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GM-CSF + Naxitamab

Phase 2

Neuroblastoma | Monoclonal antibody | Oncology |Y-mAbs Therapeutics, Inc.|Last Updated: Feb 19, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment122
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03363373Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone MarrowPHASE2 RECRUITING 122Apr 3, 2018Apr 1, 2028Feb 19, 202626 United States, Canada +8
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Study Endpoints
Primary Endpoints
Response rate during Naxitamab treatment
101 weeks

Overall objective response rate (ORR) during the Naxitamab treatment period that will be centrally assessed according to the International Neuroblastoma Response Criteria (INRC) modified with 123I-MIBG criteria and following the use of 18F FDG-PET for MIBG non-avid lesions.

Secondary Endpoints
Incidence of adverse events and serious adverse events
101 weeks
Duration of Response (DoR)
101 weeks
Complete Response Rate
101 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GM-CSF + NaxitamabEXPERIMENTALEach investigational cycle is started with 5 days of GM-CSF administered at 250 µg/m2/day in advance of the start of Naxitamab administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, Naxitamab is administered at 3 mg/kg/day on days 1, 3, and 5 totalling 9 mg/kg per cycle. Treatment cycles are repeated every 4 weeks until CR or PR followed by 5 additional cycles every 4 weeks (±1 week). Subsequent cycles are repeated every 8 weeks (±2 weeks) through 101 weeks from first infusion at the discretion of the investigator. After end of treatment patients will enter a long-term follow up for up to 3 years after end of treatment visit.
Interventions
NameTypeDescription
GM-CSF + NaxitamabBIOLOGICALGranulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody
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Eligibility Criteria
Age Range1 Year — N/A
SexALL
Healthy VolunteersNo
Study Sites26

Inclusion Criteria: * Diagnosis of neuroblastoma as defined per International Neuroblastoma Response Criteria * High-risk neuroblastoma patients with either primary refractory disease or incomplete response to salvage treatment (in both cases including stable disease, minor response and partial res...

Countries:United StatesCanadaDenmarkFranceGermanyHong KongIndiaItalySpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT03363373studyFirstPostDate: changed