Verve Therapeutics has received FDA Fast Track designation for VERVE-102, a gene editing medicine designed as a single-course treatment for hyperlipidemia in high cardiovascular risk patients. The therapy targets the PCSK9 gene in the liver to permanently lower LDL-C, currently being evaluated in the Heart-2 Phase 1b clinical trial for patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD). This designation underscores the urgent need for more effective and convenient LDL-C lowering treatments.
High LDL-C remains a significant driver of cardiovascular disease, and current therapies often face adherence challenges, leaving a substantial unmet need. A single-course treatment like VERVE-102 could revolutionize cardiovascular disease management by offering a durable solution that bypasses the limitations of chronic medication regimens. This approach may significantly improve long-term patient outcomes and reduce the burden of cardiovascular disease on the healthcare system. The Fast Track designation also validates the potential of gene editing technologies to address complex chronic diseases.
VERVE-102 is currently in Phase 1b trials, with initial safety and efficacy data expected in the second quarter of 2025. This data will include results from the first three dose cohorts, offering insights into the therapy’s potential. Verve also plans to release the full dose escalation data in the second half of 2025, along with initiating a Phase 2 trial and providing an opt-in package for the PCSK9 program to Eli Lilly and Company.
The Fast Track designation for VERVE-102 significantly accelerates its development timeline, potentially leading to earlier market availability. Positive clinical trial results could solidify base editing as a viable therapeutic strategy for cardiovascular disease and encourage further development of gene editing medicines for other chronic conditions. This advancement represents a potential paradigm shift in how cardiovascular disease is treated, moving from continuous management to a potentially curative approach.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
