Policy & Regulation
Luspatercept Phase 2 Beta-Thalassemia Study Results Published in Blood
11 January 2019 - - The journal Bloodhas published results from the Phase 2 study of luspatercept in patients with red blood cell transfusion-dependent and non-transfusion-dependent beta-thalassemia, US-based biopharmaceutical company Acceleron Pharma Inc. (NASDAQ: XLRN) said.

Luspatercept is an investigational therapy that is part of a global collaboration between Acceleron and Celgene.

In this open-label, Phase 2 study (a three-month initial stage followed by a five-year extension stage), patients were treated with luspatercept subcutaneously once every three weeks.

The primary outcome measures were a reduction in RBC transfusion burden, compared with pretreatment, in transfusion-dependent patients, and changes in hemoglobin levels from baseline in non-transfusion-dependent patients.

The extension study is ongoing and evaluates the long-term safety and tolerability of luspatercept for up to five years.

Luspatercept is being evaluated in ongoing studies in patients with non-transfusion-dependent beta-thalassemia (the Phase 2 BEYOND trial) as well as in patients with lower-risk myelodysplastic syndromes (the Phase 2 PACE-MDS trial and the Phase 3 MEDALIST and COMMANDS trials) and in a Phase 2 trial in patients with myelofibrosis.

Luspatercept is an investigational therapy that is not approved for any use in any country. 

Celgene and Acceleron are planning submission of marketing applications for luspatercept in the United States and Europe in the first half of 2019.

Luspatercept is a first-in-class erythroid maturation agent that is believed to regulate late-stage red blood cell maturation. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration.

Phase 3 clinical trials continue to evaluate the safety and efficacy of luspatercept in patients with MDS (the MEDALIST trial) and in patients with beta-thalassemia (the BELIEVE trial).

A COMMANDS Phase 3 trial in first-line, lower-risk, MDS patients, the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia, and a Phase 2 trial in myelofibrosis are ongoing.

Acceleron is a Cambridge-based, clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialisation of therapeutics to treat serious and rare diseases.

The company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases.

In hematology, the company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.

Acceleron is also advancing its neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and ACE-2494, and a phase 2 pulmonary programme with sotatercept in pulmonary arterial hypertension.