Mustang Bio and City of Hope Announce Initiation of Phase 1 Clinical Trial of MB-101 (IL13Rα2-specific CAR T cells) to Treat Leptomeningeal Brain Tumors

Trial underway at City of Hope to evaluate safety and feasibility of administering therapy in patients with brain tumors such as glioblastoma, ependymoma or medulloblastoma

WORCESTER, MA, USA I December 21, 2020 I Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, and City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that a Phase 1 single-center, two-arm clinical trial has been initiated to establish the safety and feasibility of administering MB-101 (autologous IL13Rα2-CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma). The trial will enroll up to 30 patients and take place at City of Hope, where the chimeric antigen receptor T (“CAR T”) cell therapy was initially developed.

All subjects enrolled in the trial will undergo surgery for the placement of an intraventricular (ICV) Rickham catheter for CAR T cell delivery. The Phase 1 trial will establish the safety and feasibility of administering MB-101 through the ICV Rickham catheter over four weekly cycles in patients with glioblastoma (Arm 1) and ependymoma or medulloblastoma (Arm 2). The primary endpoints that will be evaluated are toxicity and survival at three months. Secondary endpoints include overall survival, CAR T and endogenous T cell levels, cytokine levels and phenotype detection in peripheral blood, tumor cyst fluid and cerebrospinal fluid.

Lisa Feldman, M.D., Ph.D., a neurosurgeon and assistant clinical professor in the Division of Neurosurgery at City of Hope and principal investigator of the clinical trial, commented, “Leptomeningeal brain tumors are a form of metastatic brain cancer, which is currently very difficult to treat. We are encouraged by the potential of administering autologous IL13Rα2-CAR T cells intraventricularly to patients with leptomeningeal brain tumors. This CAR T cell therapy has demonstrated early safety and efficacy results in a previous clinical trial conducted at City of Hope, and we believe these preliminary results warrant further evaluation of these CAR T cells. We look forward to providing updates on the trial and to continue working closely with Mustang with the goal of bringing a safe and effective treatment option to patients with this life-threatening disease.”

Manuel Litchman, M.D., president and chief executive officer of Mustang, said, “We are pleased to further study MB-101 in leptomeningeal brain tumors as it has already demonstrated therapeutic potential when infused into the ventricular system, including delivering a complete response in a leptomeningeal glioblastoma patient that was published in the New England Journal of Medicine. Our ongoing work with City of Hope continues to advance the research of our CAR T portfolio to bring potential therapies to patients suffering from devastating diseases.”

Additional information about the trial can be found on using the identifier NCT04661384.

About MB‐101 (IL13Rα2‐specific CAR T cells)
IL13Rα2 is an attractive target for CAR T therapy as it has limited expression in normal tissue but is overexpressed on the surface of the majority of malignant glioma cells, including glioblastoma multiforme, ependymoma and medulloblastoma. CAR T cells are designed to express a membrane‐tethered IL‐13 receptor ligand (IL‐13) incorporating a single‐point mutation that provides high affinity for IL13Rα2 and reduces binding to IL13Rα1 in order to reduce healthy tissue targeting. Mustang is developing MB‐101 as an optimized CAR T product incorporating enhancements in CAR design and T cell engineering to improve antitumor potency and T cell persistence. MB‐101 includes a second‐generation hinge optimized CAR containing mutations in the IgG4 linker to reduce off‐target Fc interactions, the 4-1BB (CD137) co‐stimulatory signaling domain for improved persistence of CAR T cells and the extracellular domain of CD19 as a selection/safety marker. To further improve persistence, central memory T cells are enriched and genetically engineered using a manufacturing process that limits ex vivo expansion to reduce T cell exhaustion and maintain a memory T cell phenotype. 

About Mustang Bio
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (“SEC”). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit

About City of Hope
City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hope’s translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nation’s “Best Hospitals” in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on FacebookTwitterYouTube or Instagram.

SOURCE: Mustang Bio

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