Travere Therapeutics Announces Successful Outcome from Type A Meeting with U.S. FDA for Sparsentan in Focal Segmental Glomerulosclerosis
07 September 2021 - 10:00PM
Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced a
successful outcome from the Company’s Type A meeting with the U.S.
Food and Drug Administration (FDA) in which alignment was reached
on the Company’s plan to submit additional estimated glomerular
filtration rate (eGFR) data from the ongoing pivotal Phase 3 DUPLEX
Study of sparsentan in focal segmental glomerulosclerosis (FSGS) to
support an application for accelerated approval in 2022.
“We are very pleased with the outcome of the
Type A meeting which confirms our plan to provide FDA with
additional eGFR data from the ongoing DUPLEX Study in the first
half of 2022 to continue on the accelerated approval pathway for
sparsentan in FSGS,” said Eric Dube, Ph.D., chief executive officer
of Travere Therapeutics. “If the additional data further strengthen
the prediction of long-term benefit in the study as we expect, we
anticipate submitting a New Drug Application for accelerated
approval of sparsentan for FSGS in the middle of next year and
furthering our preparations to deliver it as a potential new
treatment standard for FSGS, if approved.”
Following the outcome of the Type A meeting, the
Company intends to continue with its plan to provide the FDA with
additional eGFR data from the ongoing DUPLEX Study in the first
half of 2022. At the time of the eGFR data cut, all patients
remaining in the DUPLEX Study will have completed one year of
treatment, and approximately 50 percent of patients will have
completed two years of treatment. The Company plans to submit an
application for accelerated approval in the U.S. in mid-2022,
pending additional supportive eGFR data. The DUPLEX Study is
continuing as planned with no changes to the statistical analysis
plan, and patients will proceed in a blinded manner to assess the
treatment effect on eGFR slope over 108 weeks in the confirmatory
endpoint analysis.
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 FSGS and IgAN 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 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 August of 2021, the
Company announced that the ongoing 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 at the time of the interim
assessment, sparsentan had been generally well-tolerated and
performed consistent with the observed safety profile to date. 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 expected timing and plan for the data cut for the
additional eGFR data from the DUPLEX Study; the Company’s
expectation that the additional data should further strengthen the
prediction of long-term benefit in the DUPLEX study; the
expectation of submitting an application for accelerated
approval of sparsentan for FSGS in mid-2022, pending the additional
eGFR data; the Company’s expectations to further its preparations
to deliver sparsentan as a potential new treatment standard for
FSGS, if approved; expectations regarding the future conduct of the
ongoing DUPLEX study and timing for topline results from the
confirmatory endpoint analysis; references to the efficacy, safety
and tolerability profile of sparsentan based on the preliminary
data from the DUPLEX and PROTECT Studies’ interim analyses; 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, and the risk that the additional eGFR
data will not support an accelerated approval submission.
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 planned
eGFR data cut will provide sufficient additional support for an
accelerated approval submission for sparsentan in FSGS, that the
FDA will accept for filing a NDA for sparsentan for FSGS under the
Subpart H approval pathway, that the FDA will grant accelerated
approval of sparsentan for FSGS 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, CFASenior Vice President, Investor Relations &
Corporate Communications888-969-7879 IR@travere.com
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