- First commercially treated ZYNTEGLO patient apheresed in Q4 2022; first revenue anticipated in Q1 2023 -

- lovo-cel BLA submission on track for Q1 2023; vector and drug product analytical comparability studies complete -

- Company enters 2023 with cash runway into Q1 2024 -

bluebird bio, Inc. (Nasdaq: BLUE) (the Company) today announced updates to be presented at the 41^st Annual J.P. Morgan Healthcare conference including commercial launch progress, 2023 program milestones and financial outlook. CEO Andrew Obenshain is scheduled to speak on Thursday, January 12 at 7:30 a.m. PT.

“In 2022, bluebird bio emerged as a commercial stage gene therapy company, with a validated lentiviral vector platform built on more than a decade of scientific leadership,” said Andrew Obenshain, chief executive officer, bluebird bio. “In a dynamic and challenging environment, bluebird has hit all its operational milestones, including nearly simultaneous launches of two first-in-class gene therapies, ZYNTEGLO and SKYSONA. We have strengthened our financial position and generated considerable momentum as we prepare for the opportunity ahead: bringing lovo-cel gene therapy to individuals living with sickle cell disease and their families.”

Highlights from the Company’s Update Include:

Significant Momentum and Early Signs of Uptake for ZYNTEGLO® (betibeglogene autotemcel) Commercial Launch

• Apheresis for first commercial ZYNTEGLO patient completed; Company entered 2023 with multiple patient cell collections scheduled. First revenue anticipated for Q1 2023 as previously guided.
• Early indicators continue to demonstrate significant patient demand for ZYNTEGLO. Approximately 40 patients have initiated benefits verification, with a significant proportion progressing to prior authorization approval across both Medicaid and commercial payer segments and zero ultimate denials to date.
• bluebird’s qualified treatment center (QTC) network is scaling as planned, with 10 activated centers to date. More than 15 additional QTCs are in the on-boarding or master service agreement negotiation stage, aligned with the Company’s expectation to scale to 40-50 centers by the end of 2023.
• Outcomes-based agreements are in place with 3 of the largest pharmacy benefit managers in the US, representing dozens of downstream national and regional plans. To date, approximately 190 million US lives are covered by a favorable coverage policy for ZYNTEGLO.
• During ZYNTEGLO’s 2023 launch year, the Company expects to assess key metrics over time as launch progresses such as number of patient starts and percent of lives covered and does not expect to provide ZYNTEGLO revenue projections this year.

SKYSONA® (elivaldogene autotemcel) Commercial Launch on Track

• Cell collection for first patient to be treated commercially with SKYSONA scheduled for January 2023.

Clear path to lovotibeglogene autotemcel (lovo-cel) BLA submission in Q1 2023

• bluebird completed vector and drug product analytical comparability studies in the 4^th quarter of 2022, as previously guided. With this progress, bluebird remains on track to submit its biologics license application (BLA) to the U.S. Food and Drug Administration this quarter.

2023 Financial Outlook

The Company’s preliminary unaudited cash and cash equivalents and marketable securities balance was approximately $182 million, excluding restricted cash of approximately $45 million, as of December 31, 2022. As bluebird bio launches two first-in-class gene therapies and readies its third investigational gene therapy for SCD for the commercial setting, full-year 2023 cash burn is expected to be in the range of $270-$300 million. bluebird expects its cash, cash equivalents, restricted cash and marketable securities, including the proceeds from the sale of its second priority review voucher (PRV) for $95 million, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the first quarter of 2024.

Presentation at the 2023 J.P. Morgan Healthcare Conference

Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update on Thursday, January 12, at 7:30 a.m. PT. 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, 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 clinical and research 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—setting the standard for 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. ​

For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube.

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, including our statements regarding our projected financial position and performance in 2022 including expected cash burn, potential cash inflows and cash runway, the timing and expectations for the commercial launches of ZYNTEGLO and SKYSONA and the number of anticipated QTCs by year end 2023, and our expectations regarding the potential BLA submission for lovo-cel in the first quarter of 2023. Such forward-looking statements are based on historical performance and current expectations and projections about our 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 our control and could cause our 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, 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 and uncertainties include, but are not limited to: the risk that regulatory approval of beti-cel and/or eli-cel will not be obtained on the timing that we expect or at all; the partial clinical hold of lovo-cel will not be resolved on the timeframe that we expect or at all; our plans and expectations with respect to our operations and financial performance, the potential commercial launches, and our ability to execute on our strategy will be negatively impacted by the ongoing COVID-19 pandemic and the effects on us and the third parties upon which we depend, including our contract manufacturing organizations and clinical trial sites. 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/20230109005284/en/