- BLAs for beti-cel for β-thalassemia and
eli-cel for cerebral adrenoleukodystrophy, to be discussed at an
FDA Advisory Committee Meeting on June 9-10, 2022 -
- Company restructuring initiated in April to
reduce operating costs by 35 to 40 percent by year-end 2022 -
- Ended quarter with $312M in restricted cash,
cash and cash equivalents and marketable securities -
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today reported financial results and business highlights
for the first quarter ended March 31, 2022, and shared recent
operational progress.
“For more than a decade, bluebird bio has helped chart the path
for the field of gene therapy and today, we are closer than ever
before to realizing our mission of bringing potentially curative
gene therapies to patients and their families,” said Andrew
Obenshain, chief executive officer, bluebird bio. “We have taken
action to sharpen our focus and our finances, as the FDA completes
its review of the beti-cel and eli-cel BLAs, and we prepare for the
launch of two gene therapies in the second half of the year. In
parallel, we are actively collecting the manufacturing
comparability data required for lovo-cel and remain focused on
submitting our BLA for sickle cell disease to the FDA in Q1
2023.”
RECENT HIGHLIGHTS
BETI-CEL AND ELI-CEL
- ADVISORY COMMITTEE MEETING ON JUNE 9-10, 2022 – On April
13, the U.S Food and Drug Administration (FDA) confirmed its plans
to review betibeglogene autotemcel (beti-cel) and elivaldogene
autotemcel (eli-cel) in an FDA Cellular, Tissue and Gene Therapies
Advisory Committee Meeting that will take place over two days on
June 9-10, 2022. The advisory committee will discuss the efficacy
and safety data supporting the benefit/risk of beti-cel and eli-cel
respectively, as well as safety information relevant to both
therapies. If approved, beti-cel and eli-cel would be the Company’s
first FDA approved therapies as an independent severe genetic
disease company. An overview of the agenda was included in the
Federal Register announcement posted on April 13. The full agenda
will be determined by the FDA and included in briefing materials
that will be posted by the FDA closer to the date of the advisory
committee meeting.
LOVO-CEL
- CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio
has enrolled more than half of the patients in the HGB-210 study
that will be needed to support manufacturing data requirements for
the lovotibeglogene autotemcel (lovo-cel) biologics licensing
application (BLA) and the Company is on track to submit a BLA to
the FDA for sickle cell disease (SCD) in the first quarter of 2023.
As previously communicated, the Company has treated all patients in
HGB-206 Group C who will form the primary basis of efficacy for BLA
submission, with the demonstration of analytical comparability and
validation of the commercial manufacturing process as the key
remaining actions prior to submission of the planned BLA.
Enrollment and dosing for patients 18 and older are continuing in
the HGB-210 study and the Company remains in active dialogue with
the FDA about the resolution of the partial clinical hold for
patients under 18.
COMPANY
- RESTRUCTURING UPDATE – On April 5, 2022, bluebird bio
announced a comprehensive restructuring intended to deliver up to
$160 million in cost savings over the next two years, reduce its
workforce by approximately 30% and extend its cash runway into the
first half of 2023. Near-term cost savings were realized in April
as bluebird began implementing the restructuring. The Company
continues to evaluate additional financing options, including
public or private equity financings and monetizing any priority
review vouchers that may be issued upon approval of beti-cel or
eli-cel.
- NEW HEADQUARTERS MOVE –bluebird bio has begun
transitioning to its new headquarters in Assembly Row. The
Company’s new HQ is designed to reflect modern ways of working and
estimated to result in more than $120 million in cost savings over
the next six years. bluebird will maintain laboratory space and
operations at 60 Binney St. in Cambridge through 2023.
UPCOMING INVESTOR EVENTS
Members of the management team will participate in the following
upcoming investor conferences:
- BofA Securities 2022 Health Care Conference, Wednesday, May 11,
at 3:20 p.m. PT at the Encore Hotel, Las Vegas, NV
- 2022 RBC Capital Markets Global Healthcare Conference, Tuesday,
May 17, at 8:00 a.m. ET at the Intercontinental NY Barclay, New
York, NY
- Goldman Sachs 43rd Annual Global Healthcare Conference,
Wednesday, June 15, at 4:00 p.m. PT at the Terranea Resort, Rancho
Palos Verdes, CA
To access the live webcast of bluebird bio’s presentations,
please visit the “Events & Presentations” page within the
Investors & Media section of the bluebird bio website at
http://investor.bluebirdbio.com. A replay of the webcasts will be
available on the bluebird bio website for 90 days following the
event.
UPCOMING ANTICIPATED
MILESTONES
LOVO-CEL
- The Company is in active communication with the FDA to resolve
the partial clinical hold and resume enrollment and treatment of
patients under the age of 18.
- The Company plans to complete manufacturing of commercial drug
product validation lots by mid-2022.
- The Company expects to confirm vector and drug product
analytical comparability by Q4 2022.
- The Company plans to submit its BLA for lovo-cel in Q1
2023.
BETI-CEL
- The FDA has set a PDUFA goal date of August 19, 2022, for a
decision on the approval of beti-cel in patients with β-thalassemia
with commercial launch expected to follow in the beginning of Q4
2022 if approved.
- An FDA advisory committee meeting for beti-cel and eli-cel will
be held over the course of two days on June 9-10, 2022.
ELI-CEL
- The FDA has set a PDUFA goal date of September 16, 2022, for a
decision on the approval of eli-cel in patients with cerebral
adrenoleukodystrophy with therapy availability expected in Q4 2022
if approved.
- An FDA advisory committee meeting for beti-cel and eli-cel will
be held over the course of two days on June 9-10, 2022.
- bluebird bio is in active communication with the FDA to resolve
the clinical hold and anticipates the FDA’s questions may be
resolved concurrent with the agency’s ongoing review of the
Company’s BLA submission.
FIRST QUARTER 2022 FINANCIAL
RESULTS
- Cash Position: The Company’s restricted cash, cash and
cash equivalents and marketable securities balance was
approximately $312 million, including restricted cash of
approximately $45 million, as of March 31, 2022. The full-year 2022
cash burn is expected to be less than $340 million with a 35 to 40
percent reduction in operating costs anticipated by year-end 2022.
The Company is exploring multiple financing opportunities,
including public or private equity financings and monetizing any
priority review vouchers that may be issued upon approval of
beti-cel or eli-cel.
- Revenues: Total revenue was $1.9 million for the three
months ended March 31, 2022, compared to $0.9 million for the three
months ended March 31, 2021.
- R&D Expenses: Research and development expenses from
continuing operations were $77.9 million for the three months ended
March 31, 2022, compared to $82.8 million for the three months
ended March 31, 2021. The decrease of $4.9 million was primarily
due to decreased employee compensation, benefit, and other
head-count related expenses, offset by increased manufacturing
costs.
- SG&A Expenses: Selling, general and administrative
expenses from continuing operations were $36.1 million for the
three months ended March 31, 2022, compared to $63.6 million for
the three months ended March 31, 2021. The decrease of $27.5
million was primarily due to decreased employee compensation,
benefit, and other head-count related expenses and decreased
commercial readiness activities due to the Company’s decision to
focus its efforts on the U.S. market for beti-cel, eli-cel, and
lovo-cel.
- Net Loss: Net loss from continuing operations was $122.2
million for the three months ended March 31, 2022, compared to
$121.5 million for the three months ended March 31, 2021.
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 clinical 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 is a trademark of bluebird bio, Inc.
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, as well as statements regarding the Company’s plans
and expectations for operations including expected timing relating
to its manufacturing plans, regulatory approvals and commercial
launches, potential cost-savings from our restructuring, expected
reductions of operating expenses, our expectations that the cost
savings from the restructuring will extend our cash runway into the
first half of 2023, and our expectations regarding the timing for a
potential BLA submission for lovo-cel, and anticipated FDA approval
of the BLAs for beti-cel and eli-cel. 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: 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 or delays in resolving existing 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, may be higher than expected which may
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 eli-cel and/or
beti-cel may not be approved within the priority review timeframe
or at all; the risk that any one or more of our product candidates,
including eli-cel and/or beti-cel, will not be successfully
developed, approved or commercialized. 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,
2022
2021
Revenue:
Product revenue
$
1,408
$
724
Other revenue
537
170
Total revenues
1,945
894
Operating expenses:
Research and development
77,875
82,843
Selling, general and administrative
36,106
63,569
Cost of product revenue
8,310
576
Total operating expenses
122,291
146,988
Loss from operations
(120,346
)
(146,094
)
Interest income, net
106
355
Other (expense) income, net
(1,912
)
24,301
Loss before income taxes
(122,152
)
(121,438
)
Income tax (expense) benefit
—
(66
)
Net loss from continuing operations
(122,152
)
(121,504
)
Net loss from discontinued operations
—
(84,304
)
Net loss
$
(122,152
)
$
(205,808
)
Net loss per share from continuing
operations—basic and diluted
$
(1.66
)
$
(1.81
)
Net loss per share from discontinued
operations—basic and diluted
$
—
$
(1.26
)
Net loss per share—basic and diluted
$
(1.66
)
$
(3.07
)
Weighted-average number of common shares
used in computing net loss per
share—basic and diluted
73,688
66,976
bluebird bio, Inc. Condensed
Consolidated Balance Sheet Data (in thousands)
(unaudited)
As of
March 31,
2022
As of
December 31,
2021
Cash, cash equivalents and marketable
securities
$
266,637
$
396,617
Total assets
$
491,071
$
593,795
Total liabilities
$
227,812
$
219,518
Total stockholders’ equity
$
263,259
$
374,277
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220509005905/en/
Investors & Media Investors: Courtney O’Leary,
978-621-7347 coleary@bluebirdbio.com Media: Sarah Alspach,
857-299-6198 sarah.alspach@bluebirdbio.com
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