Updated data from studies of beti-cel in 63
patients with transfusion-dependent beta-thalassemia showed
sustained transfusion independence and improvements in iron
management through up to 9 years of follow-up (n=1)
All four parent studies are complete, and all
patients have transitioned to a long-term follow-up study
(LTF-303)
beti-cel is U.S. Food and Drug Administration
(FDA) approved and marketed in the U.S. as ZYNTEGLO
Updated data from bluebird bio inc.’s (NASDAQ: BLUE) gene
therapy program in transfusion-dependent (TDT) beta-thalassemia
were presented today at the 65th American Society of Hematology
(ASH) Annual Meeting & Exposition. Updated follow-up data
showed sustained treatment effect, reduced iron management burden
and improved quality of life measures in patients with
beta-thalassemia who require regular red blood cell transfusions
following treatment with betibeglogene autotemcel (beti-cel) (FDA
approved and marketed in the U.S. as ZYNTEGLO™).
“Long-term results presented at ASH 2023 showed durable
transfusion independence and a continued positive safety profile in
patients with beta-thalassemia treated with our beti-cel gene
therapy through up to nine years of follow-up,” said Richard
Colvin, M.D., Ph.D., chief medical officer, bluebird bio. “These
data represent the longest follow-up with a gene therapy for
patients with beta-thalassemia requiring regular transfusions and
continue to show that beti-cel is a potentially curative therapy
across ages and genotypes, through the achievement of durable
transfusion independence and normal or near-normal hemoglobin
levels.”
As of January 30, 2023, 63 patients had received beti-cel across
four clinical studies with a median follow-up of 5 years (60.1
months; range: 23.8-109.5). These include two Phase 3 studies
(N=41) that led to the FDA approval of ZYNTEGLO in August 2022 as
the first and only gene therapy for patients with beta-thalassemia
who require regular red blood cell transfusions.
Sustained Efficacy and Safety Results, and Improved Quality
of Life Measures in Adult and Pediatric Patients With
Transfusion-Dependent β-Thalassemia Up to 9 Years Post Treatment
With Betibeglogene Autotemcel (beti-cel) (poster #1102)
Among patients in the Phase 3 studies, 90.2% (37/41) achieved
transfusion independence (TI). As of the data cutoff date, TI was
maintained through last follow-up (up to 6 years) across ages and
genotypes. Among patients in the Phase 1/2 studies, 68.2% (15/22)
achieved TI, with 14 patients sustaining TI through the last
follow-up (up to 9 years). One patient, with human immunodeficiency
virus complicated by gastrointestinal infection and bleeding, no
longer met protocol-defined TI as a result of a hemoglobin level
<9 g/dL at year 6. The patient continued to benefit from
beti-cell therapy and was not receiving chronic red blood cells
transfusions as of the last follow-up.
Among 12 adult patients and 22 pediatric patients in the Phase
1/2 and Phase 3 studies, health-related quality of life (HRQOL) was
assessed at baseline and every 6 months. Clinically meaningful
improvements in quality of life assessments, including various
quality of life assessments for mental, physical and psychosocial
health, were demonstrated in both adult and pediatric patients up
to 36 months.
As of the cutoff date, the safety results following beti-cel
treatment largely reflected the known side effects of hematopoietic
stem cell collection and the busulfan conditioning regimen.
Overall, 19% of patients experienced ≥1 beti-cel-related AE. The
most common beti-cel-related AEs (occurring in ≥3 patients) were
abdominal pain (7.9% of patients) and thrombocytopenia (4.8% of
patients). Five patients experienced serious veno-occlusive liver
disease; all 5 received defibrotide and recovered. No malignancies,
insertional oncogenesis, or vector-derived replication-competent
lentivirus were reported.
Improvement in Iron Burden in Patients With
Transfusion-Dependent β-Thalassemia (TDT) Treated With
Betibeglogene Autotemcel (beti-cel) Gene Therapy: Up to 9 Years of
Follow-Up (poster #2480)
Additionally, iron management outcomes were presented from
patients with transfusion-dependent beta-thalassemia who completed
either a Phase 1/2 or Phase 3 beti-cel parent study and
subsequently enrolled in the long-term follow-up study and were
followed for up to 9 years. Across all studies, 37/51 patients
restarted chelation, and 12 received phlebotomy post-infusion;
however, 69% (35/51) were able to stop chelation therapy,
demonstrating restoration of iron levels over time and reduced iron
management burden in those patients.
About ZYNTEGLO™ (betibeglogene autotemcel) or
beti-cel
ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy.
It is approved for the treatment of beta-thalassemia in adult and
pediatric patients who require regular red blood cell transfusions
and was launched commercially more than a year ago. ZYNTEGLO works
by adding functional copies of a modified form of the beta-globin
gene (βA-T87Q-globin gene) into a beta-thalassemia patient’s own
hematopoietic (blood) stem cells to enable the production of a
modified functional adult hemoglobin (HbAT87Q). Once a patient has
the βA-T87Q-globin gene, they have the potential to increase
ZYNTEGLO-derived adult hemoglobin (HbAT87Q) and total hemoglobin to
normal or near normal levels that can eliminate the need for
regular red blood cell (RBC) transfusions.
Indication
ZYNTEGLO is indicated for the treatment of adult and pediatric
patients with beta-thalassemia who require regular red blood cell
(RBC) transfusions.
Important Safety Information
Delayed Platelet Engraftment
Delayed platelet engraftment has been observed with ZYNTEGLO
treatment. Bleeding risk is increased prior to platelet engraftment
and may continue after engraftment in patients with prolonged
thrombocytopenia; 15% of patients had ≥ Grade 3 decreased platelets
on or after Day 100.
Patients should be made aware of the risk of bleeding until
platelet recovery has been achieved. Monitor patients for
thrombocytopenia and bleeding according to standard guidelines.
Conduct frequent platelet counts until platelet engraftment and
platelet recovery are achieved. Perform blood cell count
determination and other appropriate testing whenever clinical
symptoms suggestive of bleeding arise.
Risk of Neutrophil Engraftment Failure
There is a potential risk of neutrophil engraftment failure
after treatment with ZYNTEGLO. Neutrophil engraftment failure is
defined as failure to achieve three consecutive absolute neutrophil
counts (ANC) ≥ 500 cells/microliter obtained on different days by
Day 43 after infusion of ZYNTEGLO. Monitor neutrophil counts until
engraftment has been achieved. If neutrophil engraftment failure
occurs in a patient treated with ZYNTEGLO, provide rescue treatment
with the back-up collection of CD34+ cells.
Risk of Insertional Oncogenesis
There is a potential risk of LVV mediated insertional
oncogenesis after treatment with ZYNTEGLO.
Patients treated with ZYNTEGLO may develop hematologic
malignancies and should be monitored lifelong. Monitor for
hematologic malignancies with a complete blood count (with
differential) at Month 6 and Month 12 and then at least annually
for at least 15 years after treatment with ZYNTEGLO, and
integration site analysis at Months 6, 12, and as warranted.
In the event that a malignancy occurs, contact bluebird bio at 1
833-999-6378 for reporting and to obtain instructions on collection
of samples for testing.
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of ZYNTEGLO. The
dimethyl sulfoxide (DMSO) in ZYNTEGLO may cause hypersensitivity
reactions, including anaphylaxis.
Anti-retroviral and Hydroxyurea Use
Patients should not take prophylactic HIV anti-retroviral
medications or hydroxyurea for at least one month prior to
mobilization, or for the expected duration for elimination of the
medications, and until all cycles of apheresis are completed. If a
patient requires anti-retrovirals for HIV prophylaxis, then confirm
a negative test for HIV before beginning mobilization and apheresis
of CD34+ cells.
Interference with Serology Testing
Patients who have received ZYNTEGLO are likely to test positive
by polymerase chain reaction (PCR) assays for HIV due to integrated
BB305 LVV proviral DNA, resulting in a false-positive test for HIV.
Therefore, patients who have received ZYNTEGLO should not be
screened for HIV infection using a PCR-based assay.
Adverse Reactions
The most common non-laboratory adverse reactions (≥20%) were
mucositis, febrile neutropenia, vomiting, pyrexia, alopecia,
epistaxis, abdominal pain, musculoskeletal pain, cough, headache,
diarrhea, rash, constipation, nausea, decreased appetite,
pigmentation disorder, and pruritus. The most common Grade 3 or 4
laboratory abnormalities (>50%) include neutropenia,
thrombocytopenia, leukopenia, anemia, and lymphopenia.
Drug Interactions
Drug-drug interactions between iron chelators and the
myeloablative conditioning agent must be considered. Iron chelators
should be discontinued at least 7 days prior to initiation of
conditioning. The prescribing information for the iron chelator(s)
and the myeloablative conditioning agent should be consulted for
the recommendations regarding co-administration with CYP3A
substrates.
Some iron chelators are myelosuppressive. After ZYNTEGLO
infusion, avoid use of these iron chelators for 6 months. If iron
chelation is needed, consider administration of
non-myelosuppressive iron chelators. Phlebotomy can be used in lieu
of iron chelation, when appropriate.
Pregnancy/Lactation
Advise patients of the risks associated with conditioning
agents, including on pregnancy and fertility.
ZYNTEGLO should not be administered to women who are pregnant,
and pregnancy after ZYNTEGLO infusion should be discussed with the
treating physician.
ZYNTEGLO is not recommended for women who are breastfeeding, and
breastfeeding after ZYNTEGLO infusion should be discussed with the
treating physician.
Females and Males of Reproductive Potential
A negative serum pregnancy test must be confirmed prior to the
start of mobilization and re-confirmed prior to conditioning
procedures and before ZYNTEGLO administration.
Women of childbearing potential and men capable of fathering a
child should use an effective method of contraception (intra
uterine device or combination of hormonal and barrier
contraception) from start of mobilization through at least 6 months
after administration of ZYNTEGLO.
Advise patients of the option to cryopreserve semen or ova
before treatment if appropriate.
Please see full Prescribing Information for ZYNTEGLO.
About bluebird bio
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days.
Founded in 2010, bluebird has been setting the standard for gene
therapy for more than a decade—first as a scientific pioneer and
now as a commercial leader. bluebird has an unrivaled track record
in bringing the promise of gene therapy out of clinical studies and
into the real-world setting, having secured FDA approvals for three
therapies in under two years. Today, we are proving and scaling the
commercial model for gene therapy and delivering innovative
solutions for access to patients, providers, and payers.
With a dedicated focus on severe genetic diseases, bluebird has
the largest and deepest ex-vivo gene therapy data set in the field,
with industry-leading programs for sickle cell disease,
β-thalassemia and cerebral adrenoleukodystrophy. 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.
bluebird continues to forge new paths as a standalone commercial
gene therapy company, 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.
bluebird bio Cautionary Statement Regarding Forward-Looking
Statements
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 as
statements regarding beti-cel as a potentially curative therapy
across ages and genotypes. Such forward-looking statements are
based on historical performance and current expectations and
projections about bluebird’s 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 bluebird’s control and could cause bluebird’s future
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 for the year ended December 31, 2022, as
updated by its subsequent Quarterly Reports on Form 10-Q, Current
Reports on Form 8-K and other filings with the Securities and
Exchange Commission. These risks and uncertainties include, but are
not limited to: delays and challenges in bluebird’s
commercialization and manufacturing of its products; the internal
and external costs required for bluebird’s ongoing and planned
activities, and the resulting impact on expense and use of cash,
has been, and may in the future be, higher than expected which has
caused bluebird, and may in the future cause bluebird to use cash
more quickly than it expects or change or curtail some of its plans
or both; substantial doubt exists regarding bluebird’s ability to
continue as a going concern; bluebird’s expectations as to
expenses, cash usage and cash needs may prove not to be correct for
other reasons such as changes in plans or actual events being
different than its assumptions; the risk that the efficacy and
safety results from bluebird’s prior and ongoing clinical trials
will not continue or be seen in the commercial context; and the
risk of insertional oncogenic or other safety events associated
with lentiviral vector, drug product, or myeloablation. 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/20231209852325/en/
Investors: Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com
Media: Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
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