- Drug product vector copy number (DP VCN) and
percentage of lentiviral vector positive cells (LVV+) for initial 7
drug product lots manufactured in Northstar-2 (HGB-207) are
consistently higher than in Northstar (HGB-204) with median DP VCN
of 3.0 –- Initial results show that the three patients treated to
date have achieved in vivo VCN and HbAT87Q production as good as or
better than patients achieving transfusion independence in
Northstar –- First patient treated in Northstar-2 with 6 months
follow-up achieved normal total hemoglobin (13.3 g/dL) after
discontinuing transfusions; producing 9.5 g/dl of HbAT87Q at last
follow-up –- Safety profile to date consistent with autologous
transplantation –- Company to hold conference call and webcast
today, June 23, at 8:00 a.m. ET –
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 β-thalassemia (TDT) and
non-β0/β0 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 β-thalassemia in
Patients with non-β0/β0 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-β0/β0 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:
Patient 1
Patient 2 Patient 3 DP VCN in
each drug product lot (copies/diploid genome)
2.9 2.4 3.2, 2.4 LVV+
cells 77% 53%
77%, 82% CD34+ cell dose (x106/kg)
7.0 13.6 8.1
HbAT87Q (g/dl; at last follow-up) 9.5
1.6 4.6 Total hemoglobin
13.3 Not reported
Not reported Days since last transfusion
140 Not reported
Not reported Follow-up 6 months
3 months 2 months
- Follow-up on Patients 2 and 3 was not
sufficient for total hemoglobin or days since last transfusion to
be clinically relevant.
- The safety profile to date appears
consistent with autologous transplantation. No Grade 3 or higher
drug-product related adverse events have been observed.
Webcast Informationbluebird 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-β0/β0 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 TDTTransfusion-dependent β-thalassemia (TDT), also
called β-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 Study, for the treatment of cerebral
adrenoleukodystrophy, and its LentiGlobin™ product candidate,
currently in four clinical studies for the treatment of
transfusion-dependent β-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 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 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/
bluebird bio, Inc.Investors:Manisha Pai,
617-245-2107mpai@bluebirdbio.comorMedia:Elizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.com
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