bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to
developing potentially transformative gene therapies for severe genetic
and rare diseases and T cell-based immunotherapies, today announced that
its existing global collaboration agreement with Celgene Corporation
(Nasdaq: CELG) has been amended and restated to focus on developing
product candidates targeting B-cell maturation antigen (BCMA) during a
three-year collaboration term. The original collaboration, initiated in
2013, was focused on applying gene therapy technology to genetically
modify a patient’s own T cells to target and destroy cancer cells. BCMA
is the first target selected to advance to the clinic under this
collaboration. BCMA is a cell surface protein that is expressed in
normal plasma cells and in most multiple myeloma cells, but is absent
from other normal tissues.
Celgene and bluebird bio will work collaboratively on the initial, lead
anti-BCMA product candidate (bb2121), with a Phase 1 clinical trial
expected to begin enrollment in early 2016, and develop next-generation
anti-BCMA product candidates. bluebird bio retains sole rights to
develop all other chimeric antigen receptor (CAR) T cell programs
developed by bluebird bio under the collaboration, including ongoing
undisclosed preclinical programs with opportunities in both solid tumors
and hematologic malignancies.
Under the terms of the amended and restated collaboration agreement:
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bluebird bio will receive a $25 million payment to develop the lead
anti-BCMA product candidate through a Phase 1 clinical trial and
develop next-generation anti-BCMA product candidates.
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bluebird bio will be responsible for the development of all anti-BCMA
product candidates through the completion of Phase 1 studies.
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Additionally, on a product-by-product basis within the anti-BCMA
product program, Celgene has an option to develop and commercialize
each product candidate worldwide, and bluebird bio has the option to
share equally in the development, promotion and profits of each
product candidate in the United States. In addition to the payments
described above and consistent with the prior agreement, Celgene would
also pay bluebird bio specified development and regulatory milestone
payments as well as royalty payments on net sales.
“We have successfully achieved the initial goal of our collaboration
with Celgene —identifying a promising lead development candidate in the
CAR T cell field — and we are excited to focus our Celgene collaboration
on the development of anti-BCMA products,” said Nick Leschly, chief
bluebird. “Celgene is a leader in developing and commercializing
therapies for multiple myeloma, and we believe they are the best global
partner for our first CAR T program. Together we look forward to
entering the clinic early next year with bb2121 and continuing our
collaboration around next-generation BCMA products.”
“Our collaboration with bluebird bio has collectively made strong
progress advancing a lead product candidate, targeting BCMA, toward the
clinic in hematologic malignancies. We look forward to continuing to
work with bluebird and build on the recent success to advance the
anti-BCMA program and ultimately, to succeed on the goal of delivering a
high-impact therapeutic in the CAR T arena,” said Tom Daniel, M.D.,
President of Research and Early Development at Celgene.
A Unique Position in Immuno-oncology
Outside of BCMA,
bluebird bio is independently pursuing the development of a broad
portfolio of novel immuno-oncology therapeutics. This portfolio will
leverage bluebird bio technology, including its lentiviral vector
platform, gene editing capabilities and internal gene therapy and
immuno-oncology expertise.
“Consistent with the long-term vision for bluebird bio, we are expanding
our immuno-oncology T cell-based efforts in parallel with our late-stage
hematopoietic stem cell-based programs,” said Rob Ross, M.D., senior
vice president, clinical development, bluebird bio. “Our goal is to
initiate multiple clinical trials over the next several years against
novel targets in both solid and hematologic malignancies by integrating
our proprietary lentiviral and gene editing platforms with our
immuno-oncology expertise and experience in implementing multi-center,
industry-sponsored gene therapy studies. This is an exciting step in
bluebird bio’s evolution and an opportunity for us to further realize
the value that gene therapy can bring to patients and families in need.”
Currently, bluebird bio has active pre-clinical research programs
targeting multiple different, novel oncology antigens, including BCMA.
These programs include various T cell therapies with significant
academic and industry collaborations. bluebird bio recently announced a
strategic collaboration with Five Prime Therapeutics, which marries Five
Prime’s antigen discovery platform and certain human antibodies with
bluebird bio’s immuno-oncology and gene therapy capabilities, with the
potential for development candidates in both hematologic malignancies
and solid tumors.
Investor Conference Call and Webcast Information
bluebird
bio will host a conference call and webcast at 4:30 p.m. ET on
Wednesday, June 3, 2015 to review its strategy and development plans in
immuno-oncology. The event will be webcast live and can be accessed
under "Calendar of Events" in the Investors and Media section of the
company's website at www.bluebirdbio.com.
Alternatively, investors may listen to the call by dialing (844)
825-4408 from locations in the United States or (315) 625-3227 from
outside the United States.
About bluebird bio, Inc.
With its lentiviral-based gene
therapy and gene editing capabilities, bluebird bio has built an
integrated product platform with broad potential application to severe
genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical
programs include Lenti-D™, currently in a Phase 2/3 study, called the
Starbeam Study, for the treatment of childhood cerebral
adrenoleukodystrophy, and LentiGlobin®, currently in three
clinical studies: a global Phase 1/2 study, called the Northstar Study,
for the treatment of beta-thalassemia major; a single-center Phase 1/2
study in France (HGB-205) for the treatment of beta-thalassemia major or
severe sickle cell disease; and a separate U.S. Phase 1 study for the
treatment of sickle cell disease (HGB-206). bluebird bio also has
ongoing preclinical CAR T immuno-oncology programs, as well as discovery
research programs utilizing megaTALs/homing endonuclease gene editing
technologies.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Washington, and Paris, France. For more information, please visit www.bluebirdbio.com.
Forward-Looking Statements
This release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements regarding
the research, development and advancement of bluebird bio's
immuno-oncology product candidates and CAR T research programs. Any
forward-looking statements are based on management’s current
expectations of future events and are subject to a number of risks and
uncertainties that could cause actual results to differ materially and
adversely from those set forth in or implied by such forward-looking
statements. These risks and uncertainties include, but are not limited
to, the risk that bluebird bio's immuno-oncology research programs will
be unsuccessful and not identify any viable product candidates or will
not be safe or effective in clinical trials, the risk of cessation or
delay of any of the planned clinical studies and/or our development of
our immuno-oncology product candidates, the risk of a delay in the
enrollment of patients in the Company’s clinical studies, the risk that
our collaboration with Celgene around anti-BCMA product candidates will
not continue or will not be successful, and the risk that any one or
more of our product candidates will not be successfully developed and
commercialized. For a discussion of other risks and uncertainties, and
other important factors, any of which could cause our actual results to
differ from those contained in the forward-looking statements, see the
section entitled “Risk Factors” in our most recent annual report on Form
10-Q, as well as discussions of potential risks, uncertainties, and
other important factors in our subsequent filings with the Securities
and Exchange Commission. All information in this press release is as of
the date of the release, and bluebird bio undertakes no duty to update
this information unless required by law.
Availability of other information about bluebird bio
Investors
and others should note that we communicate with our investors and the
public using our company website (www.bluebirdbio.com),
our investor relations website (http://www.bluebirdbio.com/investor-splash.html),
including but not limited to investor presentations and FAQs, Securities
and Exchange Commission filings, press releases, public conference calls
and webcasts. You can also connect with us on Twitter @bluebirdbio,
LinkedIn
or our YouTube
channel. The information that we post on these channels and websites
could be deemed to be material information. As a result, we encourage
investors, the media, and others interested in bluebird bio to review
the information that we post on these channels, including our investor
relations website, on a regular basis. This list of channels may be
updated from time to time on our investor relations website and may
include other social media channels than the ones described above. The
contents of our website or these channels, or any other website that may
be accessed from our website or these channels, shall not be deemed
incorporated by reference in any filing under the Securities Act of 1933.
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