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 2 clinical trial of its lead compound, sparsentan, for the treatment of FSGS and expects to randomize the last patient in early 2016. The Company is also developing RE-024, its novel investigational phosphopantothenate replacement therapy, for the treatment of PKAN. RE-024 has completed dosing of a Phase 1 study in healthy volunteers and is expected to advance to the next stage of development in PKAN patients during 2016. Retrophin has also filed a provisional patent covering a novel formulation of RE-034 (a synthetic ACTH analog) for the potential treatment of several rare diseases.