On January 09, 2023 bluebird bio, Inc. (Nasdaq: BLUE) (the Company) reported updates to be presented at the 41st 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 (Press release, bluebird bio, JAN 9, 2023, View Source [SID1234626037]).

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"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 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 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 View Source A replay of the webcast will be available on the bluebird bio website for 30 days following the event.