Celgene Corporation and Acceleron Pharma Announce Submission of Luspatercept Biologics License Application to U.S. FDA
BLA submission includes both myelodysplastic syndromes and beta-thalassemia indications
EMA marketing applicationfor both indications planned for Q2:19
The submission is based on the safety and efficacy results of the pivotal phase 3 studies MEDALIST and BELIEVE, both recently presented at the
“There remains a high unmet medical need for patients with MDS or beta-thalassemia who suffer from the effects of their disease-related anemia. The primary treatment option for these patients currently is chronic transfusion of red blood cells which can be associated with complications such as iron overload,” said Jay Backstrom, M.D., Chief Medical Officer for
The companies also plan to submit a marketing application to the
“The BLA submission is a key milestone for Acceleron and a credit to our longstanding collaboration with Celgene,” said
Luspatercept is an investigational therapy that is not approved for any use in any country for any indication.
Luspatercept is a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell maturation. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. A phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing. For more information, please visit www.clinicaltrials.gov.
MEDALIST is a phase 3, randomized, double blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept in adults with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS). All patients were
BELIEVE is a phase 3, randomized, double blind, placebo-controlled multicenter study comparing luspatercept + best supportive care (BSC) versus placebo + BSC in adults with beta-thalassemia patients who require regular
Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.
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 and Celgene; the potential of luspatercept as a therapeutic drug; and the benefit of each company’s strategic plans and focus. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,” “would,” “could,” “potential,” “possible,” “hope” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs. For example, there can be no guarantee that luspatercept will be successfully developed or complete necessary clinical phases. Forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including: results of clinical trials, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; the ability to obtain and maintain requisite regulatory approvals and to enroll patients in planned clinical trials; the ability to obtain, maintain and enforce patent and other intellectual property protection for luspatercept; the ability to maintain key collaborations; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in each company’s public filings with the
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Acceleron Pharma Inc.
Todd James, IRC, (617) 649-9393
Vice President, Investor Relations and Corporate Communications
Matt Fearer, (617) 301-9557
Director, Corporate Communications