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Celgene and Acceleron Announce Luspatercept Achieved Primary and Key Secondary Endpoints in Phase III 'MEDALIST' Study in Patients with Low-to-Intermediate Risk Myelodysplastic Syndromes


Results showed significant improvement in red blood cell transfusion
independence compared to placebo

Safety profile generally consistent with previously published data

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 phase III, randomized,
double-blind, multi-center clinical study (MEDALIST). Luspatercept
achieved a highly statistically significant improvement in the primary
endpoint of red blood cell (RBC) transfusion independence of at least 8
consecutive weeks during the first 24 weeks compared to placebo.

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MEDALIST evaluated the efficacy and safety of luspatercept versus
placebo in patients with IPSS-R very low, low or intermediate risk
myelodysplastic syndromes (MDS) with chronic anemia and refractory to,
intolerant of, or ineligible for treatment with an
erythropoietin-stimulating agent (ESA), ring sideroblast-positive and
require frequent RBC transfusions.

In addition to achieving the primary endpoint of the study, luspatercept
also met the key secondary endpoint of demonstrating a highly
statistically significant improvement in RBC transfusion independence of
at least 12 consecutive weeks during the first 24 weeks. Modified
hematologic improvement-erythroid (IWG mHI-E), a meaningful secondary
endpoint, was also achieved.

Adverse events observed in the study were generally consistent with
previously published data.

"This result from the phase III MEDALIST trial demonstrates the
potential clinical benefit of luspatercept as an erythroid maturation
agent for the treatment of chronic anemia in patients with
low-to-intermediate risk MDS," said Jay Backstrom, M.D., Chief Medical
Officer for Celgene. "Based on these results, we look forward to
preparing the dossier for global regulatory submissions and also
investigating the clinical potential of luspatercept in ESA-naïve,
low-to-intermediate risk MDS patients through the initiation of our
phase III COMMANDS study."

"We are truly encouraged by the top-line results of MEDALIST and the
potential to benefit the tens of thousands of patients suffering from
low-to-intermediate risk MDS worldwide. We would like to thank the
patients and investigators involved in the trial," said Habib Dable,
President and Chief Executive Officer of Acceleron. "With other ongoing
research in beta-thalassemia and myelofibrosis, we remain committed to
exploring the potential of luspatercept to address a range of
anemia-related diseases."

Data from MEDALIST will be submitted to a future medical meeting in
2018. The companies plan to submit regulatory applications in the United
States and Europe in the first half of 2019.

Luspatercept is not approved for any indication in any geography.

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 III 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 Phase III
trial is being planned in first-line, lower-risk, MDS patients (the
COMMANDS trial). The BEYOND Phase II trial in non-transfusion-dependent
beta-thalassemia and a Phase II trial in myelofibrosis are ongoing. For
more information, please visit

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
Follow Celgene on Social Media: @CelgenePinterestLinkedInFacebook 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 pulmonary program with sotatercept in pulmonary arterial

For more information, please visit
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," "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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