– Announced long-term commercial manufacturing agreement with
– Presented data on gene editing platform at
– Received Orphan Drug Designation for bb2121 in Multiple Myeloma –
– Presented interim clinical data from Starbeam study of
Lenti-DTM in cerebral adrenoleukodystrophy
– Presented gene therapy and oncology data at
– Ended quarter with
“We continue to advance our LentiGlobinTM, Lenti-D and bb2121
programs through clinical trials, and in recent months, we’ve been
pleased to showcase some of the innovative platform work we are doing to
continuously improve upon our therapies. At the
MANUFACTURING AGREEMENT WITH LONZA – In June, bluebird and
Lonzaannounced a strategic manufacturing agreement providing for the future commercial production of bluebird bio's Lenti-D™ and LentiGlobin™ drug products. This agreement follows a successful multi-year clinical manufacturing relationship and provides bluebird bio with a path to commercial supply including dedicated production suites within Lonza'sstate-of-the-art facility. Under this multi-year agreement, Lonzawill complete the suite design, construction and validation along with process validation prior to anticipated commercial launch.
GENOME EDITING DATA PRESENTED AT ASH WORKSHOP ON GENOME EDITING – In
July, pre-clinical data from bluebird’s megaTAL genome editing
platform was presented at the
ASH Workshopon Genome Editing in Washington, DC. The data reported at the ASH workshop highlight recent progress bluebird has made in:
- Expanding the number of megaTAL targetable sites in the genome to permit the precise placement of an editing event within a target gene
- Refining the specificity of megaTALs to eliminate undesirable off-target activity
- Combining megaTALs targeting different target genes to achieve the knockout of multiple genes simultaneously
ORPHAN DRUG DESIGNATION GRANTED FOR BB2121 IN MULTIPLE MYELOMA
– In May, the
U.S. Food and Drug Administration( FDA) granted orphan drug designation for bb2121 in multiple myeloma. bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and is being developed by bluebird bio in collaboration with Celgene Corporation. In February, the first patient was infused in the CRB-401 study of anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgeneexercised its option to exclusively license bb2121.
PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN ANNUAL MEETING –
In April, Dr.
Florian Eichlerof Massachusetts General Hospital for Childrenpresented interim clinical data from the Starbeam study of Lenti-D in CALD at AAN. Initial Starbeam results suggest Lenti-D gene therapy may have similar efficacy to allogeneic hematopoietic stem cell transplant (HCT), the current standard of care, with a more favorable safety profile. As of March 31, 2016, three of the 17 patients enrolled in the study have reached two years of follow-up and remain free of major functional disabilities (MFDs), the primary endpoint of the study. Sixteen of the 17 patients had stabilization of their neurological function score (NFS), and 14 of 17 had a stable Loes score. The safety profile of Lenti-D treatment appeared consistent with myeloablative conditioning.
- TEN ABSTRACTS PRESENTED AT ASGCT 19th ANNUAL MEETING – In April, two oral presentations given by bluebird’s academic collaborators highlighted previously presented data from bluebird bio’s ongoing gene therapy clinical trials, including interim data from the Starbeam Study of Lenti-D™ in cerebral adrenoleukodystrophy, and interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and TDT. Eight additional presentations were featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities in both HSC gene therapy and T cell immunotherapy.
Second Half 2016 Anticipated Milestones
- Update on LentiGlobin process improvements
- Initiation of the HGB-207 study in patients with TDT with the non-β0/β0 genotype
Presentation of updated clinical data for LentiGlobin at the ASH
annual meeting in
Second Quarter 2016 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
June 30, 2016were $779.0 million, compared to $865.8 millionas of December 31, 2015, a decrease of $86.8 million.
Revenues: Collaboration revenue was
$1.6 millionfor the second quarter of 2016 compared to $4.9 millionfor second quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgenein June 2015.
R&D Expenses: Research and development expenses were
$41.8 millionfor the second quarter of 2016 compared to $44.3 millionfor the second quarter of 2015. The decrease in research and development expenses was primarily attributable to decreased in-licensing milestones and fees and stock-based compensation expense partially offset by increased employee payroll and facilities costs due to increased headcount, and increased manufacturing, clinical, and information technology costs to support the advancement of our clinical and pre-clinical programs.
G&A Expenses: General and administrative expenses were
$18.4 millionfor the second quarter of 2016 compared to $10.7 millionfor the second quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting costs to support our overall growth.
Net Loss: Net loss was
$58.8 millionfor the second quarter of 2016 compared to $51.8 millionfor the second quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of
$779.0 millionas of June 30, 2016will 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, 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 Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts; Seattle,
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, risks that the
preliminary results from our clinical trials 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, the risk of a delay in the enrollment of
patients in our clinical studies, the risk that our collaboration with
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.
Condensed Consolidated Statements of Operations Data
(in thousands, except per share data)
|Three months ended June 30,||Six months ended June 30,|
|Research and development||41,760||44,266||83,671||67,985|
|General and administrative||18,363||10,724||34,318||18,060|
|Change in fair value of contingent consideration||1,404||1,973||2,417||2,188|
|Total operating expenses||61,527||56,963||120,406||88,233|
|Loss from operations||(59,975)||(52,023)||(117,355)||(76,949)|
|Other income, net||905||228||1,866||367|
|Loss before income taxes||(59,070)||(51,795)||(115,489)||(76,582)|
|Income tax benefit||226||-||371||-|
|Net loss per share - basic and diluted:||$||(1.59)||$||(1.57)||$||(3.12)||$||(2.34)|
|Weighted-average number of common shares used in computing net loss per share - basic and diluted:||36,954||32,955||36,937||32,757|
bluebird bio, Inc.
Condensed Consolidated Balance Sheets Data
|June 30,||December 31,|
|Cash, cash equivalents and marketable securities||$||779,002||$||865,763|
|Total stockholders' equity||759,290||850,496|
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
bluebird bio, Inc.
Elizabeth Pingpank, 617-914-8736
Pure Communications, Inc.
Dan Budwick, 973-271-6085