Syndax Pharmaceuticals Inc
For the period ended Mar 31, 2025. Filed May 5, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 5, 2025
SNDX$9.54+1.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-05.
0000950170-24-121439
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-05.
0000950170-25-063518
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 127 previous paragraphs and 143 current paragraphs.
59 unchanged; 36 reworded; 46 added; 32 removed; 0 previous and 2 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (46)
Current report
We are a commercial-stage biopharmaceutical company advancing innovative 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 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 April 2025, we completed the submission of a supplemental New Drug Application, or sNDA, for revumenib as a treatment for R/R acute myeloid leukemia, or AML, with a nucleophosmin 1 mutation, or mNPM1, based on the positive pivotal data from our AUGMENT-101 trial. We are also studying revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring the use of revumenib as a treatment 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, 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.
Current report
Achieved $20.0 million in Revuforj net revenue in the first quarter of 2025, the first full quarter of the U.S. launch. Revuforj was launched in the U.S. in late November 2024 for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older.
Current report
As of the end of March 2025, 44% of the Company’s high priority, Tier 1 and Tier 2, accounts have ordered, up from one-third of accounts as of the end of February 2025. These accounts are the centers of excellence and the medium to large academic institutions which represent two-thirds of the patient opportunity. Among all the accounts that have ordered, two-thirds have ordered multiple times.
Current report
Formulary coverage for Revuforj continues to build. As of the end of March 2025, formal coverage policies were in place for approximately 72% of all managed care lives, which includes commercially covered plus Medicare and Medicaid lives, up from 53% at the end of February 2025.
Current report
Completed the submission of a supplemental New Drug Application (sNDA) to the U.S. FDA in April 2025, seeking Priority Review for the approval of Revuforj for the treatment of R/R mutant NPM1 (mNPM1) AML. The sNDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which allows for a more efficient review and close engagement between the sponsor and FDA throughout the submission process. The sNDA is supported by the previously reported positive pivotal data from the AUGMENT-101 trial.
Current report
Submitted the pivotal R/R mNPM1 AML data from the AUGMENT-101 trial for publication. The manuscript has been accepted and the publication is expected imminently.
Current report
Opened enrollment in the EVOLVE-2 trial, a pivotal, randomized, double-blind, placebo-controlled trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2A-rearranged (KMT2Ar) AML patients who are unfit for intensive chemotherapy in the first quarter of 2025. The trial is being conducted in collaboration with the
Current report
HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.
Current report
The Company plans to initiate multiple trials of revumenib in combination with standard of care regimens in newly diagnosed acute leukemia patients who are fit to receive intensive chemotherapy, starting in the second half of 2025.
Current report
Niktimvo achieved $13.6 million in net revenue in the first quarter of 2025, the first partial quarter of the U.S. launch. Niktimvo was launched in the U.S. in late January for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Syndax and Incyte co-commercialize Niktimvo, and Syndax records 50% of the Niktimvo net profit/loss, defined as net product revenue minus the cost of sales and commercial expenses.
Current report
More than 1,250 infusions of Niktimvo have been administered year-to-date and approximately 95% of top accounts and more than 70% of all bone marrow transplant centers have ordered as of the end of March 2025. A permanent J-code was assigned by CMS effective April 1, 2025.
Current report
Presented a post-hoc analysis evaluating the effects of prior lines of therapy on clinical outcomes for patients with chronic GVHD in the AGAVE-201 trial of axatilimab. The data show that overall response rates were consistent with axatilimab regardless of the number of prior lines of therapy and that organ-specific responses were noted regardless of the last prior therapy. The data were presented at the 2025 Tandem Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood and Marrow Transplantation Research.
Current report
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:
Current report
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multi-center trial of axatilimab in combination with corticosteriods in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
Current report
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, geopolitical tensions, including tariffs, the ongoing wars between Russia and Ukraine and Israel and Hamas as well as other conflicts in the Middle East, including between Israel and Hezbollah, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.
Current report
Product Revenue, net
Current report
Our second 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.
Current report
We generated no product revenue during the three months ended March 31, 2024.
Current report
Collaboration Revenue/Collaboration Loss
Current report
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 products will be presented as “Collaboration revenue” and its 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.
Current report
Royalty Interest Expense
Current report
Royalty interest expense consists of interest expense related to the Purchase and Sale Agreement with Royalty Pharma.
Current report
Other Interest Expense
Current report
Other Expense
Current report
Critical Accounting Policies and Estimates
Current report
Product Revenue, net
Current report
In November 2024, we began to generate product revenue from sales of Revuforj in the United States. We record product revenue net of estimated discounts, chargebacks, rebates, product returns, and other gross-to-net revenue deductions. Product revenue, net from sales of Revuforj was $20.0 million for the three months ended March 31, 2025.
Current report
Collaboration Revenue/Collaboration Loss
Current report
In February 2025, we began to generate sales of Niktimvo in the United States in collaboration with our partner Incyte. Our share of net profits in connection with commercialization of Niktimvo will be presented as “Collaboration revenue” and its 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.
Current report
Cost of Product Sales
Current report
Our cost of product sales includes the cost of goods sold and royalties associated with Revuforj sales in the United States.
Current report
A decrease in revumenib-related costs, primarily driven by an $8.0 million milestone expense in the 2024 period paid to AbbVie and a reduction in CMC expense due to the capitalization of inventory for commercial use, offset by trial expenses to expand revumenib use across the mNPM1 and KMT2a acute leukemia landscape and medical affairs activities supporting the launch of Revuforj.
Current report
An increase in axatilimab-related costs, primarily driven by the IPF trial, the frontline chronic GVHD trial with ruxolitinib being conducted in partnership with Incyte, and a $10.0 million milestone payment to UCB as a result of the first patient dosed in a Phase III study with the compound in combination with another agent for any indication
Current report
Increase in commercial-related costs compared to the prior period, primarily due to the commercialization of Revuforj and Niktimvo
Current report
Royalty Interest Expense
Current report
For the three months ended March 31, 2025, royalty interest expense increased due to the interest expense recognized related to the Royalty Pharma Purchase and Sale Agreement signed in November 2024.
Current report
Other Interest Expense
Current report
For the three months ended March 31, 2025, other interest expense decreased from the comparable period due to less interest expense recognized related to capital leases.
Current report
Interest Income
Current report
For the three months ended March 31, 2025, interest income decreased from the comparable period. The decrease of interest income was primarily due to the fluctuation of interest rates and average balance of cash equivalents and short and long-term investments.
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Removed paragraphs (32)
Previous report
We are a commercial-stage biopharmaceutical company with one commercially approved product and are developing an innovative pipeline of cancer therapies. NIKTIMVO™ (axatilimab-csfr) is our approved product and revumenib is our lead product candidate.
Previous report
We are developing revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, or KMT2Ar, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including acute lymphoblastic leukemia, or ALL, and acute myeloid leukemia, or AML, and nucleophosmin1, also known as NPM1, mutant AML. We are also exploring the use of revumenib as a treatment in solid tumors, specifically its activity in metastatic microsatellite stable, or MSS, colorectal cancer, or CRC. We continue to develop axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 receptor, or CSF-1R, for idiopathic pulmonary fibrosis, or IPF.
Previous report
Our commercially approved product, Niktimvo, received approval on August 14, 2024 from the U.S. Food and Drug Administration, or FDA, in the United States, 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 (88.2 lbs), with a recommended dosage of 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks until progression or unacceptable toxicity. While Incyte leads U.S. commercialization efforts, we are co-promoting Niktimvo with Incyte in the U.S. To facilitate patient dosing and limit product waste, following the FDA’s approval of Niktimvo, we, along with Incyte, have submitted to the FDA for the approval of two smaller vial sizes. We anticipate launching Niktimvo in the U.S. no later than early first quarter 2025. Incyte has exclusive commercialization rights for Niktimvo outside the U.S.
Previous report
The New Drug Application, or NDA, for revumenib, an oral menin inhibitor, for the treatment of adult and pediatric relapsed or refractory, or R/R, KMT2A-r acute leukemia was granted Priority Review and is being reviewed under the U.S. FDA’s Real-Time Oncology Review, or RTOR, Program with a Prescription Drug User Fee Act, or PDUFA, target action date of December 26, 2024.
Previous report
We expect to report topline data from the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin, or mNPM1, AML in the fourth quarter of 2024. Positive data could support a supplemental NDA, or sNDA, filing for revumenib in R/R mNPM1 AML in the first half of 2025.
Previous report
We announced that data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in adult and pediatric patients with R/R KMT2Ar AML and ALL have been published in the Journal of Clinical Oncology.
Previous report
We announced that a larger data set and longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in R/R KMT2Ar acute leukemia will be presented at the upcoming 66th American Society of Hematology, or ASH, Annual Meeting. Analysis of the larger efficacy population (n=97), which includes the 57 patients from the previously reported interim efficacy analysis plus an additional 40 patients. Consistent with previously reported data, the updated analysis shows that revumenib provides durable responses and robust rates of overall response, minimal residual disease, or MRD, negativity, and hematopoietic stem cell transplantation, or HSTC. With seven months of additional follow-up, the median duration of CR/CRh extended to 13 months among the 13 CR/CRh responders included in the interim analysis presented at ASH 2023.
Previous report
We plan to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy by year-end 2024.
Previous report
We announced Niktimvo was added to the latest NCCN Clinical Practice Guidelines in Oncology, or NCCN Guidelines®, as a category 2A recommendation for the treatment of GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Treatments are classified as category 2A when there is uniform NCCN consensus that the intervention is appropriate, based on lower level evidence.
Previous report
We announced that results from the pivotal Phase 2 AGAVE-201 trial of Niktimvo in adult and pediatric patients with recurrent/refractory active cGVHD who had received at least two prior lines of systemic therapy were published in the New England Journal of Medicine.
Previous report
We announced a secondary analysis of overall and organ-specific responses from the pivotal Phase 2 AGAVE-201 trial of Niktimvo in adult and pediatric patients with recurrent/refractory active cGVHD who had received at least two prior lines of systemic therapy will be presented at the 66th ASH Annual Meeting. The data demonstrated rapid responses and symptom improvement in inflammatory and fibrotic manifestations of cGVHD in heavily pretreated patients.
Previous report
Corporate
Previous report
We announced a $350 million royalty funding agreement with Royalty Pharma Development Funding, LLC, or Royalty Pharma, based on U.S. net sales of Niktimvo. Under the agreement, we received $350 million in exchange for a 13.8% royalty on U.S. net sales of Niktimvo. Royalty payments to Royalty Pharma will cease upon reaching a multiple of 2.35x.
Previous report
Revenue
Previous report
Our ability to generate sales revenue depends upon our ability to obtain marketing approval of, and successfully commercialize our product candidates. Our first commercially approved product, Niktimvo, received approval from the FDA in August 2024. We anticipate launching Niktimvo in the U.S. no later than early first quarter 2025. Our revenue for the three and nine months ended September 30, 2024 was derived from the achievement of milestones in connection with our license agreements with Incyte and Eddingpharm Investment Company Limited, or Eddingpharm. We did not generated any product sales revenue during the three and nine months ended September 30, 2024.
Previous report
Interest Expense
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Other Income (Expense), net
Previous report
Critical Accounting Estimates
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Revenue
Previous report
Revenue for the three months ended September 30, 2024, consisted of a $12.5 million milestone earned as a result of the approval of Niktimvo.
Previous report
Revenue for the nine months ended September 30, 2024, consisted of a $12.5 million milestone earned in the third quarter of 2024 as a result of the approval of Niktimvo and $3.5 million of revenue recognized in the second quarter of 2024 under the license agreement with Eddingpharm.
Previous report
The increase in costs related to revumenib for the three and nine month period ended September 30, 2024, compared to the prior year periods, was primarily driven by the continuation of the registrational trials, preparation for front-line / combination trials, companion diagnostic development costs, pre-commercial manufacturing activities, medical affairs activities in preparation for commercialization, and an $8.0 million milestone paid to Vitae in the first quarter of 2024, related to the successful completion of the first pivotal trial in the first indication.
Previous report
The increase in costs related to axatilimab for the three and nine month period ended September 30, 2024, compared to the prior year periods, was primarily driven by the continuation of the IPF trial, 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.
Previous report
The decrease in other research and development programs for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was related to a decrease in clinical trial expense, not related to the revumenib and axatilimab programs.
Previous report
The increase in commercial-related expenses for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was primarily driven by and increase in commercial readiness activities for Niktimvo and revumenib.
Previous report
The increase in other selling, general, and administrative expenses for the three and nine months ended September 30, 2024, compared to the prior year periods, was primarily due to an increase in IT, recruiting, and legal costs.
Previous report
Interest Expense and Interest Income
Previous report
For the three and nine months ended September 30, 2024, interest income, net of interest expense, increased from the comparable prior year period. The increase was primarily due to higher interest rates and an increased average balance on cash equivalents and short and long-term investments.
Previous report
Other Income (Expense)
Previous report
For the three and nine months ended September 30, 2024, other income (expense) decreased from the comparable prior year period primarily due to the decrease in foreign currency losses on short and long-term investments.
Previous report
Since our inception, our operations have been primarily financed by net proceeds from our public stock offerings, and revenue from our license agreements. We believe that our cash, cash equivalents and short- and long-term investments as of September 30, 2024, will fund our projected operating expenses and capital expenditure requirements for at least the next 12 months. In addition, we received a $350.0 million upfront payment in November 2024 pursuant to the purchase and sale agreement, or Royalty Agreement, with Royalty Pharma and are eligible to receive future aggregate payments up to $822.5 million. We are also and are eligible under our collaboration agreements to receive royalty payments, research and development funding, and milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and commercial milestones, which will continue to fund our future operations. 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.
Previous report
our ability, along with Incyte, to successfully launch and generate sales of Niktimvo;
Reworded paragraphs (36)
Previous wording
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 not generated any product revenues from product sales to date. 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 nine months ended September 30, 2024 and 2023, we reported a net loss of $224.6 million and $136.9 million, respectively. As of September 30, 2024, we had an accumulated deficit of $1.1 billion. As of September 30, 2024, we had cash, cash equivalents and short- and long-term investments of $399.6 million.
Current wording
We have begun generating product revenue from sales of Revuforj and collaboration revenue from sales of Niktimvo and 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 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, 2025 and 2024, we reported a net loss of $84.8 million and $72.4 million, respectively. As of March 31, 2025, we had an accumulated deficit of $1.3 billion. As of March 31, 2025, we had cash, cash equivalents and short- and long-term investments of $602.1 million.
Previous wording
Multiple trials evaluating the potential to expand revumenib use across the mNPM1 and KMT2Ar acute leukemia treatment landscape are ongoing. These trials include:
Current wording
Multiple trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape are ongoing. These trials include:
Previous wording
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in front-line AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML® Master Clinical Trial. We presented updated positive data from the trial at the European Hematology Association, or EHA, 2024 Congress, showing a 96% (23 of 24 pts) composite complete remission, or CRc, rate in patients with newly diagnosed mNPM1 or KMT2Ar AML. The BEAT AML trial is expanding to validate the recommended Phase 2 dose of the combination of revumenib with venetoclax and azacitidine. We anticipate that an update on the trial will be available in the fourth quarter of 2024.
Current wording
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. Updated data that showed an overall response rate (ORR) of 100% (37/37) and a composite complete remission (CRc) rate of 95% (35/37) were reported at the Company’s investor event at the 66 th ASH Annual Meeting. The Company anticipates that an update on the trial will be available at a medical meeting in the second quarter of 2025.
Previous wording
SAVE: A Phase 1 trial evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from MD Anderson Cancer Center. We announced that updated data showing an 88% ORR (23 of 26 pts) in R/R patients with KMT2Ar, mNPM1, or NUP98r leukemias will be presented at the upcoming 66th ASH Annual Meeting. In addition to the R/R cohort, a frontline cohort is now enrolling patients.
Current wording
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Updated data that showed an ORR of 82% (27/33) and a CR/CRh rate of 48% (16/33) were presented at the 66 th ASH Annual Meeting. The trial is now enrolling a cohort of newly diagnosed patients.
Previous wording
Intensive chemotherapy: A Phase 1 trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias. The trial is designed to identify the recommended Phase 2 dose for this combination to support further development.
Current wording
Intensive chemotherapy: A Phase 1 trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients. The Company expects to report data in the fourth quarter of 2025.
Previous wording
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. Preliminary results from the first eight mNPM1 patients treated with revumenib will be presented at the upcoming 66th ASH Annual Meeting.
Current wording
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. Data that showed 54% (6/11) of patients had MRD reduction at any time, including 36% (4/11) who achieved MRD negativity, were presented at the 66 th ASH Annual Meeting.
Previous wording
We are evaluating revumenib in patients with R/R metastatic MSS colorectal cancer. The trial is currently enrolling patients in the Phase 1b portion of its Phase 1/2 proof-of-concept trial.
Current wording
The Company is evaluating revumenib in patients with R/R metastatic microsatellite stable (MSS) colorectal cancer (CRC). The Phase 1b portion of this proof-of-concept trial is ongoing.
Previous wording
Our partner, Incyte, is now recruiting patients for 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 (NCT06388564). A Phase 3 trial of axatilimab in combination with steroids for the treatment for chronic GVHD is currently in preparation.
Current wording
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.
Previous wording
Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with IPF, now referred to as the MAXPIRe trial (NCT06132256). We expect to report topline data from the trial in 2026.
Current wording
Enrollment is ongoing in the MAXPIRe trial, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF). The Company expects to complete enrollment in the trial in 2025 with topline data anticipated in the second half of 2026.
Previous wording
We are subject to 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.
Current wording
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 additional indications for our approved products; 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.
Previous wording
Niktimvo received FDA approval 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). We anticipate that Niktimvo will be launched in the U.S. no later than early first quarter 2025. In the U.S., Niktimvo will be co-commercialized by Incyte and us.
Current wording
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).
Previous wording
Research and development activities are central to our business model. Drug 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 drug 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.
Current wording
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.
Previous wording
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 drug 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 product candidates for the period, if any, in which material net cash inflows from these potential drug candidates may commence. Clinical development timelines, the probability of success and development costs can differ materially from expectations.
Current wording
In addition, the probability of success for each product candidate will depend on numerous factors, including competition, manufacturing capability and commercial viability. The successful development of our 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 product candidates for the period, if any, in which material net cash inflows from these potential drug candidates may commence. Clinical development timelines, the probability of success and development costs can differ materially from expectations.
Previous wording
Selling, general and administrative expenses consist primarily of pre-commercialization costs as well as employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, information technology, business development and support functions. 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 further increase in the future as we continue to increase our headcount to support our continued research and development and anticipated commercialization of revumenib. Additionally, we expect an increase in employee-related and sales and marketing expenses and other commercial expenses as a result of Niktimvo’s anticipated commercial launch and in anticipation of revumenib’s expected regulatory approval.
Current wording
Selling, general and administrative expenses consist primarily of commercialization costs as well as employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, information technology, 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 further increase in the future as we continue to increase our headcount to support our continued research and development and anticipated commercialization of our products.
Previous wording
Interest expense consists primarily of interest expense on our operational and capital leases.
Current wording
Other interest expense consists of interest expense related to our operational and capital leases.
Previous wording
Other income (expense), net includes income (expense), consisting of revaluation of foreign currency related to trade payables.
Current wording
Other expense includes expense consisting of revaluation of foreign currency related to trade payables and amortization of debt issuance costs.
Previous wording
For a discussion of new accounting pronouncements please read Note 3 “Summary of Significant Accounting Policies,” to our unaudited condensed consolidated financial statements included in this report.
Current wording
For a discussion of new accounting pronouncements please read Note 3 - Summary of Significant Accounting Policies to our unaudited consolidated financial statements included in this Quarterly Report.
Previous wording
Comparison of the three and nine months ended September 30, 2024 and 2023:
Current wording
Comparison of the three months ended March 31, 2025 and 2024:
Previous wording
The following table summarizes the research and development expenses for the three and nine months ended September 30, 2024 and 2023:
Current wording
The following table summarizes the research and development expenses for the three months ended March 31, 2025 and 2024:
Previous wording
For the three and nine months ended September 30, 2024, our total research and development expenses increased $31.9 million and $68.2 million respectively, from the comparable prior year period. The increase was primarily due to:
Current wording
For the three months ended March 31, 2025, our total research and development expenses increased $5.1 million from the comparable prior year period. The increase was primarily due to:
Previous wording
The increase in personnel costs and other expenses, and stock-based compensation, which includes salaries, overhead, and related expenses, for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was related to support of the on-going clinical trials and NDA/sNDA activities.
Current wording
An increase in personnel costs and other expenses, and stock-based compensation, which includes salaries, overhead and related expenses compared to the prior period, have increased to support on-going clinical trials, sNDA activities and medical affairs in support of commercialization
Previous wording
The following tables summarizes the selling, general and administrative expenses for the three and nine months ended September 30, 2024 and 2023:
Current wording
The following tables summarizes the selling, general and administrative expenses for the three months ended March 31, 2025 and 2024:
Previous wording
For the three and nine months ended September 30, 2024, our total selling, general and administrative expenses increased $13.8 million and $39.0 million, respectively, from the comparable prior year period. The increase primarily is due to:
Current wording
For the three months ended March 31, 2025, our total selling, general and administrative expenses increased $18.0 million, from the comparable prior year period. The increase primarily is due to:
Previous wording
The increase in personnel costs and other expenses and stock-based compensation, which includes salaries, overhead, and related expenses, for the three and nine months ended September 30, 2024, compared to the prior year periods, was primarily due to increased headcount in preparation for commercial readiness activities.
Current wording
An increase in personnel cost and other expenses, and stock-based compensation, which includes salaries, overhead and related expenses, compared to the prior period, was primarily due to increased headcount to support the commercialization of Revuforj and Niktimvo
Previous wording
Net cash used in operating activities increased from $119.3 million for the nine months ended September 30, 2023 to $217.5 million for the nine months ended September 30, 2024, primarily due to an increase in operating net loss, prepaid expenses, and collaboration receivables, and a decrease in accounts payable.
Current wording
Net cash used in operating activities increased from $83.5 million for the three months ended March 31, 2024 to $95.2 million for the three months ended March 31, 2025, primarily due to an increase in operating net loss, accounts receivable, and inventory, and decreases in prepaid expenses, and collaboration payables.
Previous wording
Net cash provided by investing activities for the nine months ended September 30, 2024, was $49.2 million and was related to $229.9 million from the maturities of available-for-sale securities, offset by the purchase of $180.7 million of available-for-sale securities.
Current wording
Net cash provided by investing activities for the three months ended March 31, 2025, was $94.1 million and was related to $104.7 million from the maturities of available-for-sale securities, offset by the purchase of $10.5 million of available-for-sale securities.
Previous wording
Net cash provided by investing activities for the nine months ended September 30, 2023, was $133.9 million and was related to $359.2 million from the maturities of available-for-sale securities, offset by the purchase of $225.3 million of available-for-sale securities.
Current wording
Net cash used in investing activities for the three months ended March 31, 2024, was $99.4 million and was related to $68.0 million from the maturities of available-for-sale securities, offset by the purchase of $167.4 million of available-for-sale securities.
Previous wording
Net cash provided by financing activities for the nine months ended September 30, 2024, increased by $0.3 million from the comparable prior year period primarily due to proceeds from the exercise of stock options and employee participation in our 2015 Employee Stock Purchase Plan, or ESPP.
Current wording
Net cash provided by financing activities for the three months ended March 31, 2025, decreased by $1.2 million from the comparable prior year period primarily due to a decrease in proceeds from the exercise of stock options in the current period.
Previous wording
Additionally, the process of testing drug candidates in clinical trials is costly, and the timing of progress 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:
Current wording
Additionally, the process of testing product candidates in clinical trials is costly, and the timing of progress 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.
Previous wording
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;
Current wording
the outcome, timing and cost of seeking and obtaining 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;
Previous wording
the cost of establishing sales, marketing and distribution capabilities for our products and product candidates, including Niktimvo, which we are co-commercializing with Incyte, and revumenib, provided it receives regulatory approval and if we determine to commercialize it ourselves;
Current wording
the cost of maintaining sales, marketing and distribution capabilities for our products;
Previous wording
the cost of disruptions 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.
Current wording
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;
Previous wording
We have one commercially approved product, Niktimvo, which we anticipate launching in the U.S. no later than early first quarter 2025. We otherwise have no products approved for commercial sale and have not generated any product revenues from product sales to date. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings and additional funding from license and collaboration arrangements. Except for payments received under the Royalty Agreement or 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 contractual obligations and commitments as of September 30, 2024, primarily relate to our maturities of operating leases for office space and equipment and capital leases for office equipment. As of September 30, 2024, we have $0.8 million payable within 12 months.
Current wording
Although we have two FDA-approved products available for commercial sale in the U.S., we have not generated substantial product revenue to date. We believe that the combination of our available cash, cash equivalents, short-term and long-term investments as well as our Revuforj gross contribution and Niktimvo collaboration revenue is sufficient to fund our near-term existing and planned cash requirements. Until we generate substantial product revenue, we expect to finance our future cash needs through a combination of 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.
Previous wording
Except as disclosed above, we have no material non-cancelable purchase commitments with service providers, as we have generally contracted on a cancelable, purchase-order basis. We enter into contracts in the normal course of business with equipment and reagent vendors, CROs, CMOs and other third parties for clinical trials, preclinical research studies and testing and manufacturing services. These contracts are cancelable by us upon prior notice. Payments due upon cancellation consist only of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation. These payments are not determinable.
Current wording
Except as disclosed above, we have no material non-cancelable purchase commitments with service providers, as we have generally contracted on a cancelable, purchase-order basis. We enter into contracts in the normal course of business with equipment and reagent vendors, CROs, contract manufacturing organizations, and other third parties for clinical trials, preclinical research studies and testing and manufacturing services. These contracts are cancelable by us upon prior notice. Payments due upon cancellation consist only of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation. These payments are not determinable.
Previous wording
We have incurred losses and cumulative negative cash flows from operations since our inception, excluding the year ended December 31, 2021. As of September 30, 2024, we had an accumulated deficit of $1.1 billion. We anticipate that we will likely continue to incur significant losses for at least the next couple of years. We expect that our research and development and selling, general and administrative expenses will continue to increase. As a result, we will need additional capital to fund our operations, which we may raise through a combination of the sale of equity, debt financings, or other sources, including potential collaborations. 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.
Current wording
We have incurred losses and cumulative negative cash flows from operations since our inception, excluding the year ended December 31, 2021. As of March 31, 2025, we had an accumulated deficit of $1.3 billion. We anticipate that we will likely continue to incur significant losses for at least the next couple years. We expect that our research and development and selling, general and administrative expenses will continue to increase. Although we have two FDA-approved products available for commercial sale in the U.S., we have not generated substantial product revenue to date. We believe that the combination of our available cash, cash equivalents, short-term and long-term investments as well as our Revuforj gross contribution and Niktimvo collaboration revenue is sufficient to fund our near-term existing and planned cash requirements. Until we generate substantial product revenue, we expect to finance our future cash needs through a combination of equity offerings, debt financings and additional funding from license and collaboration arrangements. 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 product 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 product candidates that we would otherwise prefer to develop and market ourselves.
Previous wording
In May 2023, we entered into a sales agreement with Cowen and Company, LLC, or 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 at-the-market 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. 333-277424), which became automatically effective upon filing on February 27, 2024. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three and nine months ended September 30, 2024, we did not sell any shares of common stock under the 2023 ATM Program. As of September 30, 2024, we had $157.9 million available under the 2023 ATM Program.
Current wording
In May 2023, we entered into a sales agreement with Cowen and Company, LLC, or 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 at-the-market 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. 333-277424), which became automatically effective upon filing on February 27, 2024. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three months ended March 31, 2025, we did not sell any shares of common stock under the 2023 ATM Program. As of March 31, 2025, we had $157.9 million available under the 2023 ATM Program.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
Our future capital requirements will depend on many factors, including:
Current report — wording carried from elsewhere
Our material contractual obligations and commitments as of March 31, 2025, primarily relate to our maturities of operating leases for office space and equipment and capital leases for office equipment. As of March 31, 2025, we have $0.9 million payable within 12 months.