FDA grants fast track status to Verve's cholesterol drug

Published 11/04/2025, 12:06
FDA grants fast track status to Verve's cholesterol drug

BOSTON - The U.S. Food and Drug Administration (FDA) has awarded Fast Track designation to Verve Therapeutics' (NASDAQ:VERV) VERVE-102, a gene-editing treatment aimed at patients with hyperlipidemia and a high lifetime risk of cardiovascular disease. The biotech company, currently valued at $271 million, has seen its stock trading near 52-week lows at $3.05, though InvestingPro analysis suggests the shares may be undervalued. The designation is intended to speed up the development and review process for drugs that show the potential to address unmet medical needs in serious or life-threatening conditions.

VERVE-102 is an in vivo base editing medicine designed to permanently inactivate the PCSK9 gene in the liver, which could result in a durable reduction of low-density lipoprotein cholesterol (LDL-C), a known contributor to cardiovascular disease. Currently, VERVE-102 is undergoing evaluation in the Phase 1b Heart-2 clinical trial, focusing on safety and tolerability in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) who require additional LDL-C lowering.

Dr. Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, highlighted the necessity of sustained LDL-C lowering, citing that nearly half of patients stop taking prescribed LDL-C-lowering therapy within a year. Verve aims to transform treatment with what they believe to be a one-dose solution to this critical healthcare challenge. The company maintains a strong financial position with a current ratio of 12.65, indicating robust liquidity to fund its research programs. According to InvestingPro data, the company holds more cash than debt on its balance sheet, providing important runway for its development activities.

The Fast Track designation may lead to more frequent communications with the FDA and a rolling review of marketing applications. If certain criteria are met, it could also result in a Priority Review.

Verve anticipates releasing demographic and initial safety and efficacy data from the Heart-2 trial in the second quarter of 2025, including results from the first three dose cohorts. The company also plans to report final data for the dose escalation portion of the Heart-2 trial, deliver the opt-in package for the PCSK9 program to Eli Lilly and Company, and initiate the Phase 2 clinical trial for the PCSK9 program in the second half of 2025. Recent InvestingPro data shows four analysts have revised their earnings estimates upward for the upcoming period, suggesting growing confidence in the company's development pipeline. Discover more insights and 10+ additional ProTips about VERV with an InvestingPro subscription.

Verve Therapeutics (NASDAQ:VERV) is developing a new class of genetic medicines for cardiovascular disease, with the goal of moving from chronic therapies to single-course gene editing treatments. Their lead programs, including VERVE-102, target the main cholesterol drivers of atherosclerosis.

The information in this article is based on a press release statement.

In other recent news, Verve Therapeutics has made significant strides with its gene-editing drug, VERVE-102, as the U.S. Food and Drug Administration (FDA) granted clearance for its Investigational New Drug (IND) application. This approval allows Verve to proceed with U.S. sites for its Phase 1b clinical trial, named Heart-2, which is focused on treating heterozygous familial hypercholesterolemia (HeFH) and premature coronary artery disease. The FDA's clearance follows interim data that reported no treatment-related serious adverse events, a promising sign for the drug's safety profile. Analysts from BMO Capital Markets and Canaccord Genuity have both reiterated positive ratings on Verve, with price targets of $30 and $32, respectively, citing optimism about the drug's benefit/risk profile and its potential impact on cardiovascular diseases. H.C. Wainwright also raised its price target for Verve to $15, expressing confidence in the upcoming second-quarter data release, which could be pivotal for the company. These developments come amid broader concerns in the biotech sector following the resignation of the FDA's top vaccine official, Peter Marks, which has caused significant market reactions. Despite this shakeup, Verve's progress with VERVE-102 continues to attract attention from investors and analysts alike. The company's advancements are closely watched, especially with the anticipation of positive data readouts in the coming months.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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