The Fast Track program of the FDA is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. The designation also provides the opportunity for more frequent interaction with FDA over the course of the development program, and allows a sponsor to submit sections of the BLA on a rolling basis as they are finalized.
"The FDA's Fast Track designation for the luspatercept development program recognizes the serious unmet medical needs of patients with beta-thalassemia and the potential for luspatercept in this area," said Jacqualyn A. Fouse, President, Hematology/Oncology for Celgene. "Celgene and Acceleron are working diligently to initiate a phase 3 clinical program in 2015 to treat patients with beta-thalassemia and we look forward to continuing to work closely with health authorities and other important stakeholders to advance this program."
Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Luspatercept is currently in phase 2 clinical trials in patients with beta-thalassemia and in patients with myelodysplastic syndromes. For more information, please visit www.clinicaltrials.gov.
Beta-thalassemia is an inherited disease involving mutations in the beta globin gene leading to deficient hemoglobin levels and defective red blood cell (RBC) production in the bone marrow known as ineffective erythropoiesis. Beyond the severe anemia, many patients also suffer from multiple organ dysfunction, largely due to excess iron deposits, known as "iron overload," resulting from the ineffective erythropoiesis as well as the repeated RBC transfusions to address the anemia. Iron overload can lead to heart failure, liver fibrosis, and diabetes, among other consequences. Current clinical management for beta-thalassemia includes RBC transfusions and iron chelation therapy, which is associated with toxicities. There are no drugs approved to treat beta-thalassemia leaving healthcare providers with few options for patients.
Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair. The company is a leader in understanding the biology of the TGF-beta protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines. Acceleron has built a highly productive R&D platform that has generated innovative clinical and preclinical protein therapeutic candidates with novel mechanisms of action. These therapeutic candidates have the potential to significantly improve clinical outcomes for patients with cancer and rare diseases.
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn and YouTube.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development of luspatercept and Acceleron's TGF-beta superfamily program generally, the timeline for clinical development and regulatory approval of luspatercept, the expected timing for the reporting of data from ongoing trials, and the structure of Acceleron's planned or pending clinical trials. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include that preclinical testing of Acceleron's compounds and data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that data may not be available when we expect it to be, that Acceleron or Celgene will be unable to successfully complete the clinical development of its compounds, that the development of Acceleron's compounds will take longer or cost more than planned, that Acceleron or Celgene may be delayed in initiating or completing any clinical trials, and that Acceleron's compounds will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading "Risk Factors" included in Acceleron's Annual Report on Form 10-K which was filed with the Securities and Exchange Commission (SEC) on March 2, 2015, and other filings that Acceleron has made and may make with the SEC in the future. The forward-looking statements contained in this press release reflect Acceleron's current views with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.
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