DUET publication highlighted during Best of ASN Journals session at ASN Kidney Week 2018
“Patients with FSGS face a significant unmet need caused by a progressive decline in kidney function, which often requires a kidney transplant or dialysis,” said principal investigator
In the randomized, double-blind, controlled Phase 2 DUET Study, the overall sparsentan treatment group achieved statistical significance in the primary efficacy endpoint, demonstrating a greater than two-fold reduction in proteinuria compared to the irbesartan treatment group, after an eight-week, double-blind treatment period. Treatment with sparsentan resulted in greater reductions in urinary protein-to-creatinine ratio (UP/C) compared with irbesartan when all dose cohorts (44.8 percent versus 18.5 percent; p=0.006) were combined or when the 400 and 800 mg dose cohorts (47.4 percent vs. 19.0 percent; p=0.011) were combined.
An analysis of the secondary endpoint of the study showed that during the eight-week, double-blind treatment period, a significantly greater proportion of patients receiving sparsentan (28.1 percent) achieved the FSGS partial remission of proteinuria endpoint (FPRE), defined as UP/C: ≤1.5 g/g and >40 percent reduction of proteinuria from baseline, compared to irbesartan-treated patients (9.4 percent; p=0.040).
In addition, data from patients who were followed out to 48 weeks in the open-label sparsentan treatment period of DUET demonstrated a steady rise in the percentage of patients who achieved FPRE, reaching approximately 60 percent in patients originally randomized to either sparsentan or irbesartan.
The data also showed sparsentan was generally well-tolerated during the eight-week, double-blind period, with the overall incidences of treatment-emergent adverse events (TEAEs), drug-related TEAEs, or serious TEAEs similar between the sparsentan and irbesartan groups.
New data from an 84-week analysis of the open-label portion of the DUET Study will be presented at ASN Kidney Week 2018 during the Glomerular Diseases: Clinical, Outcomes, and Trials session on
About Focal Segmental Glomerulosclerosis
FSGS is a rare kidney disorder without an approved pharmacologic treatment option that is estimated to affect up to 40,000 patients in the U.S. with similar prevalence in
Reduction in proteinuria appears to be beneficial in the treatment of FSGS and may be associated with a decreased risk of progression to ESRD. Achieving FPRE appears to be associated with long-term preservation of renal function in patients with FSGS. Symptoms of FSGS are currently managed with angiotensin receptor blockers, angiotensin converting enzyme inhibitors, steroids or calcineurin inhibitors.
Sparsentan is an investigational product candidate that has a dual mechanism of action that 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 approved pharmacologic treatment. In
Forward Looking Statements
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 the risk that favorable results seen in the sparsentan Phase 2 DUET Study to date will not continue or be replicated in the future, 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 planned Phase 3 clinical trial of sparsentan in IgAN will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned, risk associated with enrollment of clinical trials for rare diseases and risk the clinical trial may not succeed 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 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.