ExSAR is developing EXR-101 for the treatment of Late-Onset Tay-Sachs (LOTS). EXR-101 is a small molecule pharmacological chaperone that binds to and stabilizes HexosaminidaseA (HexA). EXR-101 has been approved by the Food and Drug Administration (FDA) for other indications, can be administered orally and has shown remarkable efficacy in cell lines derived from LOTS patient fibroblasts. The body of supporting pre-clinical data and ExSAR's Phase I clinical development plan earned its Investigational New Drug Authorization (IND) and Phase I clinical trial support from the FDA in April and September 2007, respectively. ExSAR has also applied for FDA orphan designation. The Orphan Drug Act grants special status to products by providing a seven year window of market exclusivity and tax incentives. Orphan designation for EXR-101 is currently pending positive clinical outcome. 

Lysosomal Storage Disorders and Protein Misfolding | Product Pipeline | Clinical Trials
EXR-101 for Late-Onset Tay-Sachs | Tay-Sachs and EXR-101 | EXR-202 for Gaucher disease | Gaucher Disease and EXR-202