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

Dinutuximab Beta Added to Temozolomide-Based Chemotherapy for Children With Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON Immuno Phase II Trial. (PubMed, J Clin Oncol) - "Within a randomized phase II setting, results observed with addition of dB to T-based chemotherapy in RR-HR-NB warrant further evaluation."

Journal • P2 data • Neuroblastoma • Oncology • Solid Tumor

Economic Evaluation of Dinutuximab Beta in the Maintenance Therapy of High-Risk Neuroblastoma in Brazil (ISPOR-EU 2025) - "DB as LTI offers good value for money to payers in private healthcare in Brazil both in newly diagnosed and relapsed/refractory patients. As almost all total cost is attributable to DB, the results are driven by price and are broadly applicable to other countries."

Clinical • HEOR • Neuroblastoma • Solid Tumor • MYCN

Dinutuximab Beta vs. Historical Controls in the Maintenance Therapy of Relapsed Neuroblastoma: Unadjusted and Adjusted Indirect Comparison (ISPOR-EU 2025) - "Dinutuximab beta significantly extends overall survival in relapsed neuroblastoma compared to disease not treated with immunotherapy - in both unadjusted and adjusted indirect comparisons."

Clinical • Neuroblastoma • Solid Tumor • IL2 • MYCN

Prolonged overall survival in a child with multimetastatic retinoblastoma treated with anti-GD2 monoclonal antibody dinutuximab beta: a case report. (PubMed, Front Oncol) - "The patient was treated with systemic conventional and high-dose chemotherapy combined with intrathecal Topotecan. This case suggests that anti-GD2 immunotherapy, used as consolidation treatment, may improve the prognosis of patients with advanced retinoblastoma and potentially reduce the toxicity associated with standard therapies. Further clinical investigation is warranted to validate these findings."

IO biomarker • Journal • Eye Cancer • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor • RB1

CHEMOIMMUNOTHERAPY EXPERIENCE IN RELAPSED/REFRACTORY NEUROBLASTOMA: CURRENT STATUS (SIOP 2025) - " The clinical characteristics and treatment details of patients who received chemoimmunotherapy for R/R neuroblastoma between January 2020 and January 2025 were evaluated.Patients who had received first line chemotherapy or various salvage therapies prior to chemoimmunotherapy were treated with dinutuximab beta (DB) in combination with different chemotherapy regimens.Treatment responses were assessed every two to three cycles using MRI of the primary site,bone marrow aspiration and biopsy,and MIBG or PET-CT scanning. The combination of chemotherapy and immunotherapy in patients with relapsed/refractory neuroblastoma continues to yield high overall survival rates.Rapid chemotherapy modifications when an inadequate response is observed,response evaluations every three months,and continuous patient monitoring by an experienced multidisciplinary team are believed to have significantly contributed to these outcomes."

IO biomarker • Neuroblastoma • Solid Tumor • ALK • MYCN

EFFICACY AND TOXICITY OF INDUCTION REGIMENS IN HIGH-RISK NEUROBLASTOMA: A SINGLE CENTER EXPERIENCE. (SIOP 2025) - "38 patients underwent surgery and received consolidation with high-dose chemotherapy and autologous stem cell transplant, while 16 received maintenance with Dinutuximab beta, implemented in 2016... The results showed a response to the induction that is consistent with those reported in the literature. Objetive response and low toxicity were observed in RDC cohort. Large sample and prospective studies are needed to further validate these findings."

Clinical • Febrile Neutropenia • Hematological Disorders • Neuroblastoma • Neutropenia • Pediatrics • Solid Tumor • MYC • MYCN

EVALUATION OF NEW CHEMO-IMMUNOTHERAPY COMBINATIONS AFTER THERAPEUTIC FAILURE WITH IRINOTECAN-TEMOZOLAMIDE AND ANTI-GD2 IN REFRACTORY OR RELAPSED NEUROBLASTOMA (SIOP 2025) - "This study aimed to evaluate the response to switching the chemotherapy backbone to cyclophosphamide-topotecan in combination with anti-GD2 (C/T/anti-GD2) in patients who failed a first salvage strategy with irinotecan, temozolomide, and anti-GD2 (I/T/anti-GD2). This retrospective study analyzed patients with r/r HR-NB treated between 2020 and 2024, who received cyclophosphamide (250 mg/m², D1-D5) and topotecan (0.75 mg/m², D1-D5), combined with either dinutuximab beta (35 mg/m², D1-D7, continuous infusion) or naxitamab (2.25 mg/m²/day, D2, D4, D9, D11), after prior treatment with I/T/anti-GD2... Switching to C/T/anti-GD2 after failure of I/T/anti-GD2 achieved disease control in a substantial proportion of patients. Outcomes were encouraging considering the high-risk, heavily pretreated population. However, long-term durability remains challenging, underscoring the need for alternative strategies to achieve sustained responses."

Cardiovascular • Dermatology • Hematological Disorders • Hypertension • Immunology • Neuroblastoma • Solid Tumor • Urticaria

DINUTUXIMAB BETA VERSUS NAXITAMAB IN RELAPSED/REFRACTORY NEUROBLASTOMA PATIENTS WITH STABLE DISEASE, MINOR RESPONSE OR PARTIAL RESPONSE AND DISEASE IN BONE OR BONE MARROW (SIOP 2025) - "In this type of analysis, data suggest that dinutuximab beta in patients with neuroblastoma in bone or bone marrow with incomplete or no response to prior therapy mediates greater response rate and event-free survival compared to naxitamab. The benefit of DB is greater when DB is used without IL-2."

Clinical • Neuroblastoma • Solid Tumor • CSF2 • MYCN

EFFICACY OF DINUTUXIMAB BETA IN THE TREATMENT OF RELAPSED/REFRACTORY NEUROBLASTOMA IN PATIENTS WITH METASTATIC DISEASE IN BONE OR BONE MARROW –POOLED TRIAL DATA ANALYSES RESULTS (SIOP 2025) - "Response to treatment with dinutuximab beta as long term infusion in patients with relapsed/refractory neuroblastoma in bone or bone marrow is clinically significant. Treatment without IL2 has no significant impact on response or survival, but it reduces frequency of adverse events, particularly of pain."

Clinical • Metastases • Neuroblastoma • Solid Tumor • MYCN

RETROSPECTIVE EVALUATION OF THE ADMINISTRATION OF DINUTUXIMAB VIA AN ELASTOMERIC PUMP: INSIGHTS AND LESSONS LEARNED FROM A NURSING PRACTICE PERSPECTIVE (SIOP 2025) - "Elastomeric pumps were introduced in our pediatric hemato-oncology department for the administration of dinutuximab-beta (DB) in high-risk neuroblastoma patients. Close monitoring of DB-elastomer pumps at home ensures infusion safety and effectiveness. Quality of care improved through iterative problem-solving, adaptation and implementation of a systematic protocol. Key factors include: sensor fixation, catheter accessibility and knowledge by healthcare professionals."

Retrospective data • Neuroblastoma • Pediatrics • Solid Tumor

ANTI-GD2 ANTIBODY DINUTUXIMAB BETA EXTENDED CYCLES TREATMENT COMBINED WITH CHEMOTHERAPY IN PATIENTS WITH RELAPSED OR REFRACTORY NEUROBLASTOMA- A RETROSPECTIVE STUDY (SIOP 2025) - " This is a single-center, retrospective clinical study that screened children with R/R neuroblastoma, who received more than five cycles of dinutuximab beta, combined with Granulocyte-macrophage colony-stimulating factor (GM-CSF), isotretinoin, and chemotherapy. Extended cycles of chemoimmunotherapy in patients with R/R neuroblastoma is effective and well tolerated, showing promising response rates and survival outcomes."

Retrospective data • Neuroblastoma • Solid Tumor • CSF2

EXPERIENCE WITH DINUTUXIMAB BETA: EVALUATION OF ADVERSE EFFECTS (SIOP 2025) - " A total of 519 cycles of dinutuximab beta(DB) administered to 56 patients were analyzed.The male-to-female ratio was 1.1.The median age at diagnosis was 36 months(6– 132 months),while the median age at the start of DB treatment was 53 months(12–138 months).Nineteen patients were treated according to the TPOG 2009 protocol,while the remaining 37 patients received treatment under the TPOG 2020 protocol.Patients treated under the 2009 protocol received chemotherapy+DB during the relapse/refractory phase.Of the total 519 DB cycles,72 cycles were DB alone,while 447 cycles were DB combined with chemotherapy.The most frequently administered chemotherapy regimen alongside DB was irinotecan+temozolomide. Dinutuximab beta is an effective agent in the treatment of patients with high- risk or relapsed/refractory neuroblastoma.Close monitoring and a multidisciplinary approach have been shown to successfully control treatment-related adverse effects."

Adverse events • Anemia • CNS Disorders • Eosinophilia • Epilepsy • Febrile Neutropenia • Neuroblastoma • Neutropenia • Pediatrics • Solid Tumor • Thrombocytopenia

DINUTUXIMAB BETA LONG TERM INFUSION IS ASSOCIATED WITH A SURVIVAL ADVANTAGE OVER SHORT TERM INFUSION IN PATIENTS WITH HIGH-RISK NEUROBLASTOMA. RESULTS FROM THE HR-NBL1/SIOPEN TRIAL. (SIOP 2025) - P2/3 | "All patients received 160 mg/m 2 oral isotretinoin (d19-32). Conclusions We previously reported that LTI of DB increased the safety profile. Here we demonstrate that LTI is also associated with an improved outcome."

Clinical • Neuroblastoma • Solid Tumor • IL2

AFFINITY AFFECTS THE FUNCTIONAL POTENCY OF ANTI-GD2 ANTIBODIES DINUTUXIMAB BETA AND NAXITAMAB BY TARGET-MEDIATED DRUG DISPOSITION (TMDD). (SIOP 2025) - "Finally, sGD2 impaired NAXI-mediated ADCC to a significantly greater extent than DB-mediated ADCC. Conclusions DB has a higher ADCC potency over NAXI at clinically relevant concentrations, attributed to stronger TMDD effects of NAXI compared to DB."

Neuroblastoma • Solid Tumor

IMMUNOTHERAPY OF BONE AND SOFT TISSUE SARCOMAS. EXPERIENCE OF N.N.BLOKHIN NMRCO IN THE APPLICATION ANTI- GD2 - ANTIBODIES IN CHILDREN WITH RECURRENT GD2-POSITIVE SOLID TUMORS. (SIOP 2025) - "Overall one-year survival in patients with relapses was 88.8±10.5%, in patients with refractory course - 60.0±18.2% . Conclusions The using of dinutuximab beta in children with recurrent and refractory forms of bone sarcomas allows to increase survival rates in this group of patients by 30-40%, which is a promising treatment option in patients with an extremely unfavorable prognosis."

Clinical • IO biomarker • Oncology • Osteosarcoma • Sarcoma • Soft Tissue Sarcoma • 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: Jul 2025 ➔ Nov 2025

Trial completion date • Neuroblastoma • Oncology • Solid Tumor

ANBL19P1: Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P2 | N=41 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2025 ➔ Sep 2027

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

Feasibility and activity of chemo-immunotherapy in adolescents and young adults with relapsed/progressive high-risk neuroblastoma: Real-world data from SACHA-France (ESMO 2025) - P | "Eligible patients received anti-GD2 MoAbs (Dinutuximab Beta) in combination with temozolomide-based chemotherapy or topotecan/cyclophosphamide. Conclusions These findings support the safe and feasible use of chemo-immunotherapy in AYA with neuroblastoma, provided it is administered in specialized settings experienced with pediatric regimens. Despite the small sample size, this real-world study highlights the relevance of pediatric-based approaches for this underserved population."

Clinical • Real-world • Real-world evidence • Neuroblastoma • Oncology • Solid Tumor

The role of dinutuximab beta with beta cell function, apoptosis, and proliferation: new approaches to proteomic analysis of insulinoma. (PubMed, Invest New Drugs) - "Several proteins not previously associated, or only indirectly linked, with beta cell function, apoptosis, or proliferation were identified and characterized for the first time in insulinoma. These findings provide new insights and potential targets for the treatment of pancreatic cancer."

IO biomarker • Journal • Oncology • Pancreatic Cancer • Solid Tumor • Targeted Protein Degradation • BCL2 • CASP3 • FN1 • HSPA8 • NEUROD1 • PAX6 • PCNA • PDX1 • PTEN • UBE2L6

NCI-2018-03732: Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P2 | N=42 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Sep 2025 ➔ Sep 2026

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

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2026 ➔ Sep 2030 | Trial primary completion date: Sep 2026 ➔ Sep 2030

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

Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study. (PubMed, Pediatr Blood Cancer) - P | "Chemo-immunotherapy can benefit HR-NBL patients with PD before HDC, including those with progression during/at the end of the induction chemotherapy and NMYC amplification. These findings support its early inclusion in HR-NBL trials."

Journal • Real-world evidence • Neuroblastoma • Oncology • Solid Tumor • MYCN

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Active, not recruiting ➔ Recruiting

Enrollment open • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

Dinutuximab Beta for the Treatment of High-Risk Neuroblastoma: Data from the Hungarian Pediatric Oncology Network. (PubMed, J Clin Med) - "Grade 3 or 4 adverse events (including blood and lymphatic system disorders, hypoxia, hypotension, and capillary leak syndrome) were generally consistent with dinutuximab beta's known safety profile. Dinutuximab beta was an effective immunotherapy for patients with HR-NB in routine clinical practice, with a generally manageable side effect profile."

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

Dinutuximab Beta Versus Naxitamab in the Treatment of Relapsed/Refractory Neuroblastoma in Patients with Stable Disease, Minor Response or Partial Response and Disease in Bone or Bone Marrow: Systematic Review and Matching-Adjusted Indirect Comparison. (PubMed, Cancers (Basel)) - "Sensitivity analyses and unadjusted comparisons supported the results. In the indirect comparison, dinutuximab beta significantly extended PFS and increased ORR compared to naxitamab."

Journal • Neuroblastoma • Oncology • Solid Tumor • CSF2 • IL2 • MYCN