“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,
Program Updates and Anticipated 2019 Milestones
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
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. Retrophinexpects 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.
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company’s business and finances in general, success of its commercial products as well as risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with market acceptance of its marketed products including efficacy, safety, price, reimbursement and benefit over competing therapies. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company's clinical candidates will not be found to be safe or effective and that current or future clinical trials will not proceed as planned. Specifically, the Company faces the risk that the Phase 3 clinical trial of sparsentan in FSGS will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned; risk that the Phase 3 clinical trial of sparsentan in IgAN will not demonstrate that sparsentan is safe or effective or serve as the basis for accelerated approval of sparsentan as planned; risk that the Phase 3 clinical trial of fosmetpantotenate will not demonstrate that fosmetpantotenate is safe or effective or serve as the basis for an NDA filing as planned; and for each of its development programs and for its partner’s CNSA-001 program, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons and risk that the product candidates will not be approved for efficacy, safety, regulatory or other reasons. The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with its option to acquire
Vice President, Investor Relations & Corporate Communications
Source: Retrophin, Inc.