– 15/17 patients (88%) in initial study cohort remain free of major
functional disabilities (MFDs) at 24 months –
cohort enrolling additional patients to gain European manufacturing
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 26, 2017--
bluebird bio, Inc. (Nasdaq:
BLUE), a clinical-stage company committed to developing potentially
transformative gene therapies for severe genetic diseases and T
cell-based immunotherapies for cancer, announced topline interim data
from the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam
Study (ALD-102) evaluating Lenti-D™ investigational gene therapy in boys
under 18 years old with cerebral adrenoleukodystrophy (CALD).
“The hope that Lenti-D may benefit boys facing such a devastating
disease inspires all of us at bluebird,” said David Davidson, M.D.,
chief medical officer, bluebird bio. “Having this proportion of the
initial cohort of patients meet the primary endpoint is truly
gratifying, bringing us one step closer to our goal of making Lenti-D
available for patients with CALD. The two patients who did not meet the
primary endpoint underscore the devastating nature of CALD, the
importance of early diagnosis through newborn screening, and the
challenges of the current standard of care with allogeneic hematopoietic
stem cell transplant (HSCT). Patient 2016 had not experienced an MFD,
but withdrew from the study due to radiographic progression of disease
and underwent an allogeneic HSCT. He subsequently died from
complications of the allogeneic transplantation. Patient 2018, as
previously reported in April 2016, had rapid disease progression
beginning early in his participation in the study, resulting in a
neurological function score (NFS) of 5. The rapidity of his disease
progression suggests it would have been difficult to alter his early
neurological decline given that transplant takes months to exert a
therapeutic effect in CALD. Our hearts go out to these two boys and
their families. Our program would not be possible without our brave
patients and their families, and we are tremendously grateful for their
As of June 13, 2017, 17 patients with CALD have completed 2 years of
follow-up post-Lenti-D treatment, with 15/17 (88%) remaining free of
major functional disabilities (MFDs), the primary endpoint of the trial.
This exceeds the pre-defined interim efficacy benchmark for the study of
MFD-free survival of 76%, derived from the literature and based on
clinical data from an earlier observational study describing that
natural history of CALD and outcomes from allogeneic HSCT.
In the Starbeam Study, the safety profile of Lenti-D was consistent with
myeloablative conditioning. No patients treated with Lenti-D had graft
versus host disease (GvHD), and there was no graft rejection or clonal
In December 2016, bluebird bio announced that the Starbeam study had
been expanded to treat eight additional patients at sites in Europe and
the US, and the study is currently enrolling the additional patients.
The expansion of the study is intended to enable the first manufacture
of Lenti-D in Europe, the subsequent treatment of subjects in Europe,
and to bolster the overall clinical data package for potential future
regulatory filings in the United States and Europe.
About the Starbeam (ALD-102) Study
The Starbeam Study is
assessing the efficacy and safety of an investigational gene therapy in
boys up to 17 years of age with CALD. It involves transplantation with a
patient’s own stem cells, which are modified to contain functional
copies of the ABCD1 gene. This gene addition should result in the
production of functional adrenoleukodystrophy protein (ALDP), a protein
critical for the breakdown of very long chain fatty acids (VLCFAs).
Buildup of VLCFAs in the central nervous system contributes to
neurodegeneration in CALD.
Subjects enrolled in the study are:
Eligible for allogeneic hematopoietic stem cell transplant (HSCT) but
with no matched sibling donor
Confirmed early-stage, active CALD as indicated by gadolinium
enhancement on MRI
Have a Loes score between 0.5 – 9.0
Have an NFS of one or less
Also known as Lorenzo’s Oil disease,
adrenoleukodystrophy (ALD) is estimated to affect one in every 21,000
male births worldwide. The cerebral form of the disease, cerebral
adrenoleukodystrophy (CALD), is a potentially fatal form of ALD that
affects the nervous system of boys. CALD involves a breakdown of the
protective sheath of the nerve cells in the brain that are responsible
for thinking and muscle control.
Currently, the only effective treatment option for patients with CALD is
allogeneic hematopoietic stem cell transplant (HSCT). Potential
complications of allogeneic HSCT, which can be fatal, include graft
failure, graft versus host disease (GVHD) and opportunistic infections,
particularly in patients who undergo allogeneic HSCT using cells from a
donor who is not a matched, unaffected sibling.
Early diagnosis of CALD is important, as the outcome of HSCT varies with
clinical stage of the disease at the time of transplant. Favorable
outcomes have been observed in patients who undergo transplant in the
early stages of cerebral disease. In the United States, newborn
screening for ALD was added to the Recommended Universal Screening Panel
(RUSP) in February, 2016. Newborn screening for ALD is active in a
limited number of states in the US.
About bluebird bio, Inc.
With its lentiviral-based gene
therapies, T cell immunotherapy expertise and gene editing capabilities,
bluebird bio has built an integrated product platform with broad
potential application to severe genetic diseases and cancer. bluebird
bio’s gene therapy clinical programs include its Lenti-D™ product
candidate, currently in a Phase 2/3 study, called the Starbeam Study,
for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™
product candidate, currently in four clinical studies for the treatment
of transfusion-dependent β-thalassemia, and severe sickle cell disease.
bluebird bio’s oncology pipeline is built upon the company’s leadership
in lentiviral gene delivery and T cell engineering, with a focus on
developing novel T cell-based immunotherapies, including chimeric
antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird
bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program
partnered with Celgene. bb2121 is currently being studied in a Phase 1
trial for the treatment of relapsed/refractory multiple myeloma.
bluebird bio also has discovery research programs utilizing
megaTAL/homing endonuclease gene editing technologies with the potential
for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Washington and Europe.
This release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements regarding
the clinical and market potential of the Company’s Lenti-D product
candidate. 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, the risk that the preliminary efficacy and safety
data for our Lenti-D product candidate from the Starbeam Study 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, and the risk
that any one or more of our product candidates will not be successfully
developed and 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: http://www.businesswire.com/news/home/20170626006173/en/
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107