Travere Therapeutics, Inc. (TVTX) is a fully integrated biopharmaceutical company focused on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The company made significant progress in the first quarter of 2024, executing on key priorities that will drive sustainable growth.
Business Overview
The company's commercial launch of FILSPARI in the U.S. continued to reach new heights. In the first quarter of 2024, Travere reported net product sales of $39.984 million, up from $24.178 million in the same period of 2023. This increase was primarily driven by a full quarter of FILSPARI sales, which launched in February 2023. The company saw strong demand for FILSPARI, with 511 new patient start forms in the first quarter, up from 459 in the fourth quarter of 2023. Travere also saw improvements in the time it takes for patients to enroll in the REMS program and receive reimbursement, with 80% of patients enrolled within 14 days of the patient start form being received.
Travere also achieved multiple regulatory milestones with FILSPARI. The company's submission to convert FILSPARI from accelerated approval to full approval was granted priority review by the FDA, with a PDUFA target action date of September 5, 2024. Outside of the U.S., Travere's partner, CSL Vifor, recently received conditional marketing authorization (CMA) for FILSPARI in Europe for the treatment of adults with primary IgAN. Travere's partner in Japan, Renalys, also made progress with an IND submission and advancing their study to support potential approval in Japan and other Asia Pacific regions.
In addition to the progress with FILSPARI, Travere advanced its pipeline, including the pivotal Phase 3 HARMONY study of pegtibatinase for the treatment of classical homocystinuria (HCU). The company recently dosed the first patients in the HARMONY study and remains on track to report top-line results in 2026. Pegtibatinase is the only late-stage development candidate for HCU and the only medicine in development that has the potential to be disease-modifying.
Financials
Travere's financial performance in the first quarter of 2024 reflected the progress made across the business. The company reported total revenue of $41.374 million, up from $30.888 million in the same period of 2023. This increase was driven by the growth in net product sales, partially offset by a decrease in license and collaboration revenue. Research and development expenses were $49.420 million, down from $58.162 million in the prior year period, reflecting the company's focus on expense management. Selling, general and administrative expenses were $64.223 million, down from $65.950 million in the same period of 2023.
The company reported a net loss, including from discontinued operations, of $136.061 million, or $1.76 per basic share, in the first quarter of 2024, compared to a net loss of $86.331 million, or $1.27 per basic share, in the same period of 2023. The increase in net loss was primarily due to a one-time in-process research and development (IPR&D) expense of $65.205 million recognized in the first quarter of 2024, related to the achievement of the first patient dosed in the Phase 3 HARMONY study of pegtibatinase.
Liquidity
As of March 31, 2024, Travere had cash, cash equivalents and marketable securities of $441 million. The company believes its current strong cash balance, expected significant growth in FILSPARI, continued expense management and anticipated incoming future milestone payments will support its operations until 2028.
Outlook
Looking ahead, Travere is focused on executing on its key priorities in 2024, which include continuing to drive the commercial success of FILSPARI, advancing its pipeline, and maintaining a strong financial position. The company expects to see continued revenue growth for FILSPARI throughout 2024, driven by increasing patient demand, new and repeat prescribers, faster time to reimbursement, and broader payer access.
In addition to the potential full approval of FILSPARI in the U.S., Travere is also anticipating the updated KDIGO guidelines, which are expected to further support the use of FILSPARI as the foundational treatment for IgAN patients. The company also believes that the additional clinical evidence it has generated, including the positive results from the SPARTAN study in newly diagnosed IgAN patients, will provide additional support for physicians to treat patients earlier with FILSPARI.
Travere's pipeline also holds significant promise, with the pivotal Phase 3 HARMONY study of pegtibatinase for the treatment of classical HCU progressing as planned. If approved, pegtibatinase could become the first disease-modifying therapy for this devastating rare condition, affecting an estimated 7,000 to 10,000 patients worldwide.
In terms of the company's financial position, Travere's strong cash balance, expected growth in FILSPARI, and anticipated milestone payments provide a solid foundation to support its operations and continued investment in its pipeline. The company's focus on expense management has also resulted in a reduction in research and development and selling, general and administrative expenses in the first quarter of 2024 compared to the prior year period.
Conclusion
Overall, Travere Therapeutics is well-positioned to drive sustainable growth in the years ahead. The company's successful execution on the commercial launch of FILSPARI, regulatory milestones, and pipeline advancement, combined with its strong financial position, make it an intriguing investment opportunity in the rare disease space.