On September 5, 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 that The Lancet Oncology has published results from the Phase 2 study of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) (Press release, Acceleron Pharma, SEP 5, 2017, View Source [SID1234520369]).
“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,” said Uwe Platzbecker, M.D., Professor of Hematology and Head of the MDS program at the University Hospital in Dresden, Germany. “This novel investigational therapy has the potential to address a significant need in MDS patients who currently have very limited options for managing chronic anemia. We have already begun exploring luspatercept’s activity across a range of MDS patient populations.”
“We are pleased that The Lancet Oncology chose to publish these results,” said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. “The positive clinical activity demonstrated in this study informed our earlier decision to initiate the pivotal MEDALIST Phase 3 trial in lower-risk MDS, and we expect to report top-line results from this trial in mid-2018.”
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 Lancet Oncology. The journal also published online a companion Comment article, “Defeating anaemia in myelodysplastic syndromes: another step forward,” by Valeria Santini, M.D., Associate Professor of Hematology at the University of Florence Medical School in Florence, Italy.
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 ESA-treatment ineligible, based on erythropoietin levels greater than 200 units per liter at baseline. In early 2018, Acceleron and Celgene plan to initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS patients.
Luspatercept is an investigational product that is not approved for use in any country.
About Luspatercept
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.