Danyelza (naxitamab-gqgk) / SERB Pharmaceuticals, Takeda, SciClone, Nobelpharma, INPHARMUS - LARVOL DELTA

A Novel GD2-Targeted Radionuclide Probe 89 Zr-TD-hu3F8 for Melanoma Diagnosis (EANM 2026) - "A GD2-targeted radionuclide probe, ⁸⁹Zr-TD-hu3F8, has been successfully prepared with high labeling yield, high radiochemical purity, and a high target-to-background ratio. This probe offers a new candidate strategy for non-invasive diagnosis and potential radionuclide therapy of melanoma, showing promise for clinical translation."

Melanoma • Skin Cancer • Solid Tumor

Comparative evidence on the independent contribution of granulocyte-macrophage colony-stimulating factor to anti-GD2 immunotherapy in neuroblastoma: a systematic review and gap analysis. (PubMed, Front Oncol) - "No eligible comparative study was identified for dinutuximab, dinutuximab beta or naxitamab. A randomized trial holding the anti-GD2 backbone and relevant co-interventions constant is needed to isolate the effect of GM-CSF. https://www.crd.york.ac.uk/PROSPERO/, identifier CRD420251116911."

Journal • Review • Neuroblastoma • Oncology • Solid Tumor • Transplantation • CSF2

Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5 (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: Shaare Zedek Medical Center | Not yet recruiting ➔ Recruiting

Enrollment open • Neuroblastoma • Oncology • Solid Tumor • ALK • CSF2 • MYCN

Triple-costimulatory GD2-CAR T cells incorporating CD28, 4-1BB and CD27 mediate potent cytotoxicity and reduce exhaustion in solid tumour models. (PubMed, Biomed Pharmacother) - "We engineered humanised hu3F8-based GD2-CAR T cells bearing dual or triple costimulatory domains- GD2-28BBζ (CD28/4-1BB), GD2-28.27ζ (CD28/CD27) and GD2-28BB27ζ (CD28/4-1BB/CD27) - and compared their in-vitro function against GD2-positive neuroblastoma (SK-N-AS), lung adenocarcinoma (A549) and retinoblastoma (Y79 and WERI-Rb-1) cells...Under serial tumour rechallenge, GD2-28BB27ζ T cells maintained cytolytic function, acquired fewer activation and exhaustion markers (CD69, TIM-3 and, versus GD2-28BBζ, LAG-3; PD-1 unchanged), better preserved their CAR⁺ population, and retained a larger naïve-like (CD45RO⁻CD62L⁺) subset than the other constructs. Together, these in-vitro findings indicate that combining CD28, 4-1BB and CD27 within a single CAR confers potent cytotoxicity with low effector-to-target ratios, lower effector-cytokine output, reduced exhaustion-marker acquisition and greater phenotypic durability, identifying GD2-28BB27ζ as a promising configuration for..."

Journal • Eye Cancer • Lung Adenocarcinoma • Lung Cancer • Neuroblastoma • Non Small Cell Lung Cancer • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor • CD27 • CD69 • HAVCR2 • IFNG • IL2 • LAG3 • PD-1 • TNFA

Tislelizumab combined with Anti-GD2 antibody-based chemoimmunotherapy for pediatric relapsed/refractory high-risk neuroblastoma: a two-Case Report. (PubMed, Front Pharmacol) - "The anti-GD2 antibody, dinutuximab beta or naxitamab, was selected according to each patient's insurance coverage. Notably, this retrospective two-case observation cannot distinguish the independent antitumor contribution of tislelizumab alone, as multiple therapeutic components were administered simultaneously without control groups to isolate single-agent efficacy. Larger prospective studies are warranted to further evaluate the efficacy and safety of this approach."

Journal • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • CSF2

Case Report: Early introduction of anti-GD2 immunotherapy during induction chemotherapy in high-risk neuroblastoma: two pediatric cases. (PubMed, Front Oncol) - "While these preliminary findings demonstrate the feasibility and tolerability of early naxitamab introduction, the limited sample size precludes definitive conclusions regarding efficacy. This report provides exploratory data to support future prospective studies on optimizing the timing of anti-GD2 immunotherapy in HR-NB."

Journal • Hypotension • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor

Humanized Monoclonal Antibody 3F8 (Hu3F8) With Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in the Treatment of Recurrent Osteosarcoma (clinicaltrials.gov) - P2 | N=46 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Jul 2026 ➔ Jul 2027 | Trial primary completion date: Jul 2026 ➔ Jul 2027

Trial completion date • Trial primary completion date • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Quantum-Capacitance Biosensing Enables Real-Time Monitoring of Naxitamab for Therapeutic Response Stratification in Neuroblastoma. (PubMed, Small Sci) - "Application to retrospective clinical samples reveals distinct post-infusion concentration profiles, enabling early discrimination between responder groups and detection of antidrug antibody development. This work establishes quantum-capacitance biosensing as a generalizable strategy for real-time monitoring of therapeutic antibodies in precision oncology."

Journal • Neuroblastoma • Oncology • Solid Tumor

Gemcitabine and Ex Vivo Expanded Allogenic Universal Donor, TGF?i Natural Killer (NK) Cells With or Without Naxitamab (Danyelza) for the Treatment of Patients With Metastatic, GD2 Expressing, HER2 Negative Breast Cancer (clinicaltrials.gov) - P1/2 | N=42 | Recruiting | Sponsor: Margaret Gatti-Mays | Trial completion date: Dec 2026 ➔ Oct 2027 | Trial primary completion date: Dec 2026 ➔ Oct 2027

Trial completion date • Trial primary completion date • Tumor mutational burden • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • Hormone Receptor Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • CD4 • HER-2

Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Steven DuBois, MD | Trial primary completion date: Dec 2026 ➔ Jul 2027

Trial primary completion date • Neuroblastoma • Oncology • Solid Tumor

Study of Chemoimmunotherapy for High-Risk Neuroblastoma (clinicaltrials.gov) - P1 | N=48 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Jun 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • Neuroblastoma • Oncology • Solid Tumor • MYCN

Study 201: Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow (clinicaltrials.gov) - P2 | N=122 | Active, not recruiting | Sponsor: Y-mAbs Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Neuroblastoma • Oncology • Solid Tumor

Feasibility study of prolonged administration of naxitamab, irinotecan, and temozolomide for patients with relapsed or refractory neuroblastoma. (ASCO 2026) - P1 | "Secondary endpoints include response rate per revised INRC and survival outcomes. Correlative and exploratory investigations embedded within the trial include PK profiling of prolonged naxitamab infusion durations and evaluation of circulating biomarkers including circulating tumor DNA, tumor cells, and GD2 in the study population during anti-GD2 therapy."

Clinical • IO biomarker • Ganglioneuroblastoma • Neuroblastoma • Solid Tumor

BCC018: Study to find out whether adding the medicine Naxitamab to standard treatment could help children and teenagers who have just been diagnosed with high-risk neuroblastoma (clinicaltrialsregister.eu) - P1/2 | N=22 | Not yet recruiting | Sponsor: Fundacio Sant Joan De Deu

New P1/2 trial • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

Bronchiolitis Obliterans as a Fatal Late Effect in Survivors of High-Risk Neuroblastoma (ASPHO 2026) - "Per the Children's Oncology Group (COG) Long-Term Follow-Up Guidelines, pulmonary late effects in neuroblastoma typically result from radiation therapy or surgery involving the lungs or busulfan-based conditioning regimens for autologous hematopoietic stem cell transplantation (aHSCT). Less is known about long-term implications of neuroblastoma therapies such as anti-GD2 immunotherapy (Hu3F8, ch14.18), isotretinoin, and ALK inhibitors. Reversible pulmonary toxicity has been described with the combination of immunotherapy and lorlatinib, an ALK inhibitor, which improves with cessation of lorlatinib... Patient 1 was diagnosed at 4 years of age with high-risk neuroblastoma and treated with chemotherapy per COG protocol ANBL0532, adrenalectomy, tandem aHSCT (conditioning: cyclophosphamide, thiotepa; carboplatin, etoposide, melphalan [CEM]), 21.6 Gray flank radiation, and Hu3F8... We describe two survivors of high-risk neuroblastoma who developed fatal bronchiolitis..."

Bone Marrow Transplantation • Immunology • Infectious Disease • Neuroblastoma • Pulmonary Disease • Respiratory Diseases • Solid Tumor • IL2

Engineering macrophages as a novel therapeutic approach for high-risk neuroblastoma (AACR 2026) - "Fluorescent microscopy and flow cytometry confirmed a significant enhancement in the phagocytosis of GD2-high NB cells by GD2-CAR-Ms containing Hu3F8 single-chain variable fragment (scFv)...Additionally, inhibition of tumor growth was also observed in our zebrafish NB model after GD2-CAR-Ms treatment, further supporting their therapeutic potential. Together, our findings demonstrate that GD2-CAR-Ms exhibit potent antitumor activity and reshape the tumor immune microenvironment, supporting their translational potential as a therapeutic strategy for high-risk NB."

IO biomarker • Neuroblastoma • Oncology • Solid Tumor • MYCN

Clinical trial in progress: BCC022 a phase II trial of tipifarnib and naxitamab for relapsed/refractory neuroblastoma (AACR 2026) - P2 | "A projected 90 evaluable subjects (70 in cohort 1 and 20 in cohort 2) will be enrolled at up to 30 Beat Childhood Cancer Research Consortium hospitals. As of January 2026, enrollment is ongoing at 9 hospitals, with approximately 3% of the planned population enrolled."

Clinical • P2 data • Hematological Malignancies • Neuroblastoma • Oncology • Solid Tumor • HRAS

EZH2 inhibition upregulates GD2 to enhance anti-GD2 immunotherapy in Ewing sarcoma (AACR 2026) - "This data supports the use of the EZH2 inhibitor tazemetostat for ES tumors that express low or heterogeneous levels of GD2 to enhance the effectiveness of GD2-targeting immunotherapeutic approaches. This study demonstrates the capability of CyTOF to look at multiple markers simultaneously to achieve a comprehensive characterization of various samples."

IO biomarker • Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor

Single-cell transcriptomic atlas-guided NRG3-ERBB4 axis enhances the efficacy of chemo-immunotherapy during induction phase in high-risk neuroblastoma. (PubMed, Mol Ther) - "Collectively, these findings delineate an NRG3-ERBB4 axis-centered tumor-immune crosstalk that operates across pretreatment, chemotherapy, and CI, uncovering a previously unrecognized mechanism underlying naxitamab-mediated immune activation. Our results also support ERBB4 as a promising predictive biomarker and therapeutic target to optimize CI strategies for high-risk NB."

IO biomarker • Journal • Neuroblastoma • Oncology • Solid Tumor • CD8 • ERBB4 • GNLY • GZMB • NRG3

Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5 (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: Shaare Zedek Medical Center

New P2 trial • Neuroblastoma • Oncology • Solid Tumor • ALK • CSF2 • MYCN

Concurrent naxitamab and lorlatinib in a patient with refractory high-risk neuroblastoma: a case report. (PubMed, Front Oncol) - "Mild adverse events occurred during the combined immunotherapy, including transient hypotension managed with intravenous fluids and abdominal and leg pain that improved with analgesics. In conclusion, our case showed that combination therapy with naxitamab and lorlatinib may improve long-term outcomes and reduce chemotherapy-related toxicity."

IO biomarker • Journal • Hypotension • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor • ALK

M3554, a novel anti-GD2 antibody drug conjugate (AACR 2024) - "This tumor associated expression pattern makes GD2 an attractive target for therapeutic antibodies like dinutuximab (ch14.18) and naxitamab, which have been approved for neuroblastoma. Timothy Crandall is an employee of EMD Serono. Mira Toister-Achituv is an employee of Inter-Lab Ltd., Yavne, Israel, an affiliate of Merck KGaA, Darmstadt, Germany."

ADC • Brain Cancer • CNS Tumor • Glioma • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

GD3 synthase (ST8SIA1) is a key immunomodulator in GD2+ breast cancer cells (AACR 2024) - "GD3 synthase regulates macrophage-mediated phagocytosis, NK cell-induced apoptosis, and T-cell cytotoxicity in BC cells. Naxitamab enhances immune cell killing of breast cancer cells with high GD2 expression. Furthermore, naxitamab inhibited tumor growth in GD2+ TNBC PDX models."

Immunomodulating • Breast Cancer • Neuroblastoma • Oncology • Solid Tumor • Triple Negative Breast Cancer

A first-in-human phase 1, multicenter, open-label study of M3554, a novel anti-GD2 antibody-drug conjugate (ADC), in patients with advanced solid tumors (AACR 2025) - P1 | "GD2 is a clinically validated target in pediatric neuroblastoma; however, currently approved anti-GD2 antibody treatments, such as dinutuximab and naxitamab, have been associated with severe adverse events (AEs) of pain, likely due to Fcγ receptor-mediated immune activation via antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC). The study is currently recruiting and aims to enroll 52 patients in the dose escalation phase and 110 patients in the dose expansion phase (STS, n=80; GBM, n=30), across different global sites including, Japan, Belgium, France, and the US. Clinical trial information: NCT06641908."

ADC • Clinical • First-in-human • Metastases • P1 data • Brain Cancer • CNS Tumor • Glioblastoma • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Low-Dose Intravenous Ketamine for Primary Pain Control During Naxitamab Anti-GD2 Immunotherapy Treatment. (PubMed, Pediatr Blood Cancer) - "Ketamine was successfully administered in an outpatient setting and was associated with a significant opioid-sparing effect. A larger prospective study is warranted."

Journal • CNS Disorders • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor