June 9 discussion will focus on eli-cel for the
treatment of early active cerebral adrenoleukodystrophy in patients
without a matched sibling donor and overall safety of lentiviral
vector (LVV) gene therapy
June 10 discussion will focus on beti-cel for
the treatment of β-thalassemia in patients who require regular red
blood cell transfusions
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today announced that the U.S. Food and Drug
Administration (FDA) posted briefing documents for the FDA
Cellular, Tissue and Gene Therapies Advisory Committee Meeting to
review elivaldogene autotemcel (eli-cel) for early active cerebral
adrenoleukodystrophy (CALD) in patients without a matched sibling
donor and betibeglogene autotemcel (beti-cel) for the treatment of
people with β-thalassemia who require regular red blood cell
transfusions.
The advisory committee meeting will take place June 9-10, 2022.
Briefing materials, agendas and webcast information for the meeting
can be accessed here. The Company is not responsible for the
content of, nor the statements made in, the briefing materials that
were prepared by the FDA.
The Prescription Drug User Fee Act (PDUFA) goal dates for a
decision on approval of beti-cel for people with β-thalassemia who
require regular red blood cell transfusions and eli-cel for
patients with early active CALD without a matched sibling donor are
August 19, 2022, and September 16, 2022, respectively.
About elivaldogene autotemcel (eli-cel)
eli-cel (pronounced ELL-ee-cell) uses ex-vivo transduction with
the Lenti-D lentiviral vector (LVV) to add functional copies of the
ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs).
The addition of the functional ABCD1 gene allows patients to
produce the ALD protein (ALDP), which is thought to facilitate the
breakdown of very long-chain fatty acids (VLCFAs). The expression
of ALDP and effect of eli‑cel is expected to be life-long. The goal
of treatment with eli-cel is to stop the progression of CALD and,
consequently, preserve as much neurological function as possible,
including the preservation of motor function and communication
ability. Importantly, with eli-cel, there is no need for donor HSCs
from another person.
bluebird bio’s clinical development program for eli-cel includes
the completed pivotal Phase 2/3 Starbeam study (ALD-102) and the
ongoing Phase 3 ALD-104 study, which has completed enrollment and
treatment of all patients. Additionally, bluebird bio is conducting
a long-term safety and efficacy follow-up study (LTF-304) for
patients who have received eli-cel for CALD and completed two years
of follow-up in ALD-102 or ALD-104. Clinical studies of eli-cel
were placed on hold by the FDA and follow-up of all patients
continues, per protocol.
In ALD-102, 90.6% (29/32) of patients met the primary endpoint
of Major Functional Disabilities (MFD)-free survival at 24 months.
As previously reported, two patients withdrew from ALD-102 at
investigator discretion, and one additional subject experienced
rapid disease progression early after treatment, resulting in MFDs
and subsequent death. All patients who completed ALD-102 enrolled
in a long-term follow-up study (LTF-304). The median duration of
follow-up is approximately four years (49 months; 13.4, 88.1
months).
Adverse reactions attributed to eli-cel observed in clinical
trials include myelodysplastic syndrome, viral cystitis,
pancytopenia, and nausea and vomiting. There have been no reports
of graft-versus-host-disease, graft failure or rejection,
transplant-related mortality, or replication-competent lentivirus
in the 67 patients who received eli-cel and are being followed in
clinical studies (ALD-102, ALD-104, LTF-304).
About betibeglogene autotemcel (beti-cel)
betibeglogene autotemcel (beti-cel) (pronounced BEH tee cell) is
a one-time gene therapy custom-designed to treat the underlying
cause of β-thalassemia in patients who require regular red blood
cell (RBC) transfusions. beti-cel 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) in order to
correct the deficiency of adult hemoglobin that is the hallmark of
β-thalassemia. Once a patient has the βA-T87Q-globin gene, they
have the potential to produce beti-cel-derived adult hemoglobin
(HbAT87Q) at levels that may eliminate the need for transfusions.
As of the data cut in August 2021, 89% (34/38) of evaluable
patients in Phase 3 beti-cel studies achieved transfusion
independence, which is defined as no longer needing RBC
transfusions for at least 12 months while maintaining a weighted
average Hb of at least 9 g/dL. These results were observed across
all ages and genotypes, including pediatric patients as young as
four years of age and those with the most severe (β0/β0)
genotypes.
beti-cel is manufactured using the BB305 lentiviral vector
(LVV), a third-generation, self-inactivating LVV that has been
studied for more than a decade across two therapeutic areas.
Adverse reactions considered related to beti-cel were infrequent
and consisted primarily of non-serious infusion-related reactions
that occurred on the day of infusion (e.g., abdominal pain, hot
flush, dyspnea, tachycardia and non-cardiac chest pain) and
cytopenias (e.g., thrombocytopenia, leukopenia and neutropenia).
One of these adverse reactions was a serious adverse event (SAE) of
thrombocytopenia considered possibly related to beti-cel and has
resolved.
The majority of AEs and SAEs in the beti-cel clinical
development program were unrelated to beti-cel and largely reflect
the known side effects of HSC collection and busulfan conditioning
regimen (including several SAEs of veno-occlusive disease that
resolved with treatment).
The Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212)
studies evaluating beti-cel are ongoing; enrollment is complete,
and all patients have been treated. bluebird bio is also conducting
a long-term follow-up study, LTF-303, to monitor safety and
efficacy for people who have participated in bluebird bio-sponsored
beti-cel clinical studies through 15 years post-treatment.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days.
With a dedicated focus on severe genetic diseases, bluebird has
industry-leading clinical programs for sickle cell disease,
β-thalassemia and cerebral adrenoleukodystrophy and is advancing
research to apply new technologies to these and other diseases. We
custom design each of our therapies to address the underlying cause
of disease and have developed in-depth and effective analytical
methods to understand the safety of our lentiviral vector
technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo
gene therapy data set in the world—setting the standard for the
industry. Today, bluebird continues to forge new paths, combining
our real-world experience with a deep commitment to patient
communities and a people-centric culture that attracts and grows a
diverse flock of dedicated birds.
For more information, visit bluebirdbio.com or follow us
on social media at @bluebirdbio, LinkedIn,
Instagram and YouTube.
bluebird bio is a trademark of bluebird bio, Inc.
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, including
our statements regarding the Company’s plans and expectations for
anticipated FDA approval of the BLAs for beti-cel and eli-cel. Such
forward-looking statements are based on historical performance and
current expectations about our future 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, particularly those identified in the risk factors
discussion 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. These risks include, but are not limited to: we may
encounter additional delays in the development of our programs,
including the imposition of new clinical holds or delays in
resolving existing clinical holds, that may impact our ability to
meet our expected timelines; the risk that the efficacy and safety
results from our prior and ongoing clinical trials will not
continue or be seen in additional patients treated with our product
candidates; the risk that additional insertional oncogenic or other
reportable events associated with lentiviral vector, drug product,
or myeloablation will be discovered or reported over time; the risk
that our eli-cel, beti-cel and lovo-cel programs may be subject to
further delays in their development, including but not limited to
the imposition of new clinical holds; the risk that eli-cel and/or
beti-cel may not be approved within the priority review timeframe
or at all; the risk that any one or more of our product candidates,
including eli-cel and/or beti-cel, will not be successfully
developed, approved or commercialized. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220607005850/en/
Investors: Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com or Media: Jess Rowlands,
857-299-6103 jess.rowlands@bluebirdbio.com
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