- Continued strong commercial launch for
ZYNTEGLO® and SKYSONA®; 22 patient starts across both programs to
date –
- Ended quarter with $227M in cash, cash
equivalents, marketable securities and restricted cash –
- Company entered into advance agreement to
sell priority review voucher, if granted, for $103 million;
potential non-dilutive capital would strengthen cash position –
- Management to host conference call today at
8:00AM ET -
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today reported financial results and business highlights
for the third quarter ended September 30, 2023, including recent
commercial and operational progress, and regulatory updates.
“We continue to see strong, linear growth for both the ZYNTEGLO
and SKYSONA launches, with patients completing the treatment
process, and significant demand across our established qualified
treatment center network," said Andrew Obenshain, chief executive
officer, bluebird bio. "Launch preparations for lovo-cel for sickle
cell disease are well underway in anticipation of our December 2023
PDUFA date. In the third quarter, we accelerated the growth of our
QTC network, which is one hundred percent synergistic across
ZYNTEGLO and lovo-cel, and advanced conversations with payers who
recognize the value of lovo-cel and have responded favorably to our
innovative contract offerings. We are confident in our commercial
gene therapy experience, committed to continued partnership with
the sickle cell community, and excited about the opportunity to
bring gene therapy to individuals living with SCD.”
RECENT HIGHLIGHTS
Commercial launches of ZYNTEGLO® (betibeglogene autotemcel)
and SKYSONA® (elivaldogene autotemcel)
- Continued strong, linear growth for ZYNTEGLO with 16 patient
starts (cell collections) since launch. Cell collection has been
completed for 6 patients for SKYSONA to date.
- In September, bluebird announced an amendment to its agreement
with Lonza, which manufactures drug product for ZYNTEGLO and
SKYSONA. The amended agreement enables increased manufacturing
capacity for both therapies. The agreement also included modified
payment terms intended to better align with the Company’s business
objectives.
- Value of ZYNTEGLO and SKYSONA continues to be recognized among
commercial and government payers. Recently, bluebird signed
outcomes-based agreements for ZYNTEGLO with Michigan and
Massachusetts state Medicaid agencies. To date, the Company has
received zero ultimate denials for both therapies and prior
authorization approvals for drug product remain consistent at
approximately two weeks across all payers.
- bluebird’s qualified treatment center (QTC) network has scaled
to 29 activated centers (defined as a signed master service
agreement or MSA) including both adult and pediatric centers across
16 states. Of these 29 activated QTCs, four have been activated to
administer SKYSONA for patients with cerebral adrenoleukodystrophy
(CALD) in addition to ZYNTEGLO. The Company remains on track to
scale to 40-50 activated QTCs by the end of 2023.
lovo-cel FDA Review
- On August 16, 2023, bluebird bio announced that the U.S. Food
and Drug Administration (FDA) communicated that an advisory
committee meeting will not be scheduled for lovotibeglogene
autotemcel (lovo-cel).
- The lovo-cel Biologics Licensing Application (BLA) review
remains on track. The Agency previously accepted the lovo-cel BLA
for Priority Review and set a Prescription Drug User Fee Act
(PDUFA) goal date of December 20, 2023.
Advance Agreement to Sell PRV
- On October 30, 2023, bluebird entered into an agreement to sell
a Rare Pediatric Disease Priority Review Voucher (PRV), if
received, in connection with the potential approval of lovo-cel for
sickle cell disease. Under the terms of the agreement, rights to
the PRV will transfer to the buyer and the Company will receive
$103 million upon closing of the sale, which is contingent upon the
FDA’s approval of the BLA for lovo-cel and granting of the PRV.
bluebird anticipates receipt of a potential PRV should lovo-cel be
approved for patients with sickle cell disease ages 12 and older.
Proceeds from the potential sale of the PRV are not yet reflected
in the Company’s cash runway.
Data Presentations at ASH 2023
Updated long-term follow-up data from the Company’s gene therapy
programs in sickle cell disease and beta-thalassemia will be
presented at the 65th American Society of Hematology (ASH) Annual
Meeting and Exposition, taking place December 9-12, 2023 at the San
Diego Convention Center and online. bluebird continues to have the
most mature gene therapy programs for both SCD and
beta-thalassemia, presenting up to five years and up to nine years
of long-term data, respectively.
- SICKLE CELL DISEASE DATA
- Oral Presentation [#1051]:
Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in
Patients with Sickle Cell Disease (SCD) Who Have Received
lovotibeglogene autotemcel (lovo-cel) Gene Therapy: Up to 60 Months
of Follow-up
- Presenting Author: Julie Kanter, M.D., director of the UAB
Adult Sickle Cell clinic, associate professor in the Division of
Hematology and Oncology, and co-director of the UAB Comprehensive
Sickle Cell Disease Center at the University of Alabama in
Birmingham
- Date/Time: Monday, December 11, 2023, 4:30 p.m. PT
- Poster Presentation
[#1102]: Sustained, Efficacy, Safety, and Improved
Quality of Life in Adult and Pediatric Patients with
Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment
with betibeglogene autotemcel (beti-cel)
- Presenting Author: Alexis A. Thompson, M.D., M.P.H., professor
of pediatrics (hematology), Perelman School of Medicine, University
of Pennsylvania, Philadelphia, and chief, Division of Hematology,
Children’s Hospital of Philadelphia
- Date/Time: Saturday, December 9, 2023, 5:30 p.m. PT
- Poster Presentation
[#2480]: Improvement in Iron Burden in Patients with
Transfusion-Dependent β-Thalassemia (TDT) Treated with
betibeglogene autotemcel (beti-cel) Gene Therapy: Up to 9 Years of
Follow-up
- Presenting Author: Janet L. Kwiatkowski, M.D., MSCE, professor
of pediatrics (hematology), Department of Pediatrics, Perelman
School of Medicine, University of Pennsylvania, Philadelphia, and
director, Thalassemia Center, Children's Hospital of
Philadelphia
- Date/Time: Sunday, December 10, 2023, 6:00 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH 2023 are
available on the ASH conference website.
UPCOMING ANTICIPATED
MILESTONES
- For 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.
- The Company is on track to scale to 40-50 activated 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.
- For SKYSONA, the Company continues to anticipate 5-10 patient
starts this year as previously guided.
THIRD QUARTER 2023 FINANCIAL
RESULTS
- Cash Position: The Company’s cash, cash equivalents,
marketable securities and restricted cash balance was approximately
$227 million, as of September 30, 2023. bluebird anticipates
full-year 2023 net 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 and marketable
securities including anticipated cash flows from operations, and
excluding $53 million of restricted cash, will be sufficient to
meet bluebird’s planned operating expenses and capital expenditure
requirements into the second quarter of 2024.
- Revenue, net: Total revenue, net was $12.4 million for
the three months ended September 30, 2023, compared to $0.1 million
for the three months ended September 30, 2022. The increase of
$12.3 million was primarily due to SKYSONA and ZYNTEGLO product
revenue.
- SG&A Expenses: Selling, general and administrative
expenses were $40.7 million for the three months ended September
30, 2023, compared to $33.4 million for the three months ended
September 30, 2022. SG&A includes lease expense related to 50
Binney Street; however, the associated sublease income is presented
in other income (expense), net. Excluding the lease expense for 50
Binney St., SG&A expenses were $31.1 million for the three
months ended September 30, 2023, compared to $24.1 million for the
three months ended September 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 increased
employee compensation, benefit and other headcount-related
expenses, professional fees, and information technology and
facility related costs in 2023. These increased costs were
partially offset by decreased consulting fees.
- R&D Expenses: Research and development expenses were
$45.5 million for the three months ended September 30, 2023,
compared to $53.1 million for the three months ended September 30,
2022. The decrease of $7.6 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, license and milestone fees, and
information technology and facility related costs in 2023. These
decreased costs were partially offset by increased clinical costs,
lab expenses, and consulting fees.
- Net income (loss): Net loss was $71.7 million for the
three months ended September 30, 2023, compared to a net loss of
$76.5 million for the three months ended September 30, 2022.
CONFERENCE CALL DETAILS
bluebird will hold a conference call to discuss third quarter
financial results and commercial launch progress on Tuesday,
November 7 at 8:00 am ET.
To access the call via telephone, please register at this link
https://register.vevent.com/register/BIee363f414c2f4fd19da314c2b328d635
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; the Company’s ability to
obtain additional financing options to extend its cash runway; and
anticipated cash burn for 2023, as well as statements regarding the
Company’s plans and expectations for operations, including with
respect to the therapeutic potential of the Company’s products and
product candidates; the potential regulatory approval, including
the PDUFA acceptance, and commercial launch of lovo-cel; closing of
the sale of the Company’s PRV, if received in connection with the
potential approval of lovo-cel; expectations regarding receipt of
the PRV; plans to expand manufacturing capacity; anticipated growth
of its QTC network and timing thereof; the Company’s ability to
pursue curative gene therapies and the expected benefits for
patients; and the Company’s participation in upcoming events and
presentations. 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 for the
year ended December 31, 2022, 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; the risk that
we may not receive a Priority Review Voucher upon potential
approval of lovo-cel or that lovo-cel may not be approved in the
timeframe we anticipate or at all; 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 of insertional oncogenic or
other reportable events associated with lentiviral vector, drug
product, or myeloablation; the risk that any one or more of our
products or product candidates, including 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
September 30,
For the nine months ended
September 30,
2023
2022
2023
2022
Revenue:
Product revenue, net
$
12,281
$
—
$
21,414
$
2,739
Other revenue
111
71
249
795
Total revenues
12,392
71
21,663
3,534
Cost of product revenue
10,955
—
23,895
10,056
Gross margin
1,437
71
(2,232
)
(6,522
)
Operating expenses:
Selling, general and administrative
40,703
33,402
118,406
106,201
Research and development
45,463
53,149
133,881
194,864
Restructuring expenses
—
(1,699
)
—
4,940
Total operating expenses
86,166
84,852
252,287
306,005
Gain from sale of priority review voucher,
net
—
—
92,930
—
Income (loss) from operations
(84,729
)
(84,781
)
(161,589
)
(312,527
)
Interest income, net
2,454
383
7,958
663
Other income (expense), net
10,544
7,885
30,152
13,061
Income (loss) before income taxes
(71,731
)
(76,513
)
(123,479
)
(298,803
)
Income tax (expense) benefit
—
(7
)
80
(7
)
Net income (loss)
$
(71,731
)
$
(76,520
)
$
(123,399
)
$
(298,810
)
Net income (loss) per share - basic
$
(0.66
)
$
(0.94
)
$
(1.15
)
$
(3.91
)
Net income (loss) per share - diluted
$
(0.66
)
$
(0.94
)
$
(1.15
)
$
(3.91
)
Weighted-average number of common shares
used in computing net income (loss) per share - basic:
109,098
81,543
106,924
76,361
Weighted-average number of common shares
used in computing net income (loss) per share - diluted:
109,098
81,543
106,924
76,361
Other comprehensive income (loss):
Other comprehensive income (loss), net of
tax benefit (expense) of $0.0 million for the three and nine months
ended September 30, 2023 and 2022
137
(214
)
1,843
(1,719
)
Total other comprehensive income
(loss)
137
(214
)
1,843
(1,719
)
Comprehensive income (loss)
$
(71,594
)
$
(76,734
)
$
(121,556
)
$
(300,529
)
bluebird bio, Inc. Condensed
Consolidated Balance Sheet Data (in thousands)
(unaudited)
As of
September 30,
2023
As of
December 31,
2022
Cash, cash equivalents and marketable
securities
$
174,293
$
181,741
Restricted cash
$
53,022
$
45,439
Total assets
$
613,608
$
554,902
Total liabilities
$
391,072
$
358,559
Total stockholders’ equity
$
222,536
$
196,343
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231107552972/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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