Profile: Acceleron Pharma Inc (XLRN.O)
10 Mar 2014
Acceleron Pharma Inc., incorporated on June 13, 2003, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of protein therapeutics for cancer and rare diseases. The Company’s research focuses on the biology of the Transforming Growth Factor-Beta (TGF-b) protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body. By coupling its discovery and development expertise, including its knowledge of the TGF-b superfamily, with its internal protein engineering and manufacturing capabilities, it has built a productive research and development platform that has generated clinical and preclinical protein therapeutic candidates with mechanisms of action.
The Company focuses on discovering and developing protein therapeutics 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 with the potential to yield therapeutics that modulate the growth and repair of diseased cells and tissues.
The Company has three internally discovered protein therapeutic candidates that are being studied in 12 ongoing Phase 2 clinical trials, focused on cancer and rare diseases. The Company’s two advanced protein therapeutic candidates, sotatercept and ACE-536, promote red blood cell production through a novel mechanism. Together with its collaboration partner, Celgene Corporation, the Company is developing sotatercept and ACE-536 to treat anemia and associated complications in patients with b-thalassemia and myelodysplastic syndromes (MDS). These red blood cell disorders are generally unresponsive to approved drugs. The Company’s third clinical stage protein therapeutic candidate, dalantercept, is designed to inhibit blood vessel formation through a mechanism that 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 these products to produce better outcomes for cancer patients.
The Company and Celgene have conducted six human clinical trials with sotatercept in over 160 healthy volunteers and cancer patients. The Company has conducted one clinical trial with ACE-536 in healthy volunteers. In these studies, both sotatercept and ACE-536 caused a dose-dependent increase in the number of red blood cells. Based on these results, the Company and Celgene have initiated Phase II clinical trials with each of these protein therapeutic candidates in b-thalassemia and MDS. In the ongoing trial of sotatercept in patients with b-thalassemia, the Company has observed encouraging, dose-dependent increases in hemoglobin in a subset of patients at the two lowest dose levels.
Sotatercept is a soluble receptor fusion protein consisting of the extracellular domain of the activin receptor type IIA (ActRIIA) linked to the Fc domain of human IgG1. Sotatercept acts as a protein trap for TGF-b superfamily ligands that signal through the ActRIIA receptor. Sotatercept has increased red blood cells in multiple clinical trials. ACE-536 is a soluble receptor fusion protein consisting of a modified extracellular domain of the activin receptor type IIB (ActRIIB) linked to the Fc domain of human IgG1.
Acceleron Pharma Inc
128 Sidney Street
CAMBRIDGE MA 02139