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Sotatercept shows promise for anemia in patients with beta-thalassemia
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NEW ORLEANS — Sotatercept was associated with a dose-dependent hemoglobin response when used to treat anemia in patients with beta-thalassemia, according to results of an interim analysis presented at the ASH Annual Meeting and Exposition.
Researchers evaluated different doses of sotatercept (ACE-011, Celgene Corp and Acceleron Pharma Inc.) — a first-in-class activin type IIA receptor fusion protein — in patients with transfusion-dependent beta-thalassemia major, or transfusion-dependent or nontransfusion-dependent beta-thalassemia intermedia.
The interim analysis includes data from 25 patients enrolled in the dose escalation phase. Patients were assigned 0.1 mg/kg (n=8), 0.3 mg/kg (n=9) or 0.5 mg/kg (n=8) doses of sotatercept administered subcutaneously every 3 weeks.
Nineteen patients were still undergoing treatment at the cutoff date. Three patients discontinued treatment due to adverse events; of them, two were in the 0.1-mg/kg cohort and one was in the 0.5-mg/kg cohort. Three patients discontinued treatment due to a lack of efficacy; of them, two were in the 0.1-mg/kg cohort, and one was in the 0.3-mg/kg cohort.
The percentage of patients who achieved ≥1 g/dL increase of hemglobin was 0% in the 0.1-mg/kg cohort, 84% in the 0.3-mg/kg cohort and 84% in the 0.5-mg/kg cohort. The percentage of patients who achieved a ≥2 g/dL increase was 16% in the 0.3-mg/kg cohort and 33% in the 0.5-mg/kg cohort.
Treatment with sotatercept was not associated with a reduction in transfusion burden for transfusion-dependent patients in the 0.1-mg/kg and 0.3-mg/kg cohorts. Longer treatment of patients in the 0.5-mg/kg cohort is necessary to determine any change in transfusion burden of these patients, researchers said.
Assessment of pharmacokinetics parameters indicated that the area under the curve and peak concentration increased proportionally with dosing.
The researchers said sotatercept treatment generally was well tolerated. One patient in the 0.1-mg/kg cohort experienced grade ≥2 bone pain, and another patient in this cohort experienced grade ≥2 superficial thrombophlebitis. One patient in the 0.5-mg/kg cohort experienced grade ≥2 ventricular extrasystoles.
“Based on these preliminary data, sotatercept may be a safe and beneficial option for the treatment of anemia in patients with beta-thalassemia,” the researchers concluded.
For more information:
Cappellini MD. Abstract #3448. Presented at: ASH Annual Meeting and Exposition; Dec. 7-10, 2013; New Orleans.
Disclosure: The researchers report speakers’ bureau, employment and consultant roles with, research funding and honoraria from, and equity ownership in Acceleron Pharma, Celgene, Genzyme, Novartis and Shire.