ch14.18 for Neuroblastoma

In July 2010, United Therapeutics entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to collaborate on the late-stage development and commercialization of chimeric (ch)14.18 for the treatment of neuroblastoma. Pivotal Phase 3 clinical study results for Study ANBL0032 sponsored by NCI and conducted by the Children’s Oncology Group (COG) were published in the New England Journal of Medicine (September 2010).  The results described that immunotherapy with ch14.18 granulocyte-macrophage colony stimulating factor (GM-CSF) and interleukin-2 (IL-2) significantly improved patient outcome compared with standard therapy in patients with high risk neuroblastoma.  Specifically, the 2-year estimate for event-free survival was 66±5% in the immunotherapy group and 46±5% in the standard therapy group (p=0.01).  The immunotherapy group was also significantly better than the standard therapygroup in the estimated rate of overall survival (86±4% vs. 75±5% at 2 years, p=0.02 without adjustment for interim analyses).   United Therapeutics plans to manufacture ch14.18 in its Silver Spring, Maryland manufacturing facility and complete the necessary development work for filing a BLA.