Retrophin, Inc. (NASDAQ: RTRX) today announced that, based on preliminary and unaudited financial data, the Company expects net product sales for the fourth quarter of 2018 to be approximately $44 million. For the fiscal year 2018, the Company expects total net product sales of approximately $164 million. The Company also provided a general update on its development programs, including anticipated milestones for 2019.
With key data readouts from the FORT Study of fosmetpantotenate in PKAN and the CNSA-001 Phase 2 proof-of-concept study expected in 2019, Retrophin is positioned to create significant value for patients and shareholders this year, said Stephen Aselage, former chief executive officer of Retrophin. The Company closed 2018 with the initiation of the PROTECT Study of sparsentan in IgA nephropathy, our third pivotal development program, continued growth of the base commercial business and most recently hired its new president and chief executive officer, Eric Dube, Ph.D. These clinical and commercial achievements provide strong momentum entering the new year, and I look forward to working with Eric to ensure a seamless transition to drive our continued success.
Program Updates and Anticipated 2019 Milestones
In December 2018, the pivotal Phase 3 FORT Study of fosmetpantotenate in pantothenate kinase-associated neurodegeneration (PKAN) completed patient enrollment. The FORT Study is designed to be registration-enabling in the U.S. and Europe, and top-line data are expected to become available in the third quarter of 2019.
The Phase 2 proof-of-concept study evaluating CNSA-001 in patients with phenylketonuria (PKU) continues to progress, and top-line data are expected to be available in the first half of 2019. CNSA-001 is advancing under a joint development and option agreement with Censa Pharmaceuticals, and the Company expects to make a determination on its option to acquire Censa following the data readout.
The New Drug Application (NDA) for a new, more patient-friendly formulation of Thiola is currently under review by the U.S. Food and Drug Administration (FDA) with an assigned Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2019. Pending approval, the Company expects to begin marketing the new formulation in the second half of 2019.
Enrollment in the Phase 3 DUPLEX Study of sparsentan in focal segmental glomerulosclerosis (FSGS) continues. The Company expects to reach enrollment of 190 patients with FSGS during the second half of 2019, which would enable a top-line readout of the 36-week interim efficacy analysis of proteinuria in the second half of 2020. Successful achievement of the interim efficacy endpoint is expected to serve as the basis for submission of an NDA under the Subpart H accelerated approval pathway in the U.S. and Conditional Marketing Authorization (CMA) consideration in Europe.
In December 2018, the Company announced that the first patient had been dosed in the PROTECT Study, a global, pivotal Phase 3 clinical trial evaluating the long-term nephroprotective potential of sparsentan for the treatment of IgA nephropathy (IgAN). The Company expects that the study will complete enrollment of approximately 280 patients with IgAN in the first half of 2021. Top-line data from the 36-week primary endpoint efficacy analysis of proteinuria is expected to become available in the first half of 2022. Retrophin expects that successful achievement of this endpoint will support submission of an NDA under the Subpart H accelerated approval pathway in the U.S., as well as an application for CMA consideration in Europe.
In late February, the Company expects to announce final financial results from the fourth quarter and full-year 2018, as well as provide a corporate update.
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