NantCell announces FDA grant of orphan drug designation for the company’s Ganitumab monoclonal antibody therapy in Ewing sarcoma.

NantCell, Inc., part of the NantWorks system of companies, announced this week 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.

“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,” Patrick Soon-Shiong, M.D., Chairman and CEO of NantCell, commented. “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.”

(Source: Business Wire)