– Case study on first patient with severe sickle cell disease (SCD)
treated with gene therapy published in
– Appointed Derek Adams, Ph.D. Chief Technology and Manufacturing
– Refined regulatory path for LentiGlobinTM
in transfusion-dependent β-thalassemia (TDT) in
– Announced licensing of lentiviral vector patent rights for
development and commercialization of cell therapies to GlaxoSmithKline
– Ended quarter with
“In the first quarter of 2017, we have been laser-focused on continuing
to develop our commercial and manufacturing infrastructure and ensuring
that we are prepared for future MAA and BLA filings,” said
CASE STUDY PUBLISHED IN NEJM - In March, bluebird announced the
publication in the
New England Journal of Medicineof a case study on Patient 1204, the first patient with SCD to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and in that time, no SCD-related clinical events or hospitalizations have occurred, contrasting favorably with the period before the patient began regular transfusions. Adverse events (AEs) were consistent with busulfan conditioning, and no AEs related to LentiGlobin drug product had been observed.
APPOINTMENT OF NEW SENIOR EXECUTIVES – In March,
bluebird announced that
Derek Adams, Ph.D. joined the company as its Chief Technology and Manufacturing Officer and Joanne Smith-Farrell, Ph.D., joined as Senior Vice President, Corporate Development and Strategy.
REFINED REGULATORY PATH IN EU – In April, bluebird met with the
European Medicines Agency(EMA) regarding the regulatory path for LentiGlobin in TDT as part of its participation in the Adaptive Pathways Program. Based on this meeting, bluebird believes that it is possible to seek conditional approval for our LentiGlobin product candidate, with our improved manufacturing process, for the treatment of patients with TDT and a non-β0/β0 genotype on the basis of the totality of the clinical data from our ongoing Northstar Studyin patients with TDT and supportive ongoing HGB-205 study, our single-center study in patients with TDT and SCD, together with the data available from our ongoing Northstar-2 Study in patients with TDT and non-β0/β0 genotypes at the time of filing. This plan is contingent upon these studies demonstrating acceptable safety and efficacy, and in particular transfusion independence as the primary endpoint and a reduction in transfusion requirements as a secondary endpoint for efficacy analyses.
LICENSED LENTIVIRAL VECTOR PATENT RIGHTS – In May, bluebird
announced that it has entered into worldwide license agreements around
its proprietary lentiviral vector platform with
Novartis Pharma AGand GlaxoSmithKline Intellectual Development Property Limited (GSK). Financial terms of the agreements include an upfront payment to bluebird as well as potential future milestone and royalty payments.
Upcoming Anticipated Milestones:
Presentation of updated bb2121 clinical data from the CRB-401 study in
relapsed/refractory multiple myeloma at the
American Society of Clinical Oncology( ASCO) Annual Meeting
Presentation of early LentiGlobin clinical data from the Northstar-2
study in patients with TDT and non-β0/β0
genotypes, as well as data from additional patients in the HGB-205
single-center study in patients with TDT and SCD at the
European Hematology Association(EHA) Annual Meeting
- Initiation of a Phase 1 clinical study of bb21217 anti-BCMA CAR T product candidate in patients with relapsed/refractory multiple myeloma
- Initiation of HGB-212, a Phase 3 clinical study of LentiGlobin in patients with TDT and the β0/β0 genotype in the second half of 2017
- Presentation of full data from the initial 17 patients treated in the Starbeam clinical study of Lenti-D in CALD by year end 2017
Presentation of early LentiGlobin clinical data from the HGB-206 study
in patients with SCD conducted under the amended study protocol at the
American Society of Hematology(ASH) Annual Meeting
First Quarter 2017 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable
securities as of March 31, 2017 were
$799.9 million, compared to $884.8 million as of December 31, 2016, a decrease of $84.9 million.
Revenues: Collaboration revenue was $6.8 million for the first
quarter of 2017 compared to $1.5 million for the first quarter of
2016. The increase is the result of the commencement of revenue
recognition for the bb2121 license and manufacturing services under
our agreement with
- R&D Expenses: Research and development expenses were $55.0 million for the first quarter of 2017 compared to $41.9 million for the first quarter of 2016. The increase in research and development expenses was primarily attributable to increased employee compensation and facilities costs due to increased headcount, and increased manufacturing, clinical, research, and information technology costs to support the advancement of our clinical and pre-clinical programs.
- G&A Expenses: General and administrative expenses were $20.3 million for the first quarter of 2017 compared to $16.0 million for the first quarter of 2016. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount and costs to support our pre-commercial efforts.
- Net Loss: Net loss was $68.7 million for the first quarter of 2017 compared to $56.3 million for the first quarter of 2016.
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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
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, 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 risks that the changes we have made in the LentiGlobin drug product manufacturing process or the HGB-206 clinical study protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the
|bluebird bio, Inc.|
|Condensed Consolidated Statements of Operations Data|
|(in thousands, except per share data)|
Three months ended
|Research and development||55,028||41,911|
|General and administrative||20,284||15,955|
|Change in fair value of contingent consideration||1,433||1,013|
|Total operating expenses||76,745||58,879|
|Loss from operations||(69,913||)||(57,380||)|
|Other income, net||1,201||961|
|Loss before income taxes||(68,712||)||(56,419||)|
|Income tax benefit||—||145|
|Net loss per share - basic and diluted:||$||(1.68||)||$||(1.52||)|
Weighted-average number of common shares used
in computing net loss per share - basic and diluted:
|bluebird bio, Inc.|
|Condensed Consolidated Balance Sheets Data|
|March 31,||December 31,|
|Cash, cash equivalents and marketable securities||$||799,869||$||884,830|
|Total stockholders' equity||815,379||869,440|
Source: bluebird bio, Inc.