The Food and Drug Administration has authorized Verve Therapeutics to expand its clinical trial for a novel gene editing treatment targeting high cholesterol into the United States, the biotechnology company announced on Monday.
The experimental therapy, known as VERVE-102, employs base editing technology to modify a single nucleotide in the PCSK9 gene, which plays a crucial role in how liver cells process blood cholesterol. This gene editing approach aims to provide a one-time treatment that could permanently lower cholesterol levels, addressing a significant risk factor for heart disease.
The treatment is being evaluated in patients diagnosed with
heterozygous familial hypercholesterolemia, an inherited condition characterized by persistently elevated cholesterol levels, as well as those with premature coronary artery disease. The ongoing Phase 1b clinical trial, dubbed Heart-2, has already been recruiting
participants in several countries including Australia, New Zealand, Canada, and the United Kingdom.
Currently approved PCSK9-targeting treatments, including antibody drugs and RNA medicines, require repeated administration ranging from biweekly to twice-yearly injections. Among these, Amgen’s Repatha emerged as the market leader, generating over $2.2 billion in revenue during 2024.
“Traditional cholesterol-lowering medications can effectively reduce LDL-C at individual time points, but patient adherence remains a significant challenge,” explained Sekar Kathiresan, Verve’s CEO. “Our approach aims to deliver sustained cholesterol reduction through a single treatment course, potentially offering more meaningful therapeutic benefits.”
Verve anticipates releasing initial safety and efficacy data from the Heart-2 trial before the end of June, with plans to publish dose escalation results and initiate a Phase 2 study later this year.
VERVE-102 represents an improved version of the company’s earlier treatment, VERVE-101, which faced setbacks last year after a trial participant developed concerning liver enzyme elevations and reduced blood platelet counts (thrombocytopenia). While both treatments share similar genetic modification mechanisms, VERVE-102 utilizes a different lipid nanoparticle delivery system. The company had previously suggested that the delivery component may have contributed to the complications observed with VERVE-101.
The development program has attracted significant pharmaceutical industry interest, with Eli Lilly maintaining an option to participate in VERVE-102’s development costs and profit sharing following the Heart-2 trial results. However, Verve recently experienced a setback when Vertex Pharmaceuticals terminated their research collaboration in February.
The FDA clearance news sparked investor optimism, with Verve’s stock price rising up to 8% during Monday morning trading, partially recovering from losses incurred following the Vertex partnership dissolution announcement.
Beyond VERVE-102, the company has disclosed two additional base editing therapies in its pipeline, with one already advancing to Phase 1 clinical testing. This expansion of gene editing technologies into cardiovascular disease treatment represents a significant step forward in the field, potentially offering new solutions for patients with genetic cholesterol disorders who currently rely on frequent medication administration.