Drug intelligence / Profile preview

atabecestat

Development stage
Phase 3
Lead developer
Janssen Research & Development
Modality
Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral
01

Overview

Atabecestat is an oral, brain-permeable small molecule inhibitor of beta-site amyloid precursor protein cleaving enzyme 1 (BACE1), also known as β-secretase. By inhibiting BACE1, atabecestat reduces the production of amyloid beta (Aβ) peptides from amyloid precursor protein (APP), which are implicated in the pathogenesis of Alzheimer's disease. The drug was developed for the treatment of early-stage Alzheimer's disease and mild cognitive impairment due to Alzheimer's disease. Clinical trials demonstrated that atabecestat significantly reduced Aβ levels in cerebrospinal fluid and plasma, confirming target engagement. However, development was discontinued after reports of liver toxicity and cognitive worsening similar to other BACE inhibitors[1][2][6].

Brand names
atabecestat
Other names
atabecestat1200493-78-2N-(3-((4S)-2-amino-4-methyl-4H-1,3-thiazin-4-yl)-4-fluorophenyl)-5-cyano- 2-pyridinecarboxamide
02

Targets

BACE1 (Beta-site APP-cleaving enzyme 1)BACE2 (Beta-site amyloid precursor protein cleaving enzyme 2)

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