Qarziba (dinutuximab beta) / BeOne Medicines, Recordati, Ligand - LARVOL DELTA

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

Dinutuximab beta with TEMIRI chemotherapy in relapsed/refractory neuroblastoma: Real-world insights into the open question of GD2 expression. (PubMed, Transl Oncol) - "This real-world experience confirms that a short schedule is a feasible and effective option in R/R neuroblastoma. The dynamic and heterogeneous expression of GD2 deserves further evaluation, particularly in relation to selection of patients and subsequent GD2-directed treatment strategies."

IO biomarker • Journal • Real-world evidence • Hematological Disorders • Neuroblastoma • Oncology • Solid Tumor

Anti-GD2 Antibody Dinutuximab Beta and Low-Dose Interleukin 2 After Haploidentical Stem-Cell Transplantation in Patients With Relapsed Neuroblastoma: A Multicenter, Phase I/II Trial. (PubMed, J Clin Oncol) - "DB therapy after haplo-SCT in patients with rHR-NB is feasible, with low risk of inducing GvHD, and results in long-term remissions likely attributable to increased antineuroblastoma activity by donor-derived effector cells."

Journal • P1/2 data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Tumor • Graft versus Host Disease • Immunology • Neuroblastoma • Neuroendocrine Tumor • Oncology • Solid Tumor • Transplantation • IL2

Concurrent Treatment With Lorlatinib and Dinutuximab Beta Induced Acute Gastric Dilation in a Pediatric Patient With Neuroblastoma. (PubMed, Pediatr Blood Cancer) - No abstract available

Journal • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

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

GD2-targeted immunotherapy in pediatric bone sarcomas: a systematic review of emerging strategies and combination approaches. (PubMed, Front Immunol) - "Clinical evidence, although limited and heterogeneous, suggested encouraging activity of Dinutuximab beta-based chemo-immunotherapy, especially in ES...However, major translational challenges remain, including limited clinical data, heterogeneous study designs, and the lack of standardized assays for GD2 expression assessment. Further collaborative international studies are needed to optimize patient selection and accelerate the development of GD2-targeted combination immunotherapy strategies."

IO biomarker • Journal • Review • Ewing Sarcoma • Oncology • Osteosarcoma • Pediatrics • Sarcoma • Solid Tumor

Case Report: Evolution of intratumoral spatial heterogeneity assessed by [68Ga]Ga-DOTA-NOC PET/CT during dinutuximab beta therapy in pediatric high-risk neuroblastoma. (PubMed, Front Pediatr) - "Histopathological evaluation confirmed this hyper-avid region as a viable, poorly differentiated neuroblastoma subclone. Consequently, while [68Ga]Ga-DOTA-NOC PET/CT assists in localizing viable residual disease and guiding surgical navigation, elevated SSTR accumulation following immunotherapy necessitates cautious multidisciplinary evaluation."

Heterogeneity • IO biomarker • Journal • Neuroblastoma • Oncology • Pediatrics • Peritoneal Cancer • Solid Tumor • SSTR

Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse (clinicaltrials.gov) - P1/2 | N=76 | Not yet recruiting | Sponsor: National Cancer Institute (NCI) | Initiation date: Jul 2026 ➔ Oct 2026

Trial initiation date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor

Association Between Dinutuximab Beta Exposure and Post-End-of-Treatment Survival in Neuroblastoma: A Weighted Patient-Level Analysis of Three Clinical Studies. (PubMed, Pediatr Blood Cancer) - "Greater prior exposure to DB, particularly a higher number of treatment cycles, was associated with better post-EOT EFS and OS. The study did not estimate an on-treatment causal effect of DB, and the residual confounding cannot be fully excluded. Prior treatment exposure may help inform post-treatment risk stratification."

Journal • Neuroblastoma • Oncology • Solid Tumor • IL2

Management of persistent soft tissue residuals exhibiting an imaging paradox (^123I-MIBG-negative/^68Ga-DOTA-NOC-positive) in high-risk neuroblastoma following immunotherapy: a case report. (PubMed, Front Immunol) - "We report a 4-year-and-11-month-old girl with HR-NB who received multimodal therapy, including induction chemotherapy, surgery, radiotherapy, autologous stem cell transplantation, and seven cycles of dinutuximab beta-based immunotherapy...In patients with persistent masses and discordant imaging findings, multimodal functional imaging may provide complementary information to guide clinical decision-making. The favorable outcome observed in this patient, who remains in sustained remission following surgical resection without additional antitumor therapy, supports consideration of an integrated management strategy combining multimodal imaging assessment and appropriately timed surgical intervention for selected cases of persistent residual disease."

Journal • Neuroblastoma • Oncology • Solid Tumor • Transplantation

Dinutuximab-beta and Chemotherapy in Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P1 | N=30 | Not yet recruiting | Sponsor: Chang Gung Memorial Hospital

New P1 trial • Neuroblastoma • Oncology • Solid Tumor • MYCN

Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma (clinicaltrials.gov) - P1 | N=12 | Terminated | Sponsor: National Cancer Institute (NCI) | Completed ➔ Terminated; Drug supply issues

Trial termination • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Actuate Therapeutics’ Elraglusib Selected for Evaluation in BEACON2 Trial for High-Risk Pediatric Neuroblastoma (GlobeNewswire) - "BEACON2 is a landmark European multi-arm, multi-stage Phase 1/2 platform trial....Under the BEACON2 trial design, the combination of elraglusib with dinutuximab beta plus chemotherapy will initially be assessed in a dose confirmation cohort of up to 20 patients to evaluate safety and to determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and pharmacokinetics (PK) profile of the regimen. Following successful completion of the dose confirmation stage, the regimen may advance into a randomized portion of the platform trial, where approximately 75 patients will be enrolled with a planned interim analysis."

Trial status • Neuroblastoma

Dinutuximab beta in combination with chemotherapy in patients with refractory or relapsed neuroblastoma: A national prospective study (2020/ABM/01/00055). (ASCO 2026) - P1 | " Twenty-eight patients aged 1–18 years with RR-HRNB received DB combined with temozolomide/irinotecan (TemIri) or with N5/N6 chemotherapy of the German Pediatric Oncology and Hematology Group (GPOH) protocol. DB combined with TemIri or GPOH chemotherapy demonstrates high antitumor activity with acceptable and manageable toxicity in children with RR-HRNB. Response-adapted sequencing and individualized chemotherapy selection may improve disease control, including metastatic responses, in this high-risk population. Clinical trial information: 2021-003832-96."

Clinical • Combination therapy • Neuroblastoma • Solid Tumor • MYCN

Phase II Study of Chidamide-Dinutuximab Beta-Irinotecan-Temozolomide for Refractory/Relapsed Neuroblastoma in Children (clinicaltrials.gov) - P2 | N=27 | Recruiting | Sponsor: Tianjin Medical University Cancer Institute and Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Neuroblastoma • Oncology • Solid Tumor

NCI-2018-03732: Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P2 | N=42 | Completed | Sponsor: National Cancer Institute (NCI) | Active, not recruiting ➔ Completed | Trial completion date: Sep 2026 ➔ Mar 2026

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

A pilot study combining primary busulfan-based haploidentical stem cell transplantation with GD2 antibody to treat high-risk neuroblastoma. (PubMed, Bone Marrow Transplant) - "We report administration of a conditioning regimen combining myeloablative busulfan/melphalan anti-tumor therapy with the immunological advantages of haplo-SCT and GD2-directed antibody-based immunotherapy with dinutuximab beta (DB)...Prior to busulfan, melphalan, fludarabine and antithymocyte globulin conditioning followed by T/B-cell-depleted haplo-SCT and 6 DB cycles, 2 patients received [131I]MIBG therapy...Early results suggest a survival benefit for these pediatric patient subgroups at very high risk. Confirmation in a larger controlled trial is warranted."

IO biomarker • Journal • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • Transplantation

Bioequivalence and dosimetry estimates of GD2-targeting Dinutuximab-Beta labelled with indium-111, lutetium-177, and actinium-225 (SNMMI 2026) - "Radionuclide therapy with [131I]MIBG is an established treatment but clinical outcomes are heterogeneous. DOTA-conjugated Dinutuximab-Beta can be successfully labelled with alpha- and beta-emitting radionuclides, 225Ac and 177Lu, which allows its investigation as a molecular radiotherapy approach for the treatment of high-risk neuroblastoma. 111In-DB can be utilised as a tool to predict the in vivo biodistribution of 225Ac-DB and 177Lu-DB, with potential for translational SPECT imaging and dosimetry assessments. The limitations to this approach derive from the gradual accumulation of 225Ac in the bone and assumptions on daughter translocation."

Neuroblastoma • Solid Tumor

Bioequivalence and dosimetry estimates of GD2-targeting Dinutuximab-Beta labelled with indium-111, lutetium-177, and actinium-225 (SNMMI 2026) - "Radionuclide therapy with [131I]MIBG is an established treatment but clinical outcomes are heterogeneous. DOTA-conjugated Dinutuximab-Beta can be successfully labelled with alpha- and beta-emitting radionuclides, 225Ac and 177Lu, which allows its investigation as a molecular radiotherapy approach for the treatment of high-risk neuroblastoma. 111In-DB can be utilised as a tool to predict the in vivo biodistribution of 225Ac-DB and 177Lu-DB, with potential for translational SPECT imaging and dosimetry assessments. The limitations to this approach derive from the gradual accumulation of 225Ac in the bone and assumptions on daughter translocation."

Neuroblastoma • Solid Tumor

BEACON: Activity Study of Bevacizumab With Temozolomide ± Irinotecan for Neuroblastoma in Children (clinicaltrials.gov) - P2 | N=225 | Completed | Sponsor: University of Birmingham | Active, not recruiting ➔ Completed

Trial completion • Neuroblastoma • Oncology • Solid Tumor

Inbraced: Phase I Study of 131-I mIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children With Relapsed/Refractory Neuroblastoma (clinicaltrials.gov) - P1 | N=44 | Active, not recruiting | Sponsor: University Hospital Southampton NHS Foundation Trust | Trial completion date: Nov 2025 ➔ Sep 2026

Trial completion date • Neuroblastoma • Oncology • Solid Tumor

BEACON: Activity Study of Bevacizumab With Temozolomide ± Irinotecan for Neuroblastoma in Children (clinicaltrials.gov) - P2 | N=225 | Completed | Sponsor: University of Birmingham | Active, not recruiting ➔ Completed | Completed ➔ Active, not recruiting

Enrollment closed • Trial completion • Neuroblastoma • Oncology • Solid Tumor

Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse (clinicaltrials.gov) - P1/2 | N=76 | Not yet recruiting | Sponsor: National Cancer Institute (NCI) | Initiation date: May 2026 ➔ Jul 2026

Trial initiation date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor

Comprehensive molecular analyses for diagnosis and treatment guidance in an adult neuroblastoma patient. (PubMed, Oncologist) - "Comprehensive molecular analysis and MTB discussion revealed several potential treatment targets, leading to subsequent treatment including dinutuximab beta, nivolumab, cabozantinib, I-131-mIBG radionuclide therapy and alpelisib, unfortunately, all followed by disease progression. This case demonstrates the potential of comprehensive molecular analysis including methylation profiling for diagnosis and treatment guidance in rare tumors. Additional research is urgently required to improve outcomes in elderly patients with neuroblastoma."

IO biomarker • Journal • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • NTRK

End-of-Induction Response and Tolerability of High-Risk Neuroblastoma Treated with Chemoimmunotherapy—Modified N7 Regimen with Dinutuximab Beta (Multidisciplinary Digital Publishing Institute) - "The EOI objective response rate was 78% (7/9 patients) for the primary tumour site and 100% at metastatic sites. No patient exhibited progressive disease. A modified Curie score of ≤2 on MIBG scan was achieved in 78% of patients. Grade 3 or higher toxicities, including neutropenic fever, enterocolitis, and capillary leak syndrome, were observed in eight patients but were manageable."

Retrospective data • Neuroblastoma