First clinical data to be presented from Phase 2/3 Starbeam Study;
Company to host investor webcast and conference call today at
“CALD is a rare and deadly disease, and currently the only available
treatment option is allogeneic hematopoietic stem cell transplant, which
is often challenging for patients without a matched sibling donor,” said
Interim Data Summary
The Starbeam Study is a global, multi-center study assessing the
efficacy and safety of an investigational gene therapy in boys up to 17
years of age with CALD. As of
The primary efficacy endpoint for the Starbeam Study is the proportion of patients with no major functional disabilities (MFDs) at 24 months post treatment. MFDs are six components of the neurological function score (NFS) that, if present, would have a profound negative impact on patients’ lives: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence and complete loss of voluntary movement. Secondary endpoints include Loes score (a method for quantifying demyelination and atrophy in patients with CALD using brain MRI), NFS (a scoring system assessing clinical deficits across 15 functional domains), gadolinium enhancement on MRI (an indicator of neuroinflammation), and safety.
Data from these 17 patients as of
- All patients remain free of MFDs.
Sixteen of 17 patients had NFS stabilization (change of <3 points and
an absolute NFS ≤ 4).
- Two patients have an NFS of 1, due to the occurrence of stuttering in one patient, and episodic urinary incontinence in another patient.
- One patient had an early, rapidly progressive course and has an NFS of 5, reflecting deficits in speech, vision, difficulty walking and running, and episodes of urinary incontinence.
Fourteen of 17 patients had a stable Loes score (change of ≤ 5 points
or an absolute
Loes Score≤ 9).
- Sixteen of 17 had resolution of gadolinium enhancement by month six. Re-emergence of diffuse contrast enhancement was seen in five patients at month 12. Of those five patients, the two with at least 18 months of follow up showed resolution of gadolinium enhancement at month 18.
- The safety profile of Lenti-D treatment appears consistent with myeloablative conditioning with one possibly drug-related serious adverse event (Grade 3 BK-mediated viral cystitis) and one possibly drug-related adverse event (Grade 1 tachycardia). Both resolved with standard measures.
- Integration site analyses have demonstrated polyclonal reconstitution in all subjects without evidence of clonal dominance to date.
“While the interim results from the Starbeam study are early, with only
three of the 17 patients having completed the study thus far, we are
pleased to see evidence of neurologic and radiographic stabilization of
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 a functioning copy 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 (HCT) 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, cerebral adrenoleukodystrophy (CALD) is a rare and fatal, X-linked, inherited, neurodegenerative disease that primarily affects young boys. CALD involves a progressive destruction of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms usually occur in early childhood and progress rapidly if untreated, leading to severe loss of neurological function and eventual death. In boys affected by CALD, learning and behavioral problems are often observed in mid-childhood between the ages of 3 and 15 years (median age 7). The worldwide incidence rate for ALD is approximately one in 21,000 male newborns; of those, 30-40 percent are affected by CALD, the cerebral form of the disease.
Currently, the only effective treatment option for patients with CALD is allogeneic HCT. Complications of allogeneic HCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo allogeneic HCT and do not have a matched sibling donor.
Investor Webcast Information
bluebird bio will host an investor conference call and webcast with
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™ BB305 product
candidate, currently in three clinical studies for the treatment of
transfusion-dependent ß-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
bluebird bio has operations in
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
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 quarterly report on Form 10-K, as well as discussions of
potential risks, uncertainties, and other important factors in our
subsequent filings with the
Source: bluebird bio, Inc.