- 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 -
“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
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
Clear path to lovotibeglogene autotemcel (lovo-cel) BLA submission in Q1 2023
bluebird completed vector and drug product analytical comparability studies in the 4th 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 Administrationthis quarter.
2023 Financial Outlook
The Company’s preliminary unaudited cash and cash equivalents and marketable securities balance was approximately
Presentation at the 2023
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.
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
Source: bluebird bio, Inc.