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]

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]

Akebia Therapeutics Conference Summary Company Overview - **Company**: Akebia Therapeutics (NasdaqCM:AKBA) - **Focus**: Improving the lives of individuals affected by kidney disease - **Commercial Products**: - Auryxia: A phosphate binder nearing the end of its life cycle - VAFSEO: A HIF-PHI for treating anemia in chronic kidney disease patients on dialysis, launched about a year ago [4][5] Key Points on VAFSEO - **Initial Uptake**: Strong initial uptake observed post-launch, but faced challenges due to the unique nature of the dialysis market [7][8] - **Market Dynamics**: Dialysis providers control the prescription process, affecting product availability [8] - **Discontinuation Rates**: Higher than expected due to initial hemoglobin dips in patients; efforts are being made to improve patient retention [10][12] - **Dosing Strategy**: Transitioning to a three times weekly (TIW) dosing regimen has shown promising results in reducing discontinuation rates [12][13] - **Patient Access**: By the end of 2025, access to approximately 275,000 patients was achieved [13] - **Long-term Growth**: Data supporting VAFSEO's potential to become the standard of care for anemia in dialysis patients is anticipated [14][24] Clinical Trials and Studies - **VOCAL and VOICE Trials**: - VOCAL: A study comparing VAFSEO to Mircera, expected to show excellent hemoglobin control [20][29] - VOICE: A randomized comparison against Epogen, with results expected early in 2027 [29][31] - **Economic Perspective**: Demonstrating reduced hospitalization costs could enhance the economic proposition for dialysis organizations [31] Pipeline Developments - **Rare Kidney Disease Pipeline**: - Praliciguat: Phase II study initiated for FSGS, targeting rapid progression to end-stage kidney disease [5][40] - AKB-097: A tissue-targeted complement inhibitor, with a basket study planned for IgAN, C3G, and lupus nephritis [40][58] - **Mechanistic Rationale**: Praliciguat stimulates soluble guanylate cyclase, beneficial for podocytes and anti-inflammatory [42][44] - **Patient Focus**: Targeting primary FSGS and genetic mutation-related FSGS while excluding secondary causes [56] Financial Position - **Cash Reserves**: $166 million as of Q3, with at least two years of cash runway [69] Upcoming Catalysts - **Key Events**: - Continued monitoring of VAFSEO's market performance - Progress on Praliciguat and AKB-097 studies - Results from VOCAL and VOICE trials expected within the next 12 months [70][72] Conclusion - **Outlook**: The next 12 months are anticipated to be critical for Akebia, with significant clinical readouts and potential market shifts for VAFSEO and pipeline products [72]

Core Insights - Akebia Therapeutics has initiated a Phase 2 clinical trial for praliciguat, targeting biopsy-confirmed FSGS, a rare kidney disease affecting approximately 40,000 patients in the U.S. [2] - The trial aims to evaluate the efficacy and safety of praliciguat, with a primary endpoint of change in urine protein-to-creatinine ratio (UPCR) at Week 24 [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]. - The company has a mid-stage pipeline for rare kidney diseases, with praliciguat being a key component [2]. Clinical Trial Details - The Phase 2 trial is randomized, double-blind, placebo-controlled, and multicenter, involving approximately 60 patients already on maximally tolerated doses of ACE inhibitors or ARBs [2]. - Participants will be randomized 1:1 to receive either praliciguat or placebo for an initial 24-week period, followed by an open-label phase for an additional 24 weeks [2]. Safety and Efficacy - No significant safety issues were reported for praliciguat in previous Phase 1 and Phase 2 studies, indicating a favorable safety profile [3]. - Adverse events were infrequent and consistent with the known blood pressure-lowering effects of praliciguat [3].

Summary of Akebia Therapeutics FY Conference Call Company Overview - **Company**: Akebia Therapeutics (NasdaqCM:AKBA) - **Focus**: Kidney disease treatment - **Products**: - **Auryxia**: A mature phosphate binder, past patent expiry - **Vafseo**: A hypoxia-inducible factor prolyl hydroxylase inhibitor approved for treating anemia in chronic kidney disease (CKD) patients on dialysis, launched at the beginning of the year [2][3] Financial Performance - **Recent Revenue**: Reported $14.3 million in revenue for the quarter, which was below expectations despite positive underlying metrics [5] - **Market Access**: Initially had access to about 40,000 patients, which has now grown to approximately 260,000 patients due to expanded access through major dialysis providers [6] Market Dynamics - **Dialysis Providers**: - **U.S. Renal Care**: Strong clinical advocacy, significant part of revenue base - **DaVita**: Expanded access from 100 to over 4,000 dialysis centers, indicating a successful pilot program [6][14] - **Fresenius**: More challenging engagement, but potential for significant patient access if they broaden availability [20] Clinical Insights - **Patient Management**: Emphasis on educating healthcare providers about the unique dosing and administration of Vafseo compared to traditional treatments [10][12] - **Clinical Data**: Recent data presented at ASN showed a 1% decrease in mortality and an 8% lower hospitalization rate for Vafseo compared to traditional treatments [19][23] Regulatory and Reimbursement Landscape - **TDAPA**: Transitional Drug Add-on Payment Adjustment allows billing for Vafseo outside the dialysis bundle for two years, providing a higher price point and facilitating access [25][26] - **Market Size**: The anemia treatment market in dialysis is estimated at $1 billion, with potential for Vafseo to capture a significant share if it becomes standard of care [26][29] Future Outlook - **Clinical Trials**: Ongoing studies (VOICE and VOCAL) aim to provide further evidence of Vafseo's efficacy and differentiate it from existing treatments [31][32] - **Pipeline Development**: Plans to explore additional indications for HIF-PHI, including acute kidney injury and retinopathy of prematurity [38][39] Financial Position - **Cash Reserves**: Ended the quarter with $166 million, indicating a solid balance sheet and guidance towards profitability [42]

Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $58.8 million, up from $37.4 million in Q3 2024, representing an increase of over $21 million [20] - Net product revenues increased to $56.8 million from $35.6 million year-over-year, driven by Vafseo sales of $14.3 million and Auryxia sales of $42.5 million, up from $35.6 million [20] - The company generated a net income of approximately $540,000 in Q3 2025, compared to a net loss of $20 million in Q3 2024 [24] Business Line Data and Key Metrics Changes - Vafseo generated $14.3 million in revenue this quarter, while Auryxia sales increased to $42.5 million [20] - The average dose of Vafseo refills increased by 5% compared to the previous quarter and 32% above the starting dose, indicating physicians are becoming more comfortable with optimal dosing [13] Market Data and Key Metrics Changes - The number of accessible patients for Vafseo increased from 40,000 at the beginning of the year to over 260,000 by the end of Q3 2025, with significant contributions from the DaVita pilot and other dialysis providers [17][19] - Over 85% of prescriptions written for Vafseo were refills, indicating a growing acceptance among prescribers [13] Company Strategy and Development Direction - The company aims to make Vafseo the standard of care for treating anemia in dialysis patients, supported by positive clinical data and strong prescriber interest [4][11] - The strategy includes addressing operational challenges and enhancing adherence through better education and support for prescribers [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in overcoming operational issues and increasing prescribing access, with expectations of significant growth in patient access by early 2026 [19][63] - The management highlighted the importance of ongoing data generation to support Vafseo's clinical differentiation and long-term growth [8][9] Other Important Information - The company is preparing for the results of the VOICE and VOCALE studies, which are expected to be important data catalysts for Vafseo [9] - Auryxia's future sales levels are uncertain due to potential generic competition, but the company continues to perform well in the market [21] Q&A Session Summary Question: What strategies could be used to overcome operational challenges for Vafseo? - The company is focusing on improving messaging around titration strategies and working closely with anemia managers to enhance adherence [26][27] Question: What has been the physician feedback regarding Vafseo's impact on hospitalization outcomes? - Physicians expressed excitement about the data presented, indicating that Vafseo could significantly change care for patients [34][35] Question: What are the greatest hurdles for expanding Vafseo in medium dialysis organizations? - The main hurdles include navigating operational processes at dialysis providers and ensuring adherence among patients [40][42]

Core Viewpoint - The drug and biotech sector is experiencing a recovery driven by a landmark drug-pricing deal between Pfizer and the Trump administration, which includes tariff exemptions and price cuts, alongside increased M&A activity in the sector [1]. Group 1: Industry Overview - The Zacks Medical-Drugs industry consists of small to medium-sized drug companies that primarily focus on developing medicines, often relying on collaborations with larger firms for revenue [3]. - The industry is currently ranked 68 in the Zacks Industry Rank, placing it in the top 28% of 243 Zacks industries, indicating strong prospects for growth [9]. Group 2: Market Performance - The Zacks Medical-Drugs industry has seen a year-to-date stock increase of 6.2%, outperforming the Zacks Medical sector, which decreased by 0.2%, but underperforming the S&P 500, which rose by 12.2% [11]. - The industry is trading at a trailing 12-month price-to-sales ratio of 2.37, lower than the S&P 500's 5.82 and the Zacks Medical sector's 2.45 [13]. Group 3: Key Trends and Innovations - There is a strong focus on innovation in areas such as rare diseases, next-generation oncology treatments, obesity, immunology, and neuroscience, which are attracting investor interest [5]. - Investment in technology and personalized medicine is crucial for smaller companies to adapt to the evolving healthcare landscape [7]. Group 4: Company Highlights - **Ironwood Pharmaceuticals**: The company is advancing its key pipeline candidate, apraglutide, for treating short bowel syndrome, with a stock increase of 115.5% in the past three months [16][17]. - **Pyxis Oncology**: The company is progressing with its lead candidate, micvotabart pelidotin, for treating head and neck cancer, with a stock increase of 197.4% in the past three months [20][21]. - **Plus Therapeutics**: Focused on targeted radiotherapeutics for CNS cancers, the company has seen its stock rise by 113.6% in the past three months [24][26]. - **Cardiol Therapeutics**: Developing CardiolRx for heart disease, the company has experienced an 18.6% decline in stock over the past three months [29][31]. - **Akebia Therapeutics**: The company launched Vafseo for anemia due to chronic kidney disease, but its stock has declined by 28.3% in the past three months [33][34].

Company Overview - Akebia Therapeutics, Inc. is a biopharmaceutical company focused on developing therapies for kidney disease, with its primary product being Auryxia, which treats iron deficiency anemia in chronic kidney disease patients [1] Financial Performance - Akebia's Return on Invested Capital (ROIC) is 0.34%, while its Weighted Average Cost of Capital (WACC) is 10.46%, resulting in a ROIC to WACC ratio of 0.033, indicating inefficiencies in capital utilization [2][6] - Ardelyx, Inc. has a ROIC of -10.63% and a WACC of 7.62%, leading to a ROIC to WACC ratio of -1.395, which, despite being negative, is the highest among its peers [3] - FibroGen, Inc. and Karyopharm Therapeutics Inc. show concerning figures with ROICs of -122.23% and -1634.05%, respectively, and WACCs of 7.40% and 16.28%, resulting in ROIC to WACC ratios of -16.521 and -100.389 [4] - Calithera Biosciences, Inc. and Aldeyra Therapeutics, Inc. also face difficulties, with ROICs of -184.41% and -75.25%, and WACCs of 3.17% and 7.59%, leading to ROIC to WACC ratios of -58.23 and -9.913 [5][6]