Acceleron presents ACE-041 interim results for advanced cancer at EORTC-NCI-AACR Symposium

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 22^nd 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.

“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.”

"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).

Source: Acceleron Pharma, Inc.