The pharma’s $1.3 billion bet on the base editor last year when it acquired Verve Therapeutics was rewarded by a significant, long-lasting decrease in disease markers in a Phase 1b trial of a heterozygous familial hypercholesterolemia (HFH) patient population.
These data, disclosed in a May 25 release, mark Lilly’s “first Ph1b gene therapy success following several acquisitions of emerging gene therapy companies,” Leerink Partners told investors in a Monday evening note. The results paint an efficacy profile “supporting its potential as a one-time treatment for hypercholesterolemia.”
Lilly is enrolling patients in its ongoing Heart-2 Phase 1b trial to evaluate use of its base editor VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) who require additional treatment to achieve even lower LDL-C levels. Results from this population showed a single dose of 1mg/kg of VERVE-102 reduced LDL-C by up to 62% (presented Monday).
Reducing the dose of VERVE-102 also reduced LDL-C levels, although to a lesser extent.
Patients who received a single dose of VERVE-102 also had a decrease in PCSK9 levels ranging from 51% to 88% of the amount of circulating protein in the bloodstream that regulates the number of LDL-C receptors in the body; the more receptors present, the more LDL-C is removed from the bloodstream. There is a relationship between high levels of PCSK9 and high cholesterol levels. VERVE-102, which is packaged in a lipid nanoparticle for intravenous delivery, works specifically to deactivate the PCSK9 gene, according to Lilly’s Monday release. Lilly is moving VERVE-102 into mid-stage development, with a Phase 2 study set to start before the year ends.
Lilly’s $1.3 billion acquisition of Verve in June 2025 sparked doubts among analysts, who argued that there doesn’t appear to be a commercial rationale for gene therapies in the area of cholesterol reduction.
BMO Capital Markets wrote to investors on June 16, 2025, saying that they are skeptical about the actual market opportunity for other genetic medicines in these indications. “It’s in our thinking that perhaps there may be more value in investing in the company at this time.”
BMO doubled down on its apprehensions, telling investors that “questions remain around the true need for gene editing in patients with high cholesterol.”
In this context, the analysts wonder what the opportunity for gene editing would look like with effective lipid-lowering drugs that are now approved or soon will be approved for injection or oral use. Nevertheless, they acknowledged the possibility of “a clear synergistic area of development” with lipid-lowering programs, as “Lilly has strong expertise in the field of the cardio-metabolic area.”
While BMO didn’t explicitly identify the other companies in the lipid-lowering arena, the game includes some of the industry’s biggest players such as Merck, which is working on the oral drug enlicitide, and Amgen, which has the FDA-approved antibody Repatha.
Verve Demonstrates Promising Cholesterol-Lowering Results
Early-stage clinical research indicates that Verve is making significant progress in the development of next-generation cholesterol treatments. The company’s innovative approach focuses on gene-editing technology designed to reduce LDL cholesterol levels with a single treatment, potentially offering long-term cardiovascular benefits.
How Verve’s Technology Works
The Verve strategy targets genes involved in cholesterol regulation, particularly those linked to the production of PCSK9 proteins. By modifying these genetic pathways, Verve aims to provide sustained reductions in LDL cholesterol, commonly known as “bad cholesterol.” This approach differs from traditional therapies that often require lifelong medication.
Encouraging Early Clinical Findings
Recent trial data suggest that Verve has achieved substantial LDL cholesterol reductions among participants receiving higher treatment doses. Researchers also observed significant decreases in PCSK9 protein levels, supporting the effectiveness of the underlying gene-editing platform. These findings strengthen confidence in the potential of Verve to address cardiovascular disease risk factors.
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