Two patients with beta-thalassemia major with β0/βE genotype remain transfusion-independent for 23.4 and 20.1 months, respectively
Patient with severe sickle cell disease (SCD) in HGB-205 study remains free of transfusions and complications from SCD for nine months and is producing 49 percent anti-sickling hemoglobin
Early safety data presented from patients with severe SCD treated in HGB-206 study
Company to webcast investor event,
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The data from the HGB-205 study were highlighted today in an oral
presentation by Marina Cavazzana, M.D., Ph.D., lead investigator of the
HGB-205 study and professor of hematology at Paris Descartes
University, head of the department of
“The data from the HGB-205 and HGB-206 studies further demonstrate the
potential for gene therapy to make a meaningful and enduring difference
in the lives of patients with beta-thalassemia major or severe SCD,”
Abstract #202: Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q -Globin Vector (HGB-205 study)
HGB-205 is an ongoing, open-label, single-center Phase 1/2 study
designed to evaluate the safety and efficacy of LentiGlobin BB305 drug
product in the treatment of patients with beta-thalassemia major and
severe SCD. As of
- Subject 1201 with the β0/βE genotype of beta-thalassemia major has 23.4 months of transfusion independence with total hemoglobin of 10.8 g/dL, of which 7.9 g/dL was HbAT87Q. Subject 1202 with the β0/βE genotype of beta-thalassemia major has 20.1 months of transfusion independence with total hemoglobin of 13.1 g/dL, of which 10.3 g/dL was HbAT87Q.
- An additional two patients with beta-thalassemia major, Subjects 1203 and 1206, have been infused, though it is too early to draw any meaningful efficacy conclusions. At their most recent follow ups, three months and one month, respectively, these patients were producing measurable levels of HbAT87Q, which are steadily increasing in Subject 1203.
- At the 12-month post-drug infusion follow up for Subject 1204 with severe SCD, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) accounted for 49 percent of all hemoglobin production (47 percent HbAT87Q + 2 percent HbF) – well above the 30 percent threshold expected to potentially achieve a disease-modifying clinical effect. Prior to infusion, Subject 1204 required chronic blood transfusions; he was successfully weaned off of transfusions and has remained transfusion independent for more than nine months. Since infusion, this patient has had no hospitalizations or acute SCD-related events.
- No LentiGlobin-related adverse events have been observed; all of the adverse events observed are consistent with myeloablative conditioning.
- All five treated subjects successfully engrafted and insertional site analyses (ISAs) demonstrate highly polyclonal reconstitution without clonal dominance.
“These data are evidence of the durable responses we have seen in patients with beta-thalassemia major or severe SCD who have received a one-time treatment with LentiGlobin,” said Professor Cavazzana. “We are now seeing the benefit of gene therapy with LentiGlobin beyond one year in two patients with beta-thalassemia major. Clinical benefit continues to be realized in the patient with severe SCD after 12 months of follow up. We are encouraged by these results and the potential benefit treatment with LentiGlobin can have on patients living with these debilitating diseases.”
Abstract #3233: Initial Results from Study HGB-206: A Phase 1 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease (HGB-206 study)
HGB-206 is an ongoing, open-label Phase 1 study designed to evaluate the
safety and efficacy of LentiGlobin BB305 product candidate in the
treatment of subjects with severe SCD. Results, as of
- Drug product has been manufactured for four patients with severe SCD, and three patients have been infused with LentiGlobin BB305. Subjects 1301 and 1303 have three and 5.3 months of follow up post-infusion, respectively.
Drug product vector copy number (VCN) was 0.5/0.6 in Subject 1301, 1.3
in Subject 1303 and 0.6 in Subject 1306.
- VCN in peripheral blood leukocytes at three months follow up was 0.04 in Subject 1301 and 0.11 in Subject 1303.
Early data on Subjects 1301 and 1303 with at least three months of
follow up show a gradual increase in HbAT87Q levels
- At the three-month post-infusion follow up for Subject 1301, the proportion of anti-sickling hemoglobin accounted for 17 percent of all hemoglobin production (4 percent HbAT87Q + 13 percent HbF).
- At the six-month post-infusion follow up for Subject 1303, the proportion of anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12 percent HbAT87Q + 4 percent HbF).
- Longer follow up data and additional subjects are required to determine the extent of HbAT87Q production and clinical benefit of LentiGlobin BB305 in severe SCD.
- The safety profile in the infused patients is consistent with autologous transplantation and no drug product-related grade ≥3 adverse events have been reported.
Investor Webcast Information
bluebird bio will host an investor event that will be webcast live at
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
childhood cerebral adrenoleukodystrophy, and its LentiGlobin® BB305
product candidate, currently in three clinical studies for the treatment
of beta-thalassemia major 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 partnered with
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 potential efficacy and safety of the Company’s
LentiGlobin BB305 product candidate in subjects with beta thalassemia
major and severe sickle cell disease, including statements concerning
the production of HbAT87Q and the reduced or eliminated need for
transfusion support for the study subjects with beta thalassemia major,
statements concerning the Company’s future plans with respect to
LentiGlobin and its other product candidates. It should be noted that
the data announced for LentiGlobin are preliminary in nature and the
HGB-205 and HGB-206 studies of LentiGlobin are not completed. There is
limited data concerning long-term safety and efficacy following
treatment with LentiGlobin. These data may not continue for these
subjects or be repeated or observed in ongoing or future studies
involving our LentiGlobin product candidate, including the HGB-205
Study, the Northstar Study or the HGB-206 study in severe sickle cell
disease. It is possible that subjects for whom transfusion support has
been reduced or eliminated may receive transfusion support in the
future. 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 results from our clinical trials will
not continue or be repeated in our ongoing clinical trials, the risk
that previously conducted studies involving similar product candidates
will not be repeated or observed in ongoing or future studies involving
current product candidates, 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
our clinical studies, 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-Q, as well as discussions of potential risks, uncertainties, and
other important factors in our subsequent filings with the
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
Pure Communications, Inc.
Dan Budwick, 973-271-6085
Imagine Institute for Genetic Diseases (HGB-205 Study Only):
Béatrice Parinello-Froment, +33 (0)6 63 72 16 06
Pauline Rodrigue-Moriconi, +33 (0)6 77 23 71 19