– Received positive opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have β0/β0 genotype –
– Company hosting Analyst Day on
– Ended quarter with
“In the first quarter, we came one step closer to bringing ZYNTEGLO™, the
first gene therapy for transfusion-dependent β-thalassemia (TDT), to
patients with a positive opinion from the CHMP and potential approval in
- CHMP POSITIVE OPINION – In
March 2019, the Committee for Medicinal Products for Human Use(CHMP) of the European Medicines Agency(EMA) adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding β A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. If approved, ZYNTEGLO, formerly referred to as LentiGlobin™ for TDT, will be the first commercially available gene therapy to treat TDT. The CHMP’s positive opinion will now be reviewed by the European Commission(EC), which has the authority to grant marketing authorization for ZYNTEGLO in the European Union(EU).
- ALD-104 – In
April 2019, bluebird bio treated the first patient in ALD-104, the company’s international, non-randomized, open-label, multi-site Phase 3 study of Lenti-D Drug Product after myeloablative conditioning using busulfan and fludarabine in patients ≤17 years of age with cerebral adrenoleukodystrophy (CALD). The study will enroll approximately 20 patients. The primary endpoint of the study is the proportion of patients who are alive and do not have any of the 6 major functional disabilities (MFDs) at Month 24.
- NEJM PUBLICATION – In
May 2019, bluebird bio and Celgeneannounced that the New England Journal of Medicine(NEJM) has published interim results from CRB-401, the ongoing phase 1 study of idecabtagene vicleucel (ide-cel, formerly known as bb2121), the companies’ lead investigational BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy candidate for patients with relapsed and refractory multiple myeloma.
- MANAGEMENT APPOINTMENT – In
April 2019, bluebird bio announced that Joanne Smith-Farrellhas been appointed chief business officer. In this role, Joanne will lead corporate development and strategy, alliance management, and she will also continue to serve as our oncology franchise leader. Joanne has been promoted to chief business officer after joining bluebird in April 2017as our senior vice president, corporate development and strategy.
- bRT OPENING – In
March 2019, bluebird bio announced the official opening of its first wholly owned manufacturing facility in Durham, N.C., that will produce lentiviral vector for the company’s investigational gene and cell therapies, including: ide-cel and bb21217 for the treatment of multiple myeloma and potentially LentiGlobin™ for the treatment of TDT and SCD. bluebird bio purchased the facility in November 2017.
Upcoming Anticipated Milestones
- European approval of ZYNTEGLO in patients with TDT and non-β0/β0 genotypes in Q2
Submission of Biologics Licensing Application to the U.S.
FDAfor ZYNTEGLO in patients with TDT and non-β0/β0 genotypes by the end of 2019
- Presentation of ZYNTEGLO clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes by mid-2019 and by end of 2019
- Presentation of ZYNTEGLO clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype by mid-2019 and by end of 2019
- Initiation of Phase 3 HGB-210 study of LentiGlobin in patients with SCD by end of 2019
- Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD by mid-2019 and by end of 2019
- Multiple Myeloma
- Presentation of ide-cel clinical data from the registration-enabling KarMMa study and CRB-401 study in patients with relapsed/refractory multiple myeloma by end of 2019
- Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma by the end of 2019
First Quarter 2019 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities
March 31, 2019and December 31, 2018were $1.73 billionand $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of normal operating activities and cash used to purchase property, plant and equipment as the company continues the buildout of its manufacturing facility in Durham, North Carolina.
- Revenues: Total revenues were
$12.5 millionfor the three months ended March 31, 2019compared to $16.0 millionfor the three months ended March 31, 2018. The decrease was primarily attributed to decreased manufacturing services under the company’s agreement with Celgene Corporation, offset by increased license and royalty revenue.
- R&D Expenses: Research and development expenses were
$122.6 millionfor the three months ended March 31, 2019compared to $97.1 millionfor the three months ended March 31, 2018. The increase was primarily driven by costs incurred to advance and expand the company’s pipeline.
- G&A Expenses: General and administrative expenses were
$60.3 millionfor the three months ended March 31, 2019compared to $34.9 millionfor the three months ended March 31, 2018. The increase was largely attributable to overall growth of the pipeline as well as commercial-readiness activities.
- Net Loss: Net loss was
$164.4 millionfor the three months ended March 31, 2019compared to $115.1 millionfor the three months ended March 31, 2018.
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 by
researching cerebral adrenoleukodystrophy, sickle cell disease,
transfusion-dependent 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
ZYNTEGLO and LentiGlobin are trademarks of bluebird bio, Inc..
The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene.
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 anticipated development for the company’s product candidates, including anticipated regulatory milestones, potential commercial launches, planned clinical studies, as well as the company’s intentions regarding the timing for providing further updates on the development and commercialization of its product candidates. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of our product candidates will not continue or be repeated in our ongoing clinical trials, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, risks that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the
bluebird bio, Inc.
|For the three months ended March 31,|
|License and royalty revenue||$||1,294||349|
|Research and development||122,640||97,109|
|General and administrative||60,279||34,926|
|Cost of license and royalty revenue||430||17|
|Change in fair value of contingent consideration||296||534|
|Total operating expenses||183,645||132,586|
|Loss from operations||(171,174||)||(116,629||)|
|Interest income, net||10,102||1,388|
|Other (expense) income, net||(3,389||)||115|
|Loss before income taxes||(164,461||)||(115,126||)|
|Income tax benefit||15||-|
|Net loss per share - basic and diluted:||$||(2.99||)||$||(2.31||)|
|Weighted-average number of common shares used in computing net loss per share - basic and diluted:||54,957||49,923|
bluebird bio, Inc.
|As of||As of|
|March 31,||December 31,|
|Cash, cash equivalents and marketable securities||$||1,730,766||$||1,891,427|
|Total stockholders' equity||1,772,099||1,885,070|
Source: bluebird bio, Inc.
Elizabeth Pingpank, 617-914-8736
Jenn Snyder, 617-448-0281