- Lenti-D™ granted Priority Medicines (PRIME) designation from
- LentiGlobin™ in transfusion-dependent β-thalassemia (TDT) granted accelerated assessment of Marketing Authorization Application (MAA) from EMA -
- Ended quarter with
- Completed public offering of common stock in
“The clinical data presented this spring across our development programs
in TDT, SCD and multiple myeloma have further reinforced the strength of
our gene and cell therapy platforms, and we are putting tremendous
effort towards bringing all four of our clinical programs to patients as
soon as possible,” said
- LENTIGLOBIN ACCELERATED ASSESSMENT – In
July 2018, the Committee for Medicinal Products for Human Use(CHMP) of the European Medicines Agency(EMA) granted an accelerated assessment to LentiGlobin in transfusion-dependent β-thalassemia (TDT). The company is on track to submit a Marketing Authorization Application (MAA) to the EMA for LentiGlobin in 2018.
- NEW DATA FROM NORTHSTAR AND NORTHSTAR-2 PRESENTED – At the
Annual Congress of the
European Hematology Association(EHA) in June 2018, bluebird bio presented new data from its studies of LentiGlobin in patients with TDT: Northstar (HGB-204) and Northstar-2 (HGB-207). As of the data cutoff, 7/8 non-β0/β0 patients with ≥ 6 months follow-up were producing normal or near-normal amounts of total hemoglobin (11.1 – 13.3 g/dL) and were transfusion free in Northstar-2. 8/10 of non-β0/β0 patients achieved and maintained transfusion independence for up to 3 years in Northstar. Across both studies, the safety profile was consistent with myeloablative conditioning.
- FIRST PEDIATRIC PATIENT TREATED – In
April 2018, the first pediatric patient was treated in Northstar-3 (HGB-212), bluebird bio’s Phase 3 study of LentiGlobin in patients with β0/β0 genotypes.
- NEW DATA FROM HGB-206 PRESENTED – At EHA in
June 2018, bluebird bio presented new data from the HGB-206 study of LentiGlobin in patients with severe sickle cell disease (SCD). As of the data cutoff, all patients (n=4) in Group C with ≥ 3 months follow-up were consistently producing ≥ 30% anti-sickling HbAT87Q. The first Group C patient was generating a normal total hemoglobin of 14.2 g/dL with over 60% anti-sickling HbAT87Q at 6 months. Across all patients in the study, the safety profile was consistent with myeloablative conditioning.
- NEW DATA FROM CRB-401 PRESENTED – At the
American Society of Clinical Oncology( ASCO) Annual Meeting in June 2018, bluebird bio and Celgene Corporationpresented new data from the ongoing CRB-401 Phase 1 clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 43 patients with late-stage relapsed/refractory multiple myeloma. Deep and durable responses were observed at active doses (≥150 × 106 CAR+ T cells). A median progression-free survival (PFS) of approximately one year was achieved in heavily pre-treated patients in the active doses of the dose escalation cohort. Consistent response rates were observed for both low and high BCMA expression levels. Adverse events have been manageable across doses.
- PRIME DESIGNATION FOR LENTI-D – In
July 2018, the EMA granted access to its Priority Medicines (PRIME) scheme for Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD). The PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly. To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical data or nonclinical data.
- BREAKTHROUGH DESIGNATION FOR LENTI-D – In
May 2018, the U.S. Food and Drug Administration( FDA) granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with CALD. Lenti-D previously was granted Orphan Drug designation by the FDAand EMA, as well as Rare Pediatric Disease designation by the FDA.
- STRENGTHENED BALANCE SHEET – In
July 2018, bluebird bio raised approximately $600.6 millionin net proceeds through a public equity offering. bluebird bio anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations into 2022 based on the company’s current business plan.
Second Half 2018 Anticipated Milestones
- Submission of a MAA to the EMA for LentiGlobin in patients with TDT and non-β0/β0 genotypes
Submission of LentiGlobin clinical data from the Northstar-2 (HGB-207)
clinical study in patients with TDT and non-β0/β0 genotypes
American Society of Hematology(ASH) Annual Meeting
- Submission of LentiGlobin clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype to the ASH Annual Meeting
- Update on the clinical development plan and registration strategy for LentiGlobin in SCD
- Submission of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD to the ASH Annual Meeting
- Submission of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma to the ASH Annual Meeting
Celgeneof a Phase 3 clinical study of bb2121 in third line multiple myeloma
- Presentation of Lenti-D clinical data from the ongoing Starbeam clinical study in patients with CALD in the second half of 2018
Second Quarter 2018 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities
June 30, 2018and December 31, 2017were $1.46 billionand $1.61 billion, respectively.
- Revenues: Total revenues were
$7.9 millionfor the three months ended June 30, 2018compared to $16.7 millionfor the three months ended June 30, 2017. The decrease of approximately $8.9 millionwas primarily attributable to license revenue recognized in the second quarter of 2017 as a result of out-licensing arrangements entered into during that quarter. Total revenues were $23.8 millionfor six months ended June 30, 2018compared to $23.5 millionfor six months ended June 30, 2017. The increase of $0.3 millionwas primarily attributable to an overall increase in collaboration revenue for the bb2121 license and manufacturing services under the company’s agreement with Celgene, offset by a decrease in license and royalty revenue.
- R&D Expenses: Research and development expenses were
$115.0 millionfor the three months ended June 30, 2018compared to $63.9 millionfor the three months ended June 30, 2017. Research and development expenses were $212.1 millionfor six months ended June 30, 2018compared to $118.9 millionfor six months ended June 30, 2017. The increase in both periods was driven by costs incurred to advance and expand the company’s pipeline and is attributable to increased clinical trial-related costs and manufacturing costs for development programs, increased laboratory expenses, increased employee-related costs due to headcount growth, and increased license milestones and fees under the company’s strategic collaboration and license agreements.
- G&A Expenses: General and administrative expenses were
$41.2 millionfor the three months ended June 30, 2018compared to $21.2 millionfor the three months ended June 30, 2017. General and administrative expenses were $76.1 millionfor six months ended June 30, 2018compared to $41.5 millionfor six months ended June 30, 2017. The increase in both periods was attributable to increases in employee-related costs due to increased headcount to support overall growth, commercial-readiness activities, and professional and consulting fees.
- Net Loss: Net loss was
$146.0 millionfor the three months ended June 30, 2018compared to $70.9 millionfor the three months ended June 30, 2017. Net loss was $261.1 millionfor six months ended June 30, 2018compared to $139.6 millionfor six months ended June 30, 2017.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise
and gene editing capabilities,
bluebird bio's gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease.
bluebird bio's oncology pipeline is built upon the company's 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. The company’s lead oncology
programs are anti-BCMA CAR T programs partnered with
bluebird bio’s discovery research programs include utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.
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, results of
operations and sufficiency of its cash, cash equivalents and marketable
securities to fund its planned operations, as well as statements
regarding the anticipated development and regulatory milestones and
plans for the Company’s product candidates and clinical studies and
statements regarding the Company’s plans to provide updates on the
development of its product candidates. 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 risks that the
preliminary positive efficacy and safety results from our prior and
ongoing clinical trials of our product candidates 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, risks that the current
or planned clinical trials of our LentiGlobin, Lenti-D, bb2121 or
bb21217 product candidates will be insufficient to support regulatory
submissions or marketing approval in the
bluebird bio, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)
For the three months ended June
For the six months ended June
|License and royalty revenue||414||10,570||763||10,570|
|Research and development||115,014||63,891||212,123||118,919|
|General and administrative||41,168||21,197||76,094||41,481|
|Cost of license and royalty revenue||21||420||36||420|
|Change in fair value of contingent consideration||262||(970||)||796||463|
|Total operating expenses||156,465||84,538||289,049||161,283|
|Loss from operations||(148,614||)||(67,822||)||(265,241||)||(137,735||)|
|Interest income (expense), net||2,436||(2,242||)||3,824||(687||)|
|Other income (expense), net||182||(834||)||297||(1,189||)|
|Loss before income taxes||(145,996||)||(70,898||)||(261,120||)||(139,611||)|
|Net loss per share - basic and diluted:||$||(2.91||)||$||(1.73||)||$||(5.22||)||$||(3.41||)|
Weighted-average number of common shares used in
bluebird bio, Inc.
Condensed Consolidated Balance Sheet Data
|Cash, cash equivalents and marketable securities||$||1,457,243||$||1,614,302|
|Total stockholders' equity||1,457,534||1,623,432|
Source: bluebird bio