- Preliminary results show that treatment with investigational drug luspatercept increases hemoglobin and achieves durable transfusion independence in patients with lower risk myelodysplastic syndromes -
- Preliminary data in
- Acceleron to host conference call and live webcast on
"We are encouraged by the additional luspatercept data from the ongoing
Phase 2 studies," said
Luspatercept Phase 2 Data in First-line,
In lower-risk MDS patients who have not received prior treatment with an erythropoiesis-stimulating agent (ESA) and have erythropoietin (EPO) levels ≤ 500 IU, luspatercept three-month base study data demonstrated encouraging rates of transfusion independence and International Working Group Hematologic Improvement - Erythroid (IWG HI-E) response criteria.
|Transfusion Burden||IWG HI-E, n/N (%)||RBC-TI, n/N (%)|
|Base N=64||Extension N=42||Base N=49||Extension N=28|
|All||12/20 (60%)||13/16 (81%)||9/12 (75%)||8/10 (80%)|
|Low Transfusion Burden||6/13 (46%)||8/11 (73%)||5/5 (100%)||5/5 (100%)|
|High Transfusion Burden||6/7 (86%)||5/5 (100%)||4/7 (57%)||3/5 (60%)|
Luspatercept Phase 2 Data in Ring Sideroblast Positive (RS+) and Negative (RS-) in MDS Patients
|IWG HI-E, n/N (%)||RBC-TI, n/N (%)|
|< 200||RS+||18/29 (62%)||19/23 (83%)||13/19 (68%)||10/14 (71%)|
|RS-||2/5 (40%)||3/3 (100%)||1/4 (25%)||1/2 (50%)|
|≥ 200 to ≤ 500||RS+||5/11 (46%)||7/8 (88%)||3/9 (33%)||3/5 (60%)|
|RS-||0/3 (0%)||0/1 (0%)||2/2 (100%)||1/1 (100%)|
*Table includes both
Luspatercept Phase 2 Safety Data
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 poster presentation of the ongoing Phase 2 studies is available on Acceleron's website (www.acceleronpharma.com) under the Science tab.
Acceleron ASH Conference Call Information
Acceleron will host a conference call and live webcast to discuss data
presented at the ASH meeting on
To access the live webcast, please select "Events & Presentations" in the Investors 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.
About the MDS Phase 2 Studies
Data from two Phase 2 studies were presented at the conference: the base study in which patients received treatment with luspatercept for three months and the long-term 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, 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 three weeks.
The primary outcome measure for the three-month base 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 low-transfusion burden patients and for high-transfusion burden patients a reduction of either ≥ 4 units or ≥ 50% of units of RBCs transfused while receiving luspatercept compared to the patient's pretreatment transfusion burden. 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
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995. Such
forward-looking statements include those regarding the potential
benefits of, and plans relating to the collaboration between Acceleron
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
Senior Director, Investor Relations and Corporate Communications
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