--- Longitudinal analyses from the BELIEVE trial show reductions in red blood cell transfusions and iron chelation therapy in luspatercept-treated patients -
--- Longer-term update from Phase 2 trial finds durable response to luspatercept treatment in patients with myelofibrosis-associated anemia -
Acceleron Pharma Inc. (Nasdaq: XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, announced today that data from six clinical abstracts on REBLOZYL (luspatercept-aamt) will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition held virtually December 5-8, 2020.
The ASH presentations include updates from the Phase 3 placebo-controlled MEDALIST and BELIEVE trials assessing the safety and efficacy of luspatercept treatment in patients with lower-risk myelodysplastic syndromes (MDS) and in patients with transfusion-dependent beta-thalassemia, respectively. Among the results to be presented are improvements in certain health-related quality of life measures for luspatercept-treated patients in both trials as well as longitudinal trends of red blood cell transfusion reductions and reductions of iron chelation therapy in BELIEVE trial participants.
Title: Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the BELIEVE Trial Oral Presentation Presenter: Maria Domenica Cappellini, MD Session 904. Outcomes Research--Non-Malignant Conditions Title: Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Study Moderated Digital Poster Presentation Presenter: Esther Natalie Oliva, MD Session 903. Health Services Research--Malignant Conditions (Myeloid Disease) Title: Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy in Adult Patients with Beta-Thalassemia in the BELIEVE Trial Moderated Digital Poster Presentation Presenter: Olivier Hermine, MD, PhD Session 112. Thalassemia and Globin Gene Regulation Title: Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in Beta-Thalassemia with Luspatercept: Longitudinal Results of the BELIEVE Trial Moderated Digital Poster Presentation Presenter: Ali T. Taher, MD, PhD Session 112. Thalassemia and Globin Gene Regulation
Title: Efficacy and Safety of Luspatercept Treatment in Patients with Myelodysplastic Syndrome/ Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis (MDS/MPN-RS-T): A Retrospective Analysis from the MEDALIST Study Moderated Digital Poster Presentation Presenter: Rami S. Komrokji, MD Session 637. Myelodysplastic Syndromes--Clinical Studies Title: Duration of Response to Luspatercept in Patients Requiring Red Blood Cell (RBC) Transfusions with Myelofibrosis (MF) - Updated Data from the Phase 2 ACE-536-MF-001 Study Moderated Digital Poster Presentation Presenter: Aaron T. Gerds, MD Session 634. Myeloproliferative Syndromes: Clinical
The clinical abstracts can be found on the ASH Annual Meeting website (http://www.hematology.org/Annual-Meeting/Abstracts/).
The REBLOZYL ASH presentations, which will include additional information beyond the abstracts, will be posted to the "Publications" page under the "Science & Pipeline" section in the "Science" section on the Company's website, www.acceleronpharma.com, following the conference.
About REBLOZYL(R) (luspatercept-aamt)
REBLOZYL (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing REBLOZYL as part of a global collaboration. REBLOZYL is currently approved in the U.S. for the treatment of:
anemia in adult patients with beta-thalassemia who require regular red blood cell transfusions, and anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
In Europe, REBLOZYL is approved for the treatment of:
Adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy. Adult patients with transfusion-dependent anemia associated with beta-thalassemia.
In Canada, REBLOZYL is approved for the treatment of adult patients with red blood cell (RBC) transfusion-dependent anemia associated with beta-thalassemia.
REBLOZYL is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, REBLOZYL(R) (luspatercept-aamt) is the first and only erythroid maturation agent approved in the United States, Europe, and Canada for the treatment of anemia in certain blood disorders. REBLOZYL is part of a global collaboration partnership with Bristol Myers Squibb. The Companies co-promote REBLOZYL in North America and are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension (PAH), having recently presented positive topline results of the PULSAR Phase 2 trial. The Company is currently planning multiple Phase 3 trials with the potential to support its long-term vision of establishing sotatercept as a backbone therapy for patients with PAH at all stages of the disease.
For more information, please visit acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.
This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, the expected timing for reporting of data from ongoing clinical trials, and the potential of Reblozyl(R) (luspatercept-aamt) as a therapeutic drug. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "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 factors, risks and uncertainties, including, but not limited to, that the results of any clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds will take longer and/or cost more than planned, that Acceleron or its collaboration partner, Bristol Myers Squibb Company will be unable to successfully complete the clinical development of Acceleron's compounds, that Acceleron or Bristol Myers Squibb may be delayed in initiating, enrolling or completing any clinical trials, and that Acceleron'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 Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future.
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 Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.
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SOURCE: Acceleron Pharma Inc.
Investors: Jamie Bernard, IRC, 617-649-9650 Associate Director, Investor Relations Media: Matt Fearer, 617-301-9557 Director, Corporate Communications