Summary of Syndax Pharmaceuticals FY Conference Call Company Overview - **Company**: Syndax Pharmaceuticals (NasdaqGS:SNDX) - **Event**: 44th Annual JPMorgan Healthcare Conference - **Date**: January 12, 2026 - **Presenting**: CEO Michael Metzger Key Points Company Transformation and Product Launches - 2025 marked a transformational year for Syndax, transitioning to a commercial-stage oncology company with the launch of two first-in-class medicines: Revuforge and Niktimvo [2][3] - Achieved three FDA approvals within 14 months, demonstrating rapid product development and commitment to patient care [2] Financial Performance - The company reported approximately $400 million on the balance sheet, indicating strong financial health to support growth initiatives [3] - Revuforge generated $44 million in preliminary net revenue for Q4 2025, a 38% increase from the previous quarter, and $125 million for the full year [9][11] - Niktimvo generated $56 million in preliminary Q4 2025 net revenue, a 22% increase from the last quarter, and $152 million for the full year [22][23] Revuforge Insights - Revuforge is the first menin inhibitor approved for multiple acute leukemia subtypes, with a total addressable market of approximately $2 billion for relapsed refractory acute leukemia and $5 billion when considering front-line treatment [4][5] - The drug has shown unmatched efficacy and is well-tolerated, allowing for individual dosing and combination therapy with other medications [6][7] - The company has established a strong prescriber base and high reimbursement rates, with 97%-98% coverage from payers for KMT2A indications [8] Market Dynamics and Growth Strategy - The company is witnessing a shift towards earlier lines of therapy, with a growing number of patients being treated in second and third lines, which enhances response rates and treatment duration [12][13] - The approval of NPM1 has tripled the patient population eligible for treatment, further driving brand growth [14][15] - The company plans to advance clinical trials to support front-line use and explore additional indications, including myelofibrosis [18][27] Niktimvo Insights - Niktimvo is the first CSF1R blocking antibody approved for chronic GVHD, with a current market of 6,500 patients and a potential total addressable market of $2 billion [20][21] - The drug is expected to expand into front-line therapy, potentially reaching 15,000-17,000 patients [21] - The company has a robust clinical development plan for Niktimvo, including trials for chronic GVHD and idiopathic pulmonary fibrosis (IPF) [25][26] Future Outlook - Syndax is focused on generating value through data generation and clinical development in 2026, with several anticipated milestones, including top-line data for Axatilimab and pivotal trials for Revuforge [26][27] - The company aims to penetrate the KMT2A market further, targeting a 90% penetration rate over time [35] - The competitive landscape is acknowledged, but Syndax believes its best-in-class profiles and first-mover advantages will allow it to dominate the market [40] Additional Insights - Physicians have expressed strong interest in the combination of Niktimvo with existing therapies, which may drive earlier line utilization [53] - The company is confident in the efficacy of its drugs based on clinical data and physician feedback, with a significant percentage of patients remaining on therapy long-term [48] This summary encapsulates the key insights and strategic direction of Syndax Pharmaceuticals as discussed during the conference call.

Financial Data and Key Metrics Changes - Syndax reported total revenue of $45.9 million for Q3 2025, reflecting a strong 21% growth over the prior quarter [4] - Revyforge net revenue was $32 million in Q3, up 12% from the prior quarter, with a 25% increase in total prescriptions and new patient starts [5][11] - Nictimvo net revenue reached $45.8 million in Q3, marking a 27% increase over the prior quarter [9][19] Business Line Data and Key Metrics Changes - Revyforge has become the standard of care for relapsed refractory KMT2A, with approximately 50% of usage in the second line [5] - Nictimvo is annualizing at nearly $200 million within the first eight months of launch, tracking in line with first-year sales of similar therapies [9] - The approval of Revyforge for relapsed refractory NPM1 mutated AML has expanded the addressable patient population significantly [6][15] Market Data and Key Metrics Changes - The total addressable U.S. population for Revyforge has increased from approximately 2,000 to 6,500 patients with the new indication [15] - Nictimvo is targeting a market opportunity of approximately $2 billion, with 6,500 patients in the U.S. requiring three or more lines of therapy [21] Company Strategy and Development Direction - The company aims to expand Revyforge and Nictimvo into the frontline setting, unlocking a combined market opportunity exceeding $10 billion [10] - Syndax is focused on executing pivotal trials for menin inhibitors, with Evolve 2 being the first frontline trial to start enrolling patients [10][28] - The company is leveraging its strong commercial foundation and prescriber relationships to drive growth in both product lines [16][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in reaching profitability, citing strong performance from both Revyforge and Nictimvo [38] - The company anticipates that the average duration of therapy for KMT2A patients will increase to six to twelve months in 2026 as treatment patterns mature [14][44] - Management highlighted the positive feedback from healthcare providers regarding the efficacy and tolerability of both Revyforge and Nictimvo [20][22] Other Important Information - Syndax has a strong financial position with $456 million in cash equivalents and investments as of September 30, 2025 [38] - The company has received three FDA approvals and is positioned for sustained growth with its current product offerings [39] Q&A Session Summary Question: How has Revyforge's place in lines of therapy evolved? - Management noted that about 70% of Revyforge's business is in the second or third line, a shift from clinical trials where patients were typically in third or fourth line [41][42] Question: What factors will help narrow the duration of therapy range for 2026? - Management indicated that the mix of KMT2A and NPM1 patients will influence the duration of therapy, with expectations for longer durations as more patients return from transplant [44][45] Question: What is the maintenance restart rate for patients who started Revyforge during the launch? - The restart rate has increased to about 35%-40%, with expectations for further growth as more patients return from transplant [46][47] Question: How will revenue per prescription evolve with the inclusion of more NPM1 patients? - Management does not expect significant changes in average revenue per prescription as NPM1 patients are integrated into the prescribing base [49] Question: What has been the impact of the NCCN guidelines on the NPM1 launch? - Management reported strong awareness and excitement among healthcare providers, setting a positive trajectory for the NPM1 launch [54][56]