Verve Therapeutics' VERVE-102 Granted FDA Fast Track for PCSK9 Base Editing Therapy

BOSTON, April 11, 2025 - Verve Therapeutics, a pioneering company in genetic medicines for cardiovascular conditions, has announced a significant advancement in its medical research. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VERVE-102. This investigational base editing therapy is aimed at patient populations with hyperlipidemia and a high lifetime risk of cardiovascular issues, specifically to lower low-density lipoprotein cholesterol (LDL-C).

VERVE-102 represents a novel in vivo treatment method, designed to be administered as a single course, which permanently deactivates the PCSK9 gene in the liver. This process significantly reduces levels of LDL-C, which is a major factor in cardiovascular diseases. Currently, the medicine is being assessed in the Phase 1b Heart-2 clinical trial. This trial focuses on evaluating the safety and tolerability of VERVE-102 in adult patients who suffer from heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) and still need further LDL-C reduction.

Dr. Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, pointed out the ongoing challenges with existing LDL-C lowering treatments, noting that nearly half of the patients discontinue their prescribed therapies within the first year. He emphasized the necessity for a treatment that ensures profound and long-lasting LDL-C reduction, which is crucial for transforming atherosclerotic cardiovascular disease management. Dr. Kathiresan highlighted the potential of VERVE-102 as a single-dose treatment option uniquely suited to meet this need, and expressed anticipation about collaborating with the FDA to bring this innovative therapy to patients.

The FDA's Fast Track designation is designed to expedite the development and review processes for drugs intended to treat serious or life-threatening conditions where there is an unmet medical need. This status allows for more frequent interactions and communications with the FDA and may enable rolling review of the marketing application. Additionally, drugs with Fast Track status may qualify for Priority Review if they meet specific conditions.

Verve Therapeutics is set to release demographic and preliminary safety and efficacy data from the Heart-2 clinical trial in the second quarter of 2025. This initial data will involve participants from the first three dosage groups (0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg), with a minimum follow-up of 28 days for each participant.

Furthermore, in the latter half of 2025, Verve plans to announce the final results of the dose escalation phase of the Heart-2 clinical trial. The company is also preparing to deliver an opt-in package for the PCSK9 program to Eli Lilly and Company and intends to start the Phase 2 clinical trial for this program.

Verve Therapeutics is focused on advancing genetic medicines for cardiovascular diseases, aiming to shift treatment paradigms from chronic management to gene-editing solutions. Their leading programs, including VERVE-102, target the primary contributors to atherosclerosis: LDL-C, remnant cholesterol, and Lp(a). Each program is tailored to address specific genetic factors driving cardiovascular disease, with VERVE-102 specifically designed to deactivate the PCSK9 gene for individuals with conditions such as HeFH and persistent high LDL-C levels.