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Investors: Media: 888-969-7879 888-969-7879 ir@travere.com mediarelations@travere.com Travere Therapeutics Provides Corporate Update and 2024 Outlook Received 459 new patient start forms for FILSPARI (sparsentan

Key Takeaway: Travere Therapeutics provided a corporate update highlighting strong Q4 2023 performance with anticipated net sales of approximately $40 million and 459 new patient start forms for FILSPARI, indicating positive momentum. The company is preparing to submit a supplemental NDA to the FDA for full approval of FILSPARI in IgAN and expects a decision on potential European approval in early 2024. Additionally, the pivotal HARMONY Study for pegtibatinase has begun, aiming to position it as the first disease-modifying therapy for classical homocystinuria. Despite positive developments, preliminary results are unaudited, and the company must navigate the risks associated with FILSPARI's side effects.

Market Sentiment Analysis

POSITIVE FACTORS

  • 459 new patient start forms for FILSPARI indicate growing demand.
  • Expected $40 million in net product sales for Q4 2023.
  • Anticipation of converting FILSPARI from accelerated to full approval.
  • Initiation of pivotal HARMONY Study for pegtibatinase.

CONCERNS & RISKS

  • Preliminary financial results are unaudited and subject to revision.
  • Risks associated with hepatotoxicity and embryo-fetal toxicity of FILSPARI need to be monitored.
  • Approval of sparsentan in Europe is still pending.

Full Press Release Details

Contact:
Investors: Media:
888-969-7879 888-969-7879
ir@travere.com mediarelations@travere.com
Travere Therapeutics Provides Corporate Update and 2024 Outlook
Received 459 new patient start forms for FILSPARI (sparsentan) in the fourth
quarter of 2023; preliminary net product sales of FILSPARI totaled $15 million for the fourth quarter
submit sNDA to FDA in Q1 2024 for conversion of the existing U.S. accelerated approval of FILSPARI in IgAN to full approval
opinion on potential approval of sparsentan for the treatment of IgAN in Europe expected in Q1 2024
Pivotal Phase 3 study of
pegtibatinase in classical homocystinuria (HCU) underway
SAN DIEGO, January 8, 2024 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
Sparsentan Focal Segmental Glomerulosclerosis (FSGS)
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).
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.
(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 TOXICITY
Because 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.
Some 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.
Important requirements include:
Further information is available at www.filsparirems.com or 1-833-513-1325.
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
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

Frequently Asked Questions

What are the preliminary net sales for FILSPARI in Q4 2023?

Preliminary net product sales of FILSPARI totaled $15 million for Q4 2023.

When will Travere submit the sNDA for FILSPARI?

The sNDA for FILSPARI is expected to be submitted to the FDA in Q1 2024.

What therapy is pegtibatinase being developed for?

Pegtibatinase is being developed as a therapy for classical homocystinuria (HCU).

What is the indication for FILSPARI?

FILSPARI is indicated to reduce proteinuria in adults with IgAN at risk of disease progression.

What is a significant safety concern with FILSPARI?

FILSPARI has boxed warnings for hepatotoxicity and embryo-fetal toxicity.

Last updated: Jan 8, 2024