Lilly reports new Phase 1b data on PCSK9 base‑editing gene therapy from Verve buyout

Eli Lilly has reported new Phase 1b Heart-2 trial data for its PCSK9 base‑editing gene therapy VERVE‑102, a one‑time in vivo therapy aimed at permanently turning off the PCSK9 gene in the liver to durably lower LDL cholesterol (LDL‑C). The data come from Lilly’s program inherited through its acquisition of Verve Therapeutics, which had been developing VERVE‑102 and VERVE‑101 for severe hypercholesterolemia and cardiovascular disease.

In an interim analysis of 35 participants, a single intravenous infusion of VERVE‑102 produced dose‑dependent mean reductions in circulating PCSK9 protein of 51% at the lowest 0.3 mg/kg dose up to 88% at the highest 1.0 mg/kg dose. Corresponding mean LDL‑C reductions were 9% (0.3 mg/kg), 44% (0.45 mg/kg), 45% (0.6 mg/kg), 33% (0.7 mg/kg), 51% (0.8 mg/kg), and 62% (1.0 mg/kg), with effects sustained for up to 18 months. Early safety data showed no treatment‑related serious adverse events and no clinically significant liver or hematologic abnormalities, raising the potential for VERVE‑102 as a once‑and‑done therapy for high‑risk patients with elevated LDL‑C.