Data Include Updates from Phase 2/3 Starbeam
Study (ALD-102) of Lenti-D for CALD and Ongoing Observational Study
(ALD-103) of Allogeneic Hematopoietic Stem Cell Transplant for
CALD
bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data
from the clinical development program for its investigational
Lenti-D™ gene therapy in patients with cerebral
adrenoleukodystrophy (CALD) will be presented at the 13th European
Pediatric Neurology Society (EPNS) Congress, to be held September
17 – 21, 2019 in Athens, Greece.
Data will include updates from the ongoing Phase 2/3 Starbeam
study (ALD-102) which assesses the efficacy and safety of Lenti-D
gene therapy in boys 17 years of age and under with CALD. The
company will also present updated data from its ongoing
observational study (ALD-103) of allogeneic hematopoietic stem cell
transplant (allo-HSCT) in boys 17 years of age and under with
CALD.
Oral Presentations at
EPNS
Phase 2/3 Trial to Assess the Safety and Efficacy of Lenti-D
Hematopoietic Stem Cell Gene Therapy for Cerebral
Adrenoleukodystrophy
Presenter: Dr. Caroline Sevin, Hôpital Universitaire
Bicêtre-Paris Sud, Paris, France Date & Time (Parallel
Session 2D: Neurometabolic Disorders I): Wednesday,
September 18, 2019, 4:30 – 6:15 p.m. GMT+3 (9:30 – 11:15 a.m.
EDT)
An observational study of outcomes of Allogeneic
Hematopoietic Stem Cell Transplant in patients with Cerebral
Adrenoleukodystrophy (CALD)
Presenter: Dr. Florian Eichler, Center for Rare
Neurological Diseases, Associate Professor of Neurology,
Massachusetts General Hospital, Harvard Medical School, Boston, USA
Date & Time (Parallel Session 2D: Neurometabolic
Disorders I): Wednesday, September 18, 2019, 4:30 – 6:15 p.m.
GMT+3 (9:30 – 11:15 a.m. EDT)
Clinical Development Program for Lenti-D
The Lenti-D clinical development program is assessing the
efficacy and safety of an investigational gene therapy in boys up
to 17 years of age with CALD. Gene therapy for CALD consists of the
patient’s own stem cells transduced with Lenti-D lentiviral vector
that encodes ABCD1 cDNA for human adrenoleukodystrophy protein
(ALDP). This allows for the production of functional ALDP that
enables the local degradation of very long chain fatty acids
(VLCFAs) in the brain. Buildup of VLCFAs in the central nervous
system contributes to neurodegeneration in CALD.
The Phase 2/3 Starbeam study has completed enrollment. Visit
ClinicalTrials.gov for more information.
bluebird bio is currently enrolling a Phase 3 study (ALD-104)
designed to assess the efficacy and safety of Lenti-D in boys 17
years of age or younger with CALD after myeloablative conditioning
with busulfan and fludarabine. Visit ClinicalTrials.gov for more
information and a list of study sites.
bluebird bio is also conducting a long-term safety and efficacy
follow-up study (LTF-304) for patients who have completed two years
of follow-up in bluebird bio-sponsored studies of Lenti-D for CALD
(ALD-102 or ALD-104). Visit ClinicalTrials.gov for more
information.
In addition, the company continues to conduct ongoing
observational study (ALD-103) of allo‑HSCT in boys 17 years of age
or younger with CALD. Visit ClinicalTrials.gov for more
information.
The European Medicines Agency accepted Lenti-D gene therapy for
the treatment of CALD into its Priorities Medicines (PRIME) scheme
in July 2018, and previously granted Lenti-D Orphan Medicinal
Product designation for the treatment of adrenoleukodystrophy
(ALD).
The United States Food and Drug Administration granted Lenti-D
Orphan Drug status for the treatment of ALD, and Rare Pediatric
Disease designation and Breakthrough Therapy designation for the
treatment of CALD.
About Cerebral Adrenoleukodystrophy
ALD is a rare, X-linked metabolic disorder that is estimated to
affect one in 21,000 male newborns worldwide. ALD is caused by
mutations in the ABCD1 gene that affect the production of ALDP and
subsequently cause toxic accumulation of VLCFAs, primarily in the
adrenal cortex and white matter of the brain and spinal cord.
Approximately 35-40 percent of boys with ALD will develop CALD,
the most severe form of ALD. CALD is a progressive neurogenerative
disease that involves breakdown of myelin, the protective sheath of
the nerve cells in the brain that are responsible for thinking and
muscle control. Symptoms of CALD usually occur in early childhood
and progress rapidly, if untreated, leading to severe loss of
neurologic function, and eventual death, in most patients.
Currently, the only therapeutic option for patients with CALD is
allo-HSCT. Beneficial effects have been reported if allo-HSCT is
performed early in the course of cerebral progression. Potential
complications of allo-HSCT, which can be fatal, include graft
failure, GvHD, and opportunistic infections, particularly in
patients who do not receive transplant using cells from a matched
sibling donor.
Early diagnosis of CALD is important, as the outcome of
treatment varies with the clinical stage of the disease at the time
of transplant. Newborn screening for ALD is a critical enabler of
early diagnosis and successful treatment of ALD. In the U.S.,
newborn screening for ALD was added to the Recommended Universal
Screening Panel in February 2016 but is currently active in only a
limited number of states. Outside the U.S., the Minister of Health
in the Netherlands has approved the addition of
adrenoleukodystrophy to the newborn screening program, and a pilot
will start in 2019.
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 by researching cerebral adrenoleukodystrophy, sickle cell
disease, transfusion-dependent 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.
Lenti-D 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 clinical development and
commercial potential of the Company’s Lenti-D product candidate to
treat cerebral adrenoleukodystrophy. 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, risks that the
efficacy and safety results for our Lenti-D product candidate from
the Starbeam Study seen to date will not continue or persist, the
risk of cessation or delay of any of the ongoing clinical studies
and/or our development of Lenti-D, the risks regarding future
potential regulatory approvals of Lenti-D, including the risk that
the Starbeam Study will be insufficient to support regulatory
submissions or marketing approval in the U.S. and EU, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190903005276/en/
Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Media: Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com
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