Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced
that, based on preliminary and unaudited financial data, the
Company expects net product sales from continuing operations for
the fourth quarter of 2023 to be approximately $40 million. For the
fiscal year 2023, the Company expects net product sales from
continuing operations to be approximately $128 million. The Company
ended 2023 with approximately $567 million in cash, cash
equivalents, and marketable securities which is expected to support
operations into 2028. The Company also provided an update on key
corporate, clinical, and regulatory development initiatives,
including anticipated milestones for 2024.
"Our strong fourth quarter results reflect the
growing demand for FILSPARI in IgAN driven by our field teams’
education and outreach efforts to healthcare providers,” said Eric
Dube, Ph.D., president and chief executive officer of Travere
Therapeutics. “In 2024, we are well-positioned to drive sustained
growth of FILSPARI as the only non-immunosuppressive therapy for
IgAN through our continued strong commercial execution and the
potential conversion from accelerated to full approval.
Additionally, we are pleased to have recently initiated the pivotal
HARMONY Study for pegtibatinase. We are focused on building
momentum for enrollment of this important trial which is designed
to support the potential approval of pegtibatinase as the first and
only disease-modifying therapy for classical HCU. Looking forward,
we believe our efforts will ultimately position FILSPARI and
pegtibatinase as new treatment standards in IgAN and classical HCU,
clearly helping to address the needs of patients living with these
rare diseases.”
Program Updates and Anticipated 2024
Milestones
FILSPARI (sparsentan) is an endothelin and
angiotensin II receptor antagonist indicated to reduce proteinuria
in adults with primary immunoglobulin A nephropathy (IgAN) at risk
of rapid disease progression, generally a UPCR ≥1.5 g/g. In 2024,
the Company anticipates conversion of FILSPARI from accelerated
approval to full approval for IgAN as well as a potential approval
for the treatment of IgAN in Europe. The Company is also generating
additional data that is expected to support the clinical profile of
FILSPARI and it anticipates updates to treatment guidelines for
IgAN to include FILSPARI.
FILSPARI (sparsentan) –
IgAN
- In the fourth
quarter of 2023, the Company received 459 new patient start forms
(PSFs), reflecting continued strength in physician demand.
- Preliminary net
product sales of FILSPARI in the fourth quarter of 2023 were $15
million; $30 million since the beginning of commercial launch in
February 2023.
- The Company is
on track to submit a supplemental New Drug Application (sNDA) in
the first quarter of 2024 for conversion of the existing U.S.
accelerated approval of FILSPARI to full approval.
- Following
submission of the two-year results from the PROTECT Study of
FILSPARI in IgAN and a corresponding procedural review clock-stop,
the Company and its collaborator CSL Vifor anticipate a review
opinion by the Committee for Medicinal Products for Human Use
(CHMP) on the potential approval of the Conditional Marketing
Authorization (CMA) application for sparsentan for the treatment of
IgAN in Europe in the first quarter of 2024. If approved,
sparsentan would receive CMA in all member states of the European
Union, as well as in Iceland, Liechtenstein, and Norway.
- In 2024, the
Company expects additional data from its ongoing open-label studies
evaluating the safety and efficacy of sparsentan in combination
with sodium glucose cotransporter-2 inhibitors (SGLT2i) as well as
from the ongoing SPARTAN Study evaluating the potential effect of
FILSPARI as a first-line therapy in patients with newly diagnosed
IgAN.
- In 2024, the
Company anticipates inclusion of FILSPARI into the Kidney Disease
Improving Global Outcomes (KDIGO) Clinical Practice Guideline for
the Management of Glomerular Diseases.
Sparsentan – Focal Segmental
Glomerulosclerosis (FSGS)
- In 2024, the
Company is conducting additional analyses of FSGS data and will
engage with regulators to evaluate potential regulatory pathways
for a sparsentan FSGS indication.
Pegtibatinase – Classical
HCU
The Company is advancing pegtibatinase, a novel
investigational enzyme replacement therapy with the potential to
become the first and only disease-modifying therapy for people
living with classical homocystinuria (HCU).
- In December 2023, the Company
initiated the pivotal Phase 3 HARMONY Study to support the
potential approval of pegtibatinase for the treatment of classical
HCU. The HARMONY Study is a global, randomized, multi-center,
double-blind, placebo-controlled Phase 3 clinical trial designed to
evaluate the efficacy and safety of pegtibatinase as a novel
treatment to reduce total homocysteine (tHcy) levels. The trial is
expected to enroll approximately 70 patients with a diagnosis of
classical HCU and tHcy levels ≥50 μM while maintaining their
standard-of-care treatment. Participants will be randomized 1:1 to
receive 2.5 mg/kg of pegtibatinase or placebo, administered
subcutaneously, for a 24-week blinded treatment duration. The
primary endpoint is relative geometric mean change in plasma tHcy
levels from baseline compared to weeks 6 through 12. Durability of
treatment response through 24 weeks of treatment will also be
measured as a secondary endpoint. Topline results from the HARMONY
Study are expected in 2026.
- The Company will also be initiating
the ENSEMBLE Study, a Phase 3b, open-label, long-term extension,
that will evaluate the ongoing efficacy and long-term safety of
pegtibatinase in participants with HCU following their completion
of the COMPOSE Study or the HARMONY Study. ENSEMBLE will include an
optional protein tolerance modification sub-study that will
evaluate if patients can increase their natural dietary protein
intake and maintain an acceptable level of metabolic control while
receiving pegtibatinase.
The Company expects to announce complete full
year 2023 financial results and provide a corporate update in
February.
About Preliminary Financial
Results
The preliminary results set forth above are
unaudited, are based on management’s initial review of the
Company’s results for the quarter and year ended December 31, 2023,
and are subject to revision based upon the Company’s year-end
closing procedures and the completion and external audit of the
Company’s year-end financial statements. Actual results may differ
materially from these preliminary unaudited results following the
completion of year-end closing procedures, final adjustments or
other developments arising between now and the time that the
Company’s financial results are finalized. In addition, these
preliminary unaudited results are not a comprehensive statement of
the Company’s financial results for the year ended December 31,
2023, should not be viewed as a substitute for full, audited
financial statements prepared in accordance with generally accepted
accounting principles, and are not necessarily indicative of the
Company’s results for any future period.
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
About FILSPARI (sparsentan)
FILSPARI (sparsentan) is a once-daily, oral
medication designed to selectively target two critical pathways in
the disease progression of IgAN (endothelin-1 and angiotensin II)
and is the first and only non-immunosuppressive therapy approved
for the treatment of this condition. FILSPARI is a prescription
medicine indicated to reduce proteinuria in adults with primary
IgAN at risk of rapid disease progression, generally a UPCR ≥1.5
g/g.
FILSPARI (sparsentan) U.S.
Indication
FILSPARI is an endothelin and angiotensin II
receptor antagonist indicated to reduce proteinuria in adults with
primary immunoglobulin A nephropathy (IgAN) at risk of rapid
disease progression, generally a UPCR ≥1.5 g/g.
This indication is granted under accelerated
approval based on reduction in proteinuria. It has not been
established whether FILSPARI slows kidney function decline in
patients with IgAN. Continued approval for this indication may be
contingent upon verification and description of clinical benefit in
a confirmatory clinical trial.
FILSPARI (sparsentan) Important Safety
Information
BOXED WARNING:
HEPATOTOXICITY AND EMBRYO-FETAL
TOXICITYBecause of the risks of
hepatotoxicity and birth defects, FILSPARI is available only
through a restricted program called the FILSPARI REMS. Under the
FILSPARI REMS, prescribers, patients and pharmacies must enroll in
the program.
HepatotoxicitySome
Endothelin Receptor Antagonists (ERAs) have caused elevations of
aminotransferases, hepatotoxicity, and liver failure. In clinical
studies, elevations in aminotransferases (ALT or AST) of at least
3-times the Upper Limit of Normal (ULN) have been observed in up to
2.5% of FILSPARI-treated patients, including cases confirmed with
rechallenge.
Measure transaminases and bilirubin before initiating
treatment and monthly for the first 12 months, and then every 3
months during treatment. Interrupt treatment and closely monitor
patients who develop aminotransferase elevations more than 3x Upper
Limit of Normal (ULN).
FILSPARI should generally be avoided in
patients with elevated aminotransferases (>3x ULN) at baseline
because monitoring for hepatotoxicity may be more difficult and
these patients may be at increased risk for serious
hepatotoxicity.
Embryo-Fetal Toxicity
FILSPARI can cause major birth defects
if used by pregnant patients based on animal data. Therefore,
pregnancy testing is required before the initiation of treatment,
during treatment and one month after discontinuation of treatment
with FILSPARI. Patients who can become pregnant must use effective
contraception before the initiation of treatment, during treatment,
and for one month after discontinuation of treatment with
FILSPARI.
Contraindications: FILSPARI is
contraindicated in patients who are pregnant. Do not coadminister
FILSPARI with angiotensin receptor blockers (ARBs), endothelin
receptor antagonists (ERAs), or aliskiren.
Warnings and
Precautions
Hepatotoxicity: Elevations in ALT or AST of at
least 3-fold ULN have been observed. To reduce the risk of
potential serious hepatotoxicity, measure serum aminotransferase
levels and total bilirubin prior to initiation of treatment,
monthly for the first 12 months, then every 3 months during
treatment.
Advise patients with symptoms suggesting
hepatotoxicity (nausea, vomiting, right upper quadrant pain,
fatigue, anorexia, jaundice, dark urine, fever, or itching) to
immediately stop treatment with FILSPARI and seek medical
attention. If aminotransferase levels are abnormal at any time
during treatment, interrupt FILSPARI and monitor as
recommended.
Consider re-initiation of FILSPARI only when
hepatic enzyme levels and bilirubin return to pretreatment values
and only in patients who have not experienced clinical symptoms of
hepatotoxicity.
Avoid initiation of FILSPARI in patients with
elevated aminotransferases (>3x ULN) prior to drug
initiation.
- Embryo-Fetal
Toxicity: FILSPARI can cause fetal harm. Advise
patients who can become pregnant of the potential risk to a fetus.
Obtain a pregnancy test and advise patients who can become pregnant
to use effective contraception prior to, during, and one month
after discontinuation of FILSPARI treatment.
- FILSPARI
REMS: FILSPARI is available only through a
restricted program under a REMS called the FILSPARI REMS.Important
requirements include:
- Prescribers must be certified with
the FILSPARI REMS by enrolling and completing training.
- All patients must enroll in the
FILSPARI REMS prior to initiating treatment and comply with
monitoring requirements.
- Pharmacies that dispense FILSPARI
must be certified with the FILSPARI REMS and must dispense only to
patients who are authorized to receive FILSPARI.
Further information is available at
www.filsparirems.com or 1-833-513-1325.
Please see Full Prescribing Information
for FILSPARI here
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 “on-track”,
“positioned”, “look forward to”, “will,” “would,” “may”, “might”,
“believes”, “anticipates”, “plans”, “expects”, “intends,”
“potential” 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: continued progress with the FILSPARI
launch and preliminary estimates of metrics related thereto; the
planned submission of an sNDA for full approval of FILSPARI and the
anticipated timing and outcome thereof; statements regarding the
potential approval of sparsentan for the treatment of IgAN in
Europe and the anticipated timing thereof; the potential for
FILSPARI and pegtibatinase to become new treatment standards in
IgAN and HCU; additional development and regulatory milestones,
including expected data from additional studies; planned additional
analyses of FSGS data and plans and timing for re-engaging with
regulators; the advancement of the Company’s pipeline throughout
the year; expectations regarding the Phase 3 HARMONY Study; the
potential inclusion of FILSPARI in the KDIGO Clinical Practice
Guideline for the Management of Glomerular Diseases; statements
regarding financial metrics, preliminary estimates thereof, and
expectations related thereto, including but not limited to
statements regarding net product sales from continuing operations,
revenue, cash balances and cash runway. 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, as well as risks
and uncertainties associated with the Company’s business and
finances in general and its recently announced strategic
reorganization, the success of its commercial products and risks
and uncertainties associated with the Company’s preclinical and
clinical stage pipeline. Specifically, the Company faces risks
associated with market acceptance of its commercial products
including efficacy, safety, price, reimbursement, and benefit over
competing therapies, as well as risks associated with the
successful development and execution of commercial strategies for
such products, including FILSPARI. 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
anticipated future clinical trials will not proceed as planned.
Specifically, the Company faces risks related to the timing and
potential outcome of its Phase 3 HARMONY Study, the timing and
potential outcome of its planned sNDA submission for full approval
of sparsentan in IgAN, and the risk that the results from the Phase
3 DUPLEX Study of sparsentan in FSGS will not serve as a basis for
a regulatory submission for approval of sparsentan for FSGS. There
is no guarantee that regulators will grant full approval of
sparsentan for IgAN or FSGS. The Company also faces the risk that
its cash runway might not last as long as currently anticipated and
the 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, including as a result of macroeconomic
conditions; risks 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 also faces additional risks associated with
global and macroeconomic conditions, including health epidemics and
pandemics, including risks related to potential disruptions to
clinical trials, commercialization activity, supply chain, and
manufacturing operations. 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, including under the
heading “Risk Factors”, as included in the Company’s most recent
Form 10-K, Form 10-Q and other filings with the Securities and
Exchange Commission.
Contact:Investors:888-969-7879
ir@travere.com |
Media:888-969-7879 mediarelations@travere.com |
Travere Therapeutics (NASDAQ:TVTX)
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