[February 21, 2018] |
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bluebird bio Reports Fourth Quarter and Full Year 2017 Financial Results and Highlights Operational Progress
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 fourth quarter and
full year ended December 31, 2017.
"We ended 2017 in a tremendously strong position with compelling data
and progress across all four of our clinical programs. This progress
brings us closer to potentially providing transformative therapies to a
broader population of patients that we urgently seek to serve," said
Nick Leschly, chief bluebird. "We have an aggressive plan to file three
programs with regulatory authorities in the next two years: LentiGlobin
in TDT in 2018, Lenti-D in CALD in 2019 and, with our partners at
Celgene, bb2121 in multiple myeloma, also in 2019. The potential impact
that our gene and cell therapies can bring to patients drives our
commitment to execute on our strategy which is focused on operating with
discipline, expanding our capabilities for commercial success, and
levering our product engine to continue to grow and advance our
pipeline."
Recent Highlights
-
FINAL PATIENT INFUSED IN CRB-401 - In February 2018, the final
patient to be enrolled in CRB-401, the Phase 1 study of bb2121
investigational anti-BCMA CAR T therapy in patients with
relapsed/refractory multiple myeloma, was infused. A total of 43
patients (21 in the dose escalation phase and 22 in the expansion
phase) have been treated in this study.
-
FIRST PATIENT TREATED IN KARMMA - In February 2018, the first
patient was infused in KarMMa, a registration-enabling study of bb2121
in patients with relapsed/refractory multiple myeloma. This study is
being run by Celgene, bluebird's partner in the development of
anti-BCMA CAR T therapies.
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CHIEF COMMERCIAL OFFICER APPOINTED - In February 2018, bluebird
appointed Alison Finger as Chief Commercial Officer. In this role,
Alison will be responsible for shaping and delivering an integrated
global commercial strategy to make bluebird's gene therapies broadly
accessible to patients. She will oversee all commercial strategy and
operations, access management, including pricing, reimbursement and
health outcomes, as well as patient operations. Alison joined bluebird
as senior vice president, marketing and product launch in August of
2015.
-
ASH PRESENTATIONS - At the American Society of Hematology (ASH)
Annual Meeting, bluebird provided compelling clinical updates across
its ongoing studies of LentiGlobin in TDT and severe sickle cell
disease (SCD), and bb2121 anti-BCMA CAR T therapy. These data can be
found here
(HGB-204 and HGB-207 studies of LentiGlobin in TDT), here
(HGB-206 study of LentiGlobin in SCD), here
(HGB-205 single center study of LentiGlobin in patients with TDT or
SCD) and here
(bb2121). All data in these linked press releases are as of the
respective data cut-off dates described in the press releases.
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COLLABORATION WITH TC BIOPHARM - In December 2017, bluebird and
immunotherapy company TC BioPharm, Ltd., or TCB, announced the
execution of a strategic collaboration and license agreement focused
on gamma delta CAR T cells. Under the terms of the agreement, bluebird
and TCB will collaborate to discover and develop CAR-engineered gamma
delta T cells, which has the potential to be a powerful new platform
for CAR T cell therapies in cancer, with potential applicability for
both allogeneic and autologous therapies across liquid and solid
tumors. TCB is responsible for development of all programs through
Phase 1/2, at which point bluebird has the exclusive option to assume
sole responsibility for further clinical development and
commercialization on a global basis.
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MANUFACTURING SITE ACQUISITION AND AGREEMENTS - In November
2017, bluebird announced its acquisition of a 125,000 square foot
manufacturing facility in Durham, North Carolina. Once construction
and validation is complete, the site will produce lentiviral vector
for the company's gene and cell therapies. In addition, bluebird also
entered into multi-year agreements with three manufacturing partners
in the United States and Europe: Brammer Bio (Cambridge, MA), Novasep
(Gosselies, Belgium) and MilliporeSigma, the Life Science business of
Merck KGaA (Carlsbad, CA (News - Alert)). Each of these partners is collaborating
with bluebird on production of lentiviral vector across all programs.
bluebird also partners with Lonza (Houston, TX) and apceth Biopharma
(Munich, Germany) to produce drug product for its product candidates.
-
BB2121 BREAKTHROUGH AND PRIME DESIGNATIONS - In November 2017,
bluebird and Celgene announced that bb2121 had been granted
Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug
Administration (FDA) and PRIority MEdicines (PRIME) eligibility by the
European Medicines Agency (EMA (News - Alert)). BTD is designed to expedite the
development and review of drugs that are intended to treat serious or
life-threatening conditions. PRIME is a program launched by the EMA to
enhance support for the development of medicines that target an unmet
medical need.
-
FIRST PATIENT TREATED IN NORTHSTAR-3 (HGB-212) - In November
2017, the first patient was infused with LentiGlobin drug product in
Northstar-3, bluebird's Phase 3, global, multi-center study designed
to evaluate the safety and efficacy of LentiGlobin in patients with
TDT and the ß0/ß0 genotype. The target
enrollment of the study is 15 adult, adolescent or pediatric patients.
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STRENGTHENED BALANCE SHEET - In December 2017, bluebird raised
$569.8 million in net proceeds through a public equity offering. In
January 2018, bluebird raised an additional $48.6 million in net
proceeds pursuant to the partial exercise of the underwriters'
over-allotment option in connection with this public equity offering.
bluebird anticipates that its cash, cash equivalents and marketable
securities as of December 31, 2017 will be sufficient to fund
operations into 2021 based on the company's current business plan.
2018 Anticipated Milestones
-
Filing for European approval of LentiGlobin in patients with TDT and
non-ß0/ß0 genotypes in the second half of 2018
-
Update on the clinical development plan and registration strategy for
LentiGlobin in SCD by year end 2018
-
Initiation of an investigator-led Phase 1 clinical study of a
lentiviral gene therapy targeting BCL11a suppression and fetal
hemoglobin upregulation in patients with SCD
-
Initiation by Celgene of a Phase 3 clinical study of bb2121 in third
line multiple myeloma
-
Presentation of bb2121 clinical data from the CRB-401 study at the
American Society of Clinical Oncology (ASCO) Annual Meeting
-
Presentation of LentiGlobin clinical data from the Northstar-2
(HGB-207) clinical study in patients with TDT and non- ß0/ß0
genotypes at the European Hematology Association Annual Meeting
-
Presentation of LentiGlobin clinical data from the HGB-206 clinical
study in patients with SCD at the ASH Annual Meeting
-
Presentation of LentiGlobin clinical data from the Northstar-3
(HGB-212) clinical study in patients with TDT and the ß0/ß0
genotype at the ASH Annual Meeting
-
Presentation of bb21217 clinical data from the CRB-402 clinical study
in patients with relapsed/refractory multiple myeloma at the ASH
Annual Meeting
-
Presentation of Lenti-D clinical data from the ongoing Starbeam (News - Alert)
clinical study in patients with CALD by the end of 2018
Fourth Quarter and Full Year 2017 Financial Results
-
Cash Position: Cash, cash equivalents and marketable securities
as of December 31, 2017 were $1.6 billion, compared to $884.8 million
as of December 31, 2016, an increase of $729.5 million, which was
primarily driven by the company's June and December 2017 public equity
offerings.
-
Revenues: Total revenues were $4.2 million for the fourth
quarter of 2017 compared to $1.6 million for fourth quarter of 2016,
and $35.4 million for the year ended December 31, 2017 compared to
$6.2 million for the year ended December 31, 2016. The increase is
primarily attributable to the commencement of revenue recognition for
the bb2121 license and manufacturing services under the company's
agreement with Celgene and revenue recognized from the company's
out-licensing agreement with Novartis Pharma AG (Novartis).
-
R&D Expenses: Research and development expenses were $92.6
million for the fourth quarter of 2017 compared to $57.1 million for
the fourth quarter of 2016, and $273.0 million for the year ended
December 31, 2017 compared to $204.8 million for the year ended
December 31, 2016. The increase in research and development expenses
was driven by costs incurred to advance and expand the company's
pipeline and is attributable to increased clinical trial-related costs
and manufacturing costs for our development programs, as well as
increased employee-related costs due to headcount growth supporting
overall research and development activities. When comparing the fourth
quarter of 2017 to the fourth quarter of 2016, the increase in
research and development expense was also attributable to license
milestones and fees, primarily related to the company's strategic
collaboration and license agreement with TCB.
-
G&A Expenses: General and administrative expenses were
$29.1 million for the fourth quarter of 2017 compared to $16.2 million
for the fourth quarter of 2016, and $93.6 million for the year ended
December 31, 2017 compared to $65.1 million for the year ended
December 31, 2016. The increase in general and administrative expenses
was attributable to increases in employee-related costs due to
headcount to support overall growth, commercial-readiness activities,
and facility-related expenses.
-
Cost of License and Royalty Revenue: Cost of license and
royalty revenue was less than $0.1 million for the fourth quarter of
2017 and $1.5 million for the year ended December 31, 2017 and is
primarily composed of amounts payable to third party licensors in
connection with amounts received under our out-license arrangement
with Novartis. No similar costs were incurred during 2016.
-
Net Loss: Net loss was $117.2 million for the fourth quarter of
2017 compared to $71.4 million for the fourth quarter of 2016, and
$335.6 million for the year ended December 31, 2017 compared to $263.5
million for the year ended December 31, 2016.
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 for the treatment of cerebral adrenoleukodystrophy, and its
LentiGlobin® product candidate for the treatment of
transfusion-dependent ß-thalassemia, also known as ß-thalassemia major,
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 programs, bb2121
and bb21217, are anti-BCMA CAR T programs partnered with Celgene.
bluebird bio also has discovery research programs utilizing
megaTAL/homing endonuclease gene editing technologies with the potential
for use across the company's pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
Washington, Durham, North Carolina and Zug, Switzerland.
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, results of operations and sufficiency
of its cash, cash equivalents and marketable securities to fund its
planned operations, as well as statements regarding the anticipated
development and regulatory milestones and plans for to the Company's
product candidates and clinical studies and statements regarding the
Company's plans to provide updates on the development 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, 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,
risks that the current or planned clinical trials of the LentiGlobin,
Lenti-D or bb2121 product candidates will be insufficient to support
regulatory submissions or marketing approval in the United States and
European Union, 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 annual report on 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. Guidance as to the
sufficiency of our cash, cash equivalents and marketable securities to
fund our planned operations is based on current assumptions as of the
date hereof and does not include the effect of any future potential
license and collaboration agreements, business combinations or asset
acquisitions. 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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bluebird bio, Inc.
Consolidated Statements of Operations
(in thousands, except per share amounts)
(unaudited)
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Three Months Ended December 31,
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Year Ended December 31,
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|
|
|
|
|
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|
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2017
|
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2016
|
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2017
|
|
2016
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Revenue:
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue
|
|
|
|
$
|
4,018
|
|
|
$
|
1,552
|
|
|
$
|
22,207
|
|
|
$
|
6,155
|
|
License and royalty revenue
|
|
|
|
|
150
|
|
|
|
-
|
|
|
|
13,220
|
|
|
|
-
|
|
Total revenues
|
|
|
|
|
4,168
|
|
|
|
1,552
|
|
|
|
35,427
|
|
|
|
6,155
|
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Operating expenses:
|
|
|
|
|
|
|
|
|
|
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Research and development
|
|
|
|
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92,576
|
|
|
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57,133
|
|
|
|
273,040
|
|
|
|
204,775
|
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General and administrative
|
|
|
|
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29,087
|
|
|
|
16,178
|
|
|
|
93,550
|
|
|
|
65,119
|
|
Cost of license and royalty revenue
|
|
|
|
|
7
|
|
|
|
-
|
|
|
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1,527
|
|
|
|
-
|
|
Change in fair value of contingent consideration
|
|
|
|
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(730
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)
|
|
|
576
|
|
|
|
(525
|
)
|
|
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4,091
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Total operating expenses
|
|
|
|
|
120,940
|
|
|
|
73,887
|
|
|
|
367,592
|
|
|
|
273,985
|
|
Loss from operations
|
|
|
|
|
(116,772
|
)
|
|
|
(72,335
|
)
|
|
|
(332,165
|
)
|
|
|
(267,830
|
)
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Interest (expense) income, net
|
|
|
|
|
(159
|
)
|
|
|
914
|
|
|
|
(2,001
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)
|
|
|
3,782
|
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Other (expense) income, net
|
|
|
|
|
(87
|
)
|
|
|
(6
|
)
|
|
|
(1,267
|
)
|
|
|
(71
|
)
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Loss before income taxes
|
|
|
|
|
(117,018
|
)
|
|
|
(71,427
|
)
|
|
|
(335,433
|
)
|
|
|
(264,119
|
)
|
Income tax benefit (expense)
|
|
|
|
|
(210
|
)
|
|
|
63
|
|
|
|
(210
|
)
|
|
|
612
|
|
Net loss
|
|
|
|
$
|
(117,228
|
)
|
|
$
|
(71,364
|
)
|
|
$
|
(335,643
|
)
|
|
$
|
(263,507
|
)
|
Net loss per share - basic and diluted:
|
|
|
|
$
|
(2.52
|
)
|
|
$
|
(1.88
|
)
|
|
$
|
(7.71
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)
|
|
$
|
(7.07
|
)
|
Weighted-average number of common shares used in computing net
loss per share - basic and diluted
|
|
|
|
|
46,534
|
|
|
|
38,051
|
|
|
|
43,535
|
|
|
|
37,284
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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bluebird bio, Inc.
Condensed Consolidated Balance Sheet Data
(in thousands)
(unaudited)
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|
|
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As of December 31,
|
|
|
|
|
2017
|
|
2016
|
Cash, cash equivalents and marketable securities
|
|
|
|
$
|
1,614,302
|
|
$
|
884,830
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Total assets
|
|
|
|
$
|
1,900,567
|
|
$
|
1,118,122
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Total liabilities
|
|
|
|
$
|
277,135
|
|
$
|
248,682
|
Total stockholders' equity
|
|
|
|
$
|
1,623,432
|
|
$
|
869,440
|
|
|
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|
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View source version on businesswire.com: http://www.businesswire.com/news/home/20180221006147/en/
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