ExSAR Gets the Green Light from FDA to Start Clinical Trials
NEWS RELEASE - For Immediate Release
April 30, 2007 - ExSAR Corporation announced today that the U.S. Food and Drug Administration (FDA) cleared the way for the company to initiate Phase I Clinical trials of EX-101, its first clinical candidate, for the potential treatment of GM2 Gangliosidosis (Tay Sachs or Sandhoff variants).
EX-101 is a small molecule pharmacological chaperone for Hexosaminidase A (Hex A), the mutant enzyme in adult Tay-Sachs and Sandhoff diseases. Pharmacological chaperones stabilize normal and misfolded (mutant) enzymes and promote trafficking from the endoplasmic reticulum to the target organelle – in this case the lysosome, where HexA breaks down GM2, the fatty substance that accumulates in adult Tay-Sachs and Sandhoff diseases.
EX-101 can be administered orally and has shown remarkable efficacy in cell-based models of adult Tay-Sachs and Sandhoff diseases. It is already approved by the FDA for other indications.
"This is an important milestone for us and highlights the transformation of ExSAR from a service-oriented company to a drug development company," said Bijan Almassian, Ph.D., President and Chief Executive Officer at ExSAR. "In collaboration with key academic leaders using our core technology, Hydrogen Deuterium Exchange Mass Spectrometry, we have identified small molecules that can be used to treat other misfolded protein and rare diseases," Dr. Almassian said. He added, "We intend to develop these compounds further, and we anticipate filing additional INDs (Investigational New Drug applications) in the near future."
Tay Sachs Disease and Sandhoff Disease
Adult Tay-Sachs and Sandhoff disease – also known as late-onset GM2 Gangliosidosis – are autosomal recessive neurodegenerative diseases. They result from a deficiency of the enzyme Hexosaminidase A, which results in excessive accumulation of certain fats known as gangliosides in the brain and nerve cells. These disorders are characterized by progressive neurological deterioration that mainly affects motor, cerebral and spinocerebellar function. They affect fewer than 1000 people in the United States and Canada. Currently there is no effective treatment for these diseases.
About ExSAR Corporation
ExSAR Corporation was founded in March 1998 and is located near Princeton, New Jersey. ExSAR is a biopharmaceutical company that utilizes Hydrogen/Deuterium Exchange Mass spectrometry in support of in-house drug discovery efforts in the area of misfolded proteins and provides epitope mapping and drug discovery services to pharmaceutical companies and academic laboratories.
If you would like additional information on drug development licensing or H/D exchange services, please
contact Bijan Almassian, Ph.D. President & CEO.
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