Results demonstrate that treatment with
Reblozyl provides significant reduction in transfusion burden for
patients with beta thalassemia-associated anemia compared to
placebo
Bristol-Myers Squibb Company (NYSE:BMY) and Acceleron Pharma
Inc. (NASDAQ:XLRN) today announced that the New England Journal of
Medicine (NEJM) has published results from BELIEVE, the pivotal
Phase 3 study evaluating the safety and efficacy of Reblozyl
(luspatercept-aamt) for the treatment of anemia in adults with beta
thalassemia who require regular red blood cell (RBC)
transfusions.
“These results published in the New England Journal of Medicine
demonstrate the clinical benefit of Reblozyl, the first approved
treatment for anemia in beta thalassemia in the U.S.,” said Diane
McDowell, M.D., vice president, Hematology Global Medical Affairs,
Bristol Myers Squibb. “Patients with beta thalassemia experience
chronic anemia, often requiring life-long treatment with red blood
cell transfusions, which are associated with multiple medical
complications.1 The ability of Reblozyl to reduce the frequency and
burden of regular red blood cell transfusions is clinically
meaningful for these patients.”
In the BELIEVE trial, a significantly higher proportion of
patients achieved a >33% reduction
in RBC transfusion burden from baseline (with a reduction of at
least two units) during weeks 13-24 when treated with Reblozyl
compared to placebo, meeting the study’s primary endpoint. The
study also met all key secondary endpoints with a significantly
greater proportion of patients receiving Reblozyl compared to
placebo achieving a >33% reduction
in RBC transfusion burden during weeks 37-48 and a >50% reduction during weeks 13-24 or weeks
37-48.2
The most common adverse events (AEs) of any grade in greater
than 5% of the Reblozyl treatment group compared to placebo were
bone pain (19.7% vs. 8.3%), arthralgia (19.3% vs. 11.9%), dizziness
(11.2% vs. 4.6%), hypertension (8.1% vs. 2.8%) and hyperuricemia
(7.2% vs 0%). Grade >3 treatment
emergent AEs were reported in a greater proportion of patients
receiving Reblozyl compared to placebo (29.1% vs. 15.6%). The most
common Grade >3 AEs reported with
Reblozyl were anemia (3.1% vs. 0%), increased liver iron
concentration (2.7% vs. 0.9%) and hyperuricemia (2.7% vs. 0%)
compared to placebo, respectively. Serious adverse events were
reported in 15.2% of patients receiving Reblozyl and 5.5% of
patients receiving placebo.2
“We could not be more proud that the New England Journal of
Medicine has chosen to publish data from two pivotal studies of
Reblozyl, beginning this past January with the publication of the
MEDALIST trial results in a population of patients with
myelodysplastic syndromes,” said Habib Dable, President and Chief
Executive Officer, Acceleron. “With today’s publication of data
from the BELIEVE trial in a set of patients with beta thalassemia,
we are confident that physicians and patients will become
increasingly aware of a potential new treatment for severe anemia
associated with two diseases of high unmet medical need.”
Results from BELIEVE supported the U.S. Food and Drug
Administration (FDA) approval of Reblozyl for the treatment of
anemia in adult patients with beta thalassemia who require regular
RBC transfusions in November 2019, and were previously presented at
the 60th American Society of Hematology (ASH) Annual Meeting and
Exposition in December 2018. Longer-term analyses from BELIEVE were
presented at the 61st ASH Annual Meeting in December 2019.
Reblozyl is not indicated for use as a substitute for RBC
transfusions in patients who require immediate correction of
anemia.
About BELIEVE
BELIEVE is a Phase 3, randomized, double-blind,
placebo-controlled multicenter study comparing REBLOZYL plus best
supportive care (BSC) versus placebo plus BSC in adults who require
regular RBC transfusions (6-20 RBC units per 24 weeks with no
transfusion-free period greater than 35 days during that period)
due to beta thalassemia.2 Best supportive care included RBC
transfusions; iron-chelating agents; use of antibiotic, antiviral,
and antifungal therapy; and/or nutritional support as needed.2
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 Reblozyl®
(luspatercept-aamt)
Reblozyl is an erythroid maturation agent that promoted
late-stage red blood cell maturation in animal models.3 Bristol
Myers Squibb and Acceleron are jointly developing Reblozyl as part
of a global collaboration. Reblozyl is currently approved in the
U.S. for the treatment of anemia in adult patients with beta
thalassemia who require regular red blood cell transfusions.
Reblozyl is not indicated for use as a substitute for red blood
cell transfusions in patients who require immediate correction of
anemia.
A Biologics License Application for Reblozyl for the treatment
of anemia in adults with very low- to intermediate-risk
myelodysplastic syndromes (MDS) who have ring sideroblasts (MDS-RS)
and who require regular red blood cell transfusions is currently
under FDA review with a Prescription Drug User Fee Act (PDUFA), or
target action, date of April 4, 2020. In Europe, Bristol Myers
Squibb’s Marketing Authorization Application for the treatment of
anemia in adults with beta thalassemia and MDS is currently under
review.
Additional Clinical
Investigation
A Phase 2 trial (BEYOND) in adult patients with
non-transfusion-dependent beta thalassemia4; a Phase 2 trial in
pediatric patients with transfusion-dependent beta thalassemia5; a
Phase 3 trial (COMMANDS) in erythropoiesis-stimulating agent-naïve,
lower-risk MDS patients6; and a Phase 2 trial in myelofibrosis
patients are ongoing.7 The companies are also planning a Phase 3
trial known as INDEPENDENCE in myelofibrosis in 2020. For more
information, please visit www.clinicaltrials.gov.
Indication
REBLOZYL is indicated for the treatment of anemia in adult
patients with beta thalassemia who require regular red blood cell
(RBC) transfusions.
REBLOZYL is not indicated for use as a substitute for RBC
transfusions in patients who require immediate correction of
anemia.
Important Safety Information
WARNINGS AND PRECAUTIONS
Thrombosis/Thromboembolism
Thromboembolic events (TEE) were reported in 8/223 (3.6%)
REBLOZYL-treated patients. TEEs included deep vein thrombosis,
pulmonary embolus, portal vein thrombosis, and ischemic stroke.
Patients with known risk factors for thromboembolism (splenectomy
or concomitant use of hormone replacement therapy) may be at
further increased risk of thromboembolic conditions. Consider
thromboprophylaxis in patients at increased risk of TEE. Monitor
patients for signs and symptoms of thromboembolic events and
institute treatment promptly.
Hypertension
Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated
patients. Across clinical studies, the incidence of Grade 3 to 4
hypertension ranged from 1.8% to 8.6%. In patients with beta
thalassemia with normal baseline blood pressure, 13 (6.2%) patients
developed systolic blood pressure (SBP) >130 mm Hg and 33
(16.6%) patients developed diastolic blood pressure (DBP) >80 mm
Hg. Monitor blood pressure prior to each administration. Manage new
or exacerbations of preexisting hypertension using
anti-hypertensive agents.
Embryo-Fetal Toxicity
REBLOZYL may cause fetal harm when administered to a pregnant
woman. REBLOZYL caused increased post-implantation loss, decreased
litter size, and an increased incidence of skeletal variations in
pregnant rat and rabbit studies. Advise pregnant women of the
potential risk to a fetus. Advise females of reproductive potential
to use effective contraception during treatment and for at least 3
months after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 3.6% of patients on
REBLOZYL. Serious adverse reactions occurring in 1% of patients
included cerebrovascular accident and deep vein thrombosis. A fatal
adverse reaction occurred in 1 patient treated with REBLOZYL who
died due to an unconfirmed case of acute myeloid leukemia
(AML).
Most common adverse reactions (at least 10% for REBLOZYL and 1%
more than placebo) were headache (26% vs 24%), bone pain (20% vs
8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs
11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and
dizziness (11% vs 5%).
LACTATION
It is not known whether REBLOZYL is excreted into human milk or
absorbed systemically after ingestion by a nursing infant. REBLOZYL
was detected in milk of lactating rats. When a drug is present in
animal milk, it is likely that the drug will be present in human
milk. Because many drugs are excreted in human milk, and because of
the unknown effects of REBLOZYL in infants, a decision should be
made whether to discontinue nursing or to discontinue treatment.
Because of the potential for serious adverse reactions in the
breastfed child, breastfeeding is not recommended during treatment
and for 3 months after the last dose.
Please full Prescribing Information for REBLOZYL.
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 Acceleron
Acceleron is a biopharmaceutical company dedicated to the
discovery, development, and commercialization of therapeutics to
treat serious and rare diseases. Acceleron’s leadership in the
understanding of TGF-beta superfamily biology and protein
engineering generates innovative compounds that engage the body's
ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in
hematologic and pulmonary diseases. In hematology, Acceleron and
its global collaboration partner, Bristol Myers Squibb, are
co-promoting newly approved Reblozyl® (luspatercept-aamt), the
first and only approved erythroid maturation agent, in the United
States and are developing luspatercept for the treatment of chronic
anemia in myelodysplastic syndromes and myelofibrosis. Acceleron is
developing sotatercept for the treatment of pulmonary arterial
hypertension, having recently reported positive topline results of
the Phase 2 PULSAR trial and actively enrolling patients in the
Phase 2 SPECTRA trial.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social Media: @AcceleronPharma and
LinkedIn.
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 Reblozyl (luspatercept-aamt) will be
successfully commercialized for the indication for which it is
currently approved, that Reblozyl may not achieve its primary study
endpoints or receive regulatory approval for the additional
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. 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.
Acceleron Pharma Cautionary Statement
Regarding Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron’s strategy, future plans and prospects, including
statements regarding the development and commercialization of
Acceleron’s compounds, the timeline for clinical development and
regulatory approval of Acceleron’s compounds, the expected timing
for reporting of data from ongoing clinical trials, and the
potential of REBLOZYL® (luspatercept-aamt) as a therapeutic drug.
The words "anticipate," "believe," "could," "estimate," "expect,"
"goal," "intend," "may," "plan," "potential," "project," "should,"
"target," "will," "would," and similar expressions are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Actual results could differ materially from those included in
the forward-looking statements due to various factors, risks and
uncertainties, including, but not limited to, that the results of
any clinical trials may not be predictive of the results or success
of other clinical trials, that regulatory approval of Acceleron’s
compounds in one indication or country may not be predictive of
approval in another indication or country, that the development of
Acceleron’s compounds will take longer and/or cost more than
planned, that Acceleron or its collaboration partner, BMS, will be
unable to successfully complete the clinical development of
Acceleron’s compounds, that Acceleron or BMS may be delayed in
initiating, enrolling or completing any clinical trials, and that
Acceleron’s compounds will not receive regulatory approval or
become commercially successful products. These and other risks and
uncertainties are identified under the heading “Risk Factors”
included in Acceleron’s most recent Annual Report on Form 10-K, and
other filings that Acceleron has made and may make with the SEC in
the future.
The forward-looking statements contained in this press release
are based on management's current views, plans, estimates,
assumptions, and projections with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
References:
1 Galanello R, Origa R. Beta thalassemia. Orphanet Journal of
Rare Diseases. 2010;5(11). Available at:
https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11.
Accessed November 2019.
2 Cappellini, M.D., Phase 3 Study of Luspatercept for
B-Thalassemia Requiring Transfusion. New England Journal of
Medicine.
3 REBLOZYL U.S. Prescribing Information. Accessed February
2020.
4 ClinicalTrials.gov. A Study to Determine the Efficacy and
Safety of Luspatercept in Adults With Non Transfusion Dependent
Beta (β)-Thalassemia (BEYOND). Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03342404?term=BEYOND&cond=Beta-Thalassemia&rank=2.
Accessed February 2020
5 ClinicalTrials.gov. Study of Safety & PK of Luspatercept
(ACE-536) in Pediatric Subjects Who Require Regular RBC
Transfusions Due to Beta (β)-Thalassemia. Available at:
https://clinicaltrials.gov/ct2/show/NCT04143724?term=luspatercept%2C+beta+thalassemia%2C+pediatric&draw=2&rank=1.
Accessed February 2020.
6 ClinicalTrials.gov. Efficacy and Safety Study of Luspatercept
(ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to
IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes
(MDS) in ESA Naïve Subjects Who Require Red Blood Cell Transfusions
(COMMANDS). Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03682536?term=COMMANDS+luspatercept&rank=1.
Accessed February 2020.
7 ClinicalTrials.gov. A Safety and Efficacy Study to Evaluate
Luspatercept in Subjects With Myeloproliferative
Neoplasm-associated Myelofibrosis Who Have Anemia With and Without
Red Blood Cell-transfusion Dependence. Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03194542?term=luspatercept&cond=Myelofibrosis&rank=1.
Accessed February 2020.
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Bristol Myers Squibb Media: media@bms.com
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301-9557 Director, Corporate Communications
Investors: Todd James, (617) 649-9393 Senior Vice
President, Corporate Affairs and Investor Relations
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