-- Treated first patient in CRB-401 study of anti-BCMA CAR T bb2121 in relapsed/refractory multiple myeloma --
-- Announced that
-- Presented LentiGlobin clinical data in transfusion-dependent
ß-thalassemia and severe sickle cell disease at the
-- Presented pre-clinical and manufacturing data from oncology pipeline at ASH annual meeting --
-- Signed exclusive license agreement with
-- Ended year with
“In 2015 bluebird bio defined an accelerated regulatory path for
LentiGlobin in ß-thalassemia and established a powerful reason to
believe in LentiGlobin in sickle cell disease, though there is still
more to learn as we treat additional patients. We also fully enrolled
our Starbeam study of Lenti-D in cerebral adrenoleukodystrophy and made
significant advances in building a competitive T cell oncology
ADVANCED FIRST ONCOLOGY PROGRAM INTO THE CLINIC – Earlier in
February, the first patient was infused in the CRB-401 study of bb2121
in relapsed/refractory multiple myeloma. Additionally,
Celgenehas exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies. bluebird bio will receive a $10.0 millionoption exercise payment from Celgeneand is also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.
PRESENTED UPDATED CLINICAL DATA IN ß-THALASSEMIA FROM HGB-204 AND
HGB-205 CLINICAL STUDIES OF LENTIGLOBIN AT ASH – In
December 2015, investigators presented data from bluebird bio’s ongoing clinical studies in transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD). Data in patients with TDT from the HGB-204 and HGB-205 studies showed that 100% of patients with non-ß0/ß0 genotypes achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 months to 23.4 months. Patients with the ß0/ß0 genotype all saw reductions in their transfusion needs, ranging from a 33% to 100% reduction.
PRESENTED CLINICAL DATA IN SICKLE CELL DISEASE FROM HGB-205 AND
HGB-206 CLINICAL STUDIES OF LENTIGLOBIN AT ASH –Marina Cavazzana,
M.D., Ph.D., of Hospital Necker, University Paris Descartes, presented
updated data on one patient with SCD from the HGB-205 study, who
remained free of transfusions, hospitalizations and acute SCD-related
events for more than nine months as of the data cut-off. At the
12-month post-drug infusion follow-up, the proportion of anti-sickling
hemoglobin in this patient accounted for 49% (47% HbAT87Q +
2% HbF) of all hemoglobin production – well above the 30% threshold
anticipated to achieve a disease-modifying effect.
John Tisdale, M.D., of the National Institutes of Healthpresented early data from the HGB-206 study, in which two patients had at least three months of post-infusion follow-up. At the three-month post-infusion follow-up for Subject 1301, anti-sickling hemoglobin accounted for 17% of all hemoglobin production (4% HbAT87Q + 13% HbF). At the six-month post-infusion follow-up for subject 1303, anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12% HbAT87Q + 4% HbF).
- PRESENTED PRE-CLINICAL AND MANUFACTURING DATA FROM CAR T ONCOLOGY PROGRAMS AT ASH – bluebird bio scientists presented three posters at ASH, covering critical basic research, translational and manufacturing aspects of the Company’s T cell oncology programs. One poster discussed an important observation made by bluebird bio scientists: culturing anti-BCMA CAR T cells with a PI3K inhibitor generated a product with many of the properties of younger, less differentiated T cells. Consistent with a younger T cell phenotype, this product showed improved in vivo efficacy and persistence in multiple model systems.
SHARED DATA ON PLATFORM IMPROVEMENTS – In an investor event at
ASH, bluebird bio chief scientific officer
Philip Gregory, D.Phil., presented data from ongoing research to improve the cell transduction process for LentiGlobin. The presentation showed that in preclinical experiments, adding selected compounds to the transduction process resulted in substantially increased vector copy number and transduction efficiency (i.e. percentage of corrected cells). Importantly, the new process was shown to be robust with similar improvements seen across multiple donors and vector lots.
ENTERED INTO CAR T LICENSE WITH VIROMED – Signed exclusive
license agreement with
Viromed Co., Ltd., to research, develop and commercialize CAR T therapies using Viromed’s proprietary humanized antibody to an undisclosed cancer target in solid tumors.
Upcoming Anticipated Milestones
Presentation of interim data from the Starbeam study of Lenti-D in
patients with cerebral adrenoleukodystrophy (CALD) at the
American Academy of Neurologyannual meeting in April 2016
- Update on LentiGlobin process improvements in the second half of 2016
- Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016
Presentation of updated data from the HGB-204, HGB-205 and HGB-206
studies at the ASH annual meeting in
Fourth Quarter and Full Year 2015 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
December 31, 2015were $865.8 million, compared to $492.0 millionas of December 31, 2014, an increase of $373.8 million, which was primarily driven by the June 2015equity financing partially offset by cash used to fund operations.
Revenues: Collaboration revenue was
$1.5 millionfor the fourth quarter of 2015 and $14.1 millionfor the year ended December 31, 2015, compared to $6.3 millionand $25.0 millionin the comparable periods in 2014. The decrease is a result of an amendment to our collaboration agreement with Celgenein the second quarter of 2015.
R&D Expenses: Research and development expenses were
$35.7 millionfor the fourth quarter of 2015 and $134.0 millionfor the year ended December 31, 2015, compared to $20.5 millionand $62.6 millionfor the comparable periods in 2014. The increase in research and development expenses was primarily attributable to increased employee compensation expense due to increased headcount, in-licensing milestones and fees, and manufacturing and clinical trial-related costs to support our advancing pipeline.
G&A Expenses: General and administrative expenses were
$14.4 millionfor the fourth quarter of 2015 and $46.2 millionfor the year ended December 31, 2015, compared to $5.3 millionand $23.2 millionfor the comparable periods in 2014. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting and facilities-related costs to support our overall growth.
Net Loss: Net loss was
$47.3 millionfor the fourth quarter of 2015 and $166.8 millionfor the year ended December 31, 2015, compared to net loss of $19.5 millionand $48.7 millionfor the comparable periods in 2014.
Financial guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of
$865.8 millionas of December 31, 2015will be sufficient to fund its current operations through 2018.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin® BB305 product candidate, currently in three clinical studies for the treatment of transfusion-dependent ß-thalassemia, also known as ß-thalassemia major, and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with
bluebird bio has operations in
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
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 and results of operations, the sufficiency of its cash, cash equivalents and marketable securities, as well as the advancement of, and anticipated development and regulatory milestones and plans related to the Company’s product candidates and clinical studies. 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 risk of cessation or delay of any of the ongoing or planned clinical studies or development activities for our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk that our collaborations will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the
Availability of other information about bluebird bio
Investors and others should note that we communicate with our investors and the public using our company website (www.bluebirdbio.com), including but not limited to investor presentations and FAQs,
bluebird bio, Inc.
Three months ended
|Research and license fees||—||105||—||390|
|Research and development||35,659||20,530||134,038||62,574|
|General and administrative||14,444||5,302||46,209||23,227|
|Change in fair value of contingent consideration||329||168||2,869||246|
|Total operating expenses||50,432||26,000||183,116||86,047|
|Loss from operations||(48,961)||(19,614)||(169,037)||(60,626)|
|Total other income (expense), net||1,684||70||2,314||120|
|Loss before income taxes||(47,277)||(19,544)||(166,723)||(60,506)|
|Income tax (expense) benefit||—||—||(60)||11,797|
|Net loss per share - basic and diluted:||$||(1.29)||$||(0.67)||$||(4.81)||$||(1.83)|
|Weighted-average number of common shares used in computing net loss per share - basic and diluted:||36,716||29,373||34,669||26,546|
bluebird bio, Inc.
|December 31,||December 31,|
|Cash and cash equivalents||$||164,269||$||347,845|
|Prepaid expenses and other current assets||6,016||6,434|
|Total current assets||523,965||479,989|
|Property and equipment, net||82,614||15,740|
|Intangible assets, net||24,456||28,219|
|Restricted cash and other non-current assets||10,360||1,215|
|Liabilities and stockholders' equity|
|Accrued expenses and other current liabilities||28,145||14,649|
|Deferred revenue, current portion||5,889||25,375|
|Total current liabilities||40,368||42,978|
|Deferred rent, net of current portion||8,294||8,674|
|Deferred revenue, net of current portion||35,959||5,302|
|Contingent consideration, net of current portion||5,082||6,321|
|Construction financing lease obligation||61,901||—|
|Other non-current liabilities||237||2,207|
Common stock, $0.01 par value, 125,000 shares authorized;
36,894 and 32,340 shares issued and outstanding at December
31, 2015 and December 31, 2014, respectively
|Additional paid-in capital||1,166,585||638,389|
|Accumulated other comprehensive income (loss)||(2,291)||(71)|
|Total stockholders' equity||850,496||491,257|
|Total liabilities and stockholders' equity||$||1,002,337||$||556,739|
Source: bluebird bio, Inc.
Investors and Media:
bluebird bio, Inc.
Manisha Pai, 617-245-2107
Pure Communications, Inc.
Dan Budwick, 973-271-6085