Interim results from ongoing Northstar-2 (HGB-207) Phase III clinical study of LentiGlobin drug product in patients with transfusion-dependent beta-thalassemia (TDT) and non-beta0/beta0 genotypes.- bluebird bio

bluebird bio, Inc. announced early interim data from the ongoing Northstar-2 (HGB-207) Phase III clinical study of LentiGlobin drug product in patients with transfusion-dependent beta-thalassemia (TDT) and non-beta0/beta0 genotypes. These data will be presented by Mark Walters, M.D., UCSF Benioff Children�s Hospital, Oakland, California, in an oral session on Sunday, June 25 at the European Hematology Association (EHA) Annual Meeting in Madrid, Spain.

�Northstar-2 is our first study to utilize our improved LentiGlobin drug product manufacturing process to increase the drug product vector copy number and percent of cells transduced. The first patient treated in this study exemplifies the promise of gene therapy, discontinuing blood transfusions approximately a month after treatment and achieving a normal level of total hemoglobin production at six months post-treatment,� said David Davidson, M.D., chief medical officer, bluebird bio. �These early results suggest that the improved manufacturing process results in consistently higher drug product vector copy numbers (VCN) and lentiviral vector positive (LVV+) cells, which is correlated with higher production of HbAT87Q and ultimately may address known patient-to-patient variability.�

The Northstar-2 Study is an ongoing, open-label, single-dose, international, multicenter Phase III study designed to evaluate the safety and efficacy of LentiGlobin drug product for the treatment of patients with TDT and non-beta0/beta0 genotypes. As of June 2, 2017, drug product had been manufactured for six patients. The median DP VCN for these patients was 3.0 (range: 2.4 � 4.0), compared to a median DP VCN of 0.7 (range: 0.3 � 1.5) in Northstar. Follow-up on Patients 2 and 3 was not sufficient for total hemoglobin or days since last transfusion to be clinically relevant. The safety profile to date appears consistent with autologous transplantation. No Grade 3 or higher drug-product related adverse events have been observed.