FDA Approves Third-Generation PCSK9 Inhibitor for LDL Reduction

“We founded LIB Therapeutics with a mission to bring an effective and more patient-friendly treatment option to the millions of patients who still need additional LDL-C reduction in order to achieve the lower targets recommended in recent guidelines. Today’s FDA approval validates that mission and the focus of my medical career,” said Evan Stein, M.D., Ph.D., Founder, CEO and Chief Scientific Officer of LIB Therapeutics. “LEROCHOL was designed for patients who need life-long treatment to achieve and maintain the new lower LDL-C goals especially those with cardiovascular disease and the millions with inherited high cholesterol (familial hypercholesterolemia or FH). We look forward to bringing LEROCHOL to patients in the coming months. I wish to thank the more than 2,500 patients throughout the world who participated in our clinical trials and our small team of very dedicated colleagues at LIB who believed in the drug and worked tirelessly for nearly 10 years, including during the COVID-19 pandemic, to achieve this milestone. I would also like to express our thanks to the FDA Division of Diabetes, Lipid Disorders, and Obesity for their thoroughness, guidance and flexibility during the development and review process and during the government shutdown.”

Cardiovascular disease (CVD) remains the leading cause of death globally and in the United States, and lower LDL-C levels have been demonstrated to lead to better outcomes. Despite the availability of statins and other lipid-lowering therapies, millions of patients with or at risk of CVD, including those with FH, fail to achieve updated guideline-recommended LDL-C targets. Approximately 1 in 200 individuals worldwide have FH, a genetic condition characterized by severely elevated LDL-C levels from birth that requires lifelong management. PCSK9 inhibitors as a class offer the most effective and safe LDL-C lowering. However widespread adoption has been limited by inconvenience and access barriers, leaving millions of patients with a need for additional treatment options.

“The primary challenge in lipid management today is helping patients achieve and maintain the increasingly stringent LDL-C targets in current guidelines,” said Dean J. Kereiakes, M.D., FACC, MSCAI, Chairman of The Christ Hospital Heart and Vascular Institute and Professor of Medicine at the University of Cincinnati. “While PCSK9 inhibitors as a class deliver powerful cholesterol lowering potential, LEROCHOL was designed to address the barriers that have limited their use, including ease of use features like a single small monthly injection, self-administered at home with extended room-temperature stability for home storage and travel. For patients with cardiovascular disease who require lifelong cholesterol management, LEROCHOL offers an important addition to our treatment options.”

About the LIBerate Clinical Trial ProgramTM

The FDA approval of LEROCHOL was based on data from the comprehensive global Phase 3 LIBerate Clinical Trial Program, which enrolled a diverse population of over 2,900 patients with CVD, without CVD at very high and high risk for CVD, including heterozygous and homozygous familial hypercholesterolemia. Lerodalcibep was dosed once monthly for up to 52 weeks in these key registration-enabling, placebo-controlled trials, and over 2,400 patients continued in the 72-week open-label extension trial.

In clinical trials, LEROCHOL demonstrated sustained LDL-C reductions of ≥60% in patients with, or at very high or high risk of, cardiovascular disease and ≥50% in those with HeFH who have more severe LDL-C elevations. LEROCHOL was generally well tolerated across the LIBerate Clinical Trial Program, with no treatment-related serious adverse events reported in the long-term extension studies.

About LEROCHOLTM (lerodalcibep-liga)

LEROCHOL is a novel, small protein-binding, third-generation PCSK9 inhibitor, and has been developed as a more convenient, once-monthly, single small-volume, subcutaneous injection with extended room temperature stability up to three months. These features make lerodalcibep a unique alternative to other PCSK9 inhibitors. The anti-PCSK9 binding domain of lerodalcibep is an 11-kDa polypeptide called adnectin, engineered for high-affinity subnanomolar binding to human PCSK9 and fused to human serum albumin to enhance plasma half-life.