CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 9, 2015--
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, announced today the
completion of the National Institutes of Health (NIH) Recombinant DNA
Advisory Committee’s (RAC) public review of the HGB-208 pediatric study
protocol for bluebird bio’s LentiGlobin BB305 product candidate in
beta-thalassemia major. The RAC recommendation was to delay initiation
of the study in the United States for one to two years. This
recommendation has no effect on the HGB-207 protocol plan.
“We appreciate the recommendations from the RAC members regarding the
HGB-208 pediatric study protocol,” said David Davidson, M.D., chief
medical officer. “We will take the RAC feedback on the timing of
initiating HGB-208 under advisement as we advance the clinical
development of our LentiGlobin BB305 product candidate for patients with
beta-thalassemia major. The HGB-207 trial protocol did not require
further review by the RAC, and we will continue to work closely with the
regulatory authorities and our clinical study sites to pursue
appropriate accelerated regulatory approval pathways in the U.S. and the
EU.”
Background on RAC Process
The outcome of RAC review is a series of recommendations and advice. RAC
review does not constitute a formal approval of a proposed protocol. The
recommendations are captured in a summary letter, which will be sent to
the institutional review boards (IRBs) reviewing the protocol in the
United States. The recommendations are non-binding.
Background on HGB-207 and HGB-208 Clinical Studies
In May, bluebird bio announced that it had reached general agreement
with the U.S. Food and Drug Administration (FDA) on the design of its
planned clinical trials HGB-207 and HGB-208. Based on its discussions
with the FDA, bluebird bio believes that data from these trials,
together with data from the ongoing beta-thalassemia major clinical
studies (Northstar and HGB-205), could form the basis for a Biologics
License Application (BLA) submission for LentiGlobin BB305. HGB-207 and
HGB-208 share similar trial designs and are differentiated primarily by
patient age. HGB-207 will enroll adult and adolescent patients; HGB-208
is planned to enroll pediatric patients.
Highlights:
-
Sample size: 15 patients per trial
-
Duration: 24 months of follow-up per patient
-
Primary endpoint: 12 months of transfusion independence
The RAC had previously notified bluebird bio that only HGB-208 required
a public RAC discussion.
bluebird bio also announced in May that it is one of the first companies
to participate in the European Medicines Agency’s (EMA) Adaptive
Pathways (formerly referred to as Adaptive Licensing) pilot program,
which is part of the EMA’s efforts to improve timely access for patients
to new medicines. Based on several discussions involving the EMA,
European Health Technology Assessment (HTA) agencies and patient
advocacy organizations as part of this program, bluebird bio believes it
is possible to seek conditional approval for the treatment of
adults and adolescents with beta-thalassemia major on the basis of the
totality of clinical data, in particular reduction in transfusion need,
from the ongoing Northstar Study and supportive HGB-205 study.
Conversion to full approval will be subject to the successful completion
of the HGB-207 and HGB-208 clinical trials, supportive long-term
follow-up data and “real-life” post-approval monitoring data.
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 Company’s global development and regulatory strategy for
LentiGlobin BB305, including the expected protocols for planned clinical
trials and the timing of these clinical trials, whether these planned
clinical trials will be sufficient to support regulatory submissions for
marketing approval and the expected timing of any such submissions and
decisions. In particular, it should be noted that the FDA normally
requires two pivotal clinical studies to approve a drug or biologic
product. Whether the planned HGB-207 and HGB-208 trials will be
sufficient to support submission of a BLA for LentiGlobin BB305 will
likely be a review issue to be discussed with FDA following completion
of the trials. In addition, it should be noted that the EMA Adaptive
Pathways program is a pilot program, and as such there is limited
information and precedent regarding the potential outcomes for sponsors
that participate in this program. 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 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, actions of
regulatory agencies, which may affect the initiation, timing and
progress of clinical trials and regulatory submissions, the risk
that the results of previously conducted studies involving similar
product candidates will not be repeated or observed in ongoing or future
studies involving current product candidates, 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.
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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
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updated from time to time on our investor relations website and may
include other social media channels than the ones described above. The
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incorporated by reference in any filing under the Securities Act of 1933.
View source version on businesswire.com: http://www.businesswire.com/news/home/20150609006752/en/
Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc.
Manisha Pai,
617-245-2107
mpai@bluebirdbio.com
or
Media:
Pure
Communications, Inc.
Dan Budwick, 973-271-6085