Core Insights - Akebia Therapeutics reported a significant increase in net product revenues for Vafseo, reaching $14.3 million in Q3 2025, contributing to total net product revenues of $56.8 million for the quarter [1][12] - The company aims to make Vafseo the standard of care for treating anemia in dialysis patients, with expectations to quadruple prescribing access by year-end [2][4] - A recent post-hoc analysis presented at ASN Kidney Week indicated that patients receiving vadadustat had statistically more favorable outcomes in terms of all-cause mortality and hospitalization compared to those receiving erythropoiesis-stimulating agents (ESAs) [1][6] Financial Results - Total revenues increased to $58.8 million in Q3 2025 from $37.4 million in Q3 2024, driven by Vafseo and Auryxia sales [12] - Auryxia net product revenues were $42.5 million in Q3 2025, up from $35.6 million in Q3 2024, despite the loss of exclusivity in March 2025 [12] - The company reported a net income of $0.5 million in Q3 2025, a significant improvement from a net loss of $20.0 million in Q3 2024 [12][32] Operational Highlights - An operational pilot of Vafseo at DaVita is expected to complete in Q4 2025, with access anticipated for 275,000 patients by year-end [1][6] - The number of prescribers for Vafseo reached approximately 725 in Q3 2025, with an average of 12.7 prescriptions written per prescriber [6] - The average dose of refills for Vafseo increased by 5% over Q2 2025, indicating growing acceptance among healthcare providers [6] Regulatory Update - Akebia did not achieve alignment with the FDA on its proposed path for a Phase 3 clinical trial in non-dialysis patients and does not plan to initiate the VALOR clinical trial [4][29]

Core Insights - Akebia Therapeutics Inc. is experiencing a decline in stock price following an unsuccessful meeting with the U.S. FDA regarding the VALOR trial design for vadadustat, aimed at treating anemia in late-stage chronic kidney disease patients not on dialysis [1][4]. Company Developments - The company has decided not to initiate the VALOR trial and will not pursue a broad label for Vafseo for non-dialysis dependent chronic kidney disease patients [2]. - Vafseo is currently approved in the U.S. for treating anemia due to chronic kidney disease in adults who have been on dialysis for at least three months, with prescriptions starting in January 2025 [2]. Regulatory Feedback - Akebia completed a Type C meeting with the FDA, which indicated that regulatory alignment for the VALOR trial would necessitate a significantly larger patient population than initially proposed, leading to increased time and costs [3]. - The CEO of Akebia expressed disappointment with the meeting's outcome but stated that the decision not to pursue a broad label is in the best interests of shareholders [4]. Market Reaction - Following the news, Akebia Therapeutics shares fell by 25.71%, trading at $2.29 at the time of publication [4].

Core Viewpoint - Akebia Therapeutics does not plan to initiate the VALOR clinical trial for vadadustat to treat anemia in patients with late-stage chronic kidney disease (CKD) not on dialysis, following feedback from the U.S. FDA indicating a need for a larger patient population and increased time and costs for the trial [1][2][3] Company Overview - Akebia Therapeutics, Inc. is a biopharmaceutical company focused on improving the lives of individuals affected by kidney disease, founded in 2007 and headquartered in Cambridge, Massachusetts [4] Product Information - Vafseo® (vadadustat) is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor approved for treating anemia due to CKD in adults who have been on dialysis for at least three months, with approval in 37 countries [5] Regulatory Feedback - The FDA's feedback from the Type C meeting suggests that regulatory alignment for the VALOR trial would necessitate a significantly larger patient cohort than initially proposed, leading to increased time and costs [2][3] Future Directions - Despite the setback with the VALOR trial, the company remains committed to addressing the unmet need for oral anemia treatment in CKD patients not on dialysis and is exploring potential clinical trial designs for smaller subgroups of CKD patients [3]

Core Insights - Akebia Therapeutics has initiated a post-marketing study, the VOCAL trial, to evaluate the efficacy and safety of Vafseo (vadadustat) in patients with anemia due to chronic kidney disease (CKD) undergoing hemodialysis [1][2] - The trial aims to compare the three times per week dosing of Vafseo against standard erythropoiesis-stimulating agents (ESA) [1][2] - Vafseo was approved by the FDA in March 2024 and became available in the U.S. in January 2025 for treating anemia in adults on dialysis for at least three months [4] Study Design and Objectives - The VOCAL trial will enroll approximately 350 patients across 18 DaVita hemodialysis clinics, utilizing a 1:1 randomization method [2] - The study duration will be up to 33 weeks, including screening, treatment, and safety follow-up [2] - Primary endpoint is the change in hemoglobin levels, with secondary endpoints including serious adverse events, target hemoglobin range, and RBC transfusion rates compared to ESA treatment [2] Sub-Study and Research Focus - A sub-study will involve around 28 patients from three clinics, focusing on the impact of Vafseo on red blood cell (RBC) quality [3] - The sub-study aims to analyze RBC phenotypes, including deformability and resistance to oxidative stress, in collaboration with Vitalant Research Institute [3] Company Overview - Akebia Therapeutics is a biopharmaceutical company dedicated to improving the lives of individuals affected by kidney disease, founded in 2007 and headquartered in Cambridge, Massachusetts [5] - Vafseo is a once-daily oral medication that stimulates endogenous erythropoietin production to manage anemia [6] Regulatory and Safety Information - Vafseo is indicated for treating anemia due to CKD in adults on dialysis for at least three months, but it has not been shown to improve quality of life or fatigue [7] - The drug carries warnings for increased risks of death, myocardial infarction, stroke, and thrombotic events [8][10]

Vafseo Launch and Market Opportunity - Vafseo (vadadustat) has launched as a treatment for anemia due to chronic kidney disease (CKD) in adult patients on dialysis[8] - The U S market opportunity for Vafseo in the dialysis patient population is approximately $1 billion[8] - Akebia is pursuing label expansion of Vafseo for the late-stage non-dialysis CKD population, representing a multi-billion-dollar U S market opportunity[8] - Contracts are secured covering nearly 100% of U S dialysis patients for Vafseo[12, 20] Clinical and Patient Considerations - Current standard of care for dialysis patients (ESAs) has remained unchanged for 30 years, with nearly 25% of patients falling below target hemoglobin levels[13] - Approximately 20% of patients on dialysis are ESA hyporesponders, leading to higher hospitalization and mortality rates[13] - Market research indicates that over 2 out of 3 nephrologists identify an unmet need for anemia treatment in CKD, particularly for oral options and ESA-resistant patients[14] VOICE Trial and Label Expansion - The VOICE trial, with over 650 subjects enrolled as of January 10, 2025, is designed to demonstrate non-inferiority for all-cause mortality and superiority for a 10% reduction in all-cause hospitalization[23, 24] - A Phase 3 clinical trial for Vafseo in late-stage CKD patients not on dialysis is planned to begin in mid-2025[8, 46] Non-Dialysis Market - In patients with pre-hemodialysis Hb <9 0 g/dL (n=4855), 73 4% did not receive ESA pre-HD[26] - The U S non-dialysis market opportunity for anemia treatment is approximately $1 billion, driven by predominantly private and unbundled government payment structures[40, 41] HIF-based Pipeline - Akebia is advancing a HIF-based pipeline targeting high unmet needs, including ROP (rare disease) with a ~$1 5B+ U S market opportunity and ~30K patients[50]

Vafseo Launch and Market Opportunity - Vafseo (vadadustat) has launched as a treatment for anemia due to chronic kidney disease (CKD) in adult patients on dialysis[8] - The U S market opportunity for Vafseo in the dialysis patient population is approximately $1 billion[8] - Akebia is pursuing label expansion of Vafseo for the late-stage non-dialysis CKD population, representing a multi-billion-dollar U S market opportunity[8] - Contracts are secured covering nearly 100% of U S dialysis patients for Vafseo[12, 20] Clinical and Patient Considerations - Current standard of care for dialysis patients (ESAs) has remained unchanged for 30 years, with nearly 25% of patients falling below target hemoglobin levels[13] - Approximately 20% of patients on dialysis are ESA hyporesponders, leading to higher hospitalization and mortality rates[13] - Market research indicates that over 2 out of 3 nephrologists identify an unmet need for anemia treatment in CKD, particularly for oral options and ESA-resistant patients[14] Vafseo Advantages and Ongoing Trials - Vafseo is an oral HIF-PH inhibitor with the potential to become standard of care, offering a unique mechanism of action and convenient oral dosing[15] - The VOICE trial, with a target enrollment of ~2,200 patients, is underway to demonstrate Vafseo's non-inferiority for all-cause mortality and superiority for a 10% reduction in all-cause hospitalization[24] Non-Dialysis Market and Future Plans - In patients with pre-hemodialysis Hb <9 0 g/dL (n=4855), 73 4% did not receive ESA pre-HD[26] - A Phase 3 clinical trial is planned to begin in mid-2025 for Vafseo in patients with late-stage CKD anemia not on dialysis, targeting ~1,500 U S subjects[8, 46] Pipeline and Financial Outlook - Akebia expects its existing cash resources and cash from operations to be sufficient to fund its current operating plan for at least two years[8] - Akebia is advancing a HIF-based pipeline targeting high unmet needs, including ROP (~$1 5B+ U S market opportunity), ARDS (~$2B U S market opportunity), and CS associated AKI (~$1 5B+ U S market opportunity)[50]

Core Insights - Akebia Therapeutics reported strong initial sales for Vafseo® (vadadustat) with net product revenues of $12.0 million in Q1 2025, contributing to total net product revenues of $55.8 million for the quarter [1][12] - The company completed a $50 million public offering, enhancing its balance sheet with cash and cash equivalents of $113.4 million as of March 31, 2025 [1][12] - Akebia plans to initiate a Phase 3 clinical trial (VALOR) for vadadustat in late-stage CKD patients not on dialysis, expected to begin in the second half of 2025 [5][31] Vafseo U.S. Commercial Updates - Over 640 prescribers have written prescriptions for Vafseo, averaging nearly 12 prescriptions each, with about one-third being refills [5] - The top five dialysis organizations have placed orders for Vafseo, although mid-sized dialysis organizations are currently driving most of the revenue [5] - Akebia estimates it has at least 12 months of Vafseo inventory on hand in the U.S. without potential incremental tariff payments [5] Financial Results - Total revenues for Q1 2025 were $57.3 million, up from $32.6 million in Q1 2024, driven by Vafseo sales and increased Auryxia sales [12] - Auryxia net product revenues were $43.8 million in Q1 2025, compared to $31.0 million in Q1 2024 [12] - Net income for Q1 2025 was $6.1 million, a significant improvement from a net loss of $18.0 million in Q1 2024 [12] Key Business Highlights - Akebia's Vafseo was recommended for symptomatic anemia in adults undergoing dialysis for CKD by the U.K. National Institute for Health and Care Excellence (NICE) [5] - U.S. Renal Care continues enrollment in the VOICE clinical trial for Vafseo, now at 75% of the planned enrollment of approximately 2,200 patients [5] - The company is advancing its existing programs while pursuing label expansion for Vafseo [12][31]