Luspatercept for Myelodysplastic Syndromes
In the Phase 2 study, among lower-risk MDS patients, 48% of the erythropoiesis-stimulating agent-naïve[1. ESA] MDS patients treated with luspatercept achieved red blood cell transfusion independence (or RBC-TI) and 51% of ESA-naïve patients treated with luspatercept achieved a clinically meaningful erythroid hematological improvement (or HI-E). Among patients with prior ESA treatment, 51% of the patients achieved HI-E, and 33% of the patients achieved RBC-TI.
In June 2017, Celgene and Acceleron announced the completion of the enrollment of Phase 3 MEDALIST and BELIEVE trials with lupatercept in individuals with MDS and beta-thalassemia.
Initiation of MEDALIST trial
Celgene and Acceleron are conducting the Phase 3 MEDALIST trial to evaluate the safety and efficacy of luspatercept in individuals with ring sideroblasts (or RS+). These individuals are at lower-risk MDS with a baseline RBC transfusion burden of no less than two units per eight weeks over the 16-week period before the treatment.
The primary endpoint of Phase 3 MEDALIST trial is to determine the proportion of patients who are independent to red blood cell (or RBC) transfusion during any successive eight-week period through week 24.
Initiation of BELIEVE trial
Celgene and Acceleron are conducting the Phase 3 BELIEVE trial to evaluate the safety and efficacy of luspatercept in individuals with transfusion-dependent beta-thalassemia. The primary endpoint of the BELIEVE trial would be to determine the proportion of patients achieving a reduction in RBC transfusion burden ≥33% from week 13 to week 24 compared to the baseline 12-week period before therapy.
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