AGY Therapeutics was founded to apply the latest molecular and cellular research techniques to central nervous system diseases. AGY’s drug discovery and development efforts focus on neurodegeneration (functional recovery from stroke and neuroprotection) and cognitive disorders (cognition/schizophrenia). The company has identified and validated novel targets for the treatment of neurological and mental diseases using its proprietary imAGYne™ molecular cloning, bioinformatics and validation platform. The company has established a sophisticated compound screening and profiling platform, including protein expression and purification, assay development, high-throughput screening, neuronal assays and in vivo models. AGY developed a modern, high quality compound library based on high chemical diversity, lead- and drug-like features and a proprietary algorithm to predict blood-brain-barrier permeability. AGY has established and continues to develop its medicinal chemistry, pre-clinical and clinical development capabilities. All three drugs are effective in about 80% of cases. Of these, operates at very low doses - is 5-10 times lower than the competition. This slightly reduces the severity of side effects, but in diseases of the cardiovascular system and stroke drug still is contraindicated.

This novel approach to understanding disease mechanisms on a molecular level with a focus on the discovery and development of therapeutics for central nervous system disorders led to the following programs:

Stroke program: Stroke is the third leading cause of death in the industrialized world and affects annually over 2 million people worldwide. In our acute neuroprotection program, we develop compounds that are active within an extended time-window post-stroke and will be administered intravenously. In our functional recovery program we develop compounds that will enhance sensory-motor and cognitive functions after stroke that will be administered within several days after the acute manifestation of stroke with continued treatment during the recovery phase. Based on the molecular mechanisms of these targets and lead compounds we anticipate utilities in additional indications such as traumatic brain injury, Parkinson’s disease and spinal cord injury.

Cognition/schizophrenia: Glutamate is the brain’s main excitatory neurotransmitter and hypofunction of this system has been implicated in schizophrenia and cognitive disorders. A central role in glutamatergic neurotransmission is played by the NMDA receptor. The activity of this receptor is regulated by its phosphorylation state. AGY’s cognition/schizophrenia program aims to enhance NMDA receptor function in a glutamate dependent manner in pathologic situations where signaling is repressed by inhibiting intracellular phosphatases.

Oncology antibody program: Astrocytomas are the most common form of brain tumors; among these, glioblastoma multiforme (GBM) is one of the most deadly forms of cancer described. AGY identified three targets that are highly expressed in GBM and generated potent and selective monoclonal antibodies against these cell-surface antigens. These antibodies bind to and kill glioma cells when conjugated with a toxin. The utility of these antibodies may not be limited to gliomas, as these targets are highly expressed on a number of additional solid tumors.
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