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

Impact of granulocyte-colony stimulating factor use on the incidence of febrile neutropenia in acute myeloid leukemia patients undergoing high dose cytarabine consolidation therapy (HOPA 2026) - "These risks may be even higher when patients receive certain concomitant tyrosine kinase inhibitors (TKI) and gemtuzumab ozogamicin (GO). A total of 41 patients were included in the initial analysis, however; only 33 patients met inclusion criteria for data analysis. The duration of neutropenia appeared to persist longer across all cycles in patients who did not receive G-CSF vs. those who received G-CSF."

Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Over 800! Establishment of an ADC payload efficacy database across diverse tumor cell lines: Foundation for optimized in vitro ADC evaluation and drug development (AACR 2026) - "Ever since the first antibody-drug conjugate (ADC), Mylotarg, received approval back in 2000, the ADC market has expanded to include 15 approved products so far, while more than 210 others are currently in clinical trials...Up to now, we have already finished over 700 commonly used cell lines using several ADC payloads, such as MMAE, SN38, Dxd, and exatecan. As a large-scale initiative, the ADC screening platform is expected to play a positive and supportive role in the screening and evaluation of ADC drugs."

ADC • Preclinical • Tumor cell • Oncology • CLDN18 • EGFR • HER-2 • NECTIN4

A novel CD33 and TRAIL-R2 targeting bispecific antibody to treat relapsed/refractory acute myeloid leukemia (AACR 2026) - "Furthermore, current therapies such as hypomethylating agents (HMA) and venetoclax (Ven) fail in patients with TP53 mutations...Safety was assessed ex vivo using human liver microtissue and immune cells.TRIO-863 showed potent CD33-dependent killing of AML cells including high risk TP53-mutant and drug-resistant models, with over 3700-fold greater potency than Mylotarg...This approach addresses major limitations of current treatments. Clinical trials are planned for relapsed/refractory AML, including TP53-mutant and drug-resistant cases."

Bispecific • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD33 • CD34 • MCL1 • TNFRSF10B

MHC class II engager immunotherapy for the treatment of acute myeloid leukemia (AACR 2026) - "However, while ~90% of AML cases express CD33, 50% of these patients harbor a single nucleotide polymorphism (SNP) that eliminates the antibody binding epitope for existing CD33-targeted antibody therapeutics such as gemtuzumab ozogamicin (Mylotarg®)... These experiments demonstrate the potential of LTI-214 in AML and emphasize the importance and targetability of MHCII in cancer immunotherapy."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD4 • CD8 • HLA-DRB1 • PD-L1

Antibody-drug conjugates for the treatment of hematologic malignancies (AACR 2026) - "Belantamab mafodotin is composed of a monoclonal antibody targeting BCMA and is conjugated to the cytotoxic payload monomethyl auristatin F (MMAF), a microtubule disrupting agent, and is approved for multiple myeloma...Gemtuzumab oxogamicin targets the CD33 receptor on myeloid cells and is approved for AML...Inotuzumab ozogamicin targets the CD22 receptor on B-cell precursor leukemic cells and is approved for ALL in pediatric patients with R/R CD22-positive B-cell precursor ALL...Brentuximab vedotin is composed of a monoclonal antibody targeting the CD20 antigen and conjugated to microtubule disrupting agent monomethyl auristatin, which serves as the payload, for chronic Hodgkin's lymphoma...mPFS was 4.2 months (95% CI: 2.9, 7.1) with BV+R2 and 2.6 months (95% CI: 1.4, 3.1) with Pbo+R2 (HR 0.53, 95% CI: 0.38, 0.73). The ORR was 64.3% (95% CI: 54.7, 73.1) and 41.5% (95% CI: 32.5, 51.0), respectively."

ADC • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Multiple Myeloma • Oncology • CD22 • CD33

GSPT1 degrader antibody conjugate, BLB-201, for the treatment of high-risk acute myeloid leukemia (AACR 2026) - "In vivo, BLB-201 exhibited excellent synergy at low doses (0.1-0.3mg/kg) with clinical anti-AML agents (venetoclax, azacitidine and quizartinib) in systemic MV-4-11 and MOLM-13 models...We did not observe any effects of BLB-201 on normal erythroid, megakaryocytic and myeloid cell differentiation from CD34+ hematopoietic stem cells in vitro, while the CD33-targeted antibody drug conjugate, gemtuzumab ozogamacin, was highly toxic in these assays. BLB-201 DAC allows selective targeting of CD123-positive AML blasts and leukemia stem cells, minimizing direct exposure of GSPT1 degrader to healthy tissues. This unique mechanism of action may improve response rates when combined with existing targeted therapies for AML patients with high-risk or relapsed/refractory disease."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD123 • CD33 • CD34 • FLT3 • GSPT1 • IL3RA • PTPRC • TP53

ORM-1153: A novel CD123-targeting degrader antibody conjugate with proprietary GSPT1 degrading payload for the treatment of acute myeloid leukemia (AACR 2026) - "The only approved ADC for AML therapy is gemtuzumab ozogamicin (GO)...In addition, recent studies have reported that small-molecule GSPT1 degraders, such as CC-90009, exhibit potent antileukemic cytotoxicity in AML and demonstrate activity against TP53-mutant disease, one of the highest unmet needs...This in vitro activity is comparable to GO and ~3-log greater than venetoclax...We evaluated ORM-1153 in vivo MV4-11 xenograft model, and treatment with ORM-1153 demonstrated superior activity than standard of care treatment option. Our results indicate that ORM-1153 may represent a viable therapeutic agent for the treatment of AML, including in patients with TP53-mutant status who face limited options and an overall poor prognosis, and warrants further investigation in clinical trials."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD123 • GSPT1 • IL3RA • TP53

Rapid clinical diagnostic classification and risk stratification in acute myelogenous leukemia (AML) using whole genome sequencing (WGS) (AACR 2026) - "Recent evidence of clinical utility includes the incorporation of Mylotarg in induction therapy based on the identification of CBFB::MYH11 fusion gene, and the inclusion of Revumenib for a KMT2A rearrangement that could not be identified with standard breakaway FISH probes. In summary, we will describe the clinical deployment of a high-throughput, fast-turnaround WGS platform for AML, capable of delivering comprehensive genomic profiling in <48 hours from sample receipt to clinical reporting enabling earlier, more informed treatment decisions."

Clinical • Whole genome sequencing • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • FLT3 • IDH1 • IDH2 • KMT2A • MECOM • NPM1 • NUP98 • RUNX1 • RUNX1T1 • TP53

Bone marrow from acute myeloid leukemia (AML) and multiple myeloma (MM) patients can be used to evaluate target expression and on-target efficacy for novel antibody drug conjugates (ADCs) (AACR 2026) - "Gemtuzumab ozogamicin (Mylotarg), Belantamab Mofodotin (Blenrep) and Trastuzumab vedotin were evaluated for toxicity to hematopoietic progenitor cells using the colony forming cell assay (CFC) in diseased (on-target efficacy) and normal bone marrow (NBM) (off-target toxicity). Blenrap in MM patient bone marrow had IC50 values which ranged from 4.4 to 46 µg/mL with an average IC50 value of 21 µg/mL, compared to evaluation in NBM which resulted in IC50 values > 30 µg/mL (highest tested concentration) and rarely had any impact on colony growth. These data suggest that the CFC assays with primary normal and diseased bone marrow cells may provide insight as to relative potency of an ADC on a disease-specific target as well as potential off-target toxicity to normal hematopoietic progenitors."

ADC • Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • CD33 • HER-2

Low-Level Lymphoblasts in Patients with Acute Myeloid Leukemia (USCAP 2026) - "All pts received chemotherapy: 18 received HMA and venetoclax, 4 HMA alone, and 4 venetoclax alone...Targeted agents included quizartinib (n=4), gilteritinib (n=2), and gemtuzumab (n=1)... In AML patients, low-level lymphoblasts are detected rarely. When present, the lymphoblasts are predominantly of B-cell lineage, often detected at initial AML diagnosis, and are frequently associated with RUNX1 and TP53 mutations. Following AML-directed therapy, these lymphoblasts typically disappear or persist alongside AML myeloblasts without notable expansion, suggesting that the detection of lymphoblasts in the context of AML-type therapy does not require additional ALL-type therapy."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • ABL1 • ASXL1 • DNMT3A • FLT3 • IDH1 • KMT2A • MECOM • NPM1 • NRAS • NUP98 • RUNX1 • TET2 • TP53

Differentiation of Leukemic Blasts Following 7+3+GO in Core Binding Factor AML: A Case Series Highlighting Maturation Plasticity and Poor Prognosis (USCAP 2026) - "Gemtuzumab ozogamicin (GO), an anti-CD33 antibody-drug conjugate, induces cytotoxicity through calicheamicin-mediated DNA damage...Design: We retrospectively reviewed 28 newly diagnosed AML patients treated between 2017–2025 with standard 7+3 induction chemotherapy (cytarabine and daunorubicin) combined with GO... This case series provides the first human evidence of morphologic and phenotypic maturation of leukemic cells following 7+3+GO therapy in AML. This phenomenon may obscure residual disease, delay treatment escalation, and is associated with poor clinical outcomes. We propose that such cases be classified as non-response and that reinduction therapy be reconsidered in this context."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • CD34 • KIT

GEMTUZUMAB OZOGAMICIN ENABLES BRIDGING TO 2ND/3RD ALLO-HSCT WITH FAVORABLE OUTCOMES IN CD33-HIGH RELAPSED/REFRACTORY AML WITH POST–ALLO-HSCT RELAPSE (EBMT 2026) - "In heavily pretreated CD33-positive R/R AML pts relapsing after allo-HSCT, GO is an effective and well-tolerated therapy, achieving a high CR rate.Early bridging to second/third allo-HSCT in CR markedly improves survival, particularly in those with MRD-negative.The role of salvage second/third allo-HSCT in GO-refractory pts warrants further evaluation in larger cohorts."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hepatology • Infectious Disease • Pneumonia • Respiratory Diseases • CD33 • FLT3 • KMT2A • TP53

PHARMACODYNAMIC INSIGHTS INTO ADAPTER CAR TECHNOLOGIES (EBMT 2026) - "Separately, peptide tags were integrated into the amino acid sequence of the identical CD33-gemtuzumab-based AM... Our data demonstrate that the pharmacodynamic stability of the CAR-targeted epitope is essential for adapter CAR-mediated function. In addition to the loss of function when CAR-targeted epitope-devoid AMs are present, the competitive inhibition of functional AMs by the abundance of functionally inert AMs significantly disrupts overall performance of the conceptual framework. In vivo leukemia control strictly depends on the pharmacodynamic stability of the AM."

CAR T-Cell Therapy • PK/PD data • Hematological Malignancies • Leukemia • CD33

RISK FACTORS FOR HEPATIC VENO-OCCLUSIVE DISEASE AFTER ALLO-HSCT IN ACUTE LEUKEMIA PATIENTS TREATED WITH ANTIBODY-DRUG CONJUGATES (EBMT 2026) - "ADC exposure included gemtuzumab ozogamicin in acute myeloid leukemia (n=36., median doses: 3; range: 1-3) and inotuzumab ozogamicin in acute lymphoblastic leukemia (n=85, median doses: 3; range: 1-11), Patients transplanted outside first or second complete clinical and hematological remission comprised 60% (advanced disease)...Univariate analysis identified the following significant risk factors for VOD: advanced disease (38.4% vs. 17.5%, p = 0.02), use of calcineurin inhibitors vs calcineurin-free regimens (39% vs. 16.3% , p = 0.008), busulfan-based conditioning: in the pediatric subgroup (60% vs. 21.2%, p = 0.018); with no significant difference in adults (33% vs. 11%, p = 0.22), pretransplant EASIX( >3): 47.2% vs. 26.5% (p = 0.03).Multivariate logistic regression was performed for the three significant univariate predictors: advanced disease, use of calcineurin inhibitors, and busulfan-based conditioning... In patients with acute leukemia treated with ADCs prior..."

ADC • Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Leukemia

PHARMACODYNAMIC INSIGHTS INTO ADAPTER CAR TECHNOLOGIES (EBMT 2026) - "Separately, peptide tags were integrated into the amino acid sequence of the identical CD33-gemtuzumab-based AM... Our data demonstrate that the pharmacodynamic stability of the CAR-targeted epitope is essential for adapter CAR-mediated function. In addition to the loss of function when CAR-targeted epitope-devoid AMs are present, the competitive inhibition of functional AMs by the abundance of functionally inert AMs significantly disrupts overall performance of the conceptual framework. In vivo leukemia control strictly depends on the pharmacodynamic stability of the AM."

CAR T-Cell Therapy • PK/PD data • Hematological Malignancies • Leukemia • CD33

GEMTUZUMAB OZOGAMICIN ENABLES BRIDGING TO 2ND/3RD ALLO-HSCT WITH FAVORABLE OUTCOMES IN CD33-HIGH RELAPSED/REFRACTORY AML WITH POST–ALLO-HSCT RELAPSE (EBMT 2026) - "In heavily pretreated CD33-positive R/R AML pts relapsing after allo-HSCT, GO is an effective and well-tolerated therapy, achieving a high CR rate.Early bridging to second/third allo-HSCT in CR markedly improves survival, particularly in those with MRD-negative.The role of salvage second/third allo-HSCT in GO-refractory pts warrants further evaluation in larger cohorts."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hepatology • Infectious Disease • Pneumonia • Respiratory Diseases • CD33 • FLT3 • KMT2A • TP53

UNRAVELING ACUTE ST ELEVATION IN A YOUNG PATIENT WITH LEUKEMIA AND SEVERE THROMBOCYTOPENIA (ACC 2026) - "Background: A 36-year-old male with leukemic myocardial infiltrate from acute myelomonocytic leukemia developed chest pain 4 days after Cladribine, Cytarabine, Venetoclax, and Gemtuzumab. In patients with leukemic myocardial infiltrates, myocardial injury may follow oncologic treatment response. The mechanism is hypothesized as "collateral damage" of myocardial cells from inflammatory necrosis of adjacent leukemic cells. It results in STE resembling infarction, but without plaque rupture."

Clinical • Hematological Disorders • Hematological Malignancies • Leukemia • Thrombocytopenia

PRAC adopted an extension of indication for MYLOTARG (gemtuzumab ozogamicin) in combination with mitoxantrone and cytarabine for paediatric patients aged 1 to 18 years with newly diagnosed CD33-positive acute myeloid leukemia, based on results from the MyeChild 01 study. SmPC sections 4.1, 4.2, 4.4, 4.8 and 5.1 and the Package Leaflet were updated accordingly, and RMP version 2.3 was submitted. (European Medicines Agency) - Pharmacovigilance Risk Assessment Committee (PRAC) Minutes of meeting on 12 – 15 Jan 2026: [AI generated summary]

PRAC • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

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

Budget Impact of Venetoclax for Newly Diagnosed Patients with Acute Myeloid Leukemia Aged ≥ 75 Years or with Comorbidities Precluding Intensive Chemotherapy in the United States. (PubMed, Adv Ther) - "Inclusion of venetoclax combinations for newly diagnosed patients with AML aged ≥ 75 years or with comorbidities precluding intensive chemotherapy reduced the budget impact, providing potential financial benefits for US payers."

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

A Study To Learn About the Safety Medicine (Called Mylotarg) In People With Acute Myeloid Leukemia (clinicaltrials.gov) - P=N/A | N=165 | Recruiting | Sponsor: Pfizer | Trial completion date: Apr 2027 ➔ Jul 2027 | Trial primary completion date: Apr 2027 ➔ Jul 2027

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

RESOLVE trial: Registry and clinical trial comparing standard intensity and reduced intensity treatment in patients with AML or CLL who do not have residual disease. (clinicaltrialsregister.eu) - P4 | N=289 | Recruiting | Sponsor: Medizinische Hochschule Hannover | N=128 ➔ 289

Enrollment change • Minimal residual disease • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma

RESOLVE trial: Registry and clinical trial comparing standard intensity and reduced intensity treatment in patients with AML or CLL who do not have residual disease. (clinicaltrialsregister.eu) - P4 | N=128 | Recruiting | Sponsor: Medizinische Hochschule Hannover | N=52 ➔ 128

Enrollment change • Minimal residual disease • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma

Safety of gemtuzumab ozogamicin with cytarabine, daunorubicin, and midostaurin induction in FLT3-mutated AML. (PubMed, Blood Neoplasia) - P1 | "Our results show that GO can safely be combined with intensive chemotherapy with midostaurin in ND, FLT3-mutated AML. This trial was registered at www.clinicaltrials.gov as #NCT03900949."

Clinical • Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Transplantation • FLT3

REAL-WORLD PHARMACOVIGILANCE OF ANTIBODY-DRUG CONJUGATES: PAYLOAD-SPECIFIC SAFETY SIGNALS FROM DISPROPORTIONALITY ANALYSIS (ISPOR 2026) - "Potential signals were identified using the proportional reporting ratio (PRR ≥ 2), reporting odds ratio lower limit (ROR > 1), information component (IC > 0) and empirical Bayes geometric mean using the multi-item gamma Poisson Shrinker model (EBGM05 ≥ 2). A total of 11,618 reports involving four ADCs (gemtuzumab ozogamicin, inotuzumab ozogamicin, brentuximab vedotin, and polatuzumab vedotin) were analyzed, meeting all predefined criteria. This payload-stratified disproportionality analysis identified distinct and biologically plausible ADR profiles for calicheamicin and MMAE-based ADCs. The study provides a comparative, payload-stratified evaluation of real-world pharmacovigilance signals, allowing differentiation between class-wide, payload-driven, and drug-specific toxicities."

Adverse events • Clinical • Real-world • Real-world evidence • Cardiovascular • Cerebral Hemorrhage • Congestive Heart Failure • Gastroenterology • Gastrointestinal Disorder • Heart Failure • Hematological Malignancies • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases