– All patients (n=4) in Group C with ≥ 3 months follow-up consistently producing ≥ 30% anti-sickling HbAT87Q –
– First Group C patient generating a normal total hemoglobin of 14.2 g/dL with over 60% anti-sickling HbAT87Q at 6 months –
– Company to hold conference call and webcast today,
“The consistent production of increased amounts of anti-sickling HbAT87Q in
the Group C patients reflects the substantial positive impact of the
changes introduced with the amended HGB-206 study protocol and refined
manufacturing process. All four Group C patients with greater than or
equal to three months follow-up are making over 30 percent
anti-sickling HbAT87Q. The first patient treated, now
with six months of follow-up, is producing over 60 percent anti-sickling
HbAT87Q with a normal total hemoglobin level of 14.2
g/dL,” said
SCD is a genetic disease that causes the protein in red blood cells, called hemoglobin, to be misshapen. As a result of this abnormal hemoglobin, many affected individuals live with severe anemia and vaso-occlusive events which include severe, recurrent pain crises that lead to organ damage and shortened life span.
“The early data from Group C patients are very exciting and provide increasing confidence that LentiGlobin has the potential to deliver transformative benefit to patients. The longer-term data from patients treated earlier in the study show that levels of anti-sickling HbAT87Q in patients with SCD treated with LentiGlobin remain stable for at least two years,” said Dr. Kanter, a lead investigator of the HGB-206 study. “Treatment options that can address the underlying cause of sickle cell disease are limited and LentiGlobin gene therapy has the potential to prevent or substantially reduce damaging symptoms associated with this debilitating disease.”
Recent
Presenter:
Date & Time:
Location:
HGB-206 is an ongoing, open-label study designed to evaluate the safety
and efficacy of LentiGlobin gene therapy for the treatment of adults
with severe SCD. Patients in this study are divided into three cohorts:
A, B and C. Patients in Group A were treated under the original study
protocol. Patients in Group B were treated under an amended study
protocol that included a refined Drug Product (DP) manufacturing process
intended to increase DP vector copy number (VCN) as well as changes to
improve engraftment of gene-modified stem cells. Patients in both Group
A and B had DP made from stem cells collected using bone marrow harvest.
Patients in Group C were also treated under the amended study protocol,
but received LentiGlobin gene therapy made from stem cells collected
from peripheral blood after mobilization with plerixafor rather than via
bone marrow harvest. Results, as of
- Group C: 6 patients treated under the amended study protocol
and with DP manufactured using the refined process, median (range)
follow-up 3 (1.2 – 6.0) months:
- 4 of 6 patients had ≥ 3 months follow up, and were producing 3 – 6 g/dL of HbAT87Q by 3 months
- 1 patient was producing 8.8 g/dL of HbAT87Q and a total hemoglobin level of 14.2 g/dL at 6 months
- Median transduced CD34+ cells: 81%
- Median DP cell dose: 7.1 x 106 CD34+ cells
- Median DP VCN (copies per diploid genome): 4
- Overall safety profile remains generally consistent with myeloablative conditioning
- Continued feasibility of plerixafor mobilization and apheresis observed
- Group B: 2 patients treated under the amended study protocol
and with DP manufactured using stem cells from bone marrow harvest
with ≥ 9 months follow up:
- Patient 1312, who received LentiGlobin manufactured entirely using a refined manufacturing process, was producing 7.2 g/dL HbAT87Q and 12.8 g/dL of total hemoglobin (56% HbAT87Q) at 9 months of follow-up
- Patient 1313, who received LentiGlobin manufactured using a combination of the original and the refined manufacturing processes, was producing 3.2 g/dL HbAT87Q and 11.0 g/dL of total hemoglobin (29% HbAT87Q) at 15 months of follow-up
- Group A: Long-term data on 7 patients in the initial study
cohort with ≥ 2 years follow-up:
- Steady levels of LentiGlobin vector and HbAT87Q were maintained through 2 years (median follow-up: 24.2 months; range: 22.8 – 32.9)
- Median transduced CD34+ cells: 25%
- Media DP cell dose: 2.1 x 106 CD34+ cells
- Median DP VCN: 0.6
- Median (range) total hemoglobin at last study visit was 9.1 (7.1 – 11.4) g/dL
Conference Call & Webcast Information
bluebird bio will host a conference call and live webcast at
About SCD
Sickle cell disease (SCD) is a serious, progressively debilitating, and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis. As a result of this abnormal hemoglobin, many affected individuals live with severe anemia and vaso-occlusive events which include severe, recurrent pain crises that lead to organ damage and shortened life span.
Where adequate medical care is available, common treatments for patients with SCD largely revolve around prevention of infection, and management and prevention of acute sickling episodes. Chronic management includes a limited number of pharmaceutical treatment options and, in certain cases, chronic transfusions. Allogeneic hematopoietic stem cell transplant (HSCT) is currently the only available option with the potential to correct the genetic deficiency in SCD. However, its use is limited to certain pediatric patients with severe disease who have an unaffected matched sibling donor. Complications of allogeneic HSCT include a risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.
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 Lenti-D™ for the treatment of cerebral adrenoleukodystrophy, and LentiGlobin™ for the treatment of transfusion-dependent β-thalassemia, also known as β-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 programs, bb2121 and bb21217, are anti-BCMA CAR T programs partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Washington, Durham, North Carolina and Zug,
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
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 Company's research, development, manufacturing
and regulatory approval plans for its LentiGlobin product candidate to
treat severe sickle cell disease. 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 risks that the
preliminary positive efficacy and safety results from our prior and
ongoing clinical trials of LentiGlobin will not continue or be repeated
in our ongoing, planned or expanded clinical trials of LentiGlobin, the
risks that the changes we have made in the LentiGlobin manufacturing
process or the HGB-206 clinical trial protocol will not result in
improved patient outcomes, risks that the current or planned clinical
trials of LentiGlobin will be insufficient to support regulatory
submissions or marketing approval in the US and EU, 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, approved or 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
Form 10-Q, as well as discussions of potential risks, uncertainties, and
other important factors in our subsequent filings with the
View source version on businesswire.com: https://www.businesswire.com/news/home/20180615005159/en/
Source: bluebird bio, Inc.
bluebird bio, Inc.
Investors:
Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Media:
Stephanie
Fagan, 201-572-9581
sfagan@bluebirdbio.com