We partner with researchers from around the world to conduct clinical trials studying our investigational therapies.
In the second half of 2017, we are planning to initiate a pivotal Phase 3 trial evaluating sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS). Sparsentan could be the first FDA-approved pharmacologic treatment option for patients with this rare kidney disorder that often leads to end-stage renal disease.1
The trial will include an interim assessment of proteinuria that could serve as the basis for a New Drug Application seeking Subpart H accelerated approval of sparsentan. The confirmatory endpoint of the study will subsequently compare changes from baseline in estimated glomerular filtration rate, or eGFR. In the Phase 2 DUET study, which is currently in its open-label phase, the overall sparsentan treatment group experienced significant reductions in proteinuria. Sparsentan was generally safe and well tolerated in the study.2
We are also planning to initiate the Phase 3 FORT study of RE-024, our investigational therapy for pantothenate kinase-associated neurodegeneration (PKAN), and expect to begin dosing patients in mid-2017. Detailed information about this pivotal trial can be found at pkanfortstudy.com and ClinicalTrials.gov. The study will be conducted according to a Special Protocol Assessment (SPA) agreement with the FDA. As part of this process, Retrophin and the agency have agreed that the design of this pivotal trial is adequate to support a New Drug Application. RE-024 could be the first approved replacement therapy targeting the underlying cause of this rare and life-threatening neurodegenerative condition, as current therapeutic strategies are limited to symptom management.3 In 2015, we completed a Phase 1 single ascending dose study of RE-024 in 40 healthy adult volunteers. The double-blind, placebo-controlled study evaluated five oral dose levels of the compound up to 1,800 mg.
- Middleton, et al. Nephrology Rounds 2007; 5(4).
- Trachtman H, Nelson P, Radko K on behalf of the DUET Investigators. Efficacy and Safety of Sparsentan, a Dual Angiotensin II and Endothelin Type A Receptor Antagonist, in Patients with Focal Segmental Glomerulosclerosis: A Phase 2 Trial (DUET). Presented at the American Society of Nephrology Kidney Week Meeting; November 15-20 2016, Chicago, IL.
- Gregory, et al. GeneReviews. 2002.