SNDX 10-K & 10-Q changes, risk factors and insider trading
Syndax Pharmaceuticals Inc · Nasdaq · Pharmaceutical Preparations · CIK 1395937 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
New heading “Our business depends heavily on our ability to successfully commercialize Revuforj and Niktimvo in the United States and in other jurisdictions where we may obtain marketing approval. There is no assurance that our commercialization efforts with respect to Revuforj or Niktimvo will be successful or that we will be able to generate revenues at the levels or on the timing we expect.”
New heading “We have recently begun commercialization efforts and the sales, marketing, and distribution of Revuforj, Niktimvo, or any future approved products may be unsuccessful or less successful than anticipated.”
New heading “We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.”
New heading “Disruptions at the FDA and other government agencies caused by layoffs, funding shortages or global health concerns could negatively impact our business.”
New heading “We are in the process of building and maintaining our sales, marketing and distribution infrastructure.”
New heading “We currently rely, and expect to continue to rely, on third-party contract manufacturers as well as Incyte for all of our required raw materials, active pharmaceutical ingredients and finished product for our preclinical research, clinical trials, and commercial manufacturing and distribution.”
Removed heading “We have limited experience in generating revenue from product sales.”
Removed heading “We rely on third-party suppliers as well as Incyte to manufacture and distribute our clinical drug supplies for our product candidates, we intend to rely on third parties for commercial manufacturing and distribution of our product candidates and we expect to rely on third parties for manufacturing and distribution of preclinical, clinical and commercial supplies of any future product candidates.”
Removed heading “We are in the process of building our sales, marketing and distribution infrastructure.”
Largest changes
“Additionally, the U.S. Department of Justice issued a rule entitled the Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons, which places additional restriction on certain data transactions involving countries of concern (e.g., China, Russia, Iran) and covered persons (i.e., individuals and entities who are designated as such by the U.S. …”see in full comparison
“Also, there has been heightened governmental scrutiny recently over the manner in which drug manufacturers set prices for their marketed products, which have resulted in several, Presidential executive orders, Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products. …”see in full comparison
“Disruptions at the FDA and other government agencies caused by layoffs, funding shortages or global health concerns could negatively impact our business.”see in full comparison
“We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.”see in full comparison
“The current administration is also pursuing policies to reduce regulations and expenditures across government including at HHS, the FDA, CMS and related agencies. These actions, presently directed by executive orders or memoranda from the Office of Management and Budget, may propose policy changes that create additional uncertainty for our business. For example, the current administration has announced agreements with several pharmaceutical companies that require the drug manufacturers to offer, through a direct-to-consumer platform, U.S. …”see in full comparison
“Our business is reliant on revenue from behavioral, interest-based, or tailored advertising, collectively targeted advertising, but delivering targeted advertisements is becoming increasingly difficult due to changes to our ability to gather information about user behavior through third party platforms, new laws and regulations, and consumer resistance. …”see in full comparison
Full comparison: every changed paragraph (150)
Our business depends heavily on our ability to successfully commercialize Revuforj and Niktimvo in the United States and in other jurisdictions where we may obtain marketing approval. There is no assurance that our commercialization efforts with respect to Revuforj or Niktimvo will be successful or that we will be able to generate revenues at the levels or on the timing we expect.
We have recently begun commercialization efforts and the sales, marketing, and distribution of Revuforj, Niktimvo, or any future approved products may be unsuccessful or less successful than anticipated.
We have limited experience in generating revenue from product sales.
We are currently developing several product candidates. If we are unable to successfully complete clinical development of, obtain regulatory approval for, and commercialize our product candidates, our business prospects willmay be significantly harmed.
Revuforj, Niktimvo and our future product candidatesproducts may not achieve adequate market acceptance among physicians, patients, healthcare payors and others in the medical community to be commercially successful.
We currently rely, and expect to continue to rely, on third-party contract manufacturers as well as Incyte for all of our required raw materials, active pharmaceutical ingredients and finished product for our preclinical research, clinical trials, and commercial manufacturing and distribution.
We rely on third-party suppliers as well as Incyte to manufacture and distribute our clinical drug supplies for our product candidates, we intend to rely on third parties for commercial manufacturing and distribution of our product candidates and we expect to rely on third parties for manufacturing and distribution of preclinical, clinical and commercial supplies of any future product candidates.
We have incurred net losses in each period since our inception, except in 2021, and anticipate that we will continue to incur net losses for the foreseeablenear future.
While we have productproduct, collaboration and milestone revenue, we may never achieve or maintain profitability.
We may not be able to protect our intellectual property rights throughout the world.
Our business depends heavily on our ability to successfully commercialize Revuforj and Niktimvo in the United States and in other jurisdictions where we may obtain marketing approval. There is no assurance that our commercialization efforts with respect to Revuforj or Niktimvo will be successful or that we will be able to generate revenues at the levels or on the timing we expect.
In November 2024, Revuforj was approved by the FDA for the treatment of R/R acute leukemia with a 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 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. The FDA approved Niktimvo in August 2024 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.
Our business currently depends heavily on our ability to successfully commercialize Revuforj in the United States and in other jurisdictions where we may obtain marketing approval as well as commercializing Niktimvo, with our collaboration partner Incyte, in the United States. We may never be able to successfully commercialize our products or meet our expectations with respect to revenues. We have never marketed, sold, or distributed for commercial use any other products, nor have we marketed, sold or distributed for commercial use any product in any other market outside of the United States. There is no guarantee that the infrastructure, systems, processes, policies, relationships, and materials we have built for the launch and commercialization of Revuforj or Niktimvo in the United States will be sufficient for us to achieve success at the levels we expect.
We may encounter issues and challenges in commercializing Revuforj and Niktimvo and generating substantial revenues. We may also encounter challenges related to reimbursement of Revuforj or Niktimvo, including potential limitations in the scope, breadth, availability, or amount of reimbursement covering Revuforj or Niktimvo, respectively. Similarly, healthcare settings or patients may determine that the financial burdens of treatment are not acceptable. We may face other limitations or issues related to the price of Revuforj or Niktimvo. Our results may also be negatively impacted if we have not adequately sized our field teams or our physician segmentation and targeting strategy is inadequate or if we encounter deficiencies or inefficiencies in our infrastructure or processes. Other factors that may hinder our ability to successfully commercialize Revuforj, Niktimvo, or any of our future approved drugs, and generate substantial revenues, include:
the acceptance of Revuforj and Niktimvo by patients and the medical community;
the ability of our third-party manufacturer(s) to manufacture commercial supplies of Revuforj or Niktimvo at acceptable costs, to remain in good standing with regulatory agencies, and to maintain commercially viable manufacturing processes that are, to the extent required, compliant with cGMP regulations;
our ability to remain compliant with laws and regulations that apply to us and our commercial activities;
FDA-mandated package insert requirements and successful completion of any related FDA post-marketing requirements;
the actual market size for Revuforj or Niktimvo, respectively, which may be different than expected;
the length of time that patients who are prescribed our drug remain on treatment;
the sufficiency of our drug supply to meet commercial and clinical demands which could be negatively impacted if our projections regarding the potential number of patients are inaccurate, we are subject to unanticipated regulatory requirements, or our current drug supply is destroyed, or negatively impacted at our manufacturing or storage sites, or in transit;
our ability to effectively compete with other therapies that may emerge; and our ability to maintain, enforce, and defend third party challenges to our intellectual property rights in and to Revuforj and Niktimvo.
Any of these issues could impair our ability to successfully commercialize our products or to generate substantial revenues or profits or to meet our expectations with respect to the amount or timing of revenues or profits. Any issues or hurdles related to our commercialization efforts may materially adversely affect our business, results of operations, financial condition, and prospects. There is no guarantee that we will be successful in our commercialization efforts with respect to Revuforj or Niktimvo. We may also experience significant fluctuations in sales of Revuforj or Niktimvo from period to period and, ultimately, we may never generate sufficient revenues from Revuforj and Niktimvo to reach or maintain profitability or sustain our anticipated levels of operations. Any inability on our part to successfully commercialize Revuforj or Niktimvo in the United States, and any other international markets where it may subsequently be approved, or any significant delay, could have a material adverse impact on our ability to execute upon our business strategy.
We have recently begun commercialization efforts and the sales, marketing, and distribution of Revuforj, Niktimvo, or any future approved products may be unsuccessful or less successful than anticipated.
We began commercialization of Revuforj in November 2024 and Niktimvo in February 2025. As a company, we had no prior experience commercializing a product. The success of our commercialization efforts for Revuforj, Niktimvo, and any future approved products is difficult to predict and subject to the effective execution of our business plan, including, among other things, the continued development of our internal sales, marketing, and distribution capabilities and our ability to navigate the significant expenses and risks involved with the development and management of such capabilities.
For example, we have expanded in areas to support commercialization, including in sales management, sales representatives, marketing, market access and reimbursement, sales support, and distribution. There are significant risks involved with establishing our own sales, marketing, and distribution capabilities, including risks related to our ability to hire, retain, and appropriately incentivize qualified individuals, provide adequate training to sales and marketing personnel, and effectively manage geographically dispersed sales teams to generate sufficient demand. Any failure or delay in the development of these capabilities could delay or negatively affect the success of our commercialization efforts and our business. In addition, the commercialization of Revuforj and Niktimvo may not develop as planned or anticipated, which may require us to adjust or amend our business plan and incur significant expenses.
Further, given our recent launches of Revuforj and Niktimvo, we do not have a track record of successfully executing on the commercialization of approved products. If we are unsuccessful in accomplishing our objectives and executing on our business plan, or if the commercialization of Revuforj and Niktimvo or any future approved products does not develop as planned, we may require significant additional capital and financial resources, we may not become profitable, and we may not be able to compete against more established companies in our industry.
We have limited experience in generating revenue from product sales.
Our ability to generate significant revenue from product sales depends on our ability to successfully commercialize Revuforj and Niktimvo and to obtain the regulatory and marketing approvals necessary to commercialize revumenib and axatilimab for other indications. We currently have limited commercialization expertise, including sales, marketing, or distribution capabilities, and Revuforj, Niktimvo and future approved products may not remain in the market for a number of reasons, including ineffectiveness, harmful side effects, difficulty in scaling manufacturing, political and legislative changes, or competition from existing future alternatives. Our ability to generate substantial future revenue from product sales depends heavily on our success in many areas, including, but not limited to:
developing a sustainable manufacturing process for products and establishing and maintaining supply and manufacturing relationships with third parties that can conduct the processes and provide adequate (in amount and quality) product supply to support market demand for products;
launching and commercializing products;
obtaining market acceptance of products;
obtaining adequate market share, reimbursement and pricing for products;
our ability to find patients so they can be diagnosed and begin receiving treatment;
addressing any competing technological and market developments;
negotiating favorable terms, including commercial rights, in any collaboration, licensing, or other arrangements into which we may enter, any amendments thereto or extensions thereof;
maintaining, protecting, and expanding our portfolio of intellectual property rights, including patents, trade secrets, and know-how; and attracting, hiring, and retaining qualified personnel.
If the number of our addressable patients is not as significant as we estimate or the reasonably accepted population for treatment is narrowed by competition, physician choice, or treatment guidelines, we may not generate significant revenue from sales of products.
Our projections of the number of adult and pediatric patients who have the potential to benefit from treatment with either Revuforj or Niktimvo are based on our beliefs and estimates. These estimates have been derived from a variety of sources and may prove to be incorrect or new studies may change the estimated incidence or prevalence, and the number of patients may turn out to be lower than expected. Additionally, the potentially addressable patient population for Revuforj or Niktimvo may be limited or may not be amenable to treatment with Revuforj or Niktimvo, and new patients may become increasingly difficult to identify or access, which would adversely affect our results of operations and our business. If the number of our addressable patients is not as significant as we estimate or the reasonably expected population for treatment is narrowed by competition, physician choice, or treatment guidelines, revenue from sales of products may be more limited than we expect. Even if we obtain significant market share for Revuforj or Niktimvo, we may never become or remain profitable nor generate sufficient revenue growth to sustain our business.
We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
Revuforj is FDA approved for the treatment of patients one year and older with R/R acute leukemia with a KMT2A translocation or R/R AML with an NPM1 mutation. In November 2025, the FDA approved Komzifti™ (ziftomenib) for adult patients with R/R AML with an NPM1 mutation. At this time, other than Revuforj and Komzifti, there are no other drugs specifically approved for these genetic subtypes of acute leukemia, although other types of therapies can also be used in the treatment of these patient populations. While there are additional menin inhibitors in development for similar patient populations, Revuforj is the first and currently the only therapy FDA approved for both adults and pediatrics with R/R acute leukemia with a KMT2A translocation or R/R AML with an NPM1 mutation.
Existing or potential competitors have or may have substantially greater financial, technical and human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining FDA and other regulatory approvals of product candidates and the commercialization of those products. Our competitors may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours and may be more successful than us in obtaining FDA approval for drugs and achieving widespread market acceptance of their products. Our competitors’ drugs may also be more effective or more effectively marketed and sold than any drug we may commercialize and may render our product candidates obsolete or non-competitive before we can recover the expenses of developing and commercializing any of our product candidates.
the market adoption of Revuforj, Niktimvo and any future products by physicians and patients;
the efficacy and safety profile of our products and product candidates relative to marketed products and product candidates in development by third parties;
the price of Revuforj, Niktimvo and any future products, including in comparison to branded or generic competitors;
We anticipate that we will face intense and increasing competition as new drugs enter the market and advanced technologies become available. Even if we obtain regulatory approval of our other product candidates, the availability, commercial formulary placement, and price of our competitors’ products could limit the demand and the price we are able to charge. We may not be able to implement our business plan if the acceptance of our product candidates is inhibited by price competition or the reluctance of physicians to switch from existing methods of treatment, or if physicians switch to other new drug or biologic products or choose to reserve our drugs for use in limited circumstances.
OurAlthough approvedwe productshave arebegun generating revenue from product sales of Revuforj and Niktimvo.Niktimvo, Ourour financial success will also depend substantially on our ability to effectively and profitably commercialize our otherproducts and product candidates and expand the approved indications for Revuforj and Niktimvo. In order to commercialize our product candidates and expand the approved indications for Revuforj and Niktimvo, we will be required to obtain regulatory approvals by establishing that each of them is sufficiently safe and effective. The clinical and commercial success of our product candidates will depend on a number of factors, including the following:
If we fail to obtain regulatory approval for our product candidates, we will not be able to generatecommercialize our product sales,candidates, which will have a material adverse effect on our business and our prospects.
For example, there is no assurance that the parties will achieve any of theadditional regulatory development or sales milestones,milestones or that we will receive any future milestone or royalty payments under the collaborationIncyte agreement.Collaboration Agreement. Incyte’s activities may be influenced by, among other things, the efforts and allocation of resources by Incyte, which we cannot control. If Incyte does not perform in the manner we expect or fulfill its responsibilities in a timely manner, or at all, the clinical development, manufacturing, regulatory approval, and commercialization efforts related to axatilimab could be delayed or terminated. In addition, our license with Incyte may be unsuccessful due to other factors, including, without limitation, the following:
the impact of public health crises;
the impact of public health crises, or geopolitical tensions, such as the ongoing wars involving Russia and Israel;
evolving standard of care therapies in treating cancer patients;
Our collaborations, including any future strategic collaborations we enter into, could subject us to a number of risks, including that:
The FDA and comparable foreign regulatory authorities extensively and rigorously regulate and evaluate the manufacture, testing, distribution, advertising and marketing of drug products prior to granting marketing approvals with respect to such products. This approval process generally requires, at minimum, testing of any product candidate in preclinical studies and clinical trials to establish its safety and effectiveness, and confirmation by the FDA and comparable foreign regulatory authorities that any such product candidate, and any parties involved in its manufacturing, testing and development, complied with cGMP, current Good Laboratory PracticescGLP and current Good Clinical Practices,cGCP, regulations, standards and guidelines during such manufacturing, testing and development. The time required to obtain approval by the FDA and foreign regulatory authorities is unpredictable, but typically takes many years following the commencement of preclinical studies and clinical trials and depends upon numerous factors, including the substantial discretion of the regulatory authorities. In addition, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions. Other than the recent approval for marketing of entinostat tablets by the National Medical Products Administration, we have not obtained regulatory approval for any of our product candidates and it is possible that we will never obtain regulatory approval for any additional product candidates or any future product candidates.
receipt of a negative opinion from an advisory committee due to a change in the standard of care therapies regardless of the outcome of the clinical trials; or changes in the approval policies or regulations that render our preclinical and clinical data insufficient for approval.
The FDA or foreign regulatory authorities may require more information, including additional preclinical or clinical data, to support approval, which may delay or prevent approval and our commercialization plans, or may cause us to decide to abandon our development program. Even if we were to obtain approval, regulatoryRegulatory authorities may approve one or more of our product candidates for a more limited patient population than we request, may grant approval contingent on the performance of costly post-marketing trials, may impose a risk evaluation and mitigation strategy, or REMS, or foreign regulatory authorities may require the establishment or modification of a similar strategy that may, for instance, restrict distribution of one or more of our product candidates and impose burdensome implementation requirements on us, or may approve it with a label that does not include the labeling claims necessary or desirable for the successful commercialization of one or more of our product candidates, all of which could limit our ability to successfully commercialize our product candidates. Moreover, if adopted in the form proposed, the recent European Commission proposals to revise the existing European Union, or EU, laws governing authorization of medicinal products may result in a decrease in data and market exclusivity for our product candidates in the EU.
Disruptions at the FDA and other government agencies caused by layoffs, funding shortages or global health concerns could negatively impact our business.
The ability of the FDA to review proposed clinical trials or approve new products can be affected by a variety of factors, including government budget and funding levels, statutory, regulatory, and policy changes, the FDA’s ability to hire and retain key personnel and accept payment of user fees, and other events that may otherwise affect the FDA’s ability to perform routine functions. Average review times at the agency have fluctuated in recent years as a result. In addition, government funding of other government agencies that fund research and development activities is subject to the political process, including executive and congressional priorities, the impacts of which are inherently fluid and unpredictable.
Disruptions at the FDA and other agencies may slow the time necessary for new product candidates to be reviewed and/or approved, which would adversely affect our business. For example, over the last several years, including in October 2025, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA, have had to furlough critical FDA employees and stop critical activities. If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business. In addition, the current administration has implemented substantial reductions in force at various government agencies including the FDA, and has implemented layoffs at the FDA, which could significantly reduce the FDA’s capacity to perform its functions in a manner consistent with its past practices and could delay reviews and negatively impact our business.
Revuforj, Niktimvo and our future product candidatesproducts may not achieve adequate market acceptance among physicians, patients, healthcare payors and others in the medical community to be commercially successful.
Management's Discussion & Analysis (MD&A)
New heading “Cost of Product Sales”
New heading “Royalty Interest Expense”
New heading “Cost of Product Sales”
New heading “Interest Income”
Removed heading “Selling, General and Administrative”
Removed heading “Loan and Security Agreement”
Largest changes
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 geopoliticalsee in full comparisontensions as a result of the ongoing wars between Russia and Ukraine and Israel and Hamas as well as the conflicts in the Middle East, including between Israel and Hezbollah,tensions, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.
see in full comparisonWe are a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. We currently have two commercially approved products, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and a robust slate of clinical development programs designed to unlock their full potential.Revuforj is our first-in-class menin inhibitor that was approved by theFDAU.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. IntheOctober 2025, Revuforj received a secondquarterapprovaloffrom2025,thewe expect to submit a supplemental New Drug Application, or sNDA,FDA forrevumenib as athe treatmentforof R/R acute myeloid leukemia, or AML, with a susceptible nucleophosmin 1 mutation, ormNPM1,NPM1m,basedinonadulttheandpositivepediatricpivotalpatientsdataonefromyearourandAUGMENT-101oldertrial.who have no satisfactory alternative treatment options. We are also studying revumenib in combination with standard-of-care agents inmNPM1NPM1m AML and KMT2A-rearranged, orKMT2A-rearrangedKMT2Ar, acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring theusepotentialofforrevumenibmeninas a treatmentinhibition insolid tumors, specifically its activity in metastatic colorectal cancer. 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 forthe treatment ofchronic graft-versus-host disease,myelofibrosis, orcGVHD, 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, as well for the treatment of idiopathic pulmonary fibrosis, or IPF. 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.MF.
Full comparison: every changed paragraph (74)
For the discussion of the financial condition and results of operations for the year ended December 31, 20232024 compared to the year ended December 31, 2022,2023, 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 FebruaryMarch 27,3, 2024.2025.
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.
We are a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. We currently have two commercially approved products, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and a robust slate of clinical development programs designed to unlock their full potential. Revuforj is our first-in-class menin inhibitor that was approved by the FDAU.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 theOctober 2025, Revuforj received a second quarterapproval offrom 2025,the we expect to submit a supplemental New Drug Application, or sNDA,FDA for revumenib as athe treatment forof R/R acute myeloid leukemia, or AML, with a susceptible nucleophosmin 1 mutation, or mNPM1,NPM1m, basedin onadult theand positivepediatric pivotalpatients dataone fromyear ourand AUGMENT-101older trial.who have no satisfactory alternative treatment options. We are also studying revumenib in combination with standard-of-care agents in mNPM1NPM1m AML and KMT2A-rearranged, or KMT2A-rearrangedKMT2Ar, acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring the usepotential offor revumenibmenin as a treatmentinhibition in solid tumors, specifically its activity in metastatic colorectal cancer. 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,myelofibrosis, 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, as well for the treatment of idiopathic pulmonary fibrosis, or IPF. 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.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 begunincurred generatingsignificant operating losses since our inception. While we generate product revenue from sales of Revuforj and Niktimvo.collaboration Weas 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, 2023, and 2022,2023, we reported a net loss of $318.8$285.4 million and $209.4$318.8 million, and $149.3$209.4 million, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $1.2$1.5 billion, which included non-cash charges for stock-based compensation, preferred stock accretion and extinguishment charges. As of December 31, 2024,2025, we had cash, cash equivalents and short-term and long-term investments of $692.4$394.1 million.
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 as a result of the ongoing wars between Russia and Ukraine and Israel and Hamas as well as the conflicts in the Middle East, including between Israel and Hezbollah,tensions, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.
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, 2024.2025 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 yearsyear ended December 31, 2023 and 2022.2023.
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.
MilestoneMilestone, License, and LicenseRoyalty Revenue
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.
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.
We generated no milestone or license revenue during the years ended December 31, 2023 and 2022.
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. We anticipate that our selling, general and administrative expenses will increase in the future as we continue to increase our headcount to support our continued research and development and commercialization of our products.
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 consists primarily of expense related to the interest recognized for capital leases.
Interest expense consists primarily of interest expense related to the purchase and sale agreement with Royalty Pharma Development Funding, LLC, or Royalty Pharma, and our operational and capital leases and in prior years consisted of our term loan, which was paid off in 2022.
Interest income consists of income earned on our cash, cash equivalents and short and long-term investment balances.
Other (expense) income, net includes income (expense), net consistingconsists of the revaluation of foreign currency related to trade payables.
We sell Revuforj to Specialtyspecialty Distributorsdistributors and Specialtyspecialty Pharmaciespharmacies, (or collectively, Customers).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.
ProductNet product revenue
In November 2024, we began to generate product revenue from sales of Revuforj in the United States. We recordNet product revenue netfrom sales of estimatedRevuforj discounts,was chargebacks,$124.8 rebates, product returns,million and other$7.7 gross-to-netmillion revenuefor deductions.the years ended December 31, 2025 and 2024, respectively.
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.
MilestoneMilestone, 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.
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.
Milestone revenue for the fiscal year ended December 31, 2024 includes milestones achieved as part of our collaboration agreement with Incyte of $12.5 million and $3.5 million achieved as part of our licensing agreement with Eddingpharm Investment company limited Our cost of product sales includes the cost of goods sold for Revuforj and royalties associated with sales in the United States.
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.
An increase of $43.8 million in revumenib related costs due to start up activities for a pivotal frontline / combo trial, continuation of trials to expand revumenib use across the mNPM1 and KMT2A acute leukemia landscape, pre-commercial manufacturing activities, and medical affairs activities in preparation for commercialization. In addition, $18.0 million of milestone expense was recognized in 2024, comprising an $8.0 million milestone payment to AbbVie in the first quarter of 2024 for the successful completion of the first pivotal trial in the first indication as well as a $10.0 million milestone payment to AbbVie upon the first commercial sale of Revuforj in the U.S.
An increase of $17.5 million in axatilimab-related costs due to the continuation of the IPF trial, initiation of the co-development combination study of axatilimab and ruxolitinib for the treatment of cGVHD in the frontline, pre-commercial manufacturing activities, and a $15.0 million milestone payment to UCB as a result of the approval of Niktimvo in the third quarter of 2024.
A decreaseincrease in other research and development program related costs of $2.9$1.6 million due to the de-prioritization of programs not pertaining to revumenib and axatilimab.axatilimab in the prior year period.
An increase of $20.2$16.3 million in personnel costs and other expenses, including non-cash stock-based compensation which includes salaries, overhead and related expenses,expenses primarily driven by increasedto support for ongoingon-going clinical trials, preparation of a supplemental New Drug Application, and NDA/sNDAmedical activities.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.
Selling, General and Administrative
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 $30.3$32.8 million in personnel costs and other expenses, including non-cash stock-based compensation, whichrecognition includesof performance-based equity, based on certain commercial achievements. Also included are salaries, overhead and related expenses primarily due to increased headcount to support commercialthe readiness.commercialization of Revuforj and Niktimvo.
An increase of $20.4 million in sales and marketing related expenses due to commercial readiness and launch activities for Revuforj and Niktimvo.
Royalty Interest Expense and Income
For the year endended December 31, 2024,2025, royalty interest income, net of interest expense,expense increased from the comparableprior period. The increase of interest income was primarilyyear due to higher2025 interestbeing ratesthe andfirst anfull increased average balance on cash equivalents and short and long-term investments, partially offset by $4.9 millionyear of accrued interest expense recognized related to the Royalty Pharma Purchase and Sale agreement.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.
For the year ended December 31, 2024,2025, the total other (expense) income, net decreasedincreased from the comparable prior year period primarily due to the decreaseincrease in foreign currency losses on shortshort- and long-term investments.
As of December 31, 2024,2025, we had cash, cash equivalents and short-term and long-term investments totaling $692.4$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 and long-termshort-term investments as of December 31, 2024,2025, 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 and long-termshort-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 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.
Loan and Security Agreement
In February 2020, we entered into a loan and security agreement, with Hercules, as amended in December 2021, which we refer to as the Amended Loan Agreement. We terminated the Amended Loan Agreement in September 2022. On September 23, 2022, we made a prepayment of $21.5 million to satisfy in full all of our principal and interest obligations and related fees under the Amended Loan Agreement. The payoff amount paid by us in connection with the termination of the Amended Loan Agreement was pursuant to a payoff letter with Hercules and included payment of (a) $1.0 million as an end-of term fee and (b) $0.4 million as a pre-payment fee. Hercules released all security interests held on our assets.
For additional details on our Amended Loan Agreement, see "Loan Payable" (Note 15) to our consolidated financial statements in this Annual Report.
In OctoberNovember 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 will beare capped at $822.5 million or 2.35 times the funded amount.
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 long-termNiktimvo investmentscollaboration arerevenue, 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 oftiming, 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 growth rate;
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;
market acceptance of our approved products as well as our product candidates;
the cost of maintaining and expanding sales, marketing and distribution capabilities for our products;
What changed in the latest 10-Q
Risk Factors
New heading “Our indebtedness and liabilities could limit the cash flow available for our operations, expose us to risks that could adversely affect our business, financial condition and results of operations and impair our ability to satisfy our obligations under our indebtedness.”
New heading “We may not have the ability to raise the funds necessary to settle conversions of the 2031 Notes in cash, to repay the 2031 Notes at maturity or to repurchase the 2031 Notes upon a fundamental change, and our future debt may contain limitations on our ability to pay cash upon conversion or repurchase of the 2031 Notes.”
New heading “The conditional conversion feature of the 2031 Notes, if triggered, may adversely affect our financial condition and operating results.”
New heading “Certain provisions in the indenture governing the 2031 Notes may delay or prevent an otherwise beneficial takeover attempt of us.”
New heading “The accounting method for the 2031 Notes could adversely affect our reported financial condition and results.”
Largest changes
“Holders of the 2031 Notes have the right, subject to certain conditions and limited exceptions, to require us to repurchase all or a portion of their Notes upon the occurrence of a fundamental change at a fundamental change repurchase price equal to 100% of the principal amount of the 2031 Notes to be repurchased, plus accrued and unpaid interest, if any. …”see in full comparison
“We may not have the ability to raise the funds necessary to settle conversions of the 2031 Notes in cash, to repay the 2031 Notes at maturity or to repurchase the 2031 Notes upon a fundamental change, and our future debt may contain limitations on our ability to pay cash upon conversion or repurchase of the 2031 Notes.”see in full comparison
“Our indebtedness and liabilities could limit the cash flow available for our operations, expose us to risks that could adversely affect our business, financial condition and results of operations and impair our ability to satisfy our obligations under our indebtedness.”see in full comparison
“The conditional conversion feature of the 2031 Notes, if triggered, may adversely affect our financial condition and operating results.”see in full comparison
“Certain provisions in the indenture governing the 2031 Notes may delay or prevent an otherwise beneficial takeover attempt of us.”see in full comparison
“The accounting method for the 2031 Notes could adversely affect our reported financial condition and results.”see in full comparison
Full comparison: every changed paragraph (21)
In addition to the risk factors listed below and the other information contained elsewhere in this report, you should carefully consider the risks and uncertainties described in “Part I, Item 1A—Risk Factors” of our Annual Report on Form 10-K for the year ended December 31, 2025, or 2025 Form 10-K, filed with the Securities and Exchange Commission, or SEC, on February 26, 2026, which could materially and adversely affect our business, prospects, financial condition and results of operations. New risk factors can emerge from time to time, and it is not possible to predict the impact that any factor or combination of factors may have on our business, prospects, financial condition and results of operations. The risks listed below and described in our 2025 Form 10-K are not our only risks. Additional risks and uncertainties not currently known to us or that we currently deem to be immaterial also may materially adversely affect our business, financial condition or future results. ThereOther than the risk factors listed below, there have been no material changes from the risk factors previously disclosed in our 2025 Form 10-K.
Our indebtedness and liabilities could limit the cash flow available for our operations, expose us to risks that could adversely affect our business, financial condition and results of operations and impair our ability to satisfy our obligations under our indebtedness.
As of June 30, 2026, we had $250.0 million aggregate principal amount of indebtedness under the 2031 Notes. We may also incur additional indebtedness to meet future financing needs. Our indebtedness could have significant negative consequences for our security holders and our business, results of operations and financial condition by, among other things:
limiting our ability to obtain additional financing;
requiring the dedication of a substantial portion of our cash flow from operations to service our indebtedness, which will reduce the amount of cash available for other purposes;
limiting our flexibility to plan for, or react to, changes in our business;
diluting the interests of our existing stockholders as a result of issuing shares of our common stock upon conversion of the 2031 Notes;
encouraging short selling by market participants because the conversion of the 2031 Notes could be used to satisfy short positions thereby depressing the price of our common stock; and placing us at a possible competitive disadvantage with competitors that are less leveraged than us or have better access to capital.
Our ability to make scheduled payments of the principal of, to pay interest on or to refinance our indebtedness, including the 2031 Notes, depends on our future performance, which is subject to economic, financial, competitive and other factors beyond our control. Our business may not generate sufficient funds, and we may otherwise be unable to maintain sufficient cash reserves, to pay amounts due under our indebtedness, including the 2031 Notes, and our cash needs may increase in the future.
We may not have the ability to raise the funds necessary to settle conversions of the 2031 Notes in cash, to repay the 2031 Notes at maturity or to repurchase the 2031 Notes upon a fundamental change, and our future debt may contain limitations on our ability to pay cash upon conversion or repurchase of the 2031 Notes.
Holders of the 2031 Notes have the right, subject to certain conditions and limited exceptions, to require us to repurchase all or a portion of their Notes upon the occurrence of a fundamental change at a fundamental change repurchase price equal to 100% of the principal amount of the 2031 Notes to be repurchased, plus accrued and unpaid interest, if any. In addition, upon any conversion of the 2031 Notes, unless we elect to deliver solely shares of our common stock to settle such conversion (other than paying cash in lieu of delivering any fractional share), we will be required to make cash payments in respect of the 2031 Notes being converted. We may not have enough available cash or be able to obtain financing at the time we are required to make repurchases of the 2031 Notes surrendered therefor or pay cash with respect to the 2031 Notes being converted or being repaid at maturity. In addition, our ability to repurchase the 2031 Notes or to pay cash upon conversion or at maturity may be limited by law, by regulatory authority, or by agreements governing our future indebtedness. Our failure to repurchase the 2031 Notes at a time when the repurchase is required by the indenture governing the 2031 Notes or to pay any cash payable on future conversions of the 2031 Notes or at maturity of the 2031 Notes, as required by the indenture, would constitute a default under the indenture. A default under the indenture or the fundamental change itself could also lead to a default under agreements governing our then-existing indebtedness. If the repayment of the related indebtedness were to be accelerated after any applicable notice or grace periods, we may not have sufficient funds to repay the indebtedness and repurchase the 2031 Notes or make cash payments upon conversions of the 2031 Notes.
The conditional conversion feature of the 2031 Notes, if triggered, may adversely affect our financial condition and operating results.
In the event the conditional conversion feature of the 2031 Notes is triggered, holders of the notes will be entitled to convert their notes at any time during specified periods at their option. If one or more holders elect to convert their Notes, unless we elect to satisfy our conversion obligation by delivering solely shares of our common stock (other than paying cash in lieu of delivering any fractional share), we would be required to settle a portion or all of our conversion obligation in cash, which could adversely affect our liquidity. In addition, even if holders do not elect to convert their notes, we could be required under applicable accounting rules to reclassify all or any portion of the outstanding principal of the notes as a current rather than long-term liability, which would result in a material reduction of our net working capital. Holders of the existing notes have similar conversion rights.
Certain provisions in the indenture governing the 2031 Notes may delay or prevent an otherwise beneficial takeover attempt of us.
Certain provisions in the indenture that governs the 2031 Notes may make it more difficult or expensive for a third party to acquire us. For example, the indenture that governs the 2031 Notes may require us to repurchase the 2031 Notes for cash upon the occurrence of a fundamental change and, in certain circumstances, to increase the conversion rate for a holder that converts its Notes in connection with a make-whole fundamental change. A takeover of us may trigger the requirement that we repurchase the 2031 Notes and/or increase the conversion rate, which could make it costlier for a potential acquirer to engage in such takeover. Such additional costs may have the effect of delaying or preventing a takeover of us that otherwise be beneficial to investors.
The accounting method for the 2031 Notes could adversely affect our reported financial condition and results.
The accounting method for reflecting the 2031 Notes on our consolidated balance sheet, accruing interest expense for the 2031 Notes and reflecting the underlying shares of our common stock in our reported diluted earnings per share may adversely affect our reported earnings and financial condition.
In August 2020, the Financial Accounting Standards Board published Accounting Standards Update (“ASU”) 2020-06 (“ASU 2020-06”), which simplified certain of the accounting standards that apply to convertible notes. In accordance with ASU 2020-06, the 2031 Notes are reflected as a liability on our consolidated balance sheet, with the initial carrying amount equal to the principal amount of the notes, net of issuance costs. Issuance costs are treated as a debt discount for accounting purposes, which are amortized into interest expense over the term of such notes. As a result of this amortization, the interest expense that we expect to recognize for the 2031 Notes for accounting purposes will be greater than the cash interest payments we will pay on the 2031 Notes, which will result in lower reported net income or larger reported net loss.
In addition, the shares of common stock underlying the 2031 Notes are reflected in our diluted earnings per share using the “if converted” method, in accordance with ASU 2020-06, for fiscal periods in which we report net income. Under that method, diluted earnings per share would generally be calculated assuming that all the notes were converted solely into shares of common stock at the beginning of the reporting period, unless the result would be anti-dilutive. The application of the if-converted method may reduce our reported diluted earnings per share to the extent we are profitable in the future.
Furthermore, if any of the conditions to the convertibility of the 2031 Notes is satisfied, then we may be required under applicable accounting standards to reclassify the liability carrying value of such notes as a current, rather than a long-term, liability. This reclassification could be required even if no noteholders convert such notes following the occurrence of those circumstances and could materially reduce our reported working capital.
We cannot be sure whether other changes may be made to the current accounting standards related to the 2031 Notes, or otherwise, that could have a material effect on our reported financial results.
Management's Discussion & Analysis (MD&A)
New heading “Pipeline Assets”
New heading “Convertible Note Interest Expense”
New heading “Convertible Note Interest Expense”
New heading “Convertible Notes”
Largest changes
“In June 2026, we issued $250.0 million aggregate principal amount of 2.25% Convertible Senior Notes, or the 2031 Notes, due 2031 in a private placement to institutional accredited investors that were qualified institutional buyers under Rule 144A in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act. The 2031 Notes are senior unsecured obligations of ours and bear interest at 2.25% per annum, payable semi-annually in arrears on June 15 and December 15, beginning December 15, 2026. …”see in full comparison
“We expect to submit an investigational new drug (IND) application for SNDX-4321 by the end of 2026 and to initiate a Phase 1 trial in EGFRm NSCLC in 2027.”see in full comparison
Full comparison: every changed paragraph (80)
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 slatepipeline of clinicallate- and early-stage 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.
In July 2026, we announced two new pipeline assets, SNDX-4321 and SNDX-62122. SNDX-4321 is a novel, mutant-selective, allosteric epidermal growth factor receptor, or EGFR, inhibitor designed to address non-small cell lung cancer, or NSCLC, patient populations with significant unmet needs. The Company has an exclusive worldwide license to develop and commercialize SNDX-4321. SNDX-62122 is a next-generation menin inhibitor in development for myelofibrosis, or MF. SNDX-62122 is the first molecule from our library of internally developed and wholly owned next-generation menin inhibitors that we intend to advance into new areas. Our SNDX-62122 program builds off recently published revumenib preclinical data showing that menin is a novel dependency in proliferative megakaryocytes, major drivers of MF. We expect the development of SNDX-62122 to be informed and de-risked by a planned Phase 1/2 proof-of-principle trial of revumenib in MF.
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 threesix months ended MarchJune 31,30, 2026 and 2025, we reported a net loss of $42.7$92.0 million and $84.8$156.7 million, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $1.5$1.6 billion. As of MarchJune 31,30, 2026, we had cash, cash equivalents and short-termshort- and long-term investments of $352.1$575.1 millionmillion.
Recent Business Highlights and Anticipated Milestones
Achieved $54.7 million in Revuforj net revenue in the second quarter of 2026, a 91% increase over the second quarter of 2025 and a 12% increase over the first quarter of 2026. Total prescriptions were approximately 1,500 in the second quarter of 2026, an approximate 121% increase over the second quarter of 2025 and a 15% increase over the first quarter of 2026. The net revenue and prescription growth reflects an increasing average treatment duration, primarily driven by a growing pool of patients receiving Revuforj for an extended period in the post-transplant setting.
Advanced our scientific leadership in menin inhibition with the presentation of 16 revumenib abstracts at the 2026 American Society of Clinical Oncology, or ASCO, and the European Hematology Association, or EHA, annual meetings in June 2026. The data presented span multiple acute leukemia subtypes and settings, including frontline and post-transplant maintenance.
Published data from the Phase 1/2 SAVE trial of an all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax in R/R NPM1m, KMT2Ar, and NUP98r AML in the Journal of Clinical Oncology in June 2026. The results showed deep and durable remissions in a heavily pretreated patient population. The overall response rate was 88% (37/42), composite complete remission, or CRc, rate was 71% (30/42), and complete remission plus complete remission with partial hematologic recovery rate was 60% (25/42). 80% of evaluable CRc responders were measurable residual disease, or MRD, negative. 45% (19/42) of patients proceeded to a transplant and 63% (12/19) resumed revumenib post-transplant.
Published preclinical revumenib data showing that menin is a novel dependency in proliferative megakaryocytes, major drivers of MF, in Cancer Cell in July 2026. These data provide the basis for our plans to develop SNDX-62122, an internally developed, next-generation menin inhibitor, for MF.
We expect to have a major presence at upcoming medical meetings in the second half of 2026 with the presentation of new/updated revumenib data including:
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-HSCTReal-world maintenance dataevidence from multiple trials and centers.centers
Post-transplant maintenance data
R/R NUP98-rearranged, or NUP98r, acute leukemia data from patients treated in the AUGMENT-101 trial or via an expanded access program.
R/RFrontline data from the SAVEBEAT AML trial of revumenib in combination with venetoclax/azacitidine in NPM1m and decitabine/cedazuridineKMT2Ar in NPM1m, KMT2Ar, and NUP98r acute leukemia.AML.
Frontline data from the Phase 1/2 SAVE trial of an all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax 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.
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,MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break ThroughCancer,Through Cancer, a collaboration between leading U.S. cancer research centers.
We expect the MenTain Phase 2 trial to initiate around the end of 2026. MenTain will be the first randomized, placebo-controlled trial specifically focused on evaluating revumenib as post-transplant maintenance.
We expect to publish safety and efficacy data from R/R NUP98r acute leukemia patients treated with revumenib in the fourth quarter of 2026.
Achieved $55.1$60.3 million in Niktimvo net revenue in the firstsecond quarter of 2026, representinga significant67% growthincrease compared toover the $13.6second millionquarter inof net2025 revenueand generateda in9% increase over the first quarter of 20252026. fromSyndax theand firstIncyte twoare monthsco-commercializing of the launch.Niktimvo. 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$18.1 million in the firstsecond quarter of 2026.
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.
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. ToplineWe anticipate 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 2028.2026.
CompletedA enrollment in MAXPIRe, apivotal Phase 2, 26-week3, randomized, double-blinded,double-blind, placebo-controlledplacebo-controlled, multicenter trial of axatilimab onin topcombination ofwith standard of carecorticosteroids in patients with≥ IPF12 in the first quarteryears of 2026.age with newly diagnosed chronic GVHD. We expect to reportanticipate topline data in theearly fourth quarter of 2026.2028.
We anticipate topline data from 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 fourth quarter of 2026.
Pipeline Assets
SNDX-4321
In July 2026, we announced the expansion of our pipeline with SNDX-4321, a mutant-selective, CNS-penetrant, allosteric EGFR inhibitor. SNDX-4321 is in development for NSCLC patient populations with significant unmet needs, such as those with L858R mutations, CNS metastases, atypical activating mutations, or acquired resistance to current therapies. In contrast to ATP-site directed third and fourth generation EGFR inhibitors, SNDX-4321 is a novel allosteric inhibitor which binds at a pocket adjacent to the ATP site that is only accessible in the presence of L858R and certain other EGFR mutations.
We expect to submit an investigational new drug (IND) application for SNDX-4321 by the end of 2026 and to initiate a Phase 1 trial in EGFRm NSCLC in 2027.
SNDX-62122
In July 2026, we announced the selection of SNDX-62122, a next-generation menin inhibitor, for development in MF. SNDX-62122 is the first candidate from a library of internally developed, wholly owned next-generation menin inhibitors that we intend to advance into new areas.
We expect to submit an IND and initiate a Phase 1 trial of SNDX-62122 in MF in 2027. The development of SNDX-62122 will be informed by a Phase 1/2 proof-of-principle trial of revumenib in MF that is expected to initiate in the fourth quarter of 2026 with initial clinical data expected in the second half of 2027.
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 lossloss, net” 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.
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 products and product candidates and include:
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 products and 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.
In addition, the probability of success for each drugproduct 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.
Convertible Note Interest Expense
Convertible note interest expense consists of the interest recorded related to the convertible note liability under the effective interest method.
Comparison of the three and six months ended MarchJune 31,30, 2026 and 2025:
In November 2024, we began to generate product revenue from sales of Revuforj in the United States. Product revenue, net from sales of Revuforj increasedfor bythe $28.9three and six months ended June 30, 2026, was $54.7 million and $103.6 million, respectively. The increase in product revenue from the comparable prior year period primarilyend was due to increased sales volume as a result of second indication approval and increased brand awareness.
Collaboration revenue, net, representing our share of net profits in connection with commercialization of Niktimvo, for the three and six months ended MarchJune 31,30, 2026, was $15.9 million. We generated $0.2$18.1 million ofand $34.0 million, respectively. The increase in collaboration lossrevenue forfrom the threecomparable monthsprior endedperiod Marchend 31,was 2025,due to increased sales volume as Niktimvo launched in January 2025, and was not yet in a netresult revenueof position.increased brand awareness.
The following table summarizes the research and development expenses for the three and six months ended MarchJune 31,30, 2026 and 2025:
For the three and six months ended MarchJune 31,30, 2026, our total research and development expenses decreasedincreased by $2.8$5.8 million and $3.0 million from the comparable prior year period.periods, respectively. The changechanges waswere primarily due to:
Axatilimab: A decrease, due to a non-recurring $10.0 million development milestone expense recognized in the first quarter of 2025;
Revumenib: An increase,increase in the three and six months ended June 30, 2026, from the comparable prior year period, due to the initiation of frontline trials evaluating revumenib in combination with standard-of-care agents in the treatment of AML;
Axatilimab: A decrease in the three months ended June 30, 2026, from the comparable prior year period due to timing of trials and a decrease in the six months ended June 30, 2026, from the comparable prior year period due to a non-recurring $10.0 million development milestone expense recognized in the first quarter of 2025;
A decrease in other research and development programs for the three and six months ended June 30, 2026, from the comparable prior year period due to timing of research and development activities;
A decrease in personnel cost and other expenses for the three months ended June 30, 2026, from the comparable prior year period due to lower headcount and an increase for the six months ended June 30, 2026, from the comparable prior year period due to costs related to the on-going support of clinical trials and medical affairs; and An increase in stock-based compensation from the comparable prior year period driven by a higher stock price.
An increase in personnel costs and other expenses to support on-going clinical trials and medical affairs activities in support of Revuforj and Niktimvo.
The following tables summarizes the selling, general and administrative expenses for the three and six months ended MarchJune 31,30, 2026 and 2025:
For the three and six months ended MarchJune 31,30, 2026, our total selling, general and administrative expenses decreased $3.4by $2.3 million and $5.7 million, respectively, from the comparable prior year period.periods. The changechanges waswere 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;
An increase in other selling, general and administrative expenses from the comparable prior year period to further support corporate growth;
A decrease in personnel expenses from the comparable prior year period due to a decrease in overall headcount; and An increase in stock-based compensation for the six months ended June 30, 2026, from the comparable prior year period driven by a higher stock price.
SNDX insider buying and selling (Form 4)
Since 2026-04-11, insiders reported open-market purchases in 0 Form 4 filings and open-market sales in 4 filings (3 insiders, 4 trade dates, 105,914 shares, about $2.1M; 2 of these filings say the sales were made under a Rule 10b5-1 trading plan). Net open-market shares: -105,914 (purchases minus sales); net value about -$2.1M.Totals add up every open-market (code P and S) line in those filings, using the prices reported in the filings. Awards, option exercises, tax withholding and gifts are listed below but not counted.
| Trade date | Insider | Transaction | Shares | Price | Value |
|---|---|---|---|---|---|
| 2026-07-08 | Metzger Michael A |
Open-market sale |
32,375 | $24.24 | $784.8K |
| 2026-07-08 | Metzger Michael A |
Option exercise |
32,375 | $6.38 | $206.6K |
| 2026-06-12 | Botwood Nicholas A.j. |
Option exercise |
31,235 | $13.82 | $431.7K |
| 2026-06-12 | Botwood Nicholas A.j. |
Open-market sale |
31,235 | $18.14 | $566.6K |
| 2026-06-11 | Botwood Nicholas A.j. |
Option exercise |
22,515 | $13.82 | $311.2K |
| 2026-06-11 | Botwood Nicholas A.j. |
Open-market sale |
34,280 | $18.00 | $617.0K |
| 2026-06-01 | Goldan Keith A. |
Open-market sale | 1,177 | $18.99 | $22.4K |
| 2026-06-01 | Metzger Michael A |
Open-market sale | 6,847 | $18.99 | $130.0K |
Well-known investors holding SNDX (13F)
| Investor | Quarter | Shares | Reported value | % of their 13F | Change vs prior quarter |
|---|---|---|---|---|---|
| Point72 Asset Management (Steve Cohen) | 2026-06-30 | 1,985,547 | $46.4M | — | Sold out |
| Millennium Management (Israel Englander) | 2026-06-30 | 2,053,960 | $44.9M | 0.03% | Added 929% |
| Two Sigma Investments | 2026-06-30 | 1,578,125 | $34.5M | 0.03% | No change |
| D. E. Shaw & Co. | 2026-06-30 | 1,239,954 | $27.1M | 0.02% | Reduced 23% |
| AQR Capital Management (Cliff Asness) | 2026-06-30 | 853,304 | $18.7M | 0.01% | Reduced 13% |
| Renaissance Technologies | 2026-06-30 | 321,600 | $7.0M | 0.01% | Reduced 35% |
| Citadel Advisors (Ken Griffin) | 2026-06-30 | 258,278 | $5.6M | 0.0% | Reduced 59% |