bluebird bio Gets its Wings on Rare Drug Trial Results

Andrew Klips  |

Cambridge, Massachusetts-based bluebird bio (BLUE) soared to an all-time intraday high in Monday action after releasing positive preliminary clinical trial data for its experimental gene therapy to treat the rare blood disorder beta-thalassermia major.  bluebird bio (that’s right, no capital letters) reported the data on its LentiGlobin BB305 treatment at the 19th Annual Congress of the European Hematology Association in Milan, Italy.

bluebird Flies on Treatment for Cooley's Anemia

Beta-thalassermia major, sometimes called Cooley’s anemia, is a hereditary disorder caused by a genetic abnormality of the beta globin gene that results in defective red blood cells, the cells that carry oxygen to cells throughout the body via hemoglobin. The condition is characterized by patients suffering from chronic anemia, an increased risk for blood clots and generally requires a lifetime of regular transfusions. Beta-thalassermia comes in two types:  major and intermedia, with major being the more serious and more prevalent condition.

bluebird’s LentiGlobin replaces the defective gene through transplantation of a fully-functional one. The current research is on the second generation of LentiGlobin and demonstrating more robust results than the previous version.

About 290,000 people worldwide have been diagnosed with beta-thalassemia, with about 15,000 of those patients in the U.S. and Europe.

Early Clinical Trials Show Progress in Treating Beta-thalassermia Major

bluebird said that it had success in treating two Beta-thalassermia major patients in a Phase 1/2 trial conducted in France, dubbed HGB-205. The research showed that one dose of bluebird’s autologous therapy successfully replaced malfunctioning genes with functional ones, leading to substantial increases in hemoglobin levels within 2 – 4.5 months. The subjects received their last blood transfusion on day 10 and day 12, respectively, after treatment with LentiGlobin BB305 and have remained transfusion independent for approximately 3 and 6 months, respectively.

“We are encouraged by the early and high-level production of corrected betaAT87Q-globin and the rapid onset of transfusion independence in these initial subjects, as well as the absence of any gene therapy related adverse events,” said Dr. David Davidson, bluebird bio’s Chief Medical Officer, in a statement Monday morning. We look forward to providing additional data from this study and our ongoing multi-center Northstar Study later this year.” The Northstar Study is a similar study of patients and LentiGlobin, only it is being conducted in the U.S.

The trial in France is slated to enroll seven patients and the one in the U.S. up to 15.

Lenti-D Also in the Pipeline

bluebird has a more advanced study of a product it calls Lenti-D that is evaluating the therapy in a Phase 2/3 study for childhood cerebral adrenoleukodystrophy, a rare, hereditary neurological disorder affecting young boys. The process is similar, involving extracting stem cells from a patient, treating the cells with a “lentivirus” to repair the gene sequence and then re-injecting them back into the patient.

Shares of BLUE rocketed up by about 70 percent to $41.75 early in the trading day and closed the session ahead by 32.1% at $34.46. The previous all-time high for the company, which went public almost a year ago to the day, had been $36.25.


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