Acceleron and Celgene Initiate Phase 2 Study of ACE-011 to Treat Chemotherapy-Induced Anemia

Acceleron and Celgene Initiate Phase 2 Study of ACE-011 to Treat Chemotherapy-Induced Anemia

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone and muscle, and Celgene Corporation (NASDAQ: CELG) today announced the initiation of a second Phase 2 clinical study of ACE-011. This Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study designed to evaluate the potential of ACE-011 to treat chemotherapy-induced anemia in patients with metastatic breast cancer. ACE-011 is a novel therapeutic agent that has been shown to increase levels of red blood cells and hemoglobin and stimulate new bone formation. ACE-011 works through a novel mechanism by inhibiting certain members of the TGF-beta superfamily while potentially avoiding the erythropoietin pathway.

"We are pleased to build on the robust hematologic effects observed in earlier studies of ACE-011 by initiating this Phase 2 clinical trial in breast cancer patients with chemotherapy-induced anemia," said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. "This study, along with the nearly completed Phase 2 clinical trial in patients with multiple myeloma, will provide insight into the therapeutic potential of ACE-011."

"Our discovery of a novel class of anemia therapies comes at a critical time for cancer patients. Many of these patients suffer from an inadequate response to existing therapies or remain untreated due to safety concerns with erythropoietin-based therapies for chemotherapy-induced anemia," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "We are excited to be working with a pre-eminent global biopharmaceutical company like Celgene, which is experienced in maximizing the clinical and commercial potential of disease-altering compounds, to advance the development of this unique molecule, which could replenish red blood cells and has been shown to inhibit the growth and metastasis of tumors in nonclinical animal models of breast cancer."

About Chemotherapy-Induced Anemia

Anemia is one of the most common and debilitating complications associated with cancer chemotherapy, which depletes and prevents formation of red blood cells, leading to incapacitating fatigue and weakness. The vast majority of therapies approved and in development for anemia target the erythropoietin pathway. However, recent studies have suggested an increased risk of mortality associated with this class of therapy, arising from exposure to high levels of recombinant erythropoietin and its derivatives, which may stimulate tumor progression and increase the occurrence of thromboembolic events.

About ACE-011

ACE-011, a soluble form of the activin receptor type IIA (ActRIIA), is a biologic therapeutic that binds to and prevents signaling of several members of the TGF-beta protein super family, and has been shown to stimulate red blood cell production, promote bone formation, and inhibit tumor growth and metastasis. ACE-011 is the first in a novel class of anemia therapies. In Phase 1 clinical studies in healthy volunteers, ACE-011 had an encouraging safety profile, increased levels of hemoglobin and hematocrit, biomarkers of bone formation, and bone mineral density. ACE-011 is currently being studied in two Phase 2 clinical trials in cancer patients and is being jointly developed by Acceleron and Celgene Corporation.

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 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-beta superfamily of proteins. ACE-011 is currently being studied in two Phase 2 clinical trials in cancer patients. ACE-031 is currently being studied in a Phase 1 clinical trial in healthy volunteers. In addition, the company is advancing new product candidates that increase muscle mass, control angiogenesis, inhibit fat accumulation and increase hemoglobin. 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, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For more information, visit www.acceleronpharma.com.

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