A Randomized Phase II Trial of ASTX727 With Standard Duration Versus Shorter Duration of Venetoclax in Genomically Heterogenous AML Among Adults Aged 60 or Older and Less Fit for Intensive Therapy: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial compares ASTX727 with standard duration versus shorter duration of venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML). ASTX727 is a combination of decitabine and cedazuridine. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Shorter duration venetoclax may be as effective as standard duration venetoclax when given with ASTX727 for the treatment of newly diagnosed AML.

Start Date14 Oct 2026

Sponsor / Collaborator

National Cancer Institute

NCT07012044

/ RecruitingPhase 1IIT

A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727, NSC# 820631) and Filgrastim as Maintenance Therapy Post-Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Filgrastim stimulates the production of neutrophils (a type of white blood cell) which can help to prevent infection. Giving ATSX727 and filgrastim may be safe and tolerable in treating children with high risk, recurrent or refractory acute myeloid leukemia who have undergone allogenic hematopoietic stem cell transplantation.

Start Date09 Jun 2026

Sponsor / Collaborator

National Cancer Institute

NCT07472322

/ Not yet recruitingPhase 1/2IIT

UW26001 Phase I/II Study on the Safety, Tolerability, and Preliminary Efficacy of ASTX727 (Decitabine/Cedazuridine) and Retifanlimab-dlwr in Patients With Advanced Merkel Cell Carcinoma Who Have Progressed on Anti-PD-(L)1 Inhibitor

The goal of this clinical trial is to learn if ASTX727 can be combined with retifanlimab to treat Merkel cell cancer. It will also learn about the safety of combining these drugs. The main questions it aims to answer are:
* Can the combination shrink cancer and lower the chance of the cancer growing or spreading?
* Is the combination better than standard of care for Merkel cell cancer?
Participants will:
* Take oral ASTX727 and retifanlimab through a vein in the arm for about 2 years.
* Visit the clinic once every 2 weeks for checkups and tests

Start Date01 Jun 2026

Sponsor / Collaborator

University of Wisconsin-Madison

100 Clinical Results associated with Decitabine/Cedazuridine

100 Translational Medicine associated with Decitabine/Cedazuridine

100 Patents (Medical) associated with Decitabine/Cedazuridine

Literatures (Medical) associated with Decitabine/Cedazuridine

15 Apr 2026·CANCER

A phase 1 study of ASTX727 plus talazoparib in patients with triple‐negative or hormone resistant/HER2‐negative metastatic breast cancer

Article

Author: Emily Douglas MD ; Erin Conder MS, CCRP ; Kathy D. Miller MD ; Sandra Althouse MS ; Kenneth P. Nephew PhD ; Feyruz V. Rassool PhD ; Ryan Burgos BS, MBA ; H. Josh Jang PhD ; Bryan P. Schneider MD ; Woonbok Chung PhD ; Alexandra Thomas MD ; Tarah Ballinger MD ; Yong Zang PhD ; Katherine Ansley MD ; Jean-Pierre Issa PhD

Abstract:

Background:

Poly(adenosine diphosphate ribose) polymerase (PARP) is recruited to DNA damage sites along with epigenetic factors such as DNA methyltransferase 1 (DNMT1). Inhibitors of DNMT modulate reactive oxygen species (ROS)‐cyclic adenosine monophosphate (cAMP)/Protein Kinase A signaling and induce a “BRCAness phenotype” that further sensitizes cells to PARPi. In preclinical studies, combined DNMTi + PARPi therapy was effective in both triple‐negative (TNBC) and hormone resistant (HRBC) models with intact BRCA.

Methods:

The authors conducted a phase 1 study combining the oral DNMTi ASTX727 with the PARPi talazoparib in patients with previously treated TNBC or HRBC. Patients with deleterious mutations of BRCA were excluded. A classical 3+3 design guided dose escalation/de‐escalation, and 28 days constituted each cycle. Serial peripheral blood mononuclear cells (PBMCs) were analyzed for changes in methylation using the Infinium Methylation EPIC BeadChip and LINE1 sequencing.

Results:

Thirty‐four evaluable patients were enrolled and treated in eight dose cohorts. Myelosuppression was common with grade >3 neutropenia in 42% and grade 3 anemia and thrombocytopenia in 13%. Dose‐limiting toxicity was limited to neutropenia. Efficacy was assessed in 29 patients. There were no objective responses, six patients had stable disease persisting for >4 months in three patients. LINE1 demethylation ranged from ∼2%–10% and immune‐specific CpGs (methylation in immune cells) changed 1%–5% at day 15. Methylation changes were not dose‐dependent.

Conclusions:

ASTX727 plus talazoparib produces significant myelosuppression without other adverse events. Modest methylation changes in PBMCs were detected. There were no objective responses, but some heavily pretreated patients had stable disease for >4 months despite the attenuated doses.

10 Mar 2026·Blood Advances

Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AML

Article

Author: Stone, Richard M. ; DeAngelo, Daniel J. ; Fiorilla, Jade ; Auriemma, Elise ; Murdock, H. Moses ; Fell, Geoffrey ; Kelkar, Amar H. ; Panaro, Kevin M. ; Antin, Joseph H. ; Lindsley, R. Coleman ; Garcia, Jacqueline S. ; Cutler, Corey S. ; Ryan, Jeremy ; Soiffer, Robert J. ; Shapiro, Roman ; Gooptu, Mahasweta ; Lim, Felicia ; Brock, Jennifer ; Bosch-Vilaseca, Anna ; Bat-Erdene, Denbaa ; Ritz, Jerome ; Abel, Gregory A. ; Letai, Anthony ; Kim, Haesook T. ; Ho, Vincent T.

Abstract:

To improve the tolerability of posttransplant maintenance and outcomes despite poor-risk disease genetics, we conducted a phase 1 study of venetoclax (Ven) with FluBu2 (fludarabine/busulfan) reduced-intensity chemotherapy transplantation using tacrolimus/methotrexate graft-versus-host disease (GVHD) prophylaxis, followed by all-oral Ven/decitabine-cedazuridine (Ven/Dec-c) maintenance in patients with poor-risk MDS/AML (myelodysplastic syndrome/acute myeloid leukemia; N = 30). Overall, 58% had previous Ven exposure and 63% had TP53 mutation; 15/19 had TP53 multihit state. At a median of +55 days, prophylactic maintenance therapy with Ven (400 mg on days 1-14) and Dec-c (35 mg decitabine and 100 mg cedazuridine tablet on days 1, 3, and 5, or days 1, 2, and 3) was initiated for 8 cycles of 42 days each in 26 of 30 patients (87%; of the remaining patients, 3 relapsed early and 1 withdrew). On maintenance, grade 3/4 neutropenia (96%) occurred although infections were rare (n = 2). No dose-limiting toxicities occurred. The 6-month acute GVHD grade 2 to 4 rate was 13%. The 1-year moderate/severe chronic GVHD rate was 31%. At a median follow-up of 25.1 months (range, 15-33), median overall survival (OS) and progression-free survival (PFS) were not reached. On maintenance, 2-year OS was 77%, PFS was 62%, nonrelapse mortality was 0%, and cumulative incidence of relapse was 38%. Exploratory studies identified 96% of patients had pretransplant next-generation sequencing molecular residual disease (MRD) positivity, favorable survival in those with non-TP53 MRD positivity, and delayed conversion on maintenance in 11 of 18 (61%) with TP53-MRD positivity. Patient-reported outcomes assessed in the first 6 months of maintenance were stable except for emotional function, which significantly improved. This trial was registered at www.ClinicalTrials.gov as NCT03613532.

10 Feb 2026·Blood Advances

Decitabine-cedazuridine in patients with MDS and TP53 mutations

Article

Author: Urrutia, Samuel ; Griffiths, Elizabeth A. ; Garcia-Manero, Guillermo ; Sano, Yuri ; Savona, Michael ; Bataller, Alex ; McCloskey, James ; Sasaki, Koji ; Keer, Harold N. ; Oganesian, Aram ; Montalban-Bravo, Guillermo ; Kantarjian, Hagop ; Yee, Karen ; Zeidan, Amer

Patients with myelodysplastic syndromes (MDS) harboring TP53 mutations have poor outcomes. The objective of this study is to evaluate patients with TP53 mutations treated in the phase 2/3 studies of decitabine-cedazuridine (DEC-C) in MDS. We divided patients into three groups: TP53wt, TP53single-hit, and TP53multi-hit. We then performed propensity matching of patients who were treated with DEC-C vs a cohort of patients treated with parenteral hypomethylating agents (HMA). 180 patients were analyzed of which 73 (40.5%) had TP53 mutations, 23 (12%) with TP53multi-hit. Patients with TP53multi-hit more frequently had complex cytogenetics (69.5%), and had fewer median number of co-mutations (2.5, IQR 2-3) compared to TP53single-hit(3, IQR 1-5), or TP53wt (4, IQR 3-6) (p=0.002). Patients with TP53multi-hit had a higher chance of lack of response with 39.1% vs 28.1% for TP53single-hit (p=0.2). Patients with TP53mult-hit lost response earlier at 8.2 months, vs 13.2 months for TP53single-hit, and 15.1 months for TP53wt (p=0.1). Median overall survival (mOS) was 11.5 months (95% CI: 8.6 - 19.1) for TP53mult-hit, 22.1 months (95% CI: 14.6 - 35.9) for TP53single-hit, and 31.7 months (95% CI: 19.5 - 51.1) for TP53wt (log-rank, p < 0.005). Propensity scores matched 47 TP53mutpatients treated with DEC-C and 47 TP53mut patients treated with single agent parenteral HMA. Median survival was 13.1 months (95% CI: 8.4 - 21.3) for DEC-C vs 8.0 months (95% CI: 5.2 - 13.0) for single agent parenteral HMA (log-rank, p=0.047). In patients with MDS harboring TP53mut, DEC-C may improve overall survival compared to parenteral HMA.

News (Medical) associated with Decitabine/Cedazuridine

11 Jun 2026

·www.biospace.com

Syndax Showcases Revuforj® (revumenib) Clinical Activity in Multiple Acute Leukemia Subtypes and Settings at EHA 2026

– Real-world study of revumenib shows 82% (9/11) ORR and 64% (7/11) CR/CRh rate among R/R NPM1m or KMT2Ar patients treated with revumenib monotherapy or combinations – – Ph 1 trial of revumenib with intensive chemotherapy in newly diagnosed NPM1m or KMT2Ar AML shows 97% (34/35) CRc and 86% (25/29) MRD negativity among CRc responders – – Post-hoc analysis of patients with R/R NPM1m, KMT2Ar, or NUP98r acute leukemia in the AUGMENT-101 trial who resumed revumenib post-HSCT shows an observed 1-year OS of 95% – – Phase 1 trial and expanded access experience shows 28% (7/25) ORR in R/R NUP98r acute leukemia, a subtype with a poor prognosis and high unmet need – – Ph 2 SAVE trial of revumenib with venetoclax and decitabine/cedazuridine in R/R NPM1m, KMT2Ar, or NUP98r AML shows 88% (37/42) ORR, 68% (25/37) MRD negativity among responders, and 45% (19/42) transplant rate – NEW YORK, June 11, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today highlighted key Revuforj ® (revumenib) data presented at the European Hematology Association (EHA) 2026 Congress in Stockholm, Sweden. In total, 12 abstracts focused on revumenib, the Company’s first-in-class, FDA-approved menin inhibitor, were accepted for EHA 2026. The data presented span multiple acute leukemia subtypes and settings, including new real-world, post-transplant, and combination data from the relapsed/refractory (R/R) and frontline setting. “Collectively, the new data presented at EHA showcase the robust activity observed with revumenib across multiple acute leukemia subtypes and settings, highlighting the potential for revumenib to transform patient outcomes,” said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. “In particular, we are encouraged to observe deep and durable remissions among patients with NPM1m, KMT2Ar, or NUP98r acute leukemia who received revumenib in combination with standard of care therapies in clinical trials or the real-world. The breadth of data presented at EHA highlights physicians’ enthusiasm for revumenib and bolsters our conviction that our ongoing pivotal frontline combination trials are positioned to deliver paradigm-changing results.” Overview of key revumenib data presented at EHA 2026: Abstract title: Revumenib in the real world: interim findings from the ROAR study in relapsed/refractory acute leukemia Abstract #: PF542 At the first interim analysis from ROAR, a multicenter real-world study of revumenib, 11 heavily pretreated patients with R/R NPM1 mutated (NPM1m) or KMT2A-rearranged (KMT2Ar) acute leukemia (median of 2 prior lines of therapy) were response evaluable. Among this population, 64% (7/11) received revumenib monotherapy and 36% (4/11) received revumenib combination therapy. 82% (9/11) overall response rate 1 (ORR) and 64% (7/11) complete remission plus complete remission with partial hematological recovery (CR/CRh) rate among the response-evaluable population. 36% (4/11) of the response-evaluable population proceeded to a hematopoietic stem cell transplant (HSCT), of which 50% (2/4) had resumed revumenib post-HSCT at the data cut off. Revumenib was generally well-tolerated, with real-world safety findings consistent with the established safety pro revumenib. There were no events of differentiation syndrome (DS) or QTc prolongation above Grade 3 and no patient discontinued revumenib due to DS or QTc. Abstract title: Revumenib + intensive chemotherapy for newly diagnosed acute myeloid leukemia harboring genetic alterations in KMT2A, NPM1, or NUP98: updated phase 1 results from SNDX-5613-0708 Abstract #: PF489 97% (34/35) composite complete remission (CRc) and 77% (27/35) complete remission (CR) rate observed among newly diagnosed patients with NPM1m or KMT2Ar AML who received revumenib at dose level (DL) 1 or 2 in combination with intensive chemotherapy. DL1 was revumenib 110 or 220 mg q12hr with/without strong CYP3A4i. DL2 was the FDA-approved monotherapy dose of 160 or 270 mg q12hr with/without strong CYP3A4i. Response rates were similar across both dose levels. 86% (25/29) measurable residual disease (MRD) negativity among those with CRc and 87% (20/23) MRD negativity among those with CR, as determined by local testing. Durability data are still maturing. At the data cutoff, the median follow-up was 4.8 months and 2.9 months in DL1 and DL2, respectively. 60% (9/15) of patients in DL1 and 10% (2/20) in DL2 had proceeded to HSCT as of the data cutoff (this difference may be due to a higher proportion of patients with KMT2Ar and longer follow-up in DL1 than DL2); of the patients in DL1 who proceeded to HSCT, 67% (6/9) resumed revumenib post-HSCT. Safety pro broadly consistent across both revumenib dose levels and comparable with intensive chemotherapy alone. There were no treatment-emergent adverse events (TEAEs) of DS or QTc prolongation above Grade 3. No clinically meaningful pruritus was reported. Abstract Title: Revumenib therapy post hematopoietic stem cell transplant for patients with relapsed/refractory KMT2Ar, NPM1m, and NUP98r acute myeloid leukemia: post hoc analysis of outcomes from AUGMENT-101 Abstract #: PS1631 Post-hoc analysis included 19 heavily pretreated adults and children with R/R NPM1m, KMT2Ar, or NUP98r acute leukemia who resumed revumenib post-HSCT in the multicenter AUGMENT-101 trial. Median time from HSCT to resuming revumenib was 65 (range: 34-181) days. The median duration of revumenib therapy post-HSCT was 20 (range: 2-137) weeks at the data cutoff. Three patients remained on therapy, including two patients who had received more than two years of revumenib post-HSCT. Median overall survival (OS) and median relapse-free survival (RFS) were not reached, regardless of genetic subtype. The estimated 12-month OS and RFS rates were 95% and 79%, respectively. Safety pro consistent with the established safety pro revumenib. Treatment emergent adverse events leading to revumenib discontinuation were low at 5% of patients. No patients discontinued treatment due to cytopenias. These results build on data presented at EHA 2026 and ASCO 2026 from a pooled analysis of 24 adults and children who resumed revumenib post-transplant at a single-center. Abstract title: Clinical activity of revumenib in patients with relapsed/refractory NUP98-rearranged acute leukemias Abstract #: PS1607 28% (7/25) ORR observed among heavily pretreated adults and children with R/R NUP98r acute leukemia (median of 3 prior lines of therapy) who received revumenib in the Phase 1 portion of the AUGMENT-101 trial or via an expanded access program. The median duration of response was 6.7 months. 43% (3/7) of responders proceeded to HSCT and all resumed revumenib post-HSCT. Safety pro consistent with the established safety pro revumenib. No TEAEs led to treatment discontinuation. Abstract Title: Phase 1/2 study of the all-oral combination of revumenib (SNDX-5613) with decitabine/cedazuridine (ASTX727) and venetoclax (SAVE) in relapsed/refractory AML Abstract #: PF495 Results from the R/R cohort enrolled in the SAVE trial were recently published in the Journal of Clinical Oncology and simultaneously presented at EHA 2026. Key results include: 88% (37/42) ORR, 71% (30/42) CRc, and 60% CR/CRh (25/42) observed among heavily pretreated patients with R/R NPM1m, KMT2Ar, or NUP98r AML (median of two prior lines of therapy). 45% (19/42) of patients proceeded to HSCT, of which 63% (12/19) had resumed revumenib as post-HSCT maintenance at the data cutoff. The rates of MRD negativity by flow cytometry were 68% (25/37) among evaluable responders, 80% (24/30) among those with CRc, and 80% (20/25) among those with CR/CRh. With a median follow-up of 22 months, the median duration of response in patients with CR/CRh was 10.7 months among NPM1m patients, not reached among KMT2Ar patients, and 5.9 months among NUP98r patients. Revumenib was generally well-tolerated in combination with decitabine/cedazuridine and venetoclax. The most common TEAEs were consistent with the established safety pro revumenib and the combination partners. About Revuforj ® (revumenib) Revuforj (revumenib) is the first and only menin inhibitor that is FDA approved for the treatment of adult and pediatric patients one year and older with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test. 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. 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 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 . Please see Full Prescribing Information , including BOXED WARNINGS. About Syndax Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Highlights of the Company's pipeline include Revuforj ®  (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit  or follow the Company on  X  and  LinkedIn . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's or Niktimvo’s commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. References Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR; Composite complete remission (CRc) includes CR, CRh, CRp, and CRi. CR = Complete remission CRh = Complete remission with partial hematologic recovery CRp = Complete remission with incomplete platelet recovery CRi = Complete remission with incomplete count recovery MLFS = Morphologic leukemia-free state PR = Partial response Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. sklahre@syndax.com Tel 781.684.9827 SNDX-G

04 Jun 2026

·www.biospace.com

Taiho Oncology Announces New England Journal of Medicine Publication of First All-Oral Regimen in Newly Diagnosed Acute Myeloid Leukemia

Publication highlights positive results for the first all-oral decitabine-cedazuridine plus venetoclax regimen in newly diagnosed acute myeloid leukemia patients who are ineligible for intensive induction chemotherapy. PRINCETON, N.J.--(BUSINESS WIRE)--Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, today announced the publication in the New England Journal of Medicine of results from the ASCERTAIN-V Phase 1/2 clinical trial, which evaluated the first all-oral regimen of decitabine-cedazuridine plus venetoclax in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The study demonstrated favorable response rates and survival with expected myelosuppressive effects. 1 Previously presented at the 2025 ASCO Annual Meeting and the 2025 European Hematology Association Congress, the results suggest this first all-oral regimen may provide outcomes consistent with existing hypomethylating agent–venetoclax–based approaches for patients with newly diagnosed AML who cannot receive intensive induction therapy. Nearly 23,000 people in the U.S. are expected to be diagnosed with AML, a cancer of the blood and bone marrow, in 2026. 2 More than half of those patients will likely be deemed ineligible for intensive induction chemotherapy because of factors such as older age (75 or older) or poor health. 3 For these patients, the standard treatment often combines oral venetoclax with parenteral hypomethylating agents, requiring frequent injections or infusions and an average of 12.9 days per month engaging in healthcare visits. 4 Oral decitabine-cedazuridine is a hypomethylating agent that has demonstrated oral bioavailability and equivalent pharmacokinetic exposure to intravenous decitabine. 5 “We are committed to advancing therapies that improve the lives of patients, their families and their caregivers,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “An all-oral regimen for AML could help reduce the significant time and cost burden associated with treatment in hospitals or infusion centers.” The ASCERTAIN-V trial evaluated the first all-oral regimen of oral decitabine-cedazuridine plus venetoclax in 189 patients (Phase 1, n=30; Phase 2a, n=58; Phase 2b, n=101). After an initial ramp-up period, patients were treated in 28-day cycles, receiving decitabine-cedazuridine on days one through five and venetoclax daily. Key results include complete response rates, durable remissions and survival, and a safety pro with hypomethylating agent-venetoclax regimens. In the Phase 2b of ASCERTAIN-V: The complete response rate was achieved in 47% of patients (95% CI: 36-57%). The composite of complete response or complete response with incomplete hematologic recovery was 63% (95% CI: 53-73%). The median time to marrow blasts of less than 5% was 28 days (range 22 to 57), and the median time to a complete response or complete response with incomplete hematologic recovery was 35 days (range 27 to 58). Among patients achieving complete response, 80% (95% CI: 64%, 90%) had an ongoing response at six months and 75% (95% CI: 57%, 87%) at 12 months. Median overall survival was 15.5 months (95% CI 7.6-not estimable). The 30- and 60-day mortality rates were 3% and 10%, respectively. The median follow-up period was 11.2 months. Serious adverse events were reported in 84% of patients, consistent with known hypomethylating agent–venetoclax regimens. Common related Grade ≥3 adverse events were anemia (30%), neutropenia (26%) and febrile neutropenia (25%). No drug–drug interactions were observed between decitabine-cedazuridine and venetoclax. In the Phase 2b of ASCERTAIN-V, decitabine-cedazuridine and venetoclax dosing was modified once leukemic blast clearance was achieved as measured by bone marrow morphology assessments. Serious adverse events and febrile neutropenia decreased as venetoclax dosing days decreased with each treatment cycle. This information potentially supports the need to explore a treatment strategy of an initial induction followed by dose-adjusting ongoing cycles to enhance tolerability and minimize cytopenia-related complications in patients who have reached remission. “The positive results of the trial — response rates, safety, pharmacokinetics and tolerability data — suggest that an all-oral regimen could potentially become a meaningful alternative to existing treatment protocols that involve parenteral hypomethylating agents for patients with AML who can’t undergo intensive induction chemotherapy,” said Gail J. Roboz, M.D., the paper’s lead author, a professor of medicine, director of the Clinical and Translational Leukemia Program and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a hematologist oncologist at NewYork-Presbyterian/Weill Cornell Medical Center in New York City. “An all-oral treatment regimen is a highly anticipated addition to current treatment options for patients with AML.” About Decitabine and Cedazuridine Fixed-Dose Combination This product is an orally administered, fixed dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine, 6 an inhibitor of cytidine deaminase. 7 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over five days. About Taiho Oncology, Inc. The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development and commercialization of orally administered anti-cancer agents for various tumor types. Taiho Oncology has a robust pipeline of small-molecule clinical candidates targeting solid-tumor and hematological malignancies, with additional candidates in pre-clinical development. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company’s European and Canadian operations, which are located in Baar, Switzerland and Oakville, Ontario, Canada. For more information, visit , and follow us on LinkedIn and X. Taiho Oncology and the Taiho Oncology logo are registered trademarks of Taiho Pharmaceutical Co., Ltd. References Roboz G, Zeidan A, Mannis G, Montesinos P, Arnan M, Savona M, et al. All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia. Published June 3, 2026. N Engl J Med 2026;394:2107-2116. Vol 394 No. 21. American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised January 13, 2026. Accessed February 4, 2026. Heuser M, Fernandez C, Hauch O, Klibanov OM, Chaudhary T, Rives V. Therapies for acute myeloid leukemia in patients ineligible for standard induction chemotherapy: a systematic review. Future Oncol . 2022;19(11):789-810. . Zeidan A, Zhao R, Voegel A, Christensen D, Zhdanava M, Tardif-Samson A, Vanden Eynde C, Keer H, Pilon D. Time burden of treatment with parenteral hypomethylating agents in combination with venetoclax among patients with newly diagnosed Acute Myeloid Leukemia. [ASH Abstract]. Blood (2025) 146 (Supplement 1): 6990. Garcia-Manero G, McCloskey J, Griffiths EA, et al. Oral decitabine-cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study. Lancet Haematol 2024;11:e15-e26. (23)00338-1/abstract Oganesian A, Redkar S, Taverna P, Choy G, Joshi-Hangal R, Azab M. Preclinical data in cynomolgus (cyn) monkeys of ASTX727, a novel oral hypomethylating agent (HMA) composed of low-dose oral decitabine combined with a novel cytidine deaminase inhibitor (CDAi) E7727 [ASH Abstract]. Blood 2013;122(21): Abstract 2526. Ferraris D, Duvall B, Delahanty G, Mistry B, Alt, J, Rojas C, et al. Design, synthesis, and pharmacological evaluation of fluorinated tetrahydrouridine derivatives as inhibitors of cytidine deaminase. J Med Chem 2014; 57:2582-2588. Contacts Taiho Oncology Contact: Leigh Labrie +1 609.664.9878 llabrie@taihooncology.com

Clinical ResultPhase 3Phase 2

15 May 2026

·endpoints.news

Aardvark's next move after clinical hold; Alumis' verdict on Acelyrin asset

Plus, news about Tenaya’s TN-401 and Taiho’s Inqovi. 📉 Aardvark to unblind rare disease trial following clinical hold: The FDA has placed a full clinical hold on the company’s candidate for Prader-Willi syndrome, called ARD-101. Aardvark plans to unblind all studies of the drug, including the Phase 3 HERO trial, and assess data before deciding on next steps. The company previously paused enrollment in the late-stage study voluntarily. Aardvark’s share price $AARD was down about 25% when the market opened Friday. — Ayisha Sharma 🥫 Alumis finally makes decision on Acelyrin asset: A year after merging with Acelyrin, Alumis has decided to can a thyroid eye disease asset from the California biotech. Alumis will look for “strategic alternatives” for lonigutamab following a review that lasted about a year. The ValenzaBio-derived candidate had mixed Phase 2 data at the beginning of 2025, a month before Acelyrin and Alumis announced their marriage . — Kyle LaHucik ❤️‍🩹 Tenaya’s early-stage gene therapy data: The California biotech said its treatment, called TN-401, produced “dramatic” improvements in electrical stability in a rare heart condition. Six patients with arrhythmogenic right ventricular cardiomyopathy enrolled across two dose cohorts saw reductions in premature ventricular contractions ranging from 31% to 84%, Tenaya reported. — Ayisha Sharma 🟢 FDA approves Taiho’s Inqovi for certain AML patients: The agency greenlit a combination of Inqovi plus AbbVie and Roche’s Venclexta for newly-diagnosed patients with acute myeloid leukemia who are 75 years or older, or ineligible for intensive induction chemotherapy. Inqovi is already approved for certain patients with myelodysplastic syndromes and chronic myelomonocytic leukemia. — Ayisha Sharma

100 Deals associated with Decitabine/Cedazuridine

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KEGG	Wiki	ATC	Drug Bank
-	Decitabine/Cedazuridine	L01XY	DB15694

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Acute Myeloid Leukemia	European Union	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Iceland	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Liechtenstein	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Norway	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Chronic Myelomonocytic Leukemia	Australia	Otsuka Australia Pharmaceutical Pty Ltd.	29 Oct 2020
Myelodysplastic Syndromes	United States	Taiho Oncology, Inc.	07 Jul 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Anemia, Refractory	Phase 3	United States	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Austria	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Canada	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Czechia	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	France	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Germany	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Hungary	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Italy	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Spain	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	United Kingdom	Astex Pharmaceuticals, Inc.	15 Feb 2018

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASCO2026	Not Applicable	Acute Myeloid Leukemia First line	466	Decitabine/Cedazuridine + Venetoclax	cvtadzbdsu(azkiexnuuw) = wdovcajvik fkcgjeburn (frabsyyqrw ) View more	Positive	29 May 2026
				Azacitidine + Venetoclax	cvtadzbdsu(azkiexnuuw) = ebksijwmub fkcgjeburn (frabsyyqrw ) View more
EHA2026	Phase 2	Acute Myeloid Leukemia	68	ASTX727+Venetoclax (de novo AML)	nnfgzejehx(hpevhyutdb) = fipsnpoqwh lavmbzoejd (txifqvvuns ) View more	Positive	12 May 2026
				ASTX727+Venetoclax (secondary AML (sAML))	nnfgzejehx(hpevhyutdb) = bwygcllilp lavmbzoejd (txifqvvuns ) View more
NCT05360160 (EHA2026)	Phase 1/2	Refractory acute myeloid leukemia KMT2A rearrangement \| NPM1 mutation \| NUP98 rearrangement	42	Revumenib+ASTX727+Venetoclax	tjbkmzuned(usecpjxlwo) = The most common all-grade adverse events were nausea (60%), vomiting (52%), QTc prolongation (50%), and hypokalemia (45%), mostly grade 1-2. Grade ≥3 events were most frequently febrile neutropenia (35%), thrombocytopenia (22%), lung infection (20%), and respiratory failure (15%). QTc prolongation grade ≥3 occurred in 2 pts (5%). Differentiation syndrome occurred in 4 pts (10%) [grade ≥3, in 5%], all resolved with steroids. elrxqgyakq (kbgbsahils ) View more	Positive	12 May 2026
				Revumenib+ASTX727+Venetoclax (KMT2A rearrangement)
NIBIT-ML1 (AACR2026)	Phase 2	Metastatic melanoma	42	ASTX727 plus I+N	exkudnnrvm(fulbykrhff) = cfugepxgks hekzmoxfql (dptaosiaqg, 5.0 - 13.8) View more	Positive	21 Apr 2026
				I+N	exkudnnrvm(fulbykrhff) = entknbaost hekzmoxfql (dptaosiaqg, 5.0 - 6.6) View more
NCT05010772 (ASH2025)	Phase 1	Acute Myeloid Leukemia Maintenance	35	ASTX727 35/100 mg PO	kgryaewsjs(mbeuxnpjnh) = xhizjwqogn fbqacpsikz (pzivvfmbpn ) View more	Positive	06 Dec 2025
				ASTX727 35/100 mg PO + venetoclax 400 mg (azole adjusted) PO	kgryaewsjs(mbeuxnpjnh) = lvzoymycvc fbqacpsikz (pzivvfmbpn ) View more
NCT06661915 (ASH2025)	Phase 2	Myeloproliferative Disorders	50	ASTX727 + iadademstat	chllihszbm(lrnphpkpqz) = ngvbunnpyd uyhtbcsmga (uzjpslnrjw )	Positive	06 Dec 2025
				ASTX727	chllihszbm(lrnphpkpqz) = vxapuzxudp uyhtbcsmga (uzjpslnrjw )
ISRCTN30808508 (AMMO, ASH2025)	Phase 2	Myeloproliferative Disorders	77	ASTX727	lcvjjczpms(nkzhkqfxdy) = zomlmyobcf iglrtlkdcx (ulvlisvxml, 39.3 - 66.3) View more	Positive	06 Dec 2025
				Hydroxycarbamide/Best Supportive Care	lcvjjczpms(nkzhkqfxdy) = cfmshinwcw iglrtlkdcx (ulvlisvxml, 14.9 - 49.4) View more
NCT06802146 (ASH2025)	Phase 1	CCUS Clonal Cytopenia of Undetermined Significance	138	DEC/CED	zhhfynugqn(efeafaajxp) = kjiilirked qdcjlipdad (esedctnhez ) View more	Positive	06 Dec 2025
NCT04655755 (ASH2025)	Phase 1/2	High Risk Myelodysplastic Syndrome \| Chronic Myelomonocytic Leukemia First line	69	Oral decitabine/cedazuridine + venetoclax	qxdtkldrnk(bslwjramme) = Patients with ASXL1mut had a median OS not reached (vs 18 months in patients with ASXL1wt, P=0.008) tnbpjrhnhp (gnhznlndiz ) View more	Positive	06 Dec 2025
NCT05360160 (SAVE, ASH2025)	Phase 2	Acute Myeloid Leukemia NPM1 mutation \| KMT2A rearrangement	17	Decitabine/cedazuridine + venetoclax + revumenib	zsxpdatygk(sowfyzqlwt) = QTc prolongation occurred in 8 (47%) pts; 3 were grade 2 (18%), the rest were grade 1 (29%) ugrfuhvyub (gtqsotzjzy ) View more	Positive	06 Dec 2025

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