Celgene, Acceleron Offer New Results from Study with Luspatercept in Beta-Thalassemia at ASH

Celgene Corporation

and Acceleron Pharma Inc.

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.