LIB Therapeutics‘ Phase 3 LIBerate-HoFH study results for lerodalcibep, a novel PCSK9 inhibitor, in patients with homozygous familial hypercholesterolemia (HoFH) were published in Lancet Diabetes & Endocrinology. The study, a randomized, crossover trial, compared lerodalcibep to evolocumab in a diverse global population of HoFH patients aged 10 and older. The primary endpoint was the percent change from baseline in LDL cholesterol (LDL-C).
These results are critical for the HoFH patient population, which faces exceptionally high LDL-C levels and significantly increased risk of premature cardiovascular disease. Current treatment options often prove insufficient, highlighting the urgent need for new therapies. This study provides valuable data on a potential new therapeutic approach, specifically in a diverse and global cohort, offering insights that could improve treatment strategies and outcomes.
The study found that mean LDL-C reduction with lerodalcibep was -9.1% and -10.8% with evolocumab when averaged across monthly visits. At week 12, reductions were -12% and -11.5% respectively, and at week 24, -4.9% and -10.3% respectively. While there was significant variability in individual patient responses, the percent change in LDL-C over 24 weeks correlated well between the two drugs. Interestingly, genotyping and free PCSK9 suppression did not predict treatment response. Both drugs showed good tolerability profiles, with no treatment-related serious adverse events.
This study represents a crucial step forward in the development of lerodalcibep as a potential treatment option for HoFH. While the LDL-C reductions observed were less than initially hoped, the data contributes substantially to the understanding of PCSK9 inhibition in this challenging patient population. The findings underscore the need for personalized approaches to HoFH treatment and emphasize the importance of continued research in this area. The data also supports the potential use of lerodalcibep as a convenient, monthly injectable therapy for HoFH, potentially expanding the available options for patients. The company’s recent BLA submission to the FDA signifies their commitment to bringing this therapy to market and potentially improving the lives of individuals with HoFH and other forms of hyperlipidemia.
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.
