bluebird bio Reports Fourth Quarter and 2023 Annual Results and Highlights Operational Progress and 2024 Guidance
- bluebird bio secured a $175 million term loan facility with Hercules Capital to extend cash runway through Q1 2026.
- The Company expects between 85 to 105 patient starts in 2024 across its gene therapy portfolio, aiming for strong revenue growth.
- bluebird bio signed its first government outcomes-based agreement for sickle cell disease with Michigan Medicaid, demonstrating progress in access and reimbursement strategies.
- 62 Qualified Treatment Centers (QTCs) have been activated, with plans for continued network expansion in 2024.
- Full year 2023 revenue was $29.5 million, with $7.8 million generated in the fourth quarter, driven by ZYNTEGLO and SKYSONA product revenue.
- The Company's cash position was approximately $275 million as of December 31, 2023, with cash runway projected through Q1 2026.
- bluebird bio anticipates 85 to 105 patient starts (cell collections) across all three FDA approved therapies in 2024, with revenue recognition expected in the third quarter for LYFGENIA.
- The Company will restate its consolidated financial statements for 2022 and the first three quarters of 2023, with no impact on cash position or revenue.
- None.
Insights
The announcement by bluebird bio of securing a $175 million term loan facility with Hercules Capital is a strategic financial move that indicates the company's proactive approach to managing its capital structure. The staged disbursement of funds, contingent on commercial milestones, aligns the company's financing with its operational progress, potentially reducing the dilution risk for existing shareholders. The loan facility's extension of the cash runway through Q1 2026 provides bluebird with a buffer to achieve commercialization goals without the immediate pressure of raising additional capital through equity markets, which could be dilutive.
From an investor's perspective, the clear revenue trajectory with the projected 85 to 105 patient starts in 2024 for FDA approved therapies signals potential for growth. However, the discounts of 20% to 25% on gross revenue should be factored into revenue expectations, as they will affect net earnings. The restatement of financials due to lease accounting issues, although non-cash and not affecting cash position or revenue, may raise concerns about internal financial controls, necessitating a closer examination of the company's governance practices.
bluebird bio's expansion of its Qualified Treatment Centers (QTCs) and the signing of the first government outcomes-based agreement for sickle cell disease with Michigan Medicaid represent significant milestones in the healthcare market. The activation of 62 QTCs and the engagement with the Center for Medicare and Medicaid Innovation (CMMI) suggest an aggressive market penetration strategy that could set a precedent for gene therapy reimbursement models.
The company's focus on outcomes-based agreements with payers, including Medicaid, is a savvy approach to address the high costs associated with gene therapies and may facilitate broader market access. The lack of ultimate denials for ZYNTEGLO and SKYSONA therapies across Medicaid and commercial payers is indicative of effective access and reimbursement strategies, which are critical in the high-stakes gene therapy market where treatment costs can be a barrier to patient access.
bluebird bio's progress in commercializing gene therapies such as ZYNTEGLO, SKYSONA and LYFGENIA is a testament to the maturation of gene therapy as a viable treatment option in the biotechnology industry. The company's anticipation of revenue recognition from LYFGENIA infusions in Q3 2024 underscores the lengthy process from cell collection to infusion, a characteristic of the gene therapy value chain that stakeholders must understand.
The ongoing enrollment for the HGB-210 study evaluating lovo-cel for patients under the age of 12 highlights the company's commitment to expanding its therapy portfolio and addressing unmet medical needs. The company's approach to gene therapy for rare diseases like beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease demonstrates a targeted strategy aimed at diseases with significant demand for innovative treatments. This focus on rare diseases with high unmet medical needs could lead to strong market positioning given the limited competition and potential for orphan drug exclusivity.
- Cash runway through Q1 2026 following announcement of a
- 9 patient starts to date in 2024 (7 ZYNTEGLO, 2 SKYSONA); 85 to 105 patient starts anticipated across the portfolio in 2024 -
- First government outcomes-based agreement for sickle cell disease signed with Michigan Medicaid -
- 62 qualified treatment centers (QTCs) activated -
- Full year 2023 revenue of
- Management to host conference call today, March 26, 2024 at 8:00 am ET -
“In 2023, bluebird established a validated, commercial gene therapy strategy that brought ZYNTEGLO and SKYSONA to individuals living with beta-thalassemia and cerebral adrenoleukodystrophy. Building on that foundation, today we are positioned for robust commercial uptake of LYFGENIA for sickle cell disease, with a substantial QTC network in place, favorable Medicaid coverage being established, and demonstrated strong patient demand,” said Andrew Obenshain, chief executive officer, bluebird bio. “Our recent agreement with Hercules Capital meaningfully extends our cash runway, and further enables us to capitalize on our commercial head start and bring our transformative gene therapies to patients and their families. In 2024, we anticipate between 85 to 105 patient starts across our three FDA approved therapies, laying the foundation for strong revenue growth.”
RECENT COMPANY HIGHLIGHTS
Up to
-
On March 18, 2024, bluebird announced that it had entered into a five-year term loan facility with Hercules Capital. Under the terms of the agreement, the Company may draw up to
$175 million $75 million $25 million $50 million -
Based on launch estimates and current business plans, and assuming three tranches totaling
$125 million
COMMERCIAL LAUNCH UPDATES
Strong patient uptake across portfolio
- First LYFGENIA patient start imminent; multiple patients enrolled and preparing for treatment across QTC network.
- Continued strong, linear growth for ZYNTEGLO with 7 patient starts since the beginning of 2024, in addition to 20 patient starts completed for ZYNTEGLO in 2023.
- Completed 2 patient starts for SKYSONA since the beginning of 2024, in addition to 6 patient starts completed for SKYSONA in 2023.
Validated access and reimbursement strategy is driving favorable coverage landscape
-
In the first quarter of 2024, bluebird signed its first Medicaid outcomes-based agreement for LYFGENIA with the state of
Michigan . -
In addition to the Medicaid outcomes-based agreement, bluebird has signed four outcomes-based agreements for LYFGENIA with national commercial payer organizations and published coverage policies cover more than 200 million
U.S. lives. -
Discussions are ongoing with more than 15 Medicaid agencies representing
80% of Medicaid-insured individuals with sickle cell disease in theU.S. and the Company is engaged with the Center for Medicare and Medicaid Innovation (CMMI) on its Cell and Gene Therapy Access Model demonstration. - Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established
- bluebird has activated 62 QTCs for ZYNTEGLO (defined as a signed MSA); capitalizing on launch synergies, 49 centers are already receiving referrals for LYFGENIA.
- Five centers are also activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
- The Company anticipates continued QTC network expansion across its portfolio in 2024.
LOVO-CEL CLINICAL TRIAL UPDATE
- Enrollment is ongoing for the HGB-210 study evaluating lovo-cel for patients under the age of 12. The Company anticipates enrollment to be complete in Q4 2024.
2024 GUIDANCE
- The Company anticipates 85 to 105 patient starts (cell collections) combined across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts for each of its therapies.
-
Gross-to-net discounts across all three products are expected to be in the range of
20% to25% of gross revenue in 2024 and will fluctuate based on product and payer mix, and well as utilization of outcomes-based agreements for LYFGENIA and ZYNTEGLO. - Based on projected timelines from cell collection to infusion, the Company expects to recognize revenue from its first infusion of LYFGENIA in the third quarter of 2024.
FOURTH QUARTER AND ANNUAL FINANCIAL HIGHLIGHTS
-
Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately
$275 million $53 million
Based on launch trajectory and current business plans, bluebird expects its cash and cash equivalents excluding restricted cash and assuming three tranches totaling$125 million
In the fourth quarter of 2023, the Company entered into a factoring agreement which is accelerating cash collection related to patient starts across its portfolio of approved therapies.
-
Revenue, net: Total revenue, net was
$7.8 million $0.1 million
Total revenue, net was$29.5 million $3.6 million $25.9 million
For the year ended December 31, 2023, product revenues by therapy represent$16.7 million $12.4 million 19% across both products.
On March 26, 2024, bluebird announced that it will restate its consolidated financial statements for 2022, and for the first three quarters of both 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the “2023 Form 10-K"). The restatements relate to the identification of embedded leases and the treatment of non-lease components contained in lease agreements with contract manufacturers. As a result, the Company anticipates recording an increase in lease assets and lease liabilities, as well as an increase in non-cash interest expense in each restated period. The Company does not expect the restatement to result in any impact on its cash position or revenue. bluebird anticipates filing its 2023 Form 10-K, inclusive of the restatement no later than April 16, 2024.
The financial results included in this press release represent the most current information available to the Company’s management. The Company expects that its actual results to be reported in its 2023 Form 10-K will not differ materially from the results included herein, however, these results are subject to change following the completion of the Company’s financial close procedures and the audit of its consolidated financial statements for the year ended December 31, 2023.
CONFERENCE CALL DETAILS
bluebird will hold a conference call to discuss its fourth quarter and 2023 annual results and business updates today, Tuesday, March 26, 2024, at 8:00 am ET.
To access the live conference call via telephone, please register at this link to receive a dial in number and unique PIN.
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.
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, LYFGENIA, 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, such as statements regarding the number of anticipated patient starts across bluebird’s portfolio of therapies and the timing of the first LYFGENIA patient start, expectations regarding gross-to-net discounts, the Company’s anticipated cash runway, the Company’s expectations regarding its ability to access future tranches of its term loan facility, the Company’s expectations with respect to the commercialization of LYFGENIA, including without limitation, the potential for robust commercial uptake of LYFGENIA, the timing of revenue recognition, patient demand for the therapy, bluebird’s ability to establish favorable coverage for its therapies, including its ability successfully partner with payers and its expectations for expansion of its QTC network , expectations with respect to the completion of enrollment in HGB-210, and expectations regarding the Company’s restatement of certain historical financial statements and the timing for filing of its 2023 10-K. 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 the Company’s therapies; 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240326211450/en/
Investors & Media
Investors:
Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com
Media:
Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
Source: bluebird bio, Inc.
FAQ
What is the purpose of the $175 million term loan facility with Hercules Capital announced by bluebird bio?
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