# Syndax Pharmaceuticals Inc (SNDX)

Informational only - not investment advice.

CIK: 0001395937
SIC: 2834 Pharmaceutical Preparations
SIC breadcrumb: [Manufacturing](/division/D/) > [Chemicals And Allied Products](/major-group/28/) > [SIC 2834 Pharmaceutical Preparations](/industry/2834/)
Latest 10-K filed: 2026-02-26
SEC page: https://www.sec.gov/edgar/browse/?CIK=1395937
Filing source: https://www.sec.gov/Archives/edgar/data/1395937/000119312526076536/sndx-20251231.htm

## Selected Fundamentals
| Metric | Value | Unit | FY | Filed |
| --- | ---: | --- | ---: | --- |
| Revenue | 172352000 | USD | 2025 | 2026-02-26 |
| Net income | -285422000 | USD | 2025 | 2026-02-26 |
| Assets | 529706000 | USD | 2025 | 2026-02-26 |

## Financials

Annual standardized facts from SEC companyfacts as of latest extracted filing date 2026-02-26. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001395937.json. Derived margins, ratios, and free cash flow are computed from the extracted annual SEC facts.

| Metric | 2015 | 2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
| --- | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: |
| Revenue |  | 1,220,000 | 2,108,000 | 1,517,000 | 1,517,000 | 1,517,000 | 139,709,000 |  |  | 23,680,000 | 172,352,000 |
| Net income |  | -44,472,000 | -60,802,000 | -73,961,000 | -56,047,000 | -73,158,000 | 24,926,000 | -149,338,000 | -209,360,000 | -318,758,000 | -285,422,000 |
| Operating income |  | -43,766,000 | -61,954,000 | -75,876,000 | -57,539,000 | -71,423,000 | 26,220,000 | -151,757,000 | -229,954,000 | -339,672,000 | -273,084,000 |
| Diluted EPS |  |  |  |  | -1.84 | -1.87 | 0.46 | -2.46 | -2.98 | -3.72 | -3.29 |
| Operating cash flow |  | -35,157,000 | -47,371,000 | -68,531,000 | -50,612,000 | -71,260,000 | 29,131,000 | -133,675,000 | -160,601,000 | -274,903,000 | -322,980,000 |
| Capital expenditures | 49,000 | 261,000 | 84,000 | 187,000 |  | 0.00 | 129,000 | 0.00 | 0.00 | 0.00 | 187,000 |
| Assets |  | 109,013,000 | 137,186,000 | 83,938,000 | 63,525,000 | 300,613,000 | 449,657,000 | 497,236,000 | 612,880,000 | 724,816,000 | 529,706,000 |
| Liabilities |  | 24,874,000 | 32,867,000 | 30,891,000 | 31,925,000 | 48,425,000 | 41,289,000 | 29,787,000 | 58,684,000 | 436,692,000 | 465,076,000 |
| Stockholders' equity |  | 84,139,000 | 104,319,000 | 53,047,000 | 31,600,000 | 252,188,000 | 408,368,000 | 467,449,000 | 554,196,000 | 288,124,000 | 64,630,000 |
| Cash and cash equivalents |  | 23,844,000 | 35,168,000 | 33,769,000 | 24,609,000 | 115,243,000 | 221,965,000 | 74,356,000 | 295,394,000 | 154,083,000 | 134,930,000 |
| Free cash flow |  | -35,418,000 | -47,455,000 | -68,718,000 |  | -71,260,000 | 29,002,000 | -133,675,000 | -160,601,000 | -274,903,000 | -323,167,000 |

### Ratios

ROE and ROA use period-end equity/assets. Liabilities / equity uses total liabilities divided by stockholders' equity. Current ratio uses current assets divided by current liabilities when both are reported.

| Metric | 2015 | 2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
| --- | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: |
| Net margin |  |  |  |  |  |  | 17.84% |  |  |  |  |
| Operating margin |  |  |  |  |  |  | 18.77% |  |  |  |  |
| Return on equity |  | -52.86% | -58.28% | -139.43% | -177.36% | -29.01% | 6.10% | -31.95% | -37.78% | -110.63% | -441.62% |
| Return on assets |  | -40.80% | -44.32% | -88.11% | -88.23% | -24.34% | 5.54% | -30.03% | -34.16% | -43.98% | -53.88% |
| Liabilities / equity |  | 0.30 | 0.32 | 0.58 | 1.01 | 0.19 | 0.10 | 0.06 | 0.11 | 1.52 | 7.20 |
| Current ratio |  | 10.47 | 8.45 | 5.18 | 3.39 | 15.84 | 21.68 | 16.85 | 10.00 | 5.82 | 4.40 |

## Quarterly

Quarterly standardized facts from SEC companyfacts as of latest extracted filing date 2026-04-30. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001395937.json.

Flow metrics use discrete quarter-length periods from 10-Q/10-Q/A filings. Q4 revenue and net income are derived only when annual FY and nine-month YTD facts exist for the same fiscal year; derived Q4 values are labeled. EPS Q4 is not derived.

| Quarter | End date | Revenue | Net income | Diluted EPS | Method |
| --- | --- | ---: | ---: | ---: | --- |
| 2021-Q1 | 2021-03-31 | 379,000 |  |  | reported discrete quarter |
| 2021-Q2 | 2021-06-30 | 379,000 |  |  | reported discrete quarter |
| 2021-Q3 | 2021-09-30 | 12,375,000 |  |  | reported discrete quarter |
| 2021-Q4 | 2021-12-31 | 126,576,000 |  |  | derived Q4 = FY annual - nine-month YTD |
| 2022-Q2 | 2022-06-30 |  |  | -0.62 | reported discrete quarter |
| 2022-Q3 | 2022-09-30 |  |  | -0.58 | reported discrete quarter |
| 2023-Q1 | 2023-03-31 |  |  | -0.59 | reported discrete quarter |
| 2023-Q2 | 2023-03-31 |  | -41,126,000 |  | reported discrete quarter |
| 2023-Q2 | 2023-06-30 |  |  | -0.64 | reported discrete quarter |
| 2023-Q3 | 2023-06-30 |  | -44,615,000 |  | reported discrete quarter |
| 2023-Q3 | 2023-09-30 |  |  | -0.73 | reported discrete quarter |
| 2023-Q4 | 2023-12-31 |  | -72,473,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2024-Q1 | 2024-03-31 |  | -72,400,000 | -0.85 | reported discrete quarter |
| 2024-Q2 | 2024-03-31 |  | -72,400,000 |  | reported discrete quarter |
| 2024-Q2 | 2024-06-30 | 3,500,000 |  | -0.80 | reported discrete quarter |
| 2024-Q3 | 2024-06-30 |  | -68,063,000 |  | reported discrete quarter |
| 2024-Q3 | 2024-09-30 | 12,500,000 |  | -0.98 | reported discrete quarter |
| 2024-Q4 | 2024-12-31 | 7,680,000 | -94,169,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2025-Q1 | 2025-03-31 | 20,042,000 | -84,846,000 | -0.98 | reported discrete quarter |
| 2025-Q2 | 2025-03-31 |  | -84,846,000 |  | reported discrete quarter |
| 2025-Q2 | 2025-06-30 | 37,958,000 |  | -0.83 | reported discrete quarter |
| 2025-Q3 | 2025-06-30 |  | -71,847,000 |  | reported discrete quarter |
| 2025-Q3 | 2025-09-30 | 45,871,000 |  | -0.70 | reported discrete quarter |
| 2025-Q4 | 2025-12-31 | 68,728,000 | -68,014,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2026-Q1 | 2026-03-31 | 64,864,000 | -42,673,000 | -0.48 | reported discrete quarter |

## Macro Cross-References
- [CPIAUCSL](/indicator/CPIAUCSL/): Consumer Price Index for All Urban Consumers: All Items in U.S. City Average
- [UNRATE](/indicator/UNRATE/): Unemployment Rate
- [FEDFUNDS](/indicator/FEDFUNDS/): Federal Funds Effective Rate
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- [DFEDTARU](/indicator/DFEDTARU/): Federal Funds Target Range - Upper Limit
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- [DSPIC96](/indicator/DSPIC96/): Real Disposable Personal Income
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- [TTLCONS](/indicator/TTLCONS/): Total Construction Spending: Total Construction in the United States
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- [TOTALSL](/indicator/TOTALSL/): Total Consumer Credit Owned and Securitized
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- [DRCCLACBS](/indicator/DRCCLACBS/): Delinquency Rate on Credit Card Loans, All Commercial Banks
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- [NETEXP](/indicator/NETEXP/): Net Exports of Goods and Services
- [GFDEBTN](/indicator/GFDEBTN/): Federal Debt: Total Public Debt
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- [FYFSD](/indicator/FYFSD/): Federal Surplus or Deficit
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- [MANEMP](/indicator/MANEMP/): All Employees, Manufacturing
- [USCONS](/indicator/USCONS/): All Employees, Construction
- [USTRADE](/indicator/USTRADE/): All Employees, Retail Trade
- [USFIRE](/indicator/USFIRE/): All Employees, Financial Activities
- [USGOVT](/indicator/USGOVT/): All Employees, Government
- [AWHAETP](/indicator/AWHAETP/): Average Weekly Hours of All Employees, Total Private
- [DGORDER](/indicator/DGORDER/): Manufacturers' New Orders: Durable Goods
- [NEWORDER](/indicator/NEWORDER/): Manufacturers' New Orders: Nondefense Capital Goods Excluding Aircraft
- [BUSINV](/indicator/BUSINV/): Total Business Inventories
- [EXPGS](/indicator/EXPGS/): Exports of Goods and Services
- [IMPGS](/indicator/IMPGS/): Imports of Goods and Services
- [IR](/indicator/IR/): Import Price Index (End Use): All Commodities
- [PPIFIS](/indicator/PPIFIS/): Producer Price Index by Commodity: Final Demand

## Latest quarter (10-Q)

Latest 10-Q source: https://www.sec.gov/Archives/edgar/data/1395937/000119312526197154/sndx-20260331.htm

Extracted structurally from real Item 2 body heading to real Item 3/4 boundary. Published MD&A gate trimmed front/tail over-capture.
Confidence: high
Filing date: 2026-04-30
Report date: 2026-03-31

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following information should be read in conjunction with the unaudited financial information and the notes thereto included in this Quarterly Report on Form 10-Q, or the Quarterly Report, and the audited financial information and the notes thereto included in our Annual Report on Form 10-K that was filed with the Securities and Exchange Commission, or SEC, on February 26, 2026.

Company Overview

We are a commercial-stage biopharmaceutical company advancing innovative cancer therapies. We currently have two commercially approved medicines, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and a robust slate of clinical development programs.

Revuforj is our first-in-class menin inhibitor that was approved by the U.S. Food and Drug Administration, or FDA, in November 2024 for the treatment of relapsed or refractory, or R/R, acute leukemia with a lysine methyltransferase 2A gene, or KMT2A, translocation in adult and pediatric patients one year and older. In October 2025, Revuforj received a second approval from the FDA for the treatment of R/R acute myeloid leukemia, or AML, with a susceptible nucleophosmin 1 mutation, or NPM1m, in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. We are also studying revumenib in combination with standard-of-care agents in NPM1m AML or KMT2A-rearranged, or KMT2Ar, acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring the potential for menin inhibition in the treatment of myelofibrosis, or MF.

Niktimvo is our first-in-class colony stimulating factor-1 receptor, or CSF-1R, blocking antibody that was approved by the FDA in August 2024 for the treatment of chronic graft-versus-host disease, or cGVHD, after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Axatilimab is in development for the treatment of newly diagnosed cGVHD patients in combination with standard-of-care therapies, and for the treatment of idiopathic pulmonary fibrosis, or IPF.

We licensed the global rights to revumenib and axatilimab, the first two FDA approved medicines to emerge from our pipeline. We are leading the commercialization and further development of revumenib and working closely with our collaboration partner, Incyte, on the commercialization and further development of axatilimab. We plan to continue to leverage the technical and business expertise of our management team and scientific collaborators to license, acquire and develop additional therapeutics to expand our pipeline.

We have incurred significant operating losses since our inception. While we generate product revenue from sales of Revuforj and collaboration as well as milestone revenue from sales of Niktimvo, we continue to incur significant research and development and other expenses related to our ongoing operations. Except for 2021, we have not been profitable and have incurred losses in each period since our inception in 2005. For the three months ended March 31, 2026 and 2025, we reported a net loss of $42.7 million and $84.8 million, respectively. As of March 31, 2026, we had an accumulated deficit of $1.5 billion. As of March 31, 2026, we had cash, cash equivalents and short-term investments of $352.1 million

Recent Business Highlights and Anticipated Milestones

Revuforj®(revumenib)

•
Achieved $48.9 million in Revuforj net revenue in the first quarter of 2026, representing a 144% increase over the first quarter of 2025 and an 11% increase over the fourth quarter of 2025. Total prescriptions increased by approximately 160% compared to the first quarter of 2025 and approximately 13% compared to the fourth quarter of 2025. Notably, recent analysis indicates that nearly half of KMT2A patients are proceeding to a hematopoietic stem cell transplant, or HSCT, after receiving Revuforj, a significant increase from prior estimates of 33% of KMT2A patients. We expect this growing transplant rate to extend the average treatment duration as an increasing number of patients return to therapy after transplant.

•
We expect the presentation of new revumenib data from multiple ongoing studies at major medical meetings throughout 2026.

New/updated data expected in the second quarter of 2026:

•
Findings from a multicenter real-world study.

•
Post-HSCT maintenance data from multiple trials and centers.

•
R/R NUP98-rearranged, or NUP98r, acute leukemia data from patients treated in the AUGMENT-101 trial or via an expanded access program.

15

•
R/R data from the SAVE trial of revumenib in combination with venetoclax and decitabine/cedazuridine in NPM1m, KMT2Ar, and NUP98r acute leukemia.

•
Frontline data from the Phase 1 trial of revumenib in combination with intensive chemotherapy in NPM1m, KMT2Ar, or NUP98r AML.

New/updated data expected in the second half of 2026:

•
Frontline data from the BEAT AML trial of revumenib in combination with venetoclax/azacitidine in NPM1m and KMT2Ar AML.

•
R/R data from the Phase 1 trial of revumenib in combination with gilteritinib in AML patients with a FLT3 mutation and a KMT2A translocation, NPM1m, or any other mutation associated with HOX-MEIS1 overexpression.

•
Multiple clinical trials evaluating revumenib across the acute leukemia treatment continuum are ongoing, such as:

•
EVOLVE-2: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed NPM1m (primary efficacy analysis population) and KMT2Ar AML patients who are unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.

•
REVEAL-ND: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1m AML patients.

•
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with newly diagnosed and R/R AML or mixed-lineage acute leukemia harboring either NPM1m, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center.

•
Intensive chemotherapy: Two ongoing Phase 1 trials evaluating the combination of revumenib with intensive chemotherapy (7+3) in newly diagnosed NPM1m or KMT2Ar acute leukemia patients.

•
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed older adults (≥60 years) with NPM1m or KMT2Ar AML. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial.

•
Post-transplant maintenance: A Phase 1 trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after HSCT in patients with KMT2Ar or NPM1m acute leukemia. The trial is being conducted by investigators from the City of Hope Medical Center.

•
Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate measurable residual disease, or MRD, in patients with AML and extend progression-free survival. The trial is being conducted by Break ThroughCancer, a collaboration between leading U.S. cancer research centers.

•
INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial.

•
We expect the RAVEN trial to initiate in the second half of 2026. RAVEN is a Phase 2 collaborative trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed KMT2Ar patients who would be considered eligible, or fit, for intensive chemotherapy.

Niktimvo™ (axatilimab-csfr)

•
Achieved $55.1 million in Niktimvo net revenue in the first quarter of 2026, representing significant growth compared to the $13.6 million in net revenue generated in the first quarter of 2025 from the first two months of the launch. Syndax records 50% of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $15.9 million in the first quarter of 2026.

16

•
Presented data from nine axatilimab abstracts, including one oral presentation, at the Tandem Meetings (Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR®) in February 2026. The data presented included a comprehensive analysis of axatilimab in patients with chronic GVHD-related bronchiolitis obliterans syndrome in two clinical studies. The results show clinical and symptom responses across a spectrum of lung involvement.

•
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:

•
A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD. Topline data is now anticipated in the fourth quarter of 2026

•
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multicenter trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD. Topline data is anticipated in early 2028.

•
Completed enrollment in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with IPF in the first quarter of 2026. We expect to report topline data in the fourth quarter of 2026.

Financial Operations Overview

Product Revenue, net

Our FDA-approved product, Revuforj, was approved by the FDA for commercial sale in the U.S. on November 15, 2024. In accordance with GAAP, we determine net product revenue for Revuforj, with specific assumptions for variable consideration components including, but not limited to, trade discounts and allowances, co-pay assistance programs and payor rebates. We record product revenue net of estimated discounts, chargebacks, rebates, product returns, and other gross-to-net revenue deductions.

Collaboration revenue, net

In September 2021, we entered into the Incyte License and Collaboration Agreement, or the Incyte License, with Incyte covering the worldwide development and commercialization of axatilimab. In August 2024, the FDA approved Niktimvo for the treatment of cGVHD 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 accordance with Topic ASC 808, Collaboration Arrangements, Incyte has been identified as the principal in product sales, therefore, we will recognize its 50% share of any profits or losses in the amount of net product sales less cost of goods sold and shared commercial and other expenses, in the period in which the underlying sales and costs are recognized. Our share of net profits in connection with commercialization of Niktimvo will be presented as “Collaboration revenue, net” and our share of net losses will be presented as “Collaboration loss” within operating expenses. Collaboration revenue or expense is made up of our share of the 50% profit with Incyte. We record collaboration revenue net of commercial expenses, including any royalties owed on license agreements. We will continue to recognize the costs associated with ongoing development services in the R&D operating expense line, including any cost-sharing components with Incyte.

Cost of Product Sales

Our cost of product sales includes the cost of goods sold for Revuforj and license agreement royalties associated

[Excerpt truncated for page length; source filing is linked above.]

## Latest 10-K MD&A

Extracted structurally from real Item 7 body heading to real Item 7A/8 boundary.
Confidence: high

Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis should be read in conjunction with our consolidated financial statements and related notes included elsewhere in this Annual Report. This discussion and analysis and other parts of this Annual Report contain forward-looking statements based upon current beliefs, plans and expectations that involve risks, uncertainties and assumptions, such as statements regarding our plans, objectives, expectations, intentions and projections. Our actual results and the timing of selected events could differ materially from those anticipated in these forward-looking statements as a result of several factors, including those set forth under “Risk Factors” and elsewhere in this Annual Report. You should carefully read the “Risk Factors” section of this Annual Report to gain an understanding of the important factors that could cause actual results to differ materially from our forward-looking statements. Please also see the section entitled “Special Note Regarding Forward-Looking Statements.”

For the discussion of the financial condition and results of operations for the year ended December 31, 2024 compared to the year ended December 31, 2023, refer to "Management's Discussion and Analysis of Financial Condition and Results of Operations—Results of Operations" and "—Liquidity and Capital Resources" included in the Annual Report on Form 10-K filed with the SEC on March 3, 2025.

Overview

We are a commercial-stage biopharmaceutical company advancing innovative cancer therapies. We currently have two commercially approved medicines, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and a robust slate of clinical development programs designed to unlock the full potential of our first two products.

Revuforj is our first-in-class menin inhibitor that was approved by the U.S. Food and Drug Administration, or FDA, in November 2024 for the treatment of relapsed or refractory, or R/R, acute leukemia with a lysine methyltransferase 2A gene, or KMT2A, translocation in adult and pediatric patients one year old and older. In October 2025, Revuforj received a second approval from the FDA for the treatment of R/R acute myeloid leukemia, or AML, with a susceptible nucleophosmin 1 mutation, or NPM1m, in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. We are also studying revumenib in combination with standard-of-care agents in NPM1m AML and KMT2A-rearranged, or KMT2Ar, acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring the potential for menin inhibition in the treatment of myelofibrosis, or MF.

Niktimvo is our first-in-class colony stimulating factor-1 receptor, or CSF-1R, blocking antibody that was approved by the FDA in August 2024 for the treatment of chronic graft-versus-host disease, or cGVHD, after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Axatilimab is in development for the treatment of newly diagnosed cGVHD patients in combination with standard of care therapies, and for the treatment of idiopathic pulmonary fibrosis, or IPF.

We licensed the global rights to revumenib and axatilimab, the first two FDA approved medicines to emerge from our pipeline. We are leading the commercialization and further development of revumenib and working closely with our collaboration partner, Incyte, on the commercialization and further development of axatilimab. We plan to continue to leverage the technical and business expertise of our management team and scientific collaborators to license, acquire and develop additional therapeutics to expand our pipeline.

We have incurred significant operating losses since our inception. While we generate product revenue from sales of Revuforj and collaboration as well as milestone revenue from sales of Niktimvo, we continue to incur significant research and development and other expenses related to our ongoing operations. Except for 2021, we have not been profitable and have incurred losses in each period since our inception in 2005. For the years ended December 31, 2025, 2024, and 2023, we reported a net loss of $285.4 million and $318.8 million, and $209.4 million, respectively. As of December 31, 2025, we had an accumulated deficit of $1.5 billion, which included non-cash charges for stock-based compensation, preferred stock accretion and extinguishment charges. As of December 31, 2025, we had cash, cash equivalents and short-term investments of $394.1 million.

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Significant Risks and Uncertainties

Ongoing high interest rates could make it more difficult for us to obtain traditional financing on acceptable terms, if at all. Additionally, the ongoing recession risk together with the foregoing, could result in further economic uncertainty and volatility in the capital markets in the near term and, as a result could negatively affect our operations. Furthermore, such economic conditions have produced downward pressure on share prices. Although we do not believe that inflation has had a material impact on our financial position or results of operations to date, the return of a high inflationary environment could increase our operating costs, including our labor costs and research and development costs. These costs may also be negatively impacted due to supply chain constraints, global geopolitical tensions, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.

Additionally, we are subject to other challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of our late-stage product candidate; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements.

Financial Overview

Net Product Revenue

Our second FDA-approved product, Revuforj, was approved by the FDA for commercial sale in the U.S. on November 15, 2024. Net product revenue from sales of Revuforj was $124.8 million and $7.7 million for the twelve months ended December 31, 2025 and 2024 respectively. In accordance with GAAP, we determine net product revenue for Revuforj, with specific assumptions for variable consideration components including, but not limited to, trade discounts and allowances, co-pay assistance programs and payor rebates. We record product revenue net of estimated discounts, chargebacks, rebates, product returns, and other gross-to-net revenue deductions.

We generated no net product revenue during the year ended December 31, 2023.

Collaboration revenue, net

In September 2021, we entered into the Incyte License and Collaboration Agreement, or the Incyte License, with Incyte covering the worldwide development and commercialization of axatilimab. In August 2024, the FDA approved Niktimvo for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Niktimvo sales began in February 2025.

In accordance with Topic ASC 808, Collaboration Arrangements, Incyte has been identified as the principal in product sales, therefore, we will recognize its 50% share of any profits or losses in the amount of net product sales less cost of goods sold and shared commercial and other expenses, including any royalties owed on license agreements, in the period in which the underlying sales and costs are recognized. Our share of net profits in connection with commercialization of Niktimvo will be presented as “Collaboration revenue, net” and our share of net losses will be presented as “Collaboration loss” within operating expenses. We will continue to recognize the costs associated with ongoing development services in the R&D operating expense line, including any cost-sharing components with Incyte.

Milestone, License, and Royalty Revenue

We enter into license agreements for the development and commercialization of our product candidates. License agreements may include non-refundable upfront payments, contingent milestone and/or royalty payments based on the occurrence of specified events under our license arrangements, partial or complete reimbursement of research and development expenses, license fees and royalties on sales if they are successfully approved and commercialized. Our performance obligations under the license agreements may include the transfer of intellectual property rights in the form of licenses, obligations to provide research and development services and related materials and participation on certain development and/or commercialization committees.

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Revenue is recognized when, or as, performance obligations are satisfied, which occurs when control of the promised products or services is transferred to customers. Revenue is measured as the amount of consideration we expect to receive in exchange for transferring products or services to a customer, or the transaction price. To the extent that the transaction price includes variable consideration, we estimate the amount of variable consideration that should be included in the transaction price utilizing the most likely amount method. Variable consideration is included in the transaction price if, in our judgment, it is probable that a significant future reversal of cumulative revenue under the contract will not occur. Estimates of variable consideration and determination of whether to include estimated amounts in the transaction price are based largely on an assessment of our anticipated performance and all information (historical, current and forecasted) that is reasonably available.

We assessed the promises to determine if they are distinct performance obligations. Once the performance obligations are determined, the transaction price is allocated based on a relative standalone selling price basis. Milestone payments and royalties are typically considered variable consideration at the outset of the contract and are recognized in the transaction price either upon occurrence or when the constraint of a probable reversal is no longer applicable.

Cost of Product Sales

Our cost of product sales includes the cost of goods sold for Revuforj and license agreement royalties associated with its sales in the United States. Until we received regulatory approval for Revuforj in the United States in November 2024, we recorded expenses incurred for the manufacturing of pre-launch inventory that would support a U.S. launch as research and development expense. Cost of goods sold for a Revuforj may not include the full cost of manufacturing until the initial pre-launch, and previously expensed, inventory is depleted.

Research and Development

Since our inception, we have primarily focused on our clinical development programs. Research and development expenses consist primarily of costs incurred for the development of our product candidates and include:

•
expenses incurred under agreements related to our clinical trials, including the costs for investigative sites and contract research organizations, or CROs, that conduct our clinical trials;

•
employee-related expenses associated with our research and development activities, including salaries, benefits, travel and non-cash stock-based compensation expenses;

•
manufacturing process-development, clinical supplies and technology-transfer expenses;

•
license fees and milestone payments under our license agreements;

•
consulting fees paid to third parties;

•
allocated facilities and overhead expenses; and

•
costs associated with regulatory operations and regulatory compliance requirements.

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Internal and external research and development costs are expensed as they are incurred. Cost-sharing amounts received by us are recorded as reductions to research and development expense. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations or other information provided to us by our vendors.

Research and development activities are central to our business model. Product candidates in late stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of late-stage clinical trials. We plan to continue to spend a significant amount of our resources on research and development activities for the foreseeable future as we continue to advance the development of our product candidates. The amount of research and development expenses allocated to external spending will continue to grow, while we expect our internal spending to grow at a slower and more controlled pace.

It is difficult to determine, with certainty, the duration and completion costs of our current or future preclinical programs, research studies and clinical trials of our product candidates. The duration, costs and timing of research studies and clinical trials of our products and product candidates will depend on a variety of factors that include, but are not limited to, the following:

•
per patient costs;

•
the number of patients that participate;

•
the number of clinical trial sites;

•
the countries in which the trials are conducted;

•
the length of time required to enroll eligible patients;

•
the potential additional safety monitoring or other studies requested by regulatory agencies;

•
the duration of patient monitoring;

•
the efficacy and safety profile of the product candidates; and

•
timing and receipt of any regulatory approvals.

In addition, the probability of success for each drug candidate will depend on numerous factors, including competition, manufacturing capability and commercial viability. The successful development of our products and additional product candidates is highly uncertain. At this time, we cannot reasonably estimate the nature, timing or costs of the efforts that will be necessary to complete the remainder of the development of our products and additional product candidates for the period, if any, in which material net cash inflows from these potential product candidates may commence. Clinical development timelines, the probability of success and development costs can differ materially from expectations.

Selling, General and Administrative

Selling, general and administrative expenses consist primarily of employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, business development and support functions, as well as sales and marketing expenses to support the launch and commercialization of Revuforj and Niktimvo. Other selling, general and administrative expenses include facility-related costs not otherwise allocated to research and development expenses and accounting, tax, legal, information technology and consulting services.

Royalty Interest Expense

Royalty interest expense consists of the interest recorded related to the Royalty Pharma Purchase and Sale Agreement under the effective interest method.

Interest Expense

Interest expense consists primarily of expense related to the interest recognized for capital leases.

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Interest Income

Interest income consists of income earned on our cash equivalents and short and long-term investment balances.

Other (Expense) Income, net

Other (expense) income, net consists of the revaluation of foreign currency related to trade payables.

Recent Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard setting bodies and adopted by us as of the specified effective date. Unless otherwise discussed in "Summary of Significant Accounting Policies" (Note 3) to our audited consolidated financial statements included in this Annual Report, we believe that the impact of recently issued standards that are not yet effective will not have a material impact on our financial position or results of operations upon adoption.

Critical Accounting Policies and Estimates

The preparation of our consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States, or U.S. GAAP, requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenue and expense and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and assumptions. We base our estimates on historical experience, known trends and events and various other factors that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. Other significant accounting policies are outlined in "Summary of Significant Accounting Policies" (Note 3) to our consolidated financial statements included in this Annual Report.

We have listed below our critical accounting estimates that we believe to have the greatest potential impact on our consolidated financial statements. Historically, our assumptions, judgments and estimates relative to our critical accounting estimates have not differed materially from actual results.

Product Revenue Recognition

We recognize revenue when our customer obtains control of the promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services. We recognize revenue following the five – step model prescribed under FASB Accounting Standards Codification (ASC 606), Revenue from Contracts with Customers: (i) identify contract(s) with customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy the performance obligations. We only recognize revenue when it is probable that it will collect the consideration to which it is entitled in exchange for the goods or services that will be transferred to the customer.

We sell Revuforj to specialty distributors and specialty pharmacies, or collectively, Customers. These Customers subsequently resell Revuforj to healthcare providers, patients and other pharmacies. In addition to agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts for the purchase of Revuforj.

We recognize revenue from product sales at the point Customers obtain control of the product, which generally occurs upon delivery. The transaction price that is recognized as product revenue includes an estimate of variable consideration which is described below. Payment terms with Customers do not exceed one year and, therefore, we do not account for a financing component in its arrangements. We expense incremental costs of obtaining a contract with a Customer (for example, sales commissions) when incurred as the period of benefit is less than one year. Shipping and handling costs for product shipments to Customers are recorded as selling, general and administrative expenses.

Revenue from products is recognized at the estimated net sales price (transaction price), which includes estimates of variable consideration. We include estimated amounts in the transaction price to the extent it is determined probable that a significant reversal of cumulative revenue recognized will not occur when the

67

uncertainty associated with the variable consideration is resolved. Our estimates of variable consideration and determination of whether to include estimated amounts in the transaction price are based largely on an assessment of its anticipated performance and all information (historical, current and forecasted) that is reasonably available. The components of our variable consideration around product revenue include provider chargebacks and discounts, trade discounts and allowances, product returns, government rebates, patient assistance, and distribution and other fees.

Accrued Research and Development Expenses

As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued research and development expenses. This process involves reviewing contracts and vendor agreements, communicating with our applicable personnel to identify services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of actual cost. We make estimates of our accrued and prepaid clinical expenses as of each balance sheet date in our consolidated financial statements based on facts and circumstances known to us at that time. Examples of estimated accrued research and development expenses include fees paid to contract research organizations, or CROs, and investigative sites in connection with clinical studies and to vendors related to product manufacturing and development of clinical supplies.

We base our expenses related to clinical study and trial costs on our estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical studies on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows and expense recognition. Payments under some of these contracts depend on factors out of our control, such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, if our estimates of the status and timing of services performed differ from the actual status and timing of services performed, we may report amounts that are too high or too low in any particular period. To date, we have not experienced any significant adjustments to our estimates.

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Results of Operations

Comparison of the years ended December 31, 2025 and 2024:

Years Ended

December 31,

2025 - 2024 Increase

(Decrease)

(in thousands)

2025

2024

$

Revenue:

Net product revenue

$

124,844

$

7,680

$

117,164

Collaboration revenue, net

42,367

-

42,367

Milestone, license, and royalty revenue

5,141

16,000

(10,859

)

Total revenue

172,352

23,680

148,672

Operating expenses:

Cost of product sales

6,970

826

6,144

Research and development

258,784

241,647

17,137

Selling, general and administrative

179,682

120,879

58,803

Total operating expenses

445,436

363,352

82,084

Loss from operations

(273,084

)

(339,672

)

66,588

Other (expense) income, net:

Royalty interest expense

(33,779

)

(4,930

)

(28,849

)

Other interest (expense) income

(6

)

1

(7

)

Interest income

22,730

26,090

(3,360

)

Other expense, net

(1,283

)

(247

)

(1,036

)

Total other (expense) income, net

(12,338

)

20,914

(33,252

)

Net loss

$

(285,422

)

$

(318,758

)

$

33,336

Net product revenue

In November 2024, we began to generate product revenue from sales of Revuforj in the United States. Net product revenue from sales of Revuforj was $124.8 million and $7.7 million for the years ended December 31, 2025 and 2024, respectively.

Collaboration revenue, net

Collaboration revenue, net, representing our share of net profits in connection with commercialization of Niktimvo, for the year ended December 31, 2025 was $42.4 million. We generated no collaboration revenue for the years ended December 31, 2024, as Niktimvo launched in January 2025.

Milestone, license, and royalty revenue

Milestone, license, and royalty revenue for the fiscal year ended December 31, 2025 included $5.0 million milestone from Incyte, recognized for the achievement of $150.0 million total net sales of Niktimvo under the Incyte Collaboration Agreement, and $0.1 million of royalty revenue related to sales of entinostat.

In April 2024, a milestone was achieved under the Eddingpharm License Agreement for the marketing approval of entinostat in China. As a result, we recognized $3.5 million of milestone revenue in the second quarter of 2024. As the receivable remains outstanding, we have assessed the receivable as non-recoverable and recorded a full reserve against the asset.

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In August 2024, a milestone was achieved under the Incyte Collaboration Agreement for the approval of Niktimvo. As a result, we recognized $12.5 million of milestone revenue in the third quarter of 2024.

Cost of Product Sales

Our cost of product sales were $7.0 million and $0.8 million for the twelve months ended December 31, 2025 and 2024, respectively. The increase is a result of increased sales of Revuforj. Included in cost of product sales are royalties owed to AbbVie on Revuforj sales as part of the Vitae License Agreement.

Research and Development

The following table summarizes the research and development expenses for the full years ended December 31, 2025 and 2024:

Years Ended

December 31,

2025 - 2024 Increase

(Decrease)

(in thousands)

2025

2024

$

Revumenib-related costs

$

112,422

$

107,909

$

4,513

Axatilimab-related costs

48,289

49,638

(1,349

)

Other R&D programs

4,154

2,564

1,590

Personnel cost and other expenses

77,887

61,602

16,285

Stock-based compensation

16,032

19,934

(3,902

)

Total research and development expenses

$

258,784

$

241,647

$

17,137

For the year ended December 31, 2025, our total research and development expenses increased by $17.1 million from the prior year. The increase is primarily due to:

•
An increase of $4.5 million in revumenib-related costs due to the costs associated with initiation of trials evaluating revumenib in combination with standard-of-care agents in NPM1m AML and KMT2A acute leukemia in newly diagnosed patients, offset by the completion of an R/R NPM1m AML registrational trial that was ongoing in 2024 and a reduction in CMC expenses due to capitalization of inventory for commercial use. Higher development costs were also offset by a $6.0 million decrease in revumenib-related milestone expense in 2025.

•
A decrease of $1.3 million in axatilimab related costs driven by a net reduction of $5.0 million in milestone payments compared to prior year. This decrease was partially offset by higher expenses attributable to the ongoing Phase 2 IPF trial and our costs incurred in the execution of the frontline cGVHD combination trial with ruxolitinib.

•
A increase in other research and development program related costs of $1.6 million due to the de-prioritization of programs not pertaining to revumenib and axatilimab in the prior year period.

•
An increase of $16.3 million in personnel costs and other expenses, which includes salaries, overhead and related expenses to support on-going clinical trials, preparation of a supplemental New Drug Application, and medical affairs activities in support of commercialization of Revuforj and Niktimvo.

•
A decrease of $3.9 million in stock-based compensation expense related to the recognition of performance based awards in 2024 for certain regulatory achievements, as well as certain equity modifications.

The following table summarizes the selling, general and administrative expenses for the full years ended December 31, 2025 and 2024:

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Years Ended

December 31,

2025 - 2024 Increase

(Decrease)

(in thousands)

2025

2024

$

Commercial related expenses

$

53,630

$

33,541

$

20,089

Other SG&A expenses

23,310

17,353

5,957

Personnel cost and other expenses

71,271

46,893

24,378

Stock-based compensation

31,471

23,092

8,379

Total selling, general and administrative expenses

$

179,682

$

120,879

$

58,803

For the year ended December 31, 2025, our total selling, general and administrative expenses increased by $58.8 million from the prior year. The increase primarily is due to:

•
An increase of $20.1 million in commercial related expenses of which $10.0 million of the increase is related to higher selling and marketing activities to support the launches of Revuforj and Niktimvo, and $10.0 million of the increase was driven by a milestone expense triggered by the achievement of $150.0 million in Niktimvo net sales in 2025.

•
An increase of $32.8 million in personnel costs and other expenses, including non-cash stock-based compensation, recognition of performance-based equity, based on certain commercial achievements. Also included are salaries, overhead and related expenses primarily due to increased headcount to support the commercialization of Revuforj and Niktimvo.

•
An increase of $6.0 million in other SG&A expenses related to increases in IT related expenses, corporate communications, legal and recruiting costs in support of commercialization and ongoing R&D activities.

Royalty Interest Expense

For the year ended December 31, 2025, royalty interest expense increased from the prior year due to 2025 being the first full year of accrued interest expense related to the Royalty Pharma Purchase and Sale Agreement.

Interest Income

For the year ended December 31, 2025, interest income, net of interest expense, decreased from the comparable prior year period. The decrease of interest income was primarily due to lower interest rates and a decreased average balance on cash equivalents and short- and long-term investments.

Other (Expense) Income, net

For the year ended December 31, 2025, the total other (expense) income, net increased from the comparable prior year period primarily due to the increase in foreign currency losses on short- and long-term investments.

Liquidity and Capital Resources

Overview

As of December 31, 2025, we had cash, cash equivalents and short-term investments totaling $394.1 million. Since our inception, our operations have been primarily financed by net proceeds from our public stock offerings, revenue from our license agreements, and through our purchase and sale agreement with Royalty Pharma. We believe that our cash, cash equivalents and short-term investments as of December 31, 2025, will fund our projected operating expenses and capital expenditure requirements for at least the next 12 months. In addition to our existing cash, cash equivalents, short-term investments, we are eligible to receive research and development funding and to earn milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and commercial milestones, and royalty payments under our collaboration agreements. Our

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ability to earn these milestone and contingent payments and the timing of achieving these milestones is primarily dependent upon the outcome of our collaborators’ research and development activities and is uncertain at this time.

Purchase and Sale Agreement

In November 2024, we entered into a purchase and sale agreement with Royalty Pharma, pursuant to which Royalty Pharma purchased the right to receive a 13.8% royalty on quarterly net sales of Niktimvo in the United States of America and its respective territories, districts, commonwealths and possessions (including Guam and Puerto Rico) in exchange for an upfront payment of $350.0 million (gross) at closing, received in November 2024. Aggregate payments to Royalty Pharma pursuant to the Royalty Agreement are capped at $822.5 million or 2.35 times the funded amount.

For additional details on our purchase and sale agreement with Royalty Pharma, see "Purchase and Sale Agreement" (Note 16) to our consolidated financial statements in this Annual Report.

Future Funding Requirements

We believe that the combination of our available cash, cash equivalents, short-term investments, as well as our expected product revenues from sales of Revuforj and Niktimvo collaboration revenue, is sufficient to fund existing and planned cash requirements. Our primary uses of capital are, and we expect will continue to be, compensation and related expenses, third-party clinical research and development services, clinical costs, commercialization costs, legal and other regulatory expenses and general overhead costs. We have based our estimates on assumptions that may prove to be incorrect, and we could use our capital resources sooner than we currently expect.

Additionally, the process of testing product candidates in clinical trials is costly, and the timing, progress and outcomes in these trials is uncertain. We cannot estimate the actual amounts necessary to successfully complete the development and commercialization of our product candidates or whether, or when, we may achieve profitability.

Our future capital requirements will depend on many factors, including:

•
our growth rate;

•
the initiation, progress, timing, costs and results of clinical trials of our product candidates;

•
the outcome, timing and cost of seeking and obtaining additional regulatory approvals from the FDA and comparable foreign regulatory authorities, including the potential for such authorities to require that we perform more trials than we currently expect;

•
the cost to establish, maintain, expand and defend the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with licensing, preparing, filing, prosecuting, defending and enforcing any patents or other intellectual property rights;

•
market acceptance of our approved products as well as our product candidates;

•
the cost and timing of selecting, auditing and developing manufacturing capabilities, and potentially validating manufacturing sites for commercial-scale manufacturing;

•
the cost and timing for obtaining pricing and reimbursement, which may require additional trials to address pharmacoeconomic benefit;

•
the cost of maintaining and expanding sales, marketing and distribution capabilities for our products;

•
the costs of acquiring, licensing or investing in additional businesses, products, product candidates and technologies;

•
the interruption of key clinical trial activities, such as clinical trial site monitoring;

72

•
the cost of disruption to our supply chain and operations, and associated delays in the manufacturing and supply of our products, which would adversely impact our ability to continue our clinical trial operations;

•
the effect of competing technological and market developments; and

•
our need to implement additional internal systems and infrastructure, including financial and reporting systems, as we grow our company.

We expect to continue to support our future cash needs through a combination of product sales, equity offerings, debt financings and additional funding from license and collaboration arrangements. Except for any obligations of our collaborators to reimburse us for research and development expenses or to make milestone or royalty payments under our agreements with them, we will not have any committed external source of liquidity.

Our material cash requirements include the following contractual obligations as of December 31, 2025, and the effects that such obligations are expected to have on our liquidity and cash flows in future periods. For additional information, see our consolidated financial statements.

(in thousands)

Total

Less than

1 Year

1 to 3

Years

3 to 5

Years

More than

5 Years

Operating leases for office space (1)

$

1,735

$

656

$

1,079

$

—

$

—

Capital lease for office equipment (2)

2

2

—

—

—

$

1,737

$

658

$

1,079

$

—

$

—

(1)
In August 2022, we signed a 36-month extension of the lease for the office space in New York, NY. In May 2023, we signed a 27-month lease for an additional space in New York, NY. In October 2024, we signed a 36-month extension for the New York, NY office space. The minimum lease payments above do not include any related common area maintenance charges or real estate taxes.

(2)
In January 2022, we entered into two four-year non-cancelable leases for office equipment. In June 2023, we entered into one two-year non-cancelable lease for office equipment. All three leases are accounted for as a capital lease.

We have incurred losses and cumulative negative cash flows from operations since our inception, excluding year ending December 31, 2021. As of December 31, 2025, we had an accumulated deficit of $1.5 billion. We anticipate that we will continue to incur significant losses for the near future. To the extent that we raise additional capital through the future sale of equity or debt, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing common stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or drug candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market drug candidates that we would otherwise prefer to develop and market ourselves.

At-the-Market Offering Program

In May 2023, we entered into a sales agreement with TD Cowen under which we could, from time to time, issue and sell shares of our common stock having aggregate sales proceeds of up to $200.0 million, in a series of one or more ATM equity offerings, or the 2023 ATM Program. TD Cowen is not required to sell any specific share amounts but acts as the Company’s sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the sales agreement, shares will be sold under the shelf registration statement on Form S-3ASR (Registration No. 333-254661), which became automatically effective upon the filing on March 24, 2021. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. As of December 31, 2023, we sold 2,719,744 shares of common stock under the 2023 ATM Program for net proceeds of approximately $42.1 million. There was no ATM equity activity during the twelve months ended December 31, 2025 and 2024.

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Cash Flows

The following is a summary of cash flows:

Years Ended December 31,

(in thousands)

2025

2024

2023

Net cash used in operating activities

$

(322,980

)

$

(274,903

)

$

(160,601

)

Net cash provided by (used in) investing activities

290,036

(219,775

)

117,609

Net cash provided by financing activities

13,676

353,367

264,132

Net (decrease) increase in cash and cash equivalents

$

(19,268

)

$

(141,311

)

$

221,140

Net Cash Used in Operating Activities

Net cash used in operating activities for the year ended December 31, 2025, was $323.0 million and primarily consisted of our net loss of $285.4 million adjusted for non-cash items including stock-based compensation of $47.5 million, an investment increase of $10.8 million, a net increase in operating assets and liabilities of $79.3 million and non-cash operating lease expense of $0.9 million. The significant items driving the increase in operating assets and liabilities include an increase in collaboration receivable, net, of $43.0 million, an increase in accounts receivable of $30.4 million, an increase in inventory of $32.1 million, and an increase in prepaid expenses and other assets of $14.5 million. This was partially offset by an increase in accrued expense and other current liabilities of $42.2 million.

Net cash used in operating activities for the year ended December 31, 2024 was $274.9 million and primarily consisted of our net loss of $318.8 million adjusted for non-cash items including stock-based compensation of $43.0 million, an investment increase of $13.5 million, a net increase in operating assets and liabilities of $13.3 million, and non-cash operating lease expense of $1.0 million. The significant items driving the increase in operating assets and liabilities include an increase in accrued expenses and other liabilities of $19.1 million, an increase in collaboration payable of $12.0 million, an increase in accounts payable of $1.7 million, and an increase in prepaid expenses and other assets of $8.3 million.

Net Cash Provided by (Used in) Investing Activities

Net cash provided by investing activities for the year ended December 31, 2025 was $290.0 million and was due to $486.0 million in proceeds from the maturities of available-for-sale marketable securities, partially offset by the purchase of $195.8 million of available-for-sale marketable securities.

Net cash used in investing activities for the year ended December 31, 2024 was $219.8 million due to $337.3 million in proceeds from the maturities of available-for-sale marketable securities, partially offset by the purchase of $557.1 million of available-for-sale marketable securities.

Net Cash Provided by Financing Activities

Net cash provided by financing activities for the year ended December 31, 2025, was $13.7 million and was primarily due to $13.7 million of proceeds from the stock option exercises and ESPP purchases.

Net cash provided by financing activities for the year ended December 31, 2024, was $353.4 million and was primarily due to proceeds of $343.7 million from the Royalty Pharma Purchase and Sale agreement and $9.7 million of proceeds from the stock option exercises and ESPP purchases.

74

Net Operating Loss and Research and Development Tax Credit Carryforwards

At December 31, 2025, we had federal and state tax net operating loss carryforwards, or NOLs, of approximately $585.3 million and $227.7 million, respectively. We have generated federal NOLs of $563.2 million and state NOLs of $8.7 million which have an indefinite carryforward period. The remaining $22.1 million of federal NOLs and the $219.0 million of state NOLs will begin to expire at various dates starting in 2026. At December 31, 2025, we had available research and development and orphan drug tax credits of approximately $9.8 million and $27.0 million, respectively. Income tax credits began to expire in 2024.

Effective January 1, 2024, we have reclassified as an active trade or business for tax purposes. This resulted in a reclass of capitalized start-up and other costs to net operating losses and valuation allowance in the 2025 tax statements.

Utilization of the net operating losses and credits may be subject to a substantial annual limitation due to ownership change limitations provided by the Internal Revenue Code of 1986, as amended. The annual limitation may result in the expiration of our net operating losses and credits before we can use them. We have recorded a valuation allowance on all of our deferred tax assets, including our deferred tax assets related to our net operating loss and research and development tax credit carryforwards.