The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial

Jaume Mora (), Godfrey C. F. Chan, Daniel A. Morgenstern, Loredana Amoroso, Karsten Nysom, Jörg Faber, Arthur Wingerter, Melissa K. Bear, Alba Rubio-San-Simon, Blanca Martínez de Las Heras, Karen Tornøe, Maria Düring and Brian H. Kushner
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Jaume Mora: Hospital Sant Joan de Déu
Godfrey C. F. Chan: Queen Mary Hospital & Hong Kong Children’s Hospital
Daniel A. Morgenstern: University of Toronto
Loredana Amoroso: IRCCS Istituto Giannina Gaslini
Karsten Nysom: Copenhagen University Hospital – Rigshospitalet
Jörg Faber: University Medical Center of the Johannes Gutenberg University Mainz
Arthur Wingerter: University Medical Center of the Johannes Gutenberg University Mainz
Melissa K. Bear: Riley Hospital for Children
Alba Rubio-San-Simon: Hospital Infantil Universitario Niño Jesús
Blanca Martínez de Las Heras: Hospital Universitario y Politecnico La Fe
Karen Tornøe: Y-mAbs A/S
Maria Düring: Y-mAbs A/S
Brian H. Kushner: Memorial Sloan Kettering Cancer Center

Nature Communications, 2025, vol. 16, issue 1, 1-13

Abstract: Abstract In this single-arm, non-randomized, phase 2 trial (NCT03363373), 74 patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow (BM) received naxitamab on Days 1, 3, and 5 (3 mg/kg/day) with granulocyte-macrophage colony-stimulating factor (Days -4 to 5) every 4 weeks, until complete response (CR) or partial response (PR) followed by 5 additional cycles every 4 weeks. Primary endpoint in the prespecified interim analysis was overall response (2017 International Neuroblastoma Response Criteria). Among 26 responders (CR + PR) in the efficacy population (N = 52), 58% had refractory disease, and 42% had relapsed disease. Overall response rate (ORR) was 50% (95% CI: 36-64%), and CR and PR were observed in 38% and 12%, respectively. With the 95% CI lower limit for ORR exceeding 20%, the primary endpoint of overall response was met. Patients with evaluable bone disease had a 58% (29/50) bone compartment response (CR, 40%; PR, 18%). BM compartment response was 74% (17/23; CR, 74%). One-year overall survival and progression-free survival (secondary endpoints) were 93% (95% CI: 80-98%) and 35% (95% CI: 16-54%), respectively. Naxitamab-related Grade 3 adverse events included hypotension (58%) and pain (54%). Overall, naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with residual neuroblastoma in bone/BM.

Date: 2025
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DOI: 10.1038/s41467-025-56619-x

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