Product Candidates

Luspatercept

Novel erythroid maturation agent to treat serious blood diseases

Luspatercept, our lead product candidate, is a first-in-class erythroid (red blood cell) maturation agent (EMA) being developed to treat patients who have serious blood disorders marked by a late-stage defect in the maturation of red blood cells. This defect results in chronic anemia and reduced quality of life that negatively impacts a patient’s physical, social, and emotional well-being.

Discovered by scientists at Acceleron, luspatercept is the first novel approach designed to repair this red blood cell (RBC) maturation defect to restore—and increase—RBC production. As a ligand trap, it works by targeting specific TGF-beta proteins involved in late-stage red blood cell maturation. We believe luspatercept has the potential to be transformative for patients with serious RBC disorders by significantly reducing or eliminating the need for frequent and lifelong blood transfusions. 

Alongside our partner Celgene—a leader in hematology—we recently announced that luspatercept met its primary and all key secondary endpoints in both our MEDALIST Phase 3 trial in patients with lower-risk myelodysplastic syndromes (MDS) and in our BELIEVE Phase 3 trial in patients with beta-thalassemia who are transfusion-dependent. Results of the MEDALIST and BELIEVE trials were presented during the Plenary Scientific Session and an oral session, respectively, at the 2018 Annual Meeting of the American Society of Hematology.

We continue to evaluate luspatercept’s potential to address significant unmet medical needs among additional patient segments in both diseases and are pursuing other possible indications. We have initiated a Phase 3 trial, called COMMANDS, in which we are evaluating luspatercept in treatment-naïve MDS patients. We also have ongoing Phase 2 trials in non-transfusion-dependent beta-thalassemia (BEYOND) and myelofibrosis, a rare bone marrow disorder. Acceleron and Celgene are currently considering future clinical trials in a number of additional serious conditions whose patients could benefit from luspatercept’s unique mechanism of action.


Hematology

Luspatercept

Hematology

Luspatercept
PreclinicalPhase 1Phase 2Phase 3
Myelodysplastic Syndromes
Myelodysplastic
Syndromes
MEDALIST Trial*MEDALIST Trial*
COMMANDS Trial*COMMANDS Trial*
Long-Term Extension TrialLong-Term Ext Tr
Beta-Thalassemia
Beta-Thalassemia
BELIEVE Trial*BELIEVE Trial*
Long-Term Extension TrialLong-Term Ext Tr
BEYOND Trial*BEYOND Trial*
Myelofibrosis
Myelofibrosis
Ph2 Trial*Ph 2*

* Celgene-sponsored


Addressing Unmet Needs in MDS…

Myelodysplastic syndromes are cancer-like disorders in which the bone marrow produces fewer healthy, as well as abnormal red blood cells. It is estimated that more than 100,000 patients in the US and EU have lower-risk MDS. MDS patients currently have few approved therapeutic options. Erythropoiesis-stimulating agents (ESAs), although often prescribed in MDS, do not address the underlying erythroid maturation defect and, therefore, are limited in their effect. Red blood cell transfusions, while frequently necessary, are costly, often risky, and quite burdensome for patients.

…and Beta-thalassemia…

As in MDS, the red blood cell-related complications of beta-thalassemia are not adequately addressed by currently approved drugs, including ESAs. Beta-thalassemia—one of the most common, life-long genetic diseases in the world—leads to the production of fewer healthy, as well as abnormal red blood cells. Treatment options today are limited to blood transfusions and iron chelating agents, which can lead to viral infections, iron overload and other complications.

…and Myelofibrosis

Myelofibrosis (MF) is a rare, fibrotic bone marrow disease that disrupts the body’s normal production of red blood cells. Many patients will develop anemia over the course of their lifetime. There are no approved therapies for anemia in MF patients and currently available therapies only address symptoms. Patients are treated with red blood cell transfusions, ESAs, and immunomodulatory drugs—all of which produce suboptimal results and carry considerable risks.