Core Insights - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing innovative cancer therapies [2] - The company has an FDA-approved pipeline that includes Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [2] - Syndax is committed to advancing cancer care through ongoing clinical trials [2] Upcoming Events - Syndax's CEO and management team will participate in the Guggenheim SMID Cap Biotech Conference on February 6, 2025, at 10:30 a.m. ET [4] - The company will also be featured in Citi's 2025 Virtual Oncology Leadership Summit on February 19, 2025, at 9:00 a.m. ET [4] - Live webcasts of these events will be available on the company's website [1]

Company Achievements - Launched Revuforj® (revumenib), the first and only FDA-approved menin inhibitor, for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older [2][6] - Revumenib was added to the NCCN Clinical Practice Guidelines for AML and ALL as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement [2][6] - Received FDA approval for Niktimvo™ (axatilimab-csfr) for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg [2][10] - Announced a $350 million royalty funding agreement with Royalty Pharma based on U.S. net sales of Niktimvo, which is expected to help the company reach profitability [7] Clinical Trial Results - Met the primary endpoint in the Phase 2 cohort of the AUGMENT-101 trial of revumenib in 64 adults with R/R mNPM1 AML [6] - Reported an overall response rate (ORR) of 100% and a composite complete remission (CRc) rate of 95% in the BEAT AML trial evaluating revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients [6] - Presented an ORR of 82% and a CR/CRh rate of 48% in the SAVE trial evaluating revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or MPAL [6] - Published results from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD in the New England Journal of Medicine [10] 2025 Milestones - Submit a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025, with potential FDA approval around year-end 2025 [11] - Initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy in Q1 2025 [11] - Launch Niktimvo in the U.S. in early Q1 2025, co-commercialized by Syndax and Incyte [11] - Complete enrollment in the MAXPIRe Phase 2 IPF trial in 2025, with topline data expected in 2026 [11] Industry Impact - Revumenib and axatilimab are positioned to address multi-billion-dollar markets, with potential for expansion into additional indications [3] - The company's robust clinical data and first-mover advantage with Revuforj are expected to drive U.S. adoption as the preferred menin inhibitor [11] - Syndax's strong cash position, including the $350 million royalty funding agreement, is expected to fund operations through profitability [7]

Core Viewpoint - Kuehn Law, PLLC is investigating potential breaches of fiduciary duties by officers and directors of Syndax Pharmaceuticals, Inc. related to self-dealing, which may entitle shareholders to damages and corporate governance reforms [1]. Group 1 - Kuehn Law is a shareholder litigation law firm conducting an investigation into Syndax Pharmaceuticals, Inc. for possible self-dealing by its officers and directors [1]. - The investigation may lead to shareholders being entitled to damages and reforms in corporate governance [1]. - Long-term shareholders of Syndax Pharmaceuticals are encouraged to contact Kuehn Law for a free consultation regarding their rights [2]. Group 2 - The firm emphasizes the importance of shareholder participation in maintaining the integrity and fairness of financial markets [3]. - Kuehn Law offers to cover all case costs and does not charge its investor clients, highlighting a no-obligation consultation [2]. - The firm encourages immediate contact from shareholders due to potential time limitations in enforcing their rights [2].

Group 1 - Purcell & Lefkowitz LLP is investigating a potential breach of fiduciary duty claim involving the board of directors of Syndax Pharmaceuticals, Inc. [1] - The law firm is dedicated to representing shareholders nationwide who are victims of securities fraud and corporate misconduct [2] - Shareholders of Syndax Pharmaceuticals, Inc. can obtain additional information regarding the investigation free of charge [1]

–      Primary endpoint met with CR/CRh rate of 23% in pivotal Ph 2 cohort of R/R mNPM1 AML patients (p-value = 0.0014) ––      47% (30/64) ORR in a heavily pre-treated population, including 75% with prior venetoclax exposure in the efficacy population ––      Favorable safety and tolerability profile; only 5% of patients discontinued due to treatment-related adverse events ––      Results highlight consistency of revumenib's compelling clinical profile ––      sNDA filing in R/R mNPM1 AML expected in 1H25, ...

Financial Data and Key Metrics Changes - The company reported operating expenses of $102.1 million for Q3 2024, which included $71 million in research and development and $31.1 million in selling, general, and administrative expenses [55] - The company narrowed its full-year 2024 guidance, expecting research and development expenses to be between $245 million and $250 million, and total expenses to be between $365 million and $370 million [55] Business Line Data and Key Metrics Changes - The FDA approved Niktimvo for chronic graft-versus-host disease (GVHD), marking a significant milestone for the company [9] - Revumenib is anticipated to receive FDA approval for relapsed or refractory KMT2A rearranged acute leukemia by December 26, 2024, with compelling clinical data supporting its use [13][14] Market Data and Key Metrics Changes - The total addressable market for third-line or later chronic GVHD treatment in the US is estimated to be between $1.5 billion and $2 billion [43] - The combined accessible population for KMT2A and NPM1 acute leukemias in the relapsed or refractory setting is estimated to be between 5,000 and 6,500 patients, with a market opportunity approaching $2 billion in the US [50] Company Strategy and Development Direction - The company aims to leverage the recent $350 million royalty agreement for Niktimvo to strengthen its balance sheet and fund the commercialization of both Niktimvo and revumenib [8][52] - The company is focused on establishing a first-mover advantage in the menin inhibitor space, with plans for a pivotal Phase 3 trial in combination with venetoclax and azacitidine for newly diagnosed patients [34][45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launches of Niktimvo and revumenib, highlighting the strong commercial opportunities and the need for new treatment options in the market [41][49] - The management team emphasized the importance of rapid data publication and guideline inclusion following drug approvals to maximize market penetration [61][68] Other Important Information - The company has a robust clinical development program for revumenib, with multiple ongoing trials and promising data supporting its use in various settings [19][31] - The company has established partnerships, such as with Incyte, to enhance its commercial efforts and leverage existing relationships in the chronic GVHD community [41][43] Q&A Session Summary Question: What is the process for getting into guidelines post-approval? - Management explained that after drug approval, data must be published, and then submitted for inclusion in guidelines, with a quick turnaround expected based on past experiences [60] Question: What is the expected maintenance percentage for revumenib in the commercial setting? - Management indicated that the maintenance percentage observed in trials may continue in the commercial setting, with expectations for more patients to be treated post-transplant [64] Question: Does the royalty agreement accelerate any development programs? - Management confirmed that the royalty agreement strengthens the balance sheet and allows for aggressive advancement of planned trials and programs [67] Question: What is the expected clinical bar for CR/CRh rate for pivotal NPM1 data? - Management stated that a CR/CRh rate of 20% to 30% is considered approvable, with confidence in achieving results within that range based on previous data [81] Question: Is the updated KMT2A data the most recent analysis submitted to the FDA? - Management clarified that while they cannot confirm specifics about the FDA review, the updated data is consistent and supportive of approval [88]

| --- | --- | --- | --- | |-------------------------------------|-------|-------|-----------------------------------------------------------------| | | | | | | | | | | | Syndax Reimagining Cancer Treatment | | | | | | | | Third Quarter Financial Results Presentation / November 5, 2024 | Forward-looking statements disclosure This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipa ...

Syndax Pharmaceuticals (SNDX) came out with a quarterly loss of $0.98 per share versus the Zacks Consensus Estimate of a loss of $1.13. This compares to loss of $0.73 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 13.27%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $0.91 per share when it actually produced a loss of $0.80, delivering a surprise of 12.09%.Over the last four quarters, ...

Financial Performance - Syndax reported a net loss of $84.1 million, or $0.98 per share, for Q3 2024, compared to a net loss of $51.1 million, or $0.73 per share, in the same period last year[14]. - Total operating expenses for the three months ended September 30, 2024, were $102.1 million, up from $56.4 million in the same period last year, representing an increase of 81.2%[25]. - The net loss for the three months ended September 30, 2024, was $84.1 million, compared to a net loss of $51.1 million for the same period in 2023, reflecting a year-over-year increase of 64.5%[25]. - Research and development expenses for the three months ended September 30, 2024, were $71.0 million, a 81.6% increase from $39.1 million in the same period last year[25]. Expenses - Research and development expenses increased to $71.0 million in Q3 2024 from $39.1 million in the prior year, primarily due to higher clinical development and pre-commercial manufacturing costs[12]. - Selling, general and administrative expenses rose to $31.1 million in Q3 2024, up from $17.3 million in the prior year, driven by increased commercial readiness activities for revumenib and axatilimab[13]. - The company expects full-year 2024 research and development expenses to be between $245 million and $250 million, and total operating expenses to be between $365 million and $370 million[15]. Cash and Assets - As of September 30, 2024, Syndax had cash, cash equivalents, and investments totaling $399.6 million[11]. - Syndax's total assets decreased from $612.9 million on December 31, 2023, to $425.8 million on September 30, 2024, a decline of 30.5%[24]. - Total stockholders' equity decreased from $554.2 million on December 31, 2023, to $366.4 million on September 30, 2024, a decrease of 33.8%[24]. Product Development and Approvals - Revumenib's New Drug Application (NDA) is under Priority Review with a PDUFA action date set for December 26, 2024[3]. - Topline data from the AUGMENT-101 trial for R/R mNPM1 AML is expected in Q4 2024, with a potential supplemental NDA filing in the first half of 2025[4]. - Niktimvo received FDA approval for chronic GVHD treatment and is anticipated to launch in the U.S. by early Q1 2025[9]. - Niktimvo (axatilimab-csfr) is approved in the U.S. for chronic graft-versus-host disease (GVHD) after failure of at least two prior systemic therapies[20]. - The company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine by year-end 2024[8]. - The company has ongoing clinical trials for axatilimab, including a Phase 2 trial in chronic GVHD and a Phase 2 trial in idiopathic pulmonary fibrosis[20]. Revenue - Syndax reported milestone and license revenue of $12.5 million for the three months ended September 30, 2024, compared to $0 for the same period in 2023[25]. Stock Information - Syndax's common stock outstanding increased from 84,826,632 shares to 85,285,488 shares from December 31, 2023, to September 30, 2024[24]. - The weighted-average number of common stock used to compute net loss per share increased from 69,645,888 to 85,433,569 shares year-over-year[25].

–  New revumenib and Niktimvo™ clinical data will be highlighted at 66th ASH Annual Meeting –– mNPM1 AML topline data from AUGMENT-101 expected in 4Q24; potential sNDA filing in 1H25 –– Revumenib NDA in R/R KMT2Ar acute leukemia is being reviewed under RTOR; PDUFA action date of December 26, 2024 –– Niktimvo approved by U.S. FDA for treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg ––  $350 million royalty funding ...