Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced positive
topline interim results from the ongoing pivotal Phase 3 PROTECT
Study of sparsentan, an investigational product candidate for the
treatment of IgA nephropathy (IgAN). The PROTECT Study met its
pre-specified interim primary efficacy endpoint with statistical
significance, demonstrating a greater than threefold reduction of
proteinuria from baseline after 36 weeks of treatment, compared to
the active control irbesartan (p<0.0001). Preliminary results
from the interim analysis suggest that to date in the study,
sparsentan has been generally well-tolerated and consistent with
the observed safety profile to date. Based on the results from the
interim analysis, the Company plans to submit an application for
accelerated approval in the U.S. in the first half of 2022 and also
plans to submit an application for conditional marketing
authorization in Europe.
“IgAN is a leading cause of end-stage kidney
disease and there is a clear need for novel treatment options to
slow the progression of this devastating rare kidney disorder,”
said Eric Dube, Ph.D., chief executive officer of Travere
Therapeutics. “These data from the PROTECT Study further
demonstrate sparsentan’s ability to significantly reduce
proteinuria and support its potential to become a new foundational
treatment for people living with IgAN, if approved. We will
continue our efforts to maintain high quality in this ongoing
study, and we look forward to engaging with regulators as we
prepare for accelerated approval submissions beginning in the first
half of next year.”
Consistent with prior guidance, the Company is
providing limited data from the interim analysis to maintain trial
integrity in the ongoing study. In the PROTECT Study, a total of
404 patients with persistent proteinuria despite active ACE or ARB
treatment, were randomized 1:1 to receive once daily oral doses of
either sparsentan or irbesartan, the active control. The study
protocol provided for an unblinded interim analysis to evaluate the
primary efficacy endpoint – the change in proteinuria (urine
protein-to-creatinine ratio) from baseline at Week 36 –
approximately 36 weeks following randomization of the first 280
patients. After 36 weeks of treatment, patients receiving
sparsentan achieved a mean reduction in proteinuria from baseline
of 49.8 percent, compared to a mean reduction in proteinuria from
baseline of 15.1 percent for irbesartan-treated patients
(p<0.0001).
“Sparsentan has now demonstrated in one of the
largest interventional studies to date in IgAN, a statistically
robust and clinically meaningful proteinuria reduction relative to
a current standard of care,” said Noah Rosenberg, M.D., chief
medical officer of Travere Therapeutics. “These data build upon our
Phase 2 DUET and Phase 3 DUPLEX studies in FSGS and further
strengthen the support for our novel approach with sparsentan as a
dual endothelin-angiotensin receptor antagonist being developed for
rare kidney disorders. I want to thank all the patients, their
families, as well as the investigators and site staff who continue
to participate in this ongoing landmark study in IgAN.”
Secondary efficacy endpoints include the rate of
change in estimated glomerular filtration rate (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. The Company believes that preliminary eGFR data
available at the time of the interim analysis are indicative of a
potential clinically meaningful treatment effect after two years of
treatment. Per study protocol, patients will continue in a blinded
manner in the PROTECT Study to fully assess the treatment effect on
eGFR slope over 110 weeks in the confirmatory endpoint analysis.
Topline results from the confirmatory endpoint analysis are
expected in the second half of 2023.
A preliminary review of the interim safety
results indicates that to date in the study, both treatment groups
were generally well tolerated, and sparsentan appeared consistent
with the previously observed safety profile with no new safety
signals emerging.
The Company also remains on track to provide a
regulatory update on its pivotal Phase 3 DUPLEX Study of sparsentan
for the treatment of focal segmental glomerulosclerosis (FSGS)
during the third quarter of 2021.
Conference Call Information
Travere Therapeutics will host a conference call
and webcast today, Monday, August 16, 2021, at 8:30 a.m. ET to
discuss the study results. To participate in the conference call,
dial +1 (855) 219-9219 (U.S.) or +1 (315) 625-6891 (International),
confirmation code 9558913 shortly before 8:30 a.m. ET. The webcast
can be accessed at travere.com, in the Events and Presentations
section of the Investors & Media page and will be archived for
at least 30 days. A replay of the call will be available from 11:30
a.m. ET, August 16, 2021, to 11:30 a.m. ET, August 23, 2021. The
replay number is +1 (855) 859-2056 (U.S.) or +1 (404) 537-3406
(International), confirmation code 9558913.
About the PROTECT Study
The ongoing PROTECT Study is one of the largest
interventional studies to date in IgAN. It is a global, randomized,
multicenter, double-blind, parallel-arm, active-controlled clinical
trial evaluating the safety and efficacy of 400mg of sparsentan,
compared to 300mg of irbesartan, in 404 patients ages 18 years and
up with IgAN and persistent proteinuria despite available ACE or
ARB therapy. The PROTECT Study protocol provides for an unblinded
interim 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) at Week
36 from baseline. 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.
About Sparsentan
Sparsentan, a Dual Endothelin Angiotensin
Receptor Antagonist (DEARA), is a novel investigational product
candidate. Pre-clinical data have shown that blockade of both
endothelin type A and angiotensin II type 1 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 DUPLEX Study for the treatment of focal segmental
glomerulosclerosis (FSGS) and the pivotal Phase 3 PROTECT Study for
the treatment of IgAN. In February 2021, the Company announced that
the ongoing pivotal Phase 3 DUPLEX Study of sparsentan in FSGS
achieved its pre-specified interim FSGS partial remission of
proteinuria endpoint (FPRE) with statistical significance. FPRE is
a clinically meaningful endpoint defined as urine
protein-to-creatinine ratio (UP/C) ≤1.5 g/g and a >40 percent
reduction in UP/C from baseline. After 36 weeks of treatment, 42.0
percent of patients receiving sparsentan achieved FPRE, compared to
26.0 percent of irbesartan-treated patients (p=0.0094). Preliminary
results from the interim analysis suggest that at the time of the
interim assessment, sparsentan had been generally well-tolerated
and shown a comparable safety profile to irbesartan. In the Phase 2
DUET Study of sparsentan in FSGS, the combined treatment group met
its primary efficacy endpoint, demonstrating a greater than
two-fold reduction in proteinuria compared to irbesartan, and was
generally well tolerated 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. If approved for both indications, sparsentan could
potentially be the first medicine approved for both 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 plan regarding, and
expectations around the timeline for, submitting an application for
accelerated approval of sparsentan for IgAN; references to the
efficacy, safety and tolerability profile of sparsentan based on
the preliminary data from the PROTECT Study interim analysis; the
potential for sparsentan to become a new foundational treatment for
people living with IgAN, if approved; the Company’s belief that
preliminary eGFR data available at the time of the interim analysis
from the PROTECT Study are indicative of a potential clinically
meaningful treatment effect after two years of treatment;
expectations regarding the future conduct of the ongoing PROTECT
study and timing for topline results from the confirmatory endpoint
analysis; expectations around the timing for providing a regulatory
update on the Company’s pivotal Phase 3 DUPLEX Study of sparsentan
for the treatment of focal segmental glomerulosclerosis (FSGS); and
the potential for sparsentan to become the first medicine approved
for both 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 the Europe
Union, including the risk that the FDA and/or EMA could disagree
with the Company’s submission of an NDA under Subpart H for
accelerated approval, or a Marketing Approval Application (“MAA”)
under the CMA pathway. 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 FDA will accept for filing
the Company’s planned NDA 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; 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-K, Form 10-Q and other filings with
the Securities and Exchange Commission.
Contact:
Chris Cline,
CFA
Senior Vice
President, Investor Relations & Corporate
Communications888-969-7879 IR@travere.com
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