Acceleron and Celgene Announce Preliminary Results from an Investigator Initiated Phase 2 Study of Sotatercept in Myelofibrosis at the 58th Annual Meeting of the American Society of Hematology
- Preliminary results show that treatment with investigational drug sotatercept can increase hemoglobin and achieve transfusion independence in patients with myelofibrosis -
- Acceleron to host conference call and live webcast on
"These initial and encouraging data support our efforts for further
clinical development work with our Acceleron-partnered programs in
Highlights of the Sotatercept Myelofibrosis Phase 2 Data Presented at ASH
- 19 patients were enrolled and treated with sotatercept (12 patients at 0.75 mg/kg and 7 at 1 mg/kg) and 14 of these patients have received at least 5 doses of sotatercept and are evaluable for response
- 36% (5/14) of the evaluable patients achieved an anemia response, defined as a composite of RBC-transfusion-independence and hemoglobin response (increase of ≥1.5 g/dL from baseline on every determination consecutively over ≥84 days without RBC transfusions).
- Most adverse events were grades 1 or 2.
- Adverse events at least possibly related to study drug that occurred include grade 3 hypertension leading to discontinuation, grade 1 myalgia, bone pain, pain in extremity and injection site reaction.
- 13 patients have discontinued from the study: 5 due to no response, 2 proceeded to stem cell transplantation, 2 had MF progression, 1 transformed to AML, 1 each withdrew consent, had unrelated medical problems and hypertension
Sotatercept is an investigational product that is not approved for use in any country.
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 Phase 2 Study
Data were presented from an ongoing investigator sponsored Phase 2 study of sotatercept in subjects with myelofibrosis at the conference. Subjects enrolled were red blood cell (RBC) transfusion-dependent, have hemoglobin < 10 g/dL on every determination during the 84 days preceding study entry without RBC transfusions, or have hemoglobin < 10 g/dL despite intermittent RBC transfusions without fulfilling the criteria for transfusion dependence. Patients received open-label sotatercept at 0.75 mg/kg or 1 mg/kg, dosed subcutaneously once every three weeks.
The primary outcome measures for the study include anemia response and safety. Anemia response is a composite of RBC-transfusion-independence and hemoglobin response (increase of ≥1.5 g/dL from baseline on every determination consecutively over ≥84 days without RBC transfusions). Subjects must have received ≥5 cycles of sotatercept to be evaluable for response.
Sotatercept is an activin receptor type IIA fusion protein that acts as
a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β)
superfamily involved in fibrosis and late stage erythropoiesis (red
blood cell production). 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 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 sotatercept, 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
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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