Results showed a significant reduction in transfusion burden compared to placebo
Safety profile generally consistent with previously reported data
Regulatory submissions planned in
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
"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,"
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
Luspatercept is not approved for any indication in any geography.
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.
Acceleron is a
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.
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
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