Executive Summary / Key Takeaways

• Travere Therapeutics is establishing FILSPARI (sparsentan) as a foundational therapy in IgA nephropathy (IgAN) following full FDA approval, demonstrating strong commercial execution with significant revenue growth driven by its differentiated clinical profile and expanding label.
• The company is actively pursuing a potential new indication for FILSPARI in Focal Segmental Glomerulosclerosis (FSGS), having submitted an sNDA supported by existing clinical data and recent advancements in endpoint validation, representing a significant potential growth opportunity in an area of high unmet need.
• FILSPARI's dual endothelin and angiotensin receptor antagonist (DEARA) mechanism provides a key technological differentiator, offering clinically proven benefits in proteinuria reduction and kidney function preservation superior to historical standard of care, supporting its competitive positioning.
• While the pegtibatinase program for Classical Homocystinuria (HCU) faces a temporary delay due to manufacturing scale-up, it represents a potential future disease-modifying therapy in the pipeline, underscoring the company's commitment to rare metabolic diseases.
• Travere maintains a solid financial position, bolstered by recent financing and anticipated milestone payments, projecting declining operating cash use and a cash runway into 2028, providing flexibility to fund strategic priorities despite ongoing net losses.

Building a Foundational Franchise in Rare Kidney Disease

Travere Therapeutics is a biopharmaceutical company dedicated to identifying, developing, and delivering life-changing therapies for people living with rare kidney and metabolic diseases. Its strategy centers on advancing a pipeline of innovative programs, anchored by its lead commercial product, FILSPARI (sparsentan), and leveraging an established commercial infrastructure built over years of serving rare disease patients. The company's journey has involved strategic acquisitions, divestitures, and focused development efforts, culminating in its current position with a fully approved product in a significant rare kidney disease market and promising pipeline candidates.

At the core of Travere's approach is sparsentan, a molecule designed with a unique mechanism of action. Sparsentan is a dual endothelin angiotensin receptor antagonist (DEARA), simultaneously blocking two critical pathways, endothelin-1 and angiotensin II, known to contribute to glomerular injury in rare chronic kidney diseases. Pre-clinical data have shown that this dual blockade reduces proteinuria, protects podocytes, and prevents glomerulosclerosis and mesangial cell proliferation. This technological differentiation translates into tangible clinical benefits. In the Phase 3 PROTECT study in IgA nephropathy (IgAN), FILSPARI demonstrated a statistically significant treatment effect on kidney function decline over two years compared to irbesartan, with a mean eGFR slope difference of 1.2 mL/min/1.73 m²/year. It also showed rapid, profound, and durable reductions in proteinuria. This clinically proven superiority over a historical standard of care provides a strong competitive moat for FILSPARI.

The commercial launch of FILSPARI in the United States for IgAN has shown strong momentum, particularly following the full FDA approval in September 2024. Net product sales for FILSPARI reached $55.881 million in the first quarter of 2025, representing a significant 182% increase compared to the same period in 2024 and a 13% sequential growth over the fourth quarter of 2024. This performance is driven by increasing demand, expanding prescriber breadth and depth, high patient compliance and persistence rates, and improving efficiencies in the fulfillment process. The full approval removed the previous proteinuria threshold from the label, enabling the company to educate physicians on the comprehensive two-year PROTECT data, including long-term kidney function preservation. This broader label, coupled with anticipated updates to KDIGO guidelines expected to recommend earlier and more ambitious treatment targets for IgAN and highlight FILSPARI, is expected to further accelerate uptake and solidify FILSPARI's position as foundational care, replacing the traditional role of RAS inhibitors for many patients. While sales in Q1 2025 and Q1 2024 benefited from zero-cost inventory, the underlying demand trends indicate sustainable growth. International expansion is also underway through partnerships; CSL Vifor recently received standard marketing authorization for FILSPARI in Europe (April 2025) and has initiated launches, while Renalys Pharma (4582) is advancing development in Japan.

Beyond IgAN, Travere is actively pursuing a potential new indication for FILSPARI in Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disorder with no approved treatments and a high unmet need. The company submitted a supplemental New Drug Application (sNDA) to the FDA in March 2025, seeking priority review for traditional approval. This submission is supported by data from the Phase 3 DUPLEX and Phase 2 DUET studies, which demonstrated statistically significant proteinuria reduction and favorable trends in kidney function endpoints. While the DUPLEX study did not meet its primary eGFR endpoint statistically over two years, recent findings from the collaborative PARASOL initiative, which included input from regulatory bodies, suggest that proteinuria reduction over 24 months is strongly associated with a reduced risk of kidney failure in FSGS. Travere believes its clinical data align well with these findings, potentially validating proteinuria as a surrogate endpoint for traditional approval in FSGS. The company expects to receive notification regarding the FDA's acceptance of the sNDA and review timeline in the second quarter of 2025, with potential approval and launch as early as September 2025 if priority review is granted. The FSGS opportunity is viewed as potentially larger than IgAN, and Travere plans to leverage the significant overlap in the prescriber base with IgAN to drive rapid uptake if approved.

In the metabolic disease space, Travere is developing pegtibatinase for Classical Homocystinuria (HCU), a rare disorder with significant complications and no approved disease-modifying therapies. Pegtibatinase, a novel enzyme replacement candidate, has received multiple expedited designations from the FDA. The company initiated the pivotal Phase 3 HARMONY study in December 2023 but announced a voluntary pause in enrollment in September 2024 to address necessary manufacturing scale-up process improvements. While this introduces a delay, Travere remains committed to the program, viewing pegtibatinase as having the potential to be the first disease-modifying treatment for HCU. Enrollment is expected to restart in 2026.

Financially, Travere is focused on managing its resources to support these strategic priorities. Total revenue in the first quarter of 2025 was $81.732 million, up significantly from $41.374 million in the same period of 2024, driven by FILSPARI sales and an increase in license and collaboration revenue (including a $3.8 million API sale to CSL Vifor). Operating expenses include research and development ($46.889 million in Q1 2025 vs. $49.420 million in Q1 2024) and selling, general and administrative expenses ($72.838 million in Q1 2025 vs. $64.223 million in Q1 2024). The increase in SG&A reflects investment in the FILSPARI launch and intangible asset amortization. The company reported a net loss of $41.226 million in Q1 2025, compared to $136.061 million in Q1 2024 (which included a $65.2 million IPRD expense).

As of March 31, 2025, cash, cash equivalents, and marketable securities totaled $322.2 million. The company's liquidity is supported by recent financing, including $134.7 million net proceeds from a November 2024 public offering, and anticipated milestone payments, such as the $17.5 million expected from CSL Vifor in Q2 2025.

Travere projects declining operating cash use over time, driven by FILSPARI sales growth and reduced R&D spend as programs mature, which is expected to support current operations into 2028.

However, the company faces the maturity of its $68.90 million principal 2025 Convertible Notes in September 2025, which will require repayment or refinancing.

Generic competition for Thiola and Thiola EC continues to impact sales of these products.

In the competitive landscape, Travere's FILSPARI competes with other therapies in development or recently approved for IgAN, such as Calliditas Therapeutics (CALT)' Kinpeygo and Novartis (NVS)'s atrasentan. FILSPARI differentiates itself with its unique dual MOA and head-to-head superiority data against irbesartan, positioning it as a foundational therapy that can potentially be used alone or in combination with other modalities targeting immune pathways. While competitors may enter the market with accelerated approvals based on proteinuria, FILSPARI's full approval and two-year kidney function data provide a distinct advantage. In FSGS, the competitive landscape is less crowded, with no approved therapies, offering Travere a potential first-mover advantage if approved. In HCU, pegtibatinase aims to compete in a market with significant unmet need, potentially offering a disease-modifying approach compared to existing management strategies. Travere's ability to execute on its commercial strategy, advance its pipeline, and manage its financial resources will be critical in navigating this competitive environment and realizing its growth potential.

Conclusion

Travere Therapeutics is at a pivotal stage, successfully leveraging the full FDA approval of FILSPARI in IgAN to drive significant commercial growth and establish the therapy as a foundational treatment. The company's differentiated technology, demonstrated clinical superiority, and expanding label provide a strong basis for continued uptake in the IgAN market. The potential approval of FILSPARI for FSGS represents a substantial near-term growth opportunity that could significantly expand the addressable patient population and further solidify Travere's leadership in rare kidney diseases. While the pegtibatinase program faces temporary manufacturing challenges, it remains a promising long-term asset. With a solid balance sheet, declining operating cash burn, and anticipated milestone payments, Travere appears well-positioned to fund its strategic priorities and execute on key milestones in the coming year. Investors will be closely watching the FDA's decision on the FSGS sNDA, the progress in restarting pegtibatinase enrollment, and the continued trajectory of FILSPARI sales as indicators of the company's ability to deliver on its mission and create value.