Presentation of new and updated results from
ongoing Phase 1/2 HGB-206 study of LentiGlobin for sickle cell
disease will include additional patients treated in the study
New and updated data, including analysis of
healthy red blood cell production in patients with
transfusion-dependent β-thalassemia following treatment with
betibeglogene autotemcel (LentiGlobin™ for β-thalassemia) to be
shared
bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from
its gene therapy programs for sickle cell disease (SCD),
transfusion-dependent β-thalassemia (TDT) and its cell therapy
program for relapsed and refractory multiple myeloma (RRMM) will be
presented during the Virtual Edition of the 25th European
Hematology Association (EHA25) Annual Congress.
New data from the company’s Phase 1/2 HGB-206 study of
LentiGlobin™ gene therapy for SCD will be presented, including
updated data from patients in Group C.
bluebird bio will also present data from its ongoing clinical
studies of betibeglogene autotemcel (formerly LentiGlobin™ gene
therapy for β-thalassemia), including the Phase 3 Northstar-2
(HGB-207) study in patients who do not have a β0/β0 genotype and
the Phase 3 Northstar-3 (HGB-212) study in patients who have β0/β0,
β0/β+IVS-I-110, or β+IVS-I-110/β+IVS-I-110 genotypes.
Data from studies of idecabtagene vicleucel (ide-cel; bb2121),
the company’s anti-B-cell maturation antigen (BCMA) chimeric
antigen receptor (CAR) T cell therapy in development with Bristol
Myers Squibb, will be presented, including an encore presentation
of results from the pivotal Phase 2 KarMMa study.
Sickle Cell Disease Data at
EHA25
Oral Presentation: Outcomes in patients treated with
LentiGlobin for sickle cell disease (SCD) gene therapy: Updated
results from the Phase 1/2 HGB-206 group C study Presenting
Author: Julie Kanter, M.D., University of Alabama at
Birmingham, Birmingham, Ala.
Transfusion-Dependent β-Thalassemia
Data at EHA25
Oral Presentation: Improvement in erythropoiesis in patients
with transfusion-dependent β-thalassemia following treatment with
betibeglogene autotemcel (LentiGlobin for β-thalassemia) in the
Phase 3 HGB-207 study Presenting Author: John B. Porter, MA,
M.D., FRCP, FRCPath, University College London Hospital, London,
UK
Poster: Betibeglogene autotemcel (LentiGlobin) in patients
with transfusion-dependent β-thalassemia and β0/β0,
β+IVS-I-110/β+IVS-I-110, or β0/β+IVS-I-110 genotypes: Updated
results from the HGB-212 study Presenting Author: Evangelia
Yannaki, M.D., George Papanicolaou Hospital, Thessaloniki,
Greece
Multiple Myeloma Data at
EHA25
Oral Presentation: Phase II KarMMa study: Idecabtagene
vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in
patients with relapsed and refractory multiple myeloma Presenting
Author: Jesus San-Miguel, M.D., Ph.D., Clinica Universidad de
Navarra, Navarra, Spain
Poster: Quality of life in patients with relapsed and
refractory multiple myeloma treated with the BCMA-targeted CAR T
cell therapy Idecabtagene vicleucel (ide-cel; bb2121): results from
the KarMMa Trial Presenting Author: Michel Delforge, M.D.,
Ph.D., Leuven University College, Brussels, Belgium
Poster: Matching-adjusted indirect comparisons of efficacy
outcomes for idecabtagene vicleucel from the KarMMa study vs
selinexor PLUS dexamethasone (STORM part 2) and belantamab
mafodotin (DREAMM-2) Presenting Author: Paula Rodriguez-Otero,
M.D., Clinica Universidad de Navarra, Navarra, Spain
Poster: Baseline and postinfusion pharmcodynamic biomarkers
of safety and efficacy in patients treated with idecabtagene
vicleucel (ide-cel; bb2121) in the KarMMa study Presenting
Author: Justine Dell’Aringa, Bristol Myers Squibb, Seattle,
Wash.
Poster: Correlation of tumor BCMA expression with response
and acquired resistance to idecabtagene vicleucel in the KarMMa
study in relapsed and refractory multiple myeloma Presenting
Author: Nathan Martin, Bristol Myers Squibb, Seattle, Wash.
Abstracts outlining bluebird bio’s accepted data at the EHA25
Virtual Congress have been made available on the EHA25 conference
website. On Friday, June 12 at 8:30 AM CEST, the embargo
will lift for poster and oral presentations accepted for EHA25.
About betibeglogene autotemcel The European Commission
granted conditional marketing authorization (CMA) for betibeglogene
autotemcel, marketed as ZYNTEGLO™ gene therapy, for patients 12
years and older with TDT who do not have a β0/β0 genotype, for whom
hematopoietic stem cell (HSC) transplantation is appropriate, but a
human leukocyte antigen (HLA)-matched related HSC donor is not
available. On April 28, 2020, the European Medicines Agency (EMA)
renewed the CMA for ZYNTEGLO, supported by data from 32 patients
treated with ZYNTEGLO including three patients with up to five
years of follow-up.
TDT is a severe genetic disease caused by mutations in the
β-globin gene that result in reduced or significantly reduced
hemoglobin (Hb). In order to survive, people with TDT maintain Hb
levels through lifelong chronic blood transfusions. These
transfusions carry the risk of progressive multi-organ damage due
to unavoidable iron overload.
Betibeglogene autotemcel adds functional copies of a modified
form of the β-globin gene (βA-T87Q-globin gene) into a patient’s
own hematopoietic (blood) stem cells (HSCs). Once a patient has the
βA-T87Q-globin gene, they have the potential to produce HbAT87Q,
which is gene therapy-derived hemoglobin, at levels that may
eliminate or significantly reduce the need for transfusions.
Non-serious adverse events (AEs) observed during the clinical
studies that were attributed to betibeglogene autotemcel were
abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot
flush, dyspnoea, pain in extremity, and non-cardiac chest pain. One
serious adverse event (SAE) of thrombocytopenia was considered
possibly related to LentiGlobin for β-thalassemia for TDT.
Additional AEs observed in clinical studies were consistent with
the known side effects of HSC collection and bone marrow ablation
with busulfan, including SAEs of veno-occlusive disease.
The CMA for ZYNTEGLO is only valid in the 28 member states of
the EU as well as Iceland, Liechtenstein and Norway. For details,
please see the Summary of Product Characteristics (SmPC).
The U.S. Food and Drug Administration granted betibeglogene
autotemcel Orphan Drug status and Breakthrough Therapy designation
for the treatment of TDT. Betibeglogene autotemcel is not approved
in the United States.
Betibeglogene autotemcel continues to be evaluated in the
ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more
information about the ongoing clinical studies, visit
www.northstarclinicalstudies.com or
clinicaltrials.gov and use identifier NCT02906202 for
Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).
About LentiGlobin for Sickle Cell Disease LentiGlobin for
sickle cell disease is an investigational gene therapy being
studied as a potential treatment for SCD. bluebird bio’s clinical
development program for LentiGlobin for SCD includes the ongoing
Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.
SCD is a serious, progressive and debilitating genetic disease
caused by a mutation in the β-globin gene that leads to the
production of abnormal sickle hemoglobin (HbS), causing red blood
cells (RBCs) to become sickled and fragile, resulting in chronic
hemolytic anemia, vasculopathy and painful vaso-occlusive crises
(VOCs). For adults and children living with SCD, this means
unpredictable episodes of excruciating pain due to vaso-occlusion
as well as other acute complications—such as acute chest syndrome
(ACS), stroke, and infections, which can contribute to early
mortality in these patients.
LentiGlobin for SCD received Orphan Medicinal Product
designation from the European Commission for the treatment of
SCD.
The U.S. Food and Drug Administration (FDA) granted Orphan Drug
status and Regenerative Medicine Advanced Therapy designation for
LentiGlobin for the treatment of SCD.
LentiGlobin for SCD is investigational and has not been approved
by the European Medicines Agency (EMA) or FDA.
bluebird bio is conducting a long-term safety and efficacy
follow-up study (LTF-303) for people who have participated in
bluebird bio-sponsored clinical studies of betibeglogene autotemcel
and LentiGlobin for SCD. For more information visit:
https://www.bluebirdbio.com/our-science/clinical-trials or
clinicaltrials.gov and use identifier NCT02633943 for
LTF-303.
About idecabtagene vicleucel (ide-cel; bb2121) Ide-cel is
a B-cell maturation antigen (BCMA)-directed genetically modified
autologous chimeric antigen receptor (CAR) T cell immunotherapy.
The ide-cel CAR is comprised of a murine extracellular single-chain
variable fragment (scFv) specific for recognizing BCMA, attached to
a human CD8 α hinge and transmembrane domain fused to the T cell
cytoplasmic signaling domains of CD137 4-1BB and CD3-� chain, in
tandem. Ide-cel recognizes and binds to BCMA on the surface of
multiple myeloma cells leading to CAR T cell proliferation,
cytokine secretion, and subsequent cytolytic killing of
BCMA-expressing cells.
In addition to the pivotal KarMMa trial evaluating ide-cel in
patients with relapsed and refractory multiple myeloma, bluebird
bio and Bristol Myers Squibb’s broad clinical development program
for ide-cel includes clinical studies (KarMMa-2, KarMMa-3,
KarMMa-4) in earlier lines of treatment for patients with multiple
myeloma, including newly diagnosed multiple myeloma. For more
information visit clinicaltrials.gov.
Ide-cel was granted Breakthrough Therapy Designation (BTD) by
the U.S. Food and Drug Administration (FDA) and PRIority Medicines
(PRIME) designation, as well as Accelerated Assessment status, by
the European Medicines Agency for relapsed and refractory multiple
myeloma.
Ide-cel is being developed as part of a Co-Development,
Co-Promotion and Profit Share Agreement between Bristol Myers
Squibb and bluebird bio.
Ide-cel is not approved for any indication in any geography.
About KarMMa KarMMa (NCT03361748) is a pivotal,
open-label, single-arm, multicenter, multinational, Phase 2 study
evaluating the efficacy and safety of ide-cel in adults with
relapsed and refractory multiple myeloma in North America and
Europe. The primary endpoint of the study is overall response rate
as assessed by an independent review committee (IRC) according to
the International Myeloma Working Group (IMWG) criteria. Complete
response rate is a key secondary endpoint. Other efficacy endpoints
include time to response, duration of response, progression-free
survival, overall survival, minimal residual disease evaluated by
Next-Generation Sequencing (NGS) assay and safety. The study
enrolled 140 patients, of whom 128 received ide-cel across the
target dose levels of 150-450 x 10P6P CAR+ T cells after receiving
lymphodepleting chemotherapy. All enrolled patients had received at
least three prior treatment regimens, including an immunomodulatory
agent, a proteasome inhibitor and an anti-CD38 antibody, and were
refractory to their last regimen, defined as progression during or
within 60 days of their last therapy.
About bluebird bio, Inc. bluebird bio is pioneering gene
therapy with purpose. From our Cambridge, Mass., headquarters,
we’re developing gene therapies for severe genetic diseases and
cancer, with the goal that people facing potentially fatal
conditions with limited treatment options can live their lives
fully. Beyond our labs, we’re working to positively disrupt the
healthcare system to create access, transparency and education so
that gene therapy can become available to all those who can
benefit.
bluebird bio is a human company powered by human stories. We’re
putting our care and expertise to work across a spectrum of
disorders including cerebral adrenoleukodystrophy, sickle cell
disease, β-thalassemia and multiple myeloma, using three gene
therapy technologies: gene addition, cell therapy and
(megaTAL-enabled) gene editing.
bluebird bio has additional nests in Seattle, Wash.; Durham,
N.C.; and Zug, Switzerland. For more information, visit
bluebirdbio.com.
Follow bluebird bio on social media: @bluebirdbio,
LinkedIn, Instagram and YouTube.
ZYNTEGLO, LentiGlobin, and bluebird bio 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. 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:
regarding the potential for betibeglogene autotemcel to treat
transfusion-dependent β-thalassemia and the potential for
LentiGlobin for sickle cell disease (SCD) to treat SCD; and the
risk that the efficacy and safety results from our prior and
ongoing clinical trials will not continue or be repeated in our
ongoing or planned clinical trials. 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: https://www.businesswire.com/news/home/20200514005234/en/
Media: Catherine Falcetti, 339-499-9436
cfalcetti@bluebirdbio.com Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com
Investors: Ingrid Goldberg, 410-960-5022
Ingrid.goldberg@bluebirdbio.com Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
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