Hyperlipidemia is a predominant risk factor for cardiovascular disease (CVD). Mutation in low-density lipoprotein receptor ( LDLR) or apolipoprotein B (ApoB) genes are major causes of familial hypercholesterolemia. Proprotein convertase subtilisin/kexin type 9 (PCSK9) was a third gene involved in this familial disease and plays an important role in the metabolism of low-density lipoprotein cholesterol (LDL-c). PCSK9 degrades the LDL-receptor, improves the lipoprotein profile and future cardiovascular outcomes in patients with dyslipidemia.
Recent Phase 2 clinical data (Amgen) obtained using antibodies to block its action show up to 60% reduction in levels of LDL-c. However, there are no single domain antibodies under development for this target. We believe this product could position itself quite competitively in the biological landscape; providing the specificity of monoclonal antibodies but at the fraction of the price.