- First patient with severe sickle cell disease
treated with gene therapy remains free of clinical symptoms 21
months after receiving LentiGlobin Drug Product -
- Ongoing transfusion independence and
sustained production of HbAT87Q in patients with
transfusion-dependent β-thalassemia -
- Company to host event at ASH with live
webcast, Monday, December 5 at 8:30 p.m. PT -
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, announced the presentation of new data from the ongoing
HGB-205 clinical study evaluating its LentiGlobin product candidate
in patients with transfusion-dependent β-thalassemia (TDT) and
severe sickle cell disease (SCD) at the 58th American Society of
Hematology Annual Meeting.
The data from the HGB-205 study were highlighted today in a
poster presentation by Marina Cavazzana, M.D., Ph.D., lead
investigator of the HGB-205 study conducted in Necker Hospital,
AP-HP and professor of hematology at Paris Descartes University,
head of the department of Biotherapy Hospital, the clinical
research center of Biotherapy at Necker Enfants Malades - Greater
Paris University Hospitals, AP-HP and Inserm) and the
Lymphohematopoiesis Laboratory, Institute of Genetic Diseases,
Imagine, Paris, France.
“We believe the enduring responses seen in this study - in the
patients with TDT as well as the patient with SCD - demonstrate the
continued promise of LentiGlobin gene therapy in both of these
patient populations. We have seen nearly three years of transfusion
independence in TDT in certain patients, providing important data
on the long-term safety and durability of this therapy,” said David
Davidson, M.D., chief medical officer, bluebird bio. “In addition,
it is encouraging that the patient with SCD has remained free of
acute SCD-related clinical events in the 21 months since treatment,
when he previously required monthly blood transfusions to help
control his SCD symptoms. This patient’s successful outcome not
only offers hope for the potential of LentiGlobin to benefit other
patients with SCD, but also provides important insights into this
complex disease that we are applying to our ongoing HGB-206
study.”
Abstract #2311: Update from the HGB-205 Phase 1/2 Clinical
Study of LentiGlobin Gene Therapy: Sustained Clinical Benefit in
Severe Hemoglobinopathies
HGB-205 is an ongoing, open-label, single-center Phase 1/2 study
designed to evaluate the safety and efficacy of LentiGlobin drug
product in the treatment of patients with TDT and severe SCD. Four
patients with TDT and one patient with severe SCD have undergone
infusion with LentiGlobin drug product in this study as of
September 9, 2016. The patients with TDT have between 11.6 and 33.5
months of follow-up, and the patient with SCD has 22.9 months of
follow-up.
Key Results as of September 9, 2016 Data Cut-off:
- Three patients with TDT and β0/βE
genotype have remained free of transfusions since shortly after
receiving LentiGlobin treatment. Patient 1201 has been free of
transfusions for 33.1 months with total hemoglobin of 10.9 g/dL, of
which 7.7 g/dL was HbAT87Q. Patient 1202 has been free of
transfusions for 29.9 months with total hemoglobin of 13.5 g/dL, of
which 10.1 g/dL was HbAT87Q. In addition, this patient has been
able to stop iron chelation. Patient 1206 has been free of
transfusions for 11.5 months with total hemoglobin of 11.3 g/dL, of
which 8.6 g/dL was HbAT87Q.
- Patient 1203 with TDT and homozygousity
for the severe β+ mutation IVS1-110 has been free of transfusions
for 11.6 months (since approximately 3 months after receiving
LentiGlobin treatment) with total hemoglobin of 8.3 g/dL, of which
6.7 g/dL was HbAT87Q.
- Patient 1204 with severe SCD at
21-month post-drug infusion was producing 48% HbAT87Q – well above
the 30 percent threshold of anti-sickling Hb that may potentially
achieve a disease-modifying clinical effect.
- Prior to drug product infusion, Patient
1204 required monthly blood transfusions after failure of
hydroxyurea treatment to help control his SCD symptoms; he has not
received RBC transfusions since shortly after LentiGlobin infusion.
Since infusion, this patient has had no hospitalizations or acute
SCD-related events.
- No LentiGlobin-related adverse events
have been observed for the patients with either TDT or SCD; the
adverse events observed are generally consistent with myeloablative
conditioning.
- All five treated patients successfully
engrafted and insertional site analyses demonstrate highly
polyclonal reconstitution without clonal dominance.
“These data show a stable clinical and biological effect in
patients with TDT or severe SCD who have received a one-time
treatment with LentiGlobin,” said Professor Cavazzana. “We are now
seeing the benefit of gene therapy with LentiGlobin beyond two
years in TDT in certain patients, and clinical benefit continues to
be realized in the patient with severe SCD after almost 24 months
of follow-up. We are encouraged by these results and the potential
benefit treatment with LentiGlobin can have on patients living with
these debilitating diseases and without an HLA compatible sibling
donor.”
Webcast Information
bluebird bio will host a live webcast at 8:30 p.m. PT (11:30
p.m. ET) on Monday, December 5, 2016. 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.
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
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 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 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
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, including HGB-205, 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 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.
About AP-HP: AP-HP - Greater Paris University hospitals -
is a European world-renowned European university hospital. Its 39
hospitals treat 8 million people every year: in consultation,
emergency, during scheduled or home hospitalizations. The AP-HP
provides a public health service for everyone, 24 hours a day. This
mission is a duty as well as a grzeat source of pride. The AP-HP is
the leading employer un the Greater Paris area : 100 000 staff
members – doctors, researchers, paramedical staff, administrative
personnel and workers – work there. http://www.aphp.fr
About the Imagine Institute: As the leading European
center for research, care and teaching in genetic diseases, the
Imagine Institute's primary aim is to understand and cure. The
Institute's staff includes 850 of the best physicians, scientists
and healthcare professionals housed in an innovative new building
designed to realize synergies. This unprecedented continuum of
expertise available in close proximity to patients allows Imagine
to accelerate discoveries and their application at the bedside.
www.institutimagine.org
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version on businesswire.com: http://www.businesswire.com/news/home/20161203005050/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,
973-271-6085orAP-HP:Anne-Cécile Bard and Marine Leroy, +33
(0)1 40 27 37 22service.presse@aphp.frorImagine:Béatrice
Parinello-Froment, +33 (0)6 63 72 16
06beatriceparrinello@bpfconseil.comorClémence Inglard, +33 (0)1 42
75 46 44clemence.inglard@institutimagine.org
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