Financial Data and Key Metrics Changes - Total revenue for 2025 was $172.4 million, consisting of $124.8 million in Revuforj net revenue, $42.4 million of Niktimvo collaboration revenue, and $5.1 million in milestones and royalties [35] - Revuforj net revenue increased by 38% in the fourth quarter compared to the third quarter, driven by strong demand [35] - Niktimvo net revenue for 2025 reached $151.6 million, surpassing first-year launch benchmarks set by Rezurock [19][35] Business Line Data and Key Metrics Changes - Revuforj generated $125 million in net revenue in 2025, with a 38% growth in net revenue and a 35% growth in prescriptions quarter-over-quarter in Q4 [6][7] - Niktimvo saw a 22% increase in net revenue quarter-over-quarter in Q4, contributing $42 million in collaboration revenue to Syndax in 2025 [8][19] Market Data and Key Metrics Changes - The KMT2A patient population is approaching 50% penetration within the first year of Revuforj's launch, with approximately 1,050 patients treated commercially [12] - NPM1 patients represented about 30% of new patient starts, with expectations for this number to grow significantly [46][47] Company Strategy and Development Direction - Syndax aims to expand its market share in menin inhibition, targeting a total addressable market opportunity exceeding $10 billion [9] - The company is focused on advancing its pipeline, including pivotal trials for revumenib and axatilimab, to unlock new market opportunities [10][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued success of Revuforj and Niktimvo, supported by strong customer relationships and market access [7][9] - The company is well-funded with $394 million in cash and equivalents, expecting to reach profitability without additional capital [37] Other Important Information - Syndax completed enrollment in the MAXPIRe trial for axatilimab in idiopathic pulmonary fibrosis, with top-line data expected in Q4 2026 [30] - The company is positioned to deliver impactful real-world evidence supporting revumenib's clinical use [29] Q&A Session Summary Question: What factors are driving the increase in KMT2A maintenance therapy post-transplant? - Management noted that the growth in KMT2A maintenance therapy is driven by a significant number of patients returning for maintenance after transplant, with expectations for continued growth [43] Question: What is the expected steady-state percentage of NPM1 patient starts? - Management anticipates that the percentage of NPM1 patient starts will grow meaningfully throughout the year, with current estimates around 30% [46][47] Question: What are the benchmarks for the IPF trial to determine the go-forward decision? - The primary endpoint is the annualized rate of decline in forced vital capacity, with expectations for a statistically significant and clinically meaningful difference compared to historical controls [49][50] Question: What is the fastest path to a pivotal trial for IPF if the data reads out positively? - Management indicated that they would be as expeditious as possible in designing the next trial, with plans for a phase 3 trial following a positive phase 2 outcome [54][55] Question: Are physicians comfortable with the dose of Revuforj in maintenance therapy? - Management confirmed that physicians are becoming increasingly comfortable with the dosing for maintenance therapy, supported by ongoing studies [64] Question: What is the strategy for moving Revuforj into newly diagnosed fit KMT2A patients? - The strategy includes a randomized study planned in collaboration with the NCI, focusing on innovative approaches to reduce morbidity while maintaining efficacy [68][69]

Financial Data and Key Metrics Changes - Total revenue for 2025 was $172.4 million, consisting of $124.8 million in Revuforj net revenue, $42.4 million of Niktimvo collaboration revenue, and $5.1 million in milestones and royalties [34] - Revuforj net revenue increased by 38% in the fourth quarter compared to the third quarter, driven by strong demand [34] - Niktimvo net revenue for 2025 reached $151.6 million, surpassing first-year launch benchmarks set by Rezurock [18] Business Line Data and Key Metrics Changes - Revuforj generated $125 million in net revenue in 2025, with a 38% growth in net revenue and a 35% growth in prescriptions quarter-over-quarter in Q4 [4][5] - Niktimvo saw a 22% increase in net revenue quarter-over-quarter in Q4, contributing $42 million in collaboration revenue to Syndax in 2025 [6][34] Market Data and Key Metrics Changes - The KMT2A business is experiencing growth as the number of patients on therapy post-transplant increases, with approximately one-third of KMT2A patients treated with Revuforj proceeding to stem cell transplant [15] - The NPM1 market opportunity is expanding, with the addressable patient population tripling to 6,500 patients following the label expansion for Revuforj [12] Company Strategy and Development Direction - The company aims to continue investing in strategic priorities to fuel growth, including advancing life cycle and frontline programs to unlock over $10 billion in total addressable market opportunity [8] - Syndax is focused on establishing Revuforj as the menin inhibitor of choice in the relapsed refractory setting and the first menin inhibitor approved in the frontline, targeting a $5 billion-plus market opportunity [38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued success of both Revuforj and Niktimvo, supported by multiple growth drivers and a well-funded position to invest in commercial and development priorities [8][34] - The company anticipates that the average treatment duration for Revuforj will extend from 4-6 months in the first year to 6-12 months in the second year as treatment patterns mature [17] Other Important Information - The company completed enrollment in the MAXPIRe trial for axatilimab in idiopathic pulmonary fibrosis, with top-line data expected in Q4 2026 [29] - Syndax is well-funded with $394 million in cash, equivalents, and marketable securities at the end of 2025, expecting to reach profitability without the need for additional capital [37] Q&A Session Summary Question: What factors are driving the increase in KMT2A maintenance therapy post-transplant? - Management noted that the growth in KMT2A has been significant, with a rising number of patients returning for maintenance therapy post-transplant, increasing from 35%-40% to 40%-45% [42][43] Question: What is the expected steady-state percentage for NPM1 patient starts? - Management expects the percentage of NPM1 patient starts to grow significantly, potentially reaching a 50/50 split with KMT2A patients as awareness and access improve [45][48] Question: What are the criteria for determining the go-forward decision on the IPF indication? - The primary endpoint for the IPF study is the annualized rate of decline in forced vital capacity, with management looking for statistically significant and clinically meaningful results compared to historical controls [50][51] Question: What is the strategy for moving Revuforj into newly diagnosed fit KMT2A patients? - Management is pursuing an innovative approach with the RAVEN trial, aiming to combine Revuforj with venetoclax to potentially reduce morbidity while achieving similar outcomes to intensive chemotherapy [64][70]

Fourth Quarter and Full Year 2025 Financial Results February 26, 2026 Forward-looking statements disclosure This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate" and similar expressions (as well as other words or expressions referencing future events, progress, timing or circumstances) are intended to identify forward-looking statements. All statements other than statements ...

Summary of Syndax Pharmaceuticals Conference Call Company Overview - **Company**: Syndax Pharmaceuticals (NasdaqGS:SNDX) - **Date**: February 12, 2026 - **Key Speakers**: Michael Metzger (CEO), Nick Botwood (CMO, Head of R&D), Keith Goldan (CFO) Core Points and Arguments Financial Performance - **Revuforj**: Achieved $125 million in annual net sales, with a quarter-over-quarter growth of 38% in Q4 2025, indicating strong market performance and expansion in its labeled indications [2][4] - **Niktimvo**: Generated $152 million in sales within the first 11 months of launch for chronic GVHD, also showing significant quarter-over-quarter growth [2] Product Development and Market Strategy - **Revuforj**: - Focused on KMT2A and NPM1 indications, with new NCCN guidelines and approvals driving growth [4][5] - Anticipated to capture a dominant market share in NPM1, similar to its established position in KMT2A [9] - Current maintenance therapy uptake for KMT2A is 35%-40%, with expectations to grow to 70%-80% over time [10][13] - **Niktimvo**: - Currently approved for later-line chronic GVHD, with a potential market of approximately 6,500 patients in the U.S. for third-line treatment, and an estimated peak opportunity approaching $1 billion [23][24] - Collaboration with Incyte involves a 50/50 profit split, with expectations for long-term growth in revenue share [24][26] Research and Development - **IPF Phase II Trial**: - Expected to read out in the second half of 2026, with a well-designed study focusing on forced vital capacity as the primary endpoint [30][34] - Strong preclinical data supports the role of CSF1R inhibition in IPF, with significant improvements noted in related conditions [32][40] - **Future Opportunities**: - Plans to explore the role of revumenib in myeloproliferative neoplasms (MPN), with promising preclinical results [20][21][22] Path to Profitability - Syndax aims for profitability potentially by 2027, supported by strong revenue from both Revuforj and Niktimvo, while maintaining flat operating expenses at $400 million [46] Additional Important Insights - The company is fully funded and does not require additional cash, positioning it well for future growth [46] - The collaboration with Incyte extends to future trials, including potential IPF studies, with shared costs and responsibilities [45] This summary encapsulates the key points discussed during the conference call, highlighting the financial performance, product development strategies, research initiatives, and the company's path to profitability.

Financial Data and Key Metrics Changes - For Q4 2024, Syndax Pharmaceuticals reported $7.7 million in net revenue from Revuforj, marking the company's first quarter with product revenue [10][46] - Operating expenses for Q4 were $104 million, which included $65.5 million in research and development expenses and $37.7 million in selling, general, and administrative expenses [46] - The company maintained a strong financial position with $692.4 million in cash, equivalents, and investments at year-end [47] Business Line Data and Key Metrics Changes - Revuforj, the first-in-class menin inhibitor, launched in November 2024, has shown strong early demand with $7.7 million in net revenue from the first five weeks of sales [10][11] - Niktimvo, launched in January 2025 for chronic graft versus host disease, is expected to capture a significant portion of the $1.5 billion to $2 billion market for third-line or later chronic GVHD treatment in the US [32] Market Data and Key Metrics Changes - Approximately 33% of Tier 1 and Tier 2 accounts have ordered Revuforj, indicating strong early penetration in key treatment centers [21][22] - The company is seeing rapid inclusion of Revuforj in payer coverage, with 56% of commercially covered lives and 53% of all managed care lives having formal coverage policies in place [25] Company Strategy and Development Direction - The company aims to expand Revuforj's indication to include mutant NPM1 AML by filing a supplemental new drug application in the upcoming quarter [12] - Syndax is focused on building a robust pipeline in oncology, with plans to extend its products into earlier line indications and develop additional targeted therapies [99][100] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position for long-term growth, highlighting the strong market opportunities with Revuforj and Niktimvo [54] - The management team emphasized the importance of building a habit among physicians for prescribing Revuforj, which is expected to become the standard of care for KMT2A translocations [24][60] Other Important Information - The company received $350 million upfront from Royalty Pharma in exchange for a capped royalty on US net sales of Niktimvo, indicating the product's multi-billion-dollar potential [15] - The company expects R&D expenses for Q1 2025 to be between $65 million and $70 million, with total R&D plus SG&A expenses projected to be between $105 million and $110 million [48] Q&A Session Summary Question: Inventory levels and repeat prescribers for Revuforj - Management indicated that inventory levels are expected to remain stable at about two to three weeks, with revenue growth potentially increasing absolute inventory levels [62] - Approximately one-third of Tier 1 and Tier 2 accounts have ordered Revuforj, with most accounts having prescribed it more than once, indicating positive early adoption [65] Question: Activation of Tier 1/2 accounts and comparison to historic AML launches - Management expects continued activation of Tier 1 and Tier 2 accounts over the next 10 months, with a focus on the top 200 treatment centers [73] - The unique setup of Revuforj, including diagnostic testing for KMT2A patients, is expected to drive its adoption compared to other targeted therapies [78] Question: Off-label use and Expanded Access Program (EAP) - Anecdotal evidence suggests some off-label use of Revuforj, particularly in combination treatments, but specific numbers are not yet available [86] - The EAP remains lightly used, with no additional patients expected to transition from it at this time [87] Question: Future company vision and pipeline expansion - The company aims to build a larger pipeline in oncology and expects to have multiple commercial products by 2030, focusing on targeted therapies [100][101] Question: Safety feedback and physician experience with Revuforj - Feedback on Revuforj's safety profile has been positive, with comprehensive support provided to physicians through clinical nurse educators [110] Question: Gross to net adjustments and patient assistance programs - The gross to net adjustments for Revuforj are expected to remain tight due to the limited distribution channel, with a patient assistance program available for uninsured or underinsured patients [118][120] Question: Transplant dynamics and patient treatment pathways - Physicians are interested in getting patients to transplant, particularly for KMT2A, with expectations that many patients will return to maintenance therapy post-transplant [122]