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Acceleron and Collaborators Publish Two Papers on ACE-041, a Novel Angiogenesis Inhibitor, in Journal of Experimental Medicine and Molecular Cancer Therapeutics.

Preclinical Findings Demonstrate ACE-041 Inhibits Tumor Angiogenesis and Growth by Blocking Novel Target, Activin Receptor-Like Kinase 1 (ALK1)

CAMBRIDGE, Mass. -- Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone, muscle, fat, and the vasculature, announced the publication of two separate peer-reviewed articles describing preclinical data demonstrating that treatment with ACE-041, an inhibitor of signaling through the activin receptor-like kinase 1 (ALK1) receptor, slows tumor growth and progression by inhibiting angiogenesis. The paper titled "Genetic and pharmacological targeting of activin receptor-like kinase 1 impairs tumor growth and angiogenesis" was published in the Journal of Experimental Medicine in collaboration with researchers from the Karolinska Institute in Sweden. A second paper, titled "ALK1-Fc Inhibits Multiple Mediators of Angiogenesis and Suppresses Tumor Growth," was published in Molecular Cancer Therapeutics by researchers at Acceleron.

"We are excited by the results described in these papers, which establish an important role for ALK1 signaling in tumor angiogenesis and support the potential of ACE-041 as a promising new experimental drug for the treatment of cancer," said Jasbir Seehra, Ph.D., Chief Scientific Officer at Acceleron Pharma. "The discovery that the ALK1 pathway is involved as a common, critical phase of the blood vessel development process presents numerous possibilities for ACE-041, which inhibits signaling through the ALK1 receptor."

Study findings from these two publications demonstrated that treatment with an ALK1-Fc:

* Slows tumor growth and progression by inhibition of angiogenesis

* Reduces vascular endothelial growth factor (VEGF) and fibroblast growth factor (FGF)-mediated angiogenesis

* Prevents tumor angiogenesis and decreases tumor burden in several cancer models including melanoma, breast, and pancreatic islet cancer

* Conversely, ALK1 signaling is necessary for efficient tumor angiogenesis

* Tumor tissue has elevated expression of ALK-1 ligands, ALK1 receptors, and increased ALK1 signaling

About ACE-041

ACE-041 is a novel angiogenesis inhibitor that binds to and prevents members of the TGF- superfamily from signaling through the activin receptor-like kinase 1 (ALK1) receptor. Unlike current treatments, ACE-041 prevents angiogenesis by blocking a common, critical phase of the blood vessel development process termed vascular maturation. In preclinical studies, ACE-041 inhibits blood vessel formation induced by multiple angiogenesis factors, including VEGF and FGF, even though ACE-041 does not bind to these molecules. In several animal models of cancer, administration of ACE-041 inhibits tumor growth and prolongs survival by preventing tumor-induced angiogenesis.

About Acceleron

Acceleron is a privately held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases. Acceleron's scientific approach takes advantage of its unique insight into the regenerative powers of the TGF- superfamily of proteins. ACE-011 is currently being studied in a Phase 2 clinical trial in breast cancer patients. ACE-031 is currently being studied in a Phase 1 clinical trial in healthy volunteers and ACE-041 is being studied in a Phase 1 clinical trial in patients with advanced cancer. In addition, the company is developing several other new product candidates that increase muscle mass, control angiogenesis, inhibit fat accumulation and increase bone mass and strength. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company's internal GMP manufacturing capability to rapidly and efficiently advance its therapeutic programs. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For more information, visit
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Publication:Business Wire
Date:Mar 23, 2010
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