– Two of three patients with severe sickle cell
disease show >45 percent total anti-sickling hemoglobin levels
and meaningful clinical improvement; all three show rising
trajectory of HbAT87Q production in first six months post-drug
product infusion –
– Ongoing transfusion independence up to 3.8
years in patients with transfusion-dependent β-thalassemia; three
of four patients have normal or near-normal total hemoglobin levels
–
– Safety profile of LentiGlobin remains
consistent with that of myeloablative conditioning with
single-agent busulfan –
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 updated data from the ongoing HGB-205 clinical
study of its LentiGlobin gene therapy product candidate in patients
with severe sickle cell disease (SCD) and transfusion-dependent
β-thalassemia (TDT). The findings will be presented today in a
poster session at the 59th Annual Meeting of the American
Society of Hematology (ASH).
“People with SCD and TDT experience serious complications and
organ damage as a result of their disease and complications from
chronic blood transfusions. Addressing the underlying genetic
causes of these diseases has the potential to dramatically improve
patient outcomes,” said Dave Davidson, M.D., chief medical officer,
bluebird bio. “All three patients with severe SCD in the HGB-205
study showed a steady increase in HbAT87Q production in the first
six months following LentiGlobin therapy, with the longest-treated
patient showing stable hemoglobin levels over two and a half years.
All four patients with TDT are transfusion-free following therapy,
up to almost four years in the first patient treated. The durable
treatment effects observed to date in this study are encouraging,
particularly given the manufacturing process improvements that we
implemented across our subsequent clinical studies of LentiGlobin,
and additional changes to the HGB-206 study protocol that we hope
will further improve outcomes for patients with SCD.”
These data will be presented by Marina Cavazzana, M.D., Ph.D.,
Professor of Medicine at Paris Descartes University and Research
Director at the Centre for Clinical Research in Biotherapy, Necker
Hospital, and at the Institute of Genetic Diseases, Imagine, Paris,
France. Professor Cavazzana is the primary investigator of the
HGB-205 study.
“All seven patients in this study continue to experience notable
clinical improvement. Since being treated with LentiGlobin therapy,
the four patients with TDT have been free of chronic transfusions
with near normal and stable levels of total hemoglobin,” said
Professor Cavazzana. “While progress has been made with medications
to treat SCD and TDT, we are in need of better options for our
patients. This study suggests that LentiGlobin has the potential to
be a transformational one-time therapy for people with SCD and
TDT.”
Longer Term Follow-up on the First Patients with Severe
Hemoglobinopathies Treated with LentiGlobin Gene Therapy (Poster
Abstract #4609)
Presenter: Marina Cavazzana, M.D., Ph.D. Necker-Enfants
Malades Hospital, Paris, France
Poster Session Date & Time: Monday, December 11 at
6:00 p.m.
Location: Building A, Level 1, Hall A2
HGB-205 is an ongoing, open-label, single-center Phase 1/2 study
designed to evaluate the safety and efficacy of LentiGlobin drug
product (DP) in the treatment of patients with severe SCD and TDT.
The study enrolled three patients with severe SCD and four patients
with TDT, who have undergone infusion with LentiGlobin DP. Results
as of September 20, 2017 include:
SCD:
- All three treated patients showed
rising HbAT87Q levels in the first six months.
- Patient 1204 was 13 years old at study
enrollment. At last follow-up (35.2 months), this patient had a
total hemoglobin of 12.4 g/dL, of which 6.1 g/dL was HbAT87Q (52
percent anti-sickling Hb). HbAT87Q concentration in this patient
has remained stable since approximately nine months post-infusion.
The patient continues to show marked clinical improvement.
- Patient 1207 was 16 years old at study
enrollment. At last follow-up (8.9 months), this patient had a
total hemoglobin of 10.0 g/dl, of which 0.7 g/dl was HbAT87Q (14
percent anti-sickling Hb). This patient had a pre-treatment history
of frequent episodes of vaso-occlusive crisis (VOC) and acute chest
syndrome (ACS) despite hydroxyurea prior to beginning regular
transfusions. Patient 1207 had episodes of ACS and hospitalization
at six and eight months post-treatment, and received three
transfusions.
- Patient 1208 was 21 years old at study
enrollment. At last follow-up (6.0 months), this patient had a
total hemoglobin of 10.6 g/dL, of which 2.7 g/dL was HbAT87Q (46
percent total anti-sickling Hb). This patient had a pre-treatment
history of frequent episodes of VOCs and ACS prior to beginning
regular transfusions, and was still symptomatic while receiving
regular transfusions. Following LentiGlobin treatment, Patient 1208
has had no episodes of VOCs or ACS (with six months
follow-up).
TDT:
- All four patients with TDT have
remained free of chronic transfusions since shortly after receiving
LentiGlobin DP.
- Patient 1201 (β0/βE genotype) has been
free of transfusions for 45.2 months with total hemoglobin of 10.1
g/dL, of which 6.7 g/dL was HbAT87Q.
- Patient 1202 (β0/βE genotype) has been
free of transfusions for 40.1 months with total hemoglobin of 12.9
g/dL, of which 10.1 g/dL was HbAT87Q.
- Patient 1206 (β0/βE genotype) has been
free of transfusions for 23.8 months with total hemoglobin of 11.1
g/dL, of which 8.0 g/dL was HbAT87Q.
- Patient 1203, who is homozygous for the
severe β+ mutation IVS1-110, has been free of transfusions for 20.9
months with total hemoglobin of 8.7 g/dL, of which 6.7 g/dL was
HbAT87Q.
- Three of four patients (1201, 1202 and
1206) were able to begin therapeutic phlebotomy. Patient 1202
subsequently discontinued iron chelation and phlebotomy.
- The safety profile of LentiGlobin DP
continues to be consistent with myeloablative conditioning with
single-agent busulfan. No DP-related adverse events have been
observed, and there is no evidence of clonal dominance.
About SCDSickle 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 TDTTransfusion-dependent β-thalassemia (TDT) is a
severe genetic disease characterized by reduced or absent
hemoglobin levels that results in severe anemia and ineffective red
blood cell production. Supportive care for people with TDT consists
of a lifelong regimen of chronic blood transfusions to enable
survival and suppress symptoms of the disease, and iron chelation
therapy to manage iron overload that results from the transfusions.
Despite the availability of supportive care, many people with TDT
experience serious complications and organ damage due to underlying
disease and iron overload.
Allogeneic HSCT is currently the only available option to
address the underlying genetic cause of TDT, though it carries
significant risks. Complications of allogeneic HSCT include a risk
of treatment-related mortality, graft failure, GvHD and
opportunistic infections, particularly in patients who undergo
non-sibling matched allogenic HSCT.
About the HGB-205 StudyHGB-205 is an ongoing, open-label
Phase 1/2 study designed to evaluate the safety and efficacy of
LentiGlobin in the treatment of subjects with TDT and SCD. The
study enrolled seven subjects who will be followed to evaluate
safety and transfusion requirements post-transplant. Among patients
with sickle cell disease 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
clinicaltrials.gov using identifier NCT02151526.
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 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 manufacturing process
changes for LentiGlobin will improve outcomes of patients with
transfusion-dependent ß-thalassemia and severe sickle cell disease
and the potential long-term durable treatment effect of
LentiGlobin. 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20171211005582/en/
bluebird bioElizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.com
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