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Shares of Verve Therapeutics VERV surged 26.4% on Monday and continue to soar today after the company announced positive initial data from an early-stage study of its investigational candidate, VERVE-102, which is being developed for reducing cholesterol levels. VERVE-102 is a novel, in vivo, investigational base editing medicine.
The patient population enrolled in the phase Ib Heart-2 study comprises individuals with heterozygous familial hypercholesterolemia and/or premature coronary artery disease — two groups that require substantial and sustained reductions in low-density lipoprotein cholesterol (LDL-C) levels.
The data readout includes 14 patients from the first three cohorts who received doses of VERVE-102 at 0.3 mg/kg, 0.45 mg/kg, or 0.6 mg/kg, respectively, with each participant having at least 28 days of follow-up as of the data cutoff date of March 13, 2025.
More on VERV’s Phase I Cholesterol Drug Study Data
Verve Therapeutics reported that a single infusion of VERVE-102 resulted in dose-dependent reductions in blood PCSK9 protein levels and LDL-C in the Heart-2 study, with an average LDL-C decrease of 53% and a maximum reduction of 69% observed among four participants in the 0.6 mg/kg dose cohort.
Year to date, VERV stock has plunged 27% compared with the industry’s 7.1% decline.
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In cohort 1 of the study, an LDL-C reduction of 21% and s PCSK9 reduction of 46% was observed in patients receiving 0.3 mg/kg of the candidate. Patients receiving 0.45 mg/kg achieved an LDL-C reduction of 41% and a PCSK9 reduction of 53%. In cohort 3 (0.6 mg/kg dose), LDL-C reduction was 53% and PCSK9 reduction was 60%.
Additionally, Verve Therapeutics reported that VERVE-102 was well-tolerated across all dose levels. There were no reports of treatment-related serious adverse events and no clinically significant changes in alanine aminotransferase, aspartate aminotransferase, bilirubin or platelet levels among the 14 participants.
VERV’s Next Steps in Developing the Cholesterol Drug
The Heart-2 clinical study is currently enrolling participants in the fourth dose cohort of 0.7 mg/kg across multiple regions. As of April 7, 2025, Verve Therapeutics has administered the dose to two participants in this cohort. Early laboratory and clinical safety findings are consistent with those observed in the first three cohorts. VERV anticipates releasing final data from the dose escalation phase of the Heart-2 study, including durability outcomes, in the second half of 2025.
VERV also plans to initiate dosing in the phase II clinical study of VERVE-102 for evaluating cholesterol-lowering ability in the second half of 2025. Following the recent clearance of its investigational new drug application by the FDA, the company expects to begin enrolling patients for this study. VERV believes its current capital resources will support operations through mid-2027, including the completion of the impending phase II study.
