Profile: Acceleron Pharma Inc (XLRN.O)
8 Dec 2016
Acceleron Pharma Inc., incorporated on June 13, 2003, is a clinical-stage biopharmaceutical company. The Company is focused on the discovery, development and commercialization of therapeutic candidates that are based on the mechanisms that the human body uses to regulate the growth and repair of its cells and tissues. The Company's segment operates through discovery, development and commercialization of protein therapeutics for cancer and rare diseases. The Company's research focuses on key natural regulators of cellular growth and repair, particularly the Transforming Growth Factor-Beta (TGF-b) protein superfamily. The Company focuses on discovering and developing therapeutic candidates that target a group of approximately 30 secreted proteins, or ligands, that are collectively referred to as the TGF-b superfamily. These ligands bind to subsets of 12 different receptors on the surface of cells, triggering intra-cellular changes in gene expression that guide cell growth and differentiation. The TGF-b superfamily ligands and their receptors represent an under-explored and diverse set of drug targets.
The Company's therapeutic candidates that are in clinical trials include luspatercept, sotatercept, dalantercept and ACE-083. The Company's lead programs, luspatercept and sotatercept, are partnered with Celgene Corporation (Celgene). The Company's Luspatercept is designed to promote red blood cell production through a mechanism, and the Company is developing luspatercept with Celgene to treat anemia and associated complications in myelodysplastic syndromes (MDS) and beta-thalassemia. MDS is a group of heterogeneous hematologic diseases characterized by abnormal proliferation and differentiation of blood precursor cells, including red blood cell precursors, in the bone marrow. In addition to the Phase III clinical trials, the Company is conducting two Phase II clinical trials of luspatercept in patients with MDS.
The Company and Celgene are developing sotatercept for the treatment of chronic kidney disease (CKD), a disorder characterized by anemia and a mineral and bone disorder that leads to bone loss and cardiovascular disease. The mineral and bone disorder in these patients is not well-managed with current therapies. The Company and Celgene are considering refocusing the sotatercept program on the treatment of patients with earlier, pre-dialysis kidney disease. Sotatercept is also being studied through investigator-initiated clinical trials in multiple myeloma, Diamond-Blackfan anemia and myelofibrosis.
The Company's Dalantercept is designed to treat cancers by inhibiting blood vessel formation by inhibiting signaling through the ALK1 receptor. This mechanism is distinct from, and potentially synergistic with, the dominant class of cancer drugs that inhibit blood vessel formation, the vascular endothelial growth factor (VEGF), pathway inhibitors. The Company is developing dalantercept primarily for use in combination with VEGF pathway inhibitors to produce better outcomes for cancer patients. The Company's ACE-083 is designed for the treatment of focal muscle disorders, such as facioscapulohumeral dystrophy. ACE-083, is designed to promote muscle growth and function in specific, targeted muscles.
The Company competes with Amgen, Janssen Pharmaceuticals, Eli Lilly and Company, Onconova, Apogenix, Novartis, bluebird bio, Inc., Memorial Sloan Kettering Cancer Center, GlaxoSmithKline plc, Sangamo BioSciences Inc, NephroGenex, Abbvie, Bayer, Mitsubishi Pharma and Vascular Pharmaceuticals.
Acceleron Pharma Inc
128 Sidney St
CAMBRIDGE MA 02139-4239