bluebird bio Reports First Quarter 2019 Financial Results and Highlights Operational Progress

– Received positive opinion from CHMP for ZYNTEGLO™ (autologous CD34+
cells encoding β^A-T87Q-globin gene) gene
therapy for patients 12 years and older with transfusion-dependent
β-thalassemia (TDT) who do not have β⁰/β⁰
genotype –

– Company hosting Analyst Day on May 9, 2019 focused on research and
commercial strategies –

– Ended quarter with $1.73 billion in cash, cash equivalents and
marketable securities –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird
bio, Inc. (NASDAQ: BLUE) today reported financial results and
business highlights for the first quarter ended March 31, 2019.

“In the first quarter, we came one step closer to bringing ZYNTEGLO™, the
first gene therapy for transfusion-dependent β-thalassemia (TDT), to
patients with a positive opinion from the CHMP and potential approval in
Europe anticipated in the second quarter,” said Nick Leschly, chief
bluebird. “We look forward to providing more detail on our commercial
plans and launch expectations at our upcoming Analyst Day, including how
our initial country-by-country launch in TDT will lay the foundation for
our future launches. Our Analyst Day will also delve into our progress
toward our stated goals of 1-2 new INDs per year beginning in 2020, and
a deep pipeline by 2022. While the research and commercial engines are
revving, our clinical development programs continue to progress at full
speed, with the next data from our programs in TDT and sickle cell
disease (SCD) anticipated in mid-year, and studies in earlier lines of
multiple myeloma adding to the robust ide-cel (bb2121) development plan.
Our team of bluebirds and our partners have been working relentlessly to
get us to this point – and we’re only just getting started on our
journey to help patients and their families.”

Recent Highlights

TDT

• CHMP POSITIVE OPINION – In March 2019, the Committee for
  Medicinal Products for Human Use (CHMP) of the European Medicines
  Agency (EMA) adopted a positive opinion recommending conditional
  marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding
  β ^A-T87Q-globin gene), a gene therapy for patients 12 years
  and older with transfusion-dependent β-thalassemia (TDT) who do not
  have a β⁰/β⁰ genotype, for whom hematopoietic
  stem cell (HSC) transplantation is appropriate but a human leukocyte
  antigen (HLA)-matched related HSC donor is not available. If approved,
  ZYNTEGLO, formerly referred to as LentiGlobin™ for TDT, will be the
  first commercially available gene therapy to treat TDT. The CHMP’s
  positive opinion will now be reviewed by the European Commission (EC),
  which has the authority to grant marketing authorization for ZYNTEGLO
  in the European Union (EU).

CALD

• ALD-104 – In April 2019, bluebird bio treated the first patient
  in ALD-104, the company’s international, non-randomized, open-label,
  multi-site Phase 3 study of Lenti-D Drug Product after myeloablative
  conditioning using busulfan and fludarabine in patients ≤17 years of
  age with cerebral adrenoleukodystrophy (CALD). The study will enroll
  approximately 20 patients. The primary endpoint of the study is the
  proportion of patients who are alive and do not have any of the 6
  major functional disabilities (MFDs) at Month 24.

MULTIPLE MYELOMA

• NEJM PUBLICATION – In May 2019, bluebird bio and Celgene
  announced that the New England Journal of Medicine (NEJM) has
  published interim results from CRB-401, the ongoing phase 1 study of
  idecabtagene vicleucel (ide-cel, formerly known as bb2121), the
  companies’ lead investigational BCMA-targeted chimeric antigen
  receptor (CAR) T-cell therapy candidate for patients with relapsed and
  refractory multiple myeloma.

COMPANY

• MANAGEMENT APPOINTMENT – In April 2019, bluebird bio announced
  that Joanne Smith-Farrell has been appointed chief business officer.
  In this role, Joanne will lead corporate development and strategy,
  alliance management, and she will also continue to serve as our
  oncology franchise leader. Joanne has been promoted to chief business
  officer after joining bluebird in April 2017 as our senior vice
  president, corporate development and strategy.
• bRT OPENING – In March 2019, bluebird bio announced the
  official opening of its first wholly owned manufacturing facility in
  Durham, N.C., that will produce lentiviral vector for the company’s
  investigational gene and cell therapies, including: ide-cel and
  bb21217 for the treatment of multiple myeloma and potentially
  LentiGlobin™ for the treatment of TDT and SCD. bluebird bio purchased
  the facility in November 2017.

Upcoming Anticipated Milestones

• TDT
  • European approval of ZYNTEGLO in patients with TDT and non-β⁰/β⁰
    genotypes in Q2
  • Submission of Biologics Licensing Application to the U.S. FDA for
    ZYNTEGLO in patients with TDT and non-β⁰/β⁰
    genotypes by the end of 2019
  • Presentation of ZYNTEGLO clinical data from the Northstar-2
    (HGB-207) clinical study in patients with TDT and non-β⁰/β⁰
    genotypes by mid-2019 and by end of 2019
  • Presentation of ZYNTEGLO clinical data from the Northstar-3
    (HGB-212) clinical study in patients with TDT and the β⁰/β⁰
    genotype by mid-2019 and by end of 2019
• SCD
  • Initiation of Phase 3 HGB-210 study of LentiGlobin in patients
    with SCD by end of 2019
  • Presentation of LentiGlobin clinical data from the HGB-206
    clinical study in patients with SCD by mid-2019 and by end of 2019
• Multiple Myeloma
  • Presentation of ide-cel clinical data from the
    registration-enabling KarMMa study and CRB-401 study in patients
    with relapsed/refractory multiple myeloma by end of 2019
  • Presentation of bb21217 clinical data from the CRB-402 clinical
    study in patients with relapsed/refractory multiple myeloma by the
    end of 2019

First Quarter 2019 Financial Results

• Cash Position: Cash, cash equivalents and marketable securities
  as of March 31, 2019 and December 31, 2018 were $1.73 billion and
  $1.89 billion, respectively. The decrease in cash, cash equivalents
  and marketable securities is primarily related to cash used in support
  of normal operating activities and cash used to purchase property,
  plant and equipment as the company continues the buildout of its
  manufacturing facility in Durham, North Carolina.
• Revenues: Total revenues were $12.5 million for the three
  months ended March 31, 2019 compared to $16.0 million for the three
  months ended March 31, 2018. The decrease was primarily attributed to
  decreased manufacturing services under the company’s agreement
  with Celgene Corporation, offset by increased license and royalty
  revenue.
• R&D Expenses: Research and development expenses were $122.6
  million for the three months ended March 31, 2019 compared to $97.1
  million for the three months ended March 31, 2018. The increase was
  primarily driven by costs incurred to advance and expand the company’s
  pipeline.
• G&A Expenses: General and administrative expenses were
  $60.3 million for the three months ended March 31, 2019 compared to
  $34.9 million for the three months ended March 31, 2018. The increase
  was largely attributable to overall growth of the pipeline as well as
  commercial-readiness activities.
• Net Loss: Net loss was $164.4 million for the three months
  ended March 31, 2019 compared to $115.1 million for the three months
  ended March 31, 2018.

About bluebird bio, Inc.
bluebird bio is pioneering gene
therapy with purpose. From our Cambridge, Mass., headquarters, we’re
developing gene therapies for severe genetic diseases and cancer, with
the goal that people facing potentially fatal conditions with limited
treatment options can live their lives fully. Beyond our labs, we’re
working to positively disrupt the healthcare system to create access,
transparency and education so that gene therapy can become available to
all those who can benefit.

bluebird bio is a human company powered by human stories. We’re putting
our care and expertise to work across a spectrum of disorders by
researching cerebral adrenoleukodystrophy, sickle cell disease,
transfusion-dependent β-thalassemia and multiple myeloma using three
gene therapy technologies: gene addition, cell therapy and
(megaTAL-enabled) gene editing. bluebird bio has additional nests in
Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more
information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio,
LinkedIn,
Instagram
and YouTube.

ZYNTEGLO and LentiGlobin are trademarks of bluebird bio, Inc..

The full common name for ZYNTEGLO: A genetically modified autologous
CD34+ cell enriched population that contains hematopoietic stem cells
transduced with lentiviral vector encoding the β^A-T87Q-globin
gene.

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, results of operations, as well as
statements regarding the anticipated development for the company’s
product candidates, including anticipated regulatory milestones,
potential commercial launches, planned clinical studies, as well as the
company’s intentions regarding the timing for providing further updates
on the development and commercialization of its product candidates. 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, the risks that the preliminary positive efficacy and safety results
from our prior and ongoing clinical trials of our product candidates
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, risks that the
current or planned clinical trials of our product candidates will be
insufficient to support regulatory submissions or marketing approval in
the United States and European Union, the risk that we will encounter
challenges in the commercial launch of ZYNTEGLO in the European Union,
including in managing our complex supply chain for the delivery of drug
product or in the adoption of value-based payment models or in obtaining
sufficient coverage or reimbursement for our products if approved, the
risk that our collaborations, including the 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
Form 10-K, 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.

Contacts

Investors:
bluebird bio
Elizabeth Pingpank, 617-914-8736
[email protected]

Media:
bluebird bio
Jenn Snyder, 617-448-0281
[email protected]