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Acceleron Presents Positive Results from Phase 1 Study of ACE-041 in Patients with Advanced Cancer

Encouraging Anti-Tumor Activity of ALK1 Angiogenesis Inhibitor presented at the AACR 102nd Annual Meeting 2011

ORLANDO, Fla.--(BUSINESS WIRE)--Apr 5, 2011 - Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today presented positive results from a Phase 1 clinical study of ACE-041, a novel angiogenesis inhibitor that targets the activin receptor-like kinase 1 (ALK1) pathway. Study results suggest that ACE-041 monotherapy may prevent tumor growth by inhibiting ALK1-mediated angiogenesis. Findings were presented during the annual meeting of the American Association of Cancer Research (AACR) by Johanna Bendell, M.D., from the Sarah Cannon Research Institute.

“I am excited to present these interesting data of the novel anti-angiogenic agent ACE-041,” said Johanna Bendell, M.D., Director of Gastrointestinal Cancer Research and Associate Director, Drug Development at the Sarah Cannon Research Institute. “We have identified tolerable dose levels and have seen preliminary signs of antitumor activity. I look forward to the further development of this agent for cancer patients.”

“We are pleased with the successful completion of the ACE-041 phase 1 study, which establishes ALK1 as an important target for tumor angiogenesis,” said Matthew Sherman, M.D., Chief Medical Officer at Acceleron Pharma. “Based on this observed clinical activity and safety profile we plan to initiate a Phase 2 clinical program later this year, to study ACE-041 alone and in combination with other therapies, including VEGF antagonists.”

In the multiple ascending dose Phase 1 clinical study, 37 patients with advanced-stage tumors were treated to evaluate the safety, pharmacokinetics and antitumor activity of ACE-041.

Summary of results:


  • Pharmacokinetic profile supports dosing by subcutaneous injection once every three weeks.
  • Most frequently observed side effects included mild or moderate peripheral edema, fatigue, anemia, nausea, dyspnea, anorexia and headache.
  • Toxicities commonly associated with VEGF inhibition (hypertension, proteinuria, or bleeding) have not been observed.
  • One patient with advanced, recurrent head and neck cancer achieved a partial response (PR) and eight patients experienced disease stabilization (SD) of > 12 weeks.
  • Robust decline > 20% in tumor metabolic activity, assessed by FDG-PET imaging in 35% of patients.

About ACE-041

ACE-041 is a recombinant receptor fusion protein that inhibits angiogenesis by preventing BMP9 and BMP10, proteins in the TGFβ superfamily, from interacting with activin receptor-like kinase 1 (ALK1), a cell-surface receptor found on proliferating endothelial cells. ACE-041 inhibits ALK1 signaling, which is required for the development of mature, functional vasculature. In animal studies, treatment with ACE-041 inhibits tumor angiogenesis and growth and ocular neoangiogenesis. ACE-041 is being developed for the treatment of advanced-stage solid tumors and age-related macular degeneration (AMD).

About Acceleron Pharma

Acceleron is a privately-held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel protein therapeutics for orphan diseases and cancer. Acceleron's scientific approach takes advantage of its unique insight to discover first-in-class therapies based on the TGF-β protein superfamily. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company's internal GMP manufacturing capability to advance its therapeutic programs rapidly and efficiently. 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

Contact: Acceleron Pharma:
Steven Ertel, 617-649-9234
Senior Vice President, Corporate Development
Suda Communications LLC
Maureen L. Suda, 585-387-9248


Posted: April 2011