11/06/2019 | Press release | Distributed by Public on 11/06/2019 08:31
- Updated results from MEDALIST Phase 3 trial show 47.1% of patients with anemia associated with myelodysplastic syndromes treated with luspatercept achieved red blood cell transfusion independence for ≥ 8 weeks and median total duration of clinical benefit was 83.6 weeks for patients responding to luspatercept as of a January 2019 data cutoff date -
- In the BELIEVE Phase 3 trial, beta-thalassemia patients who were luspatercept responders had durable clinical benefit over the 64.1-week follow-up period -
- Interim results from the ongoing Phase 2 trial of luspatercept in patients with anemia associated with myelofibrosis suggest clinically meaningful activity, with 32% to 53% of transfusion-dependent patients achieving responses when treated in combination with ruxolitinib -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 6, 2019-- Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that data from six clinical abstracts on luspatercept will be presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Florida, on December 7-10, 2019. Luspatercept is being developed as part of a global collaboration between Acceleron and Celgene Corporation.
'It's a privilege to return to the world's largest hematology meeting to share updated results from the Phase 3 trials of luspatercept in patients with myelodysplastic syndromes and beta-thalassemia,' said Habib Dable, President and Chief Executive Officer of Acceleron. 'With the presentations of both trials having garnered 'Best of ASH' honors in 2018, we're thrilled to report that luspatercept has continued to demonstrate durable clinical benefit in both patient populations in the ongoing open-label studies. Moreover, interim Phase 2 data show clinically significant activity from luspatercept treatment in patients with myelofibrosis-associated anemia, further validating our confidence in luspatercept's potential as a therapy for a variety of chronic anemias of high unmet need.'
Oral Presentation, Monday, December 9, 4:30 p.m. EST
Session 637. Myelodysplastic Syndromes - Clinical Studies: Lower-Risk MDS and CMML
Title: Assessment of Longer-Term Efficacy and Safety in the Phase 3, Randomized, Double-Blind, Placebo-Controlled MEDALIST Trial of Luspatercept to Treat Anemia in Patients (Pts) with Revised International Prognostic Scoring System (IPSS-R) Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions
Presenter: Pierre Fenaux
Clinical benefit, defined as a patient achieving red blood cell transfusion independence (RBC-TI) ≥ 8 weeks and/or modified hematologic improvement erythroid (HI-E) response per International Working Group 2006 criteria, was assessed, along with total duration of clinical benefit (time from achieving clinical benefit to discontinuation due to loss of benefit, adverse events [AEs], or other reasons).
Summary: Data in the abstract include MEDALIST Phase 3 trial results as of the January 7, 2019 cutoff date.
Poster Presentation, Monday, December 9, 6:00 p.m. EST
Session 112. Thalassemia and Globin Gene Regulation: Poster III
Title: Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions
Presenter: Vip Viprakasit
Achievement and number of response episodes (defined as ≥ 33% reduction in RBC transfusion from baseline over any consecutive 24 weeks) were assessed at a median follow-up of 64.1 weeks. Duration of clinical benefit, defined as the time of first response (≥ 33% reduction in RBC transfusion over any 24 weeks) to discontinuation due to any cause at that episode, was also assessed.
Data in the abstract include results as of the May 11, 2018 cutoff date.
Oral Presentation,Monday, December 9, 7:00 a.m. EST
Session 634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted Therapies
Title: A Phase 2 Study of Luspatercept in Patients with Myelofibrosis-Associated Anemia
Presenter: Aaron T Gerds
Patients with myelofibrosis (MF) receiving a stable dose of ruxolitinib in combination with luspatercept treatment:
Patients with MF receiving luspatercept without concomitant treatment with ruxolitinib:
Four of 74 (5%) patients had grade 3-4 treatment-related adverse events (AEs). Treatment-related AEs occurring in ≥ 3% of patients were hypertension (11%), bone pain (8%), and diarrhea (4%). Seven (9%) patients had ≥ 1 AE resulting in treatment discontinuation.
Data in the abstract include interim results of the ongoing open-label, phase 2 trial evaluating luspatercept in patients with MF and anemia as of the data cutoff date of May 10, 2019.
Other Luspatercept Abstracts
Poster Presentation, Sunday, December 8, 6:00 - 8:00 p.m. EST
Session 637. Myelodysplastic Syndromes - Clinical Studies: Poster II
Title: Luspatercept Significantly Reduces Red Blood Cell (RBC) Transfusion Burden, Regardless of Gene Mutation Frequency, Spectrum, and Prognostic Significance, Among Patients (Pts) with LR-MDS Enrolled in the MEDALIST Trial
Presenter: Uwe Platzbecker
Poster Presentation, Monday, December 9, 6:00 - 8:00 p.m. EST
Session 637. Myelodysplastic Syndromes - Clinical Studies: Poster III
Title: Hematologic Improvement-Neutrophil and -Platelet in the MEDALIST Trial: Multilineage Data from a Phase 3, Randomized, Double-Blind, Placebo- Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions
Presenter: Guillermo Garcia-Manero
Poster Presentation, Sunday, December 8, 6:00 - 8:00 p.m. EST
Session 112: Thalassemia and Globin Regulation: Poster II
Title: Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial
Presenter: John B Porter
The clinical abstracts that contain additional trial details and results can be found on the ASH Annual Meeting website (http://www.hematology.org/Annual-Meeting/Abstracts/).
The luspatercept ASH presentations, which will include additional information beyond the abstracts, will be available in the 'Science' section on Acceleron's website, www.acceleronpharma.com, following the conference.
Luspatercept is an investigational therapy that is not approved for any use in any country.
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 Charcot-Marie-Tooth disease, and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.
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Source: Acceleron Pharma Inc.
Acceleron Pharma Inc.
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