Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer - LARVOL DELTA

Innovations in the Treatment of Pediatric AML (ASH 2026) - "Gemtuzumab ozogamicin (GO), an anti-CD33 ADC, has demonstrated improved event-free survival and reduced relapse risk, notably in subgroups with KMT2A rearrangements and high CD33 expression...The session will also highlight strategies being investigated to overcome these challenges, such as multiantigen and logic-gated CAR designs and combination approaches targeting the tumor microenvironment. Attendees will gain insight into ongoing clinical trials, current limitations, and emerging opportunities to advance CAR T-cell therapies for pediatric AML."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Pediatrics • CD123 • FLT3 • IL3RA • KMT2A

Outcomes after Transplant in Relapsed/Refractory KMT2Ar (MLLr) and mNPM1 (NPM1c) leukemia Patients Achieving Remissions after Menin Inhibition: SNDX-5613 (revumenib) Ph1 Experience (ASH 2022) - P1/2 | "1 was among the 12 patients described in Table 1: a 40 yo F with KMT2Ar AML with FLT3 TKD, and 3 prior lines of therapy including 7+3+midostaurin and HSCT...She then had a molecular relapse and received CPX-351, gemtuzumab, venetoclax, and azacytidine, a 3rd allo-HSCT, and due to persistent positive MRD by flow, she received a donor lymphocyte infusion... In SNDX-5613 patients proceeding to transplant, durable remissions occurred across a range of heavily pre-treated patients. In addition to patients achieving CR/CRh, two patients with CRp also had ongoing remissions post-transplant. AUGMENT-101 continues to enroll patients, agnostic of transplant eligibility, with the option for SNDX-5613 post-transplant maintenance."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Septic Shock • Transplantation • FLT3 • KMT2A • NPM1

Clinical Outcomes of Adults With Core Binding Factor Acute Myeloid Leukemia (CBF‑AML): A Single Center Experience (SOHO 2023) - "Other regimens included: cladribine, idarubicin and cytarabine (CLIA, n=1), fludarabine, cytarabine and gemtuzumab ozogamicin (FLAG-GO, n=1), idarubicin and cytarabine (3+7 regimen, n=1) and decitabine, venetoclax and GO (n=1). Although CBF-AML is considered a favorable risk AML, outcomes for relapsed disease remains poor. Incorporation of GO and CD117 tyrosine kinase inhibitors (i.e. dasatinib) into induction and consolidation regimens may mitigate the relapse risk and improves long-term outcomes."

Clinical • Clinical data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KIT

Combination of CPX-351 and Gemtuzumab Ozogamicin (GO) in Relapsed Refractory (R/R) Acute Myeloid Leukemia and Post Hypomethylating Agent (HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS) (ASH 2024) - P2 | "Background : Outcomes of patients (pts) with HR-MDS or AML who are refractory to or progress after HMA ± Venetoclax (Ven) based therapy is dismal, warranting evaluation of newer agents...OS was meaningful in responders, considering multiple prior lines of therapy. Infection related events were the most common serious adverse events."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Septic Shock • CD33 • KMT2A • MECOM • TP53

Updated Results of CPX-351 in Combination with Gemtuzumab Ozogamicin (GO) in Relapsed Refractory (R/R) Acute Myeloid Leukemia (AML) and Post-Hypomethylating Agent (Post-HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS) (ASH 2022) - P2 | "Background: Treatment outcomes in patients (pts) with AML and HR-MDS who progress after HMA +/- venetoclax (VEN) based regimen remains poor and warrants new therapeutic strategies... In a cohort composed predominantly of heavily pre-treated and VEN exposed adverse risk AML and HR-MDS pts, CPX-GO led to overall response (CR/CRh/MLFS/PR) rates of 52% and year-long median survival in responders (CR/CRh/MLFS) with no major non-hematological adverse event."

Combination therapy • Acute Myelogenous Leukemia • Bone Marrow Transplantation • CNS Disorders • Epilepsy • Hematological Malignancies • Infectious Disease • Mucositis • Myelodysplastic Syndrome • Transplantation • CD33 • NPM1

A multi-arm phase Ib/II study designed for rapid, parallel evaluation of novel immunotherapy combinations in relapsed/refractory acute myeloid leukemia. (PubMed, Leuk Lymphoma) - P1/2 | "Overall, 50 patients were enrolled into one of 6 arms: (A) single agent PF-04518600 (OX40 agonist monoclonal antibody), (B) azacitidine + venetoclax + gemtuzumab ozogamicin (GO), (C) azacitidine + avelumab (anti-PD-L1 monoclonal antibody) + GO, (D) azacitidine + venetoclax + avelumab, (E) azacitidine + avelumab + PF-04518600, and (F) glasdegib + GO. This study shows the feasibility of a conducting a multi-arm trial to efficiently and simultaneously evaluate novel therapies in AML, a needed strategy in light of the plethora of emerging therapies. This trial was registered at www.clinicaltrials.gov as NCT03390296."

Journal • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Long-term follow-up of a phase 2 study of all-trans retinoic acid, arsenic trioxide, and gemtuzumab ozogamicin in acute promyelocytic leukemia. (PubMed, Cancer) - P2 | "The combination of ATO-ATRA and GO was curative in 94% of patients who had APL with a favorable safety profile (ClinicalTrials.gov identifier NCT01409161)."

Journal • P2 data • Acute Promyelocytic Leukemia • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Oncology

Relationship Between Salvage Strategy and Outcomes in Patients With CBF or NPM1-mutated AML and First Molecular Relapse. (PubMed, Blood Adv) - "Given the uncertainty regarding the optimal management of molecular relapse in patients with CBF or NPM1-mutated AML, we retrospectively analyzed the outcome of 121 adults from 12 centers with CBF (n=28) or NPM1-mutated (n=93) AML and first molecular relapse according to the salvage strategy used (upfront allogeneic HCT [n=19], intensive chemotherapy [IC; n=21], venetoclax and azacitidine [VEN-AZA]; n=70), and other strategies (n=11; including AZA, gemtuzumab ozogamicin, selective inhibitors). In patients who received allogeneic HCT, type of salvage therapy was not statistically associated with post-HCT relapse, relapse-free survival, or OS. Our data suggests that upfront allogeneic is a valuable option, if feasible, while other salvage strategies are associated with favorable outcomes and relatively low non-relapse mortality after allogeneic HCT."

Journal • Acute Myelogenous Leukemia • Transplantation • NPM1

Safety and efficacy of pracinostat in combination with gemtuzumab ozogamicin (PraGO) in patients with relapsed/refractory acute myeloid leukemia. (PubMed, Leuk Res) - No abstract available

Combination therapy • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

VINCENT: Venetoclax Plus Azacitidine Versus Intensive Chemotherapy for Fit Patients With Newly Diagnosed NPM1 Mutated AML (clinicaltrials.gov) - P2 | N=146 | Recruiting | Sponsor: Technische Universitt Dresden | Trial completion date: Sep 2028 ➔ Sep 2030 | Trial primary completion date: Sep 2028 ➔ Sep 2030

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD33 • NPM1

Early Real-World Outcomes of Revumenib Versus Venetoclax-Based Salvage Therapy in Relapsed or Refractory Acute Leukemia (SOHO 2026) - "All patients had prior AML-directed therapy before index, including hypomethylating agents, venetoclax, cytarabine, anthracyclines, gemtuzumab ozogamicin, FLT3 inhibitors, IDH inhibitors, or glasdegib. In this early real-world matched analysis, revumenib demonstrated 90-day mortality, infection risk, and acute care utilization comparable to venetoclax-based salvage therapy. In evolving AML treatment algorithms, these findings are reassuring: menin inhibition appears to be entering practice without an early excess safety signal relative to a commonly used salvage backbone. The absence of superiority should not imply equivalence but supports revumenib as a reasonable sequencing option in selected patients."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • KMT2A • NPM1

Optimizing Low-Intensity Therapeutic Strategies in Older Acute Myeloid Leukemia: A Systematic Review and Network Meta-Analysis (SOHO 2026) - "Low-intensity regimens, including hypomethylating agents (HMAs) and low-dose cytarabine (LDAC), have transformed management; however, the optimal therapeutic approach remains uncertain...Interventions: Low-intensity regimens including HMAs (azacitidine, decitabine), LDAC, and combination therapies (eg, venetoclax- or glasdegib-based regimens)...LDAC plus glasdegib showed a significant survival advantage over several LDAC-based regimens, including LDAC plus gemtuzumab ozogamicin and vosaroxin... Azacitidine combined with venetoclax appears to be the most effective low-intensity regimen for older AML patients, offering significant improvements in survival and response without substantial additional toxicity. LDAC plus glasdegib represents a promising alternative. Further head-to-head randomized trials are required to refine treatment selection and address resistance mechanisms."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intensive chemotherapy with or without gemtuzumab ozogamicin in patients with NPM1-mutated acute myeloid leukaemia (AMLSG 09-09): a randomised, open-label, multicentre, phase 3 trial. (PubMed, Lancet Haematol) - P3 | "The primary endpoints of the trial of event-free survival and overall survival were not met. However, an anti-leukaemic efficacy of gemtuzumab ozogamicin in participants with NPM1-mutated acute myeloid leukaemia is shown by a significantly lower cumulative incidence of relapse rate, suggesting that the addition of gemtuzumab ozogamicin might reduce the need for salvage therapy in these participants. The results from this study provide further evidence that gemtuzumab ozogamicin should be added in the standard of care treatment in adults with NPM1-mutated acute myeloid leukaemia."

Journal • P3 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Septic Shock • Thrombocytopenia • NPM1

FLAG-Ida Combined with Gemtuzumab Ozogamicin (GO) Improves Event Free Survival in Younger Patients with Newly Diagnosed Acute Myeloid Leukaemia (AML) and Shows an Overall Survival Benefit in NPM1 and FLT3 mutated Subgroups. Results from the UK NCRI AML19 Trial (ASH 2022) - "The MRC AML15 trial suggested a higher response rate and reduced relapse risk with FLAG-Ida compared to a Daunorubicin-araC (DA) +etoposide but did not show an overall survival (OS) benefit (Burnett, JCO,2013,31,3360). Furthermore this survival benefit was associated with a reduction in the requirements for transplant in CR1 and overall. Given the benefit observed in FLT3 mutated AML in the absence of a FLT3 inhibitor, studies combining FLAG-Ida-GO with Midostaurin are warranted."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • FLT3 • NPM1

Safety and Efficacy of Combining Midostaurin and Gemtuzumab Ozogamicin with Induction Chemotherapy in FLT3 mutated AML. (PubMed, Blood Adv) - "We evaluated the safety and efficacy of the combination of daunorubicin, cytarabine (DA), gemtuzumab ozogamicin (GO) and midostaurin (DAGO+m) for younger patients with newly diagnosed FLT3mut AML in the UK NCRI AML19 trial...DAGO2+m will now be evaluated in a randomised study (OPTIMISE-FLT3, ISRCTN 34016918). Trial: ISRCTN78449203."

Journal • Acute Myelogenous Leukemia • Transplantation • FLT3 • NPM1

Sinusoidal Obstruction Syndrome in Children with CD22+ B-Cell Precursors Acute Lymphoblastic Leukemia (BCP-ALL) Treated with Inotuzumab Ozogamicin in Trial ITCC-059: Risk Factors and Outcomes (ASH 2024) - P2 | "In phase IA, phase II and stratum III, patients received single agent InO; while in phase IB InO was combined with vincristine and dexamethasone...Overall, 14 (13%) patients developed SOS, 2 during treatment, 10 after HSCT, and 2 after subsequent chemotherapy including high-dose methotrexate and cyclophosphamide...All SOS cases received treatment with defibrotide, except one patient in which SOS resolved with supportive management...The time between last InO and HSCT seems a significant risk factor for developing SOS, as in previous studies in adults treated with gemtuzumab ozogamicin. Taking into account also the longer median half-life of InO in pediatrics (T1/2 423 h,17.6 days; Trial NCT02981628), a minimum interval of 35 days after last InO dose and HSCT could be considered based on these data."

Clinical • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia • Oncology • Pediatrics • Septic Shock • CD22

Integrated analysis of genomics, molecular responses and outcomes of CBF-AML with FLAG based therapy on a phase 2 trial. (PubMed, Blood Cancer Discov) - P2 | "Fludarabine, cytarabine, and G-CSF-based therapy (FLAG) yields approximately 60% 5-year overall survival (OS) in core-binding factor (CBF) AML, with potential added benefit with gemtuzumab ozogamicin (GO)...We interrogated these factors in 219 frontline patients with CBF-AML (median age 52 years; range 19-80) treated on a phase 2 trial (NCT00801489); 51% received FLAG-GO and 49% FLAG-idarubicin...On multivariate analysis, baseline mutations did not affect OPR or survival, while FLAG-GO favored both. In CBF-AML, FLAG-based therapy possibly attenuates the prognostic impact of concurrent baseline genomics."

Journal • P2 data • Acute Myelogenous Leukemia • ASXL1 • DNMT3A • TET2

Anti-CD33 Antibody-Drug Conjugate (Gemtuzumab ozogamicin) in Adult Acute Myeloid Leukemia: Real Life Data from Iraq. (PubMed, Indian J Hematol Blood Transfus) - "Go served as a target therapy and served as an antibody-based target therapies, attained significant rate of response and improved survival outcomes in both treatment- naïve and those with relapsed/refractory AML patients, that emphasizes its role as part of induction and salvage regimens in parallel enhanced supportive care could further improve outcome. The online version contains supplementary material available at https://doi.org/10.1007/s12288-025-02247-w."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD33

A Randomized Comparison of the Fractionated Versus Single Dose Schedule of Gemtuzumab Ozogamicin at Induction with Determinants of Benefit for Older AML Patients: UK NCRI AML18 Trial Results (ASH 2022) - "Methods The NCRI AML18 trial randomised older AML or high-risk MDS patients (>10% blasts) who were not known to have adverse cytogenetics at trial entry to receive course 1 DA induction (Daunorubicin 60mg/m2 d1, 3, 5, AraC 100mg/m2 bd d1-10) with either GO 3mg/m2 on day 1 (GO1), or fractionated GO on days 1 and 4, maximum 5mg per dose (GO2). Conclusions Compared to single dose GO, the fractionated schedule (GO2) was associated with greater reduction in MRD, and improved overall survival in older adults with non-adverse risk genetics; comparable differences by molecular subtype were observed for MRD response and survival. AML18 trial results suggest that the differential benefit from GO2 for older adults is dependent on delivery of ASCT in order to translate the better leukemia clearance from induction into a significantly higher 5yr survival."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • DNMT3A • IDH1 • IDH2 • NPM1 • TP53

FLAG-IDA COMBINED WITH GEMTUZUMAB OZOGAMICIN (GO) REDUCED MRD LEVELS AND IMPROVED OVERALL SURVIVAL IN NPM1MUT AML INDEPENDENT OF FLT3 AND MRD STATUS, RESULTS FROM THE AML19 TRIAL (EHA 2023) - "FLAG-Ida-GO improved the OS of patients with NPM1 mut AML and reduced the number who were PB PC2 MRD+ve compared to DA-GO. This survival benefit was independent of FLT3 mutation status and was seen in both PB PC2 MRD+ve and MRD-ve patients and was supported by the finding that BM MRD levels in both groups were lower with FLAG-Ida-GO including those testing PB post C2 negative. For those patients who were PB MRD- ve after 2 cycles of FLAG-Ida-GO, this treatment appears sufficient."

Clinical • Acute Myelogenous Leukemia • Transplantation • FLT3 • NPM1

A RANDOMISED ASSESSMENT OF THE SEQUENTIAL ADDITION OF THE KINASE INHIBITOR QUIZARTINIB TO INTENSIVE CHEMOTHERAPY IN OLDER ACUTE MYELOID LEUKAEMIA (AML) PATIENTS: RESULTS FROM THE NCRI AML18 TRIAL (EHA 2023) - "Following recovery from course 1, comprising daunorubicin 60mg/m 2 d1, 3, 5, AraC 100mg/m 2 bd d1-10 with 1 or 2 doses of gemtuzumab ozogamicin, patients were randomised (1:1) to receive Quiz or not irrespective of FLT3 status; those allocated Quiz were additionally randomised (1:1) between short and long therapy. In older AML patients, the sequential addition of Quiz to IC was well-tolerated but did not lead to improved survival. Although not powered to assess benefit, a sub-group analysis of FLT3 -mutated patients showed a non-significant trend to survival benefit consistent with the results of the QuANTUM-First trial (Erba et al, EHA,2022). FLT3, Clinical trial, Tyrosine kinase inhibitor, Acute myeloid leukemia"

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • FLT3 • NPM1

Fludarabine, Cytarabine, Granulocyte Colony-Stimulating Factor, and Idarubicin With Gemtuzumab Ozogamicin Improves Event-Free Survival in Younger Patients With Newly Diagnosed AML and Overall Survival in Patients With NPM1 and FLT3 Mutations. (PubMed, J Clin Oncol) - "Overall, FLAG-Ida + GO significantly reduced relapse without improving OS. However, exploratory analyses show that patients with NPM1 and FLT3 mutations had substantial improvements in OS. By contrast, in patients with core binding factor AML, outcomes were excellent with DA + GO with no FLAG-Ida benefit."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Transplantation • FLT3 • NPM1

Gemtuzumab ozogamicin plus midostaurin in combination with standard '7 + 3' induction therapy in newly diagnosed AML: Results from the SAL-MODULE phase I study. (PubMed, Br J Haematol) - "Three dose levels of midostaurin and one to three sequential doses of 3 mg/m2 GO in combination with '7 + 3' induction were evaluated. Based on safety findings in 12 patients, our results show that 3 mg/m2 GO on Days 1 + 4 and 100 mg midostaurin on Days 8-21 can be safely combined with IC in newly diagnosed AML."

Combination therapy • Journal • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

Gemtuzumab Ozogamicin Added to Fludarabine, Cytarabine and G-CSF (FLAG-GO) Leads to Superior Molecular Response and Survival Outcomes Than Idarubicin (FLAG-IDA): 200 Patients Long-Term Follow up (ASH 2024) - P2 | "Even in pts with EOT OPR, FLAG-GO leads to better RFS and OS than FLAG-IDA. KIT mutation had no independent prognostic impact on survival in our analysis."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KIT

Retrospective comparison of survival and responses to Fludarabine, Cytarabine, GCSF (FLAG) in combination with gemtuzumab ozogamicin (GO) or Idarubicin (IDA) in patients with newly diagnosed core binding factor (CBF) acute myelogenous leukemia: MD Anderson experience in 174 patients. (PubMed, Am J Hematol) - "FLAG-GO regimen was superior in optimal disease specific fusion transcript reduction at end of induction (p=0.002,), mid-consolidation (p<0.01) and end of consolidation (p<0.001) therapy. Induction/consolidation with FLAG-GO regimen results in better clinical outcomes in newly diagnosed patients with CBF-AML compared to FLAG-IDA and achieves deeper molecular clearance by qPCR assessment of the fusion transcripts."

Combination therapy • Journal • Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CSF3