Transfusion independence observed in patients with non-β0/β0 genotypes
Transfusion reduction between 33 percent and 100 percent observed in patients with β0/β0 genotype
Company to webcast investor event,
“The growing body of data for LentiGlobin continues to show substantial
clinical benefit in patients with beta-thalassemia major, with high
levels of HbAT87Q production across patients regardless of
Abstract #201: Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q-Globin Vector (LentiGlobin BB305 Drug Product)
The Northstar Study is an ongoing, open-label, single-dose,
international, multicenter Phase 1/2 study designed to evaluate the
safety and efficacy of LentiGlobin BB305 drug product for the treatment
of subjects with beta-thalassemia major. As of
- Median HbAT87Q production at six months follow up was 4.9 g/dL among patients of all genotypes (n=9), 4.9 g/dL among patients with non-β0/β0 genotypes (n=5) and 5.0 g/dL among patients with the β0/β0 genotype (n=4).
- All of the patients with non-β0/β0 genotypes with at least six months follow up (n=5) have achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 to 16.4 months of ongoing transfusion independence; total hemoglobin at last follow up for these patients ranged from 9.1 to 12.2 g/dL.
- Patients with the β0/β0 genotype (n=4) demonstrated a reduction in transfusion volume ranging from 33 percent to 100 percent.
- The safety profile was consistent with autologous transplantation. No Grade 3 or higher drug-product related adverse events have been observed, and there is no evidence of clonal dominance.
“The updated data from the Northstar Study continue to show acceptable patient safety with regard to myeloablation, drug product infusion risks, and genotoxicity at this early follow up,” said Dr. Walters. “All of the subjects treated in the study had a clinical benefit, which was most pronounced in the patients with non-β0/β0 genotypes, whose endogenous beta-globin expression combined with HbAT87Q expression was sufficient for transfusion independence. The patients with the β0/β0 genotype also had a benefit, with transfusion volume reduction ranging from 33 percent to 100 percent, though longer follow up is needed to understand the clinical significance of this data. The results of the Northstar Study to date suggest that it is safe, feasible and potentially efficacious to treat patients with beta-thalassemia major with LentiGlobin BB305.”
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, 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
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
Pure Communications, Inc.
Dan Budwick, 973-271-6085