-- Initial clinical data from HGB-205 Study in beta-thalassemia major
patients to be presented at the 19th
-- IND active for HGB-206 Study of LentiGlobin product candidate for the treatment of sickle cell disease
"The first quarter of 2014 has been very productive for bluebird bio,"
said
First Quarter 2014 and Recent Business Highlights:
-- Announced that the first patient with beta-thalassemia major had been transplanted in the Northstar Study of our product candidate LentiGlobin
-- Filed an investigational new drug application (IND) with the
-- Our clinical abstract has been accepted for presentation at the 19th
-- Filed an IND amendment to conduct the ALD-103 Study. The ALD-103 Study is an observational study of subjects with childhood cerebral adrenoleukodystrophy (CCALD) treated by allogeneic hematopoietic stem-cell transplant. This study, along with our phase 2/3 Starbeam Study evaluating our product candidate Lenti-D, is intended to provide us with additional information regarding the current standard of care for CCALD and the potential safety and efficacy advantages of Lenti-D
-- Added
Anticipated 2014 Milestones:
-- Present initial clinical results in beta-thalassemia major from our
HGB-205 Study at the 19th
-- First patient with sickle cell disease will be transplanted with our LentiGlobin product candidate in 2014
-- Present additional beta-thalassemia major data from the HGB-205 and Northstar Studies in late 2014.
Financial Results:
Revenue was
Net cash used in operating activities during the three months ended
Total operating expenses for the three months ended
bluebird bio reported a net loss of
About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing
potentially transformative gene therapies for severe genetic and orphan
diseases. bluebird bio has two clinical-stage programs in development.
The most advanced product candidate, Lenti-D, is in a recently-initiated
phase 2/3 study, the Starbeam Study, for the treatment of childhood
cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological
disorder affecting young boys. The next most advanced product candidate,
LentiGlobin, is currently in two phase 1/2 studies, one in the US (the
Northstar Study) and one in
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in collaboration with
bluebird bio has operations in
Forward-Looking Statements
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 and the advancement of, and anticipated milestones related to
the Company’s product candidates and clinical studies, and anticipated
milestones for 2014. 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 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),
our investor relations website (http://www.bluebirdbio.com/investor-splash.html),
including but not limited to investor presentations and FAQs,
| bluebird bio, Inc. | ||||||||||||
| Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||||||||||
| (unaudited) | ||||||||||||
| (in thousands, except per share data) | ||||||||||||
| Three months ended | ||||||||||||
| March 31, | ||||||||||||
| 2014 | 2013 | |||||||||||
| Revenue: | ||||||||||||
| Collaboration revenue | $ | 6,250 | $ | 1,042 | ||||||||
| Research and license fees | 85 | 85 | ||||||||||
| Total revenue | 6,335 | 1,127 | ||||||||||
| Operating expenses: | ||||||||||||
| Research and development | 11,463 | 5,284 | ||||||||||
| General and administrative | 5,540 | 2,324 | ||||||||||
| Total operating expenses | 17,003 | 7,608 | ||||||||||
| Loss from operations | (10,668 | ) | (6,481 | ) | ||||||||
| Other income (expense), net | 59 | (63 | ) | |||||||||
| Net loss | $ | (10,609 | ) | $ | (6,544 | ) | ||||||
| Net loss per share - basic and diluted: | $ | (0.44 | ) | $ | (19.94 | ) | ||||||
| Weighted-average number of common shares used | ||||||||||||
| in net loss per share: | 24,148 | 328 | ||||||||||
| Comprehensive loss | $ | (10,609 | ) | $ | (6,544 | ) | ||||||
| bluebird bio, Inc. | ||||||||||
| Condensed Consolidated Balance Sheets | ||||||||||
| (unaudited) | ||||||||||
| (in thousands, except per share data) | ||||||||||
| March 31, | December 31, | |||||||||
| 2014 | 2013 | |||||||||
| Assets | ||||||||||
| Current assets: | ||||||||||
| Cash and cash equivalents | $ | 192,499 | $ | 206,279 | ||||||
| Deferred tax assets | 693 | 693 | ||||||||
| Prepaid expenses and other current assets | 2,728 | 5,015 | ||||||||
| Total current assets | 195,920 | 211,987 | ||||||||
| Property and equipment, net | 11,984 | 10,920 | ||||||||
| Restricted cash and other non-current assets | 1,606 | 1,483 | ||||||||
| Total assets | $ | 209,510 | $ | 224,390 | ||||||
|
Liabilities and stockholders' equity |
||||||||||
| Current liabilities: | ||||||||||
| Accounts payable | $ | 2,601 | $ | 4,359 | ||||||
| Accrued expenses and other current liabilities | 5,190 | 5,175 | ||||||||
| Deferred revenue, current portion | 25,255 | 25,340 | ||||||||
| Total current liabilities | 33,046 | 34,874 | ||||||||
| Deferred rent, net of current portion | 7,263 | 6,740 | ||||||||
| Deferred revenue, net of current portion | 23,958 | 30,208 | ||||||||
| Deferred tax liabilities | 693 | 693 | ||||||||
| Other non-current liabilities | 385 | 208 | ||||||||
| Total liabilities | 65,345 | 72,723 | ||||||||
| Stockholders' equity: | ||||||||||
| Common stock, $0.01 par value, 125,000 shares authorized; | ||||||||||
| 24,279 and 23,940 shares issued and outstanding at March 31, 2014 and | ||||||||||
| December 31, 2013, respectively | 243 | 239 | ||||||||
| Additional paid-in capital | 253,206 | 250,103 | ||||||||
| Accumulated deficit | (109,284 | ) | (98,675 | ) | ||||||
| Total stockholders' equity | 144,165 | 151,667 | ||||||||
| Total liabilities and stockholders' equity | $ | 209,510 | $ | 224,390 | ||||||
Source: bluebird bio, Inc.
bluebird bio, Inc
Investor Relations:
Richard E. T.
Smith, Ph.D., 339-499-9382
or
Pure Communications, Inc.
Media
Contact:
Dan Budwick, 973-271-6085