Verve Therapeutics Touts Positive Data From Gene Editing Medicine To Lower Cholesterol After Single Dose

Verve Therapeutics Inc VERV on Monday released initial data from the Heart-2 Phase 1b trial of VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD), which requires reducing low-density lipoprotein cholesterol (LDL-C) levels in the blood.

VERVE-102 was well-tolerated among 14 participants across three dose levels. No treatment-related serious adverse events (SAEs) and no clinically significant laboratory abnormalities were observed.

A single infusion of VERVE-102 led to dose-dependent decreases in blood PCSK9 protein (regulates cholesterol metabolism) levels and LDL-C, with a mean reduction in blood LDL-C of 53% and a maximum LDL-C reduction of 69% observed among four participants in the 0.6 mg/kg dose cohort.

VERVE-102 is a novel, in vivo, investigational base editing medicine designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver and durably reduces disease-driving LDL-C.

VERVE-102 comprises an adenine base editor and a guide RNA (gRNA) targeting the PCSK9 gene. Both are encapsulated in a lipid nanoparticle (LNP) and administered as a single intravenous infusion over two to four hours.

VERVE-102 has been well-tolerated, with no treatment-related SAEs, dose-limiting toxicities, or cardiovascular events observed.

Following a single infusion of VERVE-102, dose-dependent reductions in two pharmacodynamic (PD) measures were observed: blood LDL-C and PCSK9 protein levels.

Cohort 1; 0.3 mg/kg (n=4): LDL-C reduction was 21% and PCSK9 reduction was 46%.
Cohort 2; 0.45 mg/kg (n=6): LDL-C reduction was 41% and PCSK9 reduction was 53%.
Cohort 3; 0.6 mg/kg (n=4): LDL-C reduction was 53% and PCSK9 reduction was 60%.

Across all 14 participants, VERVE-102 demonstrated a strong dose-dependent response between the amount of total RNA administered and LDL-C reductions.

The Heart-2 trial is enrolling participants in the fourth dose cohort of 0.7 mg/kg. Verve has dosed two participants in this cohort.

Verve expects to announce the final data from the dose escalation portion of the Heart-2 clinical trial, including durability data, in the second half of 2025.
Verve plans to dose the first patient in the Phase 2 clinical trial of VERVE-102 in the second half of 2025, subject to regulatory clearance.

Verve expects its current capital position to be sufficient to fund its operations into mid-2027, which includes the completion of the Phase 2 clinical trial.

Under the PCSK9 program collaboration agreement with Verve, Eli Lilly And Co LLY holds the right to opt-in to share worldwide development expenses (33% contributed by Lilly) and to jointly commercialize and share profits and expenses related to commercialization in the U.S. on a 50/50 basis.

Verve retains control of the development and commercialization of all collaboration products in the U.S., and Verve holds all product rights outside the U.S.
Verve expects to deliver the opt-in package for the PCSK9 program and receive a decision from Lilly in the second half of 2025.

Last week, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to VERVE-102 for hyperlipidemia and high lifetime cardiovascular risk to reduce low-density lipoprotein cholesterol (LDL-C).

Price Action: VERV stock is up 13% at $3.69 at last check Monday.

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