Trial investigators at City of Hope announce
first-reported complete response from a CAR T therapy in a BPDCN
patient, additional complete response achieved in AML
Mustang Bio, Inc. (“Mustang”) (NASDAQ:MBIO), a Fortress Biotech
(NASDAQ:FBIO) Company focused on the development of novel
immunotherapies based on proprietary chimeric antigen receptor
engineered T cell (“CAR T”) technology, today announced that
investigators at City of Hope have reported that Mustang’s MB-102
(CD123 CAR) CAR T therapy is safe and well tolerated and achieved
the first-ever complete response (CR) from a CAR T therapy in
blastic plasmacytoid dendritic cell neoplasm (BPDCN), as well as a
CR in acute myeloid leukemia (AML), in an ongoing Phase 1 clinical
trial (NCT02159495). The data were presented by City of Hope today
in an oral session at the 59th American Society of Hematology (ASH)
Annual Meeting.
Manuel Litchman, M.D., President and Chief
Executive Officer of Mustang, said, “According to investigators at
City of Hope, these data demonstrate MB-102’s potential to be a
safe, well tolerated and effective CAR T therapy that can achieve
complete disease response. In addition, MB-102’s promising
anti-leukemic activity in both AML and BPDCN supports further
evaluation in clinical trials in transplant eligible and ineligible
patients. We are thrilled to report that our CAR Ts have now
achieved complete responses in three disease areas, MB-102 in AML
and BPDCN, and our MB-101 IL13Rα2-specific CAR T in glioblastoma,
which was published in December 2016 in the New England Journal of
Medicine.”
Elizabeth Lihua Budde, M.D., Ph.D., assistant
professor in the department of Hematology & Hematopoietic Cell
Transplantation at City of Hope and principal investigator for the
Phase 1 trial, said, “Current treatment options in AML are
associated with low rates of complete response and limited
progression to allogeneic hematopoietic stem cell transplantation.
Moreover, BPDCN is a rare and incurable blood cancer with no
standard of care. CD123 is overexpressed in both AML and BPDCN,
making it an attractive target in these diseases, which have clear
unmet therapeutic needs. We are encouraged by these interim data
that demonstrate MB-102’s potential to be a new or improved
treatment option in BPDCN and AML, and look forward to continuing
to evaluate the clinical benefits of MB-102 in our ongoing Phase 1
clinical trial.”
Key Efficacy and Safety Findings
This single center, first-in-human Phase 1
dose-escalation clinical trial is evaluating the safety and
activity of escalating doses of MB-102 in patients with relapsed or
refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a
single dose of MB-102 with an option for a second infusion if they
continue to meet safety and eligibility criteria and still have
CD123+ disease. To date, 14 patients have been enrolled and seven
have been treated (six with AML, one with BPDCN) in this first
in-human trial for AML and BPDCN patients using a CD123 CAR T
therapy.
In the AML cohort, two patients were treated at
dose level 1 (50M CAR+ T). Trial investigators reported that one
achieved a morphologic leukemic-free state at day 28 post-infusion.
Four patients received dose level 2 (200M CAR+ T), with a CR
observed at day 28 in one patient, and a CR with incomplete blood
count recovery demonstrated at day 28 in a second patient. Both
patients proceeded to a second allogeneic hematopoietic stem cell
transplantation.
In the BPDCN cohort, one patient received a
single dose of 100M CAR+ T and achieved a CR at day 28, which
lasted at least 60 days, according to investigators. Of note, this
patient had previously experienced disease progression following
five cycles of treatment with a CD123-targeted recombinant fusion
protein.
Investigators found MB-102 infusions of up to
200M CAR T cells were safe, with no graft-versus-host disease,
myeloablative effects, neurologic toxicity or dose-limiting
toxicities. Adverse events (AEs) included: cytokine release
syndrome (six grade 1, one grade 2), neurotoxicity (dizziness: one
grade 1, two grade 2; headache: five grade 1, two grade 2;
somnolence: one grade 1, two grade 2), three cases of infection
(lung infection: two, other: one). The most common ≥ grade 3
AEs included lymphopenia (seven), thrombocytopenia (seven) and
febrile neutropenia (six).
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the myeloid line of
blood cells characterized by rapid growth of abnormal white blood
cells that accumulate in the bone marrow. Although AML is rare,
there are approximately 20,000 new cases in the U.S. each year and
10,000 deaths. Current treatment of relapsed or refractory AML with
chemotherapy or hematopoietic stem cell transplantation is
associated with low rates of complete response and considerable
complications.
CD123 is overexpressed on AML blasts and
leukemic stem cell-enriched cell subpopulations compared to normal
hematopoietic stem cells and myeloid progenitors, making CD123 an
attractive target for T cell-based adoptive immunotherapy.
About Blastic Plasmacytoid Dendritic
Cell Neoplasm Blastic plasmacytoid dendritic cell neoplasm
(BPDCN) is a rare and incurable blood cancer with a median survival
of less than 18 months and no standard of care. High levels of
CD123 expression is one of the diagnostic hallmarks of BPDCN,
making CD123 an attractive target for T cell-based adoptive
immunotherapy.
About MB-102 (CD123 CAR)MB-102 (CD123CAR) is a
CAR T cell therapy that engineers patient T cells to recognize and
eliminate CD123-expressing tumors. CD123 is widely expressed on
human hematologic malignancies including acute myeloid leukemia
(AML), B cell acute lymphoblastic leukemia, hairy cell leukemia,
blastic plasmacytoid dendritic cell neoplasm (BPDCN), chronic
myeloid leukemia and Hodgkin’s lymphoma.
MB-102 has demonstrated anti-AML activity in
preclinical studies (Mardiros, Blood 2013), and is currently being
evaluated in a Phase 1 clinical trial in AML and BPDCN at City of
Hope (NCT02159495).
About Mustang BioMustang Bio, Inc., a
subsidiary of Fortress Biotech, Inc., is a clinical‐stage
biopharmaceutical company focused on the development and
commercialization of novel cancer immunotherapy products designed
to leverage 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, funding
research and development, and outlicensing or bringing the
technologies to market. Mustang has partnered with the City of Hope
National Medical Center (“COH”) and the Fred Hutchinson Cancer
Research Center in the development of proprietary chimeric antigen
receptor (“CAR”) engineered T cell (“CAR T”) therapies across many
cancers, and with Harvard Medical School’s Beth Israel Deaconess
Medical Center and the Harvard Stem Cell Institute for the
development of CRISPR/Cas9-enhanced CAR T therapies in hematologic
malignancies and solid tumors. Mustang’s lead programs are in Phase
1 clinical trials at COH: MB-101 for the treatment of brain cancer
and MB-102 as a therapeutic agent in acute myeloid leukemia and
blastic plasmacytoid dendritic cell neoplasm. Mustang is registered
under the Securities Exchange Act of 1934, as amended, and files
periodic reports with the U.S. Securities and Exchange Commission.
For more information, visit www.mustangbio.com.
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. 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 licensing arrangements, 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, each as amended. Such
statements include, but are not limited to, any statements relating
to our growth strategy and product development programs and any
other statements that are not historical facts. Forward-looking
statements are based on management’s current expectations and are
subject to risks and uncertainties that could negatively affect our
business, operating results, financial condition and stock value.
Factors that could cause actual results to differ materially from
those currently anticipated include: risks relating to our growth
strategy; our ability to obtain, perform under and maintain
financing and strategic agreements and relationships; risks
relating to the results of research and development activities;
risks relating to the timing of starting and completing clinical
trials; uncertainties relating to preclinical and clinical testing;
our dependence on third-party suppliers; our ability to attract,
integrate and retain key personnel; the early stage of products
under development; our need for substantial additional funds;
government regulation; patent and intellectual property matters;
competition; as well as other risks described in our SEC filings.
We expressly disclaim any obligation or undertaking to release
publicly any updates or revisions to any forward-looking statements
contained herein to reflect any change in our expectations or any
changes in events, conditions or circumstances on which any such
statement is based, except as required by law.
Contacts: Jaclyn JaffeMustang Bio, Inc.(781)
652‐4500ir@mustangbio.com
Fortress Biotech Media RelationsLaura Bagby6 Degrees(312)
448-8098lbagby@6degreespr.com
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