Acceleron and its global partner, Bristol-Myers Squibb—the industry leader in hematology—recently launched REBLOZYL® (luspatercept-aamt). The product was approved by the U.S. FDA for the treatment of adult patients with beta-thalassemia-associated anemia who require red blood cell (RBC) transfusions. The FDA is also evaluating REBLOZYL® in adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require RBC transfusions and set a PDUFA target action date of April 4, 2020 for the MDS indication.
Discovered by Acceleron, luspatercept-aamt is a first-in-class erythroid (red blood cell) maturation agent, which is also being developed in additional patient segments in both diseases, as well as in myelofibrosis and other possible indications. All of these serious blood disorders are marked by a late-stage defect in the maturation of red blood cells, and this defect results in chronic anemia that can affect quality of life and negatively impact a patient’s physical, social, and emotional well-being.
Acceleron is also collaborating with Adimab, an innovator in antibody discovery and optimization, in addition to Fulcrum Therapeutics, a biotech with expertise in identifying drug targets based on modulation of genetic pathways associated with disease. Adimab is working to identify fully human antibodies that Acceleron may develop and commercialize, the research for which we are funding, with the option to exclusively license newly discovered antibodies. In our agreement with Fulcrum, we will have access to their unique, proprietary product engine and target identification platform with the potential to identify small molecules that control the expression of genes known to impact specific pathways associated with a pulmonary disease of interest.
Acceleron is dedicated to the discovery and development of innovative, life-changing medicines for patients with a wide range of serious and rare diseases. Our research focuses on harnessing the power of the TGF-beta protein superfamily, the target-rich area of biology behind the body’s remarkable capacity for cellular growth and repair.
We would welcome discussions on meaningful strategic collaborations. You can reach us at BD@acceleronpharma.com.