Travere Therapeutics Announces Completion of Patient Enrollment in Pivotal Phase 3 DUPLEX Study of Sparsentan in Focal Segmen...
30 Noviembre 2020 - 3:05PM
Travere Therapeutics (NASDAQ: TVTX) today announced completion of
patient enrollment in the Phase 3 DUPLEX Study. The pivotal DUPLEX
Study is evaluating the safety and efficacy of sparsentan for the
treatment of focal segmental glomerulosclerosis (FSGS), a rare
kidney condition that often leads to end-stage kidney disease
(ESKD). Topline efficacy data from the interim 36-week proteinuria
endpoint analysis are expected in the first quarter of 2021.
“Many people living with FSGS are unable to
delay progression to end-stage kidney disease and ultimately face
transplant or dialysis,” said Noah Rosenberg, M.D., chief medical
officer of Travere Therapeutics. “New treatment options are
desperately needed. By completing enrollment in the DUPLEX Study,
we have achieved the next milestone on our path to potentially
delivering sparsentan as the first therapy indicated for the
treatment of FSGS. We are maintaining focus on high quality trial
conduct over the course of the study and we look forward to
reporting interim topline results in the first quarter of next
year.”
The DUPLEX Study is a global, randomized,
multicenter, double-blind, parallel-arm, active-controlled Phase 3
clinical trial of approximately 300 patients with FSGS. Patients
are randomized to receive either sparsentan or irbesartan, the
active control. The DUPLEX Study protocol provides for an unblinded
analysis of at least 190 patients to be performed after 36 weeks of
treatment to evaluate the interim efficacy endpoint – the
proportion of patients achieving a FSGS partial remission of
proteinuria endpoint (FPRE), which is defined as urine
protein-to-creatinine ratio (Up/C) ≤1.5 g/g and a >40 percent
reduction in Up/C from baseline, at Week 36. Successful achievement
of the interim 36-week proteinuria endpoint is expected to serve as
the basis for submission of a New Drug Application (NDA) under the
Subpart H accelerated approval pathway in the U.S. and Conditional
Marketing Authorization (CMA) consideration in Europe. The
confirmatory endpoint of the study is the slope of estimated
glomerular filtration rate (eGFR) from baseline to Week 108, in
approximately 300 patients.
About Sparsentan
Sparsentan is an investigational product
candidate in Phase 3 clinical development that has a dual mechanism
of action combining endothelin type A receptor antagonism with
angiotensin II receptor blockade. Travere Therapeutics is
developing sparsentan for the treatment of FSGS and IgA nephropathy
(IgAN), rare kidney disorders that often lead to ESKD. In several
forms of chronic kidney disease, such as FSGS and IgAN, endothelin
receptor blockade has been shown to have an additive beneficial
effect on proteinuria in combination with renin-angiotensin
blockade via angiotensin receptor blockers or angiotensin
converting enzyme inhibitors. Sparsentan has been granted orphan
drug designation for the treatment of FSGS by the FDA and European
Commission.
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. Travere Therapeutics is currently advancing the pivotal
Phase 3 DUPLEX Study of sparsentan for the treatment of FSGS and
continuing to enroll patients in the pivotal Phase 3 PROTECT Study
of sparsentan for the treatment of IgAN (IgANprotect.com). Both
studies contain 36-week proteinuria-based interim endpoints, which
if successfully achieved, are expected to 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. If approved
for both indications, sparsentan could potentially be the first
medicine approved for FSGS and IgAN.
About Travere Therapeutics
At Travere Therapeutics we are in rare for life.
We are a biopharmaceutical company that comes together every day to
help patients, families and caregivers of all backgrounds as they
navigate life with a rare disease. On this path, we know the need
for treatment options is urgent – that is why our global team works
with the rare disease community to identify, develop and deliver
life-changing therapies. In pursuit of this mission, we
continuously seek to understand the diverse perspectives of rare
patients and to courageously forge new paths to make a difference
in their lives and provide hope – today and tomorrow. For more
information, visit travere.com
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 include, but are not limited to,
references to the Company’s current expectations around the
timeline for reporting top-line data from the proteinuria endpoint
in the DUPLEX study, expectations regarding potential regulatory
submissions for sparsentan under the Subpart H accelerated approval
pathway in the U.S. and CMA consideration in Europe, the Company’s
path to potentially delivering sparsentan as the first therapy
indicated for the treatment of FSGS and the potential future
regulatory approval of sparsentan for FSGS and IgAN. 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; and risk
that sparsentan 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 or may
be delayed for safety, regulatory or other reasons. Also, there is
no guarantee that the positive results from the DUET Study of
sparsentan in FSGS will be repeated in the currently ongoing Phase
3 DUPLEX study. 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 regulatory interactions;
risks and uncertainties relating to competitive products, including
potential generic competition with certain of the Company’s
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
Securities and Exchange Commission.
Contact:Chris Cline, CFASenior Vice President,
Investor Relations & Corporate
Communications888-969-7879IR@travere.com
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