bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today reported business highlights and financial results for the first quarter ended March 31, 2016.

“In early 2016 we achieved two crucial clinical milestones: treating the first patient in the Phase 1 study of our anti-BMCA CAR T therapy bb2121, and presenting the first clinical data from our Starbeam study of Lenti-D in boys with CALD. We are very pleased with this significant progress as we continue to build our T cell immunotherapy and HSC gene therapy platforms,” said Nick Leschly, chief bluebird. “On the LentiGlobinTM program, we are on track to achieve our remaining milestones this year, which include initiation of the HGB-207 study in non-ß0/ß0 transfusion-dependent thalassemia (TDT) as well as integration of manufacturing process improvements into our LentiGlobin clinical trials.”

Recent Highlights

  • PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN – In April, Dr. Florian Eichler ofMassachusetts General Hospital for Children presented interim clinical data from the Starbeam study of Lenti-D in CALD at AAN. Initial Starbeam results suggest Lenti-D gene therapy may have similar efficacy to allogeneic hematopoietic stem cell transplant (HCT), the current standard of care, with a more favorable safety profile. As of March 31, 2016, three of the 17 patients enrolled in the study have reached two years of follow-up and remain free of major functional disabilities (MFDs), the primary endpoint of the study. Sixteen of the 17 patients had stabilization of their neurological function score (NFS), and 14 of 17 had a stable Loes score. The safety profile of Lenti-D treatment appeared consistent with myeloablative conditioning.
  • TEN ABSTRACTS ACCEPTED FOR PRESENTATION AT ASGCT 19th ANNUAL MEETING – Two oral presentations given by bluebird’s academic collaborators will highlight previously presented data from bluebird bio’s ongoing gene therapy clinical trials, including interim data from the Starbeam Study of Lenti-D in cerebral adrenoleukodystrophy, and interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and TDT. Eight additional presentations will be featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities in both HSC gene therapy and T cell immunotherapy.
  • TREATED FIRST PATIENT IN PHASE 1 STUDY OF BB2121 IN MULTIPLE MYELOMA – In February, the first patient was infused in the CRB-401 study of anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene exercised its option to exclusively license bb2121. Under the terms of the collaboration agreement between the two companies, bluebird bio received a $10.0 million option exercise payment from Celgene and may now elect to co-develop and co-promote the product candidate in the United States withCelgene. We are also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.
  • FULLY ENROLLED EXPANDED NORTHSTAR STUDY – Achievement of 18 patient enrollment target in Northstar Study of LentiGlobin in patients with transfusion-dependent thalassemia, including three additional adolescent patients.

Upcoming Anticipated Milestones

  • Update on LentiGlobin process improvements in the second half of 2016
  • Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016
  • Presentation of updated clinical data for LentiGlobin at the ASH annual meeting in December 2016

First Quarter 2016 Financial Results and Financial Guidance

  • Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2016 were$826.9 million, compared to $865.8 million as of December 31, 2015, a decrease of $38.9 million.
  • Revenues: Collaboration revenue was $1.5 million for the first quarter of 2016 compared to$6.3 million for first quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.
  • R&D Expenses: Research and development expenses were $41.9 million for the first quarter of 2016 compared to $23.7 million for the first quarter of 2015. The increase in research and development expenses was primarily attributable to increased employee compensation and facilities costs due to increased headcount, and increased manufacturing, clinical, research, and information technology costs to support the advancement of our clinical and pre-clinical programs.
  • G&A Expenses: General and administrative expenses were $16.0 million for the first quarter of 2016 compared to $7.3 million for the first quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting costs to support our overall growth.
  • Net Loss: Net loss was $56.3 million for the first quarter of 2016 compared to $24.8 million for the first quarter of 2015.
  • Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $826.9 million as of March 31, 2016 will be sufficient to fund its current operations through 2018. (Original Source)

Shares of bluebird closed today at $40.73, down $1.96 or -4.59%. BLUE has a 1-year high of $197.35 and a 1-year low of $37.40. The stock’s 50-day moving average is $45.80 and its 200-day moving average is $57.72.

On the ratings front, bluebird has been the subject of a number of recent research reports. In a report issued on April 21, Goldman Sachs analyst Salveen Richter maintained a Hold rating on BLUE, with a price target of $149, which implies an upside of 252.2% from current levels. Separately, on April 20, BTIG’s Dane Leone maintained a Buy rating on the stock and has a price target of $72.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Salveen Richter and Dane Leone have a total average return of 30% and 6% respectively. Richter has a success rate of 67.9% and is ranked #58 out of 3838 analysts, while Leone has a success rate of 63.6% and is ranked #502.

The street is mostly Bullish on BLUE stock. Out of 6 analysts who cover the stock, 4 suggest a Buy rating and 2 recommend to Hold the stock. The 12-month average price target assigned to the stock is $110.50, which implies an upside of 161.2% from current levels.

bluebird bio, Inc. is a clinical-stage biotechnology company. The company develops next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. It has two clinical-stage programs in development for childhood cerebral adrenoleukodystrophy and beta-thalassemia/sickle cell disease and a preclinical oncology program in the chimeric antigen receptor T cells field. bluebird bio was founded by Philippe Leboulch and Ronald C. Dorazio on April 16, 1992 and is headquartered in Cambridge, MA.