Core Insights - The management team of Biomea has a proven track record in value creation through the development of novel agents for treating conditions like Chronic Lymphocytic Leukemia (CLL) [2] - Biomea's lead program, icovamenib, is the first menin inhibitor aimed at treating both type 1 and type 2 diabetes, with the potential to preserve and enhance pancreatic beta cell function, indicating a significant market opportunity despite the company's small market capitalization [3] Company Overview - Biomea is focused on innovative treatments for diabetes, particularly through its lead program icovamenib, which could redefine diabetes treatment [3] - The company operates in a market that has seen limited innovation prior to the introduction of its novel agents, such as ibrutinib for CLL [2]

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]

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 of historical facts contained in this presentation, including stateme ...

Core Insights - Syndax Pharmaceuticals presented new data on Revuforj (revumenib) at the 67th ASH Annual Meeting, highlighting its efficacy and safety in treating acute leukemia, particularly in patients with NPM1m, KMT2Ar, and NUP98r mutations [1][2][3] Efficacy and Safety Data - The overall response rate (ORR) for revumenib was reported at 77% (10/13) in a retrospective review, with a complete remission (CR) rate of 31% (4/13) and 75% (9/12) achieving measurable residual disease (MRD) negativity [1][4][5] - In a Phase 2 SAVE trial, the ORR was 86% (18/21) and the CR rate was 76% (16/21), with 100% MRD negativity among responders [6][11] - Revumenib demonstrated a favorable safety profile, with only 24% (4/17) experiencing Grade 3 or 4 non-hematological adverse events, and low rates of dose reductions and discontinuations at 6% each [5][13] Clinical Trials and Future Directions - Syndax has initiated pivotal frontline trials for revumenib in patients unfit for intensive chemotherapy and in fit patients, aiming to expand clinical data supporting its use [2][23] - Multiple ongoing and planned trials are evaluating revumenib in combination with standard therapies for newly diagnosed patients with specific genetic mutations [22][23] Pediatric Applications - A retrospective review of revumenib as post-HSCT maintenance in children showed that all ten patients were alive at a median follow-up of 19 months, with a 90% relapse-free survival rate [7][8] Company Overview - Syndax Pharmaceuticals is focused on advancing innovative cancer therapies, with Revuforj being a first-in-class menin inhibitor approved for treating relapsed or refractory acute leukemia [22][40]

Financial Data and Key Metrics Changes - In Q2 2025, Syndax Pharmaceuticals reported net product sales of nearly $100 million for Revuforge and Nyktymbo, significantly exceeding expectations [5] - Revuforge net revenue increased by 43% quarter over quarter to $28.6 million, despite one-third of patients pausing treatment for stem cell transplants [5][39] - Nyktymbo generated $36.2 million in net revenue in its first full quarter, up from $13.6 million in the first two months of launch [13][39] Business Line Data and Key Metrics Changes - Revuforge has treated over 500 patients, with approximately 90% of usage in KMT2A patients, and has penetrated 25% of the annual 2,000 patient incidents [8][18] - Nyktymbo is already profitable for Syndax, contributing $9.4 million in collaboration revenue in Q2 2025 [14][40] - The average treatment duration for Revuforge is expected to increase to 6-12 months as treatment patterns mature [21][66] Market Data and Key Metrics Changes - The market opportunity for Revuforge is projected to exceed $5 billion across relapsed refractory and frontline settings [24] - The addressable market for Revuforge in the U.S. is expected to increase to $2 billion with the anticipated approval of the supplemental new drug application (sNDA) for relapsed-refractory mutant NPM1 AML [12] - Nyktymbo targets a $2 billion total addressable market in chronic GVHD, with a current indication for 6,500 patients requiring three or more lines of therapy [26] Company Strategy and Development Direction - Syndax aims to expand Revuforge into earlier lines of therapy and additional patient populations, enhancing its market position [6][12] - The company is focused on maintaining a stable operating expense base while fully funding strategic priorities [39][42] - Syndax is advancing its leadership in menin inhibition and exploring additional indications for Nyktymbo, including idiopathic pulmonary fibrosis [37][48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in reaching profitability with current resources, citing strong performance from both product launches [43][45] - The company anticipates continued growth driven by strong physician enthusiasm and robust adoption of Revuforge and Nyktymbo [45][48] - Management highlighted the importance of clinical data and physician feedback in driving future growth and market penetration [29][31] Other Important Information - Syndax has a strong financial position with $518 million in cash and equivalents as of June 30, 2025 [43] - The company has strengthened its leadership team with the addition of Dr. Nick Botwood as Head of R&D and Chief Medical Officer [15] Q&A Session Summary Question: Path to profitability and assumptions for REVIVORGE and Nyktymbo - Management indicated that profitability is expected with existing resources, and both product launches are outperforming expectations [51][52] Question: Patients proceeding to transplant and maintenance therapy - Management noted that patients typically proceed to transplant after 2-3 cycles of treatment, and they expect a high percentage of patients to return for maintenance therapy [55][60] Question: Treatment duration observations in real-world practice - Management confirmed that the average treatment duration is expected to be 4-6 months in the first year, potentially increasing to 6-12 months as treatment patterns mature [62][66] Question: KMT-2A patient penetration and revenue growth - Management stated that they expect to reach 50% penetration of the identified KMT-2A population by the end of the year, with significant growth potential remaining [76][80] Question: FDA approval confidence and remaining open items - Management expressed confidence in the FDA approval process, indicating good progress and consistent dialogue with the agency [92][96]

Financial Performance - Revuforj generated $20.0 million in net revenue in 1Q25[3] - Niktimvo generated $13.6 million in net revenue in 1Q25 (reported by Incyte)[3] - The company held $602.1 million in cash and cash equivalents as of March 31, 2025[3] - Total revenue for 1Q25 was $20.0 million[24] - Net loss for 1Q25 was $84.8 million[24] Product Development and Milestones - Syndax initiated a pivotal frontline trial of revumenib with ven/aza in mNPM1 and KMT2Ar patients unfit for intensive chemo in 1Q25[3] - The company completed the submission of Revuforj sNDA for R/R mNPM1 AML in April 2025[3] - Syndax anticipates reporting Phase 1 data from a frontline trial evaluating revumenib with I C (7+3) in 4Q25[27] - Over 1,250 Niktimvo infusions have been ordered YTD[18] Market Access and Adoption - Two-thirds of accounts ordering Revuforj have ordered multiple times[10] - Approximately 95% of all Bone Marrow Transplants (BMTs) ordered are from top accounts[18]