Early data from Phase III Northstar-2 study of LentiGlobin

US clinical-stage biotech firm bluebird bio’s (Nasdaq: BLUE) shares closed down 5.5% at $112.00, having fallen as low as $105.90 earlier on Friday, after the firm released what was apparently positive data on its lead candidate to be presented at the European Hematology Association (EHA) Annual Meeting in Madrid, Spain on June 25.

The company 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-β0/β0 genotypes.

“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, chief medical officer of 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,” Dr Davidson pointed out.