Maintenance treatment with CC-486 resulted
in a significant improvement in overall survival compared with
placebo for front-line AML patients
CC-486 had a manageable safety
profile
Data presented at the 2019 American Society
of Hematology (ASH) Annual Meeting
Bristol-Myers Squibb Company (NYSE: BMY) today announced
clinical results from the QUAZAR AML-001 study, evaluating
investigational agent CC-486 as maintenance therapy in a broad
population of patients with front-line, newly diagnosed acute
myeloid leukemia (AML) who have achieved remission with intensive
induction chemotherapy. Data were presented during a late-breaker
oral presentation at the 2019 ASH Annual Meeting in Orlando, Fla.
In the QUAZAR AML-001 study, treatment with CC-486 in the
maintenance setting provided patients a statistically significant
and clinically meaningful improvement in overall survival (OS) and
relapse-free survival (RFS), as compared to those patients treated
with placebo.
Patients in the phase 3, international, randomized,
double-blind, placebo-controlled study QUAZAR AML-001 were at least
55 years old, had de novo or secondary AML with intermediate or
poor-risk cytogenetics and had achieved their first complete
remission (CR) or complete remission with incomplete count recovery
(CRi) after intensive induction chemotherapy. Patients had received
intensive induction chemotherapy, with or without consolidation
chemotherapy per investigator’s choice and were deemed not
candidates for hematopoietic stem-cell transplant prior to study
entry.
“Despite a number of recent advances in the treatment of AML,
the prognosis remains poor, as most patients will relapse and
ultimately die of their disease,” said Dr. Andrew Wei, MBBS, Ph.D.,
from Alfred Hospital and Monash University, Melbourne, Australia.
“The role of maintenance therapy in AML has historically been a
contentious issue. Based on the results of the QUAZAR study, we are
excited about the clinical development of CC-486 and the potential
to establish maintenance therapy as a new treatment paradigm for
patients with AML in first remission.”
Following intensive induction chemotherapy, 81% of patients had
achieved a CR and 19% of patients had achieved a CRi. Eighty
percent of patients had received at least one cycle of
consolidation therapy prior to enrollment in the study. Four
hundred seventy-two patients were then randomized 1:1 to receive
initially either investigational CC-486 300mg (n=238) or placebo
(n=234) once daily for 14 days of each 28-day cycle. Patients
remained on treatment until unacceptable toxicity or disease
progression.
At a median follow-up of 41.2 months, the primary endpoint of OS
was significantly improved for patients receiving CC-486 compared
to placebo. Median OS from time of randomization was 24.7 months in
the CC-486 arm compared to 14.8 months for placebo (p=0.0009; HR
0.69 [95% CI: 0.55, 0.86]). Median RFS, the key secondary endpoint,
was 10.2 months for those receiving CC-486 compared to 4.8 months
for those receiving placebo (p=0.0001; HR 0.65 [95% CI: 0.52,
0.81]). Improvements in OS and RFS for those treated with CC-486
compared to placebo were demonstrated, regardless of cytogenetic
risk category, prior consolidation or CR/CRi status at enrollment.
Health-related quality of life (HRQoL) was preserved from baseline
for patients receiving CC-486 compared to placebo during
treatment.
The median duration of treatment was 12 cycles (1-80) for CC-486
and 6 cycles with placebo (1-73). The most commonly occurring
adverse events (AEs) of all grades with CC-486 and placebo,
respectively, were nausea (65% vs. 24%), vomiting (60% vs. 10%) and
diarrhea (50% vs. 22%). The most common grade 3-4 AEs for CC-486
and placebo, respectively, were neutropenia (41% vs. 24%),
thrombocytopenia (23% vs. 22%) and anemia (14% vs. 13%). Serious
AEs were reported in 34% of CC-486 patients and 25% of placebo
patients, and were mainly infections, which occurred in 17% and 8%
of CC-486 and placebo patients, respectively. There were 13% of
CC-486 patients and 4% of placebo patients who discontinued
treatment due to AEs.
“We are extremely encouraged by the results of the QUAZAR
AML-001 study as a part of our continuing commitment to both
epigenetic research and myeloid diseases,” said Samit Hirawat,
M.D., Chief Medical Officer of Bristol-Myers Squibb. “We now look
forward to taking the next steps to bring CC-486 to eligible AML
patients in need.”
Based on the results of QUAZAR AML-001, Bristol-Myers Squibb is
planning regulatory submissions in the first half of 2020.
CC-486 is not approved for any use in any country.
About AML
Acute myeloid leukemia (AML) is the most common type of acute
leukemia. AML starts in the bone marrow but moves quickly into the
blood. Unlike in normal blood cell development, in AML the rapid
build up of abnormal white blood cells in the bone marrow may
interfere with the production of normal blood cells, resulting in
decreased healthy white blood cells, red blood cells and platelets.
AML is a complex, diverse disease associated with multiple genetic
mutations and usually gets worse quickly if not treated. There will
be an estimated 21,450 new cases of AML in the United States this
year, accounting for 1.2% of all cancer cases, with an estimated
10,920 deaths resulting from the disease. There are an estimated
61,048 people living with AML in the United States.
About QUAZAR AML-001
QUAZAR AML-001 is a phase 3, international, randomized,
double-blind, placebo-controlled study of CC-486 as AML maintenance
therapy in patients who achieved first complete remission (CR) or
complete remission with incomplete blood count recovery (CRi)
following intensive induction chemotherapy (with or without
consolidation). The primary endpoint of the study was overall
survival. Secondary endpoints included relapse-free survival (RFS),
safety and tolerability, healthcare resource utilization and
patient-reported outcomes per the FACIT-Fatigue Scale and EQ-5D
questionnaire. The study enrolled 472 patients, randomized 1:1 to
receive initially either oral CC-486 300mg or placebo once daily
for 14 days of a 28-day cycle plus best supportive care. Patients
remained on treatment until unacceptable toxicity or disease
progression.
About CC-486
CC-486 is an oral hypomethylating agent that incorporates into
DNA and RNA allowing for sustained epigenetic regulation due to
prolonged exposure. The main mechanism of action is thought to be
hypomethylation of DNA, as well as direct cytotoxicity to abnormal
hematopoietic cells in the bone marrow. Hypomethylation may restore
normal function to genes that are critical for differentiation and
proliferation.
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
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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 results from future studies involving CC-486
will be consistent with the results to date, that CC-486 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.
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