[June 23, 2017] |
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bluebird bio Announces Early Data from Phase 3 Northstar-2 (HGB-207) Study of LentiGlobinTM Drug Product at European Hematology Association (EHA) Annual Meeting
bluebird bio, Inc. (Nasdaq:
BLUE), a clinical-stage company committed to developing potentially
transformative gene therapies for serious genetic diseases and T
cell-based immunotherapies for cancer, announced early interim data from
the ongoing Northstar-2 (HGB-207) Phase 3 clinical study of LentiGlobin
drug product in patients with transfusion-dependent ß-thalassemia (TDT)
and non-ß0/ß0 genotypes. These data will be
presented by Mark Walters, M.D., UCSF Benioff Children's Hospital,
Oakland, California, in an oral session on Sunday, June 25 at the
European Hematology Association (EHA) Annual Meeting in Madrid, Spain.
"Northstar-2 is our first study to utilize our improved LentiGlobin drug
product manufacturing process to increase the drug product vector copy
number and percent of cells transduced. The first patient treated in
this study exemplifies the promise of gene therapy, discontinuing blood
transfusions approximately a month after treatment and achieving a
normal level of total hemoglobin production at six months
post-treatment," said David Davidson, M.D., chief medical officer,
bluebird bio. "These early results suggest that the improved
manufacturing process results in consistently higher drug product vector
copy numbers (VCN) and lentiviral vector positive (LVV+) cells, which is
correlated with higher production of HbAT87Q and ultimately
may address known patient-to-patient variability."
"Although early, these data add to the growing body of clinical evidence
that indicate that LentiGlobin may offer a transformative benefit for
patients with TDT," said Alexis Thompson, MD, MPH, Ann & Robert H. Lurie
Children's Hospital of Chicago, Illinois and a primary investigator on
the study. "Patients with TDT are dependent on a burdensome cycle of
transfusion and chelation, and for these patients, gene therapy with
LentiGlobin may offer a long-term solution with a one-time therapy that
alleviates many of the complications of the current treatment paradigm."
A Phase 3 Study to Evaluate Safety and Efficacy of LentiGlobin Gene
Therapy for Transfusion-Dependent ß-thalassemia in Patients with non-ß0/ß0
Genotypes: The Northstar-2 (HGB-207) Trial (Abstract S814)
The Northstar-2 Study is an ongoing, open-label, single-dose,
international, multicenter Phase 3 study designed to evaluate the safety
and efficacy of LentiGlobin drug product for the treatment of patients
with TDT and non-ß0/ß0 genotypes. As of June 2,
2017, drug product had been manufactured for six patients. The median DP
VCN for these patients was 3.0 (range: 2.4 - 4.0), compared to a median
DP VCN of 0.7 (range: 0.3 - 1.5) in Northstar. Results in treated
patients, aged 20-22 years, as of June 2, 2017, include:
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Patient 1
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Patient 2
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Patient 3
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DP VCN in each drug product lot (copies/diploid genome)
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2.9
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2.4
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3.2, 2.4
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LVV+ cells
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77%
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53%
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77%, 82%
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CD34+ cell dose (x106/kg)
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7.0
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13.6
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8.1
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HbAT87Q (g/dl; at last follow-up)
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9.5
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1.6
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4.6
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Total hemoglobin
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13.3
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Not reported
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Not reported
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Days since last transfusion
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140
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Not reported
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Not reported
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Follow-up
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6 months
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3 months
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2 months
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Follow-up on Patients 2 and 3 was not sufficient for total hemoglobin
or days since last transfusion to be clinically relevant.
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The safety profile to date appears consistent with autologous
transplantation. No Grade 3 or higher drug-product related adverse
events have been observed.
Webcast Information bluebird bio will host a live webcast at
8:00 a.m. ET on Friday, June 23, 2017. The live webcast 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. Please refer to conference ID number 39917037.
About Northstar-2 (HGB-207) Northstar-2 is a Phase 3,
global, multi-center study designed to evaluate the safety and efficacy
of LentiGlobin drug product in patients with transfusion-dependent
beta-thalassemia and non-ß0/ß0 genotypes. For this
study, the manufacturing process by which the patient's cells are
transduced with the LentiGlobin viral vector has been improved, with the
intent of increasing vector copy number and the percentage of cells
successfully transduced.
The target enrollment of the study is 15 adult and adolescent patients
and 8 pediatric patients. The study's primary endpoint is the proportion
of treated subjects who meet the definition of "transfusion
independence," defined as total hemoglobin levels of at least 9g/dL
without any red blood cell (RBC) transfusions for a continuous period of
at least 12 months at any time during the study.
About TDT Transfusion-dependent ß-thalassemia (TDT), also
called ß-thalassemia major or Cooley's anemia, is an inherited blood
disease that can be fatal within the first few years of life if not
treated.
Despite advances in the supportive conventional management of the
disease, which consists of frequent and lifelong blood transfusions and
iron chelation therapy, there is still a significant unmet medical need,
including the risk for significant morbidity and early mortality.
Currently, the only advanced treatment option for TDT is allogeneic
hematopoietic stem cell transplant (HSCT). Complications of allogeneic
HSCT include a significant risk of treatment-related mortality, graft
failure, graft vs. host disease and opportunistic infections,
particularly in patients who undergo non-sibling-matched allogeneic HSCT.
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 (News - Alert) Study,
for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™
product candidate, currently in four clinical studies for the treatment
of transfusion-dependent ß-thalassemia, 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 program, bb2121, is an anti-BCMA CAR T program
partnered with Celgene. bb2121 is currently being studied in a Phase 1
trial for the treatment of relapsed/refractory multiple myeloma.
bluebird bio also has discovery research programs utilizing
megaTAL/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.
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, and 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, the
risks that the changes we have made in the LentiGlobin manufacturing
process will not result in improved patient outcomes, 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,
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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170623005066/en/
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