bluebird bio, Inc. (Nasdaq: BLUE) announced today
business and program updates across its severe genetic disease
portfolio including a revised diagnosis for the previously reported
case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of
LentiGlobin for sickle cell disease (SCD) (bb1111), the company’s
decision to withdraw ZYNTEGLO™ (betibeglogene autotemcel, beti-cel)
for transfusion-dependent β-thalassemia (TDT) from the German
market and a targeted reshaping of its workforce intended to enable
the company to advance its late-stage gene therapy programs.
The case of MDS reported in February in a patient from Group C
of the Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD
has been further assessed following the review of results from
additional tests. The treating investigator has concluded this is
not a case of MDS and has revised the diagnosis to
transfusion-dependent anemia. bluebird bio has reported this update
to regulatory agencies and study investigators. The company
continues to work with the treating investigator to determine the
potential cause of this patient’s anemia.
Last month, the company reported that it is very unlikely the
suspected unexpected serious adverse reaction (SUSAR) of acute
myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin
for SCD was related to the BB305 lentiviral vector (LVV). This
assessment, along with the re-classification of the originally
reported MDS case to transfusion-dependent anemia are important
steps in bluebird bio’s path to seeking removal of the clinical
hold on studies HGB-206 and HGB-210 of LentiGlobin for SCD.
bluebird bio continues to work with regulators to resume its
clinical studies in sickle cell disease as well as to remove the
clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel
for β-thalassemia, with potential lift of all clinical holds in
mid-2021.
In Europe, reimbursement negotiations in Germany did not result
in a price for ZYNTEGLO that reflects the value of this one-time
gene therapy with potential life-long benefit for people living
with TDT. The price proposed by the German health authorities fails
to recognize the severe burden of living with TDT or the innovation
and benefit ZYNTEGLO brings to patients who are impacted every day,
throughout their lives by this severe genetic disease.
bluebird bio continues with productive negotiations across
countries in Europe and we plan to continue to provide updates on
the negotiation processes in the second half of 2021.
In response to these and other events and shifts related to the
business over the past year, bluebird bio plans to reduce and
reshape its workforce, primarily in Europe. This reduction and
reallocation of resources will allow the company to focus on
priority European markets and streamline global operations going
forward to ensure its ability to deliver gene therapies to patients
based on bluebird bio’s current business plans.
“We remain committed to our pioneering mission to deliver
one-time gene therapies with life-long benefits to our patients. We
are grateful for the clinical investigators and healthcare
providers helping us better understand the recent safety events in
our sickle cell disease studies. We are confident that working with
the FDA and EMA, we will be able to determine a positive path
forward as we seek to re-open our clinical studies. Further,
through our continued engagement across Europe, we are optimistic
that countries will reach pricing decisions that recognize the
value of one-time gene therapies and provide the necessary access
to the people who need them,” said Andrew Obenshain, president,
severe genetic diseases, bluebird bio. “In terms of operations, we
have faced challenges over the last year that have resulted in the
difficult decision to reduce our workforce and say goodbye to some
valued bluebirds. We want to express our gratitude for their
contributions and commitment to patients. As we move into the
future, we look forward to bluebird bio advancing as a strong,
thriving organization that is dedicated to developing treatments
for rare genetic diseases.”
About HGB-206 and HGB-210
HGB-206 is a Phase 1/2 open-label study designed to evaluate the
efficacy and safety of LentiGlobin gene therapy for sickle cell
disease (SCD) that includes three treatment cohorts: Groups A, B
and C. A refined manufacturing process designed to increase vector
copy number (VCN) and further protocol refinements made to improve
engraftment potential of gene-modified stem cells were used for
Group C. Group C patients also received LentiGlobin for SCD made
from HSCs collected from peripheral blood after mobilization with
plerixafor, rather than via bone marrow harvest, which was used in
Groups A and B of HGB-206.
HGB-210 is a Phase 3 single-arm open-label study designed to
evaluate the efficacy and safety of LentiGlobin gene therapy for
SCD in patients between two years and 50 years of age with sickle
cell disease.
About LentiGlobin for SCD (bb1111)
LentiGlobin gene therapy for sickle cell disease (bb1111) is an
investigational treatment being studied as a potential treatment
for SCD. bluebird bio’s clinical development program for
LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study,
the Phase 1/2 HGB-206 study, and the Phase 3 HGB-210 study.
The U.S. Food and Drug Administration (FDA) granted orphan drug
designation, fast track designation, regenerative medicine advanced
therapy (RMAT) designation and rare pediatric disease designation
for LentiGlobin for SCD.
LentiGlobin for SCD received orphan medicinal product
designation from the European Commission for the treatment of SCD,
and Priority Medicines (PRIME) eligibility by the European
Medicines Agency (EMA) in September 2020.
bluebird bio is conducting a long-term safety and efficacy
follow-up study (LTF-307) for people who have participated in
bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For
more information visit:
https://www.bluebirdbio.com/our-science/clinical-trials or
clinicaltrials.gov and use identifier NCT04628585 for
LTF-307.
LentiGlobin for SCD is investigational and has not been approved
in any geography.
About Zynteglo (betibeglogene autotemcel)
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy
that 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 adult Hb, at levels that may eliminate or
significantly reduce the need for transfusions. In studies of
beti-cel, transfusion independence (TI) is defined as no longer
needing red blood cell transfusions for at least 12 months while
maintaining a weighted average Hb of at least 9 g/dL.
The European Commission granted conditional marketing
authorization (CMA) for beti-cel, marketed as ZYNTEGLO™ gene
therapy, for patients 12 years and older with transfusion-dependent
β-thalassemia (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.
Non-serious adverse events (AEs) observed during clinical
studies that were attributed to beti-cel included abdominal pain,
thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain
in extremity, tachycardia and non-cardiac chest pain. One serious
adverse event (SAE) of thrombocytopenia was considered possibly
related to beti-cel.
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.
For details, please see the Summary of Product Characteristics
(SmPC).
On April 28, 2020, the European Medicines Agency (EMA) renewed
the CMA for beti-cel. The CMA for beti-cel is valid in the 27
member states of the EU as well as the UK, Iceland, Liechtenstein
and Norway.
The U.S. Food and Drug Administration (FDA) granted beti-cel
Orphan Drug status and Breakthrough Therapy designation for the
treatment of TDT. Beti-cel is not approved in the U.S. Beti-cel
continues to be evaluated in the ongoing Phase 3 Northstar-2
(HGB-207) and Northstar-3 (HGB-212) studies.
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 ZYNTEGLO.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
Cambridge, Mass., headquarters, we’re developing gene and cell
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: cerebral adrenoleukodystrophy, sickle cell disease,
β-thalassemia and multiple myeloma, using gene and cell therapy
technologies including gene addition, 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, betibeglogene autotemcel, beti-cel, 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,
including statements regarding the Company’s timing and
expectations regarding regulatory interactions to lift the clinical
hold on its HGB-206 and HGB-210 studies, anticipated timing for the
submission of the BLA to the FDA for LentiGlobin for SCD in the
United States, ongoing pricing & reimbursement negotiations for
Zynteglo in Europe, and regarding the Company’s business strategy
and investments, including the cause and impact of the Company’s
reduction in force and related activities. 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,
many of which are beyond the Company’s control. These risks and
uncertainties include, but are not limited to: the risk that we may
not be able to address regulatory authorities’ concerns quickly or
at all regarding the risk of insertional oncogenesis or MDS from
the use of our product candidates; the risk that we may not resume
patient treatment with Zynteglo in the commercial context in a
timely manner or at all; the risk that we may not reach agreement
with payors in Europe on the price for Zynteglo; the risk that we
may not be able to execute on our business plans, including our
commercialization plans, meeting our expected or planned regulatory
milestones, submissions, and timelines, research and clinical
development plans, and in bringing our product candidates to
market; the difficulties in and effect of implementing the
Company’s reduction in force, such as claims arising out of the
reduction; the risk that the planned reduction in force does not
have the anticipated outcomes or impacts, including the risk that
the actual financial and other impacts of the reduction could vary
materially from the outcomes or impacts anticipated; and the risk
that with the impact on the execution and timing of our business
plans, we may not successfully execute our previously announced
plans to spin off our oncology programs into an independent
publicly-traded entity. 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-K, 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/20210420005511/en/
Media: Jenn Snyder, 617-448-0281
jsnyder@bluebirdbio.com
Catherine Falcetti, 617-583-3411 cfalcetti@bluebirdbio.com
Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
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