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FDA Grants Orphan Drug Status to NantCell’s Ganitumab in Ewing Sarcoma

CULVER CITY, Calif.–(BUSINESS WIRE)–NantCell, Inc., part of the NantWorks ecosystem of companies, announced today that the FDA has granted Orphan Drug Designation to the company’s Ganitumab, Insulin Growth Factor-1R (IGF-1R) monoclonal antibody therapy for the treatment of patients with Ewing sarcoma.

Ganitumab is a fully human monoclonal antibody directed against IGF-1R that is being investigated in a Phase III human clinical trial titled “Combination Chemotherapy with or without Ganitumab in Treating Patients with Newly Diagnosed Metastatic Ewing Sarcoma” (NCT02306161) by the National Cancer Institute (NCI) in partnership with Children’s Oncology Group (COG) and NantCell at clinical sites across the United States and Canada.

IGF-1R plays a role in the development and progression of many cancers, possibly due to its anti-apoptotic properties, which allow cancerous cells to resist the cytotoxic properties of chemotherapeutic drugs or radiation therapy. Ganitumab inhibits cancer cell proliferation through disruption of the P13K/Akt and MAPK pathways. Signaling through IGF-1R plays an important role in the regulation of cell growth and survival, and has been shown to be a critical promoter of anchorage independent growth, a well-recognized mechanism for malignancy.

Patrick Soon-Shiong, MD, Chairman and CEO of NantCell, commenting on the FDA’s grant of Orphan Drug Designation, said: “We are very pleased to be granted Orphan Drug Designation for the use of Ganitumab in the treatment of Ewing sarcoma.” Dr. Soon-Shiong continued: “While improvements in treatment options for patients with Ewing sarcoma have improved outcomes, there remains a significant unmet medical need for many patients, particularly those with newly diagnosed metastatic Ewing sarcoma, which is first-line therapy. With the FDA’s award in place, we believe we are in a solid position to accelerate the clinical development of this program and address this significant unmet medical need, especially in children afflicted with this disease.”

About NantCell

NantCell, a wholly-owned subsidiary of NantWorks, LLC, is an immuno-oncology company focused on the discovery of innovative antibody, T cell and NK cell based treatments by developing molecularly targeted therapeutics, based on the proteomic profile of the patient’s tumor, independent of the cancer’s anatomical type.

NantCell’s mission is to make obsolete the standard method of clinical trial design of “trial and error” and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. The Company will tap into comprehensive “omic” analytic tools and “big data” generated from supercomputing to develop molecularly designed drugs in this era of genomics and proteomics and identify patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive “omic” analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects. Through these integrated diagnostic methods, the company is pursuing the vision of treating the biology of cancer rather than the anatomy, and drive the immune system inherited by all to defeat cancer. For more information please visit and follow Dr. Soon-Shiong on Twitter @Dr.PatSoonShiong.

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