Celgene Corporation and Acceleron Pharma Announce Results of the Phase 3 BELIEVE Trial Evaluating Luspatercept in Adult Patients with Beta-Thalassemia at ASH 2018
Pivotal phase 3 data demonstrated treatment with investigational luspatercept resulted in significant reduction of transfusion burden compared to placebo
Regulatory submissions planned in
“Currently, the standard of care to help patients with beta-thalassemia manage their anemia is regular, lifelong red blood cell transfusions, which over time can result in iron overload and life-threatening co-morbidities,” said Professor Cappellini, M.D., Professor of Medicine,
BELIEVE met the primary endpoint of erythroid response, defined as a ≥33% reduction in
RBC Transfusion Burden Reduction of ≥ 33% Response Rates1
|Response Time Interval||Luspatercept||Placebo||P-value|
|Weeks 13-24||21.4% (48/224)||4.5% (5/112)||< 0.0001|
|Weeks 37-48||19.6% (44/224)||3.6% (4/112)||< 0.0001|
|Any 12 weeks during the entire treatment period||70.5% (158/224)||29.5% (33/112)||< 0.0001|
|Any 24 weeks during the entire treatment period||41.1% (92/224)||2.7% (3/112)||< 0.0001|
RBC Transfusion Burden Reduction of ≥ 50% Response Rates1
|Response Time Interval||Luspatercept||Placebo||P-value|
|Weeks 13-24||7.6% (17/224)||1.8% (2/112)||0.0303|
|Weeks 37-48||10.3% (23/224)||0.9% (1/112)||0.0017|
|Any 12 weeks during the entire treatment period||40.2% (90/224)||6.3% (7/112)||< 0.0001|
|Any 24 weeks during the entire treatment period||16.5% (37/224)||0.9% (1/112)||< 0.0001|
BELIEVE Safety Summary (Safety Population)
Grade 3 or higher treatment-emergent adverse events (TEAEs) were reported in 29.1% (65/223) of patients receiving luspatercept and 15.6% (17/109) of patients receiving placebo. Serious adverse events were reported in 15.2% (34/223) of patients receiving luspatercept and 5.5% (6/109) of patients receiving placebo. A TEAE of acute cholecystitis resulted in death in one placebo-treated patient (0.9%). No luspatercept-treated patients died due to TEAEs.
Grade 3 or 4 TEAEs in at least 1% of patients in either arm
|Increased liver iron concentration||2.7%||0.9%|
|Blood uric acid increased||1.3%||0.0%|
|Increased aspartate aminotransferase||1.3%||0.0%|
|Increase alanine aminotransferase||0.9%||2.8%|
*All grades of thromboembolic events, including DVT, PE, portal vein thrombosis, ischemic stroke, thrombophlebitis, and superficial phlebitis were reported in 8 of 223 (3.6%) luspatercept-treated versus 1 of 109 (0.9%) placebo-treated patients
“The BELIEVE results demonstrate the potential of luspatercept to help adults living with beta-thalassemia better manage their anemia and reduce their transfusion burden,” said
“These outcomes of the BELIEVE trial increase our confidence in the potential of luspatercept to become an important new treatment option for patients suffering from beta-thalassemia,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Our focus now is to work diligently with health authorities to help ensure that this underserved patient population can gain access to luspatercept as quickly as possible.”
Luspatercept is not approved in any region for any indication. The companies are planning regulatory application submissions of luspatercept in
BELIEVE is a phase 3, randomized, double blind, placebo-controlled multicenter study comparing luspatercept + best supportive care (BSC) versus placebo + BSC in adult beta-thalassemia patients who require regular
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 3 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 COMMANDS phase 3 trial in first-line, 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.
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 phase 2 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,” “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
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