Akebia Therapeutics, Inc.

Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company focused on developing and commercializing innovative therapeutics for patients impacted by kidney disease. The company's core business model revolves around generating revenue from two commercial products, Vafseo and Auryxia, and advancing a pipeline of mid-stage and early-stage programs. Revenue is generated through product sales in the U.S. and through license and collaboration agreements with partners for sales in international territories, which include upfront payments, commercial milestone payments, and tiered royalties on net sales ¹. The primary customer segments for Vafseo and Auryxia in the U.S. are dialysis organizations, with the top five U.S. dialysis organizations treating 82% of the total dialysis patient population ².

The company's product portfolio includes Vafseo (vadadustat) and Auryxia (ferric citrate). Vafseo is an orally administered medicine approved by the U.S. FDA in March 2024 for the treatment of anemia due to CKD in adult patients on dialysis for at least three months ³. The U.S. market opportunity for this indication is approximately $1 billion based on current erythropoiesis stimulating agent (ESA) pricing ⁴. Vafseo became available in the U.S. in January 2025, with commercial supply agreements in place with dialysis organizations caring for nearly 100% of dialysis patients in the U.S. ⁵. As of December 31, 2025, approximately 290,000 dialysis patients in the U.S. have prescribing access to Vafseo ⁶. Vafseo is also approved for use in adults in 37 countries and marketed by partners outside the U.S. ⁷. Auryxia (ferric citrate) is an orally administered medicine approved and marketed in the U.S. for two indications: control of serum phosphorus levels in adult patients with dialysis-dependent chronic kidney disease (DD-CKD) and treatment of iron deficiency anemia (IDA) in adult patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) ⁸. Auryxia reached loss of exclusivity (LoE) in March 2025 ⁹. Ferric citrate is also approved and marketed by partners in certain countries outside the U.S. ¹⁰.

For the fiscal year ended December 31, 2025, Akebia Therapeutics, Inc. reported a net loss of $5.3 million ¹¹. The company had an accumulated deficit of $1.7 billion as of December 31, 2025 ¹². Cash and cash equivalents stood at $184.8 million as of December 31, 2025 ¹³. During the year ended December 31, 2025, the company recorded $5.7 million in license revenue related to royalties earned on net sales of Riona in Japan ¹⁴ and $11.3 million in royalties due to Panion relating to sales of Auryxia in the U.S. and Riona in Japan ¹⁵.

In terms of year-over-year comparisons, the filing indicates that Auryxia has historically contributed meaningful revenue to the business ¹⁶. However, with Auryxia reaching LoE in March 2025 and the expectation of additional generic competition in 2026, the company anticipates an adverse impact on its revenue ¹⁷. The filing does not provide specific year-over-year revenue growth by segment or margin expansion/contraction figures for the reported fiscal period.

Significant operational developments during the period include the U.S. FDA approval of Vafseo in March 2024 and its commercial launch in January 2025 ¹⁸. The company secured reimbursement for Vafseo under the Transitional Drug Add-on Payment Adjustment (TDAPA) and established commercial supply agreements with dialysis organizations covering nearly 100% of dialysis patients in the U.S. ¹⁹. In November 2025, Akebia acquired AKB-097, an anti-C3d-Factor H fusion protein complement inhibitor, through an asset purchase agreement with Q32 Bio Inc. ²⁰. The company also initiated a Phase 2 clinical trial for praliciguat, an oral sGC stimulator, for the treatment of biopsy-confirmed focal segmental glomerulosclerosis (FSGS) in December 2025 ²¹. In October 2025, the company announced it would not initiate the VALOR trial for Vafseo in NDD-CKD patients due to FDA feedback indicating a need for a significantly larger and more costly trial ²².

The company believes its existing cash resources and the cash it expects to generate from product, royalty, supply, and license revenues are sufficient to fund its current operating plan for at least two years, including to commercialize Vafseo and Auryxia and advance existing programs ²³. However, the company anticipates continued net losses and cannot guarantee when, if ever, it will become and remain profitable ²⁴.

A major growth area for the company is driving Vafseo to be the standard of care for the treatment of anemia due to CKD for patients on dialysis in the U.S. ²⁵. Vafseo, an oral HIF-PH inhibitor, became available in the U.S. in January 2025, with commercial supply agreements in place with dialysis organizations caring for nearly 100% of dialysis patients ²⁶. As of December 31, 2025, approximately 290,000 dialysis patients in the U.S. have prescribing access to Vafseo ²⁷. The company's strategy includes increasing prescribing access and the breadth and depth of prescribing among dialysis organizations, particularly targeting home dialysis patients as an underserved group ²⁸. The company also plans to generate additional data to educate physicians on Vafseo's potential clinical benefits and differentiation ²⁹. Enrollment was completed in the Vafseo Outcomes In-Center Experience (VOICE) collaborative clinical trial with USRC in June 2025, with top-line data expected in early 2027 ³⁰. The VOCAL trial, a post-marketing study in DaVita clinics, has enrolled 353 patients, with top-line data expected in late 2026 ³¹.

Another growth area is building on the company's commitment to kidney disease patients through its mid-stage rare kidney disease pipeline assets, praliciguat and AKB-097 ³². Praliciguat, an oral sGC stimulator, is being evaluated for the treatment of biopsy-confirmed focal segmental glomerulosclerosis (FSGS) in a Phase 2 clinical trial, with the first patient dosed in December 2025 ³³. The company also plans to assess praliciguat in other rare podocytopathies ³⁴. AKB-097, an anti-C3d-Factor H fusion protein complement inhibitor acquired in November 2025, is intended for a wide range of complement-mediated rare kidney diseases ³⁵. A Phase 2 basket study for AKB-097 in IgA Nephropathy (IgAN), C3 Glomerulopathy (C3G), and Lupus Nephritis (LN) is expected to initiate in the second half of 2026, with initial data anticipated in 2027 ³⁶.

The company's early-stage pipeline includes AKB-9090 and AKB-10108, which are HIF molecules ³⁷. AKB-9090 is planned for initial evaluation in cardiac surgery-related acute kidney injury (CS-AKI), with a Phase 1 study in healthy volunteers expected to initiate in the first half of 2026 ³⁸. The company may also study AKB-9090 in acute respiratory distress syndrome (ARDS) ³⁹. AKB-10108 is in preclinical development and will potentially be evaluated for retinopathy of prematurity (ROP) in neonates and other indications ⁴⁰.

Operationally, the company expects to continue to incur substantial additional operating expenses, including additional R&D expenses related to its pipeline, including AKB-097 and praliciguat, and additional R&D and selling, general and administrative expenses for ongoing development, post-marketing requirements, and commercialization of Auryxia and Vafseo ⁴¹. The company's ability to achieve and maintain profitability depends on managing these expenses and the overall success of its products and pipeline ⁴². The company has halted further enrollment in a post-approval pediatric study for Auryxia's Hyperphosphatemia Indication due to slow enrollment and retention, and has requested a meeting with the FDA for further discussions ⁴³.

Planned capital allocation includes continued investment in R&D for its pipeline, including AKB-097 and praliciguat ⁴⁴. Under the Q32 Purchase Agreement, the company made an upfront payment of $7.0 million on the APA Closing Date ⁴⁵ and will make an additional upfront payment of $3.0 million on the sixth-month anniversary of the APA Closing Date ⁴⁶. Milestone payments for AKB-097 could aggregate up to $94.5 million for development and regulatory events, including a $2.0 million development milestone payment upon the earlier of initiation of a Phase 2 clinical trial and December 31, 2026 ⁴⁷. Commercial milestone payments for AKB-097 could aggregate up to $487.5 million ⁴⁸, and royalty payments will range from low single digits to mid-teen percentages of net sales ⁴⁹. In February 2026, a $1.0 million regulatory milestone payment was made to Cyclerion for praliciguat upon the first patient dosed in a Phase 2 clinical trial in the U.S. ⁵⁰. Cyclerion is eligible for up to an aggregate of $197.5 million in additional specified development and regulatory milestone payments for praliciguat ⁵¹, as well as tiered royalties ranging from a mid-single-digit percentage to twenty percent of net sales ⁵². The company also sold 9,437,364 shares of common stock under an at-the-market offering with gross proceeds of $18.7 million during the year ended December 31, 2025 ⁵³. Additionally, on March 21, 2025, 25,000,000 shares of common stock were sold in an underwritten public offering with net proceeds of $46.5 million ⁵⁴, and on April 22, 2025, an additional 850,000 shares were sold with net proceeds of $1.6 million ⁵⁵.

The company explicitly flagged several structural headwinds and execution risks. The loss of exclusivity for Auryxia in March 2025 and the expectation of additional generic competition in 2026 are expected to adversely impact Auryxia revenue ⁵⁶. The company's ability to maintain contracts with dialysis organizations for the sale of Auryxia and Vafseo on favorable terms is critical ⁵⁷. There is a risk that, in the post-TDAPA period for Vafseo, reduced reimbursement could limit provider adoption, restrict patient access, and adversely impact revenue, especially if the Kidney Care Access Protection Act (KCAPA) is not adopted ⁵⁸. The company remains cautious about a path forward for Vafseo in smaller subpopulations of CKD non-dialysis dependent patients due to FDA feedback ⁵⁹. The ongoing litigation regarding CMS's plan to include oral-only phosphate lowering therapies in the ESRD PPS could also reduce anticipated revenue for Auryxia if Ardelyx is successful in its claims ⁶⁰. Geopolitical events, such as tariffs, rising inflation, increasing interest rates, slower economic growth or recession, global trade policies, global supply chain disruptions, ongoing conflicts including the Russia-Ukraine war, hostilities between Israel and Hamas, instability in the Middle East, and tensions between China and Taiwan, could negatively impact the company's ability to raise capital and its business operations ⁶¹.

The company faces material risks including significant losses since inception and the anticipation of continued losses, with an accumulated deficit of $1.7 billion as of December 31, 2025 ⁶². There is a substantial need for additional financing, and failure to obtain it could force delays or termination of product development or commercialization efforts ⁶³. Raising capital may dilute existing stockholders, restrict operations, or require relinquishing rights to products on unfavorable terms ⁶⁴. The company's obligations under the BlackRock Credit Agreement, including financial covenants requiring maintenance of cash and cash equivalents greater than or equal to $15.0 million or consolidated revenue of $150.0 million for the trailing twelve-month period ⁶⁵, could adversely affect financial condition and restrict operations ⁶⁶. The Royalty Interest Acquisition Agreement with HCR also contains covenants that, if violated, could materially adversely affect financial condition ⁶⁷. The business is substantially dependent on the commercial success of Auryxia and Vafseo, and inability to maintain successful commercialization, including contracts with dialysis organizations, will materially harm results ⁶⁸. The loss of exclusivity for Auryxia in March 2025 and expected additional generic competition in 2026 will adversely impact revenue ⁶⁹. The company faces substantial competition from other pharmaceutical and biotechnology companies ⁷⁰. International operations for ferric citrate and Vafseo expose the company to risks such as political and economic instability, changes in reimbursement policies, and diminished intellectual property protection ⁷¹. Clinical drug development is lengthy and expensive with uncertain outcomes, and delays or failures in trials for product candidates like praliciguat and AKB-097 are possible ⁷². Undesirable side effects or other properties of products, such as the increased risk of thromboembolic events and drug-induced liver injury noted in Vafseo's label ⁷³, may delay or prevent marketing approval or limit commercial potential ⁷⁴. Obtaining marketing approval for label expansion for Vafseo or for other product candidates is uncertain and may be significantly delayed ⁷⁵. The company may not obtain orphan drug exclusivity for praliciguat or future product candidates, and even if obtained, it may not prevent competition ⁷⁶. Products are subject to extensive post-marketing regulatory requirements, including post-approval pediatric studies for Auryxia and Vafseo, and non-compliance could lead to penalties or market withdrawal ⁷⁷. Compliance with complex regulatory schemes, including fraud and abuse laws, anti-kickback statutes, and false claims laws, requires significant resources, and failure to comply could result in costly investigations, fines, or sanctions ⁷⁸. Legislative and regulatory healthcare reform, such as the Inflation Reduction Act of 2022, may increase the difficulty and cost of obtaining approval and affect pricing ⁷⁹. Dependence on third-party collaborations for Auryxia, Riona, and Vafseo means that unsuccessful collaborations or terminations could materially harm the business ⁸⁰. Reliance on third parties to conduct clinical trials means their failure to perform could substantially harm the business ⁸¹. Inadequate intellectual property protection or challenges to existing rights could adversely affect the ability to compete ⁸².

Management's message to shareholders emphasizes a deep understanding of kidney disease and a commitment to serving the unmet needs of kidney patients, which informs business decisions and long-term planning. The company aims to leverage its two revenue-generating products and R&D capabilities to discover, advance, and commercialize new therapies, with a purpose to better the lives of those impacted by kidney disease. Three strategic imperatives are highlighted: driving Vafseo to be the standard of care for anemia due to CKD in dialysis patients in the U.S., building on the commitment to those impacted by kidney disease by supporting Auryxia and advancing the rare kidney disease pipeline, and creating a future for Akebia beyond kidney disease by leveraging HIF science expertise and exploring strategic growth opportunities. Management explicitly states that the company believes its existing cash resources and the cash it expects to generate from product, royalty, supply, and license revenues are sufficient to fund its current operating plan for at least two years ⁸³, including to commercialize Vafseo and Auryxia and advance existing programs. However, the company has incurred a net loss of $5.3 million for the year ended December 31, 2025 ⁸⁴, and anticipates continued losses, without guaranteeing when, if ever, it will become and remain profitable ⁸⁵.