On June 4, 2017 Y-mAbs Therapeutics, Inc. (YmAbs), an immunotherapy company discovering and developing innovative treatments for patients with cancer, reported positive top line results from a pivotal study of 131I-burtomab in Refractory Leptomeningeal Metastasis from Neuroblastoma (Press release, Y-mAbs Therapeutics, JUN 4, 2017, View Source;study-of-burtomab-in-refractory-leptomeningeal–metastasis-from-neuroblastoma/ [SID1234519467]). Results showed a 58 months average survival for the patients treated with 131I-burtomab in the study, compared to an average of 4.7 month and no long term survival or cure, for a contemporary cohort in the Central German Childhood Cancer Registry . After more than a decade of follow-up, data shows more than 40% overall long term survival indicating that the treated children have been cured.
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At ASCO (Free ASCO Whitepaper), Dr. Kim Kramer from Memorial Sloan Kettering Cancer Center (MSK) presented the overall survival data from 80 pediatric patients with CNS and leptomeningeal metastasis from neuroblastoma treated at MSK with 131I-burtomab, and Prof. Dr. med. Frank Berthold from the University of Cologne presented comparable historical data from the Central German Childhood Cancer Registry, thereby demonstrating that the use of 131I-burtomab led to a multi-fold increase of the average survival in this patient population, where no other established therapy exists.
YmAbs Founder, President and Head of Business Development and Strategy, Thomas Gad said, "Having witnessed this out-patient treatment first hand as a parent, these data provide a potential curative treatment addressing an unmet medical need for a life-threatening disease to children suffering from Refractory Leptomeningeal Metastasis from Neuroblastoma. YmAbs is now positioned to seek the most efficient route to approval. This could be a potential game changer for pediatric patients and their families."
Dr. Claus Møller, Chief Executive Officer further notes, "In a setting, where no other therapies are approved, this survival data represents a true breakthrough and hope for these children."