bluebird bio Reports First Quarter 2016 Financial Results and Recent Operational Progress

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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
LentiGlobin^TM 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 of Massachusetts 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 19^th
  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 with Celgene. 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.

About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise
and gene editing capabilities, bluebird bio has built an integrated
product platform with broad potential application to severe genetic
diseases and cancer. bluebird bio’s gene therapy clinical programs
include its Lenti-D™ product candidate, currently in a Phase 2/3 study,
called the Starbeam Study, for the treatment of cerebral
adrenoleukodystrophy, and its LentiGlobin™ BB305 product candidate,
currently in three clinical studies for the treatment of
transfusion-dependent ß-thalassemia, and severe sickle cell disease.
bluebird bio’s oncology pipeline is built upon the company’s leadership
in lentiviral gene delivery and T cell engineering, with a focus on
developing novel T cell-based immunotherapies, including chimeric
antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird
bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program
partnered with Celgene. bb2121 is currently being studied in a Phase 1
trial for the treatment of relapsed/refractory multiple myeloma.
bluebird bio also has discovery research programs utilizing
megaTALs/homing endonuclease gene editing technologies with the
potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Washington, and Paris, France.
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
Forward-Looking Statements
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the Company’s financial condition and results of
operations, the sufficiency of its cash, cash equivalents and marketable
securities, as well as the advancement of, and anticipated development
and regulatory milestones and plans related to the Company’s product
candidates and clinical studies. Any forward-looking statements are
based on management’s current expectations of future events and are
subject to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in or
implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to, risks that the
preliminary results from our clinical trials will not continue or be
repeated in our ongoing clinical trials, the risk of cessation or delay
of any of the ongoing or planned clinical studies and/or our development
of our product candidates, the risk of a delay in the enrollment of
patients in our clinical studies, the risk that our collaboration with
Celgene will not continue or will not be successful, and the risk that
any one or more of our product candidates will not be successfully
developed, approved or commercialized. For a discussion of other risks
and uncertainties, and other important factors, any of which could cause
our actual results to differ from those contained in the forward-looking
statements, see the section entitled “Risk Factors” in our most recent
quarterly report on Form 10-Q, as well as discussions of potential
risks, uncertainties, and other important factors in our subsequent
filings with the Securities and Exchange Commission. All information in
this press release is as of the date of the release, and bluebird bio
undertakes no duty to update this information unless required by law.
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