- Longer term data with investigational drug luspatercept show sustained increases in hemoglobin levels, reduced transfusion burden, and improved patient reported quality of life measures
in patients with beta-thalassemia -
- Acceleron to host conference call and live webcast today at
"The results from the 3-month luspatercept clinical trial were very
promising and the data from longer-term treatment are even more
encouraging," said Professor
Highlights of the Luspatercept Beta-Thalassemia Data Presented at EHA
Data from two Phase 2 studies will be presented at the conference: the completed dose-escalation study in which patients received treatment with luspatercept for three months and the ongoing long-term extension study in which patients receive treatment with luspatercept for up to an additional 24 months. In both the 3-month 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 3 weeks. The primary outcome measure of the 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 measure of the long-term extension was the safety and tolerability assessed by adverse events.
Results in Transfusion Dependent (TD) Beta-Thalassemia Patients
RBC transfusion reduction
|Response rate (% of patients)|
3-month base study
Long-term extension study
|≥ 20%||80% (24/30)||96% (23/24)|
|≥ 33%||67% (20/30)||83% (20/24)|
|≥ 50%||53% (16/30)||67% (16/24)|
Durability of Response:
In the long-term extension study, the duration of the reduction in transfusion burden of ≥ 33% ranged from 12 to 48+ weeks.
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||64% (14/22)||78% (21/27)|
|Increase in mean Hb ≥ 1.5 g/dL||36% (8/22)||56% (15/27)|
Durability of Response:
In the long-term extension study, the duration of hemoglobin increase (≥ 1.0 g/dL) ranged from 113 to 505+ days.
Improvement in patient-reported quality of life (QoL) measures in NTD patients:
Safety Results in TD and NTD Patients
Luspatercept is an investigational product that is not approved for use in any country.
The BELIEVE Trial, a global Phase 3 study in regularly transfused beta-thalassemia patients, is currently enrolling.
The slides from the EHA beta-thalassemia presentations will be available immediately following the presentations at the conference on Acceleron's website (www.acceleronpharma.com) under the Science tab.
Acceleron EHA Conference Call Information
Acceleron will host a conference call and live webcast from EHA today at
To access the live webcast, please select "Events & Presentations" in the Investor section on Acceleron's website (www.acceleronpharma.com) at least 10 minutes beforehand to ensure time for any downloads that may be required.
An archived webcast recording will be available on the Acceleron website beginning approximately two hours after the event.
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 discovers and develops novel therapies to treat a wide range
of 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 innovative approach to drug discovery has
generated four therapeutic candidates currently in clinical trials.
Acceleron'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
For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development of luspatercept, the timeline for clinical development and regulatory approval of Acceleron's compounds, the expected timing for the reporting of data from ongoing trials, and the structure of Acceleron's planned or pending clinical trials. The words "anticipate," "appear," "believe," "continue," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Each forward-looking statement is subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statement. Applicable risks and
uncertainties include the risks that preclinical testing of Acceleron's
compounds and data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that data may
not be available when Acceleron expects it to be, that Acceleron or its
Other risks and uncertainties include those identified under the
heading "Risk Factors" included in Acceleron's Annual Report on Form
10-K which was filed with the
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.
Senior Director, Investor Relations and Corporate Communications
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