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 chronic anemia associated within a wide range of blood diseases. Discovered by Acceleron, luspatercept is being developed alongside the leader in hematology, Celgene, in multiple Phase 2 and Phase 3 trials in myelodyspastic syndromes (MDS) and beta-thalassemia, as well as a Phase 2 trial in myelofibrosis. 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 treat 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 trial in patients with lower-risk, ring sideroblast-positive MDS and in our BELIEVE Phase 3 trial 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 recently initiated a Phase 2 trial in myelofibrosis, a rare bone marrow disorder.

The COMMANDS trial will be evaluating luspatercept versus erythropoiesis-stimulating agent (ESA) treatment, and will include patients regardless of ring sideroblast (RS) status. We are also beginning a Phase 2 trial in non-transfusion-dependent (NTD) beta-thalassemia patients known as the BEYOND trial, which is expected to begin later in 2017, and the Phase 2 trial in myelofibrosis, a rare bone marrow disorder, is underway.


Hematology

Luspatercept

Hematology

Luspatercept
PreclinicalPhase 1Phase 2Phase 3
 
Myelodysplastic Syndromes
Myelodysplastic
Syndromes
MEDALIST TrialMEDALIST Trial
COMMANDS Trial (Planned)COMMANDS Trial
Long-Term Extension TrialLong-Term Ext Tr
Beta-Thalassemia
Beta-Thalassemia
BELIEVE TrialBELIEVE Trial
Long-Term Extension TrialLong-Term Ext Tr
BEYOND Trial (Planned)BEYOND Trial
Myelofibrosis
Myelofibrosis
Ph2 TrialPh 2

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 trial 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 trial achieving a 50% reduction in transfusion burden in any 12 weeks of treatment, compared to the 12 weeks prior to treatment.

…and Myelofibrosis

Myelofibrosis (MF) is a rare bone marrow disorder 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, erythropoiesis-stimulating agents (ESAs), and immunomodulatory drugs—all of which produce suboptimal results and also carry risks.

Acceleron and Celgene have initiated a Phase 2 trial in myelofibrosis, and plan to enroll the first MF patient by the end of 2017.