Summary of Syndax Pharmaceuticals Conference Call on Revuforge Approval Company Overview - **Company**: Syndax Pharmaceuticals (NasdaqGS: SNDX) - **Product**: Revuforge, a first-in-class menin inhibitor - **Date of Call**: October 24, 2025 Key Industry and Company Insights FDA Approval and Indications - Revuforge received FDA approval for its second indication, targeting adults and pediatric patients with relapsed or refractory NPM1 mutated acute myeloid leukemia (AML) [6][10] - This approval follows the initial FDA approval in November 2024 for relapsed or refractory acute leukemia patients with KMT2A translocation [6][10] - Revuforge is now the first and only menin inhibitor approved for multiple acute leukemia subtypes in both adults and pediatrics [6][10] Clinical Efficacy and Data - The approval is based on pivotal data showing a 47% overall response rate (ORR) and an 11% transplant rate in patients treated with Revuforge [8][19] - The median overall survival for responders was nearly two years, with a median duration of response of 4.5 months [19][20] - Revuforge demonstrated compelling efficacy across multiple genetic subtypes of acute leukemia, establishing a strong clinical profile [8][20] Market Opportunity - The total addressable population for Revuforge has expanded from approximately 2,000 KMT2A incident patients to 6,500 patients, representing a market opportunity exceeding $2 billion in the relapsed refractory setting [25][26] - Approximately 40-45% of AML patients have either an NPM1 mutation or a KMT2A translocation, indicating a significant market potential [26] Commercial Strategy and Execution - Syndax has established a strong commercial foundation with a trusted presence in leading cancer centers across the U.S. [10][28] - The company has achieved nearly $50 million in net revenue in the first two full quarters post-launch, despite some KMT2A patients pausing treatment for transplants [27][28] - Revuforge is on formulary for 97% of covered lives, facilitating rapid access for patients [29][30] Safety Profile and Monitoring - The updated label includes a boxed warning for QTc prolongation and torsades de pointes, based on a non-fatal case identified during clinical trials [16][51] - Management of QTc prolongation remains straightforward, with physicians already familiar with monitoring protocols [41][51] Future Outlook - Syndax plans to expand Revuforge into additional indications and frontline settings, with ongoing clinical trials [11][35] - The company is positioned for continued growth, with a strong pipeline and a focus on innovative therapies for difficult-to-treat diseases [24][35] Additional Important Points - The NCCN Guideline Committee has added Revuforge as a recommended treatment option for relapsed refractory NPM1 mutated AML, validating the strength of the clinical data [10][11] - The company has a comprehensive clinical development plan aimed at unlocking a market opportunity exceeding $5 billion in the U.S. [11] - Syndax's commercial team has extensive experience in hematology and oncology, enhancing their ability to engage with healthcare providers effectively [32][34] This summary encapsulates the critical insights from the conference call regarding Syndax Pharmaceuticals, its product Revuforge, and the broader implications for the acute leukemia treatment landscape.

Financial Data and Key Metrics Changes - In Q2 2025, Syndax reported net revenue of $28.6 million for Revuforge, a 43% increase quarter over quarter, with combined net product sales for Revuforge and Nyktymbo reaching nearly $100 million in the first half of the year [6][7][19] - Nyktymbo generated $36.2 million in net revenue in its first full quarter, significantly up from $13.6 million in the first two months of launch [15][40] - The company maintains a strong financial position with $518 million in cash and equivalents as of June 30, 2025, and expects to reach profitability with current resources [43][44] Business Line Data and Key Metrics Changes - Revuforge has treated over 500 patients, with approximately 90% of usage in KMT2A patients, and is increasingly being used in earlier lines of therapy [10][20] - Nyktymbo is already profitable for Syndax, contributing $9.4 million in collaboration revenue in Q2 2025 [16][41] - The average treatment duration for Revuforge is expected to build to 4-6 months in the first year and potentially 6-12 months in the second year as treatment patterns mature [23][68] Market Data and Key Metrics Changes - Revuforge has penetrated 25% of the annual 2,000 patient incidents of relapsed-refractory KMT2A acute leukemia, with expectations to reach 50% by year-end [20][83] - Nyktymbo is positioned in a $2 billion market for chronic GVHD treatments, with a total addressable market of $2 billion based on current indications [28][49] - The company anticipates expanding Revuforge's market opportunity to over $2 billion with the approval of its supplemental new drug application for relapsed-refractory mutant NPM1 AML [13][14] Company Strategy and Development Direction - Syndax aims to extend its leadership in the menin inhibition space, with ongoing trials for Revuforge in both frontline and relapsed settings [35][37] - The company is focused on developing Nyktymbo for additional patient populations, including idiopathic pulmonary fibrosis (IPF) [38] - Syndax's strategy includes maintaining stable operating expenses while investing in the successful launches of Revuforge and Nyktymbo [41][54] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's trajectory towards profitability, driven by strong physician enthusiasm and robust adoption of both products [45][46] - The anticipated approval of the supplemental new drug application for Revuforge is expected to significantly enhance its market position [13][96] - Management highlighted the importance of treatment duration and patient outcomes as key drivers for future growth [90] Other Important Information - The company has strengthened its leadership team with the addition of Nick Botwood as Head of R&D and Chief Medical Officer [17] - Syndax has retained worldwide rights to Revuforge, with patent protection extending through at least the late 2030s [48] Q&A Session Summary Question: Path to profitability and assumptions for top line growth - Management indicated that profitability is expected with existing resources, and both product launches are outperforming expectations [52][53] Question: Patients proceeding to transplant and maintenance therapy - Management noted that patients typically proceed to transplant after 2-3 cycles of treatment, with expectations for a high percentage of patients to return to maintenance therapy [57][62] 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 extending to 6-12 months as more patients are treated earlier [64][68] Question: KMT-2A patient penetration and revenue growth - Management clarified that there is still significant growth potential in the KMT-2A population, with expectations to reach 50% penetration by year-end [78][88] Question: FDA approval confidence and remaining open items - Management expressed confidence in the FDA approval process, indicating that discussions with the agency have been positive [95][99]