– First sickle cell patient treated with drug
product manufactured with suspension-based lentiviral vector in
HGB-206 –
– Elivaldogene autotemcel (eli-cel,
Lenti-D gene therapy) in cerebral adrenoleukodystrophy (CALD)
granted accelerated assessment of Marketing Authorization
Application (MAA) from EMA –
– In partnership with Bristol-Myers Squibb,
completed submission of Biologics License Application (BLA) to FDA
for ide-cel –
– Ended quarter with $1.6 billion in cash, cash
equivalents and securities, extending cash runway into 2023 –
bluebird bio, Inc. (NASDAQ: BLUE) today reported financial
results and business highlights for the second quarter ended June
30, 2020 and shared recent operational progress.
“I am incredibly proud of the progress made at bluebird this
quarter, and the way in which our employees have continued to
execute on behalf of patients in the midst of an ongoing global
pandemic,” said Nick Leschly, chief bluebird. “It is especially
gratifying that despite these challenges, we have continued to
treat patients in our clinical studies at levels consistent with
prior quarters. Within the quarter, we presented compelling
clinical data across three of our core four programs: sickle cell
disease, β-thalassemia, and multiple myeloma, and made important
progress across all of our programs. In sickle cell disease, we
reached an important milestone on our transition to commercial
manufacturing process with the successful dosing of the first
sickle cell patient using drug product manufactured with
suspension-based lentiviral vector. The European launch of ZYNTEGLO
continues to progress, with positive ongoing discussions with
payers across Europe and we expect to treat our first commercial
patients in the second half of this year. Additionally, our
multiple myeloma program, partnered with BMS, continues to advance
with our submission of the BLA to the FDA and BMS’ validated MAA
submission in Europe. With this foundation and additional cash
runway, we are confident in our ability to bring our core four
programs to patients in the commercial setting by 2022 and grow our
sustainable pipeline of transformative gene and cell therapies. All
of this progress is made possible by our undaunted bluebirds, who
have shown time and again their resourcefulness and ingenuity even
under the most challenging of circumstances to bring our therapies
to patients.”
RECENT HIGHLIGHTS
- SUSPENSION LVV MANUFACTURING FOR SCD - Today, bluebird
bio announced that it has treated the first sickle cell patient
with drug product manufactured with suspension-based lentiviral
vector (sLVV). This process is intended to allow for larger scale
and more efficient manufacturing of LVV. The company intends to
submit data supporting the use of sLVV to the FDA as part of its
submission for regulatory approval of LentiGlobin™ gene therapy for
SCD in the second half of 2021.
- ELI-CEL ACCELERATED ASSESSMENT – In July 2020, the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) granted an accelerated assessment
to elivaldogene autotemcel (eli-cel, Lenti-D gene therapy) for the
treatment of cerebral adrenoleukodystrophy (CALD). The company
plans to submit a Marketing Authorization Application (MAA) to the
EMA for eli-cel in 2020. Accelerated assessment reduces the
timeframe for the EMA to review an MAA to 150 evaluation days
rather than 210. The CHMP grants review under the accelerated
assessment procedure if the medicinal product is of major interest
for public health, especially from the point of view of therapeutic
innovation.
- NEW ZYNTEGLO QTC – Today, bluebird bio announced that it
has contracted with a second qualified treatment center for
ZYNTEGLO. The center, in Essen, Germany, is prepared to treat
patients with β-thalassemia in 2020.
- IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION –
On July 29, 2020, bluebird bio and BMS announced the submission of
their Biologics License Application (BLA) to the U.S. Food and Drug
Administration (FDA) for idecabtagene vicleucel (ide-cel; bb2121),
the companies’ investigational B-cell maturation antigen
(BCMA)-directed chimeric antigen receptor (CAR) T cell
immunotherapy. This submission provides further details on the
Chemistry, Manufacturing and Controls (CMC) module to address the
outstanding regulatory requests from the FDA in May 2020 following
the original BLA submission.
- SCD DATA AT EHA – On June 12, 2020, bluebird bio
presented new data showing a near elimination of sickle cell
disease-related vaso-occlusive crises and acute chest syndrome in
the phase 1/2 clinical study of bluebird bio’s LentiGlobin™ gene
therapy for sickle cell disease at 25th EHA Congress. The company
plan to submit its BLA to the FDA based on an analysis of clinical
data from this study using complete resolution of severe
vaso-occlusive events (VOEs) as the primary endpoint with at least
18 months of follow-up post-treatment with LentiGlobin for SCD. The
company continues to plan to submit the U.S. BLA for SCD in the
second half of 2021.
- TDT DATA AT EHA – On June 12, 2020, bluebird bio
presented new data showing that the majority of evaluable patients
across genotypes achieve transfusion independence and maintain it
with near-normal hemoglobin levels in phase 3 Studies of
betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for
β-thalassemia) gene therapy presented at EHA Congress. The company
presented data from the Northstar-2 (HGB-207) clinical study of
beti-cel in patients with transfusion-dependent β-thalassemia who
do not have a β0/β0 genotype and the Northstar-3 (HGB-212) clinical
study of beti-cel in patients with transfusion-dependent
β-thalassemia who have a β0/β0 genotype or IVS-I-110 mutation.
- IDE-CEL MARKETING AUTHORIZATION APPLICATION (MAA)
VALIDATION – On May 22, 2020, BMS announced that the European
Medicines Agency (EMA) has validated its Marketing Authorization
Applications (MAA) for ide-cel. Validation of the application
confirms the submission is complete and begins the EMA’s
centralized review process.
- KARMMA DATA AT ASCO – On May 13, 2020, Bristol Myers
Squibb (NYSE: BMY) and bluebird announced positive updated results
from the pivotal, Phase 2 KarMMa study evaluating the efficacy and
safety of the companies’ investigational B-cell maturation antigen
(BCMA)-directed chimeric antigen receptor (CAR) T cell
immunotherapy, ide-cel, in patients with relapsed and refractory
multiple myeloma. The data from this study formed the basis of
recent regulatory submissions.
- EXTENDED CASH RUNWAY – In June 2020, bluebird bio raised
approximately $541.5 million in net proceeds through a public
equity offering. bluebird bio anticipates that its cash, cash
equivalents and marketable securities as of June 30, 2020, together
with projected revenue generated under our collaborative
arrangements and projected sales of products, will be sufficient to
fund operations into 2023 based on the company’s current business
plan.
UPCOMING ANTICIPATED
MILESTONES
Regulatory
- Submission of a Marketing Authorization Application to the
European Medicines Agency for eli-cel in patients with cerebral
adrenoleukodystrophy by the end of 2020.
Clinical
- Updated data presentation from HGB-206 clinical study in
patients with SCD by the end of 2020.
- Presentation of ide-cel clinical data from the CRB-401 study in
patients with multiple myeloma in 2020, in partnership with
BMS.
- Updated data presentation from ALD-102 clinical study in
patients with CALD by the end of 2020.
Commercial and Foundation Building
- ZYNTEGLO first commercial patients treated in Europe in the
second half of 2020.
- ZYNTEGLO access and reimbursement in additional EU countries
established by the end of 2020.
SECOND QUARTER 2020 FINANCIAL
RESULTS
- Cash Position: Cash, cash equivalents and marketable
securities as of June 30, 2020 and December 31, 2019 were $1.60
billion and $1.24 billion, respectively. The increase in cash, cash
equivalents and marketable securities is primarily a result of
proceeds received from the May 2020 public offering of the
Company’s common stock and a one-time upfront payment received in
connection with the Company’s amended collaboration with BMS,
partially offset by cash used in support of ordinary course
operating and commercial-readiness activities.
- Revenues: Total revenues were $198.9 million for the
three months ended June 30, 2020 compared to $13.3 million for the
three months ended June 30, 2019. Total revenues were $220.8
million for the six months ended June 30, 2020 compared to $25.8
million for the six months ended June 30, 2019. The increase for
both periods was primarily attributable to the recent amended BMS
collaboration and monetization for ex-U.S. milestones and royalties
from ide-cel and bb21217, with the majority of the revenue
recognized relating to ide-cel license and manufacturing services.
Deferred revenue under our BMS collaboration will be recognized
over time as the associated obligation to provide vector
manufacturing through development is satisfied.
- R&D Expenses: Research and development expenses were
$156.3 million for the three months ended June 30, 2020 compared to
$146.5 million for the three months ended June 30, 2019. Research
and development expenses were $310.4 million for the six months
ended June 30, 2020 compared to $269.2 million for the six months
ended June 30, 2019. The increase in both periods was primarily
driven by costs incurred to advance and expand the company’s
pipeline.
- SG&A Expenses: Selling, general and administrative
expenses were $68.6 million for both the three months ended June
30, 2020 and June 30, 2019. Selling, general and administrative
expenses were $141.9 million for the six months ended June 30, 2020
compared to $128.9 million for the six months ended June 30, 2019.
The increase in the six month period was largely attributable to
costs incurred to support the Company’s ongoing operations and
growth of its pipeline as well as commercial-readiness
activities.
- Net Loss: Net loss was $21.5 million for the three
months ended June 30, 2020 compared to $195.8 million for the three
months ended June 30, 2019. Net loss was $224.1 million for the six
months ended June 30, 2020 compared to $360.2 million for the six
months ended June 30, 2019.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
Cambridge, Mass., headquarters, we’re developing gene 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 including cerebral adrenoleukodystrophy, sickle cell
disease, β-thalassemia and multiple myeloma, using three gene
therapy technologies: gene addition, cell therapy 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, and bluebird 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 plans
for regulatory submissions for beti-cel (marketed as ZYTENGLO in
the European Union), eli-cel, ide-cel, and LentiGlobin for SCD,
including anticipated endpoints to support regulatory submissions
and timing expectations; the company’s expectations regarding the
potential for the suspension manufacturing process for lentiviral
vector; the company’s expectations and execution under its revised
operating plan, including its cash runway; its expectations for
commercialization efforts for ZYNTEGLO in Europe; and the company’s
expectations for the amended collaboration agreement with BMS; as
well as the company’s intentions regarding the timing for providing
further updates on the development and commercialization of
ZYNTEGLO and the company’s 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 COVID-19 pandemic and resulting economic conditions
will have a greater impact on the company’s operations and plans
than anticipated; that our amended collaboration with BMS will not
continue or 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 our plans for submitting a BLA for LentiGlobin for
SCD may be delayed if the FDA does not accept our comparability
plans for the use of the suspension manufacturing process for LVV;
the risk that the submission of BLA for ide-cel is not accepted for
filing by the FDA or approved in the timeline we expect, or at all;
the risk of cessation or delay of any of the ongoing or planned
clinical studies and/or our development of our product candidates,
including due to delays from the COVID-19 pandemic’s impact on
healthcare systems; 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 challenges in the commercial launch of
ZYNTEGLO in the European Union, including in managing our complex
supply chain for the delivery of drug product, in the adoption of
value-based payment models, 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
June 30,
For the six months ended
June 30,
2020
2019
2020
2019
Revenue:
Service revenue
$
78,357
$
11,093
$
95,190
$
20,304
Collaborative arrangement revenue
109,674
465
111,976
2,431
Royalty and other revenue
10,859
1,738
13,587
3,032
Total revenues
198,890
13,296
220,753
25,767
Operating expenses:
Research and development
156,308
146,540
310,431
269,180
Selling, general and administrative
68,628
68,631
141,876
128,910
Cost of royalty and other revenue
1,554
613
2,579
1,043
Change in fair value of contingent
consideration
(1,655)
214
(4,763)
510
Total operating expenses
224,835
215,998
450,123
399,643
Loss from operations
(25,945)
(202,702)
(229,370)
(373,876)
Interest income, net
2,939
9,387
8,294
19,489
Other income (expense), net
1,551
(2,936)
(2,896)
(6,325)
Loss before income taxes
(21,455)
(196,251)
(223,972)
(360,712)
Income tax (expense) benefit
(10)
469
(104)
484
Net loss
$
(21,465)
$
(195,782)
$
(224,076)
$
(360,228)
Net loss per share - basic and
diluted:
$
(0.36)
$
(3.55)
$
(3.86)
$
(6.54)
Weighted-average number of common shares
used in computing net loss per share - basic and diluted:
60,384
55,165
57,987
55,062
bluebird bio, Inc. Condensed
Consolidated Balance Sheet Data (in thousands, except per share
data) (unaudited)
As of June 30,
2020
As of December 31,
2019
Cash, cash equivalents and marketable
securities
$
1,598,793
$
1,237,966
Total assets
$
2,107,790
$
1,727,424
Total liabilities
$
425,759
$
442,431
Total stockholders' equity
$
1,682,031
$
1,284,993
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200805005907/en/
Investors: Ingrid Goldberg, 410-960-5022
igoldberg@bluebirdbio.com
Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com
Media: Jenn Snyder, 617-448-0281 jsnyder@bluebirdbio.com
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