– Presented interim clinical data from Starbeam study of Lenti-DTM
in cerebral adrenoleukodystrophy (CALD) at
– Ten abstracts accepted for presentation at
– Advanced Company’s first CAR T oncology program into the clinic –
– Fully enrolled expanded Northstar study –
– Ended quarter with
“In early 2016 we achieved two crucial clinical milestones: treating the
first patient in the Phase 1 study of our anti-BMCA CAR T therapy
bb2121, and presenting the first clinical data from our Starbeam study
of Lenti-D in boys with CALD. We are very pleased with this significant
progress as we continue to build our T cell immunotherapy and HSC gene
therapy platforms,” said
PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN – In April,
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 ACCEPTED FOR PRESENTATION AT ASGCT 19th ANNUAL MEETING – Two oral presentations given by bluebird’s academic collaborators will highlight 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 will be 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.
TREATED FIRST PATIENT IN PHASE 1 STUDY OF BB2121 IN MULTIPLE MYELOMA
– In February, the first patient was infused in the CRB-401 study of
anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple
Celgeneexercised its option to exclusively license bb2121. Under the terms of the collaboration agreement between the two companies, bluebird bio received a $10.0 millionoption exercise payment from Celgeneand may now elect to co-develop and co-promote the product candidate in the United Stateswith Celgene. We are also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.
FULLY ENROLLED EXPANDED NORTHSTAR STUDY – Achievement of 18
patient enrollment target in
Northstar Studyof LentiGlobin in patients with transfusion-dependent thalassemia, including three additional adolescent patients.
Upcoming Anticipated Milestones
- Update on LentiGlobin process improvements in the second half of 2016
- Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016
Presentation of updated clinical data for LentiGlobin at the ASH
annual meeting in
First Quarter 2016 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
March 31, 2016were $826.9 million, compared to $865.8 millionas of December 31, 2015, a decrease of $38.9 million.
Revenues: Collaboration revenue was
$1.5 millionfor the first quarter of 2016 compared to $6.3 millionfor first quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgenein the second quarter of 2015.
R&D Expenses: Research and development expenses were
$41.9 millionfor the first quarter of 2016 compared to $23.7 millionfor the first quarter of 2015. 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
$16.0 millionfor the first quarter of 2016 compared to $7.3 millionfor the first 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
$56.3 millionfor the first quarter of 2016 compared to $24.8 millionfor the first quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of
$826.9 millionas of March 31, 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
bluebird bio has operations in
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 March 31,|
|Research and development||41,911||23,719|
|General and administrative||15,955||7,336|
|Change in fair value of contingent consideration||1,013||215|
|Total operating expenses||58,879||31,270|
|Loss from operations||(57,380)||(24,926)|
|Other income, net||961||139|
|Loss before income taxes||(56,419)||(24,787)|
|Income tax benefit||145||-|
|Net loss per share - basic and diluted:||$||(1.52)||$||(0.76)|
Weighted-average number of common shares used in computing net loss per share - basic and diluted:
bluebird bio, Inc.
Condensed Consolidated Balance Sheets Data
|March 31,||December 31,|
|Cash, cash equivalents and marketable securities||$||826,865||$||865,763|
|Total stockholders' equity||806,507||850,496|
Source: bluebird bio, Inc.