LIB Therapeutics has submitted a Biologics License Application (BLA) to the U.S. FDA for Lerodalcibep, a novel PCSK9 inhibitor designed to lower LDL-C in patients with atherosclerotic cardiovascular disease (ASCVD) or at high risk for ASCVD, and primary hyperlipidemia, including familial hypercholesterolemia. Lerodalcibep is administered as a once-monthly, self-administered subcutaneous injection with long ambient stability, offering a potentially more convenient alternative to existing PCSK9 inhibitors. A Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) is planned for mid-2025.
This advancement is potentially transformative for cardiovascular disease management. Current oral therapies are often insufficient for patients to achieve optimal LDL cholesterol levels, leaving a significant unmet need. Lerodalcibep’s demonstrated robust and sustained LDL-C lowering capabilities, coupled with its convenient administration and storage, could significantly improve patient adherence to long-term treatment, a crucial factor in managing LDL cholesterol and reducing cardiovascular risk. This is particularly important for the substantial population of patients with familial hypercholesterolemia, who often struggle to manage their cholesterol levels effectively.
The BLA submission is based on data from a comprehensive development program involving 2,900 patients, encompassing five global Phase 3 studies. These studies included over 2,300 patients already on maximally tolerated statins and other oral agents but still requiring further LDL-C reduction. The trials evaluated Lerodalcibep’s safety and efficacy in patients with or at high risk for CVD, including those with heterozygous and homozygous familial hypercholesterolemia. Participants received once-monthly doses for up to 52 weeks in the placebo-controlled trials, with over 2,400 continuing into a 72-week open-label extension. Lerodalcibep is a third-generation PCSK9 inhibitor designed as a small-volume, subcutaneous injection with no refrigeration requirement. Its anti-PCSK9 binding domain, an 11-kDa polypeptide called an adnectin, is engineered for high-affinity binding and fused to human serum albumin to enhance plasma half-life.
The submission of the BLA for Lerodalcibep marks a significant step towards potentially reshaping the landscape of LDL-C management. If approved, Lerodalcibep’s patient-friendly profile could significantly improve adherence to therapy, leading to better long-term outcomes for patients with CVD or at high risk, particularly those with familial hypercholesterolemia. The anticipated EMA submission further underscores the potential for this treatment to address a substantial global need. The development of a more convenient and efficacious PCSK9 inhibitor may ultimately lead to wider adoption of this class of drugs and a greater impact on cardiovascular health worldwide.
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.