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Acceleron's ACE-041 Demonstrates Clinical Efficacy in Advanced Cancer Patients

Acceleron Announces Interim Findings from the ACE-041 Phase 1, First-in-Human Clinical Trial at 22nd EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics

Novel Angiogenesis Inhibitor with Unique Mechanism of Action Demonstrates Clinical Activity in Patients with Advanced Cancer

CAMBRIDGE, Mass – November 19, 2010 – Acceleron Pharma, Inc., a biopharmaceutical company developing novel protein therapeutics that regulate the growth and development of tissues and cells, including muscle, bone, red blood cells, and vasculature, today announced the presentation of interim results from the first-in-human clinical study of ACE-041 in patients with advanced cancer at the 22nd EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Berlin, Germany. The presentation was given by Professor Sunil Sharma, the Jon and Karen Huntsman Presidential Professor of Cancer Research at the Huntsman Cancer Institute, University of Utah, Salt Lake City, Utah.

“It has been very encouraging to see so many signals of efficacy in this trial, in particular because these are end-stage cancer patients,” said Dr. Sunil Sharma. “It is also important to note that while we have demonstrated significant activity with ACE-041 monotherapy in this study, we might expect to see even more efficacy in future studies with ACE-041 used in combination with other therapies.”

“The clinical activity and safety profile of ACE-041 confirms our understanding that the ALK1 pathway plays a fundamental and distinct role compared to the VEGF-axis in tumor angiogenesis,” said Dr. Matthew Sherman, M.D., Chief Medical Officer at Acceleron Pharma. “This has significant implications because nearly all patients treated with VEGF inhibitors eventually either fail therapy or develop resistance to these drugs. There is a tremendous need for novel therapies that go beyond VEGF to target the tumor blood supply.”

In a phase 1, first-in-human clinical study, patients with advanced-stage tumors were treated with multiple ascending doses of ACE-041, to evaluate the safety and pharmacokinetics of ACE-041. This study also explored antitumor activity of ACE-041.

Summary of interim findings presented:

· ACE-041 subcutaneously (SC) injected once every three weeks (q3w) is generally well-tolerated · Common side effects include mild or moderate peripheral edema, fatigue, nausea, headache, anorexia, and anemia. A single case of Grade 3 congestive heart failure was reported · Toxicities commonly associated with VEGF inhibition (hypertension, proteinuria, or bleeding) have not been observed · ACE-041 pharmacokinetics support dosing every 3 weeks · One patient with refractory head and neck cancer achieved a partial response and three patients had prolonged disease stabilization · Rapid reduction in tumor metabolic activity observed in several patients, measured by FDG-PET imaging · An expanded cohort study is ongoing at the dose level intended for Phase 2 studies,

ACE-041 is being developed for the treatment of advanced cancer and age-related macular degeneration (AMD).

About ACE-041

ACE-041 is a recombinant receptor fusion protein that inhibits angiogenesis by preventing BMP9 and BMP10, members of the TGFβ protein superfamily, from interacting with activin receptor-like kinase 1 (ALK1), a receptor that is found on proliferating endothelial cells. ACE-041 inhibits ALK1 signaling, which is required for the development of mature, functional capillary networks. In animal studies, treatment with ACE-041 inhibits tumor angiogenesis and growth and ocular neoangiogenesis. In a clinical study of patients with advanced, refractory solid tumors, treatment with ACE-041 was generally well-tolerated and antitumor activity was observed, resulting in tumor shrinkage and stabilization of disease. ACE-041 is being developed for the treatment of advanced-stage cancer and age-related macular degeneration (AMD).

About Acceleron Pharma

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-β superfamily of proteins. 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 further information on Acceleron Pharma, please visit


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Posted: November 2010