verve Therapeutics, a Boston-based drugmaker Lilly purchased in July 2025.
Many investors are focused on Lilly's portfolio of weight-loss drugs, a catchall term for its oral and injectable GLP-1 medications, but the company has been on an acquisition spree in a bid to deepen its leadership in other fields.
The company announced Tuesday that it had agreed to acquire a trio of vaccine developers -- Curevo, LimmaTech Biologics, and Vaccine Co. -- to broaden its infectious disease portfolio.
Write to Mackenzie Tatananni at mackenzie.tatananni@barrons.com
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May 26, 2026 08:00 ET (12:00 GMT)
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