- Interim longer term data with investigational drug luspatercept show sustained increases in hemoglobin levels, and reduced transfusion burden in patients with beta-thalassemia -
"Beta-thalassemia is a severe, chronic disease with no pharmaceutical
treatment options to correct or improve the underlying anemia in
Luspatercept Beta-Thalassemia Data Presented at ASH
Results in Transfusion Dependent (TD) Beta-Thalassemia Patients
RBC transfusion reduction
|Response rate (% of patients)|
3-month base study
Long-term extension study
|≥ 20%||81% (25/31)||96% (23/24)|
|≥ 33%||71% (22/31)||83% (20/24)|
|≥ 50%||55% (17/31)||71% (17/24)|
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia Patients
Hemoglobin (Hb) response over
Response rate (% of patients)
in patients treated with ≥ 0.6 mg/kg
3-month base study
Long-term extension study
|Increase in mean Hb ≥ 1.0 g/dL||62% (14/21)||78% (21/27)|
|Increase in mean Hb ≥ 1.5 g/dL||33% (7/21)||52% (14/27)|
In the long-term extension study, the median duration of a hemoglobin increase ≥ 1.0 g/dL maintained for at least 12 weeks in responders is 13.5 months (N=21) with treatment still ongoing.
Luspatercept is an investigational product that is not approved for use in any country.
The BELIEVE Trial, a global Phase 3, double-blind, randomized, placebo-controlled, multicenter study in transfusion dependent beta-thalassemia patients, is currently enrolling.
The slides from the ASH beta-thalassemia presentation will be available immediately following the presentation at the conference on Acceleron's website (www.acceleronpharma.com) under the Science tab.
About the Phase 2 Study
Data from two open-label Phase 2 studies were presented at the conference: the base study in which patients received treatment with luspatercept for three months and the ongoing long-term safety extension study in which patients may receive treatment with luspatercept for up to an additional five years. In both the three-month base study and the long-term extension study, red blood cell (RBC) transfusion dependent patients (≥ 4 units RBC / 8 weeks) and non-transfusion dependent patients ( < 4 units RBC / 8 weeks) were enrolled and treated with open-label luspatercept, dosed subcutaneously once every three weeks.
The primary outcome measure of the three-month base study was the proportion of patients who have an erythroid response, defined as 1) a hemoglobin increase of ≥ 1.5 g/dL from baseline for ≥ 14 days (in the absence of RBC transfusions) in non-transfusion dependent patients, or 2) ≥ 20% reduction in RBC transfusion burden compared to pretreatment in transfusion dependent patients. The primary outcome for the long-term extension study is to evaluate the long-term safety and tolerability of 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-beta) 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
Acceleron is a clinical stage biopharmaceutical company focused on the
discovery, development and commercialization of innovative therapeutics
to treat serious and rare diseases. Its pioneering research platform
leverages the powerful biology behind the body's ability to rebuild and
repair its own cells and tissues. This approach to drug discovery has
generated four therapeutic candidates that are currently in clinical
trials. The Company's lead therapeutic candidate, luspatercept, is being
evaluated in Phase 3 studies for the treatment of the hematologic
diseases, myelodysplastic syndromes (MDS) and beta-thalassemia under a
global partnership with
For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.
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heading "Risk Factors" included in Acceleron's Annual Report on Form
10-K which was filed with the
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