Drug intelligence / Profile preview

scyllo-inositol

Development stage
Phase 2
Lead developer
OPKO Health
Modality
Small Molecules
Administration
Oral
01

Overview

Scyllo-inositol is a naturally occurring stereoisomer of inositol and a sugar alcohol with the molecular formula C6H12O6. It has been investigated primarily as an oral small molecule for the treatment of neurodegenerative diseases such as Alzheimer's disease and Down syndrome-related cognitive decline. Its mechanism involves binding to beta-amyloid (Aβ) peptides, inhibiting their aggregation into toxic oligomers and fibrils, thereby reducing amyloid plaque burden in the brain. Preclinical studies demonstrated that scyllo-inositol can cross the blood-brain barrier and ameliorate cognitive deficits in animal models of Alzheimer's disease by decreasing insoluble Aβ40/Aβ42 levels and preserving synaptic function. Clinical trials have explored its safety and efficacy under code names such as AZD103 or ELND005; however, results have been inconclusive or negative for efficacy endpoints in Alzheimer's disease populations[1][2][3][4][7]. The compound was developed by Transition Therapeutics (later acquired by OPKO Health), with development partnerships including Elan Corporation[2].

Other names
scyllitolcocositolquercinitol1,3,5/2,4,6-cyclohexanehexol
02

Targets

Amyloid-beta aggregate (N-terminal epitope)

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