umibecestat

Umibecestat (CNP520) is an oral small-molecule inhibitor of beta-secretase 1 (BACE1) developed by Novartis in collaboration with Amgen and the Banner Alzheimer's Institute. It was designed to reduce the production of amyloid-beta (Aβ) peptides by inhibiting the enzyme responsible for the initial cleavage of amyloid precursor protein (APP). The drug was primarily investigated for the prevention of Alzheimer's disease in cognitively unimpaired adults at high risk due to the APOE4 genotype. In 2019, the Phase 2/3 Generation Program was terminated after interim analyses revealed that umibecestat was associated with a worsening of cognitive function, increased brain atrophy, and weight loss compared to placebo. Subsequent follow-up studies suggested that the cognitive decline was reversible after treatment discontinuation.

Other names

umibecestat