Significant momentum behind LYFGENIA launch
with 35 Qualified Treatment Centers accepting patient referrals and
payer agreements in place covering approximately 200 million U.S.
lives
Anticipate 85 to 105 patient starts (cell
collections) across commercial portfolio in 2024 with first patient
start for LYFGENIA expected in Q1 2024
Cash, cash equivalents and marketable
securities expected to fund current operations into Q1 2025
bluebird bio, Inc. (Nasdaq: BLUE) (the Company; bluebird) today
announced updates to be presented at the 42nd Annual J.P. Morgan
Healthcare conference including commercial launch progress, 2024
program milestones and financial outlook. Andrew Obenshain, chief
executive officer, is scheduled to present Tuesday, January 9 at
10:30 a.m. PT/1:30 p.m. ET.
“In 2023 bluebird cemented our status as a gene therapy leader,
securing our third FDA approval in under two years and establishing
a commercial footprint that will support growth in the coming year
and beyond,” said Andrew Obenshain, chief executive officer,
bluebird bio. “In 2024 we are leveraging our validated commercial
strategy to accelerate the launch of LYFGENIA and drive continued
strong uptake for ZYNTEGLO. We are extremely pleased with the
indicators of demand from both patients and providers in the weeks
following FDA approval of LYFGENIA and are focused on using our
real-world experience to support timely and equitable access and
deliver a positive treatment experience.”
Highlights from the Company’s update include:
Synergies with ZYNTEGLO commercial network are accelerating
LYFGENIA commercial launch in 2024
- Established Qualified Treatment Center (QTC) network in place,
with 48 centers activated for ZYNTEGLO for beta-thalassemiai as of
January 5, 2024.
- 35 of 48 centers ready to receive referrals for LYFGENIA for
sickle cell disease as of January 5, 2024.
- All centers are anticipated to be ready to treat with both
ZYNTEGLO and LYFGENIA by end of Q1 2024.
Validated access and reimbursement strategy is driving favorable
coverage landscape for LYFGENIA and ZYNTEGLO
- bluebird has signed outcomes-based agreements for LYFGENIA with
national payer organizations representing dozens of downstream
plans and covering approximately 200 million U.S. lives.
- Advanced discussions are ongoing with additional commercial
payers and with more than 15 Medicaid agencies representing 80% of
individuals with sickle cell disease in the U.S.
- bluebird has designed outcomes-based agreements that are unique
to LYFGENIA and offer payers meaningful risk sharing tied to
VOE-related hospitalizations, with patients followed for three
years. Informed by input from state Medicaid agencies, the Company
has designed an offering specifically for Medicaid that addresses
the need for predictability and operational ease that is essential
for states grappling with resource constraints.
- Outcomes-based agreements are in place for ZYNTEGLO with both
commercial and Medicaid payers, and more than 200 million U.S.
lives are covered through contracts or favorable coverage policies.
Timely access to ZYNTEGLO for people living with beta-thalassemia
continues, with zero ultimate denials across both Medicaid and
commercial payers.
- bluebird also continues to engage with Center for Medicare and
Medicaid Innovation (CMMI) on its Cell and Gene Therapy Access
Model, which is anticipated to be implemented in 2025.
Strong commercial momentum is poised to translate into sustained
revenue recognition
- 26 patient starts were completed in 2023 across bluebird’s
commercial portfolio, including 20 for ZYNTEGLO and 6 for SKYSONA.
2023 patient starts will drive revenue recognition in 2024 as
patients complete the gene therapy treatment journey.
- bluebird anticipates the first patient start for LYFGENIA in Q1
2024.
- The Company anticipates 85 to 105 patient starts combined
across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO,
SKYSONA) in 2024.
Liquidity and Cash Runway Update
The Company’s preliminary unaudited cash and cash equivalents
and marketable securities balance was approximately $275 million,
including restricted cash of approximately $53 million, as of
December 31, 2023. bluebird expects its cash, cash equivalents, and
marketable securities, excluding restricted cash, will be
sufficient to meet bluebird’s planned operating expenses and
capital expenditure requirements into the first quarter of 2025 as
bluebird progresses its launch of LYFGENIA gene therapy for sickle
cell disease and continues to scale its launches of ZYNTEGLO and
SKYSONA for beta-thalassemia and cerebral adrenoleukodystrophy,
respectively.
The Company has taken additional steps to strengthen its
financial position by entering into an accounts receivable
factoring agreement which will accelerate cash collection related
to patient starts across its portfolio of approved therapies.
Presentation at the 2024 J.P. Morgan Healthcare Conference
Andrew Obenshain, chief executive officer, bluebird bio, will
present a corporate update on Tuesday, January 9 at 10:30 a.m.
PT/1:30 p.m. ET. A live webcast of the presentation will be
available on the “Events & Presentations” page within the
Investors & Media section of the bluebird bio website at
http://investor.bluebirdbio.com. A replay of the webcast will be
available on the bluebird bio website for 30 days following the
event.
About bluebird bio
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days.
Founded in 2010, bluebird has been setting the standard for gene
therapy for more than a decade—first as a scientific pioneer and
now as a commercial leader. bluebird has an unrivaled track record
in bringing the promise of gene therapy out of clinical studies and
into the real-world setting, having secured FDA approvals for three
therapies in under two years. Today, we are proving and scaling the
commercial model for gene therapy and delivering innovative
solutions for access to patients, providers, and payers.
With a dedicated focus on severe genetic diseases, bluebird has
the largest and deepest ex-vivo gene therapy data set in the field,
with industry-leading programs for sickle cell disease,
β-thalassemia and cerebral adrenoleukodystrophy. 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.
bluebird continues to forge new paths as a standalone commercial
gene therapy company, 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 Cautionary Statement Regarding 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, such as
statements regarding the number of anticipated patient starts
across bluebird’s portfolio of therapies, the Company’s anticipated
cash runway, the Company’s expectations with respect to the
commercialization of LYFGENIA, including without limitation,
patient demand for the therapy, bluebird’s ability to establish
commercial infrastructure to support timely, equitable access to
LYFGENIA, its ability to successfully partner with payers and CMMI,
its expectations on timing for activating QTCs, its expectations on
the timing and size of its QTC network and the timing of LYFGENIA’s
availability at its QTCs. Such forward-looking statements are based
on historical performance and current expectations and projections
about bluebird’s future 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 bluebird’s control and could cause bluebird’s future 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 its
subsequent Quarterly Reports on Form 10-Q, Current Reports on Form
8-K and other filings with the Securities and Exchange Commission.
These risks and uncertainties include, but are not limited to:
delays and challenges in bluebird’s commercialization and
manufacturing of its products; the internal and external costs
required for bluebird’s 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 bluebird, and
may in the future cause bluebird to use cash more quickly than it
expects or change or curtail some of its plans or both; substantial
doubt exists regarding bluebird’s ability to continue as a going
concern; bluebird’s 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 bluebird’s
assumptions; the risk that the efficacy and safety results from
bluebird’s prior and ongoing clinical trials will not continue or
be seen in the commercial context; the risk that bluebird is not
able to activate QTCs on the timeframe that it expects; the risk
that the QTCs experience delays in their ability to enroll or treat
patients; the risk that bluebird experiences delays in establishing
operational readiness across its supply chain following approval to
support treatment in the commercial context; the risk that there is
not sufficient patient demand or payer reimbursement to support
continued commercialization of LYFGENIA; the risk of insertional
oncogenic or other safety events associated with lentiviral vector,
drug product, or myeloablation, including the risk of hematologic
malignancy; and the risk that bluebird’s products, including
LYFGENIA, will not be successfully 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.
Financial Disclosure Advisory
The cash and cash equivalents and marketable securities balance
disclosed in this press release is based on preliminary unaudited
information and management estimates for the year ended December
31, 2023, is not a comprehensive statement of the Company’s
financial results as of and for the fiscal year ended December 31,
2023, and is subject to completion of the Company’s financial
closing procedures. The Company’s independent registered public
accounting firm has not conducted an audit or review of, and does
not express an opinion or any other form of assurance with respect
to, this preliminary estimate.
_________________________ i Defined as signed MSA
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version on businesswire.com: https://www.businesswire.com/news/home/20240108221653/en/
Investors: Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com
Media: Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
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