A single 1-mg/kg dose of Eli Lilly’s base editor can lower LDL cholesterol levels by 62% in patients with a heritable form of hypercholesterolemia. The pharma acquired the asset last year in the $1.3 billion Verve Therapeutics buy.
Eli Lilly’s investigational base editor substantially and sustainably reduced disease biomarkers in a Phase 1b study of heterozygous familial hypercholesterolemia, vindicating the pharma’s $1.3 billion gamble on the modality last year when it acquired Verve Therapeutics.
These data, disclosed in a May 25 release, mark Lilly’s “first Ph1b gene therapy success following a number 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 testing its base editor, dubbed VERVE-102, in the ongoing Phase 1b Heart-2 trial, focused on patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) who need support to further lower their low-density lipoprotein cholesterol (LDL-C) levels. In this population, a single high dose (1 mg/kg) of VERVE-102 lowered LDL-C by as much as 62%, according to results presented on Monday.
Lower doses of VERVE-102 likewise decreased LDL-C levels, though the magnitudes of effect were weaker.
Patients given one infusion of VERVE-102 also saw a 51% to 88% reduction in PCSK9, a key protein that affects how many LDL-C receptors are present and active in the body. High PCSK9 levels are linked to higher cholesterol. VERVE-102, which is packaged in a lipid nanoparticle for intravenous delivery, works specifically to deactivate the PCKS9 gene, according to Lilly’s Monday release.
With Monday’s data, Lilly is moving VERVE-102 into mid-stage development, with a Phase 2 study set to start before the year ends.
When Lilly dropped $1.3 billion for Verve in June 2025, analysts expressed skepticism, contending that the commercial case is weak for gene therapies to lower cholesterol levels.
“We are skeptical about the true market need of additional genetic medicines in these indications,” BMO Capital Markets wrote to investors on June 16, 2025. “We think there may be better uses of capital for the company at this time.”
BMO doubled down on its apprehensions on Monday, telling investors that “questions remain around the true need for gene editing in patients with high cholesterol.”
“With effective injectable and oral lipid lowering agents approved or likely to be approved in the near term, we wonder what the opportunity for gene editing would really look like in this setting,” the analysts said. They nevertheless conceded lipid-lowering programs “could represent a clear synergistic area of development” to complement Lilly’s deep cardio-metabolic expertise.
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.
