Synonyms: BACE1 inhibitor
Therapy Type: Small Molecule
Target Type: Amyloid-Related (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 1)
AZD3293 is an inhibitor of BACE1, the β-secretase sheddase that cleaves the APP protein to release APP's C99 fragment. This fragment then becomes a substrate for subsequent γ-secretase cleavage and Aβ peptide generation. The rationale is that inhibiting BACE1 will reduce amyloid-related toxicity in Alzheimer's disease. AZD3293 is one of several BACE1 inhibitors currently in development. Preclinical data have not been formally published. AZD3293 is administered in solution.
In December 2012, AstraZeneca started a Phase 1 study that evaluated the safety and pharmacological effects of single doses of oral AZD3293 in 72 healthy volunteers. Doses ranged from 1–1,000 mg. This trial ended in May 2013, but results have not been reported. In August 2013, a second Phase 1 study started. This one will test multiple ascending doses of AZD3293 solution in 56 healthy elderly volunteers and patients with mild to moderate Alzheimer's disease for safety, tolerability, pharmacokinetics, and effects on biomarkers in plasma and CSF.
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