Syndax (NASDAQ: SNDX) posts 224% Q1 revenue surge and lower loss
Rhea-AI Filing Summary
Syndax Pharmaceuticals reported first quarter 2026 revenue of $64.9 million, a 224% year-over-year increase driven by strong launches of Revuforj and Niktimvo. Revuforj net revenue reached $48.9 million, reflecting rapid uptake in relapsed or refractory NPM1-mutated and KMT2A-rearranged acute leukemias and growing use around stem cell transplant.
Niktimvo generated $55.1 million in net revenue at partner Incyte, producing $15.9 million of collaboration revenue for Syndax. Operating expenses declined modestly, and the net loss narrowed to $42.7 million, or $0.48 per share, compared with $84.8 million a year earlier.
The company ended the quarter with $352.1 million in cash, cash equivalents and short-term investments and 88.8 million common shares and prefunded warrants outstanding. Syndax guides to about $400 million in 2026 R&D plus SG&A (excluding $50 million non-cash stock compensation) and expects its current cash, investments, product and collaboration revenue, and interest income to support a path to profitability.
Positive
- Revenue surged 224% year over year in Q1 2026 to $64.9 million, driven by rapid uptake of Revuforj and Niktimvo.
- Net loss roughly halved to $42.7 million (from $84.8 million), reflecting higher revenue and slightly lower operating expenses.
- Strong launch metrics for Revuforj ($48.9M net revenue) and Niktimvo ($55.1M net revenue at Incyte, $15.9M collaboration revenue) support the commercial outlook.
- Robust liquidity with $352.1 million in cash, cash equivalents and short-term investments and guidance that current resources and expected revenues should enable a path to profitability.
Negative
- Despite strong growth, Syndax remains loss-making, with a Q1 2026 net loss of $42.7 million and significant ongoing R&D and SG&A spending.
- Royalty interest expense increased to $11.8 million, reducing operating leverage from higher product and collaboration revenue.
Insights
Explosive revenue growth from two launches, losses shrinking, cash still strong.
Syndax posted $64.9M in Q1 2026 revenue, up 224% year over year, powered by Revuforj and Niktimvo. Revuforj net revenue was $48.9M, while Niktimvo’s collaboration contribution was $15.9M, showing both products are scaling meaningfully.
Expenses remain high but trended down: R&D was $58.8M and SG&A $37.6M, each lower than in Q1 2025. Net loss improved to $42.7M from $84.8M, with basic and diluted loss per share at $0.48. Royalty interest expense of $11.8M is a notable ongoing drag.
Cash, cash equivalents and short-term investments of $352.1M as of March 31, 2026 provide runway alongside growing product and collaboration revenue. Management expects about $400M of 2026 R&D plus SG&A (excluding $50M stock comp) and indicates this cost base, combined with revenue growth, should enable a transition to profitability over time.
Commercial uptake supports an ambitious leukemia and fibrosis pipeline.
Revuforj’s $48.9M Q1 2026 net revenue and 144% growth over Q1 2025 reinforce leadership in menin inhibition for acute leukemia. The drug is being studied across the treatment continuum, including pivotal Phase 3 frontline trials like EVOLVE-2 and REVEAL-ND.
Niktimvo’s $55.1M in net revenue at Incyte and strong chronic GVHD demand underpin Syndax’s share of commercial profit. Multiple axatilimab trials target earlier-line chronic GVHD and idiopathic pulmonary fibrosis, with Phase 2 topline data in IPF and newly diagnosed chronic GVHD anticipated in 4Q 2026.
This combination of growing commercial revenue and late-stage studies in AML, GVHD and IPF provides several clinical and regulatory catalysts. Actual value creation will hinge on pivotal trial outcomes and sustained physician adoption, which upcoming data presentations and topline readouts throughout 2026–2028 will further clarify.
8-K Event Classification
Key Figures
Key Terms
menin inhibitor medical
collaboration revenue financial
chronic graft-versus-host disease medical
idiopathic pulmonary fibrosis medical
Orphan Drug Designation regulatory
Breakthrough Therapy Designation regulatory
Earnings Snapshot
For full-year 2026, Syndax expects approximately $400 million in combined R&D and SG&A expenses, excluding an estimated $50 million of non-cash stock compensation, and believes its cash, investments and anticipated revenues will enable it to reach profitability.
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:
Securities registered pursuant to Section 12(b) of the Act:
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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 April 30, 2026, Syndax Pharmaceuticals, Inc. (the “Company”) issued a press release and presentation announcing its financial results for the quarter and year ended March 31, 2026. 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 |
Press Release, dated April 30, 2026 |
99.2 |
Corporate Presentation, dated April 30, 2026 |
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: April 30, 2026
Exhibit 99.1
Syndax Reports First Quarter 2026 Financial Results and Provides Business Update
NEW YORK, NY, April 30, 2026 (GLOBE NEWSWIRE) – Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today reported its financial results for the first quarter ended March 31, 2026, and provided a business update.
“We delivered over $100 million in combined Revuforj and Niktimvo net sales in the first quarter, highlighting strong demand for our medicines and advancing the company towards profitability. Revuforj net revenue totaled $49 million, underscoring our leadership in menin inhibition and strong adoption in both R/R NPM1m AML and KMT2Ar acute leukemia. Notably, recent analysis indicates that Revuforj is enabling nearly half of KMT2A patients to receive a stem cell transplant, providing the best chance for durable remission and positioning the franchise for long-term growth as an increasing number of patients return to therapy post-transplant,” said Michael A. Metzger, Chief Executive Officer. “We are poised for continued commercial growth with robust prescriber bases, excellent payer coverage, and multiple evolving treatment patterns that should extend the average duration of treatment for both medicines.”
Mr. Metzger continued, “We are nearing multiple important catalysts this year, including new Revuforj data which will further highlight its best-in-class profile and topline data from Phase 2 trials of Niktimvo in frontline chronic GVHD and IPF. As we look ahead, we are focused on
unlocking the multi-billion-dollar opportunities for our medicines and are well-positioned to be first to frontline AML with a menin inhibitor, with strong global site initiation and patient enrollment underway in our pivotal trials.”
Recent Business Highlights and Anticipated Milestones
Revuforj® (revumenib)
New/updated data expected in the second quarter of 2026:
New/updated data expected in the second half of 2026:
Niktimvo™ (axatilimab-csfr)
First Quarter 2026 Financial Results
As of March 31, 2026, Syndax had cash, cash equivalents, and short-term investments of $352.1 million and 88.8 million common shares and prefunded warrants outstanding.
Total revenue for the first quarter of 2026 was $64.9 million, which consisted of $48.9 million in Revuforj net revenue and $15.9 million in Niktimvo collaboration revenue. The Niktimvo collaboration revenue is derived from the $55.1 million in Niktimvo net revenue that was previously reported by the Company's partner Incyte for the first quarter 2026. Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses.
First quarter 2026 research and development expenses decreased to $58.8 million from $61.6 million for the comparable prior year period. The year-over-year decrease was primarily due to a decrease in Niktimvo related development milestone expense recognized in the first quarter of 2025, offset by an increase in Revuforj related clinical trial and personnel expenses.
First quarter 2026 selling, general and administrative expenses decreased to $37.6 million from $41.0 million for the comparable prior year period. The year-over-year decrease was primarily due to a decrease in commercial-related expenses due to launch costs incurred in the first quarter of 2025 for Revuforj and Niktimvo that were not incurred in the same period in 2026 offset by a decrease in personnel expenses related to higher accrued compensation costs in 2025 for the achievement of corporate objectives.
For the three months ended March 31, 2026, Syndax reported a net loss attributable to common stockholders of $42.7 million, or $0.48 per share, compared to a net loss attributable to common stockholders of $84.8 million, or $0.98 per share, for the comparable prior year period.
Financial Guidance
For the full year of 2026, the Company expects total research and development plus selling, general and administrative expenses to be approximately $400 million, excluding the impact of $50 million in estimated non-cash stock compensation expense.
Syndax expects that its operating expense base will remain stable over the next couple of years. As a result, Syndax expects that its cash, cash equivalents and short-term investments, combined with its anticipated product revenue, collaboration 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, April 30, 2026.
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: Syndax1Q26
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: https://sndx-1q26.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.
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
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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March 31, |
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December 31, |
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(In thousands, except share data) |
2026 |
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2025 |
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Cash, cash equivalents and short -term investments |
$ |
352,064 |
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$ |
394,070 |
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Total assets |
$ |
472,668 |
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$ |
529,706 |
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Total liabilities |
$ |
431,046 |
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$ |
465,076 |
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Total stockholders' equity |
$ |
41,622 |
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$ |
64,630 |
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Common stock outstanding |
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88,543,881 |
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87,405,979 |
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Common stock and common stock equivalents* |
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106,213,646 |
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103,437,561 |
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*Common stock and common stock equivalents: |
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Common stock |
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88,543,881 |
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87,405,979 |
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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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88,829,595 |
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87,691,693 |
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Options to purchase common stock |
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13,949,602 |
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13,128,306 |
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Restricted Stock Units |
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3,434,449 |
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2,617,562 |
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Total common stock and common stock equivalents |
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106,213,646 |
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103,437,561 |
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SYNDAX PHARMACEUTICALS, INC. |
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(unaudited) |
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CONSOLIDATED STATEMENTS OF OPERATIONS |
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Three Months Ended March 31, |
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(In thousands, except share and per share data) |
2026 |
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2025 |
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Revenue: |
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Product revenue |
$ |
48,923 |
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$ |
20,042 |
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Collaboration revenue |
$ |
15,941 |
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$ |
- |
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Total revenue |
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64,864 |
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20,042 |
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Operating expenses: |
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Cost of product sales |
$ |
2,633 |
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$ |
885 |
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Research and development |
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58,845 |
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61,636 |
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Selling, general and administrative |
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37,588 |
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41,031 |
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Collaboration loss |
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- |
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247 |
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Total operating expenses |
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99,066 |
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103,799 |
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Loss from operations |
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(34,202 |
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(83,757 |
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Other (expense) income, net: |
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Royalty interest expense |
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(11,846 |
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(8,049 |
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Other interest expense |
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- |
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(2 |
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Interest income |
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3,561 |
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7,183 |
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Other (expense) income, net |
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(186 |
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(221 |
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Total other (expense) income, net |
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(8,471 |
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(1,089 |
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Net loss |
$ |
(42,673 |
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$ |
(84,846 |
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Net loss attributable to common stockholders |
$ |
(42,673 |
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$ |
(84,846 |
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Net loss per share: |
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Basic loss per share attributable to common stockholders |
$ |
(0.48 |
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$ |
(0.98 |
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Diluted loss per share attributable to common stockholders |
$ |
(0.48 |
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$ |
(0.98 |
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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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88,255,636 |
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86,171,889 |
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Diluted loss per share attributable to common stockholders |
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88,255,636 |
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86,171,889 |
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First Quarter 2026 Financial Results and Business Update April 30, 2026 Exhibit 99.2
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.
$49M Revuforj net revenue in 1Q26, highlighting leadership in menin inhibition $55M Niktimvo net revenue in 1Q26, resulting in $16M in collaboration revenue $65M in total revenue to Syndax in 1Q26, up 224% vs. 1Q25 Positioned to be 1st to frontline (1L) AML with a menin inhibitor Major presence planned at upcoming medical meetings with new real-world, 1L, R/R, and post-HSCT data expected in 2Q26 and 2H26 Topline data from Phase 2 trials of axatilimab in IPF and newly diagnosed cGVHD anticipated in 4Q26 Growing product revenues from two first- and best-in-class medicines Robust balance sheet and stable expense outlook On the road to profitability Strong 1Q26 results with multiple drivers for continued growth in 2026 and beyond 3 ROBUST COMMERCIAL DEMAND EXCELLENT PIPELINE PROGRESS STRONG FINANCIAL POSITION
1Q26 Revuforj results highlight leadership in menin inhibition and growing use in R/R NPM1m AML TRx, total prescriptions; HSCT, hematopoietic stem cell transplant; Tx, treatment EXCEPTIONAL LAUNCH >$180M net revenue ~1,380 patients treated Cumulative since launch in Nov ‘24: 1Q26 Revuforj results $48.9M net revenue ≥30% >1,300 TRx Growth in new patient starts driven by expanding uptake in R/R NPM1m AML of net revenue from NPM1 business 11% growth vs. 4Q25 ~13% growth vs. 4Q25, even with nearly 50% of KMT2A pts pausing Tx to proceed to HSCT New patients per quarter ~300 ~250 ~200 ~330 ~200 ≥40% of 1Q26 new patients were NPM1m
Revuforj is positioned for success with an outstanding commercial foundation All covered lives includes all commercially covered lives plus Medicare and Medicaid lives. Excellent payer coverage with no meaningful barriers to access of all covered lives with formulary coverage for both Revuforj indications 97% Robust and growing prescriber base with >85% of Tier 1+2 accounts activated Tier 1+2 account activation
Evolving clinical practice will drive continued Revuforj growth in 2026 1 2 Robust transplant rate in KMT2Ar and usage post-transplant Growing adoption in R/R NPM1m AML 3 patient population Tx durations Nearly 50% of KMT2A patients proceed to HSCT and ~45% have resumed Tx thus far R/R, relapsed/refractory; 2L, second line; 3L, third line; HSCT, hematopoietic stem cell transplantation; Tx, treatment Use in early lines of R/R treatment and in combinations Tx durations ~70% of use in 2L/3L ~40% of use in combination Average duration of therapy expected to extend as treatment patterns mature and an increasing number of patients return to therapy post-HSCT Significant room for further growth within a ~4,500 annual incident patient population
Current Revuforj indications unlock $2B+ TAM in R/R acute leukemia With the largest addressable population and anticipated duration of therapy, Revuforj is poised to become the largest targeted AML therapy R/R acute leukemia with KMT2A translocation R/R AML with NPM1m 1L “Unfit” AML with KMT2Ar or NPM1m 1L “Fit” AML with KMT2Ar or NPM1m ~2,000 ~4,500 ~3,500 ~5,500 U.S. Market Opportunity ($ B) $5B+ TAM across R/R and 1L $2B+ TAM in R/R Addressable AML population Revuforj FLT3 inhibitors IDH inhibitors Comprehensive clinical development program underway to unlock $5B+ TAM across acute leukemia Tx continuum Est. annual incidence
Building off an excellent first year, Niktimvo delivered strong 1Q26 results driven by robust demand ~300 1Q26 new patient starts ~5,000 1Q26 infusions administered Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses. $55.1M 1Q26 net revenue to INCY Compared to $13.6M in 1Q25, the first partial quarter of launch $15.9M 1Q26 collaboration revenue to SNDX Compared to -$0.2M in 1Q25, the first partial quarter of launch REMARKABLE LAUNCH $207M net revenue >1,700 patients treated Cumulative since launch in late Jan ‘25: Performance reflects strong, consistent new patient starts and solid persistency, offset by natural attrition among the large cohort of predominantly later-line patients who started in the first quarter of launch last year
Multiple drivers support continued Niktimvo growth in 2026 1 Continued adoption in 4L and growing usage in 3L cGVHD 2 Potential for extended treatment durations to address chronic disease Broad and growing prescriber base & strong commercial synergies 3 3L, third line; 4L, fourth line; BMT, bone marrow transplant centers; Tx, treatment ~32% share of 3L+ cGVHD market within 1 year of launch ~60-70% of pts who started in 1Q25 remained on Tx in 1Q26 Tx durations population and Tx durations Nearly every U.S. BMT center has ordered and become a repeat customer utilization 3
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 1L cGVHD Ph 2 IPF trial underway, the first of several potential areas for further pipeline expansion Ph 3 cGVHD trial underway outside the U.S. $5B+ TAM
Revumenib (select trials) Ph 1 Ph 2 Ph 3 FDA Approved Setting Study Name Regimen NPM1m KMT2Ar NUP98r R/R AUGMENT-101 Rev mono ● ● AUGMENT-102 Rev + IC ● ● ● SAVE Rev + ven/oral HMA ● ● ● Borate study Rev + gilt in FLT3 co-mutated ● ● ● Post-HSCT Maintenance Ball study Rev mono ● ● 1L Unfit for IC BEAT AML Rev + ven/aza ● ● SAVE Rev + ven/oral HMA ● ● ● EVOLVE-2 Rev + ven/aza ● ● Fit for IC 708 and NCI Rev + IC ● ● ● REVEAL-ND Rev + IC ● RAVEN Rev + ven/aza ● Focused on unlocking revumenib’s full potential Rev, revumenib; IC, intensive chemotherapy; 1L, frontline; R/R, relapsed or refractory; ven/aza, venetoclax and azacitidine; HMA, hypomethylating agent; HSCT, hematopoietic stem cell transplantation; RWE, real-world evidence; gilt, gilteritinib Global enrollment underway in pivotal 1L trials Positioned to be 1st to the 1L with a menin inhibitor Strong presence planned at ASCO, EHA, and ASH and other key meetings ANTICIPATED UPCOMING DATA Additional real-world evidence in 2Q26 New post-HSCT maintenance data in 2Q26 Updated Ph 1 frontline rev + IC data in 2Q26 Updated SAVE R/R and NUP98r R/R data in 2Q26 Updated BEAT AML frontline data in 2H26 Updated Ph 1 R/R rev + gilt data in 2H26
Axatilimab (select trials) Ph 1 Ph 2 Ph 3 FDA Approved Setting Study Name Regimen R/R cGVHD AGAVE-201 Axatilimab (axa) monotherapy 1L cGVHD AXemplify-357* Axatilimab + corticosteroids NCT06388564* Axatilimab + ruxolitinib IPF MAXPIRe Axatilimab on top of SOC Robust clinical development plan underway to unlock the potential for axatilimab in 1L cGVHD, IPF, and beyond *Trials led by Incyte. List is not inclusive of ongoing ex-U.S. trials. ANTICIPATED UPCOMING DATA Topline Ph 2 MAXPIRe IPF data in 4Q26 Topline Ph 2 axa + rux data now in 4Q26 Topline Ph 3 axa + steroids data in early 2028 Near-term Phase 2 data readouts in IPF and newly diagnosed cGVHD could open transformative opportunities
A growing body of evidence points to CSF1-dependent monocyte-derived alveolar macrophages as a promising new target in IPF Key discoveries: Monocyte-derived alveolar macrophages drive lung fibrosis Colony stimulating factor-1 receptor (CSF1R) signaling is a key regulator of monocytes and macrophages Multiple studies implicate the CSF1R pathway in IPF: Higher CSF1R levels observed in IPF patients vs. healthy controls Higher CSF1R levels predict shorter survival in IPF patients Higher monocyte levels predict shorter survival in IPF patients Misharin J Exp Med 2017; Byrne Trends Mol Med 2016; Joshi Eur Respir J 2020; Scott Respir Med 2019; Oldham ERJ Open Res 2023; Michalaki ERS 2024.
Development in IPF supported by lung responses observed in cGVHD Axatilimab is a CSF1R-blocking antibody targeting monocyte-derived alveolar macrophages in IPF Blocking CSF-1R with axatilimab: Reduces levels of circulating profibrotic and proinflammatory monocytes and monocyte-derived macrophages Inhibits the activity of pathogenic macrophages in tissues Among cGVHD pts with lung involvement who received axa 0.3 mg/kg Q2W, nearly 50% achieved a lung response and >90% reported improvements in shortness of breath at rest Lung responses were observed across all subgroups, including patients with difficult-to-treat, severe disease Salhotra et al., oral Presentation at ATS 2025; Salhotra et al., poster presentation at ERS 2024.
Topline data from MAXPIRe Phase 2 trial of axatilimab in IPF anticipated in 4Q26 A randomized, double-blind, placebo-controlled, multi-center international trial Key eligibility criteria: ≥40 yrs of age HRCT confirming IPF diagnosis FVC ≥45% of predicted normal (PN) FEV1/FVC ≥0.7 DLCO ≥30% and ≤90% PN Stable background use of pirfenidone or nintedanib allowed (N≈135) Placebo Q2W (n≈45) ClinicalTrials.gov ID: NCT06132256; FEV1, forced expiratory volume in 1 second; FVC, forced vital capacity; SGRQ, St. George's Respiratory Questionnaire; DLCO, diffusion capacity for carbon monoxide; HRCT, high-resolution computed tomography PRIMARY ENDPOINT: Annualized rate of decline in FVC over 26 weeks (ml) Axatilimab 0.3 mg/kg Q2W (n≈90) Randomized 2:1 to axatilimab or placebo; stratified by background antifibrotic therapy (pirfenidone, nintedanib, or none) SECONDARY ENDPOINTS: Disease progression, SGRQ (quality of life measures), change in FVC % predicted, DLCO 26-week treatment
Strong financial position driven by growing revenue and stable expense outlook Financial Summary ($ in millions) Three Months Ended March 31 2026 2025 Product revenue, net 48.9 20.0 Collaboration revenue, net 15.9 — Total revenues 64.9 20.0 Cost of product sales (2.6) (0.9) Research & development (R&D) (58.8) (61.6) Selling, general and administrative (SG&A) (37.6) (41.0) Total operating expenses (99.1) (103.8)* Other (expense) income, net (8.5) (1.1) Net loss (42.7) (84.8) Totals may not sum due to rounding; 1. Includes short-term investments; 2. Includes pre-funded warrants to purchase 285,714 common shares (rounded). *Total operating expenses includes $0.2M in collaboration loss associated with a Niktimvo net commercial loss in 1Q25. AS OF 31 MAR 2026: $352.1M in cash and equivalents1 88.8M shares outstanding2 2026 R&D + SG&A EXPENSE GUIDANCE: $400M, excluding $50M in expected stock option expense On the road to profitability
Continued focus on driving revenue growth and pipeline progress, with another data-rich year ahead 2026 Anticipated Milestones 2025 Key Accomplishments Advance global enrollment in pivotal 1L trials of revumenib Advance leadership in menin inhibition with new 1L, maintenance, R/R, and real-world evidence for revumenib Report topline Ph 2 axatilimab data in IPF and newly diagnosed cGVHD in 4Q26 Initiate RAVEN 1L trial of revumenib in fit KMT2Ar in 2H26 Initiate a program to generate proof-of-principle clinical data with revumenib in myelofibrosis Executed two strong product launches Expanded Revuforj into 2nd indication Initiated 1st pivotal 1L trial of a menin inhibitor Presented first RWE for a menin inhibitor Initiated managed access program, expanding access to Revuforj in certain OUS regions 1L, frontline; RWE, real-world evidence; OUS, outside the United States
