Oral presentation details efficacy, safety and
health-related quality of life data in adult and pediatric patients
with sickle cell disease treated with lovo-cel in HGB-206 Group C
and HGB-210 (n=47) through 5 years of follow-up (n=4)
Updated long-term efficacy, safety, quality of
life data and iron management outcomes from adult and pediatric
patients with transfusion-dependent beta-thalassemia (n=63) treated
with beti-cel through 9 years of follow-up (n=1) will be delivered
in two poster presentations
bluebird bio, Inc. (Nasdaq: BLUE) today announced that new
long-term efficacy, safety and health-related quality of life
(HRQoL) follow-up data from its lentiviral vector (LVV) gene
therapy programs in patients with sickle cell disease who have a
history of vaso-occlusive events and patients with beta-thalassemia
who require regular red blood cell transfusions will be presented
at the 65th American Society of Hematology (ASH) Annual Meeting and
Exposition. The meeting will take place December 9-12, 2023 at the
San Diego Convention Center and online.
“The bluebird bio data to be presented at ASH 2023 underscore
the maturity of LVV gene therapies for hemoglobinopathies, with up
to 9 years of follow-up data in transfusion-dependent
beta-thalassemia and 5 years of follow-up in sickle cell disease,”
said Richard Colvin, M.D., Ph.D., chief medical officer, bluebird
bio. “The robustness of this dataset is unparalleled, giving us
continued confidence that the transformational impact these
therapies may have for patients is sustained over time. We
especially look forward to the first ever presentation of data from
our long-term follow-up study of lovo-cel, the most deeply studied
gene therapy in development for sickle cell disease.”
bluebird bio will present updated follow-up data for
lovotibeglogene autotemcel (lovo-cel) in patients from the HBG-206
Group C and HGB-210 studies with sickle cell disease followed for
up to 60 months (median of 35.5 months), demonstrating sustained
hemoglobin AT87Q production and near-complete resolution of
vaso-occlusive events (VOEs) and severe VOEs, as well as sustained
improvements in HRQoL. lovo-cel treatment regimen largely reflects
known side effects of hematopoietic stem cell collection and
busulfan conditioning regimen and underlying sickle cell
disease.
Updated analyses of efficacy, safety, and HRQoL data from Phase
1/2 and Phase 3 studies of betibeglogene autotemcel (beti-cel) in
patients with transfusion-dependent beta-thalassemia will also be
presented, demonstrating sustained transfusion independence with up
to nine years of follow-up and improvements in quality of life
reported at Month 36. Updated iron management outcomes
demonstrating sustained improvements in iron burden, with the
majority of patients able to stop iron chelation therapy, will also
be presented. Safety of beti-cel treatment largely reflects the
known side effects of hematopoietic stem cell collection and
busulfan conditioning regimen.
Sickle Cell Disease Data
Oral Presentation [#1051]: Efficacy, Safety, and Health-Related
Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD)
Who Have Received lovotibeglogene autotemcel (lovo-cel) Gene
Therapy: Up to 60 Months of Follow-up Presenting Author:
Julie Kanter, M.D., director of the UAB Adult Sickle Cell clinic,
associate professor in the Division of Hematology and Oncology, and
co-director of the UAB Comprehensive Sickle Cell Disease Center at
the University of Alabama in Birmingham Date/Time: Monday,
December 11, 2023, 4:30 p.m. PT
Beta-Thalassemia Data
Poster Presentation [#1102]: Sustained, Efficacy, Safety,
and Improved Quality of Life in Adult and Pediatric Patients with
Transfusion-Dependent β-Thalassemia up to 9 Years Post
Treatment with betibeglogene autotemcel (beti-cel)
Presenting Author: Alexis A. Thompson, M.D., M.P.H.,
professor of pediatrics (hematology), Perelman School of Medicine,
University of Pennsylvania, Philadelphia, and chief, Division of
Hematology, Children’s Hospital of Philadelphia Date/Time:
Saturday, December 9, 2023, 5:30 p.m. PT
Poster Presentation [#2480]: 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 Presenting Author: Janet L.
Kwiatkowski, M.D., MSCE, professor of pediatrics (hematology),
Department of Pediatrics, Perelman School of Medicine, University
of Pennsylvania, Philadelphia, and director, Thalassemia Center,
Children's Hospital of Philadelphia Date/Time: Sunday,
December 10, 2023, 6:00 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH 2023 are
available on the ASH conference website.
The U.S. Food and Drug Administration previously accepted the
lovo-cel Biologics Licensing Application (BLA) for Priority Review
and set a Prescription Drug User Fee Act (PDUFA) goal date of
December 20, 2023. beti-cel was approved by the FDA in August 2022
and is commercially available in the United States as ZYNTEGLO.
About sickle cell disease (SCD) Sickle cell disease (SCD)
is a complex and progressive genetic disease associated with
debilitating and unpredictable pain crises, anemia, irreversible
damage to vital organs, and early death. In SCD, high
concentrations of sickle hemoglobin (HbS) in red blood cells (RBCs)
cause RBCs to become sickled, sticky, and rigid with a shorter life
span, which manifests acutely as hemolytic anemia, vasculopathy,
and vaso-occlusion. Pain onset can be sudden and unpredictable,
often requiring hospitalization. Fifty to sixty percent of adults
with SCD have end organ damage, with 24 percent experiencing damage
in multiple organs, and one in four patients have a stroke by the
age of 45. The impact of SCD is pervasive and affects every aspect
of life for patients and their families and caregivers – from
missed work and school, decreased quality of life and mental
health, and ability to complete daily tasks. In the U.S., there are
approximately 100,000 people living with SCD, and the median age of
death is 45 years of age.
While SCD was the first disease to have a genetic cause
identified, treatment advances have lagged – since that discovery
in 1949,i only four therapies have been approved,ii none of which
address the underlying genetic cause of disease.
About lovotibeglogene autotemcel (lovo-cel)
lovotibeglogene autotemcel (lovo-cel) gene therapy is an
investigational one-time treatment being studied for sickle cell
disease (SCD), that is designed to add 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). Once
patients have the βA-T87Q-globin gene, their red blood cells (RBCs)
can produce anti-sickling hemoglobin (HbAT87Q) that decreases the
proportion of HbS, with the goal of reducing sickled RBCs,
hemolysis, and other complications. bluebird bio’s clinical
development program for lovo-cel includes the completed Phase 1/2
HGB-205 and ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.
bluebird bio is also conducting a long-term safety and efficacy
follow-up study (LTF-307) for people who have been treated with
lovo-cel in bluebird bio-sponsored clinical studies.
In the BLA submission, as of August 2022, the majority of
adverse events in treated patients were attributed to underlying
sickle cell disease or conditioning with busulfan. Nonserious
adverse events related to lovo-cel included infusion reactions (hot
flush and decreased blood pressure) in two patients (2% each).
Serious adverse events related to lovo-cel included anemia in two
patients (4%) with concurrent alpha-thalassemia trait. Three cases
of hematologic malignancy have been reported, including one case of
myelodysplastic syndrome that remains under investigation. No cases
have been associated with insertional oncogenesis. Three of 50
patients (6%) died, one due to sudden cardiac death and two due to
aforementioned hematologic malignancy.
The U.S. Food and Drug Administration accepted the lovo-cel
Biologics Licensing Application (BLA) for Priority Review and set a
Prescription Drug User Fee Act (PDUFA) goal date of December 20,
2023. The FDA previously granted lovo-cel orphan drug designation,
fast track designation, regenerative medicine advanced therapy
(RMAT) designation, and rare pediatric disease designation.
About ZYNTEGLO® (betibeglogene autotemcel) or beti-cel
ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy
approved for the treatment of beta-thalassemia in adult and
pediatric patients who require regular red blood cell transfusions.
ZYNTEGLO works by adding functional copies of a modified form of
the beta-globin gene (βA-T87Q-globin gene) into a 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, 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 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
therapeutic potential of bluebird bio’s therapies, including the
potential for its therapies to have a sustained transformational
impact on patients, and bluebird bio’s ability to pursue curative
gene therapies to give patients and their families more bluebird
days. Such forward-looking statements are based on historical
performance and current expectations and projections about our
future financial results, 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 for the year ended December 31, 2022, 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, which may
impact our ability to meet our expected timelines and increase our
costs; 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
of insertional oncogenic or other reportable events associated with
lentiviral vector, drug product, or myeloablation; and the risk
that any one or more of our products or product candidates 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.
____________________ i Pauling L, Itano HA, Singer SJ, Wells IC.
Sickle cell anemia, a molecular disease. Science.
1949;110(2865):543-548. doi:10.1126/science.110.2865.543. ii Rai P,
Ataga KI. Drug therapies for the management of sickle cell disease.
F1000Res. 2020; 9:F1000 Faculty Rev-592. doi:
10.12688/f1000research.22433.1. PMID: 32765834; PMCID:
PMC7388199.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231102164140/en/
Investors: Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com
Media: Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
bluebird bio (NASDAQ:BLUE)
Historical Stock Chart
From Apr 2024 to May 2024
bluebird bio (NASDAQ:BLUE)
Historical Stock Chart
From May 2023 to May 2024