“Over the last year, bluebird bio’s efforts advanced the field of gene
therapy, driving us closer to our goal of transforming the lives of
patients with severe genetic and rare diseases,” said
Recent bluebird Highlights
Presented data from beta-thalassemia program at the
American Society of Hematology(ASH) annual meeting. In December 2014, bluebird bio presented data from its Northstar and HGB-205 studies demonstrating that the first four beta-thalassemia major patients treated with LentiGlobin® were transfusion free. The Northstar Study is an ongoing, open-label, single-dose, international, multi-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin for the treatment of subjects with beta-thalassemia major. The HGB-205 study is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin in the treatment of subjects with beta-thalassemia major and severe sickle cell disease. The data presented at ASH build upon data presented in June 2014from the HGB-205 study at the European Hematology Association(EHA) annual meeting.
Advanced sickle cell disease program. In
October 2014, as part of the HGB-205 study, bluebird bio became the first company to treat a sickle cell disease patient with gene therapy. The company also initiated the HGB-206 study, an open-label, multi-center, U.S.-based Phase 1 study designed to evaluate the safety and efficacy of LentiGlobin for the treatment of subjects with severe sickle cell disease.
Received Breakthrough Therapy designation for LentiGlobin. In
January 2015, the U.S. Food and Drug Administration( FDA) granted Breakthrough Therapy designation to LentiGlobin for the treatment of transfusion-dependent patients with beta-thalassemia major. The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar and HGB-205 studies of LentiGlobin.
- Advanced enrollment in CCALD trial. bluebird bio continued to enroll patients in its Starbeam Study, a Phase 2/3 study designed to evaluate the safety and efficacy of Lenti-D™ in the treatment of subjects with childhood cerebral adrenoleukodystrophy (CCALD).
Strengthened balance sheet. In 2014, bluebird bio raised
$353 millionin net proceeds to fund operations, including an equity financing in December 2014that raised approximately $243 millionand an equity financing in July 2014that raised approximately $110 million.
Acquired new gene editing capabilities. In
June 2014, bluebird bio acquired Precision Genome Engineering, Inc., or Pregenen, a privately held biotechnology company headquartered in Seattle. This acquisition provides bluebird bio with cutting-edge gene editing capabilities, including expertise in homing endonucleases and MegaTALs.
Expanded the bluebird leadership team. In 2014, bluebird bio
expanded its executive team with the appointments of
Jason Coleas senior vice president and general counsel, and James M. DeToreas chief financial officer and treasurer. The company also added two independent members to its board of directors: James Mandell, M.D., former chief executive officer of Boston Children’s Hospital and Mark Vachon, former president and chief executive officer of GE Healthcare Americas. Additionally, bluebird bio announced that Mitchell Finer, Ph.D. would transition from his role as chief scientific officer to a role as a member of the company’s scientific advisory board.
Continued to advance
Celgene/Baylor CAR T program. bluebird bio continued to make progress under its broad, global strategic collaboration with Celgene Corporation, which is focused on discovering, developing and commercializing novel disease-altering gene therapies in oncology by utilizing a patient’s own genetically modified T cells, known as chimeric antigen receptor (CAR) T cells, to selectively target and destroy cancer cells. The company expects the first product candidate from this program to enter the clinic in early 2016.
Upcoming Anticipated Milestones
bluebird bio has outlined certain key goals for 2015, including:
- Completing enrollment for the Starbeam Study, as well as the Northstar and HGB-205 studies. Continuing enrollment for the HGB-206 study and presenting early clinical efficacy and safety data in patients with severe sickle cell disease at a major medical conference.
- Presenting additional data on beta-thalassemia major from the Northstar and HGB-205 studies at a major medical conference. Based on these additional data, the company looks forward to defining the regulatory path forward for LentiGlobin in beta-thalassemia major this year.
Fourth Quarter and Full Year 2014 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
December 31, 2014were $492.0 million, compared to $206.3 millionas of December 31, 2013. The increase was primarily driven by net proceeds of $353.0 millionfrom equity financings partially offset by cash used to fund operations.
Revenues: Collaboration revenue was
$6.3 millionfor the fourth quarter of 2014 and $25.0 millionfor the year ended December 31, 2014, compared to $6.3 millionand $19.8 millionin the comparable periods in 2013. Collaboration revenue is primarily comprised of the amortization of deferred revenue related to the $75 millionupfront payment received in 2013 under bluebird bio’s collaboration agreement with Celgene.
R&D Expenses: Research and development expenses were
$20.5 millionin the fourth quarter of 2014 and $62.6 millionfor the year ended December 31, 2014, compared to $9.8 millionand $31.0 millionin the comparable periods in 2013. The increase in research and development expenses was largely due to increased spending on clinical and manufacturing activities related to the LentiGlobin and Lenti-D product candidates, as well as research and development efforts on the CAR T and gene editing programs.
G&A Expenses: General and administrative expenses were
$5.3 millionin the fourth quarter of 2014 and $23.2 millionin the year ended December 31, 2014, compared to $4.7 millionand $14.1 millionin the comparable periods in 2013. The increase in general and administrative expenses was largely due to incremental expenses to support public company operations and additional expenses associated with the acquisition of Pregenen.
Net Loss: Net loss was
$19.5 millionfor the fourth quarter of 2014 and $48.7 millionfor the year ended December 31, 2014, compared to net loss of $8.1 millionand $25.3 millionfor the comparable periods in 2013.
- Financial Guidance: bluebird bio expects that its cash, cash equivalents and marketable securities will be sufficient to fund its operations through 2017.
About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities,
bluebird bio has built an integrated product platform with broad
potential application to severe genetic diseases and T cell-based
immunotherapy. bluebird bio’s clinical programs include Lenti-D™,
currently in a Phase 2/3 study, called the Starbeam Study, for the
treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®,
currently in three clinical studies: a global Phase 1/2 study, called
the Northstar Study, for the treatment of beta-thalassemia major; a
single-center Phase 1/2 study in
bluebird bio has operations in
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 milestones
related to the Company’s product candidates and clinical studies, and
anticipated milestones for 2015. 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 and/or our
development of 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 collaboration
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),
our investor relations website (http://www.bluebirdbio.com/investor-splash.html),
including but not limited to investor presentations and FAQs,
bluebird bio, Inc.
Consolidated Statements of Operations Data
(in thousands, except per share data)
|Three months ended|
|December 31,||Year ended December 31,|
|Research and license fees||105||85||390||389|
|Research and development||20,531||9,765||62,574||31,002|
|General and administrative||5,303||4,685||23,227||14,126|
|Change in fair value of contingent consideration||168||—||246||—|
|Total operating expenses||26,002||14,450||86,047||45,128|
|Loss from operations||(19,616||)||(8,115||)||(60,626||)||(24,947||)|
|Total other income (expense), net||72||34||120||(374||)|
|Loss before income taxes||(19,544||)||(8,081||)||(60,506||)||(25,321||)|
|Benefit from income taxes||—||—||11,797||—|
|Net loss per share - basic and diluted:||$||(0.67||)||$||(0.34||)||$||(1.83||)||$||(2.02||)|
Weighted-average number of common shares used in computing net loss per share - basic and diluted:
bluebird bio, Inc.
Consolidated Balance Sheets Data
(in thousands, except par value amounts)
|December 31,||December 31,|
|Cash and cash equivalents||$||347,845||$||206,279|
|Deferred tax assets||1,913||693|
|Prepaid expenses and other current assets||4,521||5,015|
|Total current assets||479,989||211,987|
|Property and equipment, net||15,740||10,920|
|Intangible assets, net||28,219||—|
|Restricted cash and other non-current assets||1,215||1,483|
|Liabilities and stockholders' equity|
|Accrued expenses and other current liabilities||14,649||5,175|
|Deferred revenue, current portion||25,375||25,340|
|Total current liabilities||42,978||34,874|
|Deferred rent, net of current portion||8,674||6,740|
|Deferred revenue, net of current portion||5,302||30,208|
|Contingent consideration, net of current portion||6,321||—|
|Deferred tax liabilities||1,913||693|
|Other non-current liabilities||294||208|
Common stock, $0.01 par value, 125,000 shares authorized; 32,340 and 23,940 shares issued and outstanding at December 31, 2014 and December 31, 2013, respectively
|Additional paid-in capital||638,389||250,103|
|Accumulated other comprehensive loss||(71||)||—|
|Total stockholders' equity||491,257||151,667|
|Total liabilities and stockholders' equity||$||556,739||$||224,390|
Source: bluebird bio, Inc.
bluebird bio, Inc.
Jim DeTore, 339-499-9355
Chief Financial Officer
Pure Communications, Inc.
Dan Budwick, 973-271-6085