On April 27, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported updates on the luspatercept clinical programs under its collaboration with Celgene (Press release, Acceleron Pharma, APR 27, 2017, View Source [SID1234518699]). Luspatercept is an investigational compound being evaluated in two pivotal Phase 3 trials for the treatment of myelodysplastic syndromes (MDS) and beta-thalassemia.

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"Our pivotal luspatercept studies continue to gain momentum as enrollment nears completion, and new clinical data support the drug’s expanded development in patient groups in need of more treatment options," commented Habib Dable, President and Chief Executive Officer of Acceleron. "We now look forward to reporting topline results from the MEDALIST and BELIEVE Phase 3 studies in mid-2018 as we work together with Celgene to evaluate the full clinical potential of luspatercept."

Updated Phase 3 Clinical Trials Enrollment Guidance

Acceleron and Celgene provided updated guidance on the anticipated completion of patient enrollment for the MEDALIST and BELIEVE Phase 3 studies of luspatercept in MDS and beta-thalassemia, respectively. Based on accelerating rates of patient recruitment, the companies expect to achieve full enrollment of both studies in the second quarter of 2017, versus previous guidance of the second half of 2017.

New Phase 3 Clinical Trial

Acceleron and Celgene also plan to initiate a third Phase 3 trial of luspatercept. The new Phase 3 trial, expected to be initiated in early 2018, will evaluate luspatercept treatment versus standard-of-care in the first-line treatment setting for MDS patients. Current first-line, standard-of-care treatment for lower-risk MDS patients includes erythropoiesis-stimulating agents (ESAs) and/or regular red blood cell transfusions. The ongoing MEDALIST Phase 3 trial is evaluating luspatercept in ESA-refractory or -ineligible MDS patients.

Luspatercept Data at the Upcoming MDS Symposium

Acceleron and Celgene plan to report updated Phase 2 data on luspatercept in first-line, lower-risk MDS patients in an oral presentation at the 14th International Symposium on MDS on Saturday, May 6, 2017, in Valencia, Spain.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-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 erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Acceleron and Celgene are enrolling Phase 3 clinical trials that are designed 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.

Luspatercept is an investigational product that is not approved for use in any country.