Luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia
SUMMIT, NJ, USA & CAMBRIDGE, MA, USA I December 7, 2015 I Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced preliminary results from two Phase 2 clinical trials of luspatercept in patients with beta-thalassemia were presented at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition. Results highlighted in an oral presentation showed that luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia. Celgene and Acceleron are jointly developing luspatercept.
“These luspatercept results are very exciting as they show positive effects across a range of clinically challenging complications of beta-thalassemia,” said Professor Antonio Piga, M.D., Ph.D., Director of Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and coordinating principal investigator of the study. “A therapy that could potentially treat the anemia, complications of beta-thalassemia, such as iron overload, and improve measures of health-related quality of life would be a huge advance to help address the substantial unmet need of patients with beta-thalassemia.”
Luspatercept Data Presented at ASH
Luspatercept was evaluated in a Phase 2, multicenter, open-label study in adults with non-transfusion-dependent (NTD) and transfusion-dependent (TD) beta-thalassemia patients. The primary objectives were to assess the proportion of NTD patients that achieved a hemoglobin increase ≥ 1.5 g/dL from baseline and the proportion of TD patients that achieved at least a 20% reduction in transfusion burden. A total of 64 patients, of which 59 were efficacy evaluable (31 NTD and 28 TD), were enrolled in the dose escalation and expansion stages of the Phase 2 clinical trial. In this study, patients received up to 5 doses via subcutaneous injection once every 3 weeks. 51 of the 64 patients from this 3-month study enrolled in the long-term Phase 2 extension trial in which these patients may receive up to two years of treatment with luspatercept. Data was presented from both the 3-month study and the long-term extension study.
Improvement of anemia and transfusion burden:
- Of the 17 NTD patients that received at least 5 cycles of luspatercept at dose levels of 0.8 mg/kg or higher
- 65% (11/17) increased hemoglobin levels ≥ 1.0 g/dL over a 12-week period
- 47% (8/17) increased hemoglobin levels ≥ 1.5 g/dL over a 12-week period
- Data presented showed sustained increases in hemoglobin with the longest-treated patients having received nearly six months of luspatercept
- Of the 28 TD patients
- 79% (22/28) patients had ≥ 20% reduction of transfusion burden
- 75% (21/28) patients had ≥ 33% reduction of transfusion burden
- 57% (16/28) patients had ≥ 50% reduction of transfusion burden
Changes in iron overload:
- Liver iron concentration (LIC), a measure of iron overload, was maintained or reduced in both non-transfusion dependent and transfusion-dependent patients
- 50% (4/8) TD patients with baseline LIC ≥ 5 mg/g dry weight (dw) had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) TD patients with baseline LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
- 36% (5/14) NTD patients with baseline LIC ≥ 5 mg/g dw had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) NTD patients with baseline LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
Improvement in health-related quality of life (QoL) measures in NTD patients:
- Improved health-related QoL (FACT-An anemia subscore) correlated with increases in hemoglobin in NTD Patients
Safety:
- The most common related adverse events were bone pain, myalgia, headache, arthralgia, musculoskeletal pain, asthenia, injection site pain, back pain and pain in jaw
- Related grade 3 adverse events included headache, bone pain, asthenia, and myalgia
- There were no drug-related serious adverse events
Celgene and Acceleron are in the process of initiating a global Phase 3 study in regularly transfused beta-thalassemia patients.
Luspatercept is an investigational product that is not approved for any use in any country.
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. For more information, please visit www.clinicaltrials.gov.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair. The company is a leader in understanding the biology of the Transforming Growth Factor-Beta (TGF-β) protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines. Acceleron has built a highly productive R&D platform that has generated innovative clinical and preclinical therapeutic candidates with novel mechanisms of action. These therapeutic candidates have the potential to significantly improve clinical outcomes for patients with cancer and rare diseases.
For more information, please visit www.acceleronpharma.com.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company’s website at www.celgene.com. Follow us on Twitter @Celgene as well.
SOURCE: Celgene
Post Views: 97
Luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia
SUMMIT, NJ, USA & CAMBRIDGE, MA, USA I December 7, 2015 I Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced preliminary results from two Phase 2 clinical trials of luspatercept in patients with beta-thalassemia were presented at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition. Results highlighted in an oral presentation showed that luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia. Celgene and Acceleron are jointly developing luspatercept.
“These luspatercept results are very exciting as they show positive effects across a range of clinically challenging complications of beta-thalassemia,” said Professor Antonio Piga, M.D., Ph.D., Director of Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and coordinating principal investigator of the study. “A therapy that could potentially treat the anemia, complications of beta-thalassemia, such as iron overload, and improve measures of health-related quality of life would be a huge advance to help address the substantial unmet need of patients with beta-thalassemia.”
Luspatercept Data Presented at ASH
Luspatercept was evaluated in a Phase 2, multicenter, open-label study in adults with non-transfusion-dependent (NTD) and transfusion-dependent (TD) beta-thalassemia patients. The primary objectives were to assess the proportion of NTD patients that achieved a hemoglobin increase ≥ 1.5 g/dL from baseline and the proportion of TD patients that achieved at least a 20% reduction in transfusion burden. A total of 64 patients, of which 59 were efficacy evaluable (31 NTD and 28 TD), were enrolled in the dose escalation and expansion stages of the Phase 2 clinical trial. In this study, patients received up to 5 doses via subcutaneous injection once every 3 weeks. 51 of the 64 patients from this 3-month study enrolled in the long-term Phase 2 extension trial in which these patients may receive up to two years of treatment with luspatercept. Data was presented from both the 3-month study and the long-term extension study.
Improvement of anemia and transfusion burden:
- Of the 17 NTD patients that received at least 5 cycles of luspatercept at dose levels of 0.8 mg/kg or higher
- 65% (11/17) increased hemoglobin levels ≥ 1.0 g/dL over a 12-week period
- 47% (8/17) increased hemoglobin levels ≥ 1.5 g/dL over a 12-week period
- Data presented showed sustained increases in hemoglobin with the longest-treated patients having received nearly six months of luspatercept
- Of the 28 TD patients
- 79% (22/28) patients had ≥ 20% reduction of transfusion burden
- 75% (21/28) patients had ≥ 33% reduction of transfusion burden
- 57% (16/28) patients had ≥ 50% reduction of transfusion burden
Changes in iron overload:
- Liver iron concentration (LIC), a measure of iron overload, was maintained or reduced in both non-transfusion dependent and transfusion-dependent patients
- 50% (4/8) TD patients with baseline LIC ≥ 5 mg/g dry weight (dw) had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) TD patients with baseline LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
- 36% (5/14) NTD patients with baseline LIC ≥ 5 mg/g dw had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) NTD patients with baseline LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
Improvement in health-related quality of life (QoL) measures in NTD patients:
- Improved health-related QoL (FACT-An anemia subscore) correlated with increases in hemoglobin in NTD Patients
Safety:
- The most common related adverse events were bone pain, myalgia, headache, arthralgia, musculoskeletal pain, asthenia, injection site pain, back pain and pain in jaw
- Related grade 3 adverse events included headache, bone pain, asthenia, and myalgia
- There were no drug-related serious adverse events
Celgene and Acceleron are in the process of initiating a global Phase 3 study in regularly transfused beta-thalassemia patients.
Luspatercept is an investigational product that is not approved for any use in any country.
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. For more information, please visit www.clinicaltrials.gov.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair. The company is a leader in understanding the biology of the Transforming Growth Factor-Beta (TGF-β) protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines. Acceleron has built a highly productive R&D platform that has generated innovative clinical and preclinical therapeutic candidates with novel mechanisms of action. These therapeutic candidates have the potential to significantly improve clinical outcomes for patients with cancer and rare diseases.
For more information, please visit www.acceleronpharma.com.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company’s website at www.celgene.com. Follow us on Twitter @Celgene as well.
SOURCE: Celgene
Post Views: 97
