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1. (WO2017034994) COMPOSITION AND METHODS OF USE OF NOVEL PHENYLALANINE SMALL ORGANIC COMPOUNDS TO DIRECTLY MODULATE PCSK9 PROTEIN ACTIVITY

Pub. No.:    WO/2017/034994    International Application No.:    PCT/US2016/047810
Publication Date: Fri Mar 03 00:59:59 CET 2017 International Filing Date: Sat Aug 20 01:59:59 CEST 2016
IPC: A61K 38/17
Applicants: PORTOLA PHARMACEUTICALS, INC.
Inventors: BARTA, Thomas, E.
BOURNE, Jonathan, William
MONROE, Kyle, D.
MUEHLEMANN, Michael, M.
PANDEY, Anjali
BOWERS, Simeon
Title: COMPOSITION AND METHODS OF USE OF NOVEL PHENYLALANINE SMALL ORGANIC COMPOUNDS TO DIRECTLY MODULATE PCSK9 PROTEIN ACTIVITY
Abstract:
This invention is related to the field of PCSK9 biology and the composition and methods of use of small organic compounds as ligands for modulation of PCSK9 biological activity. In particular, the invention provides compositions of small organic compounds that modulate circulating levels of low density lipoproteins by altering the conformation of the protein PCSK9. Binding these small organic compound ligands to PCSK9 alters the conformation of the protein, modifying the interaction between PCSK9 and an endogenous low density lipoprotein receptor, and can lead to reduced or increased levels of circulating LDL-cholesterol. High LDL- cholesterol levels are associated with increased risk for heart disease. Low LDL-cholesterol levels may be problematic in other conditions, such as liver dysfunction; thus, there is also utility for small organic compound ligands that can raise LDL levels.