The BACE1 enzyme has a rate-limiting role in the amyloidogenic pathway (see Glossary) and has been extensively studied for its neuronal functions[1]. Since 2000, intensive efforts have focused on developing small-molecule BACE1 inhibitors to reduce amyloid β (Aβ) production in Alzheimer’s disease (AD) brains. However, human clinical trials involving most BACE1 inhibitors were stopped at Phase 2/3 due to limited therapeutic benefits[2]. BACE1 inhibitors act by reducing Aβ-related pathologies in AD brains, that is, they are used to treat the symptoms rather than the underlying disease.
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