Company Update (NASDAQ:BLUE): bluebird bio Announces First Patient Treated with LentiGlobinTM Drug Product in Phase 3 Trial of Patients with Transfusion-Dependent β-Thalassemia

bluebird bio Inc (NASDAQ: BLUE) announced treatment of the first patient in Northstar-2, the Phase 3 study of its LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β00 genotypes. This study will use LentiGlobin drug product manufactured with the addition of transduction enhancers intended to increase the drug product vector copy number and percent of cells transduced.

The study’s primary endpoint is the proportion of treated subjects who meet the definition of “transfusion independence,” defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study.

“The opening of our first Phase 3 trial in TDT is an exciting milestone for bluebird,” said David Davidson, chief medical officer. “At ASH we presented interim LentiGlobin data from the Northstar study showing substantial and durable treatment effects, with all patients with non-β00 genotypes and at least twelve months of follow-up achieving freedom from transfusions. We are hopeful that our new manufacturing process incorporating transduction enhancers will build upon these results and may provide even better outcomes for patients. It is an encouraging start for Northstar-2 to have achieved a robust drug product VCN of 2.9 c/dg and 77% LVV+ for our first treated patient. We are tremendously grateful to the patients, families, study investigators, and site staff who have participated in our LentiGlobin program and enabled progress toward our goal of improving the lives of patients with TDT.”

“The current results of the HGB-204 and HGB-205 LentiGlobin studies show a reduction, and in some cases, elimination of RBC transfusions in patients with TDT,” said Mark C. Walters, M.D., UCSF Benioff Children’s Hospital Oakland, a principal investigator on the study. “The current study, Northstar-2, will test a new manufacturing method to increase the VCN in the drug product. If it is successful, this new enhanced transduction method could expand the number of patients who no longer need transfusions after the gene therapy treatment.”

bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β00genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction. (Original Source)

Shares of Bluebird bio Inc closed yesterday at $68.55, down $1.45 or -2.07%. BLUE has a 1-year high of $79.70 and a 1-year low of $35.37. The stock’s 50-day moving average is $59.11 and its 200-day moving average is $55.31.

On the ratings front, Bluebird bio Inc has been the subject of a number of recent research reports. In a report issued on December 12, Jefferies Co. analyst Gena Wang reiterated a Buy rating on BLUE, with a price target of $88, which represents a potential upside of 28% from where the stock is currently trading. Separately, on December 6, J.P. Morgan’s Cory Kasimov maintained a Buy rating on the stock and has a price target of $120.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Gena Wang and Cory Kasimov have a yearly average loss of -8.1% and -12.5% respectively. Wang has a success rate of 34% and is ranked #4061 out of 4285 analysts, while Kasimov has a success rate of 31% and is ranked #4192.

Overall, one research analyst has rated the stock with a Sell rating, 2 research analysts have assigned a Hold rating and 11 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $82.00 which is 19.6% above where the stock closed yesterday.

bluebird bio, Inc. is a clinical-stage biotechnology company. Its integrated product platform includes gene therapy, cancer immunotherapy, and gene editing. 

Stay Ahead of Everyone Else

Get The Latest Stock News Alerts