bluebird bio, Inc. (Nasdaq: BLUE) today announced that
the U.S. Food and Drug Administration (FDA) has lifted the clinical
holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of
LentiGlobin for sickle cell disease (SCD) gene therapy (bb1111) for
adult and pediatric patients with SCD, and the Phase 3 Northstar-2
(HGB-207) and Northstar-3 (HGB-212) studies of betibeglogene
autotemcel gene therapy (beti-cel; licensed as ZYNTEGLO™ in the EU
and the UK) for adult, adolescent and pediatric patients with
transfusion-dependent β-thalassemia (TDT). The company is working
closely with study investigators and clinical trial sites to resume
all study activities as soon as possible.
“Patient safety continues to be our utmost priority, and we are
grateful for the close partnership with the FDA, investigators,
scientists and most importantly, patients, who all contributed to
the assessments of the adverse events in HGB-206 that ultimately
led to today’s announcement,” said Andrew Obenshain, president,
severe genetic diseases, bluebird bio. “As pioneers in gene
therapy, we remain committed to advancing the field through our
learnings. Over the past four months, we have gained deeper
knowledge and understanding of the pathophysiology of sickle cell
disease that will allow us to better serve patients and the broader
community. We look forward to resuming our clinical programs and
continuing to advance toward major regulatory submissions for
sickle cell disease and β-thalassemia.”
Previously Reported Safety Events
On March 10, 2021, bluebird bio 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). No cases of hematologic malignancy have been reported in any
patient who has received treatment with beti-cel. On April 20,
2021, bluebird bio announced a revised diagnosis for the previously
reported case of myelodysplastic syndrome (MDS) in its Phase 1/2
study of LentiGlobin for SCD. Upon further assessment, the treating
investigator concluded this is not a case of MDS and revised the
diagnosis to transfusion-dependent anemia.
About LentiGlobin for SCD (bb1111)
LentiGlobin gene therapy for sickle cell disease (bb1111) is an
investigational treatment being studied as a potential one-time
therapy for SCD. bluebird bio’s clinical development program for
LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 and
the ongoing phase 1/2 HGB-206 and Phase 3 HGB-210 studies. In
addition, 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 on the studies, visit:
https://www.bluebirdbio.com/our-science/clinical-trials or
clinicaltrials.gov.
The FDA has 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.
LentiGlobin for SCD is investigational and has not been approved
in any geography.
About betibeglogene autotemcel (beti-cel)
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 hemoglobin (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 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 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. In November 2020,
bluebird bio submitted to the EMA an application for the second
renewal of the CMA. This procedure is currently on hold while the
EMA's Pharmacovigilance Risk Assessment Committee (PRAC) reviews
the safety of ZYNTEGLO. The CMA is valid while the renewal
application review is ongoing by the regulatory agency.
The FDA granted beti-cel Orphan Drug status and Breakthrough
Therapy designation for the treatment of TDT.
bluebird bio is on track to complete its rolling Biologics
License Application (BLA) submission to the FDA for beti-cel in
mid-2021. This submission is anticipated to include adult,
adolescent and children with transfusion dependent β-thalassemia
across all genotypes (including non-β0/β0 genotypes and β0/β0
genotypes). 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 beti-cel.
About bluebird bio, Inc.
bluebird bio, Inc. (NASDAQ: BLUE) 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 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, betibeglogene autotemcel, beti-cel, LentiGlobin for
SCD, bb1111 and bluebird bio are trademarks of bluebird bio,
Inc.
bluebird bio Cautionary Statement Regarding Forward-Looking
Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements that are not statements of historical facts
are, or may be deemed to be, forward-looking statements. Such
forward-looking statements are based on historical performance and
current expectations and projections about our future financial
results, goals, plans and objectives and involve inherent risks,
assumptions and uncertainties, including internal or external
factors that could delay, divert or change any of them in the next
several years, that are difficult to predict, may be beyond our
control and could cause our future financial results, goals, plans
and objectives to differ materially from those expressed in, or
implied by, the statements. No forward-looking statement can be
guaranteed. Forward-looking statements in this press release should
be evaluated together with the many risks and uncertainties that
affect bluebird bio’s business, which include but are not limited
to: the risk that insertional oncogenic events associated with
lentiviral vector or additional MDS events associated with
transplant or myeloablation will be discovered or reported over
time; the risk that insertional oncogenic events associated with
lentiviral vector in other programs may result in a clinical hold
of our programs in SCD, TDT or cerebral adrenoleukodystrophy; the
risk that we may experience delays in our ability to restart the
enrollment and conduct of our HGB-206 and HGB-210 clinical trials;
the risk that we may not be able to execute on our business plans,
including meeting our expected or planned regulatory milestones,
submissions or timelines, such as in the completion of our BLA
submission for beti-cel; the risk that LentiGlobin for SCD or
beti-cel will not be approved for marketing by the FDA, and the
risk that we will not successfully bring LentiGlobin for SCD or
beti-cel to market in the United States; the risk that we may not
resume patient treatment with ZYNTEGLO in the commercial context in
a timely manner or at all; 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
portfolio and programs into an independent publicly-traded company
on the timeline that we expect, or at all. 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 bluebird bio’s Annual Report on Form 10-K, as updated
by our subsequent Quarterly Reports on Form 10-Q, Current Reports
on Form 8-K and other filings with the Securities and Exchange
Commission. The forward-looking statements included in this
document are made only as of the date of this document and except
as otherwise required by applicable law, bluebird bio undertakes no
obligation to publicly update or revise any forward-looking
statement, whether as a result of new information, future events,
changed circumstances or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20210607005267/en/
Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Media: Jenn Snyder, 617-448-0281
jsnyder@bluebirdbio.com
Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com
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