Retrophin, Inc. (NASDAQ:RTRX) is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company is focused on advancing the development of its lead pipeline assets: sparsentan for focal segmental glomerulosclerosis (FSGS), a serious kidney disorder that often leads to end-stage renal disease; and RE-024 for pantothenate kinase associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood.
The Companys most advanced pipeline candidate is sparsentan, which could be the first approved pharmacologic treatment for FSGS. In September 2016, Retrophin reported positive top-line data from the Phase 2 DUET study of sparsentan, and will work with the U.S. Food and Drug Administration (FDA) to find the most expeditious path forward. RE-024 also has pioneering potential, as it could be the first approved replacement therapy targeting the underlying cause of PKAN. The Company expects to initiate an efficacy trial of RE-024 in patients with PKAN during the second half of 2016.