Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 21st Congress of the European Hematology Association
- Preliminary results show that treatment with investigational drug luspatercept results in clinically meaningful increases in hemoglobin and durable transfusion independence in patients with lower risk myelodysplastic syndromes -
- Acceleron to host conference call and live webcast today at
"The results for luspatercept in lower risk MDS patients are
increasingly encouraging as we gain longer term safety and efficacy
experience with this agent," said
Highlights of the Luspatercept MDS Phase 2 Data Presented at EHA
Data from two Phase 2 studies were 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 an additional 24 months. In both the 3-month base study and the long-term extension study, high transfusion burden patients (≥ 4 units RBC / 8 weeks) and low transfusion burden patients ( < 4 units RBC / 8 weeks) were enrolled and treated with open-label luspatercept, dosed subcutaneously once every 3 weeks. The primary outcome measure for the 3 month study was the proportion of patients who had an erythroid response. Erythroid response was defined as hemoglobin ≥ 1.5 g/dL from baseline for ≥ 14 days in non-transfusion dependent patients or a reduction of either ≥ 4 units or ≥ 50% of units of RBCs transfused 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 with low or intermediate-1 risk MDS who were previously enrolled in the 3-month study.
|Response rate (% of patients)|
3-month base study
International Working Group Hematologic
|51% (25/49)||81% (26/32)|
RBC Transfusion Independence (RBC-TI)
|35% (14/40)||50% (11/22)|
|Duration of RBC-TI||Range: 9-80+ weeks|
For reference, results presented six months ago at the
- IWG HI-E response rate was 69% (22/32)
- RBC-TI response rate was 50% (11/22)
- Duration of RBC-TI ranged from 9 to 50+ weeks
- There were three grade 3 adverse events possibly/probably related to study drug (blast cell count increase, myalgia and worsening of general condition).
- Adverse events at least possibly related to study drug that occurred in at least 2 patients during studies were fatigue, bone pain, diarrhea, myalgia, headache, hypertension and injection site erythema.
Luspatercept is an investigational product that is not approved for use in any country.
The MEDALIST Trial, a global Phase 3 study in patients with very low, low, or intermediate risk, MDS with ring sideroblasts who require red blood cell transfusions, is currently enrolling.
The slides from this oral presentation are available 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
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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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