MB-101 Receives FDA Orphan Drug Designation for Astrocytoma and Glioblastoma

The FDA has granted orphan drug designation to the IL13Rα2-targeted CAR T-cell therapy MB-101 for the treatment of patients with recurrent diffuse and anaplastic astrocytoma and glioblastoma.1 “We are thrilled that MB-101 received orphan drug designation on time and with a designation that is broader than the indication proposed,” Manuel Litchman, MD, president and chief executive officer of Mustang Bio, stated in a news release. “The orphan drug designation for MB-101, coupled with the orphan drug designation granted previously for MB-108, is strong validation for our science, as we hope to advance MB-101, in combination with MB-108, as a potential treatment option for patients living with malignant glioma, including patients with recurrent glioblastoma and high-grade astrocytomas. Our novel therapeutic strategy, combining our MB-101 CAR-T cell therapy with our MB-108 oncolytic virus, leverages MB-108 to reshape the tumor microenvironment to make cold tumors ‘hot,’ thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. This progress demonstrates our dedication to exploring new possibilities for improving outcomes in patients with challenging-to-treat cancers.” MB-101 and MB-108 together are referred to as MB-109; the CAR T-cell therapy was developed at City of Hope in Duarte, California, and MB-108—an HSV-1 oncolytic virus—is being licensed from Nationwide Children’s Hospital in Columbus, Ohio. MB-109 is intended to turn immunologically cold tumors into hot tumors via MB-108 through the recruitment of endogenous CD8- and CD3-positive effector T cells, thus increasing the efficacy of subsequent administration of MB-101.