First two patients in the HGB-205 Study achieved transfusion
independence within two weeks of an autologous transplant with
bluebird’s lentiviral gene therapy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 14, 2014--
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to
developing potentially transformative gene therapies for severe genetic
and orphan diseases, today released initial positive clinical data from
its HGB-205 clinical study of its LentiGlobin BB305 product candidate in
beta-thalassemia major subjects at the 19th Annual Congress
of the European Hematology Association (EHA) in Milan, Italy.
“We are gratified that the improvements we introduced into the BB305
lentiviral vector design and manufacturing process appear to have
translated into clinical results that we believe support the potential
for our LentiGlobin BB305 gene therapy to transform the lives of
patients with beta-thalassemia major,” stated David Davidson, M.D.,
bluebird bio’s Chief Medical Officer. “We are encouraged by the early
and high-level production of corrected betaAT87Q-globin and
the rapid onset of transfusion independence in these initial subjects,
as well as the absence of any gene therapy related adverse events. We
look forward to providing additional data from this study and our
ongoing multi-center Northstar Study later this year.”
The principal investigator of the HGB-205 Study, Marina Cavazzana, M.D
delivered an oral presentation at the EHA Congress entitled “Improving
gene therapy for beta-thalassemia major: initial results from Study
HGB-205” on June 14, 2014 at 04:15 pm CET (10:15 am EDT). The
presentation included data from the prior LG001 Clinical Study and the
ongoing HGB-205 Study.
Summary of the clinical data presented at EHA were:
LG001 Clinical Study
Clinical update provided on two subjects treated in the prior LG001
Study (subjects 3 and 4) using the prior lentiviral HPV569 product
Subject 3 remains blood transfusion independent 72 months after being
transplanted with the lentiviral HPV569 product candidate
Subjects 3 and 4 are producing 2.7 g/dL and 0.4 g/dL of therapeutic
betaA-T87Q-globin post-transplant, respectively
No drug product related adverse events were reported in the LG001
HGB-205 Clinical Study
Clinical data were presented on two subjects (subjects 1 and 2), both
with beta-thalassemia major and the Beta E/Beta 0 genotype who were
treated using the new lentiviral vector BB305
At 4.5 months following autologous transplant subject 1 had a total
hemoglobin of 10.1 g/dL of which 6.6 g/dL was therapeutic betaAT87Q-globin,
and at 2 months post-transplant subject 2 had a total hemoglobin of
11.6 g/dL of which 4.2 g/dL was betaAT87Q-globin
Subjects 1 and 2 received their last blood transfusion on day 10 and
12, respectively, post-transplant and both subjects remain blood
Vector copy number in the drug product for subjects 1 and 2 were 1.5
and 2.1, respectively; multiple times higher than the drug product
vector copy numbers reported in the prior LG001 Study (VCN 0.6 and 0.3
for Subjects 3 and 4, respectively)
No drug product related adverse events were reported, and the
integration site analysis performed on subject 1 at the 3-month time
point showed polyclonal reconstitution.
We anticipate reporting additional data from the HGB-205 Study and from
our ongoing Northstar Study in late 2014.
Conference Call and Webcast
bluebird bio will host a conference call at 8:00 am EDT on Monday, June
16, 2014 to discuss the initial results from its HGB-205 Study.
Investors may listen to the webcast of the conference call live on the
"Calendar of Events" section of bluebird bio’s website, www.bluebirdbio.com.
Alternatively, investors may listen to the call by dialing: (844)
825-4408 from locations in the U.S. and (315) 625-3227 from outside the
U.S. The webcast replay will be available for at least 72 hours
following the call.
Beta-thalassemia major is a rare hereditary blood disorder caused by a
genetic abnormality of the beta globin gene resulting in defective red
blood cells. Symptoms of beta-thalassemia include severe anemia and
splenomegaly. It is estimated that about 288,000 patients with
beta-thalassemia are alive, of which an estimated 15,000 live in the
United States and Europe. The majority of beta-thalassemia patients have
About the HGB-205 Study
The phase 1/2 study is designed to evaluate the safety and efficacy of
LentiGlobin BB305 drug product in the treatment of subjects with
beta-thalassemia major and severe sickle cell disease. The study is
designed to enroll up to seven subjects. Subjects will be followed to
evaluate safety and blood transfusion requirements post-transplant. In
sickle cell disease patients only, efficacy will also be measured based
on the number of vaso-occlusive crises or acute chest syndrome events.
For more information on the HGB-205 Study, please visit
www.clinicaltrials.gov using identifier NCT02151526.
About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing
potentially transformative gene therapies for severe genetic and orphan
diseases. bluebird bio has two clinical-stage programs in development.
The most advanced product candidate, Lenti-D, is in a recently-initiated
phase 2/3 study, the Starbeam Study, for the treatment of childhood
cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological
disorder affecting young boys. The next most advanced product candidate,
LentiGlobin, is currently in two phase 1/2 studies, one in the US (the
Northstar Study) and one in France (HGB-205), for the treatment of
beta-thalassemia major. The phase 1/2 HGB-205 study also allows
enrollment of patient(s) with sickle cell disease, and bluebird bio is
planning a separate U.S. sickle cell disease trial (HGB-206).
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in collaboration with Celgene
bluebird bio has operations in Cambridge, Massachusetts and Paris,
France. For more information, please visit www.bluebirdbio.com.
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the potential efficacy and safety of the Company’s
LentiGlobin product candidate, the Company’s plans with respect to
LentiGlobin and its other product candidates and anticipated clinical
and business milestones and announcements for 2014. In addition it
should be noted that the data for LentiGlobin announced from the HGB-205
study at the EHA Congress are preliminary in nature and the HGB-205
trial is not completed. These data may not continue for these
subjects or be repeated or observed in ongoing or future studies
involving our LentiGlobin product candidate, including the HGB-205
Study, the Northstar Study or the HGB-206 study in 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 risk that the preliminary results from our clinical trials will
not continue or be repeated in our ongoing clinical trials, the risk
that previously conducted studies involving similar product candidates
will not be repeated or observed in ongoing or future studies involving
current product candidates, the risk of cessation or delay of any of the
ongoing or planned clinical studies and/or our development of our
product candidates, the risk of a delay in the enrollment of patients in
the Company’s clinical studies, the risk that our collaboration with
Celgene will not continue or will not be successful, and the risk that
any one or more of our product candidates will not be successfully
developed and 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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Source: bluebird bio, Inc.
bluebird bio, Inc.
Richard E. T.
Smith, Ph.D., 339-499-9382
Dan Budwick, 973-271-6085