Retrophin, Inc. (NASDAQ: RTRX) is a publicly-traded, San Diego-based biopharmaceutical company focused on the discovery, acquisition, development and commercialization of drugs for the treatment of debilitating and often life-threatening diseases for which there are currently limited patient options. The Company is currently focused on several catastrophic diseases affecting children, including Focal Segmental Glomerulosclerosis (FSGS), Pantothenate Kinase-Associated Neurodegeneration (PKAN), and others.
Retrophin is currently enrolling patients in a potentially pivotal Phase II clinical trial of its lead compound, sparsentan, for FSGS. The Company intends to advance RE-024 for the treatment of PKAN by filing a U.S. IND in the first half of 2015 to support the initiation of a Phase I study. Retrophin has also filed a provisional patent covering a novel formulation of RE-034 for the potential treatment of several rare diseases.