Pivotal phase 3 data demonstrated treatment
with investigational luspatercept resulted in statistically
significant increased red blood cell transfusion independence
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 the pivotal, phase 3
MEDALIST trial evaluating the efficacy and safety of
investigational luspatercept to treat patients with ring
sideroblast (RS+) myelodysplastic syndromes (MDS)-associated anemia
who require red blood cell transfusions and who had failed, were
intolerant to, or ineligible for erythropoietin therapy. Results
were presented by Alan F. List, M.D. during the Plenary Scientific
Session at the 60th American Society of Hematology (ASH) Annual
Meeting and Exposition in San Diego, C.A. (Abstract #1).
“Severe anemia resulting in red blood cell transfusion
dependence is a significant challenge for patients with low- and
intermediate-risk MDS. Those who become resistant or refractory to
currently available treatments have limited alternatives,” said Dr.
List, President and CEO of Moffitt Cancer Center. “The findings
from MEDALIST are very exciting as they support the hypothesis that
targeting red blood cell precursor maturation could help to address
patients’ anemia and allow them to achieve transfusion
independence.”
MEDALIST met the primary endpoint of red blood cell transfusion
independence (RBC-TI) for 8 or more weeks during the first 24 weeks
of the study. Treatment with luspatercept resulted in a
statistically significantly greater proportion of patients
achieving RBC-TI ≥ 8 weeks compared to placebo. The study also
found in secondary endpoints that treatment with luspatercept
resulted in a statistically significant higher percentage of
patients achieving RBC-TI of 12 or more weeks in the first 24 or 48
weeks of the study, as well as hematologic improvement-erythroid
(HI-E) of 8 or more weeks.
Endpoints Luspatercept
Placebo P-Value RBC-TI ≥8 weeks (weeks
1-24) 37.9 % (58/153) 13.2 % (10/76) <
0.0001 RBC-TI ≥12 weeks (weeks 1-24) 28.1 % (43/153)
7.9 % (6/76) 0.0002 RBC-TI ≥12 weeks (weeks 1-48)
33.3 % (51/153) 11.8 % (9/76) 0.0003 HI-E ≥ 8 weeks
(IWG 2006, weeks 1-24) 52.9 % (81/153) 11.8 % (9/76)
< 0.0001
MEDALIST Safety Summary
Treatment-emergent adverse events (TEAEs) of Grade 3 or 4 were
reported in 42.5% (65/153) of patients receiving luspatercept and
44.7% (34/76) of patients receiving placebo. Progression to acute
myeloid leukemia (AML) occurred in four patients, three patients
(2.0%) receiving luspatercept and one patient (1.3%) receiving
placebo. Five patients receiving luspatercept (3.3%) and four
patients receiving placebo (5.3%) experienced one or more TEAE that
resulted in death.
Most common TEAEs of any Grade in
Greater than 10% of Patients in Either Arm
Luspatercept
N=153
Placebo
N=76
Fatigue 26.8 % 13.2 % Diarrhea 22.2 %
9.2 % Asthenia 20.3 % 11.8 % Nausea 20.3 %
7.9 % Dizziness 19.6 % 5.3 % Back pain
19.0 % 6.6 %
“The MEDALIST results demonstrate the potential clinical benefit
of luspatercept in achieving red blood cell transfusion
independence in patients with low-to-intermediate risk RS+ MDS, an
area in need of new treatments,” said Alise Reicin, MD, President,
Global Clinical Development for Celgene. “Based on these results,
we are encouraged that this first-in-class erythroid maturation
agent may help these patients address the underlying cause of their
disease-related chronic anemia.”
“It’s truly an honor to showcase the results from the
MEDALIST trial as the first presentation of the ASH Plenary
Session,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “The results from the MEDALIST
trial increase our confidence in the potential of
luspatercept to provide a meaningful treatment option for patients
suffering from lower-risk RS+ MDS worldwide. We’re excited to
continue our clinical development program in MDS, beta-thalassemia,
and myelofibrosis, while also exploring additional applications for
luspatercept in a range of diseases associated with anemia.”
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 MEDALIST
MEDALIST is a phase 3, randomized, double blind,
placebo-controlled, multi-center study evaluating the safety and
efficacy of luspatercept in patients with very low-, low-, or
intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All
patients were RBC transfusion dependent and were either refractory
or intolerant to prior erythropoiesis-stimulating agent (ESA)
therapy, or were ESA naïve with endogenous serum erythropoietin ≥
200 U/L, and had no prior treatment with disease modifying agents.
The median age of the patients enrolled in the trial was 71 years
in the luspatercept treatment group and 72 years in the placebo
group. Median transfusion burden in both treatment arms was 5 RBC
units/8 weeks. 229 patients were randomized to receive either
luspatercept 1.0 mg/kg (153 patients) or placebo (76 patients) via
subcutaneous injection once every 21 days. The study was conducted
at 65 sites in 11 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). The 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 any
product candidate will be successfully developed or complete
necessary preclinical and clinical phases, that the results of any
clinical study will be predictive for other clinical studies of the
same product candidate, or that development of any of product
candidates will successfully continue. There can be no guarantee
that any positive developments will result in stock price
appreciation. Management's expectations and, therefore, any
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 and
preclinical studies, 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; unplanned cash requirements and
expenditures; competitive factors; the ability to obtain, maintain
and enforce patent and other intellectual property protection for
any product candidates ; 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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Celgene
CorporationInvestors:+1-908-673-9628ir@celgene.com
Media:+1-908-673-2275media@celgene.com
Acceleron Pharma Inc.Todd James, IRC, (617) 649-9393Vice
President, Investor Relations and Corporate Communications
Candice Ellis, (617) 649-9226Manager, Investor Relations and
Corporate Communications
Media:Matt Fearer, (617) 301-9557Director, Corporate
Communications
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