-- Presented promising new beta-thalassemia major and severe sickle cell disease data from HGB-205 study at EHA annual meeting --
-- Announced global regulatory strategy for LentiGlobin BB305 in beta-thalassemia major, with plans to pursue conditional and accelerated registration strategies in the E.U. and U.S., respectively --
-- Announced broad T cell oncology strategy and related collaborations --
-- Completed successful public offering of common stock, raising net
“The first half of this year was a period of exceptional progress for
bluebird bio across all fronts, and we made significant strides toward
our goal of delivering transformative gene therapy products to change
the lives of patients,” said
SICKLE CELL DISEASE (SCD) AND BETA-THALASSEMIA DATA PRESENTATION AT
THE 20TH CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION – Presented
positive data from two patients with beta-thalassemia major and the
first patient with severe sickle cell disease ever treated with our
LentiGlobin BB305 product candidate. As of
May 2015, Subjects 1201 and 1202 with beta-thalassemia major remained transfusion-independent for 16 and 14 months, respectively. Subject 1204 with severe SCD demonstrated increasing HbAT87Q production at six months' follow-up and was free of transfusions for more than three months. At the six-month visit post-drug product infusion, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) in the patient with SCD accounted for 45 percent of all hemoglobin production. As of May 2015, the patient with SCD had no hospitalizations for sickle cell complications post-transplant, despite weaning of transfusions. LentiGlobin BB305 was well-tolerated, with no drug product-related adverse events observed as of the May 2015data cut-off.
- LENTIGLOBIN BETA-THALASSEMIA GLOBAL REGULATORY STRATEGY – Announced plan to pursue conditional approval of our LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major in the E.U. through the Adaptive Pathways Pilot Program based on data from the ongoing Northstar and HGB-205 studies and plan to pursue accelerated approval in the U.S. based on our planned HGB-207 and HGB-208 studies. Completed NIH RAC review of HGB-207 and HGB-208 study protocols in adult and adolescent patients with beta-thalassemia major and pediatric patients with beta-thalassemia major, respectively.
IMMUNO-ONCOLOGY STRATEGY – Announced strategy to build a broad
T cell-based immuno-oncology portfolio based on our immuno-oncology,
gene therapy clinical development and lentiviral vector manufacturing
expertise and genome editing capabilities. Revised
Celgenecollaboration to focus exclusively on anti-BCMA product candidates in multiple myeloma, initiated a strategic collaboration with Kite Pharmafocused on second-generation T cell receptor (TCR) therapies in HPV-associated cancers and entered into an exclusive license agreement with Five Prime Therapeuticsaround chimeric antigen receptor (CAR) T cell therapies against an undisclosed cancer target for hematologic malignancies and solid tumors.
CHIEF SCIENTIFIC OFFICER – Hired Philip Gregory, D. Phil., as
Chief Scientific Officer. Formerly Chief Scientific Officer and Senior
Vice President, Research at
Sangamo BioSciences, Dr. Gregory brings extensive expertise in the fields of genome editing and gene therapy.
STRENGTHENED BALANCE SHEET – Raised
$477.2 millionin net proceeds in an equity financing in June 2015. Our cash, cash equivalents and marketable securities are sufficient to fund our operations through 2018, based on the company’s current business plan. Proceeds from the equity financing will fund advancement of our immuno-oncology programs, development of a commercial infrastructure to support a potential conditional commercial launch of LentiGlobin in Europe, expansion of manufacturing capabilities to support ongoing and anticipated development and commercial efforts, and initiation of clinical studies of LentiGlobin in adult, adolescent and pediatric subjects with beta-thalassemia major.
Second Quarter 2015 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
June 30, 2015were $936.4 million, compared to $492.0 millionas of December 31, 2014, an increase of $444.4 million, which was primarily driven by the June 2015equity financing.
Revenues: Collaboration revenue was
$4.9 millionfor the second quarter of 2015 compared to $6.3 millionfor the second quarter of 2014. Collaboration revenue is primarily comprised of the amortization of deferred revenue related to our collaboration agreement with Celgene.
R&D Expenses: Research and development expenses were
$44.3 millionfor the second quarter of 2015, compared to $13.9 millionfor the same period in 2014, an increase of $30.4 million. The increase in research and development expenses was primarily attributable to an $11.0 millionincrease in stock-based compensation expense, of which $8.5 millionis non-recurring, a $10.7 millionincrease in one-time in-license milestones and fees, and an increase in expenses necessary to support the advancement of our clinical and pre-clinical programs.
G&A Expenses: General and administrative expenses were
$10.7 millionfor the second quarter of 2015, compared to $5.7 millionfor the same period in 2014, an increase of $5.0 million. The increase in general and administrative expenses was primarily attributable to a $3.5 millionincrease in employee- and contractor-related costs to support our overall growth.
Net Loss: Net loss was
$51.8 millionfor the second quarter of 2015, compared to net loss of $1.5 millionfor the second quarter of 2014.
Financial guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of
$936.4 millionas of June 30, 2015will be sufficient to fund its operations through 2018, based on the company’s current business plan.
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 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
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.
|Three months ended June 30,||Six months ended June 30,|
|Research and license fees||—||85||—||170|
|Research and development||44,266||13,931||67,985||25,394|
|General and administrative||10,724||5,738||18,060||11,277|
Change in fair value of contingent
|Total operating expenses||56,963||19,669||88,233||36,671|
|Loss from operations||(52,023||)||(13,334||)||(76,949||)||(24,001||)|
|Other income, net||228||11||367||69|
|Loss before income taxes||(51,795||)||(13,323||)||(76,582||)||(23,932||)|
|Benefit from income taxes||—||11,797||—||11,797|
|Net loss per share - basic and diluted:||$||(1.57||)||$||(0.06||)||$||(2.34||)||$||(0.50||)|
Weighted-average number of common
bluebird bio, Inc.
|Cash, cash equivalents and marketable securities||$ 936,445||$ 492,003|
|Total stockholders' equity||918,966||491,257|
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
Pure Communications, Inc.
Dan Budwick, 973-271-6085