Core Insights - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [2] - The company has FDA-approved products in its pipeline, including Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [2] - Syndax is actively conducting clinical trials to maximize the potential of its pipeline [2] Upcoming Events - Michael A. Metzger, CEO of Syndax, and the management team will participate in investor conferences [1] - A fireside chat at the Bank of America Merrill Lynch Vegas Health Care Conference is scheduled for May 15, 2025, at 9:20 a.m. PT/12:20 p.m. ET [3] - A virtual fireside chat at the TD Cowen 6th Annual Oncology Innovation Summit will take place on May 28, 2025, at 10:30 a.m. ET [3]

Core Insights - Syndax Pharmaceuticals announced positive results from the pivotal Phase 2 portion of the AUGMENT-101 trial for revumenib in relapsed or refractory mutant NPM1 acute myeloid leukemia (mNPM1 AML) patients, achieving nearly 50% overall response rate [1][2] - The company submitted a supplemental New Drug Application (sNDA) for revumenib in April 2025 under the FDA's Real-Time Oncology Review (RTOR) program [1][2] Group 1: Trial Results - The primary efficacy endpoint was met with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% among the first 64 adult patients [5] - The overall response rate (ORR) was reported at 47%, with 17% of responders proceeding to hematopoietic stem cell transplant (HSCT) while in remission [6] - The median overall survival (OS) for all patients was 4.0 months, while responders had a median OS of 23.3 months [7] Group 2: Patient Demographics and Treatment Background - The efficacy-evaluable population had a median age of 65, with 36% having received three or more prior lines of therapy [4] - 75% of patients were previously treated with venetoclax, indicating a heavily pretreated population [4] Group 3: Safety Profile - The safety population included 84 patients, with treatment-emergent serious adverse events occurring in ≥5% of patients, including febrile neutropenia (21%) and differentiation syndrome (13%) [8] - The safety profile of revumenib was consistent with previously reported data, with 12% of patients experiencing dose reductions due to adverse events [8] Group 4: Background on mNPM1 AML - Mutations in the NPM1 gene are the most common genetic alteration in adult AML, observed in approximately 30% of cases [9] - There are currently no approved targeted therapies specifically for mNPM1 AML, highlighting a significant unmet medical need [9] Group 5: Revumenib Overview - Revumenib is an oral, first-in-class selective menin inhibitor, previously approved for R/R acute leukemia with KMT2A translocation [10][11] - The drug is in development for R/R mNPM1 AML, with ongoing trials planned for combination therapies [11]

Core Viewpoint - Syndax Pharmaceuticals reported a quarterly loss of $0.98 per share, which was better than the Zacks Consensus Estimate of a loss of $1.04, indicating an earnings surprise of 5.77% [1] Financial Performance - The company posted revenues of $20.04 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 36.69%, compared to zero revenues a year ago [2] - The current consensus EPS estimate for the upcoming quarter is -$0.99 on revenues of $17.81 million, and for the current fiscal year, it is -$3.99 on revenues of $81.74 million [7] Stock Performance - Syndax shares have increased by approximately 3.9% since the beginning of the year, while the S&P 500 has declined by 3.3% [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating it is expected to perform in line with the market in the near future [6] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Syndax belongs, is currently in the top 34% of over 250 Zacks industries, suggesting a favorable outlook [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

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]

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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37708 Syndax Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 32-0162505 (State or Other J ...

Financial Performance - Syndax reported $20.0 million in net revenue from Revuforj in the first quarter of 2025, marking the first full quarter of its U.S. launch[5] - Niktimvo generated $13.6 million in net revenue in its first partial quarter of launch, with Syndax recording a $0.2 million share of the net commercial loss[10] - Product revenue for the first quarter of 2025 was $20,042,000, compared to $0 in the same period of 2024[34] - Net loss for Q1 2025 was $84,846,000, compared to a net loss of $72,400,000 in Q1 2024, indicating a 17.2% increase in losses[34] - Basic loss per share attributable to common stockholders was $0.98 in Q1 2025, compared to $0.85 in Q1 2024, reflecting a 15.3% increase[34] Expenses - Research and development expenses increased to $61.6 million in Q1 2025, up from $56.5 million in the same period last year, primarily due to axatilimab-related costs[12] - Selling, general and administrative expenses rose to $41.0 million in Q1 2025, compared to $23.0 million in the prior year, driven by increased employee-related expenses[13] - The company expects R&D expenses for Q2 2025 to be between $70 million and $75 million, with total expenses (R&D plus SG&A) projected at $110 million to $115 million[15] - Total operating expenses increased to $103,799,000 in Q1 2025, up from $79,514,000 in Q1 2024, representing a 30.5% increase[34] - Research and development expenses rose to $61,636,000 in Q1 2025, compared to $56,492,000 in Q1 2024, marking an increase of 9.5%[34] - Selling, general and administrative expenses increased significantly to $41,031,000 in Q1 2025 from $23,022,000 in Q1 2024, a rise of 78.3%[34] Cash and Assets - The company had cash, cash equivalents, and investments totaling $602.1 million as of March 31, 2025, expected to fund operations to profitability[9] - Cash, cash equivalents, and investments decreased to $602,135,000 as of March 31, 2025, down from $692,404,000 as of December 31, 2024, a decline of 13.0%[32] - Total assets decreased to $640,707,000 as of March 31, 2025, compared to $724,816,000 as of December 31, 2024, a reduction of 11.6%[32] - Total stockholders' equity fell to $215,059,000 as of March 31, 2025, down from $288,124,000 as of December 31, 2024, a decrease of 25.3%[32] - Common stock outstanding increased to 86,047,032 shares as of March 31, 2025, compared to 85,694,443 shares as of December 31, 2024[32] Product Development and Market Opportunities - Syndax plans to initiate multiple trials of revumenib in combination with standard care regimens for newly diagnosed acute leukemia patients starting in the second half of 2025[8] - The company submitted a supplemental New Drug Application (sNDA) for revumenib for the treatment of R/R mNPM1 AML in April 2025, seeking Priority Review[5] - Revuforj and Niktimvo are positioned to unlock multi-billion-dollar opportunities in their respective markets, supported by strong clinical data and ongoing trials[3]

Financial Performance - Syndax Pharmaceuticals reported $20.0 million in net revenue from Revuforj® (revumenib) in the first full quarter of its launch [1][4] - Niktimvo™ (axatilimab-csfr) generated $13.6 million in net revenue in its first partial quarter of launch, as reported by Incyte [1][8] - The company had cash, cash equivalents, and investments totaling $602.1 million as of March 31, 2025, which is expected to fund operations until profitability [1][7] Business Developments - The company submitted a supplemental New Drug Application (sNDA) for revumenib for the treatment of relapsed or refractory (R/R) mutant NPM1 acute myeloid leukemia (AML) [1][4] - A pivotal frontline trial of revumenib in combination with venetoclax and azacitidine for mNPM1 and KMT2Ar AML has been initiated [1][4] - Multiple ongoing trials are evaluating revumenib in various combinations and settings, including a Phase 1 trial showing a 100% overall response rate in newly diagnosed mNPM1 or KMT2Ar AML patients [4][5] Research and Development - Research and development expenses for the first quarter of 2025 increased to $61.6 million from $56.5 million in the prior year, driven by costs related to axatilimab and ongoing clinical trials [10] - Selling, general, and administrative expenses rose to $41.0 million from $23.0 million, primarily due to increased employee-related expenses and marketing efforts for Revuforj [11] - The company plans to initiate multiple trials of revumenib in combination with standard care regimens for newly diagnosed acute leukemia patients starting in the second half of 2025 [5][10] Future Outlook - For the second quarter of 2025, the company expects research and development expenses to be between $70 million and $75 million, with total operating expenses projected to be between $110 million and $115 million [13] - The company anticipates that its current cash position, along with expected product revenue, will enable it to reach profitability [14]

Core Viewpoint - Syndax Pharmaceuticals will report its first quarter 2025 financial results and provide a business update on May 5, 2025 [1] Financial Results Announcement - The management will host a conference call and live audio webcast at 4:30 p.m. ET on May 5, 2025, to discuss financial results and business updates [2] - Access to the live audio webcast and slides will be available on the Company's website, with a replay accessible approximately 24 hours after the call for 90 days [2] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing an innovative pipeline of cancer therapies [3] - Key products in the pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody targeting the CSF-1 receptor [3] - The company is committed to advancing cancer care through various clinical trials [3]