Phase 1 data suggests MB-101 is safe and
well‐tolerated, capable of eliciting potent anti-tumor response
Mustang Bio, Inc. (“Mustang”), a Fortress Biotech (NASDAQ:FBIO)
Company, today announced that Phase 1 clinical data and
pre-clinical data on its MB-101 (IL13Rα2-specific Chimeric Antigen
Receptor–engineered CAR T cells (CAR T cells)) product candidate in
development for the treatment of glioblastoma were presented by
investigators from the City of Hope (“COH”) in oral sessions at the
21st Annual Meeting and Education Day of the Society for
Neuro-Oncology (“SNO”) in Scottsdale, AZ.
Dr. Lindsay A. Rosenwald, Fortress Biotech’s
Chairman, President and Chief Executive Officer commented, “CAR T
cell therapy has shown promise in treating certain forms of
hematological cancers. However, translating that activity
into solid tumors has been challenging to date. The clinical
data presented at SNO by the investigators from COH suggest MB-101
is safe and well‐tolerated, and capable of eliciting a potent
anti-tumor response in patients with glioblastoma (GBM), a disease
that is almost universally fatal. We believe this is the
first evidence of activity of CAR T cells in the treatment of GBM.
We are very encouraged by the early evidence of anti-tumor activity
with MB-101 with five of seven post-surgical resection patients
showing stable disease for >8 weeks and, in particular, one
patient showing complete response for 7.5 months. Interestingly,
this patient was the only patient of the seven to receive, on a
compassionate use basis, the dual delivery of CAR T cells by both
intracavitary and via intraventricular administration, which
provides systemic CNS delivery of the CAR T cells. We look
forward to continuing to advance MB-101 in Phase 1 studies and
further exploring the dual delivery approach beyond this single
patient experience.”
The following summarizes the oral presentations
on November 18, 2016 at SNO:
Phase I Study of Chimeric Antigen
Receptor–Engineered T cells Targeting IL13Rα2 for the Treatment of
GlioblastomaPresenter: Christine E. Brown, PhD, Heritage Provider
Network Professor of Immunotherapy, Associate Director, T Cell
Therapeutics Research Laboratory, City of Hope National Medical
Center/Beckman Research Institute
The Phase I study presented showed early
clinical data evaluating IL13Rα2-targeted CAR T cell therapy for
the treatment of glioblastoma. On this study, patients are
treated on a four-week therapeutic regimen consisting of three
weekly intracranial infusions of IL13Rα2-specific CAR T cells
followed by one rest week for toxicity and disease
assessment. To date, seven patients have been treated with
local intracavitary delivery of the CAR T cells following surgical
resection.
Some highlights from the presentation
included:
- The treatment was well-tolerated in all patients treated – with
No DLTs or therapy-related SAEs
- No grade 3 or higher toxicities attributed to the therapy were
observed
- No CRS or Neurotoxicity was observed
- Only grade ≤2 fevers, headaches, myalgia, chills
- Best Response: 2 PD, 4 SD for >8-weeks, 1 SD→ CR following
intraventricular CAR T therapy for 7.5 months
Development of murine IL13Rα2-targeted CAR T
cells (mIL13BBζ) for assessment of CAR T cell therapy in syngeneic
glioma modelsPresenter: Darya Alizadeh, PhD, City of Hope National
Medical Center/Beckman Research Institute
The pre-clinical research program presented
discussed a murine IL13Rα2-targeted CAR T cell platform that was
developed to evaluate parameters that impact the efficacy of CAR T
cell therapy. Overall, the development of mIL13BBζ CAR T cells and
its applications will allow researchers to assess factors that may
impact the efficacy of CAR T cells and provide invaluable
information critical for combination therapies and clinical trial
design. These studies may also provide important insights for
improving therapeutic outcomes for patients with 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 percent of all primary
brain tumors and 55.1 percent of all gliomas. There will be an
estimated 12,120 new glioblastoma cases in the U.S. in 2016.
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 EU), it is quite lethal with five-year
survival rates historically less than 10 percent. Chemotherapy with
temozolomide and radiation are shown to extend mean survival from
approximately 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 (IL13Rα2-specific CAR T
cells)IL13Rα2 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 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 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.
About Mustang BioMustang 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 out-licensing or
bringing the technologies to market. Mustang is currently
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, plans to develop
CARs across multiple cancers. Its lead programs in acute myeloid
leukemia and brain cancer are in Phase 1 clinical trials. Mustang
is registered under the Securities Exchange Act of 1934, as
amended, and files periodic reports with the US Securities and
Exchange Commission. For more information, visit
www.mustangbio.com.
Mustang Bio is a majority-owned subsidiary of Fortress
Biotech.
About Fortress BiotechFortress
Biotech, Inc. (“Fortress”) is a biopharmaceutical company dedicated
to acquiring, developing and commercializing novel pharmaceutical
and biotechnology products. Fortress develops and commercializes
products both within Fortress and through certain of its subsidiary
companies, also known as Fortress Companies. Additionally,
Fortress recently acquired a controlling interest in National
Holdings Corporation (NASDAQ:NHLD), a diversified independent
brokerage company (together with its subsidiaries, “NHLD”). In
addition to its internal development programs, Fortress leverages
its biopharmaceutical business expertise and drug development
capabilities and provides funding and management services to help
the Fortress Companies achieve their goals. Fortress and the
Fortress Companies may seek licensings, 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.
Forward-Looking StatementsThis
press release may contain “forward-looking statements” within the
meaning of Section 27A of the Securities Act of 1933 and Section
21E of the Securities Exchange Act of 1934. Such statements
include, but are not limited to, any statements relating to our
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Factors that could cause actual results to differ materially from
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relating to preclinical and clinical testing; our dependence on
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as well as other risks described in our SEC filings. We expressly
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Contacts:
Jaclyn Jaffe
Mustang Bio, Inc.
781-652-4500; ir@mustangbio.com
Fortress Biotech Media Relations
Laura Bagby
6 Degrees
(312) 448-8098
lbagby@6degreespr.com
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