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]

Summary of Akebia Therapeutics (AKBA) 2025 Conference Call Company Overview - **Company**: Akebia Therapeutics (AKBA) - **Product**: Vafsio, a treatment for anemia in dialysis patients - **Conference Date**: June 05, 2025 Key Points Product Launch and Sales Performance - **Successful Launch**: Vafsio had a strong first quarter launch with 640 physicians prescribing the product, indicating broad reach [2][4] - **Prescribing Depth**: Average of over 12 prescriptions per physician in Q1, indicating high adoption [4] - **Patient Population**: Broad prescribing across various patient types, not limited to initially identified profiles [4] Market Dynamics - **Payer Mix**: - 40% of prescriptions are from fee-for-service Medicare patients - 40% coverage from Medicare Advantage Plans, with an additional 20% coverage seen in Q1 [7][8] - **Home Patients**: Approximately 12% of prescriptions are for home patients, aligning with the dialysis community's demographics [7] Growth Strategy - **Transitional Drug Add-On Payment Adjustment (TDAPA)**: - Provides additional reimbursement outside the bundled payment system for dialysis treatments [17] - Expected to help cover costs of innovation during the two-year TDAPA period [18] - **Post-TDAPA Pricing**: Anticipated to be competitive at approximately $2,500 per year, targeting a billion-dollar market with 500,000 anemic patients [18][19] Clinical Trials and Data Generation - **VOICE Trial**: Aims to demonstrate a 10% reduction in hospitalization and mortality, crucial for economic viability [27] - **Enrollment Status**: 75% enrollment of 2,200 patients completed, with expectations to finish soon [32] Non-Dialysis Dependent (NDD) Market Opportunity - **Significant Unmet Need**: NDD population represents a multi-billion dollar market opportunity, with approximately 500,000 patients in the U.S. [25][23] - **FDA Engagement**: Plans to initiate a Phase 3 trial for NDD in the second half of the year, focusing on cardiovascular outcomes [38][40] Competitive Landscape - **Unique Position**: Akebia is the only company with a novel HIF inhibitor therapy in the U.S. market, differentiating itself from previous competitors [46][50] - **Clinical Profile**: Vafsio has a broader label without the heart failure warning present in previous products, enhancing its marketability [50] Financial Position - **Cash Reserves**: Ended Q1 with $113 million in cash, indicating a strong financial position to support ongoing operations and trials [72] - **Expense Management**: Efficient management of expenses, with a focus on reaching profitability while supporting pipeline activities [73][75] Pipeline and Future Opportunities - **Emerging Programs**: Plans to advance programs for acute kidney injury (AKI) and retinopathy of prematurity (ROP) into clinical trials [70][71] Additional Insights - **Market Dynamics**: The transition from traditional erythropoietin-stimulating agents (ESAs) to HIF inhibitors represents a significant shift in treatment paradigms for anemia management in chronic kidney disease [58] - **Physician Engagement**: Continuous engagement with physicians to highlight the benefits of Vafsio and its mechanism of action is crucial for adoption [58][59] This summary encapsulates the key insights and strategic directions discussed during the Akebia Therapeutics conference call, highlighting the company's growth potential and market positioning.

Core Insights - Akebia Therapeutics has published significant data regarding Vafseo (vadadustat) in the Journal of the American Society of Nephrology, focusing on its safety and efficacy in treating anemia related to chronic kidney disease (CKD) [1][2][4] Company Overview - Akebia Therapeutics, Inc. is a biopharmaceutical company dedicated to improving the lives of individuals affected by kidney disease, founded in 2007 and headquartered in Cambridge, Massachusetts [5] - The company aims to advance the understanding and treatment of kidney diseases through high-impact research [4] Product Information - Vafseo (vadadustat) is a once-daily oral medication approved by the U.S. FDA in March 2024 for treating anemia due to CKD in adults on dialysis for at least three months, and it has been available in the U.S. since January 2025 [3][6] - Vafseo functions as a hypoxia-inducible factor prolyl hydroxylase inhibitor, stimulating the production of erythropoietin to manage anemia [6] Clinical Trial Insights - The global phase 3 clinical trials for vadadustat were open-label, randomized, and noninferiority trials comparing its safety and efficacy to darbepoetin alfa in adult patients with CKD-related anemia [2] - In the U.S. patient subgroup with dialysis-dependent CKD, the safety and efficacy of vadadustat were found to be similar to darbepoetin alfa, while in non-dialysis-dependent CKD patients, vadadustat showed a higher risk for major adverse cardiovascular events (MACE) outside the U.S. [2] Research Publication - The analyses published in JASN reflect the importance of geographic-specific data in understanding treatment outcomes for CKD-related anemia, highlighting how regional differences can affect clinical trial results [4]

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 [3]. Upcoming Events - Akebia Executive Management will participate in a Fireside Chat at the Jefferies Global Healthcare Conference on June 5, 2025, at 8:45 AM EDT [1]. - The Jefferies Global Healthcare Conference is scheduled to take place from June 3-5, 2025, in New York City [2]. Investor Relations - A webcast of the presentation will be available in the "Investors" section of Akebia's website following the conference [1].

Core Insights - Akebia Therapeutics, Inc. will present clinical data on Vadadustat at the 62nd European Renal Association Congress from June 4-7, 2025, in Vienna, both virtually and live [1] Company Overview - Akebia Therapeutics is a biopharmaceutical company founded in 2007, headquartered in Cambridge, Massachusetts, focused on improving the lives of individuals affected by kidney disease [2] - The company is fully integrated and aims to address the needs of patients with chronic kidney disease (CKD) [2] Presentation Highlights - Dr. Glenn Chertow will present a win-ratio analysis of the cardiovascular safety of Vadadustat in patients with CKD-related anemia undergoing dialysis on June 6 at 8:15 am CEST [2] - An e-poster will discuss the combination of Vadadustat with Ferric Citrate, highlighting its protective effects against inflammation-induced anemia and anemia caused by chronic kidney disease [2] - Insights from a Japanese database study on the real-world use of HIF-PH inhibitors and erythropoiesis-stimulating agents (ESAs) in non-dialysis-dependent CKD with anemia will also be presented [2]

Financial Data and Key Metrics Changes - The first quarter marked the initial launch of Vafsio in the U.S. for anemia in CKD patients receiving dialysis, with nearly 640 physicians having prescribed the product by the end of the quarter [8][9] - Average prescriptions per prescriber were about 12, indicating a transition from trial to adoption [9] Business Line Data and Key Metrics Changes - Vafsio is positioned as a hypoxia-inducible factor prolyl hydroxylase inhibitor, with a focus on becoming the standard of care for all chronic kidney disease patients, including those not on dialysis [4][5] - Auryxia, another commercial product, is a phosphate binder that has positively impacted patients [3] Market Data and Key Metrics Changes - The dialysis market is significant, with 150,000 patients in small and medium providers, and all five top dialysis providers, representing approximately 85% of treatment, have ordered the product [11][12] - The non-dialysis market for CKD anemia is equally substantial, with over 550,000 anemic patients in stages four and five, presenting a larger revenue opportunity compared to dialysis [55][56] Company Strategy and Development Direction - The company aims to expand its pipeline within kidney disease and explore applications of HIF technology beyond the kidney space [5][6] - The strategy includes engaging with the FDA for a path forward to make Vafsio available for non-dialysis patients [5][60] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early launch of Vafsio, highlighting the enthusiasm from nephrologists and the potential for broader adoption [7][19] - The company anticipates significant growth in Q4 as pilot programs expand, with expectations for increased prescriber engagement [46][48] Other Important Information - The TDAPA program allows for reimbursement outside the bundled payment system, facilitating the launch of Vafsio [36][38] - The company plans to initiate a Phase III trial for Vafsio in non-dialysis patients by the end of the year [60][61] Q&A Session Summary Question: How is the launch of Vafsio progressing? - The launch is progressing well, with nearly 640 physicians prescribing the product and an average of 12 prescriptions per prescriber [8][9] Question: What is the strategy for engaging with dialysis organizations? - The strategy involves creating contracts and protocols to ensure positive economics for dialysis organizations, which are financially liable for treatment costs [25][31] Question: What are the market opportunities for Vafsio? - The non-dialysis market presents a significant opportunity, with over 550,000 anemic patients in stages four and five, potentially yielding higher revenue compared to the dialysis market [55][56] Question: What are the timelines for upcoming trials? - The pilot for larger dialysis organizations is expected to start in Q3, with broader usage anticipated in Q4 [44][46]

Company Performance - Akebia Therapeutics reported quarterly earnings of $0.03 per share, exceeding the Zacks Consensus Estimate of a loss of $0.03 per share, and showing improvement from a loss of $0.09 per share a year ago, resulting in an earnings surprise of 200% [1] - The company posted revenues of $57.34 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 26.83%, compared to year-ago revenues of $32.61 million [2] - Over the last four quarters, Akebia has surpassed consensus EPS estimates two times and topped consensus revenue estimates twice [2] Stock Performance - Akebia Therapeutics shares have increased approximately 29.5% since the beginning of the year, contrasting with the S&P 500's decline of -4.3% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.04 on revenues of $44.05 million, and for the current fiscal year, it is -$0.12 on revenues of $192.75 million [7] Industry Outlook - The Medical - Drugs industry, to which Akebia belongs, is currently ranked in the top 26% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

Financial Data and Key Metrics Changes - Total revenues increased to $57.3 million in Q1 2025 from $32.6 million in Q1 2024, primarily driven by net product revenues which rose to $55.8 million from $31 million [22][25] - Net income for Q1 2025 was $6.1 million compared to a net loss of $18 million in Q1 2024, reflecting strong revenue growth [25][26] - Cost of goods sold decreased to $7.6 million in Q1 2025 from $11.6 million in Q1 2024, attributed to the full amortization of a non-cash charge related to Auryxia [23][24] Business Line Data and Key Metrics Changes - Vafcio generated $12 million in net product revenues in Q1 2025, exceeding guidance of $10 million to $11 million [6][22] - Auryxia net product revenues increased to $43.8 million in Q1 2025 from $31 million in Q1 2024, despite losing IP exclusivity [22][23] Market Data and Key Metrics Changes - The top five dialysis organizations have ordered Vafcio, with revenues primarily coming from small to mid-sized dialysis organizations treating approximately 150,000 patients [8][14] - The VOICE study is progressing towards full enrollment, with about 75% of the target 2,200 subjects enrolled [10] Company Strategy and Development Direction - The company aims to expand the use of Vafcio in both dialysis and non-dialysis patient populations, with plans to initiate a Phase III VALOR trial in the second half of 2025 [10][11] - The focus remains on increasing the breadth and depth of prescribing among current customers while unlocking new customers [21][27] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong early performance of Vafcio, highlighting the importance of prescriber education and patient retention [6][21] - The company is confident in its financial position and ability to achieve profitability based on its current operating plan [26][27] Other Important Information - The company ended Q1 2025 with $113.4 million in cash and cash equivalents, bolstered by a public offering that raised over $46 million [26] - The company has at least 12 months of Vafcio inventory on hand in the U.S. and does not expect significant tariff-related impacts [24] Q&A Session Summary Question: Update on timelines for adding Vafcio to protocols at large dialysis organizations - Management indicated that a large pilot is planned to operationalize protocols, with training expected to take about a month, followed by a pilot duration of two to three months [29][31][34] Question: Shift from Auryxia prescriptions to authorized generic - Management noted that the impact of the authorized generic has been minimal in Q1 2025, with expectations that it will not significantly affect business in Q2 [46][48][49] Question: Net price dynamics as large dialysis providers come on board - Management acknowledged that net prices may decrease as volumes increase, but there will be variability in pricing due to the competitive environment [53][55] Question: Demand drivers for Vafcio and reimbursement trends - Management highlighted that demand is driven by clinical efficacy and economic incentives, with positive trends in Medicare Advantage coverage [70][72][78] Question: Inventory stocking trends with large dialysis organizations - Management expects initial stocking for pilots but does not anticipate significant issues with inventory levels due to efficient distribution networks [84][88] Question: Market landscape for label expansion opportunities - Management expressed excitement about the non-dialysis market, which is comparable in size to the dialysis market, and emphasized the potential for significant revenue growth [90][92][94]

Financial Data and Key Metrics Changes - Total revenues increased to $57.3 million in Q1 2025 from $32.6 million in Q1 2024, driven by net product revenues of $55.8 million compared to $31 million in the prior year [22][25] - Net income for Q1 2025 was $6.1 million, a significant improvement from a net loss of $18 million in Q1 2024 [25] - Cost of goods sold decreased to $7.6 million in Q1 2025 from $11.6 million in Q1 2024, attributed to the absence of a non-cash amortization charge related to Auryxia [23][24] Business Line Data and Key Metrics Changes - Vafcio generated $12 million in net product revenues in Q1 2025, exceeding guidance of $10 million to $11 million [6][22] - Auryxia net product revenues increased to $43.8 million in Q1 2025 from $31 million in Q1 2024, despite the entry of an authorized generic [22][23] Market Data and Key Metrics Changes - The top five dialysis organizations have ordered Vafcio, with revenues primarily coming from small to mid-sized dialysis organizations treating approximately 150,000 patients collectively [7][14] - The VOICE study is progressing towards full enrollment, with about 75% of the target 2,200 subjects enrolled [9] Company Strategy and Development Direction - The company aims to expand the use of Vafcio in both dialysis and non-dialysis patient populations, with plans to initiate a Phase III VALOR trial in the second half of 2025 [10][11] - The focus remains on increasing the breadth and depth of prescribing among current customers while unlocking new customers [21][22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong early performance of Vafcio, highlighting the importance of prescriber education and patient retention [6][7] - The company is confident in its financial position and believes it is financed to achieve profitability based on its current operating plan [25][26] Other Important Information - The company has at least 12 months of Vafcio inventory on hand in the U.S. and does not expect significant tariff-related impacts [24] - The average compliance rate in dialysis is expected to be around 65%, which will influence refill rates over time [60] Q&A Session Summary Question: Update on timelines for adding Vafcio to protocols at large dialysis organizations - Management indicated that a large pilot is planned to operationalize protocols, with training expected to take about a month before broader rollout [29][31][34] Question: Shift from Auryxia prescriptions to authorized generic - Management noted that the impact of the authorized generic has been minimal in Q1 2025, with expectations of maintaining significant revenue until more generics are approved [47][50] Question: Net price dynamics as large dialysis providers come on board - Management acknowledged the competitive environment and indicated that net price per patient is expected to decrease as volumes increase, with some lumpiness in pricing [55][57] Question: Demand drivers for Vafcio and reimbursement trends - Management highlighted that demand is driven by clinical value and economic incentives, with positive trends in Medicare Advantage coverage [70][78] Question: Inventory stocking trends with large dialysis organizations - Management expects initial stocking for pilots but does not anticipate significant issues with inventory levels due to efficient distribution networks [84][86] Question: Market landscape for label expansion opportunities - Management expressed excitement about the non-dialysis market, estimating a significant patient population and potential revenue opportunities [90][92]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q Commission File Number 001-36352 (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________to__________ AKEBIA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) (State or other juris ...