Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced the initiation of a phase 2 study of its novel, investigational protein therapeutic, ACE-536, to treat patients with beta-thalassemia, a genetic hematologic disorder causing chronic and life-threatening anemia and serious complications affecting the spleen, liver and heart. Patients and healthcare providers currently have limited options for the treatment of beta-thalassemia. This is the second ongoing Phase 2 trial for ACE-536, which is being developed by Acceleron as part of a global collaboration with Celgene Corporation (NASDAQ: CELG).
"The unmet medical need in beta-thalassemia is enormous as treatment options are limited mainly to blood transfusions and iron chelating agents," said Professor Antonio Piga, M.D., Ph.D., Director of Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and coordinating principal investigator of the study. "ACE-536 could address this important unmet need and I am excited to explore the potential benefits of this innovative therapeutic in a phase 2 study."
"We are excited to start our second phase 2 trial for ACE-536," said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. "Both the preclinical and clinical data assembled to date and our understanding of the protein's novel mechanism of action suggest that ACE-536 may become an important new treatment option for those suffering with beta-thalassemia or other diseases that negatively impact the body's ability to produce a sufficient number of functional red blood cells."
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