bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today announced long-term follow up of two patients with beta-thalassemia major and early safety and efficacy data in the first patient with severe sickle cell disease (SCD) treated with LentiGlobin BB305 product candidate in the HGB-205 study. These data were presented today at the 20th Congress of theEuropean Hematology Association (EHA) in Vienna, Austria by Marina Cavazzana, M.D., Ph.D., lead investigator of the HGB-205 study and professor of hematology at Paris Descartes University, head of the department of Biotherapy Hospital, the clinical research INSERM center of Biotherapy at Necker Enfants Malades, (Assistance Publique-Hôpitaux de Paris) and theLymphohematopoiesis Laboratory, Institute of Genetic Diseases, Imagine, Paris, France.

Key findings:

  • Beta-thalassemia:
    • As of May 2015, Subjects 1201 and 1202 with beta-thalassemia major remained transfusion-independent for 16 and 14 months, respectively, with persistent stable expression of HbAT87Q. Neither has experienced a LentiGlobin-related adverse event.
  • Sickle Cell Disease:
    • The proportion of anti-sickling hemoglobin being produced by the first-ever patient with severe SCD treated with gene therapy (Subject 1204) is rising steadily and accounted for 45% of all hemoglobin production (40% HbAT87Q + 5% HbF) at the patient’s six-month visit post-drug product infusion; this is above the 30% threshold expected to potentially achieve a disease-modifying clinical effect.
    • As of May 2015, Subject 1204 had been free of transfusions for more than three months without complications or hospitalizations for SCD-related events post-transplant, and with improvement in hemolysis markers.

“These data are promising for patients living with beta-thalassemia major and severe sickle cell disease, two devastating, genetically-based hematologic diseases that have a profound impact on both quality of life and life expectancy,” said Professor Cavazzana. “The steady rise and high-level of HbAT87Q production in our patient with severe sickle cell disease is cause for optimism as we expect levels of anti-sickling hemoglobin of 30 percent or more could significantly improve and potentially eliminate the serious and life-threatening complications associated with sickle cell disease.”

bluebird bio also announced that the first patient with severe SCD has been infused in the HGB-206 U.S.-based clinical study at the National Institutes of Health Clinical Center in Bethesda, Maryland. HGB-206 is a Phase 1 clinical trial evaluating the safety and efficacy of bluebird bio’s LentiGlobin BB305 product candidate in subjects with severe SCD.

“Today’s data further demonstrate the potentially transformative effects of gene therapy for the treatment of beta-hemoglobinopathies and support our global regulatory strategy for LentiGlobin in beta-thalassemia major, with the goal of potentially accelerated approvals in the EU and U.S.,” said David Davidson, M.D., chief medical officer, bluebird bio. “We are excited that the promising early results for LentiGlobin in beta-thalassemia major are now extending to the first treated patient with severe sickle cell disease. With the treatment of the first patient with severe sickle cell disease in the HGB-206 study, we look forward to gaining increasing clarity on the potential clinical benefit of LentiGlobin for patients with severe sickle cell disease.”

HGB-205 Study Data
HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin BB305 product candidate for the treatment of patients with beta-thalassemia major and severe SCD. As of May 2015, two patients with beta-thalassemia major and one patient with severe SCD have undergone infusion with LentiGlobin BB305 product candidate in this study.

  • Patients with beta-thalassemia major demonstrated continued transfusion independence: The data presented today are an update on those initially presented at EHA in June 2014 and demonstrate a long-term stable and durable response. As reported at ASH in December 2014, Subjects 1201 and 1202 achieved rapid transfusion independence with near-normal hemoglobin levels. As of May 2015, Subjects 1201 and 1202 remained transfusion independent for 16 and 14 months, respectively.
  • Early efficacy data is promising in the first subject with severe SCD treated with gene therapy:The production of anti-sickling hemoglobin is consistent with a level expected to potentially achieve a disease-modifying clinical effect in Subject 1204. At the six-month visit post-drug product infusion, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) accounted for 45% of all hemoglobin production (40% HbAT87Q + 5% HbF). The patient’s vector copy number in peripheral blood leukocytes was 2.2.
    • Subject 1204 had his last red blood cell (RBC) transfusion at day 88. Since his infusion with LentiGlobin, this patient has had no hospitalizations, and has demonstrated improvements in markers of hemolysis, including normalization of reticulocyte count (from 283.3 x 109/L to 131.7 x 109/L) and lactate dehydrogenase (LDH) (from 626 U/L to 254 U/L). (Original Source)

Shares of bluebird bio closed yesterday at $180.81, down $5.37 or 2.88%. BLUE has a 1-year high of $197.35 and a 1-year low of $24.70. The stock’s 50-day moving average is $165.80 and its 200-day moving average is $115.90.

On the ratings front, bluebird has been the subject of a number of recent research reports. In a report issued on June 10, BMO analyst Jim Birchenough reiterated a Buy rating on BLUE, with a price target of $200, which represents a potential upside of 10.6% from where the stock is currently trading. Separately, on the same day, Roth Capital’s Debjit Chattopadhyay maintained a Buy rating on the stock and has a price target of $192.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Jim Birchenough and Debjit Chattopadhyay have a total average return of 39.4% and 32.2% respectively. Birchenough has a success rate of 62.7% and is ranked #21 out of 3624 analysts, while Chattopadhyay has a success rate of 69.5% and is ranked #16.

In total, 7 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 $206.40 which is 14.2% above where the stock closed yesterday.

bluebird bio Inc is a clinical-stage biotechnology company. It is engaged in developing potentially transformative gene therapies for severe genetic and rare diseases and in the field of T cell-based immunotherapy.