Drug intelligence / Profile preview

AST-004

Development stage
Unknown
Lead developer
Astrocyte Pharmaceuticals
Modality
Small Molecules
Administration
Intravenous (iv), Oral (tablet Formulation Under Development)
01

Overview

AST-004 is an investigational small-molecule drug developed as a dual adenosine A1 and A3 receptor agonist, designed to provide cerebroprotection in acute central nervous system injuries. Its mechanism of action involves agonism at both the adenosine A1 and A3 receptors, which are implicated in reducing neuroinflammation, tissue damage, and brain swelling following events such as stroke, traumatic brain injury (TBI), and concussion. Preclinical studies have demonstrated significant reductions in brain lesion size and neuroprotective effects across multiple animal models. In clinical trials to date—including completed Phase 1 safety studies—AST-004 has shown a favorable safety profile with no serious adverse events reported. The drug is being advanced by Astrocyte Pharmaceuticals for use in acute ischemic stroke (currently entering Phase 2), TBI, concussion (Phase 2 ongoing for sports-related concussion), with additional preclinical work ongoing for Alzheimer’s disease and other neurodegenerative disorders[1][2][3][4][5][6][7][8].

02

Targets

ADORA1 (Adenosine receptor A1 subtype)ADORA3 (Adenosine A3 receptor)

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