"The efficacy and safety results in this Phase 2 study support continued
research into luspatercept for the treatment of refractory anemia which
often requires red blood cell transfusions in lower-risk MDS patients,"
"We are pleased that The Lancet Oncology chose to publish these
The article, entitled "Luspatercept for the treatment of anaemia in
patients with lower-risk myelodysplastic syndromes: a phase 2
dose-finding study with long-term extension study" is now available
online and will be published in a future print issue of The
Phase 2 presentations of luspatercept in MDS presented at recent medical conferences include updated longer-term follow-up and new expansion cohort preliminary results beyond that incorporated in this publication. Presentations outlining these results are available online under the science page on the Company's website at www.acceleronpharma.com.
The MEDALIST trial, a global Phase 3 study of luspatercept in lower-risk
MDS patients, is fully enrolled and top-line results are expected in
mid-2018. The MEDALIST trial enrolled patients who are ring
sideroblast-positive, red blood cell transfusion dependent, and are
erythropoiesis-stimulating agent (ESA)-refractory or
Luspatercept is an investigational product that is not approved for use in any country.
Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of 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 erythropoiesis stimulating agents (ESAs), which stimulate the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Phase 3 clinical trials are underway to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the MEDALIST study) and in patients with beta-thalassemia (the BELIEVE study). For more information, please visit www.clinicaltrials.gov.
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. 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 Celgene. Acceleron is also advancing its ACE-083 clinical program in the field of neuromuscular disease, and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about the Company's strategy, future plans and prospects, including statements regarding the development of the Company's compounds, the timeline for clinical development and regulatory approval of the Company's compounds and the expected timing for reporting of data from ongoing clinical trials. The words "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "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.
Actual results could differ materially from those included in the
forward-looking statements due to various risks and uncertainties,
including, but not limited to, that preclinical testing of the Company's
compounds and data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that the
development of the Company's compounds will take longer and/or cost more
than planned, that the Company or its collaboration partner, Celgene,
will be unable to successfully complete the clinical development of the
Company's compounds, that the Company or Celgene may be delayed in
initiating, enrolling or completing any clinical trials, and that the
Company's compounds will not receive regulatory approval or become
commercially successful products. These and other risks and
uncertainties are identified under the heading "Risk Factors" included
in the Company's most recent Annual Report on Form 10-K, and other
filings that the Company has made and may make with the
The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions and projections with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.
Vice President, Investor Relations and Corporate Communications
Manager, Investor Relations and Corporate Communications
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