CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 2, 2015--
bluebird bio, Inc. (Nasdaq:BLUE) a clinical-stage company committed to
developing potentially transformative gene therapies for severe genetic
and rare diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted Breakthrough Therapy designation to
LentiGlobin® BB305 Drug Product for the treatment of
transfusion-dependent patients with beta-thalassemia major.
LentiGlobin BB305 Drug Product aims to treat beta-thalassemia major and
severe sickle cell disease by inserting a functional human beta-globin
gene into the patient's own hematopoietic stem cells ex vivo and
then returning those modified cells to the patient through an autologous
stem cell transplantation.
"The FDA's Breakthrough designation of LentiGlobin highlights that new
therapies are needed for the treatment of patients with beta-thalassemia
major, especially treatments with the potential to meaningfully reduce
or liberate patients from transfusion dependence," said David Davidson,
M.D., chief medical officer of bluebird bio. "Our early clinical data
investigating the use of LentiGlobin in patients with multiple genotypes
of beta-thalassemia major, including beta-0/beta-0, the most severe
genotype, are very encouraging, and we remain on track to complete
enrollment in the Northstar and HGB-205 studies in 2015. In light of the
Breakthrough designation, we look forward to working even more closely
with the FDA to expedite the development of LentiGlobin for the
treatment of beta-thalassemia major."
The FDA's Breakthrough Therapy designation is intended to expedite the
development and review of a drug candidate that is planned for use to
treat a serious or life-threatening disease or condition when
preliminary clinical evidence indicates that the drug may demonstrate
substantial improvement over existing therapies on one or more
clinically significant endpoints. The benefits of Breakthrough Therapy
designation include the same benefits as Fast Track designation, plus an
organizational commitment involving FDA’s senior managers with more
intensive guidance from the FDA. Breakthrough Therapy designation does
not however change the standards for approval.
The Breakthrough Therapy designation is supported by data from the
ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of
LentiGlobin. Findings in eight subjects with beta-thalassemia major were
presented at the 56th Annual Meeting of the American Society of
Hematology (ASH) in December 2014. In the first four subjects, each of
whom had at least three months of follow up, treatment resulted in
sufficient hemoglobin production to reduce or eliminate the need for
transfusion support among patients with beta-thalassemia major who would
otherwise require chronic blood transfusions. These data consisted of
the first five subjects treated in bluebird bio's ongoing Northstar
Study and the first three subjects from its HGB-205 study. These
included the first beta-thalassemia subjects with the beta-0/beta-0
genotype to be treated with LentiGlobin BB305 drug product. The HGB-205
study also included the first subject with sickle cell disease to be
treated with gene therapy.
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 a preclinical CAR T cancer immunotherapy program
in collaboration with Celgene Corporation, 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 potential efficacy and safety of the Company’s
LentiGlobin product candidate and the regulatory pathway afforded by
Breakthrough Designation by the FDA, in particular statements concerning
the reduced or eliminated need for transfusion support in the four
initial subjects treated with LentiGlobin drug product, statements
concerning the Company’s future plans with respect to LentiGlobin and
its other product candidates and statements concerning anticipated
enrollment rates and clinical milestones in 2015. It should be noted
that the data for LentiGlobin announced from the Northstar and HGB-205
studies at the ASH Annual Meeting are preliminary in nature and the
Northstar and HGB-205 studies are not completed. There is limited data
concerning long-term safety and efficacy following treatment with
LentiGlobin drug product. These data may not continue for these subjects
or be repeated or observed in ongoing or future studies involving our
LentiGlobin product candidate, including the HGB-205 Study, the
Northstar Study or the HGB-206 study in sickle cell disease. It is
possible that subjects for whom periodic transfusion support has been
reduced or temporarily eliminated may receive transfusion support in the
future. It should also be noted that Breakthrough designation does not
change the standards for approval and is not a guarantee of success. 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 the preliminary results from our clinical trials will
not continue or be repeated in our ongoing clinical trials, the risk
that previously conducted studies involving similar product candidates
will not be repeated or observed in ongoing or future studies involving
current product candidates, the risk of cessation or delay of any of the
ongoing or planned clinical studies and/or our development of our
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 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
quarterly 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.
Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc.
Jim DeTore,
339-499-9355
Chief Financial Officer
or
Media:
Pure
Communications, Inc.
Dan Budwick, 973-271-6085
