Additional follow-up from the completed Phase
1/2 Northstar (HGB-204) study of LentiGlobin in adolescents and
adults with TDT
New data from ongoing Phase 3 Northstar-2
(HGB-207) study of LentiGlobin for TDT in patients who do not have
β0/β0 genotype and Phase 3 Northstar-3 (HGB-212) of patients with
β0/β0 genotype or an IVS-I-110 mutation
New data from ongoing Phase 1/2 HGB-206 study
of investigational LentiGlobin for patients with SCD
Company to hold conference call and
webcast, Friday, June 14 at 8:00 a.m. EDT
bluebird bio, Inc. (Nasdaq: BLUE) announced today that new data
from its investigational gene therapy programs for
transfusion-dependent β-thalassemia (TDT) and sickle cell disease
(SCD) will be presented during the 24th European Hematology
Association (EHA) Congress in Amsterdam, the Netherlands, June
13-16.
bluebird bio will present data from its clinical studies of
LentiGlobin™ gene therapy for TDT including updated results up to
54 months from the long-term follow-up period of the completed
Phase 1/2 Northstar (HGB-204) study. The company will also present
new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in
patients who do not have a β0/β0 genotype and from the ongoing
Phase 3 Northstar-3 (HGB-212) study in patients who have β0/β0
genotype or an IVS-I-110 mutation.
New data from the company’s Phase 1/2 HGB-206 study of
LentiGlobin gene therapy for SCD will include additional patients
treated in the study and updated data for those previously
reported.
Oral Presentations:
Transfusion-Dependent β-Thalassemia
Results from the Phase 3 Northstar-3 Study Evaluating
LentiGlobin Gene Therapy in Patients with Transfusion-Dependent
β-Thalassaemia and a β0 or IVS-I-110 Mutation at Both
Alleles of the HBB Gene (HGB-212)Presenting Author: Andreas
Kulozik, M.D., Ph.D., University Hospital Heidelberg, Heidelberg,
GermanyDate & Time: Friday, June 14, 2019, 11:30 – 11:45 a.m.
CEST (5:30 – 5:45 a.m. EDT)
Clinical Outcomes of LentiGlobin Gene Therapy for
Transfusion-Dependent β-Thalassaemia (TDT) Following Completion of
the Northstar (HGB-204) StudyPresenting Author: Mark Walters,
M.D., Benioff Children’s Hospital, Oakland, Calif., U.S.Date &
Time: Friday, June 14, 2019, 11:45 a.m. – 12:00 p.m. CEST (5:45 –
6:00 a.m. EDT)
Safety and Efficacy of LentiGlobin Gene Therapy in Patients
with Transfusion-Dependent β-Thalassaemia and
Non-β0/β0 Genotypes in the Phase 3
Northstar-2 Study (HGB-207)Presenting Author: Franco Locatelli,
M.D., Ph.D. University of Pavia, Pavia, Lombardy, ItalyDate &
Time: Sunday, June 16, 2019, 8:00 – 8:15 a.m. CEST (2:00 – 2:15
a.m. EDT)
Oral Presentation: Sickle Cell
Disease
Updated Results from the HGB-206 Study in Patients with
Severe Sickle Cell Disease Treated Under a Revised Protocol with
LentiGlobin Gene Therapy Using Plerixafor-Mobilised Haematopoietic
Stem Cells (HGB-206)Presenting Author: Julie Kanter, M.D.,
Division of Hematology and Oncology, University of Alabama at
Birmingham, Birmingham, Ala., U.S.Date & Time: Sunday, June 16,
2019, 8:15 – 8:30 a.m. CEST (2:15 – 2:30 a.m. EDT)
Abstracts outlining bluebird bio’s accepted data at EHA have
been made available on the EHA conference website.
Investor Event & Webcast Information
bluebird bio will host a conference call and live webcast at
8:00 a.m. EDT Friday, June 14, 2019. To access the webcast, please
visit the "Events & Presentations" page within the Investors
& Media section of the bluebird bio website
at http://investor.bluebirdbio.com/. A replay of the webcast
will be available on the bluebird bio website for 90 days following
the call.
About LentiGlobin for Transfusion-Dependent
β-Thalassemia
In March 2019, the Committee for Medicinal Products for
Human Use (CHMP) of the European Medicines Agency (EMA)
adopted a positive opinion recommending conditional marketing
authorization for LentiGlobin gene therapy for TDT, which if
approved will be commercialized as ZYNTEGLO™ (autologous CD34+
cells encoding βA-T87Q-globin gene) gene therapy for patients 12
years and older with transfusion-dependent β-thalassemia (TDT) who
do not have a β0/β0 genotype, for whom hematopoietic stem cell
(HSC) transplantation is appropriate but a human leukocyte antigen
(HLA)-matched related HSC donor is not available. ZYNTEGLO adds
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 a patient has the βA-T87Q-globin gene they
have the potential to produce HbAT87Q, which is gene therapy-
derived-hemoglobin, at levels that significantly reduce or
eliminate the need for transfusions.
Non-serious adverse events (AEs) observed during clinical trials
that were attributed to ZYNTEGLO were hot flush, dyspnoea,
abdominal pain, pain in extremities and non-cardiac chest pain. One
serious adverse event (SAE) of thrombocytopenia was considered
possibly related to ZYNTEGLO.
Additional AEs observed in clinical studies were consistent with
the known side effects of HSC collection and bone marrow ablation
with busulfan including SAEs of veno-occlusive disease.
ZYNTEGLO continues to be evaluated in the ongoing Phase 3
Northstar-2 and Northstar-3 studies and the long-term follow-up
study LTF-303. If approved, ZYNTEGLO will be the first gene therapy
to treat TDT.
About LentiGlobin for Sickle Cell Disease
LentiGlobin for sickle cell disease is an investigational gene
therapy being studied as a potential treatment to address the
underlying genetic cause of SCD. bluebird bio’s clinical
development program for LentiGlobin for SCD includes the ongoing
Phase 1/2 HGB-206 study.
bluebird bio is conducting a long-term safety and efficacy
follow-up study (LTF-303) for people who have participated in
bluebird bio-sponsored clinical studies of LentiGlobin for TDT and
LentiGlobin for SCD. For more information
visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/.
The European Medicines Association (EMA) granted Priority
Medicines (PRIME) eligibility and Orphan Medicinal Product
designation to LentiGlobin for the treatment of TDT. LentiGlobin
for TDT was also part of the EMA’s Adaptive Pathways pilot program,
which is part of the EMA’s effort to improve timely access for
patients to new medicines.
The U.S. Food and Drug Administration granted Orphan Drug
status and Breakthrough Therapy designation to LentiGlobin for TDT;
as well as Orphan Drug status and Regenerative Medicine Advanced
Therapy designation for LentiGlobin for the treatment of SCD.
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.
ZYNTEGLO and LentiGlobin are trademarks of bluebird bio.
The full common name for ZYNTEGLO: A genetically modified
autologous CD34+ cell enriched population that contains
hematopoietic stem cells transduced with lentiviral vector encoding
the βA-T87Q-globin gene.
Forward-Looking Statements
This release contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995.
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 our MAA submitted for
LentiGlobin for TDT may not be approved by the European
Commission when expected, or at all; the risk that the
efficacy and safety results from our prior and ongoing clinical
trials of LentiGlobin for TDT will not continue or be repeated in
our ongoing or planned clinical trials of LentiGlobin for TDT; the
risk that the current or planned clinical trials of LentiGlobin for
TDT will be insufficient to support regulatory submissions or
marketing approval in the US and EU; the risk that the production
of HbA-T87Q may not be sustained over extended periods of time; and
the risk that we may not secure adequate pricing or reimbursement
to support continued development or commercialization of
LentiGlobin for TDT following regulatory approval. 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/20190516005626/en/
bluebird bioInvestors:Elizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.comorMedia:Catherine Falcetti,
339-499-9436cfalcetti@bluebirdbio.com
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