Significant momentum behind LYFGENIA launch with 35 Qualified Treatment Centers accepting patient referrals and payer agreements in place covering approximately 200 million
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
“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
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
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
- 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
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
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
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
i Defined as signed MSA
Courtney O’Leary, 978-621-7347
Source: bluebird bio, Inc.