U.S. FDA set a target action date of August 17,
2020
Application based on results from the TRANSCEND
NHL 001 trial, the largest study of CD19-directed CAR T cells to
support a BLA to date
Liso-cel is a CD19-directed CAR T-cell product
with a defined composition of CAR+ viable T cells consisting of
purified CD8+ and CD4+ T cells
Bristol-Myers Squibb Company (NYSE: BMY) today announced that
the U.S. Food and Drug Administration (FDA) has accepted for
Priority Review its Biologics License Application (BLA) for
lisocabtagene maraleucel (liso-cel), the company’s autologous
anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with
a defined composition of purified CD8+ and CD4+ CAR T cells for the
treatment of adult patients with relapsed or refractory (R/R) large
B-cell lymphoma after at least two prior therapies. The FDA has set
a Prescription Drug User Fee Act (PDUFA) goal date of August 17,
2020.
“There remains a critical need for additional therapies in large
B-cell lymphoma, particularly for relapsed or refractory patients,”
said Stanley Frankel, M.D., senior vice president, Cellular Therapy
Development, Bristol-Myers Squibb. “Based on the TRANSCEND NHL 001
data, liso-cel has the potential to expand treatment options for
those affected by this aggressive blood cancer who did not respond
to initial therapies or whose disease has relapsed. This BLA
acceptance and Priority Review designation is an important step as
we work to improve treatment for these patients in need.”
The BLA, submitted by Juno Therapeutics, a wholly owned
subsidiary of Bristol-Myers Squibb Company, is based on the safety
and efficacy results from the TRANSCEND NHL 001 trial, evaluating
liso-cel in 268 patients with R/R large B-cell lymphoma, including
diffuse large B-cell lymphoma (DLBCL), high-grade lymphoma, primary
mediastinal B-cell lymphoma and Grade 3B follicular lymphoma.
TRANSCEND NHL 001 is the largest study of CD19-directed CAR T cells
to support a BLA to date and was recently the subject of an oral
presentation at the 61st American Society of Hematology Annual
Meeting and Exposition.
According to the FDA, a Priority Review designation will direct
overall attention and resources to the evaluation of applications
for drugs that, if approved, would be significant improvements in
the safety or effectiveness of the treatment, diagnosis, or
prevention of serious conditions when compared to standard
applications.
Liso-cel was previously granted Breakthrough Therapy and
Regenerative Medicine Advanced Therapy designations by the FDA for
R/R aggressive large B-cell non-Hodgkin lymphoma, including DLBCL,
not otherwise specified (de novo or transformed from indolent
lymphoma), PMBCL or Grade 3B FL, and Priority Medicines (PRIME)
scheme by the European Medicines Agency for R/R DLBCL.
Liso-cel is an investigational compound that is not approved for
use in any country.
About Large B-cell
Lymphoma
Diffuse large B-cell lymphoma (DLBCL) is the most common of
large B-cell lymphomas. It is an aggressive form of non-Hodgkin
lymphoma (NHL), accounting for three out of every five cases.
Approximately one-third of patients with DLBCL relapse after
receiving first-line treatment, and about 10% have refractory
disease. Historically, median life expectancy for patients who
relapse or are refractory to current standard of care treatments is
approximately six months.
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 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 Lisocabtagene Maraleucel
(liso-cel)
Liso-cel is an investigational chimeric antigen receptor (CAR)
T-cell therapy designed to target CD19, which is a surface
glycoprotein expressed during normal B-cell development and
maintained following malignant transformation of B cells. Liso-cel
aims to target CD19-expressing cells through a CAR construct that
includes an anti-CD19 single-chain variable fragment (scFv)
targeting domain for antigen specificity, a transmembrane domain, a
4-1BB costimulatory domain hypothesized to increase T-cell
proliferation and persistence, and a CD3-zeta T-cell activation
domain. The defined composition of CAR-positive viable T-cells
(consisting of CD8 and CD4 components) in liso-cel may reduce
product variability; however, the clinical significance of defined
composition is unknown.
About TRANSCEND NHL 001
TRANSCEND NHL 001 is an open-label, multicenter, pivotal phase 1
study to determine the safety, antitumor activity, and
pharmacokinetics of liso-cel in patients with R/R B-cell NHL,
including DLBCL, HGL, PMBCL, Grade 3B FL. Mantle cell lymphoma is
investigated in a separate cohort. The primary outcome measures
included treatment-related adverse events, dose-limiting toxicities
and objective response rate. Key secondary outcome measures
included complete response rate, duration of response, and
progression-free survival. The TRANSCEND program is a broad
clinical program evaluating liso-cel in multiple disease states and
treatment stages.
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.
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 studies involving liso-cel, that such product candidate
may not receive regulatory approval for the indications described
in this release in the currently anticipated timeline or at all
and, if approved, whether such product candidate for such
additional indications described in this release will be
commercially successful. No forward-looking statement can be
guaranteed. It should be noted that a priority review designation
does not change the standards for FDA approval. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20200213005238/en/
Bristol-Myers Squibb Company Media Inquiries:
609-252-3345 media@bms.com Rose Weldon rose.weldon@bms.com
Investors: Tim Power 609-252-7509
timothy.power@bms.com
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