“With no approved therapies indicated for IgA nephropathy, dosing the first patient in the pivotal PROTECT Study brings us closer to our goal of ultimately delivering sparsentan as the first approved therapy for patients living with this rare disorder,” said
About the PROTECT Study
The PROTECT Study is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled Phase 3 clinical trial evaluating the safety and efficacy of sparsentan for the treatment of IgAN. Approximately 280 patients with IgAN, aged 18 years or older, are expected to be randomized to receive either sparsentan (initial dose of 200 mg daily for two weeks, titrating up to a target dose of 400 mg daily) or an active control, irbesartan (initial dose of 150 mg daily for two weeks, titrating up to a target dose of 300 mg daily).
The primary efficacy endpoint in the study is the change in proteinuria (urine protein-to-creatinine ratio) from baseline after 36 weeks of treatment.
About IgA Nephropathy
IgAN, also known as Berger’s disease, is a rare chronic kidney disorder in which an estimated 20 to 40 percent of patients progress to ESRD within 10 to 20 years of diagnosis. IgAN is often defined by progressive proteinuria, hematuria and acute onset of nephrotic syndrome. The disorder is estimated to affect more than 100,000 people in the U.S. and is one of the leading causes of acute nephritis in
Sparsentan’s dual mechanism of action combines angiotensin receptor blockade with endothelin receptor type A blockade.
The Phase 2 DUET Study of sparsentan in FSGS met its primary efficacy endpoint for the combined treatment group, demonstrating a greater than two-fold reduction in proteinuria compared to irbesartan, after the eight-week, double-blind treatment period. Irbesartan is part of a class of drugs used to manage FSGS and IgAN in the absence of an
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, with respect to sparsentan, the Company faces the risk that the Phase 3 clinical trial of sparsentan in IgAN (the “PROTECT Study”) will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned, the Company faces the risk that the Phase 3 clinical trial of sparsentan in FSGS (the “DUPLEX Study”) will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned; and more generally, risk that the Company’s product candidates will not be approved for efficacy, safety, regulatory or other reasons, and for each of the programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed, may not progress as expected, or may be delayed for 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; and risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the
Vice President, Investor Relations & Corporate Communications
Source: Retrophin, Inc.