bluebird bio, Inc. (Nasdaq: BLUE) today announced planned updates to be presented at the 40th Annual J.P. Morgan Healthcare conference including 2022 program milestones and financial outlook.
In 2022, bluebird is focused on the FDA review of two gene therapies – betibeglogene autotemcel (beti-cel), for beta-thalassemia and elivaldogene autotemcel (eli-cel), for cerebral adrenoleukodystrophy (CALD) – and is prepared to launch both beti-cel and eli-cel for patients in the U.S. in mid-2022, if approved by the FDA. An FDA Advisory Committee to discuss the Biologics License Application (BLA) for beti-cel is anticipated on March 9, 2022.
Additionally, bluebird bio expects to complete the manufacturing of commercial drug product validation lots for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for sickle cell disease in mid-2022. The Company is evaluating what impact, if any, the ongoing partial clinical hold of lovo-cel for patients under the age of 18 may have on the projected timing of the first quarter of 2023 for the submission of the BLA.
“In 2021, bluebird bio relaunched as an independent company focused on severe genetic diseases,” said Andrew Obenshain, chief executive officer, bluebird bio. “We believe in the potential of our one-time therapies to transform the lives of patients and their families and to give them more bluebird days together. This year, we intend to realize the promise of lentiviral vector gene therapy in the U.S. with the launch of beti-cel for beta-thalassemia and eli-cel for cerebral adrenoleukodystrophy, and to lay the foundation for lovo-cel to be the first gene therapy for people living with sickle cell disease.”
2022 Financial Outlook
The Company’s preliminary unaudited restricted cash, cash and cash equivalents and marketable securities balance was approximately $442 million, including restricted cash of approximately $46 million, as of December 31, 2021. As bluebird bio advances its late-stage pipeline assets to the commercial setting, full-year 2022 cash burn is expected to be less than $400 million. The Company plans to explore the sale of priority review vouchers which would be expected to be issued with the U.S. approvals of BLAs for beti-cel and eli-cel. Approximately $150-200 million in non-dilutive cash inflows could potentially be realized from the sales, which would extend the current cash runway into 2023. The Company is also exploring multiple additional financing opportunities, while focusing on further cost efficiencies.
Presentation at the 2022 J.P. Morgan Healthcare Conference
The bluebird bio management team will present on Wednesday, January 12, at 3:45 p.m. ET. 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.
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