-- Treated first patient in CRB-401 study of anti-BCMA CAR T
bb2121 in relapsed/refractory multiple myeloma --
-- Announced that Celgene has exercised its option to license
bb2121 --
-- Presented LentiGlobin clinical data in transfusion-dependent
ß-thalassemia and severe sickle cell disease at the American Society of
Hematology (ASH) annual meeting --
-- Presented pre-clinical and manufacturing data from oncology
pipeline at ASH annual meeting --
-- Signed exclusive license agreement with Viromed for solid tumor
CAR T target --
-- Ended year with $865.8 million in cash, cash equivalents, and
marketable securities --
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, 2015.
“In 2015 bluebird bio defined an accelerated regulatory path for
LentiGlobin in ß-thalassemia and established a powerful reason to
believe in LentiGlobin in sickle cell disease, though there is still
more to learn as we treat additional patients. We also fully enrolled
our Starbeam study of Lenti-D in cerebral adrenoleukodystrophy and made
significant advances in building a competitive T cell oncology
franchise,” said Nick Leschly, chief bluebird. “In 2016 we are excited
to learn even more across all of our programs and continue to innovate
and improve. We are particularly looking forward to sharing data from
the Starbeam study for the first time and presenting more data with
longer follow-up from all three of our LentiGlobin clinical studies.”
Recent Highlights
-
ADVANCED FIRST ONCOLOGY PROGRAM INTO THE CLINIC – Earlier in
February, the first patient was infused in the CRB-401 study of bb2121
in relapsed/refractory multiple myeloma. Additionally, Celgene has
exercised its option to exclusively license bb2121, under the terms of
the collaboration agreement between the two companies. bluebird bio
will receive a $10.0 million option exercise payment from Celgene and
is also eligible to receive specified development and regulatory
milestone payments and royalty payments on net sales.
-
PRESENTED UPDATED CLINICAL DATA IN ß-THALASSEMIA FROM HGB-204 AND
HGB-205 CLINICAL STUDIES OF LENTIGLOBIN AT ASH – In December 2015,
investigators presented data from bluebird bio’s ongoing clinical
studies in transfusion-dependent ß-thalassemia (TDT) and severe sickle
cell disease (SCD). Data in patients with TDT from the HGB-204 and
HGB-205 studies showed that 100% of patients with non-ß0/ß0 genotypes
achieved sustained transfusion independence as of the data cut-off,
ranging from 7.1 months to 23.4 months. Patients with the ß0/ß0
genotype all saw reductions in their transfusion needs, ranging from a
33% to 100% reduction.
-
PRESENTED CLINICAL DATA IN SICKLE CELL DISEASE FROM HGB-205 AND
HGB-206 CLINICAL STUDIES OF LENTIGLOBIN AT ASH –Marina Cavazzana,
M.D., Ph.D., of Hospital Necker, University Paris Descartes, presented
updated data on one patient with SCD from the HGB-205 study, who
remained free of transfusions, hospitalizations and acute SCD-related
events for more than nine months as of the data cut-off. At the
12-month post-drug infusion follow-up, the proportion of anti-sickling
hemoglobin in this patient accounted for 49% (47% HbAT87Q +
2% HbF) of all hemoglobin production – well above the 30% threshold
anticipated to achieve a disease-modifying effect. John Tisdale, M.D.,
of the National Institutes of Health presented early data from the
HGB-206 study, in which two patients had at least three months of
post-infusion follow-up. At the three-month post-infusion follow-up
for Subject 1301, anti-sickling hemoglobin accounted for 17% of all
hemoglobin production (4% HbAT87Q + 13% HbF). At the
six-month post-infusion follow-up for subject 1303, anti-sickling
hemoglobin accounted for 16 percent of all hemoglobin production (12%
HbAT87Q + 4% HbF).
-
PRESENTED PRE-CLINICAL AND MANUFACTURING DATA FROM CAR T ONCOLOGY
PROGRAMS AT ASH – bluebird bio scientists presented three posters
at ASH, covering critical basic research, translational and
manufacturing aspects of the Company’s T cell oncology programs. One
poster discussed an important observation made by bluebird bio
scientists: culturing anti-BCMA CAR T cells with a PI3K inhibitor
generated a product with many of the properties of younger, less
differentiated T cells. Consistent with a younger T cell phenotype,
this product showed improved in vivo efficacy and persistence
in multiple model systems.
-
SHARED DATA ON PLATFORM IMPROVEMENTS – In an investor event at
ASH, bluebird bio chief scientific officer Philip Gregory, D.Phil.,
presented data from ongoing research to improve the cell transduction
process for LentiGlobin. The presentation showed that in preclinical
experiments, adding selected compounds to the transduction process
resulted in substantially increased vector copy number and
transduction efficiency (i.e. percentage of corrected cells).
Importantly, the new process was shown to be robust with similar
improvements seen across multiple donors and vector lots.
-
ENTERED INTO CAR T LICENSE WITH VIROMED – Signed exclusive
license agreement with Viromed Co., Ltd., to research, develop and
commercialize CAR T therapies using Viromed’s proprietary humanized
antibody to an undisclosed cancer target in solid tumors.
Upcoming Anticipated Milestones
-
Presentation of interim data from the Starbeam study of Lenti-D in
patients with cerebral adrenoleukodystrophy (CALD) at the American
Academy of Neurology annual meeting in April 2016
-
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 data from the HGB-204, HGB-205 and HGB-206
studies at the ASH annual meeting in December 2016
Fourth Quarter and Full Year 2015 Financial Results and Financial
Guidance
-
Cash Position: Cash, cash equivalents and marketable securities
as of December 31, 2015 were $865.8 million, compared to $492.0
million as of December 31, 2014, an increase of $373.8 million, which
was primarily driven by the June 2015 equity financing partially
offset by cash used to fund operations.
-
Revenues: Collaboration revenue was $1.5 million for the fourth
quarter of 2015 and $14.1 million for the year ended December 31,
2015, compared to $6.3 million and $25.0 million in the comparable
periods in 2014. 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 $35.7
million for the fourth quarter of 2015 and $134.0 million for the year
ended December 31, 2015, compared to $20.5 million and $62.6 million
for the comparable periods in 2014. The increase in research and
development expenses was primarily attributable to increased employee
compensation expense due to increased headcount, in-licensing
milestones and fees, and manufacturing and clinical trial-related
costs to support our advancing pipeline.
-
G&A Expenses: General and administrative expenses were
$14.4 million for the fourth quarter of 2015 and $46.2 million for the
year ended December 31, 2015, compared to $5.3 million and $23.2
million for the comparable periods in 2014. The increase in general
and administrative expenses was primarily attributable to increased
employee compensation expense due to increased headcount, and
consulting and facilities-related costs to support our overall growth.
-
Net Loss: Net loss was $47.3 million for the fourth quarter of
2015 and $166.8 million for the year ended December 31, 2015, compared
to net loss of $19.5 million and $48.7 million for the comparable
periods in 2014.
-
Financial guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of $865.8 million as of December
31, 2015 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, 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
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, the risk of cessation or delay of any of the
ongoing or planned clinical studies or development activities for our
product candidates, the risk of a delay in the enrollment of patients in
the Company’s clinical studies, the risk that the results of previously
conducted studies involving similar product candidates will not be
repeated or observed in ongoing or future studies involving current
product candidates, the risk that our collaborations 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 and
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 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. 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.
Availability of other information about bluebird bio
Investors
and others should note that we communicate with our investors and the
public using our company website (www.bluebirdbio.com),
including but not limited to investor presentations and FAQs, Securities
and Exchange Commission filings, press releases, public conference calls
and webcasts. You can also connect with us on Twitter @bluebirdbio,
LinkedIn
or our YouTube
channel. The information that we post on these channels and websites
could be deemed to be material information. As a result, we encourage
investors, the media, and others interested in bluebird bio to review
the information that we post on these channels, including our investor
relations website, on a regular basis. This list of channels may be
updated from time to time on our investor relations website and may
include other social media channels than the ones described above. The
contents of our website or these channels, or any other website that may
be accessed from our website or these channels, shall not be deemed
incorporated by reference in any filing under the Securities Act of 1933.
|
bluebird bio, Inc. Consolidated Statements of
Operations Data (unaudited) (in thousands,
except per share data)
|
|
|
|
|
|
|
|
|
|
|
Three months ended December 31,
|
|
|
Year ended December 31,
|
|
|
|
|
|
|
|
|
2015
|
|
|
2014
|
|
|
2015
|
|
|
2014
|
Revenue:
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue
|
|
|
$
|
1,471
|
|
|
$
|
6,281
|
|
|
$
|
14,079
|
|
|
$
|
25,031
|
Research and license fees
|
|
|
|
—
|
|
|
|
105
|
|
|
|
—
|
|
|
|
390
|
Total revenue
|
|
|
|
1,471
|
|
|
|
6,386
|
|
|
|
14,079
|
|
|
|
25,421
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
|
35,659
|
|
|
|
20,530
|
|
|
|
134,038
|
|
|
|
62,574
|
General and administrative
|
|
|
|
14,444
|
|
|
|
5,302
|
|
|
|
46,209
|
|
|
|
23,227
|
Change in fair value of contingent consideration
|
|
|
|
329
|
|
|
|
168
|
|
|
|
2,869
|
|
|
|
246
|
Total operating expenses
|
|
|
|
50,432
|
|
|
|
26,000
|
|
|
|
183,116
|
|
|
|
86,047
|
Loss from operations
|
|
|
|
(48,961)
|
|
|
|
(19,614)
|
|
|
|
(169,037)
|
|
|
|
(60,626)
|
Total other income (expense), net
|
|
|
|
1,684
|
|
|
|
70
|
|
|
|
2,314
|
|
|
|
120
|
Loss before income taxes
|
|
|
|
(47,277)
|
|
|
|
(19,544)
|
|
|
|
(166,723)
|
|
|
|
(60,506)
|
Income tax (expense) benefit
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(60)
|
|
|
|
11,797
|
Net loss
|
|
|
$
|
(47,277)
|
|
|
$
|
(19,544)
|
|
|
$
|
(166,783)
|
|
|
$
|
(48,709)
|
Net loss per share - basic and diluted:
|
|
|
$
|
(1.29)
|
|
|
$
|
(0.67)
|
|
|
$
|
(4.81)
|
|
|
$
|
(1.83)
|
Weighted-average number of common shares used in computing net loss
per share - basic and diluted:
|
|
|
|
36,716
|
|
|
|
29,373
|
|
|
|
34,669
|
|
|
|
26,546
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
bluebird bio, Inc. Consolidated Balance Sheets Data (unaudited) (in
thousands)
|
|
|
|
|
|
|
|
|
|
|
December 31,
|
|
|
December 31,
|
|
|
|
2015
|
|
|
2014
|
Assets
|
|
|
|
|
|
|
Current assets:
|
|
|
|
|
|
|
Cash and cash equivalents
|
|
|
$
|
164,269
|
|
|
$
|
347,845
|
Marketable securities
|
|
|
|
353,680
|
|
|
|
125,710
|
Prepaid expenses and other current assets
|
|
|
|
6,016
|
|
|
|
6,434
|
Total current assets
|
|
|
|
523,965
|
|
|
|
479,989
|
Marketable securities
|
|
|
|
347,814
|
|
|
|
18,448
|
Property and equipment, net
|
|
|
|
82,614
|
|
|
|
15,740
|
Intangible assets, net
|
|
|
|
24,456
|
|
|
|
28,219
|
Goodwill
|
|
|
|
13,128
|
|
|
|
13,128
|
Restricted cash and other non-current assets
|
|
|
|
10,360
|
|
|
|
1,215
|
Total assets
|
|
|
$
|
1,002,337
|
|
|
$
|
556,739
|
Liabilities and stockholders' equity
|
|
|
|
|
|
|
Current liabilities:
|
|
|
|
|
|
|
Accounts payable
|
|
|
$
|
6,334
|
|
|
$
|
2,954
|
Accrued expenses and other current liabilities
|
|
|
|
28,145
|
|
|
|
14,649
|
Deferred revenue, current portion
|
|
|
|
5,889
|
|
|
|
25,375
|
Total current liabilities
|
|
|
|
40,368
|
|
|
|
42,978
|
Deferred rent, net of current portion
|
|
|
|
8,294
|
|
|
|
8,674
|
Deferred revenue, net of current portion
|
|
|
|
35,959
|
|
|
|
5,302
|
Contingent consideration, net of current portion
|
|
|
|
5,082
|
|
|
|
6,321
|
Construction financing lease obligation
|
|
|
|
61,901
|
|
|
|
—
|
Other non-current liabilities
|
|
|
|
237
|
|
|
|
2,207
|
Total liabilities
|
|
|
|
151,841
|
|
|
|
65,482
|
Stockholders' equity:
|
|
|
|
|
|
|
Common stock, $0.01 par value, 125,000 shares authorized; 36,894
and 32,340 shares issued and outstanding at December 31, 2015
and December 31, 2014, respectively
|
|
|
|
369
|
|
|
|
323
|
Additional paid-in capital
|
|
|
|
1,166,585
|
|
|
|
638,389
|
Accumulated other comprehensive income (loss)
|
|
|
|
(2,291)
|
|
|
|
(71)
|
Accumulated deficit
|
|
|
|
(314,167)
|
|
|
|
(147,384)
|
Total stockholders' equity
|
|
|
|
850,496
|
|
|
|
491,257
|
Total liabilities and stockholders' equity
|
|
|
$
|
1,002,337
|
|
|
$
|
556,739
|
|
|
|
|
|
|
|
|
|
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