Eli Lilly announced on May 25, 2026 that VERVE-102 produced dose-dependent and durable reductions in PCSK9 and LDL‑C in an interim analysis of the Phase 1b Heart-2 study.
In 35 adults with heterozygous familial hypercholesterolemia or premature coronary artery disease a single infusion of VERVE-102 cut mean PCSK9 levels by 51% to 88% and lowered LDL‑C by 9% to 62% across doses of 0.3 mg/kg, 0.45 mg/kg, 0.6 mg/kg, 0.7 mg/kg, 0.8 mg/kg and 1.0 mg/kg. The reductions were sustained for up to 18 months after treatment. VERVE-102 was well tolerated at every dose level; there were no treatment-related serious adverse events or dose-limiting toxicities, all participants received their full planned dose and no one withdrew from the study.
"These early data give us encouraging evidence that in vivo base editing of PCSK9 may offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment," said Riyaz S. Patel. He added: "Many patients with elevated LDL-C struggle to achieve sustained control despite ongoing efforts with the medicines available today, putting them at significant risk for cardiovascular events."
The Heart-2 results were presented as a late-breaking oral presentation at the European Atherosclerosis Society Congress and published simultaneously in The New England Journal of Medicine. VERVE-102 is an investigational in vivo base editing medicine designed to durably turn off the PCSK9 gene in the liver and lower blood LDL‑C following a single infusion. Lilly, which acquired the therapy in 2025 in a $1 billion purchase of Verve Therapeutics, said it plans to begin enrolling a Phase 2 clinical study of VERVE-102 by the end of 2026. The company positions the program as intended to broadly prevent heart disease in patients who struggle with conventional cholesterol-lowering medicines.
Geneticists have long pointed to PCSK9 loss-of-function variants as proof that life‑long suppression of the gene protects against heart attack. "Twenty years ago, genetics showed us that people born with PCSK9 naturally turned off have low LDL-C for life and are remarkably protected from heart attack, yet today's chronic therapies struggle to deliver this lifelong lowering," Sekar Kathiresan said. "The Heart-2 results provide early clinical evidence that a single dose of VERVE-102 may mimic the LDL-C lowering effects of PCSK9 cardioprotective variants, potentially transforming cardiovascular care from chronic management to a one-time treatment."
The data carry immediate promise but also clear limits. This was a small, 35‑participant interim analysis and LDL‑C responses varied widely across the tested dose range, with some dose groups showing more modest reductions. The safety profile to 18 months is reassuring, but longer follow-up and a larger, more diverse population will be required to understand rare risks and how durable the effect will be beyond what has been observed so far.
The most consequential question now is whether the Phase 2 trial, set to begin enrollment by the end of 2026, will confirm that a single in vivo base-editing infusion can reproducibly deliver substantial, long-term LDL‑C lowering and a safety profile strong enough to support replacing chronic lipid-lowering therapies with a one-time treatment.
