BLA submission includes both myelodysplastic
syndromes and beta-thalassemia indications
EMA marketing application for both indications
planned for Q2:19
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced that Celgene has submitted a
Biologics License Application (BLA) for luspatercept, an erythroid
maturation agent, for the treatment of adult patients with very low
to intermediate risk myelodysplastic syndromes (MDS)-associated
anemia who have ring sideroblasts and require red blood cell (RBC)
transfusions and for the treatment of adult patients with
beta-thalassemia-associated anemia who require RBC
transfusions.
The submission is based on the safety and efficacy results of
the pivotal phase 3 studies MEDALIST and BELIEVE, both recently
presented at the American Society of Hematology annual meeting,
where MEDALIST was included in the plenary session.
“There remains a high unmet medical need for patients with MDS
or beta-thalassemia who suffer from the effects of their
disease-related anemia. The primary treatment option for these
patients currently is chronic transfusion of red blood cells which
can be associated with complications such as iron overload,”
said Jay Backstrom, M.D., Chief Medical Officer for Celgene.
“New treatment options are urgently needed for these patients. With
this submission, we look forward to working with the Agency to
deliver luspatercept to patients with these serious blood
diseases.”
The companies also plan to submit a marketing application to the
European Medicines Agency in the second quarter of 2019.
“The BLA submission is a key milestone for Acceleron and a
credit to our longstanding collaboration with Celgene,” said Habib
Dable, President and Chief Executive Officer of Acceleron. “We
believe luspatercept’s positive clinical trial results demonstrate
its potential as a novel treatment for patients with lower-risk MDS
as well as in beta-thalassemia. All involved have worked diligently
to develop luspatercept for patients with chronic anemias
associated with these serious blood disorders.”
Luspatercept is an investigational therapy that is not approved
for any use in any country for any indication.
About Luspatercept
Luspatercept is a first-in-class erythroid maturation agent
(EMA) that regulates late-stage red blood cell maturation.
Acceleron and Celgene are jointly developing luspatercept
as part of a global collaboration. A phase 3 trial (COMMANDS) in
ESA-naïve, 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 MEDALIST
MEDALIST is a phase 3, randomized, double blind,
placebo-controlled, multi-center study evaluating the safety and
efficacy of luspatercept in adults with very low-, low-, or
intermediate-risk 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) by subcutaneous
injection once every 21 days. The study was conducted at 65 sites
in 11 countries.
About BELIEVE
BELIEVE is a phase 3, randomized, double blind,
placebo-controlled multicenter study comparing luspatercept + best
supportive care (BSC) versus placebo + BSC in adults with
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 to receive either luspatercept 1.0 mg/kg
(224 patients) or placebo (112 patients) by subcutaneous injection
every 21 days for up to 48 weeks. Crossover to the luspatercept
treatment groups was allowed after unblinding based on the
recommendation of an independent Data Safety Monitoring Committee;
patients treated with luspatercept will be followed for up to 3
years. The study was conducted at 65 sites in 15 countries.
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 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 program with ACE-083,
a locally-acting Myostatin+ agent in Phase 2 development in
facioscapulohumeral muscular dystrophy and 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
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 and
with respect to Celgene includes risk factors related to the
proposed transaction between Bristol-Myers Squibb and Celgene, such
as, but not limited to, the risks that: management’s time and
attention is diverted on transaction related issues; disruption
from the transaction makes it more difficult to maintain business,
contractual and operational relationships; and Bristol-Myers
Squibb, Celgene or the combined company is unable to retain key
personnel. 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.comAcceleron
Pharma Inc.Investors:Todd James,
IRC, (617) 649-9393Vice President, Investor Relations and Corporate
CommunicationsorMedia:Matt Fearer, (617) 301-9557Director,
Corporate Communications
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