– Completed enrollment in
– Presented updated clinical results from studies in
relapsed/refractory multiple myeloma, TDT and severe sickle cell disease
– Announced topline interim clinical data from Starbeam Study of Lenti-DTM drug product in cerebral adrenoleukodystrophy (CALD) –
– Appointed John O. Agwunobi, M.D. and
– Completed public offering of common stock, raising net proceeds of
“In the first half of 2017, we’ve made tremendous progress against our
stated goals and continue to build momentum to the end of the year. New
data from our bb2121 anti-BCMA CAR T program reinforced our confidence
in this program, and we and
COMPLETED ENROLLMENT IN NORTHSTAR-2 – In June, bluebird bio
completed the enrollment of the adult and adolescent patient cohort in
Northstar-2 study of LentiGlobin drug product in patients with TDT and non-β0/β0 genotypes.
UPDATED DATA FROM BB2121 ANTI-BCMA CAR T PROGRAM PRESENTED – At
ASCOin June, bluebird bio presented updated results from the ongoing CRB-401 Phase 1 clinical study of bb2121, an investigational anti-BCMA CAR T cell therapy, in 18 patients with relapsed/refractory multiple myeloma. 100% of the 15 evaluable patients in active dose cohorts (doses above 50 x 106) achieved an objective response; overall response rate (ORR) across all cohorts (n=18) was 89%. 73% of evaluable patients in active dose cohorts achieved a very good partial response (VGPR) or better; 27% complete response (CR) rate across active dose cohorts. All patients tested for minimal residual disease (MRD) status (n=4) were found to be MRD-negative. No disease progression had been observed in active dose cohorts as of the May 4, 2017data cut-off; range of follow-up was 8 to 54 weeks. No dose-limiting toxicities had been observed. The objective of this Phase 1 dose-escalation study is to evaluate the safety and efficacy of bb2121 and determine a recommended Phase 2 dose. bluebird bio and Celgeneare jointly developing bb2121.
EARLY DATA FROM NORTHSTAR-2 PRESENTED – At EHA in June,
bluebird bio presented early data from its Phase 3 Northstar-2
(HGB-207) study of LentiGlobin drug product in patients with
transfusion-dependent β-thalassemia (TDT) and non-β0/β0
genotypes. Drug product vector copy number (DP VCN) and
percentage of lentiviral vector positive cells (LVV+) for the initial
7 drug product lots manufactured in
Northstar-2 were consistently higher than in Northstar(HGB-204), with a median DP VCN of 3.0. Initial results show that the three patients treated to date had achieved in vivo VCN and HbAT87Q production as good as or better than patients achieving transfusion independence in Northstar. The first patient treated in Northstar-2 with 6 months of follow-up achieved normal levels of total hemoglobin (13.3 g/dL) after discontinuing transfusions, producing 9.5 g/dl of HbAT87Q at last follow-up. The safety profile was consistent with autologous transplantation.
- NEW DATA FROM HGB-205 PRESENTED – At EHA in June, bluebird bio presented new data from the HGB-205 study of LentiGlobin drug product in patients with TDT and severe sickle cell disease (SCD). Ongoing transfusion independence up to 3.5 years was observed in patients with TDT; three patients have discontinued iron chelation. The first patient with SCD treated with gene therapy (Patient 1204) continues to show clinically meaningful improvement in symptoms of SCD and stable vector copy number and HbAT87Q in peripheral blood. Two recently treated patients with severe SCD show increasing levels of HbAT87Q and stable in vivo VCN. As with Patient 1204, the first patient with SCD treated in HGB-205, these two patients received a more stringent busulfan conditioning regimen and regular blood transfusions prior to stem cell harvest.
TOPLINE INTERIM LENTI-D DATA ANNOUNCED – In June, bluebird bio
announced topline interim clinical data on the initial 17 patients
treated in the Starbeam study of Lenti-D drug product in CALD. As of
June 13, 15/17 patients (88%) in initial study cohort remain free of major functional disabilities (MFDs) at 24 months, the primary endpoint of the trial. This exceeds bluebird’s pre-defined interim efficacy benchmark for the study of MFD-free survival of 76%, derived from the literature and based on clinical data from an earlier observational study describing that natural history of CALD and outcomes from allogeneic hematopoietic stem cell transplant. The safety profile of Lenti-D was consistent with myeloablative conditioning. No patients treated with Lenti-D had graft versus host disease, and there was no graft rejection or clonal dominance. An expansion cohort is enrolling additional patients to gain European manufacturing experience.
NEW BOARD APPOINTMENTS – In June, bluebird bio appointed
John O. Agwunobi, M.D. and Douglas A. Melton, Ph.D. to its Board of Directors.
DUKE COLLABORATION – In May, bluebird bio announced that it has
entered into a collaboration with Duke University’s Robert J.
Center for Health Policyto develop a broadly-supported path for value-based payment reform models for gene therapies and other innovative treatments.
STRENGTHENED BALANCE SHEET – In June, bluebird raised
$436.8 millionin net proceeds in an equity financing. The company’s cash, cash equivalents and marketable securities are sufficient to fund operations into 2020 based on the company’s current business plan. Proceeds from the equity financing will fund the potential exercise of the option to co-develop and co-promote bb2121; planned clinical studies in oncology and severe genetic diseases; and to further expand the company’s manufacturing platform and capabilities to support ongoing and anticipated product development efforts and in anticipation of a potential commercial launch; and general and administrative expenses.
Second Quarter 2017 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
June 30, 2017were $1.2 billion, compared to $884.8 millionas of December 31, 2016, an increase of $312.2 million.
Revenues: Total revenue was
$16.7 millionfor the second quarter of 2017 compared to $1.6 millionfor second quarter of 2016. The increase is primarily attributable to revenue recognized under bluebird bio’s out-licensing agreements with Novartis Pharma AGand GlaxoSmithKline Intellectual Property Development Limited (GSK) and the commencement of revenue recognition for the bb2121 license and manufacturing services under the company’s agreement with Celgene.
R&D Expenses: Research and development expenses were
$64.3 millionfor the second quarter of 2017 compared to $41.8 millionfor the second quarter of 2016. The increase in research and development expenses was primarily attributable to increased manufacturing expenses, clinical trial expenses, and employee-related costs due to increased headcount to support overall growth.
G&A Expenses: General and administrative expenses were
$21.2 millionfor the second quarter of 2017 compared to $18.4 millionfor the second quarter of 2016. The increase in general and administrative expenses was primarily attributable to increased employee-related costs due to increased headcount, and increased facility-related expenses to support overall growth.
Net Loss: Net loss was
$70.9 millionfor the second quarter of 2017 compared to $58.8 millionfor the second quarter of 2016.
Financial Guidance: bluebird bio expects that its cash, cash
equivalents and marketable securities of
$1.2 billionas of June 30, 2017will be sufficient to fund its current operations into 2020.
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 studies, 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 studies 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
Six Months Ended
|Research and development||64,311||41,760||119,339||83,671|
|General and administrative||21,197||18,363||41,481||34,318|
Change in fair value of contingent consideration
|Total operating expenses||84,538||61,527||161,283||120,406|
|Loss from operations||(67,822||)||(59,975||)||(137,735||)||(117,355||)|
|Interest (expense) income, net||(2,242||)||981||(687||)||1,932|
|Other (expense) income, net||(834||)||(76||)||(1,189||)||(66||)|
|Loss before income taxes||(70,898||)||(59,070||)||(139,611||)||(115,489||)|
|Income tax benefit||—||226||—||371|
|Net loss per share - basic and diluted:||$||(1.73||)||$||(1.59||)||$||(3.41||)||$||(3.12||)|
Weighted-average number of common shares used in computing net loss per share - basic and diluted:
bluebird bio, Inc.
Condensed Consolidated Balance Sheets Data
|Cash, cash equivalents and marketable securities||$||1,197,059||$||884,830|
|Total stockholders' equity||1,200,019||869,440|
Source: bluebird bio, Inc.