– Consistently demonstrated improved vector
copy number (VCN) in transfusion-dependent β-thalassemia (TDT) and
severe sickle cell disease (SCD) patient cells in vitro with
manufacturing Process 2 –
–Implementing plan to optimize patient outcomes
in severe SCD –
–Achieved general agreement on regulatory path
for TDT across all genotypes, ages –
–Advanced suspension manufacturing process
–
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, will outline today key activities underway intended to
advance the company’s LentiGlobin programs in transfusion-dependent
β-thalassemia (TDT) and severe sickle cell disease (SCD). The
presentation will focus on three key aspects of these activities:
1) potential improvements in transduction efficiency and
manufacturing; 2) updates to the protocol for the company’s ongoing
HGB-206 clinical trial in SCD; and 3) regulatory plans for the
company’s LentiGlobin drug product candidate in TDT. The company
will webcast its presentation beginning at 9:00 am ET today on the
Investors & Media section of www.bluebirdbio.com.
“At bluebird bio we have an incredibly ambitious goal: to
deliver one-time, transformative therapies to patients with rare
genetic diseases and cancer. We are relentless in our efforts to
continue innovating in pursuit of that goal, and we have made
substantial advances in the transduction and manufacturing
processes, translational research, and clinical development,” said
Nick Leschly, chief bluebird. “By incorporating manufacturing
Process 2 into our LentiGlobin clinical trials, we believe we can
achieve our ultimate goal of increasing hemoglobin production in
patients treated with LentiGlobin drug product. In sickle cell
disease we have used our early clinical data to identify and
implement multiple distinct improvements with the potential to
overcome the unique challenges presented by this complex disease.
We are hopeful that the tremendous work done by our research and
development teams will yield improved outcomes for patients in the
clinic in 2017 and beyond, and that the regulatory progress we are
making will enable us to bring these treatments to patients as
quickly as possible. We anticipate seeing many catalysts across our
programs in the next 15 months, including initial clinical data
from our Phase 1 clinical study of our bb2121 product candidate in
relapsed/refractory multiple myeloma and updates on our Lenti-D
program in cerebral adrenoleukodystrophy.”
LentiGlobin Manufacturing Data: A Head-to-Head In Vitro
Comparison of Process 1 and Process 2bluebird bio has
recently modified the process by which the patient’s cells are
transduced in LentiGlobin clinical studies with the addition of
enhancers during the manufacturing process. The goal of
manufacturing Process 2 is to increase the percentage of cells
successfully transduced, thereby increasing vector copy number
(VCN) in the drug product that is given to the patient.
Using retained samples of CD34+ stem cells collected from
patients in the HGB-204 (Northstar) and HGB-206 studies, the
company was able to demonstrate in a head-to-head in vitro
comparison that manufacturing Process 2 substantially increased the
percentage of cells transduced and VCN, as compared to
manufacturing Process 1.
This in vitro data from Process 2 showed an average increase of
approximately three-fold in vector-positive cells and VCN across
all patient samples tested. Process 2 has been successfully scaled
up for clinical manufacturing, and all LentiGlobin clinical trials
moving forward will use manufacturing Process 2, including the
Phase 3 HGB-207 (Northstar-2) trial and the Phase 1 HGB-206
clinical trial.
bluebird bio also highlighted progress it has made in moving
from adherent manufacturing of lentiviral vectors to potentially
more efficient suspension manufacturing, consistently achieving the
targeted potency, purity and VCNs at increased scale.
LentiGlobin in Sickle Cell Disease: Addressing the Challenges
and Promise of Gene TherapyBased on an assessment of patient
data presented at ASH 2015 and the underlying biology of SCD, the
company believes that the following specific amendments to the
protocol of the ongoing HGB-206 clinical trial may lead to improved
patient outcomes through:
- Improving or enhancing stem cell
collection by both:(i) Suppression of sickle cell bone marrow
pathology through required pre-stem cell harvest red blood cell
transfusions, and(ii) Use of improved cell separation techniques
and increase of the required minimum cell dose
- Increasing percentage of cells
transduced and VCN through implementation of manufacturing Process
2 for LentiGlobin drug product
- Enhancing the engraftment of the
LentiGlobin drug product by adjusting the target level of exposure
to busulfan for pre-infusion conditioning; and
- Additional exploratory alternative cell
collection approaches through the mobilization and apheresis of
patient CD34+ cells using plerixafor
To accommodate these changes to the protocol, the study
enrollment has been expanded for a total enrollment of up to 29
patients.
LentiGlobin in Transfusion-Dependent β-thalassemia:
Regulatory Progressbluebird bio is working closely with
regulatory agencies in the United States and Europe to bring
LentiGlobin to patients with TDT who can benefit, as quickly as
possible, and has reached general agreement with the U.S. Food and
Drug Administration (FDA) and the European Medicines Agency (EMA),
respectively, on the regulatory paths forward. Key elements of the
company’s regulatory process include:
- General agreement with the FDA on
pivotal clinical trial designs across patient genotypes and age
groups:
- HGB-207 (Northstar-2) Phase 3 study in
15 adult and adolescent patients with TDT who do not have β0/β0
genotypes, with an additional pediatric cohort of 8 patients for a
total enrollment of approximately 23 patients
- HGB-212 Phase 3 study in 15 adult,
adolescent and pediatric patients with TDT who have β0/β0 genotypes
to launch in 2017, with a primary endpoint of transfusion
reduction
- Both studies will be conducted using
manufacturing Process 2 and are designed to provide the basis for
BLA submissions in the United States
- Confirmation that, as part of the EMA
Adaptive Pathways and PRIME programs, application for conditional
approval for LentiGlobin in the EU would be based on data from the
HGB-204 (Northstar) and HGB-205 studies of LentiGlobin, as well as
available data from the HGB-207 (Northstar-2) and HGB-212
studies
To access the live webcast, please visit the “Events &
Presentations” page within the Investors and Media section of the
bluebird bio website at http://investor.bluebirdbio.com. Replays of
the webcast will be available on the bluebird bio website for 90
days following the event.
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 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
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 Paris,
France.
Forward-Looking StatementsThis 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 severe sickle cell
disease, including statements whether the planned manufacturing
process changes for LentiGlobin will reduce costs and improve
outcomes of patients with transfusion-dependent ß-thalassemia and
severe sickle cell disease, whether the planned changes to the
HGB-206 clinical trial protocol will improve outcomes in patients
with severe sickle cell disease. 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, risks that the
preliminary positive results from our prior and ongoing clinical
trials of LentiGlobin will not continue or be repeated in our
ongoing or planned clinical trials of LentiGlobin, the risks that
the changes we have made in the LentiGlobin manufacturing process
or the HGB-206 clinical trial protocol will not result in reduced
costs or 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20161013005754/en/
Investors:bluebird bio, Inc.Manisha Pai,
617-245-2107mpai@bluebirdbio.comorMedia:bluebird bio, Inc.Elizabeth
Pingpank, 617-914-8736epingpank@bluebirdbio.comorPure
Communications, Inc.Dan Budwick, 973-271-6085
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