Y-mAbs Therapeutics announced interim Phase 2 trial data published in Nature Communications, showing naxitamab combined with GM-CSF is effective and safe for relapsed/refractory high-risk neuroblastoma with residual bone/bone marrow disease. The trial involved administering naxitamab on days 1, 3, and 5 with GM-CSF until a complete or partial response, followed by five additional cycles. The primary endpoint, overall response rate, was achieved.
This positive data is crucial for children with high-risk neuroblastoma, a challenging cancer with limited treatment options, especially for those with persistent disease in bone and bone marrow. The trial focused on this specific patient population, offering a potential new therapeutic avenue for those who haven’t responded to other treatments, including other anti-GD2 therapies. This opens the possibility of improved outcomes for these young patients.
The trial met its primary endpoint with a 50% overall response rate, comprised of 38% complete responses and 12% partial responses. Importantly, responses were observed even in patients previously treated with other anti-GD2 antibodies. One-year overall survival was 93%, and progression-free survival was 35%. Side effects were primarily infusion-related and manageable, mainly hypotension and bronchospasm.
This data reinforces naxitamab’s position as a valuable treatment option for this patient population. The results support further investigation and potential expansion of naxitamab’s use in high-risk neuroblastoma, offering renewed hope for improved survival and quality of life for affected children. This could lead to changes in treatment protocols and ultimately improve the prognosis for this aggressive childhood cancer.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
