Study met its primary endpoint and key
secondary endpoint, demonstrating deep and durable responses in a
heavily pre-treated multiple myeloma patient population
Safety results are consistent with the data
presented in CRB-401 study
Bristol-Myers Squibb Company (NYSE: BMY) and bluebird bio, Inc.
(Nasdaq: BLUE) today announced positive top-line results from
KarMMa, a pivotal, open-label, single arm, multicenter, Phase 2
study of idecabtagene vicleucel (ide-cel; bb2121). KarMMa, which
evaluated the efficacy and safety of the companies’ lead
investigational BCMA-targeted chimeric antigen receptor (CAR) T
cell therapy candidate for patients with relapsed and refractory
multiple myeloma, met its primary endpoint and key secondary
endpoint.
KarMMa enrolled 140 patients, of whom 128 patients were treated
with ide-cel across the target dose levels of 150-450 x 106 CAR+ T
cells. All treated patients were exposed to at least three prior
therapies, including an immunomodulatory (IMiD) agent, a proteasome
inhibitor (PI) and an anti-CD38 antibody, and all were refractory
to their last regimen. Ninety-four percent of patients were
refractory to an anti-CD38 antibody and 84% percent were triple
refractory (refractory to an IMiD agent, PI and anti-CD38
antibody).
Results for the primary endpoint (overall response rate [ORR])
and key secondary endpoint (complete response rate [CR]), as well
as duration of response (DoR) and progression-free survival (PFS)
across the target dose levels and at each of the three target doses
explored in the study are presented in the table below. The median
follow-up duration for all subjects was 11.3 months.
Ide-cel Treated
Population
150 x 106 CAR+ T cells
(N=4)
300 x 106 CAR+ T cells
(N=70)
450 x 106 CAR+ T cells
(N=54)
150-450 x 106 CAR+ T cells
(N=128)
ORR, n (%)
2 (50.0)
48 (68.6)
44 (81.5)
94 (73.4)
CR/sCR, n (%)
1 (25.0)
20 (28.6)
19 (35.2)
40 (31.3)
Median DoR, months
---
9.9
11.3
10.6
Median PFS, months
---
5.8
11.3
8.6
Median DOR and median PFS are not reported
for the 150 x 106 CAR+ T cells
dose group due to the small number of
evaluable patients
Overall, the safety results were consistent with those observed
in the phase 1 CRB-401 study, which evaluated the preliminary
safety and efficacy of ide-cel. Instances of grade 3 or higher
cytokine release syndrome (CRS) occurred in 5.5% (7/128) of
patients, including one fatal CRS event. Investigator identified
grade 3 or higher neurotoxicity events (iiNT) occurred in 3.1%
(4/128) of patients and there were no Grade 4 iiNT events reported.
Grade 3 or higher CRS and iiNT events were reported in <6% of
subjects at each target dose. CRS of any grade occurred in 83.6%
(107/128) of patients and iiNT of any grade occurred in 18%
(23/128) of patients.
“For multiple myeloma patients who have relapsed and become
refractory to current treatment options, there remains a high unmet
need, as these patients typically experience low response rates,
short response durations and poor survival,” said Kristen Hege,
M.D., Senior Vice President, Hematology/Oncology and Cell Therapy,
Early Clinical Development for Bristol-Myers Squibb. “The KarMMa
study provides further support for ide-cel as a potential
therapeutic option in this heavily pre-treated patient population,
and we are encouraged by these data, especially the outcomes
observed at the highest target dose of 450 x 106 CAR+ T cells. We
are actively preparing for submission of these data to Health
Authorities for proposed initial registration of ide-cel as a
first-in-class BCMA-targeted CAR T cell therapy.”
“Multiple myeloma is a relentless disease and there is
significant need to find new treatment options for patients who
advance through the current therapies available to them,” said
Joanne Smith-Farrell, Ph.D., oncology franchise lead and chief
business officer, bluebird bio. “With these data in hand, bluebird
bio and Bristol-Myers Squibb remain fully focused on advancing
ide-cel as quickly as possible for patients in late-line myeloma,
while continuing to execute our broad development program to
understand the potential benefits of ide-cel across earlier lines
of therapy.”
More comprehensive data from KarMMa will be submitted for
presentation at a future medical meeting.
About KarMMa
KarMMa (NCT03361748) is a pivotal, open-label, single-arm,
multi-center phase 2 study evaluating the efficacy and safety of
ide-cel in adult patients 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
and minimal residual disease evaluated by Next-Generation
Sequencing (NGS) assay. 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 IMiD agent, a PI 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 Ide-cel
Ide-cel is a CAR T cell therapy targeting B-cell maturation
antigen (BCMA), which is expressed on the surface of normal and
malignant plasma cells. The ide-cel CAR construct includes an
anti-BCMA scFv-targeting domain for antigen specificity, a
transmembrane domain, a CD3-zeta activation domain, and a 4-1BB
co-stimulatory domain hypothesized to increase T-cell activation,
proliferation and persistence. Ide-cel CAR T cells are proposed to
recognize and bind to BCMA on the surface of multiple myeloma cells
leading to apoptosis.
In November 2017, ide-cel was granted Breakthrough Therapy
Designation (BTD) by the U.S. Food and Drug Administration and
PRIority Medicines (PRIME) eligibility by the European Medicines
Agency based on preliminary clinical data from the phase 1 CRB-401
study.
Bristol-Myers Squibb and bluebird bio’s broad clinical
development program for ide-cel includes clinical studies
(KarMMa-2, KarMMa-3) in earlier lines of treatment for patients
with 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 BMS and bluebird
bio.
Ide-cel is not approved for any indication in any geography.
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
quality, 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 chimeric antigen receptor (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 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.
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 by researching 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, the possibility of unfavorable results from
additional clinical trials of ide-cel or from subsequent analysis
of existing data from the KarMMa study or existing or new data
received from additional ongoing and future studies of ide-cel,
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 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, 2018, 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 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, the possibility of unfavorable results from
additional clinical trials of ide-cel or from subsequent analysis
of existing data from the KarMMa study or existing or new data
received from additional ongoing and future studies of ide-cel,
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 such product candidate for such
indication described in this release will be commercially
successful, 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, 2018, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191206005463/en/
Bristol-Myers Squibb Company Media Inquiries:
media@bms.com 609-252-3345
Rose Weldon Rose.weldon@bms.com
Investors: Tim Power 609-252-7509
timothy.power@bms.com
Nina Goworek ngoworek@celgene.com 908-673-9711
For bluebird bio Investors: Elizabeth Pingpank,
617-914-8736 epingpank@bluebirdbio.com
Media: Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com
Bristol Myers Squibb (NYSE:BMY)
Historical Stock Chart
From Apr 2024 to May 2024
Bristol Myers Squibb (NYSE:BMY)
Historical Stock Chart
From May 2023 to May 2024