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Dec 31, 2019
Bluebird Bio Q4 2019 Earnings Report
Reported financial results for the fourth quarter and full year ended December 31, 2019, and highlighted operational progress, including the first conditional approval of ZYNTEGLO in Europe and positive top-line data from the pivotal Phase 2 KarMMa study of ide-cel.
Key Takeaways
Bluebird Bio reported fourth-quarter financial results, with collaboration and license and royalty revenues of $10.0 million and a net loss of $223.3 million. The company ended the quarter with $1.24 billion in cash, cash equivalents, and marketable securities, and made significant progress in its clinical programs, including the launch of ZYNTEGLO in Germany and positive data from the KarMMa study.
First conditional approval of ZYNTEGLO achieved in Europe, with Germany launch underway.
Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma.
Presented clinical data across studies of LentiGlobin gene therapy for β-thalassemia and sickle cell disease, and bb21217 in multiple myeloma at ASH Annual Meeting.
Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities.
Bluebird Bio
Bluebird Bio
Forward Guidance
bluebird bio anticipates several milestones in regulatory submissions, clinical data presentations, and commercial and foundation building.
Positive Outlook
- Submission of a BLA to the U.S. FDA for ide-cel in patients with relapsed and refractory multiple myeloma in the first half of 2020, in partnership with Bristol-Myers Squibb.
- Submission of a BLA to the U.S. FDA and a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
- Submission for presentation of ide-cel clinical data from the KarMMa study in the first half of 2020, in partnership with Bristol-Myers Squibb.
- Initiation of the Phase 3 HGB-210 study of LentiGlobin for SCD in patients with a history of vaso-occlusive crises in the first half of 2020.
- ZYNTEGLO first commercial patients treated in the first half of 2020.
Challenges Ahead
- Risk that preliminary efficacy and safety results from clinical trials will not continue or be repeated.
- Risk of cessation or delay of ongoing or planned clinical studies and/or product development.
- Risk that current or planned clinical trials will be insufficient to support regulatory submissions or marketing approval.
- Risk that regulatory authorities will require additional information, delaying anticipated timelines for regulatory submissions.
- Risk of challenges in the commercial launch of ZYNTEGLO in the European Union.