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ExSAR Receives $400K FDA Grant for Phase I Clinical Trials
September 26, 2007 - ExSAR Corporation announced today that they are the recipients of a $400,000 U.S. Food and Drug Administration (FDA) grant supporting Phase I clinical studies of EXR-101, ExSAR’s first clinical candidate for the treatment of Adult Tay-Sachs and Sandhoff Diseases. $200,000 has been awarded in support of first year activities, with the second $200,000 to be awarded in the second year following review of progress and availability of funds.

"This FDA award is an important milestone for ExSAR in that it reflects a favorable degree of confidence in our pre-clinical data and Phase I clinical development plan," said Bijan Almassian, Ph.D., principal investigator of the study and ExSAR’s President and Chief Executive Officer. "In collaboration with key academic leaders using our core technology, Hydrogen Deuterium Exchange Mass Spectrometry (H/D-Ex), we have identified small molecules that can be used to treat other protein misfolding disorders and rare diseases." He added, “We intend on filing additional INDs (Investigational New Drug applications) and advancing them into the clinic in the near future."

About EXR-101
EXR-101 has already been approved by the FDA for other indications, can be administered orally and has shown remarkable efficacy in cell-based models of Adult Tay-Sachs and Sandhoff diseases. It is a small molecule pharmacological chaperone of Hexosaminidase A (HexA), the mutant enzyme at the core of Adult Tay-Sachs and Sandhoff Diseases etiology. EXR-101 facilitates the folding and trafficking of HexA to the lysosome where HexA is responsible for catabolizing GM2 ganglioside.

Tay Sachs Disease and Sandhoff Disease
Adult Tay-Sachs and Sandhoff disease are autosomal recessive neurodegenerative diseases resulting from deficiencies in the ability of HexA to catabolize GM2 ganglioside. The excessive neuronal accumulation of GM2 ganglioside is accompanied by progressive neurological deterioration affecting motor, cerebral and spinocerebellar functions. These diseases are rare genetic conditions (<1000 people in the US and Canada) for which there is no effective treatment.

About ExSAR Corporation
ExSAR is a biopharmaceutical company utilizing Hydrogen Deuterium Exchange Mass Spectrometry (H/D-Ex) to validate and then in-license small molecule candidates for the treatment of protein misfolding diseases. ExSAR will also on a contractual basis provide epitope mapping and drug discovery services to pharmaceutical and academic clients interested in utilizing ExSAR’s H/D-Ex technology.

For more information contact Bijan Almassian, PhD or call 732-438-6500.

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