Pivotal phase 3 data demonstrated treatment
with investigational luspatercept resulted in significant reduction
of transfusion burden compared to placebo
Regulatory submissions planned in the United
States and Europe in the first half of 2019
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced results from a pivotal, phase 3
trial (BELIEVE) evaluating the safety and efficacy of luspatercept
for the treatment of adults with beta-thalassemia-associated anemia
who require regular red blood cell (RBC) transfusions. The data
were presented by Maria Domenica Cappellini, M.D. in an oral
session of the 60th American Society of Hematology (ASH) Annual
Meeting and Exposition in San Diego, CA (Abstract #163).
“Currently, the standard of care to help patients with
beta-thalassemia manage their anemia is regular, lifelong red blood
cell transfusions, which over time can result in iron overload and
life-threatening co-morbidities,” said Professor Cappellini, M.D.,
Professor of Medicine, University of Milan - Fondazione IRCCS.
“These findings from the BELIEVE study are exciting because they
suggest that luspatercept may help patients reduce their dependence
on red blood cell transfusions.”
BELIEVE met the primary endpoint of erythroid response, defined
as a ≥33% reduction in RBC transfusion burden (with a reduction of
≥ 2 units of RBC) during weeks 13–24 compared to the baseline
12-week interval prior to randomization. The study also included
secondary endpoints that evaluated the impact of treatment on RBC
transfusion burden. Mean change in transfusion burden from baseline
to weeks 13-24 (luspatercept vs. placebo) was -1.35 RBC units.
RBC Transfusion Burden Reduction of ≥ 33% Response
Rates1
Response Time Interval Luspatercept
Placebo P-value Weeks
13-24 21.4% (48/224) 4.5% (5/112)
< 0.0001 Weeks 37-48 19.6% (44/224)
3.6% (4/112) < 0.0001 Any 12 weeks
during the entire treatment period 70.5% (158/224)
29.5% (33/112) < 0.0001 Any 24 weeks
during the entire treatment period 41.1% (92/224)
2.7% (3/112) < 0.0001
RBC Transfusion Burden Reduction of ≥ 50% Response
Rates1
Response Time Interval Luspatercept
Placebo P-value Weeks
13-24 7.6% (17/224) 1.8% (2/112)
0.0303 Weeks 37-48 10.3% (23/224)
0.9% (1/112) 0.0017 Any 12 weeks during the
entire treatment period 40.2% (90/224)
6.3% (7/112) < 0.0001 Any 24 weeks during the
entire treatment period 16.5% (37/224)
0.9% (1/112) < 0.0001
1RBC transfusion burden reduction response rates are calculated
versus baseline (i.e., the 12 weeks prior to randomization)
BELIEVE Safety Summary (Safety
Population)
Grade 3 or higher treatment-emergent adverse events (TEAEs) were
reported in 29.1% (65/223) of patients receiving luspatercept and
15.6% (17/109) of patients receiving placebo. Serious adverse
events were reported in 15.2% (34/223) of patients receiving
luspatercept and 5.5% (6/109) of patients receiving placebo. A TEAE
of acute cholecystitis resulted in death in one placebo-treated
patient (0.9%). No luspatercept-treated patients died due to
TEAEs.
Grade 3 or 4 TEAEs in at least 1% of
patients in either arm
Luspatercept
N= 223
Placebo
N= 109
Anemia 3.1% 0.0% Increased liver iron
concentration 2.7% 0.9% Hyperuricemia
2.7% 0.0% Hypertension
1.8% 0.0% Syncope 1.8%
0.0% Back pain 1.3% 0.9% Bone pain
1.3% 0.0% Blood uric acid increased
1.3% 0.0% Increased aspartate
aminotransferase 1.3% 0.0% Increase
alanine aminotransferase 0.9% 2.8%
Thromboembolic events* 0.9% 0.0%
*All grades of thromboembolic events, including DVT, PE, portal
vein thrombosis, ischemic stroke, thrombophlebitis, and superficial
phlebitis were reported in 8 of 223 (3.6%) luspatercept-treated
versus 1 of 109 (0.9%) placebo-treated patients
“The BELIEVE results demonstrate the potential of luspatercept
to help adults living with beta-thalassemia better manage their
anemia and reduce their transfusion burden,” said Alise Reicin,
M.D., President, Global Clinical Development for Celgene. “These
results further our understanding of the luspatercept clinical
profile, which will continue to inform our plans to advance this
promising investigational therapy.”
“These outcomes of the BELIEVE trial increase our confidence in
the potential of luspatercept to become an important new treatment
option for patients suffering from beta-thalassemia,”
said Habib Dable, President and Chief Executive Officer of
Acceleron. “Our focus now is to work diligently with health
authorities to help ensure that this underserved patient population
can gain access to luspatercept as quickly as possible.”
Luspatercept is not approved in any region for any indication.
The companies are planning regulatory application submissions of
luspatercept in the United States and Europe in the first half of
2019.
About BELIEVE
BELIEVE is a phase 3, randomized, double blind,
placebo-controlled multicenter study comparing luspatercept + best
supportive care (BSC) versus placebo + BSC in adult
beta-thalassemia patients who require regular RBC transfusions. The
median age of the patients was 30 years in both treatment arms. 336
patients were randomized 2:1 to receive either luspatercept 1.0
mg/kg + BSC (224 patients) or placebo + BSC (112 patients) every 3
weeks for up to 48 weeks. Patients in the luspatercept + BSC arm
were able to be titrated up to 1.25 mg/kg of luspatercept every 3
weeks. BSC was defined as RBC transfusions and iron chelation
therapy to maintain each patient’s baseline hemoglobin level.
Crossover to the luspatercept treatment group was allowed after
unblinding and assessment by an independent Data Safety Monitoring
committee; patients receiving luspatercept + BSC will be followed
for up to 3 years. The study was conducted at 65 sites in 15
countries.
About Luspatercept
Luspatercept is a first-in-class erythroid maturation agent
(EMA) that is believed to regulate late-stage red blood cell
maturation. Acceleron and Celgene are jointly developing
luspatercept as part of a global collaboration. Phase 3 clinical
trials continue to evaluate the safety and efficacy of luspatercept
in patients with MDS (the MEDALIST trial) and in patients with
beta-thalassemia (the BELIEVE trial). A COMMANDS phase 3 trial in
first-line, lower-risk, MDS patients, the BEYOND phase 2 trial in
non-transfusion-dependent beta-thalassemia, and a phase 2 trial in
myelofibrosis are ongoing. For more information, please
visit www.clinicaltrials.gov.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global biopharmaceutical company engaged primarily in
the discovery, development and commercialization of innovative
therapies for the treatment of cancer and inflammatory diseases
through next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com.
Follow Celgene on Social Media: Twitter, Pinterest, LinkedIn,
Facebook and YouTube.
About Acceleron
Acceleron is a Cambridge-based, clinical-stage
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
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,
the Company and its global collaboration partner, Celgene, are
developing luspatercept for the treatment of chronic anemia in
myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.
Acceleron is also advancing its neuromuscular franchise with two
distinct Myostatin+ agents, ACE-083 and ACE-2494, and 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
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. Such forward-looking statements include those regarding the
potential benefits of, and plans relating to the collaboration
between Acceleron and Celgene; the potential of luspatercept
as a therapeutic drug; and the benefit of each company’s strategic
plans and focus. The words “anticipate,” “believe,” “estimate,”
“expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,”
“would,” “could,” “potential,” “possible,” “hope” and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Such statements are subject to numerous
important factors, risks and uncertainties that may cause actual
events or results to differ materially from current expectations
and beliefs. For example, there can be no guarantee that
luspatercept will be successfully developed or complete necessary
clinical phases. Forward-looking statements in this press release
could also be affected by risks and uncertainties relating to a
number of other important factors, including: results of clinical
trials, including subsequent analysis of existing data and new data
received from ongoing and future studies; the content and timing of
decisions made by the U.S. FDA and other regulatory
authorities, investigational review boards at clinical trial sites
and publication review bodies; the ability to obtain and maintain
requisite regulatory approvals and to enroll patients in planned
clinical trials; the ability to obtain, maintain and enforce patent
and other intellectual property protection for luspatercept; the
ability to maintain key collaborations; and general economic and
market conditions. These and other risks are described in greater
detail under the caption "Risk Factors" included in each company’s
public filings with the Securities and Exchange Commission.
Any forward-looking statements contained in this press release
speak only as of the date hereof, and neither company has any
obligation to update any forward-looking statements, whether as a
result of new information, future events or otherwise, except as
may be required by law.
Hyperlinks are provided as a convenience and for informational
purposes only. Neither Celgene nor Acceleron bears
responsibility for the security or content of external websites or
websites outside of their respective control.
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version on businesswire.com: https://www.businesswire.com/news/home/20181201005028/en/
Celgene
CorporationInvestors:+1-908-673-9628ir@celgene.comorMedia:+1-908-673-2275media@celgene.com
Acceleron Pharma Inc.Todd James, IRC, (617) 649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, (617) 649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:Matt Fearer, (617) 301-9557Director,
Corporate Communications
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