- beti-cel for beta-thalassemia PDUFA goal date
is set for August 19, 2022 -
- eli-cel for cerebral adrenoleukodystrophy
PDUFA goal date is set for September 16, 2022 -
- Ended quarter with $218M 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 second quarter ended June 30, 2022, and shared recent
operational progress.
“The second quarter marked significant progress for bluebird bio
and a precedent-setting moment for the field of gene therapy,” said
Andrew Obenshain, chief executive officer, bluebird bio. “With the
FDA advisory committee’s unanimous support for beti-cel and eli-cel
for their target indications, we are now laser-focused on
commercial readiness, and if approved, we anticipate launching both
therapies in the fourth quarter of this year. Additionally, this
quarter we advanced the remaining CMC steps ahead of our lovo-cel
BLA submission, and we remain on track to submit the BLA in the
first quarter of next year.”
RECENT HIGHLIGHTS
BETI-CEL
- UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING
– On June 10, the U.S. Food and Drug Administration’s (FDA)
Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC)
voted (13-0) that the benefits of betibeglogene autotemcel
(beti-cel) gene therapy outweigh the risks for people with
beta-thalassemia who require regular red blood cell transfusions.
If approved, beti-cel will be the first ex-vivo LVV gene therapy
available in the U.S.
- ICER REVIEW – The Institute for Clinical and Economic
Review (ICER) completed its review of beti-cel for people with
beta-thalassemia and determined in its final report that beti-cel
will be cost effective at a price up to $3.0 million. bluebird
anticipates setting a price for beti-cel upon potential FDA
approval.
ELI-CEL
- UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING
– On June 9, the FDA CTGTAC voted (15-0) that the benefits of
elivaldogene autotemcel (eli-cel) gene therapy outweigh the risks
for the treatment of any sub-population of children with early
active cerebral adrenoleukodystrophy (CALD). If approved, eli-cel
will be the first and only gene therapy for the treatment of early
active CALD, a rare neurodegenerative disease that primarily
affects young children and leads to irreversible loss of neurologic
function and death.
LOVO-CEL
- CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio
remains on track to submit a biologics licensing application (BLA)
to the FDA for lovotibeglogene autotemcel (lovo-cel) for sickle
cell disease 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.
This quarter, the Company completed manufacturing of commercial
drug product validation lots, marking significant progress on CMC
requirements and final steps to BLA submission. Additionally,
bluebird completed enrollment of all patients in the HGB-210 study
necessary to support manufacturing data requirements for the BLA
submission. The remaining step prior to BLA submission is
completion of vector and drug product analytical comparability,
which the Company expects to complete in the fourth quarter of
2022. The Company remains in active dialogue with the FDA about the
resolution of the partial clinical hold for patients under 18. The
Company is continuing to enroll and treat patients 18 and older in
the HGB-210 study.
COMPANY
- TOM KLIMA APPOINTED CHIEF COMMERCIAL & OPERATING OFFICER
– Effective August 8, Tom Klima will serve as Chief Commercial
& Operating Officer. Klima joined bluebird in May 2021 as Chief
Commercial Officer to hone the Company’s commercial strategy and
oversee launch execution plans for its gene therapy portfolio. His
new role reflects expanded responsibilities for program management
and patient supply chain in addition to sales, marketing and market
access.
UPCOMING INVESTOR EVENT
Members of the management team will participate in the 2022
Wedbush PacGrow Virtual Healthcare Conference, Wednesday, August
10, at 9:10 a.m. ET as part of the panel titled Miss
Con-GENE-iality - Updates in Gene Tx.
UPCOMING ANTICIPATED
MILESTONES
BETI-CEL
- The FDA has set a PDUFA goal date for August 19, 2022, and if
approved, the Company anticipates first apheresis in the fourth
quarter of 2022.
- beti-cel is being reviewed under Priority Review for the
treatment of beta-thalassemia in patients requiring regular red
blood cell transfusions. bluebird bio anticipates receiving a
Priority Review Voucher (PRV) upon potential approval of
beti-cel.
ELI-CEL
- The FDA has set a PDUFA goal date of September 16, 2022, and if
approved, the Company anticipates therapy availability in the
fourth quarter of 2022.
- eli-cel is being reviewed under Priority Review for the
treatment of cerebral adrenoleukodytrophy in patients less than 18
years of age who do not have an available and willing human
leukocyte antigen (HLA)-matched sibling hematopoietic stem cell
(HSC) donor. bluebird bio anticipates receiving a PRV upon
potential approval of eli-cel.
- bluebird bio is in active communication with the FDA to resolve
the eli-cel clinical hold and anticipates the FDA’s questions may
be resolved concurrent with the agency’s ongoing review of the
Company’s BLA submission.
LOVO-CEL
- The Company is in active communication with the FDA to resolve
the lovo-cel partial clinical hold and resume enrollment and
treatment of patients under the age of 18.
- The Company expects to complete vector and drug product
analytical comparability in the fourth quarter of 2022.
- The Company plans to submit its BLA for lovo-cel in Q1
2023.
SECOND QUARTER 2022 FINANCIAL
RESULTS
- Cash Position: The Company’s restricted cash, cash and
cash equivalents and marketable securities balance was
approximately $218 million, including restricted cash of
approximately $45 million, as of June 30, 2022. The full-year 2022
cash burn is expected to be less than $340 million. As of today,
the Company has raised approximately $24.7 million in gross
proceeds through its At-the-Market (ATM) equity facility. Of this
$24.7 million, $8.0 million in net proceeds were realized in the
second quarter and are reflected in the restricted cash, cash and
cash equivalents and marketable securities balanced as of June 30,
2022. The Company is exploring additional 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 $1.5 million for the three
months ended June 30, 2022, compared to $0.1 million for the three
months ended June 30, 2021.
- R&D Expenses: Research and development expenses from
continuing operations were $63.8 million for the three months ended
June 30, 2022, compared to $84.6 million for the three months ended
June 30, 2021. The decrease of $20.8 million was primarily due to
decreased employee compensation, benefits, other head-count related
expenses, information technology and facility-related costs, lab
expenses and clinical trial costs. These decreased costs were
partially offset by increased manufacturing costs.
- SG&A Expenses: Selling, general and administrative
expenses from continuing operations were $36.7 million for the
three months ended June 30, 2022, compared to $55.0 million for the
three months ended June 30, 2021. The decrease of $18.3 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.
These decreased costs were partially offset by increased
information technology and facility-related costs due to the
addition of the Company’s office lease in Somerville,
Massachusetts.
- Net Loss: Net loss from continuing operations was $100.1
million for the three months ended June 30, 2022, compared to
$155.8 million for the three months ended June 30, 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, and anticipated cash burn for 2022, as well as
statements regarding the Company’s plans and expectations for
operations including expected timing relating to its regulatory
approvals, commercial launches including the initiation of patient
apheresis in the commercial context following potential approval,
expectations regarding the price of any therapy if approved by the
FDA, plans for future regulatory submissions, expectations
regarding the timing of completion of vector and drug product
analytical comparability for lovo-cel, expectations regarding the
receipt of any Priority Review Vouchers upon potential approval of
beti-cel or eli-cel, and our expectations regarding the timing for
a potential BLA submission for lovo-cel, anticipated PDUFA goal
dates 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
June 30,
For the six months ended June
30,
2022
2021
2022
2021
Revenue:
Product revenue
$
1,331
$
—
$
2,739
$
724
Other revenue
188
143
725
313
Total revenues
1,519
143
3,464
1,037
Operating expenses:
Research and development
63,841
84,645
141,716
167,488
Selling, general and administrative
36,694
54,984
72,800
118,553
Cost of product revenue
1,745
15,215
10,055
15,791
Restructuring expenses
6,639
—
6,639
—
Total operating expenses
108,919
154,844
231,210
301,832
Loss from operations
(107,400
)
(154,701
)
(227,746
)
(300,795
)
Interest income, net
174
218
280
573
Other (expense) income, net
7,088
(1,274
)
5,176
23,027
Loss before income taxes
(100,138
)
(155,757
)
(222,290
)
(277,195
)
Income tax (expense) benefit
—
(216
)
—
(282
)
Net loss from continuing operations
(100,138
)
(155,973
)
(222,290
)
(277,477
)
Net loss from discontinued operations
—
(85,729
)
—
(170,033
)
Net loss
$
(100,138
)
$
(241,702
)
$
(222,290
)
$
(447,510
)
Net loss per share from continuing
operations - basic and diluted
$
(1.36
)
$
(2.31
)
$
(3.02
)
$
(4.13
)
Net loss per share from discontinued
operations - basic and diluted
$
—
$
(1.27
)
$
—
$
(2.53
)
Net loss per share - basic and diluted
$
(1.36
)
$
(3.58
)
$
(3.02
)
$
(6.66
)
Weighted-average number of common shares
used in computing net loss per share - basic and diluted:
73,767
67,487
73,727
67,233
bluebird bio, Inc.
Condensed Consolidated Balance
Sheet Data
(in thousands)
(unaudited)
As of June 30, 2022
As of December 31,
2021
Cash, cash equivalents and marketable
securities
$
173,150
$
396,617
Total assets
$
573,592
$
593,795
Total liabilities
$
393,476
$
219,518
Total stockholders’ equity
$
180,116
$
374,277
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220804005831/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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