On November 3, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that 23 abstracts, including six oral presentations, showcasing compelling Revuforj (revumenib) and Niktimvo (axatilimab-csfr) data were accepted for presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in Orlando, Florida, December 6-9, 2025.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"The breadth of the upcoming data presentations reflects the tremendous promise that Revuforj and Niktimvo hold across the treatment continuum for acute leukemia and chronic GVHD, respectively," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "In particular, we are excited to present new frontline datasets showcasing the tolerability of Revuforj in combination with standard of care therapies along with high rates of complete remission and MRD negativity, as well as the first real-world evidence for a menin inhibitor, and a retrospective review of usage in the post-transplant setting. We also look forward to the presentation of new data that highlight the potential for Niktimvo to provide long-term benefits in chronic GVHD and the feasibility of combining with ruxolitinib in newly diagnosed chronic GVHD."
The Company will host an in-person investor event, along with a live webcast, at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 8, 2025, at 7:00 a.m. ET to discuss key data presented at the meeting. The live webcast will be available on the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.
Key abstracts accepted for presentation at ASH (Free ASH Whitepaper) 2025:
Revumenib:
An oral presentation will highlight results from a cohort of newly diagnosed patients in the Phase 2 SAVE trial of revumenib in combination with venetoclax and decitabine/cedazuridine in NPM1 mutated (NPM1m), KMT2A-rearranged (KMT2Ar), or NUP98-rearranged (NUP98r) acute myeloid leukemia (AML).
An oral presentation will report efficacy and safety by leukemia type (AML, ALL, or MPAL) in patients with R/R KMT2Ar acute leukemia in the Phase 2 portion of the pivotal AUGMENT-101 trial.
A poster presentation will highlight the first real-world experience with revumenib outside of a clinical trial setting, including in patients with KMT2Ar, NPM1m, or NUP98r acute leukemia.
Two poster presentations will report preliminary results from Phase 1 trials of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1m, KMT2Ar, or NUP98r AML.
A poster presentation will highlight results from a retrospective review of pediatric patients with KMT2Ar, NUP98r, or NPM1m acute leukemia who received revumenib as a maintenance therapy following hematopoietic stem cell transplantation (HSCT).
Axatilimab:
An oral presentation will describe the safety and feasibility observed among patients with recurrent or refractory chronic graft-versus-host disease (GVHD) who transitioned from 0.3 mg/kg every 2 weeks dosing of axatilimab (FDA-approved dose) to 0.6 mg/kg every 4 weeks in the pivotal Phase 2 AGAVE-201 trial.
A poster presentation will highlight the long-term duration of therapy and safety of axatilimab among patients with recurrent or refractory chronic GVHD in the pivotal Phase 2 AGAVE-201 trial.
A poster presentation will report an interim safety analysis from a Phase 2 trial of axatilimab in combination with ruxolitinib in patients with newly diagnosed chronic GVHD.
The accepted abstracts listed below are now available online at the ASH (Free ASH Whitepaper) conference website. Copies of the oral and poster presentations will be made available in the ‘Publications & Meetings Presentations’ section of the Syndax website after the relevant embargoes lift.
Full list of abstracts accepted for presentation at ASH (Free ASH Whitepaper) 2025 (all times in ET):
Revumenib
Abstract Titles Presentation Details
Phase II study of the all-oral combination of revumenib (SNDX-5613) with decitabine/cedazuridine (ASTX727) and venetoclax (SAVE) in newly diagnosed AML Oral presentation
Abstract #: 47
Saturday, December 6
Session: 9:30-11:00 am
Revumenib for patients with relapsed or refractory (R/R) KMT2Ar acute leukemia: Outcomes by leukemia type in the Phase 2 AUGMENT-101 study Oral presentation
Abstract #: 1001
Monday, December 8
Session: 4:30-6:00 pm
Early real-world experience with revumenib outside of a clinical trial setting: A single center retrospective review of efficacy and tolerability Poster presentation
Abstract #: 3448
Sunday, December 7
Session: 6:00-8:00 pm
Phase 1 study of revumenib in combination with intensive chemotherapy (IC) in patients (pts) with newly diagnosed (ND) acute myeloid leukemia (AML) harboring genetic alterations in KMT2A, NPM1, or NUP98: SNDX-5613-0708 Poster presentation
Abstract #: 3425
Sunday, December 7
Session: 6:00-8:00 pm
Revumenib in combination with intensive induction and consolidation for newly diagnosed patients with NPM1-mutated or KMT2A-rearranged acute myeloid leukemia: Preliminary results from the Phase 1b ETCTN 10596 study Poster presentation
Abstract #: 5206
Monday, December 8
Session: 6:00-8:00 pm
Revumenib for patients with relapsed or refractory (R/R) nucleophosmin 1–mutated (NPM1m) acute myeloid leukemia (AML): Outcomes by prior treatment in the Phase 2 AUGMENT-101 study Poster presentation
Abstract #: 3418
Sunday, December 7
Session: 6:00-8:00 pm
Post-transplant maintenance with revumenib in children with HOX pathway-mutated AML Poster presentation
Abstract #: 3461
Sunday, December 7
Session: 6:00-8:00 pm
Trial in progress: A multicenter Phase I trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after allogeneic hematopoietic cell transplant in patients with KMT2A-rearranged or NPM1-mutated acute leukemia Poster presentation
Abstract #: 5207
Monday, December 8
Session: 6:00-8:00 pm
Preliminary results of a Phase 1 study of the safety and tolerability of the combination of revumenib (REV) with gilteritinib (GILT) in relapsed/ refractory (R/R) acute myeloid leukemia (AML) Poster presentation
Abstract #: 3427
Sunday, December 7
Session: 6:00-8:00pm
Real-world treatment patterns and outcomes among patients with newly diagnosed NPM1-mutated acute myeloid leukemia in the United States Poster presentation
Abstract #: 3385
Sunday, December 7
Session: 6:00-8:00 pm
Menin inhibition as a new therapeutic option for the myeloproliferative neoplasms Oral presentation
Abstract #: 67
Saturday, December 6
Session: 9:30-11:00 am
Co-targeting menin and RAS in KMT2A-r/NPM1c AML with activated RTK//RAS/MAPK signaling Poster presentation
Abstract #: 5060
Monday, December 8
Session: 6:00-8:00 pm
Axatilimab
Abstract Titles Presentation Details
Safety and feasibility of 0.6 mg/kg every 4 weeks dosing of axatilimab in patients treated in the AGAVE-201 study Oral presentation
Abstract #: 272
Saturday, December 6
Session: 2:00-3:30 pm
Long-term treatment duration and safety of axatilimab among patients with chronic graft-versus-host disease in AGAVE-201 Poster presentation
Abstract #: 6010
Monday, December 8
Session: 6:00-8:00 pm
Axatilimab in combination with ruxolitinib in patients with newly diagnosed chronic graft-versus-host disease: Interim safety analysis of a randomized, Phase 2 study Poster presentation
Abstract #: 6012
Monday, December 8
Session: 6:00-8:00 pm
CSF-1R+ macrophages orchestrate human cutaneous chronic graft-versus-host disease Oral presentation
Abstract #: 588
Sunday, December 7
Session: 12:00-1:30 pm
Safety analysis of axatilimab in patients with chronic graft-versus-host disease in an expanded access program Poster presentation
Abstract #: 6008
Monday, December 8
Session: 6:00-8:00 pm
Trial in progress: A Phase 3, randomized, double-blind, placebo-controlled study of axatilimab and corticosteroids as initial treatment for moderate to severe chronic graft-versus-host disease Poster presentation
Abstract #: 4256
Sunday, December 7
Session: 6:00-8:00 pm
Pharmacodynamic analysis of AGAVE-201 indicates changes in CSF-1R-expressing cells and associated biomarkers potentially contributing to chronic graft-versus-host disease resolution Poster presentation
Abstract#: 2458
Saturday, December 6
Session: 5:30-7:30pm
Clinical and disease characteristics of initial participants at time of enrollment in THRIVE, a prospective, observational cohort study of patients at risk for chronic graft versus host disease Poster presentation
Abstract#: 2446
Saturday, December 6
Session: 5:30-7:30pm
CSF-1R inhibition and lenalidomide synergize to promote myeloma control after autologous stem cell transplantation Oral presentation
Abstract #: 689
Sunday, December 7
Session: 4:30-6:00 pm
CSF1R-CSF1 axis blockade with axatilimab effectively targets leukemia stem cells and monocytes in AML resistant to BH3 mimetics Poster presentation
Abstract #: 3276
Sunday, December 7
Session: 6:00-8:00 pm
Phase 1b/2 study of axatilimab in combination with azacitidine in advanced phase MPN, MDS/MPN overlap and high-risk CMML Poster presentation
Abstract #: 5607
Monday, December 8
Session: 6:00-8:00 pm
About Revuforj (revumenib)
Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.
Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.
Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.
About Niktimvo (axatilimab-csfr)
Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).
In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.
Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).
Niktimvo is a trademark of Incyte.
All other trademarks are the property of their respective owners.
Revuforj (revumenib)
IMPORTANT SAFETY INFORMATION
WARNING: DIFFERENTIATION SYNDROME, QTc PROLONGATION, and TORSADES DE POINTES
Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.
QTc prolongation and Torsades de Pointes have occurred in patients receiving Revuforj. Correct hypokalemia and hypomagnesemia prior to and during treatment. Do not initiate Revuforj in patients with QTcF > 450 msec. If QTc interval prolongation occurs, interrupt, reduce, or permanently discontinue Revuforj.
WARNINGS AND PRECAUTIONS
Differentiation Syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, rash, and/or hypotension.
In clinical trials, DS occurred in 60 (25%) of 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia. Among those with a KMT2A translocation, DS occurred in 33% of patients with acute myeloid leukemia (AML), 33% of patients with mixed-phenotype acute leukemia (MPAL), and 9% of patients with acute lymphoblastic leukemia (ALL); DS occurred in 18% of patients with NPM1m AML. DS was Grade 3 or 4 in 12% of patients and fatal in 2 patients. The median time to initial onset was 9 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.
Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours in adults or dexamethasone 0.25 mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.
QTc Interval Prolongation and Torsades de Pointes: Revuforj can cause QT (QTc) interval prolongation and Torsades de Pointes.
Of the 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia in clinical trials, QTc interval prolongation was reported as an adverse reaction in 86 (36%) patients. QTc interval prolongation was Grade 3 in 15% and Grade 4 in 2%. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 10%, and the increase from baseline QTcF was greater than 60 msec in 24%. Revuforj dose reduction was required for 7% due to QTc interval prolongation. QTc prolongation occurred in 21% of the 34 patients less than 17 years old, 35% of the 146 patients 17 years to less than 65 years old, and 46% of the 61 patients 65 years or older. One patient had a fatal outcome of cardiac arrest, and one patient had non-sustained Torsades de Pointes.
Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to and throughout treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.
Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia
Embryo-Fetal Toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.
ADVERSE REACTIONS
Fatal adverse reactions occurred in 9 (4%) patients who received Revuforj, including 4 with sudden death, 2 with differentiation syndrome, 2 with hemorrhage, and 1 with cardiac arrest.
Serious adverse reactions were reported in 184 (76%) patients. The most frequent serious adverse reactions (≥10%) were infection (29%), febrile neutropenia (20%), bacterial infection (15%), differentiation syndrome (13%), and hemorrhage (11%).
The most common adverse reactions (≥20%) including laboratory abnormalities, were phosphate increased (51%), hemorrhage (48%), nausea (48%), infection without identified pathogen (46%), aspartate aminotransferase increased (44%), alanine aminotransferase increased (40%), creatinine increased (38%), musculoskeletal pain (37%), febrile neutropenia (37%), electrocardiogram QT prolonged (36%), potassium decreased (34%), parathyroid hormone intact increased (34%), alkaline phosphatase increased (33%), diarrhea (29%), bacterial infection (27%), triglycerides increased (27%), phosphate decreased (25%), differentiation syndrome (25%), fatigue (24%), edema (24%), viral infection (23%), decreased appetite (20%), and constipation (20%).
DRUG INTERACTIONS
Drug interactions can occur when Revuforj is concomitantly used with:
Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec
SPECIFIC POPULATIONS
Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.
Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.
Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.
Pediatric: monitor bone growth and development in pediatric patients.
Geriatric: no overall differences were observed in the effectiveness of Revuforj between patients who were 65 years and older, and younger patients. Compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.
To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see Full Prescribing Information, including BOXED WARNINGS.
Niktimvo (axatilimab-csfr)
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
Niktimvo (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.
Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.
Embryo-Fetal Toxicity
Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.
The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.
Clinically relevant adverse reactions in <10% of patients who received Niktimvo included:
Eye disorders: periorbital edema
Skin and subcutaneous skin disorders: pruritus
Vascular disorders: hypertension
Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions
Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.
USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.
Females and Males of Reproductive Potential
Pregnancy Testing
Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.
Contraception
Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.
DOSAGE AND ADMINISTRATION
Dosage Modifications for Adverse Reactions
Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.
(Press release, Syndax, NOV 3, 2025, View Source [SID1234659281])