Submission based on results from Phase 2
BEYOND study of Reblozyl plus best supportive care in adults with
NTD beta thalassemia
Bristol Myers Squibb (NYSE: BMY) today announced that the U.S.
Food and Drug Administration (FDA) has accepted for priority review
the supplemental Biologics License Application (sBLA) for Reblozyl®
(luspatercept-aamt), a first-in-class erythroid maturation agent,
for the treatment of anemia in adults with non-transfusion
dependent (NTD) beta thalassemia. The FDA has set a Prescription
Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition,
the European Medicines Agency has validated the Type II variation
for Reblozyl in NTD beta thalassemia. Reblozyl is being
co-developed and co-commercialized with Merck & Co., Inc.,
known as MSD outside the United States and Canada, following
Merck’s recent acquisition of Acceleron Pharma, Inc.
These applications were based on safety and efficacy results
from the pivotal Phase 2 BEYOND study evaluating Reblozyl plus best
supportive care in patients with NTD beta thalassemia.
“Patients with non-transfusion dependent beta thalassemia may
not require lifelong blood transfusions for survival, but their
need for effective treatment options is significant as they face a
range of clinical complications due to chronic anemia and iron
overload,” said Noah Berkowitz, M.D., Ph.D., senior vice president,
Hematology Development, Bristol Myers Squibb. “Reblozyl is an
important therapy approved for anemia associated with beta
thalassemia and lower-risk myelodysplastic syndromes in multiple
countries, including the United States and within the European
Union. Along with our partners at Merck, we are committed to
continuing to advance our clinical program for Reblozyl and look
forward to working with the FDA during its review of our
application for this underserved patient population.”
Updated analyses from the BEYOND study will be presented at the
63rd American Society of Hematology Annual Meeting and Exposition
from December 11-14.
About BEYOND
BEYOND (NCT03342404) is a Phase 2, double-blind, randomized,
placebo-controlled, multicenter study to determine the efficacy and
safety of luspatercept-aamt (ACE-536) versus placebo in adults with
non-transfusion dependent beta thalassemia. The study is divided
into the Screening Period, Double-blind Treatment Period (DBTP) and
Post-Treatment Follow-up Period (PTFP) and randomized 145 subjects
at a 2:1 ratio of Reblozyl versus placebo. The primary endpoint of
the study is the proportion of subjects who have an increase from
baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous
12-week interval from Week 13 to Week 24 of treatment in the
absence of transfusions. Key secondary endpoints include mean
change in non-transfusion dependent beta thalassemia-patient
reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain
score and baseline hemoglobin (Hb).
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.1 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.1 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.2
About Reblozyl®
Reblozyl, the first and only erythroid maturation agent,
promotes late-stage red blood cell maturation in animal models.1
Bristol Myers Squibb and Merck & Co., Inc., through Merck’s
acquisition of 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
syndrome 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.
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 3-4
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 occurring in 1% of patients included
cerebrovascular accident and deep vein thrombosis. A fatal adverse
reaction occurred in one patient treated with REBLOZYL who died due
to an unconfirmed case of 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%).1
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 for REBLOZYL
Bristol Myers Squibb: Creating a Better
Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision —
transforming people’s 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,
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company to look at cancer from every angle. Cancer can have a
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Myers Squibb is committed to taking actions to address all aspects
of care, from diagnosis to survivorship. Because as a leader in
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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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Juno Therapeutics, Inc. is a wholly owned subsidiary 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 that Reblozyl (luspatercept-aamt) may
not receive regulatory approval for the additional indication
described in this release in the currently anticipated timeline or
at all, that any marketing approvals, if granted, may have
significant limitations on their use, and, if approved, whether
such product candidate 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.
corporatefinancial-news
References:
- 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 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 November
2021.
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