– Promising early results from two
patients treated under amended study protocol and with refined
manufacturing process show 51% and 28% anti-sickling HbAT87Q at six
and nine months, respectively, exceeding levels seen previously in
the HGB-206 study –
– Plerixafor mobilization and apheresis
cell collection for LentiGlobin manufacture now implemented in the
study, first patient treated with this method had a peripheral VCN
of 2.5 copies/diploid genome at month 1 –
– Company to hold webcast today, December
10, 8:30 p.m. ET –
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 updated clinical results from HGB-206,
the company’s ongoing Phase 1 multicenter study of its LentiGlobin
gene therapy product candidate in patients with severe sickle cell
disease (SCD), will be discussed in an oral presentation during the
59th Annual Meeting of the American Society of Hematology (ASH). In
addition, a poster on the feasibility and potential benefits of
plerixafor-mediated peripheral blood stem cell collection and drug
product (DP) manufacturing in patients with SCD was presented
yesterday at ASH.
“The promising early results from the first two patients treated
under the amended HGB-206 study protocol indicate that the
manufacturing and patient management changes we implemented may
have a meaningful impact on patient outcomes,” said Dave Davidson,
chief medical officer, bluebird bio. “These two patients have
maintained higher levels of gene-marked cells in the blood
following treatment compared to the previous patients in HGB-206.
This improvement corresponds with increased production of the
anti-sickling hemoglobin, HbAT87Q, made from LentiGlobin. We are
hopeful that this high-level expression of HbAT87Q will lead to a
sustained clinical benefit for these patients. The next group of
patients in the study will be treated using LentiGlobin made from
stem cells obtained from plerixafor-mobilized peripheral blood.
Plerixafor mobilization in place of direct bone marrow harvest is
less burdensome for patients, and our results suggest that this
approach may be able to obtain a greater quantity of higher quality
cells.”
Interim Results from a Phase 1/2 Clinical Study of
LentiGlobin Gene Therapy for Severe Sickle Cell Disease (Oral
Abstract #527)Presenter: Julie Kanter, M.D., Medical
University of South Carolina, Charleston, SCDate & Time:
Sunday, December 10 at 5:30 p.m.Location: Bldg C, Level 1,
C101 Auditorium
“People with sickle cell disease have a genetic disease that
causes the protein in red blood cells, called hemoglobin, to be
misshapen. As a result of this abnormal hemoglobin, many affected
individuals live with low blood counts and severe, recurrent pain
crises that lead to organ damage and shortened life spans,” said
Dr. Kanter. “It is also a disease that has been historically
under-researched and under-resourced, with few treatment options
beyond pain management. These early results with the revised study
protocol indicate that gene therapy with LentiGlobin may allow
people with SCD to produce substantial levels of normal,
anti-sickling, adult hemoglobin. We are hopeful about the
possibility that this could substantially reduce the painful and
damaging crises that are a hallmark of this disease, potentially
allowing patients to live longer, healthier lives.”
HGB-206 is an ongoing, open-label study designed to evaluate the
safety and efficacy of LentiGlobin DP in the treatment of adults
with severe SCD. Patients in this study are divided into three
cohorts: A, B and C. Patients in Group A were treated under the
original study protocol. Patients in Group B were treated under an
amended study protocol that included changes intended to increase
DP vector copy number (VCN) and improve engraftment of
gene-modified stem cells. Patients in both Group A and B had DP
made from stem cells collected using bone marrow harvest. Patients
in Group C are also treated under the amended study protocol, but
receive LentiGlobin made from stem cells collected from peripheral
blood after mobilization with plerixafor rather than via bone
marrow harvest. As of November 30, 2017, ten patients had been
treated in the study and follow-up data were available on nine
patients from groups A and B, with a median of 21 (6-27) months
since transplantation. Key results include:
Group A
N=7
Median (min-max)
Group B
N=2
Patient 1312
Patient 1313 Transduced CD34+ cells (%) 25 (8-42)
951, 901
46, 831 Drug product Cell Dose (x106 CD34+ cells)
2.1 (1.6-5.1) 3.2 2.2 Drug product VCN
(copies per diploid genome) 0.6 (0.3-1.3)
2.91, 5.01 1.4, 3.31 VCN in peripheral blood (copies
per diploid genome at last measurement) 0.1 (0.1-0.2)
2.5 (M6) 0.5 (M9) HbAT87Q (g/dL at last
measurement) 0.7 (0.5-2.0) 6.4 (M6)
3.0 (M9) HbAT87Q (% of total, at last measurement)
7.9 (5.3-18.2) 51% (M6) 28% (M9)
__________________________
1 LentiGlobin DP manufactured using refined process
- Both patients in Group B were treated
with two DP lots. Information from each of these LentiGlobin DP
lots is reflected in the chart above.
- Patient 1312 received LentiGlobin
manufactured entirely using the refined manufacturing process.
- Patient 1313 received LentiGlobin
manufactured using a combination of the original and the refined
manufacturing processes.
- LentiGlobin DP has been manufactured
for four patients in Group C:
- Median transduced CD34+ cells: 80%
- Median DP cell dose: 6.9 x106 CD34+
cells
- Median DP VCN (copies per diploid
genome): 3.3
- The first patient treated with
LentiGlobin (Group C) made using plerixafor-mobilized stem cells
had a VCN in peripheral blood of 2.5 at one month.
- The toxicity profile observed from drug
product infusion to latest follow-up was generally consistent with
myeloablative conditioning with single-agent busulfan.
Successful Plerixafor-Mediated Mobilization, Apheresis, and
Lentiviral Vector Transduction of Hematopoietic Stem Cells in
Patients with Severe Sickle Cell Disease (Poster Abstract
#990)Presenter: John Tisdale, M.D., National Heart, Lung
and Blood Institute (NHLBI), Bethesda, MDDate & Time:
Saturday, December 9 at 5:30 p.m.Location: Bldg A, Level 1,
Hall A2
“Historically, harvesting stem cells from people with SCD
required bone marrow harvest, a painful approach for obtaining
cells that often yields a suboptimal dose level and cell quality,”
said Dr. Tisdale. “The data we presented at ASH suggest that not
only is this new approach using plerixafor mobilization generally
tolerable for patients, but it may enable us to obtain a larger
cell dose with a higher concentration of primitive stem cells.
Cells with this primitive phenotype are more likely to become
long-term sources of gene-modified red blood cells. We believe that
providing more primitive hematopoietic stem cells that carry more
copies of the gene therapy vector may be critical to realizing the
full promise of gene therapy for people with SCD, and we look
forward to getting more data on this new cohort of patients in the
coming months.”
Results as of November 30, 2017:
Bone Marrow Harvest Plerixafor Number of
Patients 9 (26 BMHs) 7 (10 mobilization
cycles) Adverse Events
17 Grade 3 AEs following BMH in 5
patients, 4 were SAEs (1 procedural pain, 3 SCD pain crisis)
5 Grade 3 events included 2 non-serious
(hypomagnesemia and non-cardiac chest pain) and 3 SAEs (1 patient
each) of SCD pain crisis CD34+ cells collected per harvest, median
(min-max) cells/kg 5.0 (0.3-10.8) x 106
10.4 (5.1-20.0) x 106
Webcast Information
bluebird bio will host a webcast at 8:30 p.m. ET today, December
10, 2017. The webcast can be accessed under "Calendar of Events" in
the Investors and Media section of the company's website at
www.bluebirdbio.com.
About SCD
Sickle cell disease (SCD) is an inherited disease caused by a
mutation in the beta-globin gene, that produces βS-globin. High
levels of HbS in patients with SCD are responsible for the
characteristic chronic anemia, vaso-occlusive crises, and other
acute and chronic manifestations of SCD which lead to significant
morbidity and early mortality.
Where adequate medical care is available, common treatments for
patients with SCD largely revolve around prevention of infection
and management and prevention of acute sickling episodes. Chronic
management may include hydroxyurea and, in certain cases, chronic
transfusions. Allogeneic hematopoietic stem cell transplant (HSCT)
is currently the only available option to address the underlying
genetic cause of SCD, though it carries significant risk.
Complications of allogeneic HSCT include a risk of
treatment-related mortality, graft failure, graft versus host
disease (GvHD) 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 Study, for the treatment
of cerebral adrenoleukodystrophy, and its LentiGlobin® product
candidate, currently in five 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, Durham, North Carolina
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 whether the manufacturing process changes for
LentiGlobin will 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,
including plerixafor mobilization, 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, 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 or the HGB-206
clinical trial protocol 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, 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20171210005109/en/
bluebird bio, Inc.Investors & MediaElizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.com
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