“Undoubtedly the highlight of last quarter at bluebird was the approval of Abecma, the first and only CAR T therapy approved for the treatment of relapsed or refractory multiple myeloma,” said
BUSINESS SEPARATION UPDATE
Today, bluebird bio is providing additional detail regarding the company’s planned business separation, which is targeted for completion by year-end 2021.
bluebird bio
bluebird bio is announcing that
Additional members of the bluebird bio leadership team focused on severe genetic diseases will include:
-
Andrew Obenshain , chief executive officer (previously-disclosed) -
Jason Cole , chief business officer -
Rich Colvin , interim chief medical officer -
Anne-Virginie Eggiman , senior vice president, regulatory science
“As we move towards separation, I’m pleased to have key leadership team members in place that are poised to bring bluebird bio to its next phase of success,” said
Oncology NewCo – 2seventy bio
Today, the company is announcing that Oncology NewCo will be named 2seventy bio and members of the leadership team will include:
-
Nick Leschly , chief executive officer (previously-disclosed) -
Chip Baird , chief financial officer -
Philip Gregory , chief scientific officer -
Nicola Heffron , chief operating officer
“Two hundred seventy miles per hour is the maximum speed of human thought,” said
RECENT HIGHLIGHTS
MULTIPLE MYELOMA
-
ABECMA FDA APPROVAL – On
March 26, 2021 , bluebird bio and Bristol-Myers Squibb announced that theU.S. Food and Drug Administration (FDA) approved Abecma (idecabtagene vicleucel; ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is a personalized immune cell therapy approved as a one-time infusion with a recommended dose range of 300 to 460 x 106 CAR-positive T cells.
-
KARMMA NEJM PUBLICATION– On
February 24, 2021 , bluebird bio and Bristol-Myers Squibb announced that results from the pivotal Phase 2 KarMMa study were published inThe New England Journal of Medicine . The KarMMa study met its primary endpoint of overall response rate and key secondary endpoint of complete response rate. The data from the study demonstrates deep and durable responses with ide-cel treatment in triple-class exposed RRMM patients (n=128).
SICKLE CELL DISEASE
-
CLINICAL STUDIES UPDATE – On
April 20, 2021 , bluebird bio announced a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111). Upon further assessment, the treating investigator concluded this is not a case of MDS and revised the diagnosis to transfusion-dependent anemia. In addition, onMarch 10, 2021 , bluebird bio reported that it is very unlikely the suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector (LVV). bluebird bio continues to work with regulators to resume its clinical studies in sickle cell disease as well as to remove the clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel for β-thalassemia, with potential lift of all clinical holds in mid-2021.
CEREBRAL ADRENOLEUKODYSTROPHY
-
ELI-CEL DATA AT EBMT– On
March 15, 2021 , bluebird bio presented new data suggesting durability of response and a strong safety profile post elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 47th Annual Meeting of theEuropean Society for Blood and Marrow Transplantation (EBMT 2021). Long-term results from the Phase 2/3 Starbeam study of eli-cel, showed that ninety percent of patients (27/30) are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up. In the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104) there were no reports of graft failure, graft rejection, GVHD, replication competent lentivirus or insertional oncogenesis.
PIPELINE
-
BILL & MELINDA GATES FOUNDATION GRANT TO EXPLORE IN-VIVO LVV APPLICATIONS - bluebird bio is announcing today that it has received a grant from the
Bill & Melinda Gates Foundation to explore new, potentially transformative in vivo treatments for SCD using the Company’s proprietary lentiviral vector (LVV) platform. The funding will support the research and development of novel LVVs that target hematopoietic stem cells (HSCs) for in vivo administration to bring gene-based therapies for SCD and other potential indications to patients around the world who may have limited access to ex vivo and other emerging therapies. This research may also enable the application of in-vivo LVV approaches in other severe genetic diseases.
-
PSIOXUS PRE-CLINICAL DATA - On
April 14, 2021 , bluebird bio andPsiOxus Therapeutics presented preclinical data at theAmerican Association for Cancer Research (AACR) Annual Meeting 2021. The results showed synergistic activity between PsiOxus’ T-SIGn vector and bluebird bio’s CAR-T therapy in primary and metastatic solid tumors. A single IV cycle of PsiOxus’ T-SIGn vector enabled an otherwise non-effective dose of CAR-T cell therapy to clear primary and metastatic tumors in vivo.
COMPANY
-
BUSINESS OPERATIONS – On
April 20, 2021 bluebird bio announced its decision to withdraw ZYNTEGLO™ (betibeglogene autotemcel, beti-cel) for transfusion-dependent β-thalassemia (TDT) from the German market because reimbursement negotiations inGermany did not result in a price for ZYNTEGLO that reflects the value of this one-time gene therapy with potential life-long benefit for people living with TDT. Due in part to this decision, the company also announced a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs. This reduction and reallocation of resources will allow the company to focus on priority European markets and streamline global operations going forward to ensure its ability to deliver gene therapies to patients.
UPCOMING ANTICIPATED MILESTONES
Regulatory Outlook
- SCD: The company is investigating the recently-reported safety events and plans to continue to work closely with the FDA in their review of these events to provide an update on the Company’s development plan and timeline for submission for regulatory approval by year end.
-
TDT: The company is on track to complete its rolling BLA submission to the
U.S. FDA for beti-cel in mid-2021, contingent upon successful resolution of anyU.S. FDA concerns applicable to the program arising out of the recently-reported safety events in the SCD program. This submission is anticipated to include adult, adolescent and pediatric patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes). -
CALD: The company is on track to complete its BLA submission to the
U.S. FDA for eli-cel in mid-2021. The company plans to receive European approval for eli-cel in patients with CALD in mid-2021.
Clinical Updates and Milestones
- Updated data from ongoing clinical study in patients with SCD by the end of 2021.
- Updated data from ongoing clinical studies in patients with TDT in mid-2021.
-
Updated clinical data from the ongoing pivotal Phase 2 KarMMa study of Abecma (ide-cel, bb2121) in patients with relapsed and refractory multiple myeloma to be presented at the
American Society of Clinical Oncology 2021 (ASCO21) Virtual Scientific Program onJune 4 . - bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2021.
- Submission of 1 - 2 investigational new drug (IND) applications by the end of 2021.
- Abecma first commercial patients treated in the first half of 2021.
Company
- bluebird bio anticipates the separation of its severe genetic disease and oncology businesses into two independent, publicly traded companies (bluebird bio and 2seventy bio) to be completed by the end of 2021.
FIRST QUARTER 2021 FINANCIAL RESULTS
-
Cash Position: Cash, cash equivalents and marketable securities as of
March 31, 2021 andDecember 31, 2020 were$1.09 billion and$1.27 billion , respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities. -
Revenues: Total revenues were
$12.8 million for the three months endedMarch 31, 2021 compared to$21.9 million for the three months endedMarch 31, 2020 . The decrease was primarily driven by a decrease in ide-cel license and manufacturing services revenue and a decrease in revenue recognized in connection with treating patients in the Phase 1 CRB-402 study of bb21217 under our agreements with BMS. -
R&D Expenses: Research and development expenses were
$154.5 million for the three months endedMarch 31, 2021 compared to$154.1 million for the three months endedMarch 31, 2020 . The increase was primarily driven by increased costs incurred through the amended BMS collaboration as well as an increase in employee compensation, benefit, and other headcount related expenses. These increased costs were partially offset by a decrease in manufacturing costs. -
SG&A Expenses: Selling, general and administrative expenses were
$86.9 million for the three months endedMarch 31, 2021 compared to$73.2 million for the three months endedMarch 31, 2020 . The increase was primarily driven by increased employee compensation, benefit, and other headcount related expenses, as well as an increase in consulting fees associated with the ongoing project to separate the Company’s severe genetic disease and oncology programs into two independently traded companies. These increased costs were partially offset by a decrease in costs related to commercial readiness activities due to delays in commercialization as a result of the COVID-19 pandemic and in light of safety events in the HGB-206 study of LentiGlobin gene therapy for SCD. -
Net Loss: Net loss was
$205.8 million for the three months endedMarch 31, 2021 compared to$202.6 million for the three months endedMarch 31, 2020 .
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.
ZYNTEGLO, LentiGlobin, bluebird bio, 2seventy and 2seventy bio are trademarks of bluebird bio, Inc.
Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company’s financial condition, results of operations, as well as statements regarding the Company’s timing and expectations regarding its investigation of the relationship of the safety events in HGB-206 to the use of lentiviral vector BB305 in LentiGlobin gene therapy for SCD, and any regulatory interactions for lifting the clinical hold; its expectations for commercialization efforts for ZYNTEGLO in
bluebird bio, Inc. Condensed Consolidated Statements of Operations (in thousands, except per share data) (unaudited) |
|||||||
|
For the three months ended
|
||||||
|
2021 |
|
2020 |
||||
Revenue: |
|
|
|
||||
Service revenue |
$ |
5,918 |
|
|
$ |
16,833 |
|
Collaborative arrangement revenue |
1,519 |
|
|
2,302 |
|
||
Royalty and other revenue |
5,357 |
|
|
2,728 |
|
||
Total revenues |
12,794 |
|
|
21,863 |
|
||
Operating expenses: |
|
|
|
||||
Research and development |
154,478 |
|
|
154,123 |
|
||
Selling, general and administrative |
86,874 |
|
|
73,248 |
|
||
Cost of royalty and other revenue |
2,281 |
|
|
1,025 |
|
||
Change in fair value of contingent consideration |
369 |
|
|
(3,108) |
|
||
Total operating expenses |
244,002 |
|
|
225,288 |
|
||
Loss from operations |
(231,208) |
|
|
(203,425) |
|
||
Interest income, net |
710 |
|
|
5,355 |
|
||
Other income (expense), net |
24,756 |
|
|
(4,447) |
|
||
Loss before income taxes |
(205,742) |
|
|
(202,517) |
|
||
Income tax expense |
(66) |
|
|
(94) |
|
||
Net loss |
$ |
(205,808) |
|
|
$ |
(202,611) |
|
Net loss per share - basic and diluted: |
$ |
(3.07) |
|
|
$ |
(3.64) |
|
Weighted-average number of common shares used in computing net loss
|
66,976 |
|
|
55,590 |
|
bluebird bio, Inc. Condensed Consolidated Balance Sheet Data (in thousands, except per share data) (unaudited) |
|||||
|
As of
|
|
As of
|
||
Cash, cash equivalents and marketable securities |
$ |
1,093,551 |
|
$ |
1,274,142 |
Total assets |
$ |
1,637,279 |
|
$ |
1,781,252 |
Total liabilities |
$ |
436,946 |
|
$ |
426,196 |
Total stockholders’ equity |
$ |
1,200,333 |
|
$ |
1,355,056 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20210505006054/en/
Investors & Media
Investors:
epingpank@bluebirdbio.com
Media:
jsnyder@bluebirdbio.com
cfalcetti@bluebirdbio.com
Source: bluebird bio, Inc.