Product Candidates

Product Candidates

Luspatercept

Potential first-in-class anemia treatment for rare blood diseases

Luspatercept, our lead product candidate, is being developed to treat patients with the chronic anemia associated with a wide range of blood diseases. Discovered by Acceleron, luspatercept is being developed with leading hematology company Celgene in multiple Phase 2 and Phase 3 trials in myelodyspastic syndromes (MDS) and beta-thalassemia. The therapy is designed to promote production of healthy red blood cells by targeting and regulating specific TGF-beta proteins in late-stage red blood cell differentiation and maturation. It is the first novel approach to treating anemia in more than 20 years, with potential to vastly improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.

We are advancing luspatercept in our MEDALIST Phase 3 study in patients with lower-risk, ring sideroblast-positive MDS and in our BELIEVE Phase 3 study in beta-thalassemia patients who are transfusion dependent, as well as in Phase 2 extension studies in an effort to learn more about luspatercept’s long-term use. We continue to evaluate its potential in additional patient segments in both diseases, and are planning a Phase 2 study in myelofibrosis, a rare bone marrow disorder. We expect to begin a new Phase 3 trial, in early 2018, which will evaluate luspatercept versus standard-of-care in the first-line treatment setting for MDS patients.


Hematology

Luspatercept

Hematology

Luspatercept
PreclinicalPhase 1Phase 2Phase 3
 
Myelodysplastic Syndromes
Myelodysplastic
Syndromes
MEDALIST StudyMEDALIST Study
Long-Term Extension StudyLong-Term Ext St
First-Line Lower-Risk MDS Cohorts1st Line Lower-Risk MDS
Beta-Thalassemia
Beta-Thalassemia
BELIEVE StudyBELIEVE Study
Long-Term Extension StudyLong-Term Ext St
NTD Ph 2 Study (Planned)NTD Ph 2 Planned
Myelofibrosis
Myelofibrosis
Ph 2 Study (Planned)Ph 2 Planned

Addressing Unmet Need in MDS…

Myelodysplastic syndromes are a family of rare, cancer-like disorders afflicting the elderly, in which the bone marrow fails to produce sufficient healthy red blood cells. With no approved treatment for the chronic anemia in lower-risk MDS patients, off-label use of erythropoiesis-stimulating agents (ESAs), such as Epogen® (Amgen) and Procrit® (J&J), are often prescribed. However, Phase 3 study results show that only a minority of patients respond to ESA treatment and only for short durations. We believe luspatercept has potential to address this unmet need based on encouraging clinical results to date, showing reduction or elimination of patients’ red blood cell transfusion burden. We presented data from an ongoing Phase 2 study at the 14th Annual International Symposium on Myelodysplastic Syndromes, in which luspatercept demonstrated meaningful erthyroid response rates and transfusion independence in first-line, lower-risk myelodysplastic syndromes patients.

…and Beta-thalassemia

Similar to MDS, the red blood cell-related complications of beta-thalassemia are not adequately addressed by currently approved drugs, including ESAs. Caused by the body’s inability to produce enough hemoglobin, beta-thalassemia is one of the most common genetic diseases in the world, with an estimated 70,000 to 100,000 children born transfusion-dependent each year. Treatment options today are limited to blood transfusions and iron chelating agents, which can lead to viral infections, iron overload and other complications. Luspatercept’s first-in-class potential was highlighted by Phase 2 clinical results presented at major medical meetings, with a majority of patients in the study achieving a 50% reduction in transfusion burden in any 12 weeks of treatment, compared to the 12 weeks prior to treatment.