- Alignment with FDA on an accelerated regulatory path for LentiGlobin for sickle cell disease based on HGB-206 study with targeted submission in 2H 2021 -
- Completed submission with Bristol Myers Squibb of Biologics License Application (BLA) for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121) to FDA -
- Amended BMS collaboration including
- Revised Operating Plan achieves cash savings of over
- Company extends cash runway into 2022 -
- Company to host conference call today,
“We remain grounded in our core values and priorities: our patients, our people, our community and our business,” said
SCD REGULATORY PATH
Today, bluebird bio announced general agreement with FDA that the clinical data package required to support a BLA submission for LentiGlobin™ for sickle cell disease (SCD) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint and at least 18 months of follow-up post drug product infusion. Globin response will be used as a key secondary endpoint. The company anticipates additional guidance from FDA regarding the commercial manufacturing process, including suspension lentiviral vector. The company is planning to seek an accelerated approval and expects to submit the
Additionally, to enhance its strategic, clinical and commercial manufacturing platform, bluebird bio has entered into expanded relationships with two subsidiaries of Hitachi Chemical Co., Ltd. (Hitachi Chemical Advanced Therapeutics Solutions and apceth
FINANCIAL AND BUSINESS UPDATES
IDE-CEL ROYALTY MONETIZATION AND BCMA RELATIONSHIP AMENDMENT
Today, bluebird bio announced in a separate press release that it has amended its existing co-promotion/co-development agreement with Bristol Myers Squibb (BMS) to enable the companies to focus their efforts on efficient commercialization of idecabtagene vicleucel (ide-cel; bb2121) in the
REVISED BUSINESS PRIORITIES AND OPERATING PLAN
Given the ongoing impact of the COVID-19 global pandemic and recent shifts in regulatory timelines, bluebird bio has undertaken a comprehensive business review with the goal of ensuring the ability to achieve its 2022 vision with a path towards financial sustainability. Under the revised business priorities and operating plan, bluebird remains on track for potential regulatory approval and commercial launch for ZYNTEGLO, ide-cel, Lenti-D for CALD, and LentiGlobin for SCD by 2022.
Through this comprehensive business review, bluebird bio has prioritized key research and development programs and has made a number of changes to the future cost structure relative to the prior long-range plan, including:
-
Reduced investment in selling, general and administrative expenses, including a deferred investment in building a
U.S. commercial organization, reduced facilities and IT infrastructure, and other cost-reduction measures. - Prioritized investment in R&D expenses, including an indefinite pause of the HGB-211 clinical study in SCD patients at high risk of stroke, adjustment to the timing of investment in ongoing clinical studies to reflect COVID-19 related delays in enrollment, reduction or elimination of investment in certain preclinical programs, and other cost-reduction measures.
-
Nick Leschly , chief bluebird, will decline nearly 100% of his salary for the next 12 months. Similarly, additional members of the bluebird bio senior leadership team and all members of the Company’s Board of Directors will forgo 20% of their salaries or Board cash retainers for the next 12 months. All will receive a grant of restricted stock units equal to 80% of the value of the released cash compensation, which will vest over one year.
In total, these changes are expected to result in over
PREVIOUSLY DISCLOSED RECENT HIGHLIGHTS
-
IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On
March 31, 2020 , bluebird bio and BMS announced the submission of their BLA to theU.S. FDA for ide-cel, the companies’ lead investigational BCMA-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The BLA submission includes results from pivotal KarMMa study evaluating ide-cel in a heavily pre-treated patient population with relapsed and refractory multiple myeloma. -
COVID-19 IMPACT – On
March 26, 2020 , bluebird bio provided an assessment of the impact of the COVID-19 pandemic and outlined steps the company has taken to ensure the safety of its patients and employees, while working to ensure the sustainability of its business operations as this unprecedented situation continues to evolve. Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months and expects timing shifts to vary by clinical trial and by program.
UPCOMING ANTICIPATED MILESTONES
-
Regulatory
-
Submission of a Marketing Authorization Application to the
European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
-
Submission of a Marketing Authorization Application to the
-
Clinical
-
Presentation of ide-cel clinical data from the KarMMa study at the
American Society of Clinical Oncology meeting later this month, in partnership with Bristol-Myers Squibb. -
Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes at the 2020 Annual Congress of the
European Hematology Association (EHA). -
Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-
I-110 mutation at the 2020 AnnualCongress of EHA . -
Updated data presentation from HGB-206 clinical study in patients with SCD at the 2020 Annual
Congress of EHA . - Presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.
- Updated data presentation from ALD-102 in patients with CALD by the end of 2020.
-
Presentation of ide-cel clinical data from the KarMMa study at the
-
Commercial and Foundation Building -
ZYNTEGLO first commercial patients treated in
Europe in the second half of 2020. - ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
-
ZYNTEGLO first commercial patients treated in
FIRST QUARTER 2020 FINANCIAL RESULTS
-
Cash Position: Cash, cash equivalents and marketable securities as of
March 31, 2020 andDecember 31, 2019 were$1.02 billion and$1.24 billion , respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities. -
Revenues: Total revenues were
$21.9 million for the three months endedMarch 31, 2020 compared to$12.5 million for the three months endedMarch 31, 2019 . The increase was primarily attributable to an increase in ide-cel license and manufacturing service revenue under our agreement with BMS, as well as an increase in royalty revenue. -
R&D Expenses: Research and development expenses were
$154.1 million for the three months endedMarch 31, 2020 compared to$122.6 million for the three months endedMarch 31, 2019 . The increase was primarily driven by costs incurred to advance and expand the company’s pipeline. -
SG&A Expenses: Selling, general and administrative expenses were
$73.2 million for the three months endedMarch 31, 2020 compared to$60.3 million for the three months endedMarch 31, 2019 . The increase was largely attributable to costs incurred to support the company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities. -
Net Loss: Net loss was
$202.6 million for the three months endedMarch 31, 2020 compared to$164.4 million for the three months endedMarch 31, 2019 .
CONFERENCE CALL DETAILS
bluebird bio will hold a conference call to discuss business updates and first quarter 2020 financial results on
Investors may listen to the call by dialing (844) 825-4408 from locations in
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 including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.
ZYNTEGLO, LentiGlobin, and bluebird 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 plans for regulatory submissions for the company’s product candidates LentiGlobin for β-thalassemia, LentiGlobin for SCD, and Lenti-D, including anticipated endpoints to support regulatory submissions and timing expectations; the company’s expectations and execution under its revised operating plan, including its cash runway; 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
|
|||||||
2020 |
2019 |
||||||
Revenue: |
|||||||
Service revenue |
$ |
16,833 |
|
$ |
9,211 |
|
|
Collaborative arrangement revenue |
|
2,302 |
|
|
1,966 |
|
|
Royalty revenue |
|
2,728 |
|
|
1,294 |
|
|
Total revenues |
|
21,863 |
|
|
12,471 |
|
|
Operating expenses: |
|||||||
Research and development |
|
154,123 |
|
|
122,640 |
|
|
Selling, general and administrative |
|
73,248 |
|
|
60,279 |
|
|
Cost of royalty revenue |
|
1,025 |
|
|
430 |
|
|
Change in fair value of contingent consideration |
|
(3,108 |
) |
|
296 |
|
|
Total operating expenses |
|
225,288 |
|
|
183,645 |
|
|
Loss from operations |
|
(203,425 |
) |
|
(171,174 |
) |
|
Interest income, net |
|
5,355 |
|
|
10,102 |
|
|
Other expense, net |
|
(4,447 |
) |
|
(3,389 |
) |
|
Loss before income taxes |
|
(202,517 |
) |
|
(164,461 |
) |
|
Income tax (expense) benefit |
|
(94 |
) |
|
15 |
|
|
Net loss |
$ |
(202,611 |
) |
$ |
(164,446 |
) |
|
Net loss per share - basic and diluted: |
$ |
(3.64 |
) |
$ |
(2.99 |
) |
|
Weighted-average number of common shares used in computing net loss per
|
|
55,590 |
|
|
54,957 |
|
bluebird bio, Inc. |
|||||
Condensed Consolidated Balance Sheet Data |
|||||
(in thousands, except per share data) |
|||||
(unaudited) |
|||||
As of |
As of |
||||
|
|
||||
2020 |
2019 |
||||
Cash, cash equivalents and marketable securities |
$ |
1,018,357 |
$ |
1,237,966 |
|
Total assets |
$ |
1,529,104 |
$ |
1,727,424 |
|
Total liabilities |
$ |
408,671 |
$ |
442,431 |
|
Total stockholders' equity |
$ |
1,120,433 |
$ |
1,284,993 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20200511005289/en/
Investors:
igoldberg@bluebirdbio.com
epingpank@bluebirdbio.com
Media:
jsnyder@bluebirdbio.com
Source: bluebird bio, Inc.