bluebird bio, Inc. (NASDAQ: BLUE) today reported financial
results and business highlights for the first quarter ended March
31, 2021 and shared recent operational progress.
“Undoubtedly the highlight of last quarter at bluebird was the
approval of Abecma, the first and only CAR T therapy approved for
the treatment of relapsed or refractory multiple myeloma,” said
Nick Leschly, chief bluebird. “We and our colleagues at BMS are now
full speed ahead and on track to begin treating patients this
quarter. It has been an amazing journey and in many ways, we’re
just getting started. While the oncology team has been delivering
on Abecma, the severe genetic disease team met the moment. We
quickly completed an investigation of the SUSAR of AML in our
HGB-206 study of LentiGlobin gene therapy for SCD and determined
that it was highly unlikely to be due to BB305 lentiviral vector.
With these data and the other event changed from an MDS diagnosis
to transfusion-dependent anemia, we are now quickly moving to
engage with regulators with a goal of lifting the clinical holds in
mid-2021. Amidst these challenges and work towards the planned
separation, I want to commend and thank all birds for truly
demonstrating anti-fragility and continuing to keep patients at the
center of everything we do.”
BUSINESS SEPARATION
UPDATE
Today, bluebird bio is providing additional detail regarding the
company’s planned business separation, which is targeted for
completion by year-end 2021.
bluebird bio
bluebird bio is announcing that Tom Klima will join the company
as chief commercial officer. Tom brings a track record of success
across multiple commercial roles in oncology and rare diseases,
most recently at Gamida Cell Ltd., where he served as chief
commercial officer.
Additional members of the bluebird bio leadership team focused
on severe genetic diseases will include:
- Andrew Obenshain, chief executive officer
(previously-disclosed)
- Jason Cole, chief business officer
- Rich Colvin, interim chief medical officer
- Anne-Virginie Eggiman, senior vice president, regulatory
science
“As we move towards separation, I’m pleased to have key
leadership team members in place that are poised to bring bluebird
bio to its next phase of success,” said Andrew Obenshain. “With the
addition of Tom Klima to lead our commercial efforts, we are
rounding out our team of experts with a deep level of expertise
from early stage clinical development to commercial delivery. I’m
excited about our path forward and look forward to continuing to
expand the bluebird team to bring gene therapy to patients with
severe genetic diseases.”
Oncology NewCo – 2seventy bio
Today, the company is announcing that Oncology NewCo will be
named 2seventy bio and members of the leadership team will
include:
- Nick Leschly, chief executive officer
(previously-disclosed)
- Chip Baird, chief financial officer
- Philip Gregory, chief scientific officer
- Nicola Heffron, chief operating officer
“Two hundred seventy miles per hour is the maximum speed of
human thought,” said Nick Leschly, chief executive officer. “The
name 2seventy was selected to signify this speed and our team’s
translation of thought to action as we advance our next generation
pipeline of transformative cell therapies to help cancer patients
urgently in need.”
RECENT HIGHLIGHTS
MULTIPLE MYELOMA
- ABECMA FDA APPROVAL – On March 26, 2021, bluebird bio
and Bristol-Myers Squibb announced that the U.S. Food and Drug
Administration (FDA) approved Abecma (idecabtagene vicleucel;
ide-cel) as the first B-cell maturation antigen (BCMA)-directed
chimeric antigen receptor (CAR) T cell immunotherapy for the
treatment of adult patients with relapsed or refractory multiple
myeloma (RRMM) after four or more prior lines of therapy, including
an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38
monoclonal antibody. Abecma is a personalized immune cell therapy
approved as a one-time infusion with a recommended dose range of
300 to 460 x 106 CAR-positive T cells.
- KARMMA NEJM PUBLICATION– On February 24, 2021, bluebird
bio and Bristol-Myers Squibb announced that results from the
pivotal Phase 2 KarMMa study were published in The New England
Journal of Medicine. The KarMMa study met its primary endpoint of
overall response rate and key secondary endpoint of complete
response rate. The data from the study demonstrates deep and
durable responses with ide-cel treatment in triple-class exposed
RRMM patients (n=128).
SICKLE CELL DISEASE
- CLINICAL STUDIES UPDATE – On April 20, 2021, bluebird
bio announced a revised diagnosis for the previously reported case
of myelodysplastic syndrome (MDS) in its Phase 1/2 study of
LentiGlobin for sickle cell disease (SCD) (bb1111). Upon further
assessment, the treating investigator concluded this is not a case
of MDS and revised the diagnosis to transfusion-dependent anemia.
In addition, on March 10, 2021, bluebird bio reported that it is
very unlikely the suspected unexpected serious adverse reaction
(SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206
study of LentiGlobin for SCD was related to the BB305 lentiviral
vector (LVV). bluebird bio continues to work with regulators to
resume its clinical studies in sickle cell disease as well as to
remove the clinical hold for HGB-207 and HGB-212 clinical studies
of beti-cel for β-thalassemia, with potential lift of all clinical
holds in mid-2021.
CEREBRAL ADRENOLEUKODYSTROPHY
- ELI-CEL DATA AT EBMT– On March 15, 2021, bluebird bio
presented new data suggesting durability of response and a strong
safety profile post elivaldogene autotemcel (eli-cel, Lenti-D™)
gene therapy in patients with cerebral adrenoleukodystrophy (CALD)
at the 47th Annual Meeting of the European Society for Blood and
Marrow Transplantation (EBMT 2021). Long-term results from the
Phase 2/3 Starbeam study of eli-cel, showed that ninety percent of
patients (27/30) are alive and free of major functional
disabilities (MFDs) at 24 months or more of follow-up. In the 51
patients treated with eli-cel in clinical studies (ALD-102/LTF-304
and ALD-104) there were no reports of graft failure, graft
rejection, GVHD, replication competent lentivirus or insertional
oncogenesis.
PIPELINE
- BILL & MELINDA GATES FOUNDATION GRANT TO EXPLORE IN-VIVO
LVV APPLICATIONS - bluebird bio is announcing today that it has
received a grant from the Bill & Melinda Gates Foundation to
explore new, potentially transformative in vivo treatments for SCD
using the Company’s proprietary lentiviral vector (LVV) platform.
The funding will support the research and development of novel LVVs
that target hematopoietic stem cells (HSCs) for in vivo
administration to bring gene-based therapies for SCD and other
potential indications to patients around the world who may have
limited access to ex vivo and other emerging therapies. This
research may also enable the application of in-vivo LVV approaches
in other severe genetic diseases.
- PSIOXUS PRE-CLINICAL DATA - On April 14, 2021, bluebird
bio and PsiOxus Therapeutics presented preclinical data at the
American Association for Cancer Research (AACR) Annual Meeting
2021. The results showed synergistic activity between PsiOxus’
T-SIGn vector and bluebird bio’s CAR-T therapy in primary and
metastatic solid tumors. A single IV cycle of PsiOxus’ T-SIGn
vector enabled an otherwise non-effective dose of CAR-T cell
therapy to clear primary and metastatic tumors in vivo.
COMPANY
- BUSINESS OPERATIONS – On April 20, 2021 bluebird bio
announced its decision to withdraw ZYNTEGLO™ (betibeglogene
autotemcel, beti-cel) for transfusion-dependent β-thalassemia (TDT)
from the German market because reimbursement negotiations in
Germany did not result in a price for ZYNTEGLO that reflects the
value of this one-time gene therapy with potential life-long
benefit for people living with TDT. Due in part to this decision,
the company also announced a targeted reshaping of its workforce
intended to enable the company to advance its late-stage gene
therapy programs. This reduction and reallocation of resources will
allow the company to focus on priority European markets and
streamline global operations going forward to ensure its ability to
deliver gene therapies to patients.
UPCOMING ANTICIPATED
MILESTONES
Regulatory Outlook
- SCD: The company is investigating
the recently-reported safety events and plans to continue to work
closely with the FDA in their review of these events to provide an
update on the Company’s development plan and timeline for
submission for regulatory approval by year end.
- TDT: The company is on track to
complete its rolling BLA submission to the U.S. FDA for beti-cel in
mid-2021, contingent upon successful resolution of any U.S. FDA
concerns applicable to the program arising out of the
recently-reported safety events in the SCD program. This submission
is anticipated to include adult, adolescent and pediatric patients
with transfusion dependent β-thalassemia across all genotypes
(including non-β0/β0 genotypes and β0/β0 genotypes).
- CALD: The company is on track to
complete its BLA submission to the U.S. FDA for eli-cel in
mid-2021. The company plans to receive European approval for
eli-cel in patients with CALD in mid-2021.
Clinical Updates and Milestones
- Updated data from ongoing clinical study in patients with SCD
by the end of 2021.
- Updated data from ongoing clinical studies in patients with TDT
in mid-2021.
- Updated clinical data from the ongoing pivotal Phase 2 KarMMa
study of Abecma (ide-cel, bb2121) in patients with relapsed and
refractory multiple myeloma to be presented at the American Society
of Clinical Oncology 2021 (ASCO21) Virtual Scientific Program on
June 4.
- bb21217 clinical data from the ongoing CRB-402 study in
patients with multiple myeloma by the end of 2021.
- Submission of 1 - 2 investigational new drug (IND) applications
by the end of 2021.
Commercial and Foundation Building
- Abecma first commercial patients treated in the first half of
2021.
Company
- bluebird bio anticipates the separation of its severe genetic
disease and oncology businesses into two independent, publicly
traded companies (bluebird bio and 2seventy bio) to be completed by
the end of 2021.
FIRST QUARTER 2021 FINANCIAL
RESULTS
- Cash Position: Cash, cash equivalents and marketable
securities as of March 31, 2021 and December 31, 2020 were $1.09
billion and $1.27 billion, respectively. The decrease in cash, cash
equivalents and marketable securities is primarily related to cash
used in support of ordinary course operating activities.
- Revenues: Total revenues were $12.8 million for the
three months ended March 31, 2021 compared to $21.9 million for the
three months ended March 31, 2020. The decrease was primarily
driven by a decrease in ide-cel license and manufacturing services
revenue and a decrease in revenue recognized in connection with
treating patients in the Phase 1 CRB-402 study of bb21217 under our
agreements with BMS.
- R&D Expenses: Research and development expenses were
$154.5 million for the three months ended March 31, 2021 compared
to $154.1 million for the three months ended March 31, 2020. The
increase was primarily driven by increased costs incurred through
the amended BMS collaboration as well as an increase in employee
compensation, benefit, and other headcount related expenses. These
increased costs were partially offset by a decrease in
manufacturing costs.
- SG&A Expenses: Selling, general and administrative
expenses were $86.9 million for the three months ended March 31,
2021 compared to $73.2 million for the three months ended March 31,
2020. The increase was primarily driven by increased employee
compensation, benefit, and other headcount related expenses, as
well as an increase in consulting fees associated with the ongoing
project to separate the Company’s severe genetic disease and
oncology programs into two independently traded companies. These
increased costs were partially offset by a decrease in costs
related to commercial readiness activities due to delays in
commercialization as a result of the COVID-19 pandemic and in light
of safety events in the HGB-206 study of LentiGlobin gene therapy
for SCD.
- Net Loss: Net loss was $205.8 million for the three
months ended March 31, 2021 compared to $202.6 million for the
three months ended March 31, 2020.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
Cambridge, Mass., headquarters, we’re developing gene and cell
therapies for severe genetic diseases and cancer, with the goal
that people facing potentially fatal conditions with limited
treatment options can live their lives fully. Beyond our labs,
we’re working to positively disrupt the healthcare system to create
access, transparency and education so that gene therapy can become
available to all those who can benefit.
bluebird bio is a human company powered by human stories. We’re
putting our care and expertise to work across a spectrum of
disorders: cerebral adrenoleukodystrophy, sickle cell disease,
β-thalassemia and multiple myeloma, using gene and cell therapy
technologies including gene addition, and (megaTAL-enabled) gene
editing.
bluebird bio has additional nests in Seattle, Wash.; Durham,
N.C.; and Zug, Switzerland. For more information, visit
bluebirdbio.com.
Follow bluebird bio on social media: @bluebirdbio, LinkedIn,
Instagram and YouTube.
ZYNTEGLO, LentiGlobin, bluebird bio, 2seventy and 2seventy bio
are trademarks of bluebird bio, Inc.
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,
results of operations, as well as statements regarding the
Company’s timing and expectations regarding its investigation of
the relationship of the safety events in HGB-206 to the use of
lentiviral vector BB305 in LentiGlobin gene therapy for SCD, and
any regulatory interactions for lifting the clinical hold; its
expectations for commercialization efforts for ZYNTEGLO in Europe;
and the company’s expectations for the commercialization of ABECMA
through the BMS collaboration; as well as the company’s
expectations regarding the effects and impact of the resizing and
reshaping of the workforce; the timing, leadership, structure,
including the division of assets among bluebird bio and 2seventy
bio, and the impact of a separation; as well as the company’s
intention to provide further updates on the separation and the
related financing strategies for bluebird bio and 2seventy. 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 that the Company may not be able
to definitively determine whether the lentiviral vector BB305 used
in LentiGlobin gene therapy for SCD and in beti-cel is related to
the safety events in a timely manner, or at all; the risk that
insertional oncogenic or other safety events associated with
lentiviral vector, drug product, or myeloablation will be
discovered or reported over time; the risk that we may not be able
to address regulatory authorities’ concerns quickly or at all and
may not be able to resume our HGB-206 or HGB-210 studies in a
timely manner, or at all; the risk that we may not resume patient
treatment with ZYNTEGLO in the commercial context in a timely
manner or at all; the risk that our lentiviral vector platform
across our severe genetic disease programs may be implicated,
affecting the development and potential approval elivaldogene
autotemcel; the risks that we may not complete the separation on
the terms or timeline currently contemplated if at all, achieve the
expected benefits of a separation, and a separation could harm our
business, results of operations and financial condition; the risk
that the transaction might not be tax-free; we may be unable to
make, on a timely or cost-effective basis, the changes necessary to
operate as independent companies; 2seventy Bio's lack of
independent operating history and the risk that its accounting and
other management systems may not be prepared to meet the financial
reporting and other requirements of operating as an independent
public company; dedicated financial and/or strategic funding
sources may not be available on favorable terms; a separation or
announcement thereof may adversely impact our ability to attract or
retain key personnel; a separation may adversely impact the
effectiveness of development and commercialization efforts by us
and our partners; our businesses may be disrupted as a result of
the announcement or pendency of the separation; the risk that we
are unable to realize the intended benefits of resizing and
reshaping our workforce; the COVID-19 pandemic and resulting
economic conditions will have a greater impact on the company’s
operations and plans than anticipated; that our collaboration with
BMS will not continue or be successful; that the commercialization
of ABECMA will not be successful; that preliminary positive
efficacy and safety results from our prior and ongoing clinical
trials will not continue or be repeated in our ongoing or future
clinical trials; the risk that the current or planned clinical
trials of our product candidates will be insufficient to support
regulatory submissions or marketing approval in the United States
and European Union; the risk that regulatory authorities will
require additional information regarding our product candidates,
resulting in delay to our anticipated timelines for regulatory
submissions, including our applications for marketing approval; the
risk that we will encounter further challenges in the commercial
launch of ZYNTEGLO in the European Union, including in managing our
complex supply chain for the delivery of drug product, or in
obtaining sufficient coverage or reimbursement for our products;
and the risk that any one or more of our product candidates, will
not be successfully developed, approved or commercialized. For a
discussion of other risks and uncertainties, and other important
factors, any of which could cause our actual results to differ from
those contained in the forward-looking statements, see the section
entitled “Risk Factors” in our most recent Form 10-K, as well as
discussions of potential risks, uncertainties, and other important
factors in our subsequent filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and bluebird bio undertakes no duty to update this
information unless required by law.
bluebird bio, Inc.
Condensed Consolidated
Statements of Operations
(in thousands, except per
share data)
(unaudited)
For the three months ended
March 31,
2021
2020
Revenue:
Service revenue
$
5,918
$
16,833
Collaborative arrangement revenue
1,519
2,302
Royalty and other revenue
5,357
2,728
Total revenues
12,794
21,863
Operating expenses:
Research and development
154,478
154,123
Selling, general and administrative
86,874
73,248
Cost of royalty and other revenue
2,281
1,025
Change in fair value of contingent
consideration
369
(3,108)
Total operating expenses
244,002
225,288
Loss from operations
(231,208)
(203,425)
Interest income, net
710
5,355
Other income (expense), net
24,756
(4,447)
Loss before income taxes
(205,742)
(202,517)
Income tax expense
(66)
(94)
Net loss
$
(205,808)
$
(202,611)
Net loss per share - basic and
diluted:
$
(3.07)
$
(3.64)
Weighted-average number of common shares
used in computing net loss per share - basic and diluted:
66,976
55,590
bluebird bio, Inc.
Condensed Consolidated Balance
Sheet Data
(in thousands, except per
share data)
(unaudited)
As of March 31, 2021
As of December 31,
2020
Cash, cash equivalents and marketable
securities
$
1,093,551
$
1,274,142
Total assets
$
1,637,279
$
1,781,252
Total liabilities
$
436,946
$
426,196
Total stockholders’ equity
$
1,200,333
$
1,355,056
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210505006054/en/
Investors & Media Investors: Elizabeth Pingpank,
617-914-8736 epingpank@bluebirdbio.com Media: Jenn Snyder,
617-448-0281 jsnyder@bluebirdbio.com Catherine Falcetti,
617-583-3411 cfalcetti@bluebirdbio.com
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