Travere Therapeutics Completes Enrollment in Pivotal Phase 3 PROTECT Study of Sparsentan in IgA Nephropathy
June 02 2021 - 8:00AM
Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced
completion of patient enrollment in the Phase 3 PROTECT Study. The
pivotal PROTECT Study is evaluating the safety and efficacy of
sparsentan for the treatment of IgA nephropathy (IgAN), a rare
kidney disorder that often progresses to end-stage kidney disease
(ESKD). Topline efficacy data from the interim 36-week proteinuria
endpoint analysis are expected in August 2021.
“The current treatment goal for people living
with IgAN is to preserve kidney function, but with limited
treatment options available many patients remain at high risk of
progression towards end-stage kidney disease,” said Noah Rosenberg,
M.D., chief medical officer of Travere Therapeutics. “Completing
enrollment in PROTECT marks a significant milestone on our path to
potentially establishing sparsentan, if approved, as a new,
non-immunosuppressant based treatment option to slow the
progression of IgAN. We are grateful for the continued strong
support from patients and investigators in this important trial,
and we look forward to the topline results from the 36-week
proteinuria analysis in August of this year.”
The PROTECT Study is a global, randomized,
multicenter, double-blind, parallel-arm, active-controlled pivotal
Phase 3 clinical trial evaluating the safety and efficacy of
sparsentan in approximately 380 patients with IgAN. The PROTECT
Study protocol provides for an unblinded analysis of at least 280
patients to be performed after 36 weeks of treatment to evaluate
the primary efficacy endpoint – the change in proteinuria (urine
protein-to-creatinine ratio, or UPCR) at Week 36 from baseline. The
interim assessment of the PROTECT Study is designed to support
potential submissions under the Subpart H pathway for accelerated
approval in the United States, and potential Conditional Marketing
Authorization in Europe. Secondary efficacy endpoints include the
rate of change in eGFR following the initiation of randomized
treatment over 58-week and 110-week periods, as well as the rate of
change in eGFR over 52-week and 104-week periods following the
first six weeks of randomized treatment in approximately 380
patients.
About IgA Nephropathy
IgA nephropathy (IgAN), also called Berger's
disease, is a rare kidney disorder characterized by the buildup of
immunoglobulin A (IgA), a protein that helps the body fight
infections, in the kidneys. The deposits of IgA cause a breakdown
of the normal filtering mechanisms in the kidney, leading to blood
in the urine (hematuria), and protein in the urine (proteinuria).
Other symptoms of IgAN may include kidney pain, swelling (edema)
and high blood pressure.
IgAN is the most prevalent primary chronic
glomerular disease worldwide and a leading cause of ESKD. IgAN is
estimated to affect more than 100,000 people in the U.S. and is one
of the leading causes of acute nephritis in Europe and Japan. There
are currently no approved treatments indicated for IgAN.
About Sparsentan
Sparsentan is a novel investigational product
candidate, that functions as a high affinity dual-acting antagonist
of both the endothelin type A and angiotensin II type 1 receptors,
in a single molecule. Pre-clinical data have shown that blockade of
both pathways in forms of rare chronic kidney disease, reduces
proteinuria, protects podocytes and prevents glomerulosclerosis and
mesangial cell proliferation. Sparsentan has been granted Orphan
Drug Designation for the treatment of IgAN and FSGS in the U.S. and
Europe.
Sparsentan is currently being evaluated in the
pivotal Phase 3 PROTECT Study for the treatment of IgAN and the
pivotal Phase 3 DUPLEX Study for the treatment of focal segmental
glomerulosclerosis (FSGS). If approved for both indications,
sparsentan could potentially be the first medicine approved for
both IgAN and FSGS.
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 timing for
reporting top-line data from the interim proteinuria endpoint in
the PROTECT Study; the Company’s path to potentially establishing
sparsentan, if approved, as a new, non-immunosuppressant based
treatment option to slow the progression of IgAN; the potential for
the interim assessment of the PROTECT Study to support submissions
under the Subpart H pathway for accelerated approval in the United
States and Conditional Marketing Authorization in Europe; 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
regulatory review and approval process, including the Subpart H
accelerated approval pathway in the United States and the
conditional marketing authorization (CMA) pathway in Europe.
Specifically, the Company faces the risk that the Phase 3 DUPLEX
Study 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 PROTECT Study 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 Company’s 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. There is no guarantee that the interim
assessment from the PROTECT Study will support an NDA submission
for sparsentan for IgAN under the Subpart H approval pathway, that
the FDA will grant accelerated approval of sparsentan for IgAN or
that sparsentan will be approved at all. 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; and risks and uncertainties relating
to competitive products, including current and potential future
generic competition with certain of the Company’s products, and
technological changes that may limit demand for the Company's
products. The Company faces additional risks associated with the
potential impacts the COVID-19 pandemic may have on its business,
including, but not limited to (i) the Company’s ability to continue
its ongoing development activities and clinical trials, (ii) the
timing of such clinical trials and the release of data from those
trials, (iii) the Company’s and its suppliers’ ability to
successfully manufacture its commercial products and product
candidates, and (iv) the market for and sales of its commercial
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-Q, Form 10-K 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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