Full Press Release Details
Mustang Bio Announces Oral Presentation
of Initial Clinical Data for MB-101 (IL13R 2-specific CAR T cells) by City of Hope Investigators at the American Society
of Gene and Cell Therapy 19th Annual Meeting
Early clinical findings suggest IL13R 2-targeted
CAR T cells is safe and well-tolerated, and capable of eliciting potent antitumor responses against recurrent multifocal glioblastoma
New York, NY - May 5, 2016
- Mustang Bio, Inc. ("Mustang"), a Fortress Biotech (NASDAQ: FBIO) Company, today announced that an oral presentation
related to its MB-101 (IL13R 2-specific CAR T cells) product candidate in development was presented by City of Hope ("COH")
Investigators at the American Society of Gene and Cell Therapy 19th Annual Meeting (ASGCT) at the Marriott Wardman Park
Hotel in Washington, DC.
Michael S. Weiss, Mustang Bio's Executive
Chairman, Interim Chief Executive Officer and President commented on the data, "We are very encouraged by the early safety
and efficacy profile demonstrated by MB-101 (IL13R 2-specific CAR T cells) to date. We were particularly impressed with the
data presented today showing a patient treated at the first dose level who obtained an investigator designated complete response
to MB-101. We believe this is the first demonstration of a response in a GBM patient utilizing CAR-T treatment. We are eager to
continue the dose escalation to further assess the safety, activity and durability of MB-101 treatment at higher doses."
Mr. Weiss continued, "We would like to thank the investigators at City of Hope for all of their efforts on this important
The following summarizes the oral presentation
Phase I Study of Second Generation Chimeric
Antigen Receptor-Engineered T cells Targeting IL13R 2 for the Treatment of Glioblastoma
The Phase I study abstract that was presented
showed early data evaluating the safety, feasibility and bioactivity of weekly intracranial infusions of autologous IL13R 2-specific
CAR T cells in patients with recurrent IL13R 2+ GBM. On this study, patients are treated on a 4-week therapeutic regimen
consisting of 3 weekly intracranial infusions of IL13R 2-specific CAR T cells followed
by one rest week for toxicity and disease assessment. To date, treatment of the first low dose cohort of patients has been completed
demonstrating that local delivery of IL13R 2-specific CAR T cells post-surgical resection
appeared to be safe and well-tolerated, with no grade 3 or higher toxicities attributed to the therapy reported. Importantly, early
evidence for antitumor activity following CAR T cell administration was observed.
One patient of particular interest presented
with a recurrent multifocal GBM, including one metastatic site in the spine and extensive leptomeningeal disease. This patient
was initially treated per protocol with six local infusions of IL13R 2-specific CAR T cells
into the resection cavity of the largest recurrent tumor focus in the posterior temporal-occipital region. Encouragingly, this
CAR T cell injection site remained stable without evidence of disease recurrence for over 7-weeks, while other disease foci distant
from the CAR T cell injection site continued to progress. This patient was then treated on a compassionate use protocol with five
weekly intraventricular infusions of IL13R 2-specific CAR T cells without any other
therapeutic interventions. The investigator reported today that following treatment the patient achieved a complete response. Early
clinical findings suggest that intracranial delivery of second-generation IL13R 2-targeted CAR T cells is safe and well-tolerated,
and that after adoptive transfer, CAR T cells survive and maintain activity, capable of eliciting potent antitumor responses against
recurrent multifocal glioblastoma.
About Glioblastoma multiforme (GBM)
Glioblastomas (GBM) are tumors that arise
from astrocytes cells that make up the supportive tissue of the brain. These tumors are usually highly malignant (cancerous) because
the cells reproduce quickly and they are supported by a large network of blood vessels. GBM is the most common brain and central
nervous system (CNS) malignancy, accounting for 15.1% of all primary brain tumors, and 55.1% of all gliomas. There are an estimated
12,120 new glioblastoma cases predicted in 2016 in the U.S. Malignant brain tumors are the most common cause of cancer-related
deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19 year olds in the U.S. (Brain
Tumor Statistics. American Brain Tumor Association. December 2015). While GBM is a rare disease (2-3 cases per 100,000 person life
years in the U.S. and E.U.), it is quite lethal with 5-year survival rates historically less than 10%. Chemotherapy with temozolomide
and radiation are shown to extend mean survival from ~12 to ~15 months, while surgery remains the standard of care. GBM remains
difficult to treat due to the inherent resistance of the tumor to conventional therapies. Treatment is further complicated by the
susceptibility of the brain to damage, difficulty of the brain to repair itself and limitation to drugs crossing the blood brain
barrier. Immunotherapy approaches targeting brain tumors offer promise over conventional treatments.
About MB-101 (IL13R 2-specific CAR-T cells)
IL13R 2 is an attractive target for
CAR-T therapy as it has limited expression in normal tissue but is over-expressed on the surface of the majority of GBM. CAR T
cells designed to express a membrane-tethered IL-13 receptor ligand (IL-13) incorporating a single point mutation that provides
high affinity for IL13R 2 and reduces binding to IL13R 1 in order to reduce healthy tissue targeting.
We are developing an optimized CAR-T product
incorporating enhancements in CAR design and T cell engineering to improve antitumor potency and T cell persistence. We include
a second generation hinge optimized CAR containing mutations in the IgG4 linker to reduce off-target Fc interactions, as well as
the 41BB (CD137) co-stimulatory signaling domain for improved persistence of CAR T cells, and extracellular domain of CD19 as a
selection/safety marker. In order to further improve persistence, central memory T cells are enriched and genetically engineered
using a manufacturing process that limits ex vivo expansion in order to reduce T cell exhaustion and maintain a memory T cell phenotype.
Mustang Bio, Inc., a Fortress Biotech Company,
is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel cancer immunotherapy products
designed to utilize the power of the patient's own immune system to eliminate cancer cells. Mustang aims to acquire rights
to these technologies by licensing or otherwise acquiring an ownership interest in the technologies, funding their research and
development and eventually either out-licensing or bringing the technologies to market. Currently Mustang is developing proprietary
Chimeric Antigen Receptor (CAR) engineered T cells (CAR-T) technology, which was licensed from Drs. Stephen Forman and Christine
Brown's laboratory at the City of Hope National Medical Center (COH). CAR-T uses the patient's own T cells to engage
and destroy specific tumors. The process involves selecting specific T cell subtypes, genetically engineering them to express chimeric
antigen T cell receptors and placing them back in the patient where they recognize and destroy cancer cells. Mustang, through a
research agreement with COH, will develop CARs across multiple cancers, including for AML and Brain Cancer. Both of the lead programs
are in Phase I clinical trials. For more information, visit www.mustangbio.com.
Mustang Bio is a majority-owned subsidiary of Fortress Biotech.
About Fortress Biotech
Fortress Biotech, Inc. ("Fortress"
or "the Company") is a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical
and biotechnology products. Fortress plans to develop and commercialize products both within Fortress and through subsidiary companies,
also known as Fortress Companies. In addition to its internal development programs, the Company will leverage its biopharmaceutical
business expertise and drug development capabilities to help the Fortress Companies achieve their goals. Additionally, the
Company will provide funding and management services to each of the Fortress Companies and, from time to time, the Company and
the Fortress Companies will seek licensing, acquisitions, partnerships, joint ventures and/or public and private financings to
accelerate and provide additional funding to support their research and development programs. For more information, visit www.fortressbiotech.com.
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