- Continued strong commercial launch for
ZYNTEGLO® and SKYSONA®; 16 patient starts across both programs to
date –
- Biologics License Application (BLA) for
lovo-cel for sickle cell disease accepted for FDA priority review;
PDUFA goal date December 20, 2023 -
- Ended quarter with $291M in cash, cash
equivalents, marketable securities and restricted cash –
- Management to host conference call on Q2
earnings and commercial launch progress today August 8, 2023 at
8:00AM ET -
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today reported financial results and business highlights
for the second quarter ended June 30, 2023, including recent
commercial and operational progress, and regulatory updates.
"As we approach the anniversaries of the FDA approvals of
ZYNTEGLO and SKYSONA, we have continued to advance our commercial
strategy and prove the model for the gene therapy field on our path
to profitability,” said Andrew Obenshain, chief executive officer,
bluebird bio. “Additionally, with the ongoing FDA review of
lovo-cel and potential approval by the end of this year, bluebird
is preparing for our largest opportunity yet to impact the lives of
patients and families – a gene therapy for individuals living with
sickle cell disease in the US.”
RECENT HIGHLIGHTS
ZYNTEGLO® (betibeglogene autotemcel) Commercial
Launch
- bluebird continues to build on the launch of ZYNTEGLO for
beta-thalassemia. To date, there have been 11 patient starts for
ZYNTEGLO.
- To date, the Company has received zero ultimate denials from
commercial or government payers 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.
SKYSONA® (elivaldogene autotemcel) Commercial Launch
- Cell collection has been completed for 5 patients for SKYSONA
to date.
- Since approval, bluebird has activated four QTCs to administer
SKYSONA for patients with cerebral adrenoleukodystrophy
(CALD).
lovo-cel BLA Acceptance and Priority Review
- On June 21, 2023, bluebird bio announced that the FDA accepted
for priority review its BLA for lovotibeglogene autotemcel
(lovo-cel), the Company’s gene therapy for individuals living with
sickle cell disease (SCD). bluebird is pursuing FDA approval for
lovo-cel for patients ages 12 and older who have a history of
vaso-occlusive events (VOEs). The agency has set a Prescription
Drug User Fee Act (PDUFA) goal date of December 20, 2023.
lovo-cel ICER Review
- In its ongoing review of the cost-effectiveness of gene
therapies for sickle cell disease, the Institute for Clinical and
Economic Review (ICER) determined that lovo-cel will be cost
effective at a price up to $2.26 million when considering the
societal perspective. bluebird anticipates setting a price for
lovo-cel upon potential FDA approval.
UPCOMING ANTICIPATED
MILESTONES
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.
LOVO-CEL
- The FDA has set a PDUFA goal date for December 20, 2023, and if
approved, the Company anticipates commercial launch in early 2024.
bluebird estimates approximately 20,000 individuals living with SCD
(or one-fifth of the U.S. SCD population) may be eligible for gene
therapy.
SECOND QUARTER 2023 FINANCIAL
RESULTS
- Cash Position: The Company’s cash, cash equivalents,
marketable securities and restricted cash balance was approximately
$291 million, as of June 30, 2023. The Company anticipates
full-year 2023 cash burn 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, inclusive of revenue will be sufficient to meet
bluebird’s planned operating expenses and capital expenditure
requirements into the fourth quarter of 2024. Excluding $45 million
of restricted cash, which is currently unavailable for use,
bluebird estimates cash runway into the second quarter of 2024.
Please see our Quarterly Report on Form 10-Q for the quarter ended
June 30, 2023 for further information regarding our cash runway
guidance and other financial results.
- Revenue, net: Total revenue, net was $6.9 million for
the three months ended June 30, 2023, compared to $1.5 million for
the three months ended June 30, 2022. The increase of $5.4 million
was primarily due to SKYSONA and ZYNTEGLO product revenue.
- SG&A Expenses: Selling, general and administrative
expenses were $40.3 million for the three months ended June 30,
2023, compared to $36.7 million for the three months ended June 30,
2022. SG&A includes lease expense related to 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 $30.7 million for the three months ended June 30,
2023, compared to $28.2 million for the three months ended June 30,
2022. This increase is mainly attributable to commercial costs
driven by marketing activities for ZYNTEGLO and SKYSONA in the
United States and the performance of commercial readiness
activities in the United States for lovo-cel, in anticipation of
potential approval, as well as costs related to employee
compensation, benefits, and other head-count related expenses.
- R&D Expenses: Research and development expenses were
$42.3 million for the three months ended June 30, 2023, compared to
$63.8 million for the three months ended June 30, 2022. The
decrease of $21.5 million was primarily due to manufacturing costs
related to SKYSONA and ZYNTEGLO now being included in inventory and
cost of product revenue, as well as decreased employee
compensation, benefit and other headcount-related expenses and a
decrease in information technology and facility related costs in
2023.
- Net income (loss): Net loss was $72.9 million for the
three months ended June 30, 2023, compared to a net loss of $100.1
million for the three months ended June 30, 2022.
CONFERENCE CALL DETAILS
bluebird will hold a conference call to discuss second quarter
financial results and commercial launch progress on Tuesday, August
8 at 8:00 am ET.
To access the call via telephone, please register at this link
https://register.vevent.com/register/BI4fa1d86317c74333813f6827624e43ae
to receive a dial in number and unique PIN to access the live
conference call.
The live webcast of the call may be accessed by visiting the
“Events & Presentations” page within the Investors & Media
section of the bluebird website at http://investor.bluebirdbio.com.
A replay of the webcast will be available on the bluebird website
for 90 days following the event.
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 and is establishing the
commercial model for gene therapy—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 its QTC
network and timing thereof, plans for future regulatory
submissions, the expected timing for the potential PDUFA acceptance
and regulatory approval of lovo-cel by FDA, 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; 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; substantial doubt exists regarding our ability to continue
as a going concern; 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 any one or
more of our products or product candidates, including eli-cel,
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
June 30,
For the six months ended June
30,
2023
2022
2023
2022
Revenue:
Product revenue, net
$
6,837
$
1,331
$
9,133
$
2,739
Other revenue
53
188
138
725
Total revenues
6,890
1,519
9,271
3,464
Cost of product revenue
9,564
1,745
12,940
10,055
Gross margin
(2,674
)
(226
)
(3,669
)
(6,591
)
Operating expenses:
Selling, general and administrative
40,349
36,694
77,703
72,800
Research and development
42,274
63,841
88,418
141,716
Restructuring expenses
—
6,639
—
6,639
Total operating expenses
82,623
107,174
166,121
221,155
Gain from sale of priority review voucher,
net
—
—
92,930
—
Income (loss) from operations
(85,297
)
(107,400
)
(76,860
)
(227,746
)
Interest income, net
2,679
174
5,504
280
Other income (expense), net
9,630
7,088
19,608
5,176
Income (loss) before income taxes
(72,988
)
(100,138
)
(51,748
)
(222,290
)
Income tax (expense) benefit
80
—
80
—
Net income (loss)
$
(72,908
)
$
(100,138
)
$
(51,668
)
$
(222,290
)
Net income (loss) per share - basic
$
(0.67
)
$
(1.36
)
$
(0.49
)
$
(3.02
)
Net income (loss) per share - diluted
$
(0.67
)
$
(1.36
)
$
(0.49
)
$
(3.02
)
Weighted-average number of common shares
used in computing net income (loss) per share - basic:
108,685
73,767
105,819
73,727
Weighted-average number of common shares
used in computing net income (loss) per share - diluted:
108,685
73,767
105,819
73,727
Other comprehensive income (loss):
Other comprehensive income (loss), net of
tax benefit (expense) of $0.0 million for the three and six months
ended June 30, 2023 and 2022
722
43
1,706
(1,505
)
Total other comprehensive income
(loss)
722
43
1,706
(1,505
)
Comprehensive income (loss)
$
(72,186
)
$
(100,095
)
$
(49,962
)
$
(223,795
)
bluebird bio, Inc. Condensed
Consolidated Balance Sheet Data (in thousands)
(unaudited)
As of
June 30,
2023
As of
December 31,
2022
Cash, cash equivalents and marketable
securities
$
245,303
$
181,741
Restricted cash
$
45,302
$
45,439
Total assets
$
663,393
$
554,902
Total liabilities
$
374,374
$
358,559
Total stockholders’ equity
$
289,019
$
196,343
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230808850512/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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