New drug for neuroblastoma! Y-mabs company's gD2 targeted antibody, naxitamab, has applied for listing in the United States, with a total remission rate of 78% in combination with GM-CSF

November 30, 2019 news / BIOON / -- y-mabs is a biopharmaceutical company in the late clinical stage, dedicated to the development and commercialization of new antibody based cancer treatment products Recently, the company announced that it has submitted the first part of the application for approval of biological products for treatment of recurrent / refractory high-risk neuroblastoma (BLA) to the food and Drug Administration (FDA) in its rolling review process Neuroblastoma is a very aggressive tumor and one of the most common tumors in infants Although intensive multimodal therapy has improved the survival rate in recent years, survivors have a high risk of recurrence Naxitamab is a humanized monoclonal antibody targeting gD2 antigen GD2 antigen is expressed on the surface of tumor, including neuroblastoma, melanoma and osteosarcoma By binding to the gD2 antigen on the surface of tumor, naxitamab can trigger the antibody mediated cytotoxicity and activate the complement system in the immune system, so as to achieve the effect of killing tumor In the United States, the FDA has awarded the orphan drug qualification (odd) for the treatment of neuroblastoma and osteosarcoma; in August 2018, the FDA also awarded the breakthrough drug qualification (BTD) for the treatment of high-risk neuroblastoma with GM-CSF Currently, nexitamab is in clinical development for the treatment of recurrent / refractory high-risk neuroblastoma, osteosarcoma and other gD2 positive tumors Breakthrough drug qualification (BTD) certification will help y-mabs interact frequently with the FDA review team The rolling review process allows y-mabs to submit individual parts of the BLA for review, rather than waiting for all parts to be completed before they are submitted to the FDA for review If approved, y-mabs intends to commercialize nexitamab in the U.S market Mechanism of action of nexitamab (photo source: website of y-mabs company) on October 25, y-mabs company announced the latest data of phase I / II 12-230 study (nct01757626) of nexitamab combined with GM-CSF in the treatment of relapsed / refractory high-risk neuroblastoma children at the annual meeting of the international society for children's Oncology (SIOP) held in Lyon, France One subgroup of the study included 28 children with primary refractory high-risk neuroblastoma who had no effect on intensive induction therapy, and more than half of the patients were also refractory to second-line chemotherapy In the study, these patients were treated with nexitamab in combination with GM-CSF The results showed that the objective response rate (ORR) was 78%, and 50% of patients had a PFS of 24 months Another subgroup of the study included 35 children with recurrent neuroblastoma who were resistant to salvage therapy, 30 of whom were evaluable One third of the patients had relapsed twice or more before entering the study, and 89% of the patients had received anti gD2 drug treatment In this group of patients, the overall response rate (ORR) of the combination treatment of nexitamab and GM-CSF was 37%, indicating that the treatment regimen has significant clinical benefits in such refractory patients Dr Claus Moller, CEO of y-mabs, said: "I am proud and appreciative of the y-mabs team and our clinical researchers, who helped achieve this key milestone We believe that this BLA submission is an important milestone for y-mabs and neuroblastoma patients " Original source: y-mabs initiates rolling submission of biology license application to U.S FDA for toxitamab for treatment of neuroplastoma