First pivotal study of BCMA-directed CAR T cell
therapy in triple-class exposed relapsed and refractory multiple
myeloma
Biologics License Application (BLA) for ide-cel
was accepted by FDA for Priority Review, with a target action date
of March 27, 2021
Results from the pivotal Phase 2 KarMMa study evaluating the
efficacy and safety of bluebird bio, Inc. (Nasdaq: BLUE) and
Bristol Myers Squibb’s (NYSE: BMY) investigational B-cell
maturation antigen (BCMA)-directed chimeric antigen receptor (CAR)
T cell immunotherapy, idecabtagene vicleucel (ide-cel; bb2121), in
adult patients with relapsed and refractory multiple myeloma (RRMM)
who have received at least three prior therapies, including an
immunomodulatory agent, a proteasome inhibitor and an anti-CD38
antibody, were published today in The New England Journal of
Medicine.1
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).
“The publication of KarMMa, the first pivotal study of a CAR T
cell therapy in multiple myeloma, in The New England Journal of
Medicine, underscores the importance of these data and the
unprecedented outcomes observed in this triple-class exposed
patient population, following a single infusion of ide-cel,” said
David Davidson, M.D., chief medical officer, bluebird bio.
“Together with our partners at Bristol Myers Squibb, we look
forward to the prospect of bringing this first-in-class
BCMA-directed CAR T therapy to patients.”
Clinically meaningful responses were reported in heavily
pre-treated patients across all dose levels and in multiple
high-risk subgroups, including those with high-risk cytogenetics,
triple- or penta-refractory disease, high tumor burden at baseline,
and extramedullary disease. Clinically meaningful improvement was
also observed across measures for median duration of response,
median progression-free survival and overall survival in treated
patients.
In the KarMMa study, ide-cel demonstrated a safety profile
consistent with known toxicities of CAR T cell therapies,
regardless of dose level. The most frequently reported adverse
events were cytopenia and cytokine release syndrome.
“Despite the progress made in the treatment of multiple myeloma
over the past decade, long-term disease-free survival is uncommon
and relapses are inevitable. Currently, the patients who have
progressed through the three main classes of therapy do not have
very effective therapeutic options and their outcome are often
poor,” said Nikhil C. Munshi, M.D., lead author, Associate
Director, The Jerome Lipper Multiple Myeloma Center at the
Dana-Farber Cancer Institute, Boston, Massachusetts. “The deep and
durable responses observed in a large majority of patients in the
KarMMa study published today in The New England Journal of Medicine
demonstrate the potential of ide-cel to address a high unmet need
for patients with heavily pre-treated and highly refractory
multiple myeloma.”
The research included in this manuscript was first presented at
the American Society of Clinical Oncology 2020 (ASCO20) Virtual
Scientific Program in May 2020. This is the second publication in
The New England Journal of Medicine to report results of a study
with ide-cel.
Ide-cel is not approved for any indication in any geography.
About Idecabtagene Vicleucel (ide-cel,
bb2121)
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.
bluebird bio and Bristol Myers Squibb’s broad clinical
development program for ide-cel includes clinical studies
(KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for
patients with multiple myeloma, including high risk newly diagnosed
multiple myeloma. For more information visit
clinicaltrials.gov.
Ide-cel is being developed as part of a Co-Development,
Co-Promotion and Profit Share Agreement between Bristol Myers
Squibb and bluebird bio.
The European Medicines Agency (EMA) has validated the MAA for
ide-cel and it is currently under review. The U.S. Food and Drug
Administration accepted the ide-cel Biologics License Application
for priority review on September 22, 2020 and set a Prescription
Drug User Fee Act (PDUFA) goal date of March 27, 2021.
About KarMMa2-4
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 the 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.
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, including 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.
bluebird bio is a trademark of bluebird bio, Inc.
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 ide-cel may not receive the
FDA’s 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, that
ide-cel may not receive marketing approval in the EU or in any
jurisdictions outside of the US and the EU, 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, 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.
References
- Munshi NC, Anderson LD, Shah N, et al. Idecabtagene Vicleucel
in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021
February 25;384:705-716.
- 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 #39T850339T.
- Raje N, Berdeja J, Lin Y, et al. Anti-BCMA CAR T-cell therapy
bb2121 in relapsed or refractory multiple myeloma. N Engl J Med.
2019 May 2;380(18):1726-1737.
- ClinicalTrials.gov. Efficacy and safety study of bb2121 in
subjects with relapsed and refractory multiple myeloma (KarMMa).
Available at: https://clinicaltrials.gov/ct2/show/NCT03361748.
Accessed February 2021.
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version on businesswire.com: https://www.businesswire.com/news/home/20210224006141/en/
bluebird bio Media: Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com Investors: Ingrid Goldberg,
857-217-0490 igoldberg@bluebirdbio.com Elizabeth Pingpank,
617-914-8736 epingpank@bluebirdbio.com
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