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 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: @Celgene, 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 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,” “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.comorMedia:+1-908-673-2275media@celgene.comorAcceleron
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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