- Continued progress towards 2022 vision of
four marketed gene and cell therapy products with robust
development pipeline -
- ZYNTEGLO commercial launch advancing with
European Medicines Agency approval of refined commercial
manufacturing process -
- Ended quarter with $1.41 billion in cash,
cash equivalents and marketable securities –
bluebird bio, Inc. (NASDAQ: BLUE) today reported financial
results and business highlights for the third quarter ended
September 30, 2019.
“During the third quarter we advanced our country-by-country
launch plans in Europe and, with the recent approval of the
commercial drug product manufacturing specifications for ZYNTEGLO,
we moved one step closer to our goal of treating patients suffering
from TDT in early 2020,” said Nick Leschly, chief bluebird. “Also
this quarter, we presented updated data from the Phase 2/3 Starbeam
study in patients with CALD. To report that patients continued to
be free of MFDs at up to five years of follow-up is something we’re
tremendously proud to do for these families, and we look forward to
advancing that program in the regulatory process next year. Looking
ahead, we plan to provide clinical updates for ZYNTEGLO and across
the rest of our portfolio, including LentiGlobin in sickle cell
disease, bb21217 in multiple myeloma, and from our
registration-enabling KarMMa study of ide-cel in patients with
multiple myeloma by the end of this year. I’d like to thank all the
bluebirds around the globe for their tireless focus on doing the
right thing for our patients – we’ve seen amazing progress thus far
in 2019 and I look forward to ending the year on a strong
note.”
Recent Highlights:
TDT
- ZYNTEGLO COMMERCIAL READINESS – In October, bluebird bio
announced that the European Medicines Agency (EMA) approved the
refined commercial drug product manufacturing specifications for
ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a
one-time 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. With this update, apceth is in the final
stages of preparing to manufacture ZYNTEGLO for commercial use. The
company continues to proceed with discussions on value-based
payment agreements and Qualified Treatment Center contracts and
expects to treat the first commercial patient in early 2020.
CALD
- DATA FROM STARBEAM STUDY (ALD-102) AND ALD-103 PRESENTED
– At the 13th European Pediatric Neurology Society (EPNS) Congress
in September 2019, bluebird bio presented new data from the
clinical development program for its investigational studies of
Lenti-D™ gene therapy in patients with cerebral
adrenoleukodystrophy: updated data from the Phase 2/3 Starbeam
study (ALD-102) in boys 17 years of age and under with CALD and
updated data from the ongoing observational study (ALD-103) of
allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys
17 years of age and under with CALD. Long-term follow-up data as of
April 2019 showed that the 88% of patients treated in the Starbeam
study (ALD-102) were free of major functional disabilities (MFDs)
at two years, and continued to remain MFD-free at up to five years
of follow-up.
COMPANY
- FIRST PATIENT TREATED IN PHASE 1/2 TRIAL FOR MERKEL CELL
CARCINOMA (MCC) – In August 2019, Fred Hutchinson Cancer
Research Center infused the first patient in their proof-of-concept
phase 1/2 single-arm study evaluating Merkel Cell Polyomavirus
(MCPyV) TCR-engineered autologous T cells in combination with
avelumab (anti-PDL1) for the treatment of MCC. Results from the
academic phase 1/2 single-arm study are expected to inform
next-generation T cell approaches including TCR engineering and
checkpoint inhibition. The study will enroll approximately 16
patients. Development of this program is led by Fred Hutchinson
Cancer Research Center. bluebird bio retains the exclusive option
to license this program.
- NOVO NORDISK COLLABORATION – In October 2019, bluebird
bio and Novo Nordisk announced a research collaboration to jointly
develop next-generation in vivo genome editing treatments for
genetic diseases, including hemophilia. During the three-year
research collaboration, bluebird and Novo Nordisk will focus on
identifying a development gene therapy candidate with the ambition
of offering people with hemophilia A a lifetime free of factor
replacement therapy.
- MANAGEMENT UPDATE – In October 2019, bluebird bio
announced that Jeffrey T. Walsh, chief strategy officer, has
decided to transition from his current role effective January 6,
2020. Jeff has not only built a strong foundation for bluebird’s
overall growth strategy but also leaves an experienced and
passionate team. Both Chip Baird, chief financial officer, and
Joanne Smith-Farrell, chief business officer, will assume broader
corporate development and strategic responsibilities as bluebird
continues to deliver on its mission for patients.
- NEW BOARD APPOINTMENT – In September 2019, bluebird bio
announced the appointment of William R. Sellers, M.D. to its Board
of Directors.
Upcoming Anticipated Milestones:
- TDT
- Initiation of a rolling Biologics Licensing Application
submission 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 the end of 2019
- Presentation of ZYNTEGLO clinical data from the Northstar-3
(HGB-212) clinical study in patients with TDT and a β0/β0 genotype
or an IVS-I-110 mutation by the end of 2019
- SCD
- Phase 3 HGB-210 study of LentiGlobin in patients with SCD open
and enrolling by the end of 2019
- Presentation of LentiGlobin clinical data from the HGB-206
clinical study in patients with SCD by the end of 2019
- Multiple Myeloma
- Ide-cel clinical data update from the registration-enabling
KarMMa study in patients with relapsed/refractory multiple myeloma
by the 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
Third Quarter 2019 Financial Results
- Cash Position: Cash, cash equivalents and marketable
securities as of September 30, 2019 and December 31, 2018 were
$1.41 billion and $1.89 billion, respectively. The decrease in
cash, cash equivalents and marketable securities is primarily
related to cash used in support of ordinary course operating
activities and cash used to purchase property, plant and equipment,
including those purchases related to the company’s buildout of its
manufacturing facility in Durham, North Carolina.
- Revenues: Collaboration and license and royalty revenues
were $8.9 million for the three months ended September 30, 2019
compared to $11.5 million for the three months ended September 30,
2018. Collaboration and license and royalty revenues were $34.7
million for the nine months ended September 30, 2019 compared to
$35.3 million for the nine months ended September 30, 2018. The
decrease in both periods was primarily attributable to a decrease
in collaboration revenue under our arrangement with Celgene,
partially offset by an increase in license and royalty revenue and
collaboration revenue under our arrangement with Regeneron.
- R&D Expenses: Research and development expenses were
$151.4 million for the three months ended September 30, 2019
compared to $116.7 million for the three months ended September 30,
2018. Research and development expenses were $420.6 million for the
nine months ended September 30, 2019 compared to $328.9 million for
the nine months ended September 30, 2018. The increase in both
periods was primarily driven by costs incurred to advance and
expand the company’s pipeline.
- G&A Expenses: General and administrative expenses
were $66.3 million for the three months ended September 30, 2019
compared to $44.5 million for the three months ended September 30,
2018. General and administrative expenses were $195.2 million for
the nine months ended September 30, 2019 compared to $120.6 million
for the nine months ended September 30, 2018. The increase in both
periods was largely attributable to costs incurred to support the
company’s ongoing operations and growth of its pipeline as well as
commercial-readiness activities.
- Net Loss: Net loss was $206.0 million for the three
months ended September 30, 2019 compared to $145.5 million for the
three months ended September 30, 2018. Net loss was $566.3 million
for the nine months ended September 30, 2019 compared to $406.6
million for the nine months ended September 30, 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, LentiGlobin and Lenti-D 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 βA-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 plans for regulatory
submissions and commercialization for ZYNTEGLO and the company’s
product candidates, including anticipated regulatory milestones,
planned 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 ZYNTEGLO and
the company’s 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 will not continue or be repeated in our
ongoing or future 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-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.
bluebird bio, Inc.
Consolidated Statements of Operations (in thousands, except per
share data) (unaudited)
For the three months ended
September 30,
For the nine months ended
September 30,
2019
2018
2019
2018
Revenue:
Collaboration revenue
$
6,575
$
10,926
$
29,310
$
33,971
License and royalty revenue
2,335
602
5,367
1,365
Total revenues
8,910
11,528
34,677
35,336
Operating expenses:
Research and development
151,412
116,744
420,592
328,867
General and administrative
66,250
44,527
195,160
120,621
Cost of license and royalty revenue
862
29
1,905
67
Change in fair value of contingent
consideration
802
47
1,312
843
Total operating expenses
219,326
161,347
618,969
450,398
Loss from operations
(210,416
)
(149,819
)
(584,292
)
(415,062
)
Interest income, net
8,417
4,591
27,906
8,415
Other (expense) income, net
(4,298
)
(252
)
(10,623
)
45
Loss before income taxes
(206,297
)
(145,480
)
(567,009
)
(406,602
)
Income tax benefit
264
—
748
—
Net loss
$
(206,033
)
$
(145,480
)
$
(566,261
)
$
(406,602
)
Net loss per share - basic and
diluted:
$
(3.73
)
$
(2.73
)
$
(10.27
)
$
(7.95
)
Weighted-average number of common shares
used in computing net loss per share - basic and diluted:
55,292
53,277
55,139
51,130
bluebird bio, Inc. Condensed
Consolidated Balance Sheet Data (in thousands, except per share
data) (unaudited)
As of
As of
September 30,
December 31,
2019
2018
Cash, cash equivalents and marketable
securities
$
1,405,887
$
1,891,427
Total assets
1,892,218
2,242,844
Total liabilities
420,508
357,774
Total stockholders' equity
1,471,710
1,885,070
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191031005885/en/
Investors & Media Investors: bluebird bio Elizabeth
Pingpank, 617-914-8736 epingpank@bluebirdbio.com Media: bluebird
bio Jenn Snyder, 617-448-0281 jsnyder@bluebirdbio.com
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