The Food and Drug Administration has authorized Verve Therapeutics to extend its gene editing clinical trial into the United States, marking a significant expansion for the company’s cholesterol treatment program.
The experimental therapy, VERVE-102, is designed to modify a single nucleotide in the PCSK9 gene, which plays a crucial role in regulating how liver cells process blood cholesterol. This approach aims to provide a permanent solution for cholesterol management,
distinguishing it from current treatments that require repeated administration.
Currently, several approved medications target PCSK9, including antibody treatments and RNA-based therapies. Among these, Amgen’s Repatha has emerged as a market leader, generating over $2.2 billion in revenue during 2024. However, these existing treatments necessitate regular injections ranging from biweekly to twice yearly.
VERVE-102 represents a potentially transformative approach, offering the possibility of sustained cholesterol reduction through a one-time treatment that permanently deactivates the PCSK9 gene in liver cells. The therapy is being evaluated in patients with heterozygous familial hypercholesterolemia, a genetic condition causing persistent high cholesterol, and those with premature coronary artery disease.
According to Sekar Kathiresan, Verve’s CEO, while current medications can effectively lower cholesterol levels at specific points in time, patient adherence remains problematic. “Verve’s medicines are designed to deliver lifelong cholesterol lowering after a single course of treatment and, consequently, drive more meaningful efficacy,” he explained.
The company’s Phase 1b trial, dubbed Heart-2, has been ongoing in multiple countries including Australia, New Zealand, Canada, and the United Kingdom. Initial safety and efficacy data are expected to be released before the end of June, with dose escalation results and Phase 2 trial initiation planned for later this year.
VERVE-102 succeeds an earlier version, VERVE-101, whose trial was suspended following complications in a study participant who developed elevated liver enzymes and reduced blood platelet counts. While both treatments share similar mechanisms for PCSK9 gene modification, VERVE-102 employs a different lipid nanoparticle delivery system, as the previous version’s delivery method was suspected to have contributed to the adverse effects.
The company has formed a strategic partnership with Eli Lilly, which maintains the option to participate in development costs and profit sharing for VERVE-102 after reviewing Heart-2 trial data. Following the FDA’s announcement, Verve’s stock value increased by up to 8% on Monday morning, helping to offset recent losses stemming from Vertex Pharmaceuticals’ February decision to terminate their partnership.
The development of VERVE-102 represents part of a broader portfolio of base editing therapies at Verve, with one additional treatment already advancing to Phase 1 testing. This expansion into U.S. clinical trials marks a significant milestone for the company’s gene editing program and its mission to develop long-lasting solutions for cardiovascular disease management.
The advancement of this trial into the United States exemplifies the growing momentum in genetic medicine development, particularly in addressing chronic conditions that traditionally require ongoing treatment. If successful, VERVE-102 could potentially revolutionize how high cholesterol is treated, offering patients a single-treatment alternative to current therapeutic options.