Data from MEDALIST showed significant
clinical benefit of Reblozyl in treating anemia in adults with
myelodysplastic syndromes
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 MEDALIST, the pivotal
phase 3 study evaluating the use of Reblozyl® (luspatercept-aamt)
to treat anemia in patients with very low- to intermediate-risk
myelodysplastic syndromes (MDS) who have ring sideroblasts (RS+)
and require red blood cell transfusions, and who had failed, were
intolerant to, or ineligible for/unlikely to respond to treatment
with erythropoiesis-stimulating agents (ESAs).1
“This NEJM publication recognizes the importance and robustness
of the MEDALIST study, as well as the impact and potential clinical
benefit of Reblozyl in MDS patients who have ring sideroblasts,”
said Diane McDowell, M.D., Vice President, Hematology Global
Medical Affairs, Bristol-Myers Squibb. “As the first erythroid
maturation agent, Reblozyl holds the potential to help these
patients address the underlying cause of their disease-related
chronic anemia.”
MEDALIST achieved a statistically significant improvement in the
primary endpoint of red blood cell transfusion independence
(RBC-TI) for 8 or more weeks during the first 24 weeks of the
study. The study also met the key secondary endpoint of RBC-TI for
12 or more weeks during the first 24 or 48 weeks of the study, as
well as the additional secondary endpoint of hematologic
improvement-erythroid (HI-E) for 8 or more weeks as assessed by
International Working Group 2006 criteria.1
The most common treatment-emergent adverse events (TEAEs) of any
grade in greater than 10% of patients in either the treatment or
placebo arm were fatigue, diarrhea, asthenia, nausea, dizziness,
and back pain. TEAEs of Grade 3 or 4 were reported in 42.5%
(65/153) of patients receiving luspatercept-aamt and 44.7% (34/76)
of patients receiving placebo. Progression to acute myeloid
leukemia (AML) occurred in three patients (2.0%) receiving
luspatercept-aamt and one patient (1.3%) receiving placebo. Five
patients receiving luspatercept-aamt (3.3%) and four patients
receiving placebo (5.3%) experienced one or more TEAE that resulted
in death.1
Results from MEDALIST were initially presented during the
Plenary Scientific Session at the 60th American Society of
Hematology (ASH) Annual Meeting and Exposition in December 2018.
Longer-term analyses from MEDALIST were recently presented at the
61st ASH Annual Meeting in December 2019.
“It is truly an honor to see work that began in Acceleron
laboratories more than a decade ago and advanced successfully
through a collaboration with Bristol-Myers Squibb now highlighted
in the New England Journal of Medicine,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “Publication of
the MEDALIST trial results in a journal of such eminence will
certainly help to raise awareness among physicians of a potential
new therapeutic option for treating anemia in certain patients with
MDS.”
The U.S. Food and Drug Administration (FDA) is evaluating
Reblozyl for the treatment of anemia in adults with very low- to
intermediate-risk MDS who have ring sideroblasts and require red
blood cell transfusions, and has set a Prescription Drug User Fee
Act (PDUFA), or target action, date of April 4, 2020 for this
indication. 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.
In November 2019, Reblozyl was granted approval by the FDA to
treat 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.
Reblozyl is not approved to treat MDS in any geography.
About MEDALIST MEDALIST is a
phase 3, randomized, double blind, placebo-controlled, multi-center
study evaluating the safety and efficacy of luspatercept in
patients with IPSS-R-defined very low-, low-, or intermediate-risk
non-del(5q) myelodysplastic syndromes (MDS). All patients were red
blood cell transfusion-dependent and were either refractory or
intolerant to prior ESA therapy, or were ESA naïve and unlikely to
respond due to endogenous serum erythropoietin levels of ≥ 200 U/L,
and had no prior treatment with disease modifying agents.
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.2 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.
Additional Clinical Investigation A
phase 2 trial (BEYOND) in adult patients with
non-transfusion-dependent beta thalassemia3; a phase 2 trial in
pediatric patients with transfusion-dependent beta thalassemia4; a
phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients5;
and a phase 2 trial in myelofibrosis patients are ongoing.6 The
companies are also planning a phase 3 trial known as INDEPENDENCE
in myelofibrosis in 2020. For more information, please visit
www.clinicaltrials.gov.
Reblozyl has not been approved as safe and effective for use in
patients with MDS or myelofibrosis in any country.
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 3-4 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
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. The Company'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, neuromuscular, 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 also advancing its
neuromuscular program with ACE-083, a locally-acting Myostatin+
agent in Phase 2 development in Charcot-Marie-Tooth disease and is
conducting a Phase 2 pulmonary program with sotatercept in
pulmonary arterial hypertension.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social Media: @AcceleronPharma and
LinkedIn.
Forward-Looking Statements
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 preclinical
testing of Acceleron’s compounds and data from clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials, 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.
1 Fenaux P. Luspatercept in Patients with Lower-Risk
Myelodysplastic Syndromes. New England Journal of Medicine. 2
REBLOZYL U.S. Prescribing Information. Accessed November 2019. 3
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 October 2019. 4 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 December 2019. 5 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 October 2019. 6 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 October 2019.
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Bristol-Myers Squibb Company Media Inquiries:
media@bms.com 609-252-3345
Investors: Tim Power 609-252-7509
timothy.power@bms.com
Nina Goworek 908-673-9711 ngoworek@celgene.com
Acceleron Pharma Inc. Investors: Todd James, IRC,
(617) 649-9393 Vice President, Investor Relations and Corporate
Communications
Ed Joyce, (617) 649-9242 Director, Investor Relations
Media: Matt Fearer, (617) 301-9557 Director, Corporate
Communications
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