ACE-011 Phase 1 Results Published In The Journal Of Bone And Mineral Research

Main Category: Bones / Orthopaedics
Also Included In: Blood / Hematology
Article Date: 01 Jan 2009 - 4:00 PST

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Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including bone, muscle and red blood cells and Celgene Corporation, today announced the publication of the ACE-011 Phase 1 clinical study demonstrating that ACE-011 increased biomarkers of bone formation, decreased biomarkers of bone resorption and increased hemoglobin levels in postmenopausal women. The paper was published online in the Journal of Bone and Mineral Research (JBMR) and is titled "A Single-Dose, Randomized, Double-Blind, Placebo-Controlled Study of ACE-011 (ActRIIA-IgG1) in Postmenopausal Women."

The Phase 1 study results demonstrated that a single dose of ACE-011 caused a rapid, sustained, dose-dependent increase in serum levels of bone-specific alkaline phosphatase (BSAP), a marker of bone formation, while a marker of bone resorption, C-terminal type 1 collagen telopeptide (CTX), decreased. These effects were seen as early as three days following the first dose and, at the higher dose levels, were maintained for several months. Furthermore, clinically meaningful, dose-dependent increases in hemoglobin were seen following treatment with a single dose of ACE-011.

"We are encouraged to see this degree of biological activity in a single ascending dose study," said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. "The anabolic bone activity and the positive effects on hemoglobin substantiate an exciting and unique profile for ACE-011. We look forward to the ongoing development of this promising compound for a wide range of patients who could potentially benefit from treatment with ACE-011."

Acceleron and Celgene Corporation are co-developing ACE-011 which is currently being evaluated in a Phase 2 clinical study of multiple myeloma patients with osteolytic bone disease.

About ACE-011

ACE-011, a soluble form of the activin receptor type IIA (ActRIIA), is a biologic therapeutic agent that inhibits signaling through the ActRIIA receptor, a member of the Growth and Differentiation Factor (GDF) family of proteins. By blocking signaling through ActRIIA, ACE-011 stimulates bone formation and increases red blood cell production. In numerous animal models of diseases involving bone loss, ACE-011 significantly increased bone mineral density, improved bone architecture, and increased bone formation rate and bone mechanical strength. In Phase 1 clinical studies in healthy volunteers, ACE-011 had an encouraging safety profile and increased biomarkers of bone formation, bone mineral density and levels of hemoglobin and hematocrit. A Phase 2 clinical study in multiple myeloma patients with osteolytic bone disease is ongoing. ACE-011 is being jointly developed by Acceleron and Celgene Corporation.

About Celgene/Acceleron Collaboration

On February 20, 2008, under the terms of the agreement, Celgene and Acceleron announced that they will jointly develop, manufacture and commercialize Acceleron's products for bone loss. Celgene made an upfront payment to Acceleron of $50 million, which included a $5 million equity investment in Acceleron. In addition, in the event of an initial public offering of Acceleron, Celgene will purchase a minimum of $7 million of Acceleron common stock.

Acceleron is responsible for initial activities including research and development through the end of Phase 2a clinical trials as well as manufacturing the clinical supplies for these studies. In turn, Celgene will conduct the Phase 2b and Phase 3 clinical studies and will oversee the manufacture of Phase 3 and commercial supplies. Acceleron will pay a share of the development expenses and is eligible to receive development, regulatory and commercial milestones of up to $510 million for the ACE-011 program and up to an additional $437 million for each of the three discovery stage programs. Both companies will co-promote the products in North America. Acceleron will receive tiered royalties on worldwide net sales.

About Celgene

Celgene Corporation, based in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of novel therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at www.celgene.com

About Acceleron

Acceleron is a privately held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of bone, muscle, red blood cells, 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 Growth and Differentiation Factor (GDF) family of proteins. ACE-011 is the Company's lead program and is being developed across a variety of diseases. In addition, the company is advancing through preclinical development product candidates that increase muscle mass, control angiogenesis and inhibit fat accumulation. 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 are Advanced Technology Ventures, Bessemer Ventures, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For more information, visit
www.acceleronpharma.com