Core Insights - Syndax Pharmaceuticals has appointed Dr. Nicholas Botwood as the new Head of Research and Development and Chief Medical Officer, bringing over 25 years of experience in oncology drug development [1][2] - Dr. Botwood previously held senior roles at Bristol Myers Squibb, where he led medical oncology and was responsible for the medical strategy in the U.S. [2][3] - The company aims to leverage Dr. Botwood's expertise to accelerate the growth of its oncology franchises and enhance its R&D strategy [2][3] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [4] - The company's pipeline includes FDA-approved products such as Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [4] - Syndax is committed to advancing cancer care through ongoing clinical trials and aims to unlock the full potential of its product pipeline [4]

Core Viewpoint - Syndax Pharmaceuticals (SNDX) shows significant upside potential with a mean price target of $35.23, indicating a 220% increase from the current price of $11.01 [1] Price Targets and Estimates - The mean estimate consists of 13 short-term price targets with a standard deviation of $10.34, suggesting variability among analysts [2] - The lowest estimate of $17 indicates a 54.4% increase, while the highest estimate predicts a surge of 363.2% to $51 [2] - A low standard deviation indicates a high degree of agreement among analysts regarding price movement [9] Analyst Sentiment and Earnings Estimates - Analysts have shown increasing optimism about SNDX's earnings prospects, with a positive trend in earnings estimate revisions [11] - The Zacks Consensus Estimate for the current year has risen by 5.6% over the past month, with four estimates increasing and no negative revisions [12] - SNDX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] Caution on Price Targets - While price targets are commonly referenced, they can mislead investors, as empirical research shows they rarely indicate actual stock price movements [7][10] - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [8]

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]

[FORWARD-LOOKING STATEMENTS](index=2&type=section&id=FORWARD-LOOKING%20STATEMENTS) This section provides a standard disclaimer regarding forward-looking statements, emphasizing that they are predictions subject to known and unknown risks and uncertainties, advising against reliance on them as future event predictions and stating no duty to update them unless required by law - Forward-looking statements are identified by terms such as '**anticipate**,' '**believe**,' '**could**,' '**estimate**,' '**expects**,' '**intend**,' '**may**,' '**plan**,' '**potential**,' '**predict**,' '**project**,' '**should**,' '**will**,' '**would**' or similar expressions[5](index=5&type=chunk) - Key areas covered by forward-looking statements include estimates for expenses, future revenues, capital requirements, financing needs, R&D activities, clinical trial results, product candidate efficacy and safety, regulatory approvals, license agreements, collaboration success (e.g., Incyte), milestone and royalty payments, strategic plans, intellectual property, market adoption of products (REVUFORJ®, NIKTIMVO™), competitor developments, and impacts of geopolitical actions or economic conditions[8](index=8&type=chunk) - The company cannot guarantee future results, levels of activity, performance, or achievements, and is under no duty to update or revise these statements except as required by law[7](index=7&type=chunk) [PART I. FINANCIAL INFORMATION](index=4&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Unaudited Financial Statements](index=4&type=section&id=Item%201.%20Unaudited%20Financial%20Statements) This section presents the unaudited consolidated financial statements, including balance sheets, statements of comprehensive loss, stockholders' equity, and cash flows, offering a snapshot of the company's financial position and performance [Consolidated Balance Sheets](index=4&type=section&id=Consolidated%20Balance%20Sheets) This section provides a snapshot of the company's financial position, detailing assets, liabilities, and stockholders' equity at specific dates | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | Percentage Change | | :-------------------------------- | :----------------------------- | :------------------------------ | :-------------------- | :------------------ | | **ASSETS** | | | | | | Total current assets | $548,751 | $603,057 | $(54,306) | -9.00% | | Total assets | $640,707 | $724,816 | $(84,109) | -11.60% | | **LIABILITIES AND STOCKHOLDERS' EQUITY** | | | | | | Total current liabilities | $94,551 | $103,549 | $(8,998) | -8.69% | | Total long-term liabilities | $331,097 | $333,143 | $(2,046) | -0.61% | | Total liabilities | $425,648 | $436,692 | $(11,044) | -2.53% | | Total stockholders' equity | $215,059 | $288,124 | $(73,065) | -25.36% | - Cash and cash equivalents remained relatively stable at **$153,993 thousand** as of March 31, 2025, compared to **$154,083 thousand** at December 31, 2024[13](index=13&type=chunk) - Inventory significantly increased from **$366 thousand** at December 31, 2024, to **$4,664 thousand** at March 31, 2025, indicating increased product stock[13](index=13&type=chunk) [Consolidated Statements of Comprehensive Loss](index=5&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) This section details the company's financial performance, including revenues, expenses, and net loss, for the specified periods | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | Percentage Change | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | :------------------ | | Product revenue, net | $20,042 | $0 | $20,042 | N/A | | Total revenues | $20,042 | $0 | $20,042 | N/A | | Cost of product sales | $885 | $0 | $885 | N/A | | Research and development | $61,636 | $56,492 | $5,144 | 9.11% | | Selling, general and administrative | $41,031 | $23,022 | $18,009 | 78.22% | | Collaboration loss | $247 | $0 | $247 | N/A | | Total operating expenses | $103,799 | $79,514 | $24,285 | 30.54% | | Loss from operations | $(83,757) | $(79,514) | $(4,243) | 5.34% | | Royalty interest expense | $(8,049) | $0 | $(8,049) | N/A | | Interest income | $7,183 | $7,256 | $(73) | -1.01% | | Net loss | $(84,846) | $(72,400) | $(12,446) | 17.19% | | Basic loss per share | $(0.98) | $(0.85) | $(0.13) | 15.29% | | Diluted loss per share | $(0.98) | $(0.85) | $(0.13) | 15.29% | - The company began generating product revenue, net, of **$20,042 thousand** in Q1 2025, compared to none in Q1 2024, primarily from the launch of Revuforj[15](index=15&type=chunk) - Selling, general and administrative expenses saw a substantial increase of **78.22%** (**$18,009 thousand**) in Q1 2025 compared to Q1 2024, reflecting commercialization efforts for new products[15](index=15&type=chunk) [Consolidated Statements of Stockholders' Equity](index=6&type=section&id=Consolidated%20Statements%20of%20Stockholders'%20Equity) This section outlines changes in the company's equity, including additional paid-in capital, accumulated comprehensive gain, and accumulated deficit | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------ | :-------------------- | | Additional Paid-In Capital | $1,520,527 | $1,509,110 | $11,417 | | Accumulated Other Comprehensive Gain | $527 | $163 | $364 | | Accumulated Deficit | $(1,306,004) | $(1,221,158) | $(84,846) | | Total Stockholders' Equity | $215,059 | $288,124 | $(73,065) | - Stock-based compensation expense contributed **$10,487 thousand** to additional paid-in capital for the three months ended March 31, 2025[17](index=17&type=chunk) - The accumulated deficit increased by **$84,846 thousand**, reflecting the net loss for the three months ended March 31, 2025[17](index=17&type=chunk) [Consolidated Statements of Cash Flows](index=7&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes the company's cash inflows and outflows from operating, investing, and financing activities for the specified periods | Cash Flow Activity | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Net cash used in operating activities | $(95,162) | $(83,548) | $(11,614) | | Net cash provided by (used in) investing activities | $94,142 | $(99,399) | $193,541 | | Net cash provided by financing activities | $930 | $2,168 | $(1,238) | | Net (decrease) increase cash, cash equivalents and restricted cash | $(90) | $(180,779) | $180,689 | | Cash, cash equivalents and restricted cash—end of period | $154,210 | $114,832 | $39,378 | - Net cash used in operating activities increased by **$11,614 thousand**, primarily due to an increase in operating net loss, accounts receivable, and inventory[20](index=20&type=chunk)[118](index=118&type=chunk) - Investing activities shifted from using **$99,399 thousand** in Q1 2024 to providing **$94,142 thousand** in Q1 2025, mainly due to higher proceeds from maturities of available-for-sale securities[20](index=20&type=chunk)[119](index=119&type=chunk)[120](index=120&type=chunk) [Notes to Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section provides detailed disclosures and explanations for the consolidated financial statements, covering business, accounting policies, significant agreements, and specific financial line items, offering crucial context for understanding the company's financial health and operations [1. Nature of Business](index=8&type=section&id=1.%20Nature%20of%20Business) This section describes the company's core business as a commercial-stage biopharmaceutical company focused on innovative cancer therapies - Syndax Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, with two commercially approved products: Revuforj and Niktimvo[23](index=23&type=chunk) - The company operates in one segment and faces risks including revenue generation, regulatory approval for new indications, supply chain issues, intellectual property protection, and compliance with regulations[23](index=23&type=chunk)[24](index=24&type=chunk) [2. Basis of Presentation](index=8&type=section&id=2.%20Basis%20of%20Presentation) This section outlines the accounting principles and conventions used in preparing the unaudited interim consolidated financial statements - The unaudited interim consolidated financial statements are prepared in conformity with U.S. GAAP and include all normal and recurring adjustments deemed necessary by management[25](index=25&type=chunk)[26](index=26&type=chunk) - Operating results for the three months ended March 31, 2025, are not necessarily indicative of the full year, and these statements should be read in conjunction with the annual audited financial statements in the Form 10-K[26](index=26&type=chunk) [3. Summary of Significant Accounting Policies](index=8&type=section&id=3.%20Summary%20of%20Significant%20Accounting%20Policies) This section details the key accounting policies applied, including revenue recognition, deferred taxes, and recent accounting pronouncements - The company's significant accounting policies are consistent with those disclosed in the 2024 Form 10-K, with no material changes noted[28](index=28&type=chunk) - A full valuation allowance was deemed necessary for all net deferred tax assets as of March 31, 2025, and December 31, 2024, due to expected current year and historical losses[32](index=32&type=chunk) - For collaboration revenue, Incyte is identified as the principal in product sales of Niktimvo, with Syndax recognizing its **50% share of net profits** as 'Collaboration revenue' or net losses as 'Collaboration loss' within operating expenses[34](index=34&type=chunk) - ASU 2024-03, effective for the company for the year ending December 31, 2027, is expected to result in disclosure changes only, expanding disclosures around employee compensation and selling expenses[36](index=36&type=chunk) [4. Significant Collaborative Research and License Agreements](index=10&type=section&id=4.%20Significant%20Collaborative%20Research%20and%20License%20Agreements) This section details the company's key agreements with partners like Incyte, AbbVie, UCB Biopharma, and Eddingpharm, outlining terms for co-commercialization, development, milestones, and royalties - Incyte Collaboration: Syndax co-commercializes and co-promotes axatilimab (Niktimvo™) in the U.S. with Incyte, sharing profits and losses equally. Incyte holds exclusive commercialization rights outside the U.S. and is responsible for **55% of global/U.S.-specific development costs**, while Syndax covers **45%**[37](index=37&type=chunk)[38](index=38&type=chunk) - Under the Incyte Agreements, Syndax is eligible for up to **$220.0 million** in development/regulatory milestones and **$230.0 million** in commercialization milestones, plus tiered royalties on ex-U.S. net sales[39](index=39&type=chunk) - Vitae Pharmaceuticals, Inc. (AbbVie): Syndax holds an exclusive worldwide license for Menin–KMT2A binding interaction inhibitors (Menin Assets), with potential milestone payments up to **$99.0 million** and low single to low double-digit royalties on sales, plus up to **$70.0 million** in sales-based milestones[42](index=42&type=chunk) - UCB Biopharma Sprl: Syndax has an exclusive worldwide license for axatilimab (UCB6352), with potential milestone payments up to **$119.5 million** and low double-digit royalties on sales, plus up to **$250.0 million** in sales-based milestones. A **$10.0 million** milestone was paid in Q1 2025 for a Phase III study[44](index=44&type=chunk)[45](index=45&type=chunk) - Eddingpharm Investment Company Limited: A milestone was achieved in April 2024 for the marketing approval of entinostat in China, resulting in a **$3.5 million** milestone revenue recorded in 2024[47](index=47&type=chunk)[49](index=49&type=chunk) [5. Net Loss per Share Attributable to Common Stockholders](index=14&type=section&id=5.%20Net%20Loss%20per%20Share%20Attributable%20to%20Common%20Stockholders) This section presents the calculation of basic and diluted net loss per share for common stockholders | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------------------------------------------------------------------------------ | :-------------------------------- | :-------------------------------- | | Net loss attributable to common stockholders—basic and diluted (in thousands) | $(84,846) | $(72,400) | | Net loss per share attributable to common stockholders—basic and diluted | $(0.98) | $(0.85) | | Weighted-average number of common shares used to compute net loss per share—basic and diluted | 86,171,889 | 85,213,200 | - Due to net losses in all periods presented, diluted net loss per common share is the same as basic net loss per common share[48](index=48&type=chunk) [6. Other Receivables](index=15&type=section&id=6.%20Other%20Receivables) This section details other amounts owed to the company, specifically a milestone receivable from a licensing agreement - As of March 31, 2025, the company recorded a **$3.6 million** milestone receivable related to the marketing approval of entinostat in China under the Eddingpharm license agreement, with full payment expected by December 2026[49](index=49&type=chunk) [7. Fair Value Measurements](index=15&type=section&id=7.%20Fair%20Value%20Measurements) This section provides information on the fair value of financial assets, including cash, cash equivalents, and investments, categorized by valuation inputs | Asset Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--------------------- | :----------------------------- | :------------------------------ | | Cash and cash equivalents | $153,993 | $154,083 | | Short-term investments | $358,204 | $418,801 | | Long-term investments | $89,938 | $119,520 | | Total assets at fair value | $602,135 | $692,404 | - The fair value of Level 1 instruments (cash equivalents) is determined using quoted market prices in active markets, while Level 2 instruments (short and long-term investments) are valued based on observable market data or models[51](index=51&type=chunk)[53](index=53&type=chunk) - Available-for-sale securities as of March 31, 2025, included commercial paper (**$223,552 thousand**), corporate bonds (**$29,489 thousand**), and US Treasury securities (**$195,101 thousand**), totaling **$448,142 thousand**[55](index=55&type=chunk) [8. Prepaid Expenses and Other Current Assets](index=17&type=section&id=8.%20Prepaid%20Expenses%20and%20Other%20Current%20Assets) This section lists various prepaid expenses and other current assets, providing a breakdown of their balances at different reporting dates | Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------ | | Prepaid insurance | $968 | $1,276 | | Interest receivable on investments | $2,170 | $2,634 | | Prepaid subscriptions | $1,721 | $1,605 | | Prepaid state and local taxes | $329 | $298 | | Prepaid rent | $35 | $107 | | Prepaid inventory | $2,701 | $2,009 | | Other | $538 | $612 | | Total | $8,462 | $8,541 | [9. Inventory](index=17&type=section&id=9.%20Inventory) This section provides a breakdown of the company's inventory, including raw materials, work-in-process, and finished goods | Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :---------------- | :----------------------------- | :------------------------------ | | Raw materials | $1,743 | $0 | | Work-in-process | $572 | $330 | | Finished goods | $2,349 | $36 | | Total Inventory | $4,664 | $366 | - Total inventory increased significantly from **$366 thousand** at December 31, 2024, to **$4,664 thousand** at March 31, 2025, driven by increases in raw materials and finished goods[57](index=57&type=chunk) [10. Accrued Expenses and Other Current Liabilities](index=17&type=section&id=10.%20Accrued%20Expenses%20and%20Other%20Current%20Liabilities) This section details various accrued expenses and other current liabilities, including product revenue allowances, clinical study costs, and compensation | Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------ | | Product revenue allowances | $4,006 | $849 | | Accrued clinical study and trial costs | $22,939 | $23,869 | | Accrued selling, general, and administrative costs | $7,092 | $4,258 | | Accrued compensation and related costs | $7,402 | $14,477 | | Accrued professional fees | $1,109 | $385 | | Accrued milestone costs | $0 | $10,600 | | Accrued royalty interest expense | $12,857 | $4,930 | | Other | $576 | $421 | | Total | $55,981 | $59,789 | [11. Royalty Interest Financing Liability](index=18&type=section&id=11.%20Royalty%20Interest%20Financing%20Liability) This section describes the financing arrangement with Royalty Pharma, detailing the upfront payment, royalty rates, and accounting treatment as a debt instrument - In November 2024, Syndax entered into a Purchase and Sale Agreement with Royalty Pharma, selling rights to certain revenue streams from net sales of axatilimab (Niktimvo™) in the U.S. for an upfront fee of **$350 million**[60](index=60&type=chunk) - Royalty Pharma receives a **13.8% royalty rate** on quarterly net sales of Niktimvo in the U.S., capped at **$822.5 million** in aggregate payments[61](index=61&type=chunk) - The agreement is accounted for as a debt instrument, with an estimated effective annual interest rate of approximately **9.22%** for the period ended March 31, 2025. Royalty interest expense recognized for Q1 2025 was **$8.0 million**[64](index=64&type=chunk) | Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :------------------------------------------ | :----------------------------- | :------------------------------ | | Current portion of royalty interest financing liability | $14,149 | $12,116 | | Royalty interest financing liability, less current portion | $335,852 | $337,884 | | Debt issuance costs | $(6,208) | $(6,319) | | Total royalty interest financing liability, net | $343,793 | $343,681 | [12. Stock-Based Compensation](index=19&type=section&id=12.%20Stock-Based%20Compensation) This section details the company's stock-based compensation plans, including shares available for issuance and the recognized compensation expense - The number of shares available for issuance under the 2015 Omnibus Incentive Plan increased by **3,427,778 shares** in January 2025, and the 2023 Inducement Plan increased by **1,700,000 shares** in total during July and December 2024[66](index=66&type=chunk) | Expense Category | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Research and development | $3,668 | $4,144 | | Selling, general and administrative | $6,712 | $4,755 | | Total Stock-Based Compensation | $10,380 | $8,899 | - As of March 31, 2025, there were **$102.6 million** of unrecognized compensation costs related to unvested stock options and RSUs, expected to be recognized over a weighted-average remaining service period of **2.69 years**[68](index=68&type=chunk) [13. Stockholders' Equity](index=19&type=section&id=13.%20Stockholders'%20Equity) This section provides information on the company's equity structure, including outstanding pre-funded warrants - As of March 31, 2025, there were **285,714 pre-funded warrants** outstanding, which were sold in December 2021[70](index=70&type=chunk) [14. Commitments and Contingencies](index=19&type=section&id=14.%20Commitments%20and%20Contingencies) This section outlines the company's contractual obligations and potential liabilities, including royalty payments and legal proceedings - The company is obligated to pay royalties on net product sales for direct licensed products like Revuforj under the Vitae License Agreement, recorded as cost of product sales[71](index=71&type=chunk)[72](index=72&type=chunk) - As of March 31, 2025, there were no contingent liabilities recorded, and the company was not party to any material legal or arbitration proceedings[73](index=73&type=chunk)[139](index=139&type=chunk) [15. Segment Reporting](index=20&type=section&id=15.%20Segment%20Reporting) This section clarifies that the company operates as a single reportable segment focused on biopharmaceutical cancer therapeutics - The company manages its business activities on a consolidated basis and operates as a single operating and reportable segment: biopharmaceutical cancer therapeutics[74](index=74&type=chunk) - The Chief Operating Decision Maker (CODM) uses consolidated net loss to assess segment performance and for budget and forecasting processes[75](index=75&type=chunk) | Operating Financial Results | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Total Revenue | $20,042 | $0 | | Cost of product sales | $885 | $0 | | Collaboration loss | $247 | $0 | | Research and development expenses | $61,636 | $56,492 | | General and administrative expenses | $34,319 | $18,263 | | Stock-based compensation | $10,380 | $8,900 | | Royalty interest expense | $8,049 | $0 | | Interest expense (income), net | $(7,181) | $(7,201) | | Other expense, net | $221 | $87 | | Segment net loss | $(84,846) | $(72,400) | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the company's financial condition, results of operations, and liquidity, covering business updates, significant risks, and detailed analysis of revenue, expenses, and cash flows for the three months ended March 31, 2025 [Company Overview](index=21&type=section&id=Company%20Overview) This section provides a high-level summary of Syndax's business, including its commercial-stage products and overall financial position - Syndax is a commercial-stage biopharmaceutical company with two FDA-approved cancer therapies, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and ongoing clinical development programs[78](index=78&type=chunk) - The company reported a net loss of **$84.8 million** for the three months ended March 31, 2025, and an accumulated deficit of **$1.3 billion**, with **$602.1 million** in cash, cash equivalents, and investments[79](index=79&type=chunk) [Business Update](index=21&type=section&id=Business%20Update) This section highlights recent commercial and clinical developments for the company's key products, Revuforj and Niktimvo, detailing launch performance, regulatory submissions, and ongoing clinical trials [Revumenib](index=21&type=section&id=Revumenib) This section provides updates on the commercial performance, regulatory submissions, and ongoing clinical trials for Revuforj (revumenib) - Revuforj generated **$20.0 million** in net revenue in Q1 2025, its first full quarter post-U.S. launch in late November 2024[80](index=80&type=chunk) - As of March 2025, **44% of high-priority accounts** have ordered Revuforj, and formal formulary coverage policies are in place for approximately **72% of all managed care lives**[80](index=80&type=chunk) - A supplemental New Drug Application (sNDA) for Revuforj for R/R mutant NPM1 (mNPM1) AML was submitted to the FDA in April 2025, seeking Priority Review under the Real-Time Oncology Review (RTOR) program[80](index=80&type=chunk) - Enrollment began in Q1 2025 for the EVOLVE-2 trial, a pivotal Phase 3 study of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients[80](index=80&type=chunk) - Ongoing trials include BEAT AML (**100% ORR, 95% CRc rate reported**), SAVE (**82% ORR, 48% CR/CRh rate reported**), and trials evaluating revumenib with intensive chemotherapy and for MRD elimination in AML[83](index=83&type=chunk) [Niktimvo™ (axatilimab-csfr)](index=23&type=section&id=Niktimvo%E2%84%A2%20(axatilimab-csfr)) This section provides updates on the commercial launch, administration, and ongoing clinical trials for Niktimvo (axatilimab-csfr) - Niktimvo achieved **$13.6 million** in net revenue in Q1 2025, its first partial quarter post-U.S. launch in late January[83](index=83&type=chunk) - Over **1,250 infusions** of Niktimvo have been administered year-to-date, with approximately **95% of top accounts** and over **70% of bone marrow transplant centers** having ordered as of March 2025. A permanent J-code was assigned by CMS effective April 1, 2025[83](index=83&type=chunk) - Ongoing trials include two Phase 2/3 studies evaluating axatilimab in combination with standard of care therapies (ruxolitinib or corticosteroids) in newly diagnosed chronic GVHD patients[90](index=90&type=chunk) - Enrollment is ongoing in the MAXPIRe trial, a Phase 2 study of axatilimab for idiopathic pulmonary fibrosis (IPF), with topline data anticipated in the second half of 2026[90](index=90&type=chunk) [Significant Risks and Uncertainties](index=24&type=section&id=Significant%20Risks%20and%20Uncertainties) This section outlines the significant economic and business-specific risks and uncertainties that could impact the company's operations and financial results, including macroeconomic conditions and regulatory challenges - Ongoing high interest rates, recession risk, and inflation could negatively affect financing, operating costs, and capital markets[85](index=85&type=chunk) - Other risks include supply chain constraints, geopolitical tensions (e.g., wars in Russia/Ukraine, Israel/Hamas), challenges in obtaining regulatory approval for new indications, acquiring/in-licensing products, pharmaceutical development uncertainty, intellectual property protection, and regulatory compliance[85](index=85&type=chunk)[86](index=86&type=chunk) [Financial Overview](index=24&type=section&id=Financial%20Overview) This section provides an overview of the company's revenue streams and expense categories, detailing how product sales, collaboration agreements, and various operational costs are recognized and managed [Product Revenue, net](index=24&type=section&id=Product%20Revenue,%20net) This section describes the recognition of net product revenue, primarily from the sales of Revuforj - The company began generating product revenue from sales of Revuforj in the U.S. in November 2024, recording **$20.0 million** for the three months ended March 31, 2025[87](index=87&type=chunk)[106](index=106&type=chunk) [Collaboration Revenue/Collaboration Loss](index=24&type=section&id=Collaboration%20Revenue/Collaboration%20Loss) This section explains how revenue or loss from collaborative agreements, specifically for Niktimvo with Incyte, is recognized - Sales of Niktimvo began in February 2025 in collaboration with Incyte, with Syndax recognizing its **50% share of net profits** as 'Collaboration revenue' or net losses as 'Collaboration loss' within operating expenses[89](index=89&type=chunk)[107](index=107&type=chunk) [Research and Development](index=25&type=section&id=Research%20and%20Development) This section outlines the nature and recognition of research and development expenses, including clinical trial costs and employee expenses - R&D expenses are primarily incurred for product candidate development, including clinical trial costs, employee expenses, manufacturing, license fees, and regulatory costs, and are expensed as incurred[91](index=91&type=chunk)[95](index=95&type=chunk) - The company expects to continue significant R&D spending, with late-stage clinical development programs generally incurring higher costs due to increased trial size and duration[92](index=92&type=chunk) [Selling, General and Administrative](index=25&type=section&id=Selling,%20General%20and%20Administrative) This section describes the components of selling, general, and administrative expenses, including commercialization and employee-related costs - SG&A expenses primarily include commercialization costs, employee-related expenses (salaries, benefits, stock-based compensation), and sales and marketing to support the launch of Revuforj and Niktimvo[94](index=94&type=chunk) - These expenses are anticipated to increase further with continued headcount growth to support ongoing research and development and commercialization efforts[97](index=97&type=chunk) [Royalty Interest Expense](index=27&type=section&id=Royalty%20Interest%20Expense) This section explains the incurrence of royalty interest expense related to the Royalty Pharma Purchase and Sale Agreement - Royalty interest expense is incurred due to the Purchase and Sale Agreement with Royalty Pharma, signed in November 2024[98](index=98&type=chunk)[111](index=111&type=chunk) [Other Interest Expense](index=27&type=section&id=Other%20Interest%20Expense) This section details other interest expenses, primarily related to operational and capital leases - Other interest expense relates to operational and capital leases, which decreased in Q1 2025 compared to the prior year[99](index=99&type=chunk)[112](index=112&type=chunk) [Interest Income](index=27&type=section&id=Interest%20Income) This section describes the sources of interest income, including cash, cash equivalents, and investments - Interest income is earned on cash, cash equivalents, and short- and long-term investments, and decreased in Q1 2025 due to fluctuations in interest rates and average balances[100](index=100&type=chunk)[113](index=113&type=chunk) [Other Expense](index=27&type=section&id=Other%20Expense) This section covers other expenses, such as foreign currency revaluation and amortization of debt issuance costs - Other expense includes revaluation of foreign currency related to trade payables and amortization of debt issuance costs, which increased in Q1 2025 primarily due to the latter[101](index=101&type=chunk)[114](index=114&type=chunk) [Recent Accounting Pronouncements](index=27&type=section&id=Recent%20Accounting%20Pronouncements) This section directs readers to further details on new accounting pronouncements within the financial statements notes - For a discussion of new accounting pronouncements, refer to Note 3 - Summary of Significant Accounting Policies[102](index=102&type=chunk) [Critical Accounting Policies and Estimates](index=27&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section confirms the consistency of critical accounting estimates with previous annual reports - There have been no material changes to the critical accounting estimates described in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2024[104](index=104&type=chunk) [Results of Operations](index=28&type=section&id=Results%20of%20Operations) This section provides a detailed comparison of the company's financial performance for the three months ended March 31, 2025, versus the same period in 2024, highlighting key revenue and expense changes [Product Revenue, net](index=28&type=section&id=Product%20Revenue,%20net_results) This section details the product revenue generated from sales of Revuforj for the current period - Product revenue, net from sales of Revuforj was **$20.0 million** for the three months ended March 31, 2025, compared to zero in the prior year period[106](index=106&type=chunk) [Collaboration Revenue/Collaboration Loss](index=28&type=section&id=Collaboration%20Revenue/Collaboration%20Loss_results) This section explains the recognition of collaboration revenue or expense related to Niktimvo sales with Incyte - The company began generating sales of Niktimvo in February 2025, with collaboration revenue or expense reflecting its **50% share of profit/loss** with Incyte[107](index=107&type=chunk) [Cost of Product Sales](index=28&type=section&id=Cost%20of%20Product%20Sales_results) This section describes the components of cost of product sales, including cost of goods sold and royalties for Revuforj - Cost of product sales includes the cost of goods sold and royalties associated with Revuforj sales in the United States[108](index=108&type=chunk) [Research and Development](index=28&type=section&id=Research%20and%20Development_results) This section analyzes the changes in research and development expenses, attributing them to specific product programs and personnel costs | Category | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Revumenib-related costs | $20,805 | $31,441 | $(10,636) | | Axatilimab-related costs | $19,711 | $5,601 | $14,110 | | Other research and development programs | $921 | $966 | $(45) | | Personnel cost and other expenses | $16,531 | $14,340 | $2,191 | | Stock-based compensation | $3,668 | $4,144 | $(476) | | Total research and development expenses | $61,636 | $56,492 | $5,144 | - Total R&D expenses increased by **$5.1 million**, primarily due to a **$14.1 million** increase in axatilimab-related costs (driven by IPF and frontline cGVHD trials, and a **$10.0 million** milestone payment to UCB) and a **$2.2 million** increase in personnel costs, partially offset by a **$10.6 million** decrease in revumenib-related costs[109](index=109&type=chunk)[115](index=115&type=chunk) [Selling, General and Administrative](index=29&type=section&id=Selling,%20General%20and%20Administrative_results) This section examines the increase in selling, general, and administrative expenses, linking it to commercialization efforts and personnel growth | Category | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Commercial-related expenses | $10,825 | $5,833 | $4,992 | | Other selling, general and administrative expenses | $4,412 | $4,103 | $309 | | Personnel cost and other expenses | $19,082 | $8,330 | $10,752 | | Stock-based compensation | $6,712 | $4,756 | $1,956 | | Total selling, general and administrative expenses | $41,031 | $23,022 | $18,009 | - Total SG&A expenses increased by **$18.0 million**, primarily due to a **$4.9 million** increase in commercial-related costs and a **$10.7 million** increase in personnel costs, both driven by the commercialization of Revuforj and Niktimvo[110](index=110&type=chunk)[116](index=116&type=chunk) [Royalty Interest Expense](index=29&type=section&id=Royalty%20Interest%20Expense_results) This section explains the increase in royalty interest expense due to the Royalty Pharma agreement - Royalty interest expense increased due to the interest recognized related to the Royalty Pharma Purchase and Sale Agreement, signed in November 2024[111](index=111&type=chunk) [Other Interest Expense](index=29&type=section&id=Other%20Interest%20Expense_results) This section details the decrease in other interest expense, primarily from capital leases - Other interest expense decreased due to less interest recognized related to capital leases[112](index=112&type=chunk) [Interest Income](index=29&type=section&id=Interest%20Income_results) This section attributes the change in interest income to fluctuations in interest rates and investment balances - Interest income decreased due to the fluctuation of interest rates and average balance of cash equivalents and short and long-term investments[113](index=113&type=chunk) [Other Expense](index=29&type=section&id=Other%20Expense_results) This section explains the increase in other expense, mainly due to the amortization of debt issuance costs - Other expense increased primarily related to the current period amortization of debt issuance costs from the Royalty Pharma Purchase and Sale Agreement[114](index=114&type=chunk) [Liquidity and Capital Resources](index=30&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's cash flow activities, current liquidity, and future funding requirements, including details on the Royalty Pharma agreement and At-the-Market Offering Program [Cash Flows](index=30&type=section&id=Cash%20Flows) This section provides a summary of cash flows from operating, investing, and financing activities | Cash Flow Activity | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :---------------------------------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Net cash used in operating activities | $(95,162) | $(83,548) | | Net cash provided by (used in) investing activities | $94,142 | $(99,399) | | Net cash provided by financing activities | $930 | $2,168 | | Net (decrease) increase cash, cash equivalents and restricted cash | $(90) | $(180,779) | [Net Cash Used in Operating Activities](index=30&type=section&id=Net%20Cash%20Used%20in%20Operating%20Activities) This section analyzes the increase in net cash used in operating activities, attributing it to changes in net loss, receivables, and inventory - Net cash used in operating activities increased to **$95.2 million** in Q1 2025 from **$83.5 million** in Q1 2024, primarily due to an increase in operating net loss, accounts receivable, and inventory, and decreases in prepaid expenses and collaboration payables[118](index=118&type=chunk) [Net Cash Provided by Investing Activities](index=30&type=section&id=Net%20Cash%20Provided%20by%20Investing%20Activities) This section details the shift in investing activities from cash usage to cash provision, driven by maturities of available-for-sale securities - Net cash provided by investing activities was **$94.1 million** in Q1 2025, resulting from **$104.7 million** from maturities of available-for-sale securities offset by **$10.5 million** in purchases[119](index=119&type=chunk) [Net Cash Provided by Financing Activities](index=30&type=section&id=Net%20Cash%20Provided%20by%20Financing%20Activities) This section explains the decrease in net cash provided by financing activities, mainly due to lower proceeds from stock option exercises - Net cash provided by financing activities decreased by **$1.2 million** in Q1 2025 compared to the prior year, mainly due to lower proceeds from stock option exercises[121](index=121&type=chunk) [Purchase and Sale Agreement](index=30&type=section&id=Purchase%20and%20Sale%20Agreement) This section describes the agreement with Royalty Pharma, including the upfront payment and royalty terms for Niktimvo sales - In October 2024, the company entered into an agreement with Royalty Pharma, receiving an upfront payment of **$350.0 million** (gross) for the right to receive **13.8%** on quarterly net sales of Niktimvo in the U.S., capped at **$822.5 million**[122](index=122&type=chunk) [Future Funding Requirements](index=30&type=section&id=Future%20Funding%20Requirements) This section outlines the company's expected capital needs and strategies for financing future operations and growth - The company believes its available cash, cash equivalents, investments, and Revuforj/Niktimvo revenues are sufficient to fund existing and planned near-term cash requirements[124](index=124&type=chunk)[127](index=127&type=chunk)[131](index=131&type=chunk) - Primary uses of capital include compensation, third-party clinical R&D services, clinical costs, commercialization costs, legal/regulatory expenses, and general overhead[124](index=124&type=chunk) - Future capital requirements depend on factors such as clinical trial progress, regulatory approvals, intellectual property costs, market acceptance, manufacturing, pricing/reimbursement, and potential acquisitions[130](index=130&type=chunk) - The company expects to finance future cash needs through equity offerings, debt financings, and additional funding from license and collaboration arrangements until substantial product revenue is generated[127](index=127&type=chunk)[131](index=131&type=chunk) [At-the-Market Offering Program](index=32&type=section&id=At-the-Market%20Offering%20Program) This section provides details on the company's At-the-Market (ATM) Program, including its size and remaining availability - The company has a **$200.0 million** At-the-Market (ATM) Program established in May 2023. No shares were sold under this program for the three months ended March 31, 2025[132](index=132&type=chunk) - As of March 31, 2025, **$157.9 million** remained available under the 2023 ATM Program[132](index=132&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section describes the company's exposure to market risk, primarily interest rate sensitivity, and its approach to managing investment activities - The company's primary market risk exposure is interest rate sensitivity, affecting its cash, cash equivalents (**$154.0 million**), and short- and long-term investments (**$448.1 million**)[133](index=133&type=chunk) - Investment objectives prioritize liquidity and principal preservation while maximizing interest income without significantly increasing risk. Due to short-term maturities and low risk, a **100 basis point change** in interest rates would not materially affect the fair value of investments[133](index=133&type=chunk) - Inflation and changing prices have not had a significant impact on results of operations for the periods presented[134](index=134&type=chunk) [Item 4. Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) This section details management's evaluation of the effectiveness of the company's disclosure controls and procedures and reports on any changes in internal control over financial reporting [Disclosure Controls and Procedures and Internal Control over Financial Reporting](index=32&type=section&id=Disclosure%20Controls%20and%20Procedures%20and%20Internal%20Control%20over%20Financial%20Reporting) This section confirms the effectiveness of the company's disclosure controls and procedures as evaluated by management - As of March 31, 2025, management, including the CEO and CFO, concluded that the company's disclosure controls and procedures are effective[135](index=135&type=chunk) - Disclosure controls ensure that required information is recorded, processed, summarized, and reported within SEC specified time periods and communicated to management for timely decisions[135](index=135&type=chunk) [Changes in Internal Control over Financial Reporting](index=34&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) This section reports on any material changes to the company's internal control over financial reporting during the quarter - There was no change in the company's internal control over financial reporting during the quarter ended March 31, 2025, that has materially affected, or is reasonably likely to materially affect, its internal control over financial reporting[137](index=137&type=chunk) [PART II. OTHER INFORMATION](index=35&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=35&type=section&id=Item%201.%20Legal%20Proceedings) This section confirms the absence of material legal or arbitration proceedings against the company as of the reporting date - As of March 31, 2025, the company was not party to any material legal or arbitration proceedings, and no governmental proceedings are pending or contemplated[139](index=139&type=chunk) [Item 1A. Risk Factors](index=35&type=section&id=Item%201A.%20Risk%20Factors) This section refers to the comprehensive risk factors disclosed in the company's Annual Report on Form 10-K and highlights any new or updated risks - There have been no material changes in the risk factors previously disclosed in the 2024 Form 10-K[140](index=140&type=chunk) - A new risk factor includes potential disruptions at the FDA and other government agencies due to layoffs, funding shortages, or global health concerns, which could negatively impact the business by slowing product review and approval times[141](index=141&type=chunk) [Item 5. Other Information](index=35&type=section&id=Item%205.%20Other%20Information) This section provides other relevant information not covered elsewhere, specifically detailing trading arrangements adopted or terminated by directors and officers [Trading Arrangements](index=35&type=section&id=Trading%20Arrangements) This section details Rule 10b5-1 trading arrangements adopted or amended by company directors and officers | Name and Position | Action | Adoption/Termination Date | Rule 10b5-1* | Non-Rule 10b5-1** | Total Shares of Common Stock to be Sold | Total Shares of Common Stock to be Purchased | Expiration Date | | :-------------------------------- | :----------- | :------------------------ | :----------- | :---------------- | :-------------------------------------- | :------------------------------------------- | :-------------- | | Michael A. Metzger, CEO, Director | Amendment | 3/11/2025 | X | | 157,307 | - | 9/9/2025 | | Dennis Podlesak, Director | Adoption | 3/5/2025 | X | | 65,600 | - | 3/31/2026 | [Item 6. Exhibits](index=38&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed as part of the Form 10-Q, including corporate governance documents, certifications, and XBRL data [SIGNATURES](index=39&type=section&id=SIGNATURES) This section contains the official signatures of the company's authorized officers, certifying the submission of the Form 10-Q - The report was signed on May 5, 2025, by Michael A. Metzger (Chief Executive Officer) and Keith A. Goldan (Chief Financial Officer and Treasurer)[150](index=150&type=chunk)[151](index=151&type=chunk)

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]