BLA submission based on results from pivotal
Phase 2 KarMMa study evaluating ide-cel in heavily pre-treated
patient population
Companies are committed to working with the
FDA to rapidly advance ide-cel through the regulatory review
process
Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq:
BLUE) today 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, for the treatment of
adult patients with relapsed and refractory multiple myeloma. 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 from March 2020.
The submission is based on results from the pivotal Phase 2
KarMMa study evaluating the efficacy and safety of ide-cel in
relapsed and refractory multiple myeloma patients exposed to an
immunomodulatory (IMiD) agent, a proteasome inhibitor (PI) and an
anti-CD38 antibody. Results from the study were shared during an
oral presentation as part of the American Society of Clinical
Oncology 2020 (ASCO20) Virtual Scientific Program.1
Multiple myeloma is a cancer of plasma cells.2 The cause of
multiple myeloma is not known and currently there is no cure;
however, there are a number of treatment options available that can
lead to response.2 Patients who have already been treated with some
available therapies but continue to have progression of their
disease have “relapsed” and “refractory” multiple myeloma, meaning
their cancer has returned after they have received initial
treatments. Patients with relapsed and refractory multiple myeloma
that have been exposed to all three major drug classes, including
an IMiD agent, a PI and an anti-CD38 antibody, have fewer treatment
options and poor outcomes, including shorter response durations and
lower overall survival.3
Ide-cel was granted Breakthrough Therapy Designation (BTD) by
the FDA, and PRIority MEdicines (PRIME) designation and validation
of its Marketing Authorization Application (MAA) by the European
Medicines Agency for relapsed and refractory multiple myeloma.
Ide-cel is not approved for any indication in any geography.
For Holders of Contingent Value Rights
(CVR), Ticker BMY-RT
U.S. FDA approval of ide-cel by March 31, 2021 is one of the
required remaining milestones of the Contingent Value Rights issued
upon the close of the Celgene acquisition in the fourth quarter of
2019. The other is U.S. FDA approval of liso-cel by December 31,
2020. The company is committed to working with FDA to progress both
applications and achieve the remaining regulatory milestones
required by the CVR.
About Ide-cel
Ide-cel is a B-cell maturation antigen (BCMA)-directed
genetically modified autologous chimeric antigen receptor (CAR) T
cell immunotherapy. The ide-cel CAR is comprised of a murine
extracellular single-chain variable fragment (scFv) specific for
recognizing BCMA, attached to a human CD8 α hinge and transmembrane
domain fused to the T cell cytoplasmic signaling domains of CD137
4-1BB and CD3-� chain, in tandem. Ide-cel recognizes and binds to
BCMA on the surface of multiple myeloma cells leading to CAR T cell
proliferation, cytokine secretion, and subsequent cytolytic killing
of BCMA-expressing cells.
Ide-cel is being developed as part of a Co-Development,
Co-Promotion and Profit Share Agreement between Bristol Myers
Squibb and bluebird bio. Ide-cel was granted accelerated assessment
by the European Medicines Agency (EMA) on March 26, 2020, and the
MAA was validated by the EMA on May 20, 2020.
About KarMMa
KarMMa (NCT03361748) is a pivotal, open-label, single-arm,
multicenter, multinational, Phase 2 study evaluating the efficacy
and safety of ide-cel in adults with relapsed and refractory
multiple myeloma in North America and Europe. The primary endpoint
of the study is overall response rate as assessed by an independent
review committee (IRC) according to the International Myeloma
Working Group (IMWG) criteria. Complete response rate is a key
secondary endpoint. Other efficacy endpoints include time to
response, duration of response, progression-free survival, overall
survival, minimal residual disease evaluated by Next-Generation
Sequencing (NGS) assay and safety. The study enrolled 140 patients,
of whom 128 received ide-cel across the target dose levels of
150-450 x 106 CAR+ T cells after receiving lymphodepleting
chemotherapy. All enrolled patients had received at least three
prior treatment regimens, including an immunomodulatory agent, a
proteasome inhibitor and an anti-CD38 antibody, and were refractory
to their last regimen, defined as progression during or within 60
days of their last therapy.
Bristol Myers Squibb: Advancing Cancer
Research
At Bristol Myers Squibb, patients are at the center of
everything we do. The goal of our cancer research is to increase
patients’ quality of life, long-term survival and make cure a
possibility. We harness our deep scientific experience,
cutting-edge technologies and discovery platforms to discover,
develop and deliver novel treatments for patients.
Building upon our transformative work and legacy in hematology
and Immuno-Oncology that has changed survival expectations for many
cancers, our researchers are advancing a deep and diverse pipeline
across multiple modalities. In the field of immune cell therapy,
this includes registrational CAR T cell agents for numerous
diseases, and a growing early-stage pipeline that expands cell and
gene therapy targets, and technologies. We are developing cancer
treatments directed at key biological pathways using our protein
homeostasis platform, a research capability that has been the basis
of our approved therapies for multiple myeloma and several
promising compounds in early- to mid-stage development. Our
scientists are targeting different immune system pathways to
address interactions between tumors, the microenvironment and the
immune system to further expand upon the progress we have made and
help more patients respond to treatment. Combining these approaches
is key to delivering potential new options for the treatment of
cancer and addressing the growing issue of resistance to
immunotherapy. We source innovation internally, and in
collaboration with academia, government, advocacy groups and
biotechnology companies, to help make the promise of
transformational medicines a reality for patients.
About Bristol Myers
Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose
mission is to discover, develop and deliver innovative medicines
that help patients prevail over serious diseases. For more
information about Bristol Myers Squibb, visit us at BMS.com or
follow us on LinkedIn, Twitter, YouTube, Facebook and
Instagram.
Celgene and Juno Therapeutics are wholly owned subsidiaries of
Bristol-Myers Squibb Company. In certain countries outside the
U.S., due to local laws, Celgene and Juno Therapeutics are referred
to as, Celgene, a Bristol Myers Squibb company and Juno
Therapeutics, a Bristol Myers Squibb company.
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.
bluebird bio is a trademark of bluebird bio, Inc.
Bristol Myers Squibb Cautionary
Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of 1995
regarding, among other things, the research, development and
commercialization of pharmaceutical products. All statements that
are not statements of historical facts are, or may be deemed to be,
forward-looking statements. Such forward-looking statements are
based on historical performance and current expectations and
projections about our future financial results, goals, plans and
objectives and involve inherent risks, assumptions and
uncertainties, including internal or external factors that could
delay, divert or change any of them in the next several years, that
are difficult to predict, may be beyond our control and could cause
our future financial results, goals, plans and objectives to differ
materially from those expressed in, or implied by, the statements.
These risks, assumptions, uncertainties and other factors include,
among others, that future study results will be consistent with the
results to date, that ide-cel, or bb2121, may not achieve its
primary study endpoint or receive regulatory approval for the
indication described in this release in the currently anticipated
timeline or at all and, if approved, whether such product candidate
for such indication described in this release will be commercially
successful. No forward-looking statement can be guaranteed.
Forward-looking statements in this press release should be
evaluated together with the many risks and uncertainties that
affect Bristol Myers Squibb’s business and market, particularly
those identified in the cautionary statement and risk factors
discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for
the year ended December 31, 2019, as updated by our subsequent
Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and
other filings with the Securities and Exchange Commission. The
forward-looking statements included in this document are made only
as of the date of this document and except as otherwise required by
applicable law, Bristol Myers Squibb undertakes no obligation to
publicly update or revise any forward-looking statement, whether as
a result of new information, future events, changed circumstances
or otherwise.
bluebird bio Cautionary Statement
Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of 1995
regarding, among other things, the research, development and
commercialization of ide-cel. All statements that are not
statements of historical facts are, or may be deemed to be,
forward-looking statements. Such forward-looking statements are
based on historical performance and current expectations and
projections about our future financial results, goals, plans and
objectives and involve inherent risks, assumptions and
uncertainties, including internal or external factors that could
delay, divert or change any of them in the next several years, that
are difficult to predict, may be beyond our control and could cause
our future financial results, goals, plans and objectives to differ
materially from those expressed in, or implied by, the statements.
These risks, assumptions, uncertainties and other factors include,
among others, the possibility that the BLA submission may not be
accepted for filing by the FDA without the provision of further
information or responses to additional requests, if at all, that
ide-cel may not receive regulatory approval for the indication
described in this release in the currently anticipated timeline or
at all, and, if approved, whether ide-cel will be commercially
successful, that the positive results for ide-cel may not continue
in additional clinical trials, and that the collaboration with
Bristol Myers Squibb may not continue or be successful. No
forward-looking statement can be guaranteed. Forward-looking
statements in this press release should be evaluated together with
the many risks and uncertainties that affect bluebird bio’s
business, particularly those identified in the risk factors
discussion in bluebird bio’s Annual Report on Form 10-K for the
year ended December 31, 2019, as updated by our subsequent
Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and
other filings with the Securities and Exchange Commission. The
forward-looking statements included in this document are made only
as of the date of this document and except as otherwise required by
applicable law, bluebird bio undertakes no obligation to publicly
update or revise any forward-looking statement, whether as a result
of new information, future events, changed circumstances or
otherwise.
Hyperlinks are provided as a convenience and for informational
purposes only. Neither Bristol Myers Squibb nor bluebird bio bears
responsibility for the security or content of external websites or
websites outside of their respective control.
References
- Munshi NC, et al. Idecabtagene vicleucel (ide-cel; bb2121), a
BCMA-targeted CAR T cell therapy, in patients with relapsed and
refractory multiple myeloma (RRMM): initial KarMMa results. ASCO
2020 Virtual Scientific Program. Abstract #8503.
- American Cancer Society. What is Multiple Myeloma? Available
at:
https://www.cancer.org/cancer/multiple-myeloma/about/what-is-multiple-myeloma.html.
Accessed June 2020.
- Jagannath S, et al. KarMMa-RW: a study of real world treatment
patterns in heavily pretreated patients with relapsed and
refractory multiple myeloma (RRMM) and comparison of outcomes to
KarMMa. ASCO 2020 Virtual Scientific Program. Abstract #8525.
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version on businesswire.com: https://www.businesswire.com/news/home/20200729005776/en/
Bristol Myers Squibb Media Inquiries:
609-252-3345 media@bms.com
Rose Weldon rose.weldon@bms.com
Investors:
Tim Power 609-252-7509 timothy.power@bms.com
bluebird bio
Media: Victoria von Rinteln 617-914-8774
vvonrinteln@bluebirdbio.com
Investors: Ingrid Goldberg 410-960-5022
igoldberg@bluebirdbio.com
Elizabeth Pingpank 617-914-8736 epingpank@bluebirdbio.com
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