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 investigational protein therapeutic, ACE-536, to treat anemia in patients with myelodysplastic syndromes (MDS). MDS are a group of hematologic malignancies of the bone marrow that result in low levels of one or more types of blood cells resulting most commonly in severe and chronic anemia (low levels of red blood cells). Acceleron is developing ACE-536 in a global collaboration with Celgene Corporation (NASDAQ: CELG). Acceleron earned a $10 million milestone for initiating this phase 2 study and is still eligible to receive development, regulatory and commercial milestones of up to $200 million for the ACE-536 program.
"The anemia experienced by patients with MDS is clinically challenging because it is often unresponsive to administration of erythropoietin and many patients ultimately require red blood cell transfusions," said Professor Uwe Platzbecker, M.D., Director outpatient department of Hematology/Oncology at Universitatsklinikum Carl Gustav Carus in Dresden, Germany and coordinating principal investigator of the ACE-536 "PACE-MDS" phase 2 study. "We are excited to explore ACE-536 as a potential new treatment for MDS patients in this phase 2 study".
"ACE-536 has the potential to make a significant impact on the treatment of anemia in MDS," said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. "Unlike erythropoietin, ACE-536 may target the specific defect in the erythropoietic maturation process in MDS patients and we are optimistic that it could become an important new therapeutic option for this underserved patient population."
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