Celgene Corporation ו-Acceleron
Pharma Inc. הודיעו היום על תוצאות של מחקר קליני שלב 3, אקראי, עם סמיות
כפולה רב-מרכזי (BELIEVE). Luspatercept
השיגה שיפור עם מובהקות סטטיסטית גבוהה בנקודה הסופית הראשונית בתגובת כדוריות הדם
האדומות, שהוגדרה כהפחתה של לפחות 33 אחוז מקו הבסיס בנטל העירוי של כדוריות
אדומות עם הפחתה של לפחות שתי יחידות בתקופה שהוגדרה בפרוטוקול של 12 שבועות
עוקבים, משבוע 13 עד שבוע 24, בהשוואה לפלצבו.
BELIEVE
העריך את היעילות והבטיחות של luspatercept בתוספת הטיפול התומך הטוב ביותר לעומת פלצבו
בתוספת הטיפול התומך הטוב ביותר במבוגרים עם תלסמיה בטא שתלויה בעירויים.
Celgene and Acceleron Announce Luspatercept Achieved Primary and All Key Secondary Endpoints in Phase III ‘BELIEVE’ Study in Adults with Transfusion-Dependent Beta-Thalassemia
Results
showed a significant reduction in transfusion burden compared to placebo
Safety
profile generally consistent with previously reported data
Regulatory
submissions planned in the United States and Europe in the first half of 2019
SUMMIT, N.J. & CAMBRIDGE, Mass., 9 July 2018, (BUSINESS WIRE):
Celgene
Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today
announced results from a phase III, randomized, double-blind, multi-center
clinical study (BELIEVE). Luspatercept achieved a highly statistically
significant improvement in the primary endpoint of erythroid response, which
was defined as at least a 33 percent reduction from baseline in red blood cell
(RBC) transfusion burden with a reduction of at least 2 units during the
protocol-defined period of 12 consecutive weeks, from week 13 to week 24,
compared to placebo.
BELIEVE
evaluated the efficacy and safety of luspatercept plus best supportive care
versus placebo plus best supportive care in adults with transfusion-dependent
beta-thalassemia.
In
addition to achieving the primary endpoint of the study, luspatercept also met
all key secondary endpoints of demonstrating statistically significant
improvements in RBC transfusion burden from baseline of at least a 33 percent
reduction during the period from week 37 to week 48, at least a 50 percent
reduction during the period from week 13 to week 24, at least a 50 percent
reduction during the period from week 37 to week 48, and a mean change in
transfusion burden from week 13 to week 24.
Adverse
events observed in the study were generally consistent with previously reported
data.
“For
decades, the management of beta-thalassemia in adults has been limited to
transfusions and iron chelation. Reduction of transfusion burden represents an
important step forward for patients with this rare and debilitating blood
disease,” said Jay Backstrom, M.D., Chief Medical Officer for Celgene. “We
thank the patients, as well as their families and physicians, for their
participation in the BELIEVE study.”
“The
BELIEVE study marks the second positive phase III study for luspatercept and
underscores the potential of this erythroid maturation agent to impact a range
of diseases associated with chronic anemia,” said Habib Dable, President and
Chief Executive Officer of Acceleron. “We continue to explore luspatercept
across our broader development programs, including non-transfusion dependent
beta-thalassemia in the ongoing BEYOND study.”
The
companies also recently announced that luspatercept met the primary and key
secondary endpoints in the MEDALIST study, a phase III, randomized,
double-blind, multi-center clinical trial evaluating 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.
Data
from BELIEVE and MEDALIST will be submitted to a future medical meeting in
2018. The companies plan to submit regulatory applications for luspatercept 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.
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
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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