bluebird bio (BLUE) Opens Phase 3 Study of LentiGlobin in Patients with Transfusion-Dependent Beta-Thalassemia

September 8, 2016 8:17 AM EDT

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bluebird bio, Inc. (Nasdaq: BLUE) today announced the opening of HGB-207, a Phase 3, global, multi-center study in patients with transfusion-dependent beta-thalassemia with non-β0/β0 genotypes. This study will incorporate the addition of bluebird bio’s transduction enhancers to the hematopoietic stem cell (HSC) manufacturing process, and will be conducted under the same Investigational New Drug application (IND) as previous studies of LentiGlobin in beta-thalassemia.

“The opening of bluebird’s first Phase 3 study for LentiGlobin, in which we are using an improved manufacturing process that increases transduction efficiency, is an exciting step forward,” said David Davidson, M.D., chief medical officer, bluebird. “The accumulating clinical data show a correlation among vector copy number, the percentage of vector-containing cells, and the amount of hemoglobin produced by patients treated with LentiGlobin. We believe that the addition of our transduction enhancers to our manufacturing process has the potential to substantially increase the hemoglobin levels in patients with transfusion dependent beta-thalassemia and increase their likelihood of achieving clinically meaningful reductions in transfusion requirements or transfusion independence, the ultimate goal of our therapy.”

In this study, the process by which the patient’s cells are transduced with LentiGlobin will be modified by the addition of two additives during the transduction step of the manufacturing process, intended to increase vector copy number and the percentage of cells successfully transduced. bluebird also intends to incorporate these transduction enhancers into the manufacturing process for HGB-206, its ongoing study of LentiGlobin in patients with severe sickle cell disease (SCD).

About the HGB-207 Study

HGB-207 is a Phase 3, global, multi-center study designed to evaluate the safety and efficacy of LentiGlobin BB305 drug product in patients with transfusion-dependent beta-thalassemia and non-β0/β0 genotypes. The target enrollment of the study is 15 subjects. The study’s primary endpoint is transfusion independence, defined as a 12-month transfusion free period after transplant. This study is intended to be pivotal in the U.S. and confirmatory in the E.U.

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