ExSAR Therapeutics in Development
The method of use of EXR-101 and EXR-202 as pharmacological chaperones (PCs) is protected under two patent applications with rights assigned to ExSAR from the Hospital for Sick Children, Toronto, Canada. Both EXR-101 and EXR-202 are bioavailable blood-brain barrier permeable small molecules. In contrast to currently available enzyme replacement therapies, EXR-202’s indication will most likely include treatment of neuronopathic forms of Gaucher disease (GD). A large body of information, including preclinical toxicology and human clinical data for EXR-101 and EXR-202 already exists as they both have already been approved by regulatory authorities for other indications. For example, EXR-101 has been approved by both the FDA and EMEA (Europe, Middle East and Africa) for the treatment of malaria, and EXR-202 has been approved by the EMEA and some African and Asian countries for the treatment of acute and chronic diseases of upper respiratory tracts including asthmatic bronchitis.
The FDA granted EXR-101 IND authorization in April 2007 and an IND application for EXR-202 is in preparation. ExSAR has applied for orphan drug designation with the FDA for EXR-101 for the treatment of Late Onset Tay-Sachs (LOTS). The Orphan Drug Act (ODA) grants special status to products including seven years market exclusivity and tax incentives. ExSAR plans to apply for orphan drug designation for use of EXR-202 for the treatment of Gaucher disease after receiving authorization from the FDA to initiate human clinical trials.
ExSAR has established relationships with leading lysosomal disorder treatment centers (e.g., University Hospitals of Cleveland, New York University School of Medicine, the Hospital for Sick Children and Shaare Zedek Medical Center) for conducting Phase I and Phase II clinical trials of EXR-101 and EXR-202. All the clinicians involved with these studies are opinion leaders in the field of lysosomal disorders and have extensive experience in running Lysosomal Storage Disorders (LSD) related clinical trials. Phase II studies will include assessment of safety and efficacy based on biomarker and clinical end point measurement. We have identified an experienced CRO to monitor and manage these clinical trials.
Periodically, ExSAR’s therapies for Lysosomal Storage Disorders and Protein Misfolding will enter the clinical trial phase. Bookmark this page in your browser and check back for updates.