Company Plans to Pursue Conditional and Accelerated Registration
Strategies in E.U. and U.S., Respectively
Plans to Pursue Conditional Approval in E.U. Based on Data from
Ongoing Northstar and HGB-205 Studies through Adaptive Pathways Pilot
Program
NIH RAC Will Review HGB-208 Pediatric Study Protocol on June 9, 2015
Conference Call and Webcast Today at 8:00AM ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 19, 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, today announced that
it has met with regulatory authorities in Europe and the United States
to discuss potential approval pathways for its LentiGlobin BB305 product
candidate for the treatment of beta-thalassemia major. These discussions
have resulted in general agreement from both agencies regarding bluebird
bio’s development plans, which could potentially result in accelerated
approvals.
“We are very pleased with the outcome of these recent regulatory
interactions,” commented David Davidson, M.D., chief medical officer.
“We look forward to advancing our beta-thalassemia major program based
on data both from our ongoing studies as well as two planned open-label
studies with a sample size of 15 patients each. The EMA Adaptive
Pathways pilot program will allow us to pursue conditional approval for
the treatment of beta-thalassemia major on the basis of clinical data
from our ongoing HGB-204 and HGB-205 studies. This feedback brings us
closer to achieving our vision of delivering one-time, potentially
transformative gene therapy to patients.”
EMA Registration Strategy: Participate in Adaptive Pathways Pilot
Program
bluebird bio 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 discussed below, supportive long-term follow-up data and
“real-life” post-approval monitoring data.
FDA Registration Strategy: Pursue Accelerated Approval Based on
HGB-207 (n=15) and HGB-208 (n=15)
In addition, bluebird bio has 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 will enroll pediatric
patients.
bluebird bio has also reached general agreement with the FDA on:
-
Sample size: 15 patients per trial
-
Duration: 24 months of follow-up per patient
-
Primary endpoint: 12 months of transfusion independence
In the United States, if the LentiGlobin BB305 product candidate
demonstrates acceptable efficacy and safety in these patient
populations, these planned clinical trials could support an accelerated
approval, with post-approval confirmatory evidence to be provided with
longer-term follow-up of these trials. As a result of this regulatory
feedback and as required of all gene therapy clinical trials, bluebird
bio has filed both clinical study protocols with the National Institutes
of Health (NIH) Recombinant DNA Advisory Committee (RAC). The RAC has
notified bluebird bio that HGB-207 does not require an in-depth review
or public RAC discussion. The RAC has also notified bluebird bio that
the HGB-208 study protocol is scheduled for public review on June 9,
2015.
“We are grateful for the collaborative regulatory feedback from the FDA
and EMA on the design of our pivotal studies, as well as feedback from
the European HTA agencies and patient advocacy organizations that are
participating in our Adaptive Pathways pilot project,” stated
Anne-Virginie Eggimann, vice president of regulatory science. “We are
looking forward to continuing our engagement with all of these
stakeholders in the coming months to support the potential acceleration
of the LentiGlobin BB305 program.”
Background on the EMA’s Adaptive Pathways Program
In establishing the Adaptive Pathways program, the EMA stated the
following:
“The concept of Adaptive Pathways foresees either an initial approval in
a well-defined patient subgroup with a high medical need and subsequent
widening of the indication to a larger patient population, or an early
regulatory approval (e.g. conditional approval), which is prospectively
planned, and where uncertainty is reduced through the collection of
post-approval data on the medicine's use in patients. This approach is
particularly relevant for medicines with the potential to treat serious
conditions with an unmet medical need and may reduce the time to a
medicine's approval or to its reimbursement for targeted patient groups.
It involves balancing the importance of timely patient access with the
need for adequate, evolving information on a medicine's benefits and
risks. The Adaptive Pathways approach builds on regulatory processes
already in place within the existing European Union legal framework.”
The pilot was initiated in March 2014 and was called “Adaptive
Licensing” at the time. EMA changed the name to Adaptive Pathways “to
better reflect the idea of a life-span approach to bring new medicines
to patients with clinical drug development, licensing, reimbursement,
and utilization in clinical practice, and monitoring viewed as a
continuum.”
Background on the FDA Process and NIH’s RAC
FDA approval must be obtained before clinical testing of biological
products. Each clinical study protocol for a gene therapy product is
reviewed by the FDA and the NIH, through its Recombinant DNA Advisory
Committee (RAC). Within the FDA, the Center for Biologics Evaluation and
Research (CBER) regulates gene therapy products. CBER works with the NIH
and its RAC, which makes recommendations to the NIH on gene therapy
issues and engages in a public discussion of scientific, safety, ethical
and societal issues related to proposed and ongoing gene therapy
protocols.
The NIH is responsible for convening the RAC to discuss protocols that
raise novel or particularly important scientific, safety or ethical
considerations at one of its quarterly public meetings. The Office of
Biotechnology Activities (OBA) notifies the FDA of the RAC’s decision
regarding the necessity for full public review of a gene therapy
protocol. RAC proceedings and reports are posted to the OBA web site and
may be accessed by the public.
Investor Conference Call and Webcast Information
bluebird bio will host a conference call and webcast on May 19, 2015 at
8:00 AM ET to review its LentiGlobin regulatory strategy. 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 and (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 a preclinical CAR T immuno-oncology 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 Company’s global 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, 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 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20150519005556/en/
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