SNDX 8-K
Syndax Pharmaceuticals Inc (SNDX)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02. Results of Operations and Financial Condition.
On November 3, 2025, Syndax Pharmaceuticals, Inc. (the “Company”) issued a press release and presentation announcing its financial results for the quarter and six months ended September 30, 2025. A copy of the press release and presentation are furnished as Exhibits 99.1 and 99.2 to this Current Report on Form 8-K and are incorporated herein by reference.
The information contained in this Item 2.02 and in Exhibits 99.1 and 99.2 attached hereto is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any of the Company’s filings under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits.
Exhibit
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Description
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99.1 |
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99.2 |
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104 |
Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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SYNDAX PHARMACEUTICALS, INC. |
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By: |
/s/ Michael A. Metzger |
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Michael A. Metzger |
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Chief Executive Officer |
Dated: November 3, 2025
Exhibit 99.1
Syndax Reports Third Quarter 2025 Financial Results and Provides Business Update
– Revuforj FDA-approved in R/R NPM1m AML on October 24, 2025 –
company to profitability –
NEW YORK, NY, November 03, 2025 (GLOBE NEWSWIRE) – Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today reported its financial results for the third quarter ended September 30, 2025, and provided a business update.
“The third quarter was another remarkable period of commercial and pipeline execution for Syndax. Demand remained strong for Revuforj and Niktimvo with over $75 million in combined net sales for the quarter,” said Michael A. Metzger, Chief Executive Officer. “We also furthered our leadership in menin inhibition with the addition of Revuforj to the NCCN Guidelines for R/R NPM1m AML in late September followed by FDA approval in late October. Our expansion into this second indication is underway and we are making great progress driving awareness and generating demand. Additionally, we continue to advance the development of both Revuforj and Niktimvo in the frontline setting, further unlocking their multi-billion-dollar potential.”
Recent Business Highlights and Anticipated Milestones
Revuforj®(revumenib)
Niktimvo™ (axatilimab-csfr)
Third Quarter 2025 Financial Results
As of September 30, 2025, Syndax had cash, cash equivalents, and short- and long-term investments of $456.1 million and 87.2 million common shares and prefunded warrants outstanding.
Total revenue for the third quarter of 2025 was $45.9 million, which consisted of $32.0 million in Revuforj net revenue and $13.9 million in Niktimvo collaboration revenue. The collaboration revenue is derived from the $45.8 million in Niktimvo net revenue that was previously reported by
the Company's partner Incyte. Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses.
Third quarter 2025 research and development expenses decreased to $56.3 million from $71.0 million for the comparable prior year period. The decrease was primarily the result of a $15 million milestone payment paid by the Company upon Niktimvo's approval, incurred in the third quarter of 2024 and not incurred in the 2025 period. Also contributing to the decrease was lower revumenib-related costs due the completion of the registrational trial in R/R NPM1m AML that was ongoing in the prior period and a reduction in CMC expenses due to the capitalization of inventory for commercial use.
Third quarter 2025 selling, general and administrative expenses increased to $44.9 million from $31.1 million for the comparable prior year period. The increase was primarily due to higher commercial costs and personnel and stock-based compensation expenses related to the commercial launches of Revuforj and Niktimvo in the 2025 period.
For the three months ended September 30, 2025, Syndax reported a net loss attributable to common stockholders of $60.7 million, or $0.70 per share, compared to a net loss attributable to common stockholders of $84.1 million, or $0.98 per share, for the comparable prior year period.
Financial Guidance
For the full year of 2025, the Company expects total research and development plus selling, general and administrative expenses to be $380 to $385 million, compared to prior guidance of $370 to $390 million, both estimates excluding an estimated $45 million in non-cash stock compensation expense.
Syndax expects that its operating expense base will remain stable over the next few years. As a result, Syndax expects that its cash, cash equivalents and short- and long-term investments, combined with its anticipated product revenue and interest income, will enable the company to reach profitability.
Conference Call and Webcast
In connection with the earnings release, Syndax's management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, November 3, 2025.
The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following:
Conference ID: Syndax3Q25
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: https://sndx-3q25.open-exchange.net
For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company's website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.
About Revuforj® (revumenib)
Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.
Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.
Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.
About Niktimvo™ (axatilimab-csfr)
Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease
(GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).
In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.
Axatilimab is being studied in frontline combination trials in chronic GVHD, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774). Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).
About Syndax
Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit www.syndax.com/ or follow the Company on X and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution
requirements, the potential use of its product candidates to treat various cancer indications and fibrotic diseases, and Syndax's expected full year total operating expenses, including its estimated non-cash stock compensation expense. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's or Niktimvo’s commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Niktimvo is a trademark of Incyte.
All other trademarks are the property of their respective owners.
References
1. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR; Composite complete remission (CRc) includes CR, CRh, CRp, and CRi.
CR = Complete remission
CRh = Complete remission with partial hematologic recovery
CRp = Complete remission with incomplete platelet recovery
CRi = Complete remission with incomplete count recovery
MLFS = Morphologic leukemia-free state
PR = Partial response
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
[email protected]
Tel 781.684.9827
SNDX-G
SYNDAX PHARMACEUTICALS, INC. |
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(unaudited) |
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CONDENSED CONSOLIDATED BALANCE SHEETS |
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September 30, |
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December 31, |
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(In thousands) |
2025 |
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2024 |
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Cash, cash equivalents, short and long-term investments |
$ |
456,125 |
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$ |
692,404 |
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Total assets |
$ |
551,792 |
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$ |
724,816 |
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Total liabilities |
$ |
436,362 |
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$ |
436,692 |
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Total stockholders' equity |
$ |
115,430 |
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$ |
288,124 |
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Common stock outstanding |
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86,905,343 |
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85,694,443 |
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Common stock and common stock equivalents* |
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103,502,892 |
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98,972,323 |
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*Common stock and common stock equivalents: |
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Common stock |
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86,905,343 |
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85,694,443 |
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Common stock warrants (pre-funded) |
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285,714 |
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285,714 |
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Common stock and pre-funded stock warrants |
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87,191,057 |
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85,980,157 |
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Options to purchase common stock |
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13,506,676 |
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11,688,079 |
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Restricted Stock Units |
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2,805,159 |
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1,304,087 |
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Total common stock and common stock equivalents |
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103,502,892 |
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98,972,323 |
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SYNDAX PHARMACEUTICALS, INC. |
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(unaudited) |
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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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(In thousands, except share and per share data) |
2025 |
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2024 |
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2025 |
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2024 |
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Revenue |
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Product revenue, net |
$ |
32,007 |
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— |
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$ |
80,649 |
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— |
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Collaboration revenue, net |
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13,864 |
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— |
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22,975 |
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— |
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Milestone and license revenue |
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— |
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12,500 |
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— |
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16,000 |
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Total revenues |
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45,871 |
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12,500 |
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103,624 |
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16,000 |
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Operating expenses: |
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Cost of product sales |
$ |
2,100 |
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— |
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$ |
4,264 |
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— |
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Research and development |
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56,280 |
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70,971 |
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180,143 |
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176,118 |
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Selling, general and administrative |
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44,917 |
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31,106 |
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129,753 |
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83,189 |
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Total operating expenses |
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103,297 |
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102,077 |
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314,160 |
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259,307 |
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Loss from operations |
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(57,426 |
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(89,577 |
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(210,536 |
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(243,307 |
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Other (expense) income, net |
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(3,289 |
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5,451 |
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(6,872 |
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18,718 |
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Net loss |
$ |
(60,715 |
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$ |
(84,126 |
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$ |
(217,408 |
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$ |
(224,589 |
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Net loss attributable to common stockholders |
$ |
(60,715 |
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$ |
(84,126 |
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$ |
(217,408 |
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$ |
(224,589 |
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Net loss per share: |
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Basic loss per share attributable to common stockholders |
$ |
(0.70 |
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$ |
(0.98 |
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$ |
(2.51 |
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$ |
(2.63 |
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Diluted loss per share attributable to common stockholders |
$ |
(0.70 |
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$ |
(0.98 |
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$ |
(2.51 |
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$ |
(2.63 |
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Weighted-average common shares used in calculating: |
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Basic loss per share attributable to common stockholders |
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86,620,992 |
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85,433,569 |
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86,531,218 |
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85,307,660 |
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Diluted loss per share attributable to common stockholders |
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86,620,992 |
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85,433,569 |
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86,531,218 |
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85,307,660 |
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Third Quarter Financial Results Presentation / November 3, 2025
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," "anticipate" and similar expressions (as well as other words or expressions referencing future events, progress, timing or circumstances) are intended to identify forward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding future operations, financial results and the financial condition of Syndax Pharmaceuticals, Inc. (“Syndax” or the “Company”), including financial position, strategy and plans, the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax’s product candidates, the progress of regulatory submissions and approvals and subsequent commercialization and the potential use of Syndax’s product candidates to treat various cancer indications and fibrotic diseases, and Syndax’s expectations for liquidity and future operations, are forward-looking statements. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical site activation rates or clinical trial enrollment rates that are lower than expected; changes in expected or existing competition; the impact of macroeconomic conditions (the Russia-Ukraine war, inflation, among others) on Syndax’s business and that of the third parties on which Syndax depends, including delaying or otherwise disrupting Syndax’s clinical trials and preclinical studies, manufacturing and supply chain, or impairing employee productivity; failure of our collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Moreover, Syndax operates in a very competitive and rapidly changing environment. Other factors that may cause our actual results to differ from current expectations are discussed in Syndax’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein. New risks emerge from time to time. It is not possible for Syndax’s management to predict all risks, nor can Syndax assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this presentation may not occur and actual results could differ materially and adversely from those anticipated or implied. Except as required by law, neither Syndax nor any other person assumes responsibility for the accuracy and completeness of the forward- looking statements. Syndax undertakes no obligation to update publicly any forward-looking statements for any reason after the date of this presentation to conform these statements to actual results or to changes in Syndax’s expectations.
Strong 3Q25 commercial and portfolio execution position Syndax to reach profitability and be first to the frontline setting with a menin inhibitor Revuforj FDA-approved for second indication on Oct 24, 2025 Revuforj added to NCCN Guidelines® for R/R NPM1m AML on Sept 18, 2025 23 Revuforj & Niktimvo abstracts accepted for presentation at ASH 2025 Commercial Execution $32.0M net revenue $45.8M net revenue to INCY Portfolio Advancements On the road to profitability, with growing product contributions, a robust balance sheet, and stable expense outlook TRx, total prescriptions; q/q, quarter-over-quarter; R/R, relapsed/refractory $13.9M collaboration revenue to SNDX +25% TRx growth q/q
Strong 3Q25 Revuforj growth, even with a third of patients pausing Tx to proceed to stem cell transplant +12% q/q growth +25% q/q growth Cumulative since launch ~2,200 $32M 3Q25 3Q25 patient insights $88M ~850 Net revenue New patient starts ~750 ~250 TRx +25% q/q growth ~70% ~33% ~35-40% Revuforj is positioned for long-term growth with building usage observed post-HSCT and expansion into R/R NPM1m AML of use in KMT2A of KMT2A use in 2L/3L setting of KMT2A pts. proceed to HSCT of KMT2A pts. resume Revuforj post-HSCT ~90% Tx, treatment; q/q, quarter-over-quarter; 2L, second line; 3L, third line; HSCT, hematopoietic stem cell transplantation; pts, patients
Second Revuforj indication unlocks $2B U.S. market opportunity in R/R acute leukemia alone With the largest addressable population and anticipated duration of therapy, Revuforj is poised to become the largest targeted AML therapy R/R Acute Leukemia with KMT2A translocation R/R AML with NPM1m 1L “Unfit” AML with KMT2Ar or NPM1m 1L “Fit” AML with KMT2Ar or NPM1m Est. annual incidence ~2,000 ~4,500 ~3,500 ~5,500 U.S. Market Opportunity ($ B) $5B+ TAM $2B+ TAM Addressable AML population Revuforj FLT3 inhibitors IDH inhibitors NPM1 mutations and KMT2A translocations are routinely tested for, enabling efficient patient identification
Revuforj expansion into R/R NPM1m AML is well underway, leveraging solid foundation Unmatched efficacy data across multiple patient subtypes Strong prescriber base & HCP familiarity Excellent payer support & reimbursement Proven track record of delivering for patients Competitive advantages
Robust 3Q25 Niktimvo growth reflects rapid uptake across U.S. bone marrow transplant centers Cumulative since launch 8,500 $45.8M +27% q/q growth 3Q25 3Q25 insights $96M ~4,500 Net revenue to INCY New patient starts 1,100 ~400 Infusions administered 3L+ 90% of pts. that started in Q1 remain on Tx most usage in 4L; growing 3L use of U.S. bone marrow transplant centers have ordered 80% Collaboration revenue to SNDX $13.9M +48% q/q growth $23M Tx, treatment; q/q, quarter-over-quarter; 3L, third line; 4L, fourth line; pts, patients
Initial Niktimvo indication represents a $2B U.S. market opportunity, with substantial opportunities for label and geographic expansion cGVHD, chronic graft-versus-host disease; 1. Internal data on file; 2. SmartImmunology Insights cGVHD report March 2020; 3. SmartImmunology Insights IPF report March 2020. * IPF trial will be conducted and funded by Syndax. ~17,000 U.S. cGVHD patients1 ~6,500 currently treated 3L+ U.S. cGVHD patients ~35,000 cGVHD patients W.W.2 Idiopathic pulmonary fibrosis (IPF) patients3 ~150,000 U.S. | ~280,000 W.W. $2B TAM with initial Niktimvo indication Ongoing combo trials could support future expansion into newly diagnosed cGVHD Ph 2 IPF trial underway; evaluating other potential disease areas Ph 3 cGHVD trial underway outside the U.S. $5B+ TAM
First Revuforj real-world evidence highlights favorable tolerability and excellent activity across genetic subtypes and settings Early efficacy data (median follow up: 3.97 months) Patients treated for morphologic disease/relapse (n=14) ORR 79% (11/14) MRD negativity among responders by flow KMT2Ar 86% (6/7) NPM1m 67% (2/3) Proceeded to HSCT post-revumenib 29% (4/14) Received revumenib post-HSCT 3 pts (2 resumed post-HSCT, 1 started post-HSCT without prior rev Tx) Patients treated for NPM1m MRD positivity (n=2) Achieved MRD negativity 50% (1/2) ASH 2025 abstract #3448 Safety overview (n=18) No AEs led to revumenib discontinuation Low rate of revumenib dose reductions: 11% (2/18) DS in 11% (2/18) of pts (1 G2 & 1 G3) QTc prolongation: 23% (3/13) G3 and 31% (4/13) G1/G2 Demographics/characteristics (n=18) Age, median (range): 60 (23-79) Prior lines, median (range): 3 (0-6) Genetics, n (%) 9 (50%) KMT2Ar, 6 (33%) NPM1m, 3 (17%) NUP98r Setting of therapy, n (%) 15 (83%) R/R, 2 (11%) 1L, 1 (6%) post-HSCT w/out prior rev Rev usage, n (%) 14 (78%) in combination, 4 (22%) as monotherapy Abstract data cutoff: July 17, 2025. Notes: Two of the 18 patients were excluded from the efficacy analysis (no bone marrow evaluation post revumenib and use in post-HSCT maintenance setting only). The rate of QTc prolongation was reported among the subset of patients with regular EKG monitoring (n=13). 1L, frontline Real-world experience at Moffitt Cancer Center through July 2025
SAVE trial: High rates of CR & MRD negativity in newly diagnosed AML cohort treated with revumenib + venetoclax/oral HMA Demographics/characteristics in ND cohort (n=17) Age, median (range) 68 (60-83) NPM1m/KMT2Ar 11 (65%)/ 6 (35%) ELN22 risk Favorable 9 (53%) Intermediate 2 (12%) Adverse 6 (35%) ASH 2025 abstract #47 Abstract data cutoff: July 22, 2025. *Of the 17 patients, only 16 were evaluable for end of cycle 1. One patient was not yet evaluable for end of cycle 1 but continued on therapy with day 14 showing MLFS. HMA, Hypomethylating agent (decitabine/cedazuridine); AE, adverse event; DS, differentiation syndrome; MRD, minimal residual disease; HSCT, hematopoietic stem cell transplant Safety overview Most common AE was infection, occurring in 53% of pts (all G3) QTc prolongation in 8 (47%) pts (G1 or G2 only) DS in 4 (24%) pts (no events above G3) 94% Overall response rate (ORR) (16/17) Efficacy in ND cohort (n=17) 88% Complete remission (CR) (14/16*) 100% MRD negative CR by flow (14/14) At a median follow-up of 6 months, median overall survival and event free survival were not reached 29% Proceeded to HSCT (5/17)
Two Phase 1 trials of revumenib with 7+3 in newly diagnosed AML show high activity, tolerability, and rapid count recovery Demographics & baseline characteristics (n=12) Age, median (range) 55 (26-72) Genetics, n 6 high risk NPM1m, 6 KMT2Ar Efficacy evaluable pts at DL1 or DL2* (n=9) CR 89% (8/9) CR among KMT2Ar 100% (5/5) CR among high-risk NPM1m 75% (3/4) Proceeded to HSCT 44% (4/9) Time to full count recovery among pts with CR, median 25.5 days Safety overview Revumenib with 7+3 overall appears to be well-tolerated 9 pts completed induction DLT period: 1 DLT on DL2 (G5 typhlitis) 8 pts completed consolidation DLT period with no DLTs Preliminary results from Ph 1b NCI study ASH Abstract #5206 Preliminary results from Ph 1 Syndax study ASH Abstract #3425 Demographics & baseline characteristics (n=7) Age, median (range) 37 (27-56) Genetics, n 7 KMT2Ar Efficacy evaluable pts at DL1* (n=7) CR among KMT2Ar 100% (7/7) MRD negative CR 100% (6/61) Proceeded to HSCT 57% (4/7) Safety overview 6/7 pts. at DL1 were DLT evaluable; 1 DLT of G3 QTc prolongation (pt discontinued rev during cycle 1; pt achieved MRD(-) CR at end of cycle 1 and went to HSCT) Most common TEAEs were nausea and decreased neutrophils *DL1: Revumenib (rev) 110 or 220 mg q12hr with/without strong CYP3A4i with 7+3 induction and cytarabine consolidation; DL2: Revumenib 160 or 270 mg q12hr with/without strong CYP3A4i (approved monotherapy dose) with 7+3 induction and cytarabine consolidation; NCI: National Cancer Institute Abstract data cutoff: July 19, 2025; Full count recovery defined as ANC >1,000 and platelets >100,000) Abstract data cutoff: June 3, 2025; 1. All patients tested for MRD negativity achieved MRD-negative CR by local assessment (n=6)
Revumenib post-HSCT was well-tolerated with promising early efficacy in retrospective review of pediatric patients Safety overview (post-HSCT) Well-tolerated as post-HSCT maintenance in children; most common AE was thrombocytopenia (including two Gr 3 and one Gr 4, occurring primarily during first two cycles) At last follow-up (15.5 months median follow-up), all pts were alive with no relapses, yielding: 100% estimated 1-year EFS ASH 2025 abstract #3461 Abstract data: Patients who received a HSCT between November 2020 and January 2025. Demographics & baseline characteristics (n=10) Age, median (range) 10 yrs (1.4-18 yrs) Genetics, n (%) 8 (80%) KMT2Ar, 2 (20%) NUP98r Observations Prior to HSCT Cycles of rev, median (range) 2 (1-4) Rev usage, n (%) 5 (50%) combination, 5 (50%) monotherapy Post-HSCT Days post-HSCT rev reinitiated, median (range) 111 (58-175) Cycles of rev, median (range) 11 (1-25*) *Study planned for rev post-HSCT for up to 1 yr; 1 pt continued for 2 yrs due to parental preference
ASH abstracts highlight the potential for long-term axatilimab use in R/R cGVHD and feasibility of combining with ruxolitinib in newly diagnosed cGVHD 44 pts were enrolled across 3 arms (axa+rux, rux, or corticosteroids) at the interim analysis Axatilimab + ruxolitinib in newly diagnosed cGVHD patients was well tolerated with no evidence of additive toxicity Long-term treatment duration & safety from AGAVE-201 trial of axatilimab in R/R cGVHD Interim safety analysis from Ph 2 trial of axatilimab + ruxolitinib in newly diagnosed cGHVD Safety and feasibility of 0.6 mg/kg every 4-week axatilimab dosing in AGAVE-201 trial 33 pts from AGAVE-201 (N=239) were still on axa as of March 2025, with a median of 2.8 years on therapy (range 2.6–3.4 yrs) Long-term data show a continued tolerable safety profile with prolonged use 32% (19/59) of pts who had a response on axa 0.3 mg/kg Q2W (FDA-approved dose) transitioned to 0.6 mg/kg Q4W in AGAVE-201 Among the 19 pts who switched, the Q4W dosing was well tolerated, with a median of 1.7 years on therapy (range 0.2-2.7 yrs) after the dosing change ASH 2025 abstract #6010 ASH 2025 abstract #6012 ASH 2025 abstract #272
Strong financial position driven by growing Revuforj and Niktimvo contributions and stable expense outlook Key 3Q25 Financial Results (Unaudited) Three Months Ended Sept 30 ($ in millions) 2025 2024 Product revenue, net 32.0 - Collaboration revenue, net 13.9 - Milestone and license revenue - 12.5 Total revenues $45.9 $12.5 Cost of product sales (2.1) - Research & development (56.3) (71.0) Selling, general and administrative (44.9) (31.1) Total operating expenses ($103.3) ($102.1) Other (expense) income, net (3.3) 5.5 Net loss ($60.7) ($84.1) Totals may not sum due to rounding; 1. Includes short- and long-term investments; 2. Includes pre-funded warrants to purchase 285,714 common shares (rounded). Strong balance sheet, increasing contributions from Revuforj & Niktimvo, and a stable expense outlook expected to drive path to profitability AS OF SEPT 30, 2025: $456M in cash and equivalents1 87.2M shares outstanding2
15 Two first- & best-in-class drugs $5B+ TAM $5B+ TAM Two exceptional product launches Syndax is on the road to profitability with two medicines with multi-billion-dollar potential
Lilah, diagnosed with R/R AML FUELED BY A PASSION FOR PATIENTS
