- Preliminary results show that treatment with investigational drug luspatercept achieves meaningful erythroid response rates and transfusion independence in first-line, lower-risk myelodysplastic syndromes patients -
- Acceleron to review MDS Symposium presentation in conjunction with
its first quarter earnings call on
"These positive data presented in lower-risk MDS confirm our optimism in
new opportunities for luspatercept beyond our ongoing Phase 3 trials,"
"There is a high unmet medical need for a drug to treat patients earlier
in the MDS treatment paradigm," said
Luspatercept Phase 2 Data in First-Line, Lower-Risk MDS Patients
In lower-risk, erythropoiesis-stimulating agent (ESA)-naïve MDS
patients, 48% (11/23) of patients treated with luspatercept achieved red
blood cell transfusion independence (RBC-TI) and 51% (20/39) of patients
achieved a clinically meaningful erythroid hematological improvement
(HI-E) response per the International Working Group's (IWG) criteria.
The response rates were positive in patients treated with luspatercept
IWG HI-E, N=82
|ESA-Naïve||20/39 (51%)||11/23 (48%)|
||22/43 (51%)||11/33 (33%)|
Luspatercept Phase 2 Data in RS+ and RS- Lower-Risk MDS Patients
In patients with baseline erythropoietin (EPO) levels ≤ 500 international units per liter (IU/L), RBC-TI and IWG HI-E response rates were positive in both ring sideroblast-positive (RS+) and -negative (RS-) patients.
IWG HI-E, N=82
|≤ 500||RS+||30/46 (65%)||16/29 (55%)|
|RS-||6/14 (43%)||4/7 (57%)|
|> 500||RS+||5/9 (56%)||2/9 (22%)|
|RS-||1/11 (9%)||0/9 (0%)|
|Unknown||0/2 (0%)||0/2 (0%)|
*Table includes both
Luspatercept Phase 2 Safety Data
The majority of adverse events (AEs) were grade 1 or 2. AEs at least possibly related to study drug that occurred in at least 3 patients during the studies were fatigue, headache, hypertension, diarrhea, arthralgia, bone pain, injection site erythema, myalgia, and edema peripheral. Grade 3 non-serious AEs possibly or probably related to study drug were ascites, blast cell count increase, blood bilirubin increase, hypertension, platelet count increase, and pleural effusion. Grade 3 serious AEs possibly or probably related to study drug were general physical health deterioration and myalgia.
Luspatercept is an investigational product that is not approved for use in any country.
The oral presentation given at the 14th International Symposium on MDS is available on Acceleron's website (www.acceleronpharma.com) under the Science tab.
Acceleron MDS Symposium Conference Call Information
Acceleron will host a conference call and live webcast to discuss data
presented at the MDS Symposium and its first quarter operational and
financial results on
To access the live webcast, please select "Events & Presentations" in the Investors/Media 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, lower-risk MDS patients were enrolled and treated with open-label luspatercept, dosed subcutaneously once every three weeks.
The outcome measures for the studies included the proportion of patients who had an erythroid response (IWG HI-E) or achieved RBC transfusion independence (RBC-TI). IWG HI-E was defined as hemoglobin increase ≥ 1.5 g/dL sustained for ≥ 8 weeks in patients with < 4 units RBC / 8 weeks transfusion burden at baseline and hemoglobin levels below 10 g/dL. For patients with a ≥ 4 units RBC / 8 weeks transfusion burden at baseline, erythroid response was defined as a reduction of ≥ 4 units RBC sustained for ≥ 8 weeks. RBC-TI was defined as no RBC transfusions for ≥ 8 weeks in patients with a ≥ 2 units RBC / 8 weeks baseline transfusion burden.
Luspatercept is a modified activin receptor type IIB fusion protein that
acts as a ligand trap for members in the transforming growth factor-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 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
Senior Director, Investor Relations and Corporate Communications
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