bluebird bio Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Operational Progress – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ:BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2020 and shared recent operational progress.

For over ten years, bluebird bio has been pioneering gene therapies for patients with rare diseases and cancer, and our work continues with the analysis of the recent safety events identified in our HGB-206 study in patients with sickle cell disease. We have seen the transformative benefit of these therapies and are conducting a scientific and medical investigation to best inform our path forward on behalf of the patients we hope to serve with LentiGlobin, said Nick Leschly, chief bluebird. While this work is ongoing, we remain focused on the upcoming PDUFA action due date for ide-cel for multiple myeloma next month, the potential EU approval of eli-cel for CALD mid-year and the planned US filings for -thalassemia and CALD later in the year. Through these challenges, we are keeping patients at the center and were very excited to reach a major milestone of the first commercial infusion of ZYNTEGLO in Germany earlier this month.

RECENT HIGHLIGHTS

COMPANY

TRANSFUSION DEPENDENT -THALASSEMIA

SICKLE CELL DISEASE

MULTIPLE MYELOMA

UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

Clinical Updates and Milestones

FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS

About bluebird bio, Inc.bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the Companys timing and expectations regarding its investigation of the relationship of the safety events in HGB-206 to the use of lentiviral vector BB305 in LentiGlobin gene therapy for SCD, and any myeloablation regimen used in connection with treatment; the plans for regulatory submissions for beti-cel (marketed as ZYTENGLO in the European Union), eli-cel, ide-cel, and LentiGlobin for SCD, including anticipated endpoints to support regulatory submissions and timing expectations; the companys expectations regarding the potential for the suspension-based manufacturing process for lentiviral vector; the companys expectations and execution under its revised operating plan, including its cash runway; its expectations for commercialization efforts for ZYNTEGLO in Europe; and the companys expectations for the amended collaboration agreement with BMS; as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates; the timing, leadership, structure, including the division of assets among bluebird bio and Oncology Newco, and the impact of a separation; as well as the companys intention to provide further updates on the separation and the related financing strategies for bluebird bio and Oncology Newco; and the tax free nature of the separation. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company may not be able to definitively determine whether the lentiviral vector BB305 used in LentiGlobin gene therapy for SCD and in beti-cel is related to the safety events in a timely manner, or at all; the risk that the lentiviral vector BB305 has caused insertional oncogenic events, including acute myeloid leukemia; the risk that insertional oncogenic events associated with lentiviral vector or additional safety events associated with myeloablation will be discovered or reported over time; the risk that we may not be able to address regulatory authorities concerns quickly or at all and may not be able to resume our HGB-206 or HGB-210 studies in a timely manner, or at all; the risk that we may not resume patient treatment with ZYNTEGLO in the commercial context in a timely manner or at all; the risk that our lentiviral vector platform across our severe genetic disease programs may be implicated, affecting the development and potential approval elivaldogene autotemcel; the risks that we may not complete the separation on the terms or timeline currently contemplated if at all, achieve the expected benefits of a separation, and a separation could harm our business, results of operations and financial condition; the risk that the transaction might not be tax-free; we may be unable to make, on a timely or cost-effective basis, the changes necessary to operate as independent companies; Oncology Newco's lack of independent operating history and the risk that its accounting and other management systems may not be prepared to meet the financial reporting and other requirements of operating as an independent public company; dedicated financial and/or strategic funding sources may not be available on favorable terms; a separation or announcement thereof may adversely impact our ability to attract or retain key personnel; a separation may adversely impact the effectiveness of development and commercialization efforts by us and our partners; our businesses may be disrupted as a result of the announcement or pendency of the separation; the COVID-19 pandemic and resulting economic conditions will have a greater impact on the companys operations and plans than anticipated; that our amended collaboration with BMS will not continue or be successful; that preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk that our plans for submitting a BLA for LentiGlobin for SCD may be delayed if the FDA does not accept our comparability plans for the use of the suspension-based manufacturing process for LVV; the risk that the BLA for ide-cel is approved by the FDA in the timeline we expect, or at all; the risk of cessation or delay of any of the ongoing development activities, including clinical studies, and commercialization efforts due to delays from the COVID-19 pandemics impact on healthcare systems; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, or in obtaining sufficient coverage or reimbursement for our products; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.

Condensed Consolidated Statements of Operations

(in thousands, except per share data)

(unaudited)

For the three months endedDecember 31,

For the twelve months endedDecember 31,

2020

2019

2020

2019

Revenue:

Service revenue

$

5,522

$

5,827

$

114,064

$

30,729

Collaborative arrangement revenue

1,196

1,332

115,594

5,740

Royalty and other revenue

3,990

2,838

21,076

8,205

Total revenues

10,708

9,997

250,734

44,674

Operating expenses:

Research and development

137,094

161,821

587,956

582,413

Selling, general and administrative

76,974

76,202

286,896

271,362

Cost of royalty and other revenue

1,499

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bluebird bio Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Operational Progress - Business Wire