R&D Day 2026 April 2, 2026 Bettering the Lives of People Impacted by Kidney Disease NASDAQ: AKBA 1 Cautionary note on forward-looking statements Statements in this presentation regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia's plans, strategies an ...

Core Insights - Akebia Therapeutics Inc. is highlighted as one of the top penny stocks with potential for significant returns, particularly focusing on its pipeline expansion and Vafseo data catalysts in the $1 billion dialysis market [1][3] Company Overview - Akebia Therapeutics specializes in developing and commercializing medicines for kidney diseases, with key products including Vafseo (vadadustat) and Auryxia [5] - The company is also advancing other treatments, such as AKB-10108 for retinopathy of prematurity and AKB-9090 for cardiac surgery-related acute kidney injury, currently in Phase 2 clinical trials [5] Financial Performance - For the year, Akebia reported a total of $227 million in net product revenue, with Vafseo and Auryxia being significant contributors [1] - The CEO noted that the commercialization of Vafseo marked an important year for the company, with expectations for it to become a standard of care in the U.S. market [3] Pipeline and Future Prospects - The company announced a pipeline for rare kidney diseases and scheduled an R&D day for April 2 to discuss mid-stage assets like praliciguat and AKB-097 [4] - Evidence presented at the ASN meeting indicated that patients treated with Vafseo had a lower risk of dying or being hospitalized compared to those treated with ESA comparators [4] Market Dynamics - The pricing strategy for Vafseo is expected to align with ESA pricing, which could enhance its market penetration in the $1 billion U.S. opportunity post-TDAPA [3] - Despite a recent price target reduction from BTIG, which lowered it from $5 to $4, the firm maintained a Buy rating, indicating confidence in the company's long-term outlook despite short-term inventory adjustments affecting Q4 revenue [4]

Core Insights - The company is focusing on three key strategic areas for capital allocation, with an emphasis on its commercial product, Vafseo, to establish it as the standard of care [1] - There are ongoing Phase IV/IIIb trials aimed at generating data necessary for Vafseo to become the standard of care [1] - The company is committed to advancing its initiatives in kidney disease [2]

Akebia Therapeutics Conference Call Summary Company Overview - **Company**: Akebia Therapeutics (NasdaqCM:AKBA) - **Focus**: Development of therapies for kidney disease, particularly anemia in chronic kidney disease patients Key Strategic Areas 1. **Commercial Product - Vafseo**: - Aim to establish Vafseo as the standard of care for anemia in chronic kidney disease patients on dialysis [3][7] - Ongoing phase four and three B trials to generate necessary data for standardization [3] 2. **Commitment to Kidney Disease**: - Focus on expanding the pipeline related to kidney disease, including drugs like praliciguat and AKB-907 [3][4] 3. **Future Beyond Kidney Disease**: - Small investments in other areas such as aducanumab and retinopathy of prematurity [4] Vafseo Product Insights - **Mechanism**: Vafseo is a HIF-PH inhibitor that offers a new approach to managing anemia, with fewer dose titrations and easier administration for physicians [7][8] - **Clinical Data**: - Phase 3 study showed a statistically significant reduction in mortality and hospitalization rates [8] - An 8% decrease in hospitalization and a 15% cost reduction per patient per year, translating to approximately $3.7 billion savings if all eligible patients switched from ESAs to Vafseo [9][10] - **Market Potential**: - Post-TDAPA market opportunity estimated at $1 billion, with potential for Vafseo to be a $500 million product [26][30] Dosing Strategy - **TIW Dosing**: - Transitioning to TIW (three times a week) dosing to improve patient adherence and ease of use for nephrologists [12][14] - Ongoing studies (VOICE and VOCAL) to validate TIW dosing effectiveness [15][37] Pipeline Developments 1. **Praliciguat**: - Targeting focal segmental glomerulosclerosis (FSGS) with a unique mechanism of action [49][50] - Phase 2 trials initiated, with a focus on demonstrating efficacy in a high unmet need area [50] 2. **AKB-097**: - A complement inhibitor with a tissue-targeted effect, expected to have a better safety profile and dosing regimen compared to existing treatments [51][52] - Basket study planned for multiple indications, including IgAN and lupus nephritis, with data expected in 2027 [53][54] Financial Metrics and Market Perception - **Investor Metrics**: - Focus on increasing the number of prescribers and adherence rates for Vafseo [60] - Balanced capital allocation to support both Vafseo and the pipeline [61] - **Market Understanding**: - Complexity of dialysis reimbursement may lead to underappreciation of Akebia's market potential [62] - Emphasis on the importance of consistent revenue growth and pipeline visibility to attract investor confidence [63][64] Conclusion - Akebia Therapeutics is positioned to leverage its innovative product Vafseo and a robust pipeline to address significant unmet needs in kidney disease, with a clear strategy for market penetration and growth potential in a billion-dollar market.

Financial Data and Key Metrics Changes - Total revenues increased to $57.6 million in Q4 2025 from $46.5 million in Q4 2024, and for the full year, revenues rose to $236.2 million from $160.2 million in 2024, driven by Vafseo and Auryxia sales [22][25] - Vafseo net product revenues were $6.2 million in Q4 2025 and $45.8 million for the full year, while Auryxia revenues were $48.1 million in Q4 2025 and $181.5 million for the full year [22][25] - Net loss decreased to $12.2 million in Q4 2025 from $22.8 million in Q4 2024, and for the year, net loss was $5.3 million compared to $69.4 million in 2024 [25] Business Line Data and Key Metrics Changes - Vafseo faced challenges in demand growth during the second half of 2025, with a slight decrease in Q4 demand compared to Q3, attributed to a transition to an observed dosing protocol [5][16] - Auryxia sales increased, but the company anticipates a decrease in Auryxia revenues in 2026 due to expected generic competition [23] Market Data and Key Metrics Changes - Over 1,000 prescribers at 24 dialysis organizations have written prescriptions for Vafseo, with 290,000 patients having access to it in dialysis clinics [6][17] - The company noted a significant increase in prescriber access and improved adherence rates among patients on observed dosing regimens [18][19] Company Strategy and Development Direction - The strategy includes making Vafseo the standard of care in a $1 billion U.S. market, supported by ongoing clinical data generation [6][10] - The company is expanding its pipeline with rare kidney disease treatments, including praliciguat and AKB-097, which are expected to be significant value drivers [11][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth of Vafseo, despite previous challenges, and emphasized the importance of clinical data in influencing physician adoption [33][40] - The company plans to host an R&D day to discuss mid-stage assets and the potential of its pipeline [70][71] Other Important Information - Cash and cash equivalents as of December 31, 2025, were $184.8 million, compared to $51.9 million at the end of 2024, indicating a solid financial position [26] Q&A Session Summary Question: Expectations for sequential Vafseo growth in 2026 - Management did not provide specific revenue guidance but indicated that demand has been flat and they expect growth from current levels [29][33] Question: Importance of VOICE study data for uptake - Management highlighted that published data will significantly influence physician utilization and adoption of Vafseo [32][33] Question: Current operationalization of Vafseo access at DaVita - DaVita has made Vafseo widely available and is focusing on educating physicians, particularly in the home dialysis population [34][38] Question: Trends in second and third refill rates - First refill adherence improved significantly, and management noted ongoing positive trends in adherence rates [45] Question: Differentiation of AKB-9090 from prior HIFs - AKB-9090 has a different pharmacokinetic profile, allowing for broader tissue penetration compared to vadadustat [51] Question: Expectations for R&D day on April 2 - The company plans to provide detailed insights into its pipeline, focusing on praliciguat and AKB-097, and will include external experts to discuss the potential of these products [70][71]

Financial Data and Key Metrics Changes - Total revenues increased to $57.6 million in Q4 2025 from $46.5 million in Q4 2024, and for the full year, revenues rose to $236.2 million from $160.2 million in 2024, driven by Vafseo and Auryxia sales [22] - Vafseo net product revenues were $6.2 million in Q4 2025 and $45.8 million for the full year, while Auryxia revenues were $48.1 million in Q4 2025 compared to $44.4 million in Q4 2024 [22] - Net loss decreased to $12.2 million in Q4 2025 from $22.8 million in Q4 2024, and for the year, net loss was $5.3 million compared to $69.4 million in 2024 [25] Business Line Data and Key Metrics Changes - Vafseo faced challenges in demand growth during the second half of 2025, with a slight decrease in Q4 demand leading to $6.2 million in net product revenue [5][16] - Auryxia sales increased, contributing to overall revenue growth, but the company anticipates a decrease in Auryxia revenues in 2026 due to expected generic competition [23] Market Data and Key Metrics Changes - Vafseo has gained access to 290,000 patients in dialysis clinics, with over 1,000 prescribers at 24 different dialysis organizations writing prescriptions [6][17] - The company noted a shift in prescriber dynamics and improved adherence rates, with first refill adherence rising from approximately 75% to 91% among patients on an observed dosing regimen [18] Company Strategy and Development Direction - The company aims to establish Vafseo as the standard of care in a $1 billion U.S. market opportunity, focusing on generating data to support its benefits over existing treatments [6][10] - Akebia is expanding its pipeline with new rare kidney disease products, including praliciguat and AKB-097, which are expected to drive future growth [11][12] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth potential for Vafseo in 2026, despite previous challenges, and emphasized the importance of ongoing data generation to support product adoption [5][33] - The company is confident in its ability to fund operations for at least the next two years, with cash and cash equivalents at $184.8 million as of December 31, 2025 [26] Other Important Information - The company plans to host an R&D day on April 2, 2026, to discuss mid-stage assets and provide insights into its pipeline [11][70] - The VOCAL study is expected to provide important data regarding Vafseo's efficacy compared to existing treatments, which could influence its adoption [61] Q&A Session Summary Question: Expectations for sequential Vafseo growth in 2026 - Management noted that while they are not guiding for revenue, they expect growth from current demand levels, which have been flat recently [29][33] Question: Operationalization of Vafseo access at DaVita - DaVita has made Vafseo widely available and is focusing on educating physicians, particularly in the home dialysis population [34][38] Question: Trends in second and third refill rates - First refill adherence has improved significantly, and there are positive trends in adherence rates for subsequent prescriptions [45] Question: Differentiation of AKB-9090 from prior HIFs - AKB-9090 has different pharmacokinetics and broader tissue penetration compared to vadadustat, which may enhance its efficacy in certain indications [51] Question: Expectations for the R&D day on April 2 - The company plans to provide detailed insights into its pipeline, focusing on praliciguat and AKB-097, and will include external experts to discuss the products [68][70]

Core Viewpoint - Akebia Therapeutics Inc. is identified as a promising growth stock for the next 20 years, despite recent adjustments in price targets by analysts [1]. Financial Performance - In Q3, Akebia reported total revenue of $58.8 million and a net income of $540,000, marking a significant turnaround from a net loss of $20 million in Q3 2024 [2]. - The revenue from the dialysis drug Vafseo was lower than expected at $14.3 million due to operational challenges, but the company expanded patient access to 70,000 and achieved over 700 active prescribers [2]. Future Outlook - Management anticipates a temporary revenue dip in Q4 due to inventory adjustments at US Renal Care, but projects growth to resume in Q1 2026 [3]. - The company is focused on its 2026 pipeline, including the initiation of a Phase 2 study for rare kidney diseases, and asserts that current cash reserves are adequate to support its path to sustained profitability [3].

Market Overview - The S&P 500 and Dow have risen for nine consecutive months on a total return basis despite rising geopolitical tensions [2] - A potential 5% dip in the market is anticipated in the second half of February due to high optimism and put-to-call ratios, but the bull market remains intact with solid fundamentals [2] Technology Sector Insights - The market is experiencing indiscriminate selling, with a notable increase in short interest in the XLK (Tech ETF) reaching its highest level in years [3] - Software valuations are at their cheapest since 2013, presenting a buying opportunity, as the Carson Group has added to tech and software positions [3] Akebia Therapeutics Inc. (NASDAQ:AKBA) - Akebia reported total revenue of $58.8 million in Q3, with a net income of $540,000, recovering from a $20 million net loss in Q3 2024 [10] - The launch of its dialysis drug Vafseo generated lower-than-expected revenue of $14.3 million, but patient access expanded to 70,000 with over 700 active prescribers [10] - Management anticipates a temporary revenue dip in Q4 due to inventory adjustments but projects growth to resume in Q1 2026, focusing on its 2026 pipeline outlook [11] LiveRamp Holdings Inc. (NYSE:RAMP) - LiveRamp reported a revenue growth of 9% year-over-year to $212 million in FQ3 2026, exceeding expectations [14] - The company has formed a significant strategic partnership with Publicis and expanded its Data Marketplace to include AI models, positioning for double-digit growth in fiscal year 2027 [14] - Subscription net retention has compressed to 101%, and gross margins are expected to dip slightly, with a projected sequential increase in operating expenses of $15 million in FQ4 [15]

Company Overview - Akebia Therapeutics, Inc. (AKBA) primarily generates revenue through the commercialization of Vafseo, an oral therapy approved for treating anemia due to chronic kidney disease (CKD) in adults [1] Analyst Background - The analyst has a Master's degree in Cell Biology and extensive experience as a lab technician in drug discovery, providing a strong foundation in the challenges of drug development [1] - The analyst has been active in the investment space for five years, focusing on biotechnology companies that innovate through unique mechanisms, first-in-class therapies, or platform technologies [1] Investment Focus - The analyst aims to identify promising biotechnology companies and evaluates the science behind drug candidates, competitive landscape, clinical trial design, and market opportunities while balancing financial fundamentals and valuation [1] - The goal is to provide insights that help investors understand both opportunities and risks in the biotech sector, which can yield significant returns through breakthrough science [1]

分组1 - Akebia Therapeutics, Inc. (AKBA) primarily generates revenue through the commercialization of Vafseo, an oral therapy approved for treating anemia due to chronic kidney disease (CKD) in adults [1] - The company focuses on identifying promising biotechnology innovations, including novel mechanisms of action and first-in-class therapies, to reshape treatment paradigms [1] - The analysis emphasizes the importance of evaluating the science behind drug candidates, the competitive landscape, clinical trial design, and potential market opportunities while balancing financial fundamentals and valuation [1]