– Study to enroll approximately fifteen adult,
adolescent and pediatric patients –
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company
committed to developing potentially transformative gene therapies
for severe genetic diseases and T cell-based immunotherapies for
cancer, today announced that the first patient has been treated in
Northstar-3 (HGB-212), the company’s Phase 3 study of LentiGlobin
in patients with transfusion-dependent β-thalassemia (TDT) with the
β0/β0 genotype.
“We are excited to start our second Phase 3 study of LentiGlobin
in patients with TDT,” said Dave Davidson, chief medical officer.
“Early data presented at the European Hematology Association annual
meeting this year indicates that our refined manufacturing process,
now implemented for treatment across all of our active studies of
LentiGlobin, has consistently yielded improved Drug Products with
higher vector copy number and an increased proportion of transduced
cells. We are optimistic that these advances will improve our
ability to achieve the levels of HbAT87Q needed for the patients in
this study, who have the β0/β0 genotype, to see their annual RBC
transfusions dramatically reduced, if not entirely eliminated.”
“Ongoing studies of LentiGlobin in patients with TDT have
indicated the potential for a durable effect, with patients seeing
consistent production of HbAT87Q for more than three years after
treatment,” said Alexis Thompson, MD, MPH, Ann & Robert H.
Lurie Children’s Hospital of Chicago, Illinois and a primary
investigator on the study. “With the introduction of the refined
manufacturing process into the Northstar-2 and Northstar-3 pivotal
studies, we hope our patients will produce a greater amount of
hemoglobin to enhance their treatment effect, and thereby
demonstrate that LentiGlobin has the potential to address the
underlying genetic cause of TDT, regardless of genotype.”
LentiGlobin is also being investigated in Northstar-2 (HGB-207),
a Phase 3 study in patients with TDT and non-β0/β0 genotypes,
Northstar (HGB-204), a Phase 1/2 study in patients with TDT and all
genotypes, HGB-205, a Phase 1 study in patients with TDT and severe
sickle cell disease (SCD), and HGB-206, a Phase 1 study in patients
with SCD.
About Northstar-3 (HGB-212)
Northstar-3 is a Phase 3, global, multi-center study designed to
evaluate the safety and efficacy of LentiGlobin in patients with
transfusion-dependent beta-thalassemia with a β0/β0 genotype. In
this study, the manufacturing process by which the patient’s cells
are transduced with the LentiGlobin viral vector has been modified,
with the intent of increasing the percentage of cells successfully
transduced and the average vector copy number per diploid
genome.
The study’s primary endpoint is the proportion of patients who
meet the definition of "transfusion reduction" (TR). TR is defined
as demonstration of reduction of at least 60% in volume of red
blood cell (RBC) transfusion requirements (in mL/kg) in the
post-treatment time period of Months 12 to 24, as compared to the
average annual transfusion requirement in the 24 months prior to
enrollment. The target enrollment of the study is 15 adult,
adolescent and pediatric patients.
About TDT
Transfusion-dependent β-thalassemia (TDT) is a severe genetic
disease characterized by reduced or absent hemoglobin levels that
results in severe anemia and ineffective red blood cell production.
Supportive care for people with TDT consists of a lifelong regimen
of chronic blood transfusions to enable survival and suppress
symptoms of the disease, and iron chelation therapy to manage iron
overload that results from the transfusions. Despite the
availability of supportive care, many people with TDT experience
serious complications and organ damage due to underlying disease
and iron overload.
Allogeneic hematopoietic stem cell transplant (HSCT) is
currently the only available option to address the underlying
genetic cause of TDT, though it carries significant risks.
Complications of allogeneic HSCT include a risk of
treatment-related mortality, graft failure, graft vs. host disease
(GvHD) and opportunistic infections, particularly in patients who
undergo HSCT from a donor who is not a matched sibling.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy
expertise and gene editing capabilities, bluebird bio has built an
integrated product platform with broad potential application to
severe genetic diseases and cancer. bluebird bio’s gene therapy
clinical programs include its Lenti-D™ product candidate, currently
in a Phase 2/3 study, called the Starbeam Study, for the treatment
of cerebral adrenoleukodystrophy, and its LentiGlobin® BB305
product candidate, currently in three clinical studies for the
treatment of transfusion-dependent β-thalassemia, also known as
β-thalassemia major, and severe sickle cell disease. bluebird bio’s
oncology pipeline is built upon the company’s leadership in
lentiviral gene delivery and T cell engineering, with a focus on
developing novel T cell-based immunotherapies, including chimeric
antigen receptor (CAR T) and T cell receptor (TCR) therapies.
bluebird bio’s lead oncology programs, bb2121 and bb21217, are
anti-BCMA CAR T programs partnered with Celgene. bb2121 and
bb21217 are each currently being studied in Phase 1 trials for the
treatment of relapsed/refractory multiple myeloma. bluebird bio
also has discovery research programs utilizing megaTALs/homing
endonuclease gene editing technologies with the potential for use
across the company’s pipeline.
bluebird bio has operations in Cambridge,
Massachusetts, Seattle, Washington, and Europe.
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
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 research, development,
manufacturing and regulatory approval plans for its LentiGlobin
product candidate to treat transfusion-dependent ß-thalassemia,
including statements whether the manufacturing process changes for
LentiGlobin will improve outcomes of patients with
transfusion-dependent ß-thalassemia. 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 risks that
the preliminary positive efficacy and safety results from our prior
and ongoing clinical trials of LentiGlobin will not continue or be
repeated in our ongoing, planned or expanded clinical trials of
LentiGlobin, risks that the current or planned clinical trials of
LentiGlobin will be insufficient to support regulatory submissions
or marketing approval in the US and EU, the risk of a delay in the
enrollment of patients in our clinical studies, and the risk that
any one or more of our product candidates, including our bb2121
product candidate, will not be successfully developed, approved or
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 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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version on businesswire.com: http://www.businesswire.com/news/home/20171116005767/en/
Investors & Mediabluebird bioElizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.com
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