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.

Summary of Syndax Pharmaceuticals Conference Call Company Overview - **Company**: Syndax Pharmaceuticals - **Key Products**: RevuForge (Menin inhibitor for AML) and Nictimvo (for chronic GVHD) - **Market Potential**: - RevuForge: Over $5 billion market opportunity - Nictimvo: Over $3 billion market opportunity - **Current Status**: Both products are generating revenue and have FDA approval, with early launches showing positive results [3][4][7]. Core Points and Arguments RevuForge - **Product Profile**: First-in-class Menin inhibitor with a significant first-mover advantage in the market [3]. - **Clinical Data**: - Phase 1b study confirmed a dose of 160 mg in combination with Venaza, showing good tolerability and a 67% complete response (CR) rate compared to 37% historical control [11][13]. - 100% of evaluable patients achieved minimal residual disease (MRD) negativity, indicating strong efficacy [14]. - **Market Position**: Anticipated inclusion in NCCN guidelines ahead of approval, which could enhance market penetration [24][44]. Nictimvo - **Clinical Data**: - Updated data to be presented at EHA, showing a 26% CR rate and a 48% overall response rate in a larger patient population [21][22]. - Rapid symptom improvement across organ systems in chronic GVHD patients [47][48]. - **Sales Performance**: Strong initial sales with $13.6 million in net revenue for the first two months post-launch [49]. Additional Important Insights - **Regulatory Strategy**: Plans to use CR and MRD negativity as potential endpoints for accelerated approval in clinical trials [35][36]. - **Launch Metrics**: - RevuForge generated $20 million in its first full quarter, with 90% of sales from KMT2A patients and expectations for growth as NPM1 reimbursement is secured [37][42]. - High coverage rates with 72% of managed care lives covered, indicating strong market access [39]. - **Competitive Landscape**: Syndax is currently the only approved Menin inhibitor, with a best-in-class profile that physicians favor [29][30][31]. Conclusion Syndax Pharmaceuticals is well-positioned in the oncology market with its innovative products, strong clinical data, and strategic regulatory plans. The company is focused on expanding its market presence and enhancing its product offerings, which could lead to significant growth in the coming years [5][6][34].

Financial Data and Key Metrics Changes - Syndax reported net revenue of $20 million for RevuForge in Q1 2025, marking the first full quarter of its launch [8][35] - Nictimvo generated $13.6 million in net revenue for the first two months of its launch, with Syndax reporting a collaboration loss of only $200,000 for this period [8][36] - The company maintained a strong financial position with $602.1 million in cash and equivalents as of March 31, 2025 [9][38] Business Line Data and Key Metrics Changes - RevuForge's launch has seen strong adoption, with 44% of tier one and tier two accounts ordering the product as of March, up from one-third in February [16][17] - Nictimvo has been administered in over 1,250 infusions year-to-date, with approximately 95% of top accounts ordering the product [25][26] Market Data and Key Metrics Changes - The current indication for RevuForge targets an estimated 2,000 patients in the U.S. with relapsed or refractory acute leukemia, representing a market opportunity of $750 million [22] - The total addressable market for Nictimvo is estimated to be between $1.5 billion to $2 billion, targeting 6,500 chronic GVHD patients in the U.S. [27][10] Company Strategy and Development Direction - Syndax aims to position RevuForge as the first menin inhibitor included in clinical guidelines for treating relapsed or refractory mutant NPM1 AML [13] - The company is focused on executing strategic launch imperatives to ensure long-term competitive immunity ahead of potential market entrants [23] - The EVOLVE-two trial is a pivotal frontline study for Rebuminav, aiming for accelerated approval and full approval based on dual primary endpoints [29][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong commercial opportunities for both RevuForge and Nictimvo, highlighting the unmet medical needs and the compelling profiles of their medicines [8][10] - The company anticipates that RevuForge will be the first menin inhibitor approved in the frontline setting, with ongoing engagement with the FDA on their submissions [11][14] Other Important Information - The company has submitted a supplemental new drug application (sNDA) for RevuForge, seeking priority review for treating relapsed or refractory mutant NPM1 AML [10] - The FDA's real-time oncology review program is expected to facilitate quicker approvals for their submissions [11] Q&A Session Summary Question: What are you seeing regarding repeat prescribers for RevuForge? - Management noted that 44% of tier one and tier two accounts have ordered, with about 80% of those ordering more than once, indicating a growing user base [43][46] Question: Are you seeing a similar pace of patients receiving transplants with RevuForge as in clinical trials? - Management stated that anecdotal information suggests patients are being taken to transplant, but it is too early to provide definitive data [52][53] Question: Can you share any color on month-over-month trends for new patient adds for RevuForge in Q1? - Management indicated that while they are not providing specific numbers yet, they are happy with the steady stream of new patients and refill rates [58][60] Question: What portion of the $20 million revenue stems from refill dynamics versus new patient starts? - Management mentioned that both refill and new patient dynamics are building, but specific data is still maturing [101][102]

Financial Data and Key Metrics Changes - Syndax Pharmaceuticals reported net revenue of $20 million for RevuForge in Q1 2025, marking the first full quarter of its launch [6][35] - Nictimvo achieved net revenue of $13.6 million in the first two months of its launch, with Syndax reporting a collaboration loss of only $200,000 for this period [6][36] - The company maintained a strong financial position with $602.1 million in cash and equivalents as of March 31, 2025 [7][39] Business Line Data and Key Metrics Changes - RevuForge's launch has seen strong adoption, with 44% of tier one and tier two accounts ordering the product as of March, up from one-third in February [16] - Nictimvo has been administered in over 1,250 infusions year-to-date, with approximately 95% of top accounts ordering the product [24] Market Data and Key Metrics Changes - The current indication for RevuForge targets an estimated 2,000 patients in the U.S. with relapsed or refractory acute leukemia with a KMT2A translocation, representing a $750 million market opportunity [20] - The total addressable market for Nictimvo is estimated to be between $1.5 billion to $2 billion, targeting 6,500 chronic GVHD patients in the U.S. [10][26] Company Strategy and Development Direction - Syndax aims to position RevuForge as the first menin inhibitor included in clinical guidelines for treating relapsed or refractory mutant NPM1 AML, with plans for a supplemental new drug application (sNDA) to the FDA [8][11] - The company is focused on executing strategic launch imperatives to ensure long-term competitive immunity ahead of potential me-too products entering the market [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong commercial opportunities for both RevuForge and Nictimvo, highlighting the high unmet medical need and the compelling profiles of their medicines [5][6] - The company anticipates that RevuForge will be the first menin inhibitor approved in the frontline setting, with ongoing trials expected to support accelerated approval [11][12] Other Important Information - The EVOLVE-two trial, a pivotal frontline trial for a menin inhibitor, has been initiated, targeting newly diagnosed patients with high unmet medical needs [7][28] - The company has seen robust engagement with the FDA regarding its sNDA filing and overall programs [9] Q&A Session Summary Question: What are you seeing regarding repeat prescribers for RevuForge? - Management noted that 44% of tier one and tier two accounts have ordered RevuForge, with about 80% of those having ordered more than once, indicating a growing user base [44][46] Question: Can you comment on patients receiving RevuForge and their transplant rates? - Management stated that anecdotal information suggests patients are being taken to transplant, and they expect patients to return to RevuForge post-engraftment [52][53] Question: What are the month-over-month trends for new patient adds for RevuForge? - Management indicated that while they are not providing specific numbers yet, they are happy with the steady stream of new patients and refill rates [57][60] Question: Can you provide insights on trends in GVHD and any subgroup details? - Management mentioned that it is still early to provide detailed information on GVHD subgroups, but they are collecting real-world evidence [67][70] Question: What portion of the $20 million revenue for RevuForge stems from refill dynamics? - Management stated that both new patient starts and refill dynamics are building, but specific numbers are not available yet [92][95]