- Biologics License Application (BLA) for
lovo-cel for sickle cell disease submitted to FDA -
- First commercial infusions completed for both
ZYNTEGLO® and SKYSONA® -
- Ended quarter with $364M in cash, cash
equivalents, marketable securities and restricted cash –
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today reported financial results and business highlights
for the first quarter ended March 31, 2023, including recent
commercial and operational progress, and regulatory updates.
"The first quarter of 2023 saw us continue to establish and
scale the commercial model for ex-vivo gene therapy in the US
through the launches of ZYNTEGLO and SKYSONA and lay the foundation
for revenue generation for years to come for bluebird," said Andrew
Obenshain, chief executive officer, bluebird bio. "Additionally,
with the submission of the lovo-cel BLA for sickle cell disease in
April, bluebird has taken a pivotal step towards realizing its most
significant opportunity yet -- bringing a transformative gene
therapy to individuals living with sickle cell disease."
RECENT HIGHLIGHTS
lovo-cel (lovotibeglogene autotemcel) BLA Submitted
- On April 24, 2023, bluebird bio announced it submitted its
Biologics License Application (BLA) to the U.S. Food and Drug
Administration (FDA) for lovo-cel for patients with sickle cell
disease (SCD) who are 12 years and older and have a history of
vaso-occlusive events. The Company has requested Priority Review,
which, if granted, would shorten the FDA’s review of the
application to six months from the time of filing, versus a
standard review timeline of 10 months.
ZYNTEGLO® (betibeglogene autotemcel) Commercial
Launch
- In the weeks since the Company’s last commercial update,
bluebird continues to build on the launch of ZYNTEGLO for
beta-thalassemia. To date, there have been six patient starts (cell
collections) for ZYNTEGLO.
- The first commercial ZYNTEGLO infusion has been completed and
the Company anticipates recognizing revenue in Q2 2023.
- Payer uptake continues to be positive and to date, the Company
has received zero ultimate denials for ZYNTEGLO; prior
authorization approvals for drug product remain consistent at
approximately two weeks.
- As previously communicated, patient starts remain the key
commercial metric during the first year of the ZYNTEGLO launch.
bluebird does not expect to provide ZYNTEGLO revenue projections
for 2023.
SKYSONA® (elivaldogene autotemcel) Commercial Launch
- First commercial infusion with SKYSONA was completed in March
2023. In total, cell collection has been completed for three
patients.
- Since approval, bluebird has activated three qualified
treatment centers (QTCs) to administer SKYSONA for patients with
cerebral adrenoleukodystrophy (CALD). Two additional QTCs on the
West Coast are anticipated in 2023.
UPCOMING ANTICIPATED
MILESTONES
LOVO-CEL
- The Company anticipates BLA acceptance of lovo-cel for SCD in
Q2 2023.
- The Company continues to anticipate commercial launch in early
2024, if approved. bluebird estimates approximately 20,000
individuals living with sickle cell disease (or 1/5 of the SCD
population in the US) may be eligible for gene therapy.
ZYNTEGLO
- The Company is on track to scale to 40-50 QTCs by the end of
2023. bluebird’s QTC network is designed to maximize its commercial
opportunity in beta-thalassemia and to prioritize proximity to
individuals with living with SCD in anticipation of a 2024
commercial launch for lovo-cel, if approved by the FDA.
SKYSONA
- The Company continues to anticipate 5-10 patient starts this
year as previously guided.
FIRST QUARTER 2023 FINANCIAL
RESULTS
- Cash Position: The Company’s cash, cash equivalents,
marketable securities and restricted cash balance was approximately
$364 million, as of March 31, 2023. As bluebird bio launches two
first-in-class gene therapies and readies its third investigational
gene therapy for SCD for the commercial setting, full-year 2023
cash burn is expected to be in the range of $270-$300 million, as
previously guided. Based on current operating plans, bluebird
expects its cash, cash equivalents, marketable securities and
restricted cash will be sufficient to meet bluebird’s planned
operating expenses and capital expenditure requirements into the
fourth quarter of 2024. This runway includes approximately $45
million of restricted cash, which is currently unavailable for use.
Please see our Quarterly Report on Form 10-Q for the quarter ended
March 31, 2023 for further information regarding our cash runway
guidance and other financial results.
- Revenue, net: Total revenue, net was $2.4 million for
the three months ended March 31, 2023, compared to $1.9 million for
the three months ended March 31, 2022. The increase of $0.4 million
was primarily due to SKYSONA product revenue.
- SG&A Expenses: Selling, general and administrative
expenses were $37.4 million for the three months ended March 31,
2023, compared to $36.1 million for the three months ended March
31, 2022. SG&A includes lease expense related to the 50 Binney
Street; however, sublease income is presented in other income
(expense), net. Excluding the lease expense for 50 Binney St.,
SG&A expenses were $27.8 million for the three months ended
March 31, 2023, compared to $36.1 million for the three months
ended March 31, 2022. This decrease is mainly attributable to costs
related to employee compensation, benefits, and other head-count
related expenses.
- R&D Expenses: Research and development expenses were
$46.1 million for the three months ended March 31, 2023, compared
to $77.9 million for the three months ended March 31, 2022. The
decrease of $31.7 million was primarily due to decreased employee
compensation, benefit and other headcount-related expenses and a
decrease in R&D production costs in 2023.
- Gain from sale of priority review voucher: Operating
expenses were offset by the one-time gain from sale of the
Company’s second priority review voucher, for $92.9 million, net,
in the first quarter of 2023.
- Net income (loss): Net income was $21.2 million for the
three months ended March 31, 2023, compared to a net loss of $122.2
million for the three months ended March 31, 2022.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days.
With a dedicated focus on severe genetic diseases, bluebird has
industry-leading programs for sickle cell disease, β-thalassemia
and cerebral adrenoleukodystrophy and is advancing research to
apply new technologies to these and other diseases. We custom
design each of our therapies to address the underlying cause of
disease and have developed in-depth and effective analytical
methods to understand the safety of our lentiviral vector
technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo
gene therapy data set in the world—setting the standard for the
industry. Today, bluebird continues to forge new paths, combining
our real-world experience with a deep commitment to patient
communities and a people-centric culture that attracts and grows a
diverse flock of dedicated birds.
bluebird bio, ZYNTEGLO and SKYSONA are registered trademarks of
bluebird bio, Inc. All rights reserved.
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements that are not statements of historical facts
are, or may be deemed to be, forward-looking statements, including
our statements regarding the Company’s financial condition, results
of operations, commercial revenue and key metrics, including the
expected number of patient starts, and anticipated reporting and
timing thereof; anticipated cash runway, including restricted cash;
and anticipated cash burn for 2023 as well as statements regarding
the Company’s plans and expectations for operations including
expected timing relating to its regulatory approvals, plans to
expand manufacturing capacity, anticipated growth of our QTC
network and timing thereof, plans for future regulatory
submissions, our expectations regarding the possible approval of
lovo-cel by FDA, the expected timing relating to the potential
acceptance and regulatory approval of our BLA for lovo-cel and the
fact that a potential granting of Priority Review may shorten the
FDA’s review of the BLA to six months, and the timing of commercial
launch of lovo-cel, if approved. Such forward-looking statements
are based on historical performance and current expectations and
projections about our future financial results, goals, plans and
objectives and involve inherent risks, assumptions and
uncertainties, including internal or external factors that could
delay, divert or change any of them in the next several years, that
are difficult to predict, may be beyond our control and could cause
our future financial results, goals, plans and objectives to differ
materially from those expressed in, or implied by, the statements.
No forward-looking statement can be guaranteed. Forward-looking
statements in this press release should be evaluated together with
the many risks and uncertainties that affect bluebird bio’s
business, particularly those identified in the risk factors
discussion in bluebird bio’s Annual Report on Form 10-K, as updated
by our subsequent Quarterly Reports on Form 10-Q, Current Reports
on Form 8-K and other filings with the Securities and Exchange
Commission. These risks include, but are not limited to: delays and
challenges in our commercialization and manufacturing of our
products, including risks associates with demonstrating analytical
comparability with respect to our lovo-cel program; the risk that
we may not realize expected cost savings from the restructuring,
including the anticipated decrease in operational expenses, at the
levels we expect; we may encounter additional delays in the
development of our programs, including the imposition of new
clinical holds, that may impact our ability to meet our expected
timelines and increase our costs; the internal and external costs
required for our ongoing and planned activities, and the resulting
impact on expense and use of cash, has been, and may in the future
be, higher than expected which has caused us, and may in the future
cause us to use cash more quickly than we expect or change or
curtail some of our plans or both; our expectations as to expenses,
cash usage and cash needs may prove not to be correct for other
reasons such as changes in plans or actual events being different
than our assumptions; the risk that the efficacy and safety results
from our prior and ongoing clinical trials will not continue or be
seen in additional patients treated with our product candidates;
the risk that additional insertional oncogenic or other reportable
events associated with lentiviral vector, drug product, or
myeloablation will be discovered or reported over time; the risk
that our eli-cel, beti-cel and lovo-cel programs may be subject to
further delays in their development, including but not limited to
the imposition of new clinical holds; the risk that any one or more
of our products or product candidates, including eli-cel and,
beti-cel or lovo-cel, will not be successfully developed, approved
or commercialized, as applicable. The forward-looking statements
included in this document are made only as of the date of this
document and except as otherwise required by applicable law,
bluebird bio undertakes no obligation to publicly update or revise
any forward-looking statement, whether as a result of new
information, future events, changed circumstances or otherwise.
bluebird bio, Inc.
Condensed Consolidated
Statements of Operations
(in thousands, except per
share data)
(unaudited)
For the three months ended
March 31,
2023
2022
Revenue:
Product revenue, net
$2,296
$1,408
Other revenue
85
537
Total revenues
2,381
1,945
Cost of product revenue
3,376
8,310
Gross margin
(995)
(6,365)
Operating expenses:
Selling, general and administrative
37,354
36,106
Research and development
46,144
77,875
Total operating expenses
83,498
113,981
Gain from sale of priority review voucher,
net
92,930
—
Income (loss) from operations
8,437
(120,346)
Interest income, net
2,825
106
Other income (expense), net
9,978
(1,912)
Income (loss) before income taxes
21,240
(122,152)
Income tax (expense) benefit
—
—
Net income (loss)
$21,240
$(122,152)
Net income (loss) per share - basic
$0.21
$(1.66)
Net income (loss) per share - diluted
$0.21
$—
Weighted-average number of common shares
used in computing net income (loss) per share - basic:
102,920
73,688
Weighted-average number of common shares
used in computing net income (loss) per share - diluted:
103,303
—
Other comprehensive income (loss):
Other comprehensive income (loss), net of
tax benefit (expense) of $0.0 million for the three months ended
March 31, 2023 and 2022
984
(1,548)
Total other comprehensive income
(loss)
984
(1,548)
Comprehensive income (loss)
$22,224
$(123,700)
bluebird bio, Inc.
Condensed Consolidated Balance
Sheet Data
(in thousands)
(unaudited)
As of March 31,
2023
As of December 31,
2022
Cash, cash equivalents and marketable
securities
$
318,257
$
181,741
Restricted cash
$
45,354
$
45,439
Total assets
$
692,736
$
554,902
Total liabilities
$
337,996
$
358,559
Total stockholders’ equity
$
354,740
$
196,343
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230509005510/en/
Investors & Media Investors: Courtney O’Leary,
978-621-7347 coleary@bluebirdbio.com Media: Jess Rowlands,
857-299-6103 Jess.rowlands@bluebirdbio.com
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