Advancing the Promising Cerebroprotectant AST-004 to Human Clinical Trials

William Korinek, PhD Astrocyte Pharmaceuticals

Co-Principal Investigators

Theodore Liston, PhD Astrocyte Pharmaceuticals
James Lechleiter, PhD University of Texas Health Science Center


The BrightFocus Foundation and MTEC funding will support 1) preparing and conducting the PreIND meeting with the FDA, and the submission of an IND application to start the clinical research in U.S., and 2) enable Astrocyte Pharmaceuticals to produce the starting material for the manufacture of additional AST-004 drug substance to support the oral tablet formulation to be used in mild TBI clinical studies.

Project Details

Astrocyte has developed AST-004, a promising therapeutic for TBI. AST-004 has significant efficacy across a broad range of preclinical TBI models, demonstrating reductions in cell death, edema, and markers of neuronal and astrocyte injury. In a repetitive TBI mouse model, our treatment approach reduced long-term neurological deficits in motor coordination and anxiety. Importantly, AST-004 reduced edema and brain injury markers in a large animal porcine TBI model. AST-004 has shown preliminary efficacy in AD with increased brain cell viability in two AD mouse models. Thus, this support from the BrightFocus Foundation and MTEC can significantly accelerate the development of this novel and promising therapy for mild and severe TBIs that can limit the short and long-term negative effects associated with TBIs. If successful in protecting the human brain after TBI, the treatment will also be assessed for the potential to protect the degenerating brain in AD patients.