Presenting new and updated data from HGB-205 study in beta-thalassemia major and severe sickle cell disease, including first patient with sickle cell disease ever treated with gene therapy
Company to host investor call on
“The early data included in our abstract provide further validation for
our approach and important insights into the safety and mechanism of
action of LentiGlobin in both beta-thalassemia and sickle cell disease,”
said
Abstract Highlights (Data as of
- Beta-thalassemia: Beta-thalassemia major subjects (1201 and 1202) remained transfusion independent at 14 months and 11 months, respectively
-
Sickle Cell Disease: This subject (1204) entered the trial
receiving chronic transfusions and began the process of being weaned
from transfusions after day 37, receiving the last transfusion on day
88
-
Increasing production of HbAT87Q; the first-ever SCD
patient treated with gene therapy (subject 1204) had a HbAT87Q
level of 24% at 4.5 months follow up, compared to an HbAT87Q
level of 9.6% at three months post-transplant
- Note that this subject did not engraft until after month one, so their level of HbAT87Q production at months three and 4.5 are actually months two and 3.5, after engraftment
- At 4.5 months follow up, total anti-sickling hemoglobin (HbAT87Q + HbF) was 31.6%
- Subject 1204 has not had any hospitalizations for SCD-related complications post-transplant
-
Increasing production of HbAT87Q; the first-ever SCD
patient treated with gene therapy (subject 1204) had a HbAT87Q
level of 24% at 4.5 months follow up, compared to an HbAT87Q
level of 9.6% at three months post-transplant
- Safety: No subject has experienced a drug product-related adverse event, and integration site analyses demonstrate highly polyclonal reconstitution without clonal dominance
Based on historical clinical observations in patients with SCD, bluebird bio believes that individuals who achieve ≥ 30 percent of anti-sickling hemoglobin (HbAT87Q + HbF) have the potential to reduce or eliminate the serious and life-threatening events associated with SCD.
The abstract is now available online on the EHA conference website. Information contained in the abstract reflects data available as of February 2015. Details of bluebird bio’s presentation are as follows:
Title: Outcomes of Gene Therapy for B-Thalassemia Major and
Severe Sickle Cell Disease via Transplantation of Autologous
Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta
Globin Vector
Abstract Code: S466
Session Name:
Gene therapy, cellular immunotherapy and vaccination
Date:
Oral Presentation Time: 11:30 -
Location: Reed Messe Vienna, Room Stolz 2
Investor Conference Call and Webcast Information
bluebird
bio will host a conference call and webcast on June 15, 2015 at
About bluebird bio, Inc.
With its lentiviral-based gene
therapy and gene editing capabilities, bluebird bio has built an
integrated product platform with broad potential application to severe
genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical
programs include Lenti-D™, currently in a Phase 2/3 study,
called the Starbeam Study, for the treatment of childhood cerebral
adrenoleukodystrophy, and LentiGlobin®, currently in three
clinical studies: a global Phase 1/2 study, called the Northstar Study,
for the treatment of beta-thalassemia major; a single-center Phase 1/2
study in France (HGB-205) for the treatment of beta-thalassemia major or
severe sickle cell disease; and a separate U.S. Phase 1 study for the
treatment of sickle cell disease (HGB-206). bluebird bio also has a
preclinical CAR T immuno-oncology program in collaboration with Celgene
Corporation, as well as discovery research programs utilizing
megaTALs/homing endonuclease gene editing technologies.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Forward-Looking Statements
This release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements regarding
the advancement of, and anticipated milestones related to the Company’s
product candidates, including LentiGlobin. 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 of cessation or
delay of any of the ongoing or planned clinical studies and/or our
development of our product candidates, the risk of a delay in the
enrollment of patients in the Company’s clinical studies, the risk that
the results of previously conducted studies involving similar product
candidates will not be repeated or observed in ongoing or future studies
involving current product candidates, the risk that our collaboration
with
View source version on businesswire.com: http://www.businesswire.com/news/home/20150521005591/en/
Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc.
Jim DeTore,
339-499-9355
Chief Financial Officer
or
Media:
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Dan Budwick, 973-271-6085