Results showed treatment with
Reblozyl plus best supportive care improved anemia in 77% of
patients compared to placebo
Reblozyl was generally well tolerated and
improvements in hemoglobin correlated with improved
patient-reported outcomes over a continuous 12-week
interval
In the study, 89.6% of patients treated with
Reblozyl remained transfusion free vs. 67.3% of patients in the
placebo arm at weeks 1-24
Results featured in Presidential Symposium
of European Hematology Association’s Virtual Congress as one of top
six abstracts submitted
Bristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced the first data from the Phase 2
BEYOND study evaluating Reblozyl® (luspatercept-aamt), a
first-in-class erythroid maturation agent, plus best supportive
care in adult patients with non-transfusion dependent (NTD) beta
thalassemia, were presented at the European Hematology Association
(EHA) 2021 Virtual Congress as part of its Presidential Symposium
(Abstract #S101). Results demonstrated that 77.7% of patients
treated with Reblozyl achieved a hemoglobin increase (≥1.0
gram/deciliter) compared to 0% of patients in the placebo arm.
Changes in patient-reported outcomes also correlated with increases
in hemoglobin. NTD beta thalassemia is a term used to describe
patients who do not require lifelong regular red blood cell (RBC)
transfusions for survival, although they may require occasional or
even frequent transfusions, usually for defined periods of
time.
“Patients with non-transfusion dependent beta thalassemia
experience chronic anemia and iron overload, which may lead to a
range of clinical complications, and treatment options are greatly
needed,” said Ali Taher, M.D., Ph.D., FRCP, of American University
of Beirut and BEYOND study investigator. “Results from the BEYOND
study show the clinical potential of luspatercept to sustain the
elevation of hemoglobin levels in a majority of patients regardless
of their baseline hemoglobin status, and improvements were noted in
quality of life outcomes in adults with non-transfusion dependent
beta thalassemia.”
Reblozyl is the first and only erythroid maturation agent
approved in the European Union, United States and Canada to address
anemia-associated beta thalassemia and lower-risk myelodysplastic
syndromes, representing an important class of therapy for eligible
patients.1,2,3
“We are very encouraged by the magnitude of improvement seen
among Reblozyl-treated patients in the BEYOND trial,” said Habib
Dable, President and Chief Executive Officer of Acceleron. “These
data further strengthen our confidence in Reblozyl’s potential to
become a meaningful option for this important, underserved patient
population around the world.”
“The results we are presenting at EHA continue to highlight
multiple benefits observed with Reblozyl to treat anemia and
achieve transfusion independence, as well as show its potential for
patients with non-transfusion dependent disease who face a range of
serious, often lifelong health complications,” said Noah Berkowitz,
M.D., Ph.D., senior vice president, Hematology Development, Bristol
Myers Squibb. “Along with our partners at Acceleron, we are
committed to advancing our clinical program for Reblozyl for
patients living with anemia-associated blood disorders.”
BEYOND Study Results
BEYOND is a Phase 2, randomized, double-blind,
placebo-controlled multi-center study to determine the efficacy and
safety of Reblozyl versus placebo in adults with non-transfusion
dependent (NTD) beta thalassemia. Eligible patients were ≥18 years
with beta thalassemia or hemoglobin (Hb) E beta thalassemia and
received ≤5 red blood cell (RBC) units in the 24 weeks prior to
randomization, with mean baseline Hb ≤10.0 gram/deciliter
(g/dL).4
In the study, 145 patients were randomized 2:1 to receive
Reblozyl, 1 milligram/kilogram (titration up to 1.25 mg/kg) or
placebo subcutaneously every 3 weeks for ≥48 weeks. Patients in
both arms continued to receive best supportive care, including RBC
transfusions as indicated and iron chelation therapy. The primary
endpoint was achievement of ≥1.0 g/dL mean Hb increase from
baseline over a continuous 12-week interval from weeks 13-24 in the
absence of RBC transfusions. Secondary endpoints included
proportion of patients who remained transfusion free over weeks
1-24, who achieved mean Hb increase of ≥1.5 g/dL from baseline to
weeks 13-24, and mean change in NTD beta thalassemia
patient-reported outcome tiredness and weakness (NTDT-PRO T/W)
domain scores (higher scores reflect worse quality of life;
QoL).4
Over a continuous 12-week interval from weeks 13-24 in the
absence of RBC transfusions, 74 of 96 (77.1%) patients in the
Reblozyl treatment arm achieved the study’s primary endpoint, ≥1.0
g/dL mean Hb increase from baseline, versus 0 of 49 (0%) patients
in the placebo arm (P<0.0001). The primary endpoint was achieved
by 40 of 55 (72.7%) patients in the Reblozyl arm with mean baseline
Hb of <8.5 g/dL versus 0 (0%) of patients in the placebo arm
(P<0.0001), and 34 of 41 patients (82.9%) with mean baseline Hb
of ≥8.5 g/dL versus 0 patients (0%) in the placebo arm
(P<0.0001). In a key secondary endpoint of the study, during
weeks 13-24, 50 of 96 patients (52.1%) in the Reblozyl arm achieved
mean Hb increase of ≥1.5 g/dL compared to baseline versus 0
patients (0%) in the placebo arm (P<0.0001). 89.6% of patients
in the Reblozyl arm remained transfusion free at weeks 1-24 versus
67.3% of patients in the placebo arm (P=0.0013). Improvements in
patient-reported QoL outcomes (tiredness and weakness) were also
observed to correlate with Hb increases.
The most common treatment-emergent adverse events of any grade
occurring in ≥5% of patients were bone pain (36.5% Reblozyl versus
6.1% placebo), headache (30.2% versus 20.4%), and arthralgia (29.2%
versus 14.3%). No malignancies or thromboembolic events were
reported in patients treated with Reblozyl.
About Beta Thalassemia
Beta thalassemia is an inherited blood disorder caused by a
genetic defect in hemoglobin. It is one of the most common
autosomal recessive disorders, and the total annual incidence of
symptomatic individuals is estimated at 1 in 100,000 people
globally and 1 in 10,000 people in the European Union.5 The disease
is associated with ineffective erythropoiesis, which results in the
production of fewer and less healthy red blood cells (RBCs), often
leading to severe anemia—a condition that can be debilitating and
can lead to other complications for patients—as well as other
serious health issues. Treatment options for anemia associated with
beta thalassemia are limited, consisting mainly of frequent RBC
transfusions that have the potential to contribute to iron
overload, which can cause serious complications such as organ
damage.5 Non-transfusion dependent thalassemia is a term used to
describe patients who do not require lifelong regular transfusions
for survival, although they may experience a range of clinical
complications and require occasional or even frequent transfusions,
usually for defined periods of time.6
About Reblozyl®
Reblozyl (luspatercept-aamt), a first-in-class erythroid
maturation agent, promotes late-stage red blood cell maturation in
animal models. 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, and
- anemia failing an erythropoiesis stimulating agent and
requiring 2 or more red blood cell units over 8 weeks in adult
patients with very low- to intermediate-risk myelodysplastic
syndromes with ring sideroblasts (MDS-RS) or with
myelodysplastic/myeloproliferative neoplasm with ring sideroblasts
and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood
cell transfusions in patients who require immediate correction of
anemia.
U.S. Important Safety Information
WARNINGS AND PRECAUTIONS
Thrombosis/Thromboembolism
In adult patients with beta thalassemia, 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. In
adult patients with MDS with normal baseline blood pressure, 26
(29.9%) patients developed SBP ≥130 mm Hg and 23 (16.4%) patients
developed 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 final dose.
ADVERSE REACTIONS
Beta Thalassemia
- 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%).
Myelodysplastic
Syndromes
- Grade >3 (≥2%) adverse
reactions included fatigue, hypertension, syncope and
musculoskeletal pain. A fatal adverse reaction occurred in 5 (2.1%)
patients.
- The most common (≥10%) adverse reactions included fatigue,
musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity
reactions, hypertension, headache, upper respiratory tract
infection, bronchitis, and urinary tract infection.
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 see full Prescribing Information and Summary of
Product Characteristics for REBLOZYL
Bristol Myers Squibb: Creating a Better
Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision—transforming
patients’ lives through science. The goal of the company’s cancer
research is to deliver medicines that offer each patient a better,
healthier life and to make cure a possibility. Building on a legacy
across a broad range of cancers that have changed survival
expectations for many, Bristol Myers Squibb researchers are
exploring new frontiers in personalized medicine, and through
innovative digital platforms, are turning data into insights that
sharpen their focus. Deep scientific expertise, cutting-edge
capabilities and discovery platforms enable the company to look at
cancer from every angle. Cancer can have a relentless grasp on many
parts of a patient’s life, and Bristol Myers Squibb is committed to
taking actions to address all aspects of care, from diagnosis to
survivorship. Because as a leader in cancer care, Bristol Myers
Squibb is working to empower all people with cancer to have a
better future.
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 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 commercialization, research, and
development efforts in hematologic and pulmonary diseases. In
hematology, Acceleron and its global collaboration partner, Bristol
Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the
first and only approved erythroid maturation agent, in the United
States for the treatment of anemia in certain blood disorders. The
Companies are also developing luspatercept for the treatment of
chronic anemia in patient populations of MDS, beta-thalassemia, and
myelofibrosis. In pulmonary, Acceleron is developing sotatercept
for the treatment of pulmonary arterial hypertension, having
recently reported positive topline results of the Phase 2 PULSAR
trial.
For more information, please visit https://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 plus best supportive care may not
receive regulatory approval for the additional indication described
in this release in the currently anticipated timeline or at all
and, if approved, whether such combination treatment for such
additional 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, 2020, 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 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, Bristol-Myers
Squibb Company (“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, Quarterly Report on Form 10-Q, 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:
- REBLOZYL Summary of Product Characteristics. Accessed May
2021.
- REBLOZYL U.S. Prescribing Information. Accessed May 2021.
- REBLOZYL Canada Product Monograph. Accessed May 2021.
- 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 May 2021.
- 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 May 2021.
- Musallam, K. M., Rivella, S., Vichinsky, E.,
& Rachmilewitz, E. A. (2013). Non-transfusion-dependent
thalassemias. Haematologica, 98(6), 833–844.
https://doi.org/10.3324/haematol.2012.066845. Accessed May
2021.
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Bristol Myers Squibb Media Inquiries:
media@bms.com 609-252-3345
Investors: Tim Power 609-252-7509
timothy.power@bms.com
Nina Goworek 908-673-9711 ngoworek@bms.com
Acceleron Pharma Inc. Investors: Jamie Bernard,
IRC, 617-301-9650 Associate Director, Investor Relations
Media: Matt Fearer, (617) 301-9557 Senior Director,
Corporate Communication
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