Travere Therapeutics Provides Corporate Update and 2025 Outlook

Received 693 new patient start forms for FILSPARI$(R)$ (sparsentan) in the fourth quarter of 2024; approximately $50 million in preliminary net product sales of FILSPARI for the fourth quarter

sNDA requesting modification of liver monitoring for FILSPARI in IgAN accepted for review by FDA; PDUFA target action date of August 28, 2025

Company remains on track to provide regulatory update on sparsentan in FSGS by its fourth quarter 2024 earnings call

SAN DIEGO, Jan. 13, 2025 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that, based on preliminary and unaudited financial data, the Company expects net product sales for the fourth quarter of 2024 to be approximately $74 million. For the fiscal year 2024, the Company expects net product sales to be approximately $227 million. The Company ended 2024 with approximately $371 million in cash, cash equivalents, and marketable securities. The Company also provided an update on key corporate, clinical, and regulatory development initiatives, including anticipated 2025 milestones.

"The fourth quarter capped a tremendous year of execution for Travere. Following full approval of FILSPARI in September, the ongoing U.S. commercial launch resulted in nearly 700 new patient start forms in the fourth quarter as well as a 40% increase in FILSPARI net product sales compared to the third quarter," said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. "As we look ahead to the new year, we expect to help even more patients with IgAN through continued strong commercial execution, the final publication of the updated KDIGO guidelines and potential approval of the recently accepted sNDA to modify liver monitoring for FILSPARI. Beyond IgAN, we believe that sparsentan has the potential to become an important new medicine for people with FSGS, and we remain on track to provide an update on our interactions with FDA to establish a potential regulatory pathway for this additional indication by our fourth quarter 2024 earnings call."

Program Updates and Anticipated 2025 Milestones

FILSPARI(R) (sparsentan) -- IgA Nephropathy (IgAN)

   -- In the fourth quarter of 2024, the Company received 693 new patient start 
      forms (PSFs), driven by growth amongst new and repeat prescribers 
      following full approval by the U.S. Food and Drug Administration (FDA) on 
      September 5, 2024. 
   -- Preliminary net product sales of FILSPARI in the fourth quarter of 2024 
      were approximately $50 million; $132 million for the full year 2024. 
   -- The FDA recently accepted for review the Company's supplemental New Drug 
      Application (sNDA) requesting modification of liver monitoring for 
      FILSPARI in IgAN and assigned a PDUFA target action date of August 28, 
      2025. 
   -- In 2025, the Company anticipates final publication of the updated Kidney 
      Disease Improving Global Outcomes (KDIGO) clinical guidelines for IgAN. 
      The draft guidelines published in August 2024 recommended FILSPARI as a 
      foundational kidney-targeted therapy and lowered the targeted proteinuria 
      level for all IgAN patients to under 0.5 g/day or ideally complete 
      remission (under 0.3 g/day). 
   -- In 2025, the Company anticipates presenting additional data from its 
      ongoing clinical studies to further support FILSPARI as foundational 
      therapy in treating patients with IgAN. 
   -- The Company's collaborator CSL Vifor has launched FILSPARI for the 
      treatment of IgAN in Germany, Austria, and Switzerland. FILSPARI also 
      recently received approval in the UK. 
   -- In 2025, the Company and CSL Vifor anticipate the current conditional 
      marketing authorization $(CMA)$ for FILSPARI for the treatment of IgAN in 
      Europe will be converted to full approval. The Company expects to receive 
      a $17.5 million milestone payment from CSL Vifor upon conversion of the 
      CMA to full approval, and the Company remains eligible to receive 
      additional milestone payments related to market access and sales-based 
      achievements. 
   -- In the second half of 2025, Travere's partner Renalys Pharma, Inc. 
      expects topline results from its registrational Phase 3 clinical trial of 
      sparsentan for the treatment of IgA nephropathy in Japan. 

Sparsentan -- Focal Segmental Glomerulosclerosis $(FSGS)$

   -- The Company remains on track to provide an update on interactions with 
      the FDA regarding a potential regulatory pathway for a sparsentan FSGS 
      indication by its fourth quarter 2024 earnings call. 

Pegtibatinase -- Classical Homocystinuria (HCU)

   -- The Company is making progress on necessary process improvements in 
      manufacturing scale-up and is on track to restart enrollment in the Phase 
      3 HARMONY Study in 2026. 

The Company expects to announce complete full year 2024 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, 2024, 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, 2024, 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.

FILSPARI(R) (sparsentan) U.S. Indication

FILSPARI (sparsentan) is indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.

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.

Hepatotoxicity

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 3.5% 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 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), ERAs, or aliskiren.

Warnings and Precautions

   -- Hepatotoxicity: Elevations in ALT or AST of at least 3-fold ULN have been 
      observed in up to 3.5% of FILSPARI-treated patients, including cases 
      confirmed with rechallenge. While no concurrent elevations in bilirubin 
      >2-times ULN or cases of liver failure were observed in FILSPARI-treated 
      patients, some ERAs have caused elevations of aminotransferases, 
      hepatotoxicity, and liver failure. To reduce the risk of potential 
      serious hepatotoxicity, measure serum aminotransferase levels and total 
      bilirubin prior to initiation of treatment and 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.

(MORE TO FOLLOW) Dow Jones Newswires

January 13, 2025 07:01 ET (12:01 GMT)