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Dec 31, 2021
Bluebird Bio Q4 2021 Earnings Report
Reported financial results for the fourth quarter and full year ended December 31, 2021, shared recent operational progress, and provided a corporate update.
Key Takeaways
Bluebird Bio reported fourth-quarter financial results, highlighting the ongoing review of their gene therapies, beti-cel and eli-cel, by the FDA, and the planned BLA submission for lovo-cel in Q1 2023. The company ended the year with $442 million in restricted cash, cash equivalents, and marketable securities.
Beti-cel and eli-cel gene therapies are under review by the FDA.
Lovo-cel BLA submission remains on track for Q1 2023.
Completed final patient infusion in the HGB-206 study.
Ended the year with $442M in restricted cash, cash and cash equivalents and marketable securities.
Bluebird Bio
Bluebird Bio
Forward Guidance
Bluebird Bio anticipates several milestones including resolving the partial clinical hold for lovo-cel, completing manufacturing of commercial drug product validation lots, confirming vector and drug product analytical comparability, and potential FDA approval decisions for beti-cel and eli-cel.
Positive Outlook
- The Company is in active communication with the FDA to resolve the partial clinical hold and resume treating patients under the age of 18.
- The Company plans to complete manufacturing of commercial drug product validation lots by mid-2022.
- The Company expects to confirm vector and drug product analytical comparability by Q4 2022.
- The FDA has set a PDUFA goal date of August 19, 2022, for a decision on the approval of beti-cel in patients with β-thalassemia with commercial launch expected to follow in mid-2022 if approved.
- The FDA has set a PDUFA goal date of September 16, 2022, for a decision on the approval of eli-cel in patients with cerebral adrenoleukodystrophy with commercial launch expected to follow by the end of 2022 if approved.
Challenges Ahead
- The Company's expectation to generate operating losses and negative operating cash flows in the future and the need for additional funding to support its planned operations raise substantial doubt regarding its ability to continue as a going concern for a period of one year after the date that its consolidated financial statements for the year ended December 31, 2021 are issued.
- The risk that the Company may not be able to execute an orderly wind down of European operations with the timing or at a cost that we anticipated.
- The risk that additional insertional oncogenic or other safety events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time.
- The risk that the FDA may impose a clinical hold on additional programs utilizing lentiviral vectors.
- The risk that we may not address the FDA’s concerns regarding eli-cel quickly or at all.