Discovery of high affinity beta-secretase inhibitors using fragment-based lead generation and structure-based design

MEDI 243

Jeffrey S. Albert, jeffrey.albert@astrazeneca.com, CNS Discovery Research, AstraZeneca Pharmaceuticals, 1800 Concord Pike, PO Box 15437, Wilmington, DE 19850-5437
Beta-secretase is a leading target for the development of therapies to treat Alzheimer's disease. Despite intense research over the past decade, only recently have high-affinity drug-like inhibitors of beta-secretase emerged. Fragment based approaches to lead generation rely on identifying drug fragments that contain minimal functionality for binding to the target of interest. Using NMR methods, we screened a library of low molecular weight compounds to identify hits that bound to the active site of beta-secretase with affinities (IC50) of 1-5 mM. X-ray crystallography facilitated the rapid evolution of these weak hits into high affinity (IC50 <100 nM) drug leads.